A Phase 3, Multicenter, Randomized, Open-Label Study to Compare the Efficacy and Safety of Pomalidomide in Combination With Low-Dose Dexamethasone Versus High-Dose Dexamethasone in Subjects With Refractory Multiple Myeloma or Relapsed and Refractory Multiple Myeloma and Companion Study (NIMBUS)

• ClinicalTrials.gov Identifier: NCT01311687
• Recruitment Status: Completed
• First Posted: March 9, 2011
• Results First Posted: April 30, 2014
• Last Update Posted: October 24, 2018
• Sponsor: Celgene
• Information provided by (Responsible Party): Celgene

• Study Type: Interventional
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Multiple Myeloma
• Interventions: Drug: pomalidomide; Drug: Dexamethasone
• Enrollment: 455

Participant Flow

Recruitment Details	The study was conducted at 93 sites: 68 sites in Europe, 10 sites in Australia, 10 sites in Canada, 4 sites in Russia, and 1 site in the United States (US) from 18 March 2011 to 29 August 2017.
Pre-assignment Details	Participants were randomized in a 2:1 ratio. Treatment phase discontinuation occurred when a participant had confirmed progressive disease. Participants who did not progress but who were intolerant to treatment, or no longer wished to receive study treatment entered the progression-free survival (PFS) follow-up period until disease progression.

Arm/Group Title	Pomalidomide Plus Low-Dose Dexamethasone	High-Dose Dexamethasone (HD-Dex)
Arm/Group Description	Participants received 4 mg pomalidomide administered by mouth on Days 1-21 of each 28-day treatment cycle and 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1, 8, 15, and 22 of a 28-day cycle until disease progression.	Participants received 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1 through 4, 9 through 12, and 17 through 20 of a 28-day cycle until disease progression.
Period Title: Treatment Phase
Started	302	153
Received Study Drug	300	150
Crossed-over to Pomalidomide	0 [1]	11 [2]
Completed [3]	302	153
Not Completed	0	0
[1] Not applicable to participants initially randomized to receive pomalidomide; [2] After Amendment 4 participants still on HD-Dex treatment were permitted to crossover to pomalidomide; [3] Completed represents participants who discontinued from study treatment
Period Title: PFS Follow-up Phase
Started	11	8
Crossed-over to Pomalidomide [1]	11	0
Completed [2]	11	8
Not Completed	0	0
[1] After Amendment 4 participants still on HD-Dex treatment were permitted to crossover to pomalidomide; [2] Completed represents participants who discontinued from PFS follow-up

Baseline Characteristics

Arm/Group Title		Pomalidomide Plus Low-Dose Dexamethasone	High-Dose Dexamethasone	Total
Arm/Group Description		Participants received 4 mg pomalidomide administered by mouth on Days 1-21 of each 28-day treatment cycle and 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1, 8, 15, and 22 of a 28-day cycle until disease progression.	Participants received 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1 through 4, 9 through 12, and 17 through 20 of a 28-day cycle until disease progression.	Total of all reporting groups
Overall Number of Baseline Participants		302	153	455
Baseline Analysis Population Description		Intent-to-treat population (all participants who were randomized, regardless of whether they received study treatment or not)
Age, Continuous; Mean (Standard Deviation); Unit of measure: Years
	Number Analyzed	302 participants	153 participants	455 participants
		63.6 (9.33)	63.7 (9.56)	63.6 (9.40)
Age, Customized [1]; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	302 participants	153 participants	455 participants
	≤ 75 Years Old	278 92.1%	141 92.2%	419 92.1%
	> 75 Years Old	24 7.9%	12 7.8%	36 7.9%
		[1] Measure Description: Stratification Factor 1
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	302 participants	153 participants	455 participants
	Female	121 40.1%	66 43.1%	187 41.1%
	Male	181 59.9%	87 56.9%	268 58.9%
Ethnicity (NIH/OMB); Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	302 participants	153 participants	455 participants
	Hispanic or Latino	27 8.9%	14 9.2%	41 9.0%
	Not Hispanic or Latino	228 75.5%	104 68.0%	332 73.0%
	Unknown or Not Reported	47 15.6%	35 22.9%	82 18.0%
Race/Ethnicity, Customized; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	302 participants	153 participants	455 participants
	Asian	4 1.3%	0 0.0%	4 0.9%
	Black or African American	4 1.3%	3 2.0%	7 1.5%
	White	244 80.8%	113 73.9%	357 78.5%
	Other	2 0.7%	2 1.3%	4 0.9%
	Not collected	48 15.9%	35 22.9%	83 18.2%
Participants with Prior Anti-Myeloma (MM) Therapies; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	302 participants	153 participants	455 participants
		302 100.0%	153 100.0%	455 100.0%
Time from First Pathologic Diagnosis; Mean (Standard Deviation); Unit of measure: Years
	Number Analyzed	302 participants	153 participants	455 participants
		6.2 (4.02)	6.5 (3.63)	6.3 (3.89)
Stratification Factor 2: Disease Population [1]; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	302 participants	153 participants	455 participants
	Disease Population Group 1	249 82.5%	125 81.7%	374 82.2%
	Disease Population Group 2	8 2.6%	5 3.3%	13 2.9%
	Disease Population Group 3	45 14.9%	23 15.0%	68 14.9%
		[1] Measure Description: Disease Population Group 1 is defined as refractory patients who have progressed on or within 60 days of both lenalidomide and bortezomib based treatments. Disease Population Group 2 is defined as relapsed and refractory patients who achieved at least a partial response (PR) and progressed within 6 months after stopping treatment with lenalidomide and/or bortezomib. Disease Population Group 3 is defined as refractory/intolerant patients who developed intolerance/toxicity after a minimum of 2 cycles of bortezomib.
Stratification Factor 3: Number of Prior Anti-MM Therapies; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	302 participants	153 participants	455 participants
	2 Prior Anti-MM Therapies	17 5.6%	8 5.2%	25 5.5%
	> 2 Prior Anti-MM Therapies	285 94.4%	145 94.8%	430 94.5%
Multiple Myeloma Stage before Study Entry [1]; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	302 participants	153 participants	455 participants
	Stage I	81 26.8%	36 23.5%	117 25.7%
	Stage II	115 38.1%	56 36.6%	171 37.6%
	Stage III	92 30.5%	53 34.6%	145 31.9%
	Missing	14 4.6%	8 5.2%	22 4.8%
		[1] Measure Description: The International Staging System divides myeloma into 3 stages based only on the serum beta-2 microglobulin and serum albumin levels. Stage I: Serum beta-2 microglobulin is less than 3.5 (mg/L) and the albumin level is above 3.5 (g/L); Stage II: Neither stage I or III, meaning that either: ◾ The beta-2 microglobulin level is between 3.5 and 5.5 (with any albumin level), OR ◾ The albumin is below 3.5 while the beta-2 microglobulin is less than 3.5 Stage III: Serum beta-2 microglobulin is greater than 5.5.

Outcome Measures

1. Primary Outcome

Title	Progression-free Survival (PFS) - Primary Analysis
Description	Progression-free survival was calculated as the time from randomization to disease progression as determined by the Independent Response Adjudication Committee based on the International Myeloma Working Group Uniform Response criteria (IMWG), or death on study, whichever occurred earlier. Progressive disease required 1 of the following: • Increase of ≥ 25% from nadir in: o Serum M-component (absolute increase ≥ 0.5 g/dl); o Urine M-component (absolute increase ≥ 200 mg/24 hours); o Bone marrow plasma cell percentage (absolute % ≥ 10%); • Development of new or increase in the size of existing bone lesions or soft tissue plasmacytomas; • Development of hypercalcemia (corrected serum calcium > 11.5 mg/dl) attributed solely to plasma cell proliferative disease.
Time Frame	From randomization until the data cut-off date of 07 September 2012. Maximum duration of follow-up for PFS assessments was 57 weeks.

Outcome Measure Data

Analysis Population Description

Intent-to-treat population

Arm/Group Title	Pomalidomide Plus Low-Dose Dexamethasone	High-Dose Dexamethasone
Arm/Group Description:	Participants received 4 mg pomalidomide administered by mouth on Days 1-21 of each 28-day treatment cycle and 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1, 8, 15, and 22 of a 28-day cycle until disease progression.	Participants received 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1 through 4, 9 through 12, and 17 through 20 of a 28-day cycle until disease progression.
Overall Number of Participants Analyzed	302	153
Median (95% Confidence Interval); Unit of Measure: weeks
	15.7; (13.0 to 20.1)	8.0; (7.0 to 9.0)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Pomalidomide Plus Low-Dose Dexamethasone, High-Dose Dexamethasone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority or Other
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	<0.001
	Comments	[Not Specified]
	Method	Stratified Log Rank Test
	Comments	Stratified by age, disease population, and prior number of anti myeloma therapy.
Method of Estimation	Estimation Parameter	Hazard Ratio (HR)
	Estimated Value	0.45
	Confidence Interval	(2-Sided) 95%; 0.35 to 0.59
	Estimation Comments	Hazard ratio is for Pomalidomide Plus Low-Dose Dexamethasone : High Dose Dexamethasone

2. Primary Outcome

Title	Progression-free Survival (PFS) With a Later Cut-off Date
Description	Progression-free survival was calculated as the time from randomization to disease progression as determined by the Independent Response Adjudication Committee based on the International Myeloma Working Group Uniform Response criteria (IMWG), or death on study, whichever occurred earlier. Progressive disease requires 1 of the following: • Increase of ≥ 25% from nadir in: o Serum M-component (absolute increase ≥ 0.5 g/dl); o Urine M-component (absolute increase ≥ 200 mg/24 hours); o Bone marrow plasma cell percentage (absolute % ≥ 10%); • Development of new or increase in the size of existing bone lesions or soft tissue plasmacytomas; • Development of hypercalcemia (corrected serum calcium > 11.5 mg/dl) attributed solely to plasma cell proliferative disease.
Time Frame	From randomization until the data cut-off date of 01 March 2013. Maximum duration of follow-up for PFS assessments was 74 weeks.

Outcome Measure Data

Analysis Population Description

Intent-to-treat population

Arm/Group Title	Pomalidomide Plus Low-Dose Dexamethasone	High-Dose Dexamethasone
Arm/Group Description:	Participants received 4 mg pomalidomide administered by mouth on Days 1-21 of each 28-day treatment cycle and 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1, 8, 15, and 22 of a 28-day cycle until disease progression.	Participants received 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1 through 4, 9 through 12, and 17 through 20 of a 28-day cycle until disease progression.
Overall Number of Participants Analyzed	302	153
Median (95% Confidence Interval); Unit of Measure: weeks
	16.0; (13.0 to 19.6)	8.1; (7.1 to 9.4)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Pomalidomide Plus Low-Dose Dexamethasone, High-Dose Dexamethasone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority or Other
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	<0.001
	Comments	[Not Specified]
	Method	Stratified log-rank test
	Comments	Stratified by age, disease population, and prior number of anti myeloma therapy.
Method of Estimation	Estimation Parameter	Hazard Ratio (HR)
	Estimated Value	0.49
	Confidence Interval	(2-Sided) 95%; 0.39 to 0.61
	Estimation Comments	Hazard ratio is for Pomalidomide Plus Low-Dose Dexamethasone : High Dose Dexamethasone

3. Secondary Outcome

Title	Number of Participants With Adverse Events (AEs)
Description	An adverse event is any noxious, unintended, or untoward medical occurrence that may appear or worsen in a participant during the course of a study. A serious AE is any AE occurring at any dose that: • Results in death; • Is life-threatening; • Requires or prolongs existing inpatient hospitalization; • Results in persistent or significant disability/incapacity; • Is a congenital anomaly/birth defect; • Constitutes an important medical event. The Investigator assessed the relationship of each AE to study drug and graded the severity according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE, Version 4.0): Grade 1 = Mild (no limitation in activity or intervention required); Grade 2 = Moderate (some limitation in activity; no/minimal medical intervention required); Grade 3 = Severe (marked limitation in activity; medical intervention required, hospitalization possible); Grade 4 = Life-threatening; Grade 5 = Death.
Time Frame	From first dose of study drug through to 30 days after the last dose as of the end of the study (29 August 2017); maximum time on treatment was 297, 269, and 239 weeks in the Pomalidomide + LD-Dex, HD-Dex, and cross-over groups respectively.

Outcome Measure Data

Analysis Population Description

Safety population (all randomized participants who received at least one dose of study drug (either pomalidomide or dexamethasone)).

Arm/Group Title	Pomalidomide Plus Low-Dose Dexamethasone	High-Dose Dexamethasone	HD-Dex / Pomalidomide
Arm/Group Description:	Participants received 4 mg pomalidomide administered by mouth on Days 1-21 of each 28-day treatment cycle and 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1, 8, 15, and 22 of a 28-day cycle until disease progression.	Participants received 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1 through 4, 9 through 12, and 17 through 20 of a 28-day cycle until disease progression, or until cross-over to pomalidomide. Data are up to the time of cross-over.	Participants initially randomized to high-dose dexamethasone (HD-Dex) crossed over to receive 4 mg pomalidomide administered by mouth on Days 1-21 of each 28-day treatment cycle, with or without low-dose dexamethasone (40 mg for participants ≤ 75 years or 20 mg for participants > 75 years of age, administered orally once per day on Days 1, 8, 15, and 22 of each 28-day cycle) at the discretion of the investigator. Data include AEs that occurred after cross-over to pomalidomide.
Overall Number of Participants Analyzed	300	150	11
Measure Type: Count of Participants; Unit of Measure: Participants
Any adverse event	298 99.3%	149 99.3%	11 100.0%
Grade 3-4 adverse events	266 88.7%	127 84.7%	8 72.7%
AE related to pomalidomide	251 83.7%	0 0.0%	11 100.0%
AE related to dexamethasone	205 68.3%	115 76.7%	5 45.5%
AE related to either study drug	271 90.3%	115 76.7%	11 100.0%
Grade 3-4 AE related to pomalidomide	199 66.3%	0 0.0%	6 54.5%
Grade 3-4 AE related to dexamethasone	114 38.0%	70 46.7%	2 18.2%
Grade 3-4 AE related to either study drug	212 70.7%	70 46.7%	6 54.5%
Grade 5 adverse events	46 15.3%	21 14.0%	1 9.1%
Serious adverse events (SAEs)	195 65.0%	80 53.3%	4 36.4%
SAE related to pomalidomide	89 29.7%	0 0.0%	1 9.1%
SAE related to dexamethasone	73 24.3%	36 24.0%	0 0.0%
SAE related to either study drug	98 32.7%	36 24.0%	1 9.1%
SAE leading to discontinuation of pomalidomide	20 6.7%	0 0.0%	1 9.1%
SAE leading to discontinuation of dexamethasone	20 6.7%	14 9.3%	1 9.1%
SAE leading to discontinuation of either study dru	23 7.7%	14 9.3%	1 9.1%
AE leading to discontinuation of pomalidomide	30 10.0%	0 0.0%	1 9.1%
AE leading to discontinuation of dexamethasone	34 11.3%	16 10.7%	1 9.1%
AE leading to discontinuation of either study drug	38 12.7%	16 10.7%	1 9.1%

4. Secondary Outcome

Title	Overall Survival - Primary Analysis
Description	Overall survival is calculated as the time from randomization to death from any cause. Overall survival was censored at the last date that the participant was known to be alive for participants who were alive at the time of analysis and for participants who were lost to follow-up before death was documented.
Time Frame	From randomization until the data cut-off date of 07 September 2012. Maximum time on follow-up for survival was 70 weeks.

Outcome Measure Data

Analysis Population Description

Intent-to-treat

Arm/Group Title	Pomalidomide Plus Low-Dose Dexamethasone	High-Dose Dexamethasone
Arm/Group Description:	Participants received 4 mg pomalidomide administered by mouth on Days 1-21 of each 28-day treatment cycle and 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1, 8, 15, and 22 of a 28-day cycle until disease progression.	Participants received 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1 through 4, 9 through 12, and 17 through 20 of a 28-day cycle until disease progression.
Overall Number of Participants Analyzed	302	153
Median (95% Confidence Interval); Unit of Measure: weeks
	NA [1]; (48.1 to NA)	34.0; (23.4 to 39.9)

[1]

Could not be estimated due to the low number of events

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Pomalidomide Plus Low-Dose Dexamethasone, High-Dose Dexamethasone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority or Other
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	<0.001
	Comments	[Not Specified]
	Method	Log Rank
	Comments	[Not Specified]
Method of Estimation	Estimation Parameter	Hazard Ratio (HR)
	Estimated Value	0.53
	Confidence Interval	(2-Sided) 95%; 0.37 to 0.74
	Estimation Comments	Hazard ratio is for Pomalidomide Plus Low-Dose Dexamethasone : High Dose Dexamethasone

5. Secondary Outcome

Title	Overall Survival With a Later Cut-off Date
Description	Overall survival is calculated as the time from randomization to death from any cause. Overall survival was censored at the last date that the participant was known to be alive for participants who were alive at the time of analysis and for participants who were lost to follow-up before death was documented.
Time Frame	From randomization until the data cut-off date of 01 March 2013. Maximum time on follow-up for survival was 93 weeks.

Outcome Measure Data

Analysis Population Description

Intent-to-treat

Arm/Group Title	Pomalidomide Plus Low-Dose Dexamethasone	High-Dose Dexamethasone
Arm/Group Description:	Participants received 4 mg pomalidomide administered by mouth on Days 1-21 of each 28-day treatment cycle and 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1, 8, 15, and 22 of a 28-day cycle until disease progression.	Participants received 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1 through 4, 9 through 12, and 17 through 20 of a 28-day cycle until disease progression.
Overall Number of Participants Analyzed	302	153
Median (95% Confidence Interval); Unit of Measure: weeks
	54.0; (45.3 to 66.4)	34.9; (29.9 to 39.1)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Pomalidomide Plus Low-Dose Dexamethasone, High-Dose Dexamethasone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority or Other
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	0.009
	Comments	[Not Specified]
	Method	Log Rank
	Comments	[Not Specified]
Method of Estimation	Estimation Parameter	Hazard Ratio (HR)
	Estimated Value	0.70
	Confidence Interval	(2-Sided) 95%; 0.54 to 0.92
	Estimation Comments	Hazard ratio is for Pomalidomide Plus Low-Dose Dexamethasone : High Dose Dexamethasone

6. Secondary Outcome

Title	Overall Survival Based on the Final Dataset
Description	Overall survival is calculated as the time from randomization to death from any cause. Overall survival was censored at the last date that the participant was known to be alive for participants who were alive at the time of analysis and for participants who were lost to follow-up before death was documented.
Time Frame	From randomization until the data cut-off date of 29 August 2017. Maximum time on follow-up for survival was 324 weeks.

Outcome Measure Data

Analysis Population Description

Intent-to-treat

Arm/Group Title	Pomalidomide Plus Low-Dose Dexamethasone	High-Dose Dexamethasone
Arm/Group Description:	Participants received 4 mg pomalidomide administered by mouth on Days 1-21 of each 28-day treatment cycle and 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1, 8, 15, and 22 of a 28-day cycle until disease progression.	Participants received 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1 through 4, 9 through 12, and 17 through 20 of a 28-day cycle until disease progression.
Overall Number of Participants Analyzed	302	153
Median (95% Confidence Interval); Unit of Measure: weeks
	56.1; (47.7 to 67.4)	35.3; (29.9 to 39.9)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Pomalidomide Plus Low-Dose Dexamethasone, High-Dose Dexamethasone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	0.005
	Comments	[Not Specified]
	Method	Log Rank
	Comments	[Not Specified]
Method of Estimation	Estimation Parameter	Hazard Ratio (HR)
	Estimated Value	0.74
	Confidence Interval	(2-Sided) 95%; 0.60 to 0.91
	Estimation Comments	Hazard ratio is for Pomalidomide Plus Low-Dose Dexamethasone : High Dose Dexamethasone

7. Secondary Outcome

Title	Percentage of Participants With an Objective Response According to International Myeloma Working Group (IMWG) Uniform Response Criteria
Description	Objective response is defined as a best overall response of stringent complete response (SCR), complete response (CR), very good partial response (VGPR) or partial response (PR) based on the Independent Response Adjudication Committee: SCR: CR and normal free light chain (FLC) ratio and no clonal cells in bone marrow; CR: Negative serum and urine on immunofixation, disappearance of any soft tissue plasmacytomas and ≤ 5% plasma cells in bone marrow; VGPR: Serum and urine M-protein detectable by immunofixation but not on electrophoresis or ≥ 90% reduction in serum M-protein and urine M-protein level < 100 mg/24 hours; PR: ≥ 50% reduction of serum M-Protein and reduction in urinary M-protein by ≥ 90% or to < 200 mg/24 hours. In addition to the above, if present at baseline a ≥ 50% reduction in the size of soft tissue plasmacytomas is also required.
Time Frame	From randomization until the data cut-off date of 01 March 2013. Maximum time on follow-up was 93 weeks.

Outcome Measure Data

Analysis Population Description

Intent-to-treat population

Arm/Group Title	Pomalidomide Plus Low-Dose Dexamethasone	High-Dose Dexamethasone
Arm/Group Description:	Participants received 4 mg pomalidomide administered by mouth on Days 1-21 of each 28-day treatment cycle and 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1, 8, 15, and 22 of a 28-day cycle until disease progression.	Participants received 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1 through 4, 9 through 12, and 17 through 20 of a 28-day cycle until disease progression.
Overall Number of Participants Analyzed	302	153
Measure Type: Number; Unit of Measure: percentage of participants
	23.5	3.9

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Pomalidomide Plus Low-Dose Dexamethasone, High-Dose Dexamethasone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority or Other
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	< 0.001
	Comments	[Not Specified]
	Method	Fisher Exact
	Comments	[Not Specified]
Method of Estimation	Estimation Parameter	Odds Ratio (OR)
	Estimated Value	7.53
	Confidence Interval	(2-Sided) 95%; 3.19 to 17.77
	Estimation Comments	Odds ratio is for pomalidomide plus low-dose dexamethasone : high dose dexamethasone

8. Secondary Outcome

Title	Percentage of Participants With Objective Response According to European Group for Blood and Marrow Transplantation (EBMT) Criteria
Description	Objective response defined as a best overall response of complete response (CR) or partial response (PR) based on the Independent Response Adjudication Committee: CR requires all of the following: - Absence of original monoclonal paraprotein in serum and urine by immunofixation maintained at least 42 days. - <5% plasma cell in bone marrow aspirate and on bone marrow biopsy, if performed. - No increase in size or number of lytic bone lesions. - Disappearance of soft tissue plasmacytomas. PR requires all of the following: - ≥ 50% reduction in level of serum monoclonal paraprotein, maintained at least 42 days. - Reduction in 24-hour urinary light chain extraction by ≥ 90% or to < 200 mg, maintained at least 42 days. - For patients with non-secretory myeloma, ≥ 50% reduction in plasma cells in bone marrow aspirate and on biopsy, if performed, for at least 42 days. - ≥ 50% reduction in the size of soft tissue plasmacytomas. - No increase in size or number of lytic bone lesions.
Time Frame	From randomization until the data cut-off date of 01 March 2013. Maximum time on follow-up was 93 weeks.

Outcome Measure Data

Analysis Population Description

Intent-to-treat population

Arm/Group Title	Pomalidomide Plus Low-Dose Dexamethasone	High-Dose Dexamethasone
Arm/Group Description:	Participants received 4 mg pomalidomide administered by mouth on Days 1-21 of each 28-day treatment cycle and 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1, 8, 15, and 22 of a 28-day cycle until disease progression.	Participants received 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1 through 4, 9 through 12, and 17 through 20 of a 28-day cycle until disease progression.
Overall Number of Participants Analyzed	302	153
Measure Type: Number; Unit of Measure: percentage of participants
	22.2	3.3

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Pomalidomide Plus Low-Dose Dexamethasone, High-Dose Dexamethasone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority or Other
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	< 0.001
	Comments	[Not Specified]
	Method	Fisher Exact
	Comments	[Not Specified]
Method of Estimation	Estimation Parameter	Odds Ratio (OR)
	Estimated Value	8.44
	Confidence Interval	(2-Sided) 95%; 3.32 to 21.42
	Estimation Comments	Odds ratio is for Pomalidomide Plus Low-Dose Dexamethasone : High Dose Dexamethasone

9. Secondary Outcome

Title	Time to Progression
Description	Time to progression (TTP) is calculated as the time from randomization to the first documented progression confirmed by a blinded, independent Response Adjudication Committee and based on the International Myeloma Working Group Uniform Response criteria (IMWG). Progressive disease requires 1 of the following: • Increase of ≥ 25% from nadir in: o Serum M-component (absolute increase ≥ 0.5 g/dl); o Urine M-component (absolute increase ≥ 200 mg/24 hours); o Bone marrow plasma cell percentage (absolute % ≥ 10%); • Development of new or increase in the size of existing bone lesions or soft tissue plasmacytomas; • Development of hypercalcemia (corrected serum calcium > 11.5 mg/dl) attributed solely to plasma cell proliferative disease.
Time Frame	From randomization until the data cut-off date of 01 March 2013. Maximum time on follow-up was 93 weeks.

Outcome Measure Data

Analysis Population Description

Intent-to-treat population

Arm/Group Title	Pomalidomide Plus Low-Dose Dexamethasone	High-Dose Dexamethasone
Arm/Group Description:	Participants received 4 mg pomalidomide administered by mouth on Days 1-21 of each 28-day treatment cycle and 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1, 8, 15, and 22 of a 28-day cycle until disease progression.	Participants received 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1 through 4, 9 through 12, and 17 through 20 of a 28-day cycle until disease progression.
Overall Number of Participants Analyzed	302	153
Median (95% Confidence Interval); Unit of Measure: weeks
	20.0; (16.1 to 24.0)	9.0; (8.0 to 10.9)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Pomalidomide Plus Low-Dose Dexamethasone, High-Dose Dexamethasone
	Comments	[Not Specified]
	Type of Statistical Test	Superiority or Other
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	< 0.001
	Comments	[Not Specified]
	Method	Stratified Log Rank Test
	Comments	Stratified by age, diseases population, and prior number of anti myeloma therapy.
Method of Estimation	Estimation Parameter	Hazard Ratio (HR)
	Estimated Value	0.46
	Confidence Interval	(2-Sided) 95%; 0.36 to 0.59
	Estimation Comments	Hazard ratio is for Pomalidomide Plus Low-Dose Dexamethasone : High Dose Dexamethasone

10. Secondary Outcome

Title	Time to Response
Description	Time to response is calculated as the time from randomization to the initial documented response (partial response or better) based on IMWG criteria. SCR: CR and normal free light chain (FLC) ratio and no clonal cells in bone marrow; CR: Negative serum and urine on immunofixation, disappearance of any soft tissue plasmacytomas and ≤ 5% plasma cells in bone marrow; VGPR: Serum and urine M-protein detectable by immunofixation but not on electrophoresis or ≥ 90% reduction in serum M-protein and urine M-protein level < 100 mg/24 hours; PR: ≥ 50% reduction of serum M-Protein and reduction in urinary M-protein by ≥ 90% or to < 200 mg/24 hours. If present at baseline a ≥ 50% reduction in size of soft tissue plasmacytomas is also required.
Time Frame	From randomization until the data cut-off date of 01 March 2013. Maximum time on follow-up was 93 weeks.

Outcome Measure Data

Analysis Population Description

Intent-to-treat responder population

Arm/Group Title	Pomalidomide Plus Low-Dose Dexamethasone	High-Dose Dexamethasone
Arm/Group Description:	Participants received 4 mg pomalidomide administered by mouth on Days 1-21 of each 28-day treatment cycle and 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1, 8, 15, and 22 of a 28-day cycle until disease progression.	Participants received 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1 through 4, 9 through 12, and 17 through 20 of a 28-day cycle until disease progression.
Overall Number of Participants Analyzed	71	6
Median (Full Range); Unit of Measure: weeks
	8.1; (4.0 to 48.0)	10.5; (4.1 to 42.1)

11. Secondary Outcome

Title	Duration of Response
Description	Duration of response (calculated for responders only) is defined as time from the initial documented response (partial response or better) to confirmed disease progression, based on IMWG criteria assessed by the Independent Response Adjudication Committee.
Time Frame	From randomization until the data cut-off date of 01 March 2013. Maximum time on follow-up was 93 weeks.

Outcome Measure Data

Analysis Population Description

Intent-to-treat responder population

Arm/Group Title	Pomalidomide Plus Low-Dose Dexamethasone	High-Dose Dexamethasone
Arm/Group Description:	Participants received 4 mg pomalidomide administered by mouth on Days 1-21 of each 28-day treatment cycle and 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1, 8, 15, and 22 of a 28-day cycle until disease progression.	Participants received 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1 through 4, 9 through 12, and 17 through 20 of a 28-day cycle until disease progression.
Overall Number of Participants Analyzed	71	6
Median (95% Confidence Interval); Unit of Measure: weeks
	35.1; (28.4 to 52.9)	28.1; (20.1 to 37.1)

12. Secondary Outcome

Title	Time to the First Hemoglobin Improvement
Description	Time to increased hemoglobin, defined as the time from randomization to at least one category improvement from Baseline in common terminology criteria for adverse events (CTCAE) grade for hemoglobin level. Hemoglobin categories are: 1) Normal; 2) CTCAE Grade 1: < lower limit of normal (LLN) to 10.0 g/dL; 3) CTCAE Grade 2: < 10.0 to <8.0 g/dL. Participants with CTCAE Grade 3 anemia or worse at Baseline were excluded from the study.
Time Frame	From randomization until the data cut-off date of 01 March 2013. Maximum time on follow-up was 93 weeks.

Outcome Measure Data

Analysis Population Description

Intent-to-treat population with improvement in hemoglobin during the study

Arm/Group Title	Pomalidomide Plus Low-Dose Dexamethasone	High-Dose Dexamethasone
Arm/Group Description:	Participants received 4 mg pomalidomide administered by mouth on Days 1-21 of each 28-day treatment cycle and 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1, 8, 15, and 22 of a 28-day cycle until disease progression.	Participants received 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1 through 4, 9 through 12, and 17 through 20 of a 28-day cycle until disease progression.
Overall Number of Participants Analyzed	144	50
Median (Full Range); Unit of Measure: weeks
	3.4; (1.1 to 49.3)	1.3; (0.9 to 24.3)

13. Secondary Outcome

Title	Time to Improvement in Bone Pain
Description	Time to improvement in bone pain is defined as the time from randomization to at least one category improvement from Baseline in bone pain category. Bone pain was categorized (from best to worst) according to answers to the European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire for patients with Multiple Myeloma Module (QLQ-MY20), Question 1, "Have you had bone aches or pain?": 1) Not at all, 2) A little, 3) Quite a bit, or 4) Very much.
Time Frame	From randomization until the data cut-off date of 01 March 2013. Maximum time on follow-up was 93 weeks.

Outcome Measure Data

Analysis Population Description

Intent-to-treat population with improvement in bone pain

Arm/Group Title	Pomalidomide Plus Low-Dose Dexamethasone	High-Dose Dexamethasone
Arm/Group Description:	Participants received 4 mg pomalidomide administered by mouth on Days 1-21 of each 28-day treatment cycle and 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1, 8, 15, and 22 of a 28-day cycle until disease progression.	Participants received 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1 through 4, 9 through 12, and 17 through 20 of a 28-day cycle until disease progression.
Overall Number of Participants Analyzed	101	37
Median (Full Range); Unit of Measure: weeks
	5.7; (3.7 to 88.6)	4.1; (3.7 to 27.3)

14. Secondary Outcome

Title	Time to Improvement in Renal Function
Description	Time to improvement in renal function is defined as the time from randomization to at least one category improvement from Baseline in renal function. Renal Function was categorized as (from best to worst): - Normal: creatinine clearance ≥80 mL/min; - Grade 1: creatinine clearance ≥60 to <80 mL/min; - Grade 2 : creatinine clearance ≥45 to < 60 mL/min. Participants with creatinine clearance < 45 mL/min at baseline were excluded from the study.
Time Frame	From randomization until the data cut-off date of 01 March 2013. Maximum time on follow-up was 93 weeks.

Outcome Measure Data

Analysis Population Description

Intent-to-treat population with improvement in renal function

Arm/Group Title	Pomalidomide Plus Low-Dose Dexamethasone	High-Dose Dexamethasone
Arm/Group Description:	Participants received 4 mg pomalidomide administered by mouth on Days 1-21 of each 28-day treatment cycle and 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1, 8, 15, and 22 of a 28-day cycle until disease progression.	Participants received 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1 through 4, 9 through 12, and 17 through 20 of a 28-day cycle until disease progression.
Overall Number of Participants Analyzed	94	45
Median (Full Range); Unit of Measure: weeks
	4.6; (3.3 to 45.6)	4.1; (3.3 to 28.1)

15. Secondary Outcome

Title	Time to Improvement in Eastern Cooperative Oncology Group (ECOG) Performance Status
Description	Time to improvement in ECOG performance status defined as the time from randomization until at least a one category improvement from Baseline in ECOG performance status score. The categories of the ECOG Performance Status Scale are as follows: -0: Fully active, able to carry on all pre-disease performance without restriction; -1: Restricted in physically strenuous activity but ambulatory and able to carry our work of a light or sedentary nature, e.g., light housework, office work; -2: Ambulatory and capable of all self-care but unable to carry out any work activities. Up and about more than 50% of waking hours. Patients with a score of 3, 4 or 5 were excluded from participating in the study.
Time Frame	From randomization until the data cut-off date of 01 March 2013. Maximum time on follow-up was 93 weeks.

Outcome Measure Data

Analysis Population Description

Intent-to-treat population with improvement in ECOG performance status during the study

Arm/Group Title	Pomalidomide Plus Low-Dose Dexamethasone	High-Dose Dexamethasone
Arm/Group Description:	Participants received 4 mg pomalidomide administered by mouth on Days 1-21 of each 28-day treatment cycle and 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1, 8, 15, and 22 of a 28-day cycle until disease progression.	Participants received 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1 through 4, 9 through 12, and 17 through 20 of a 28-day cycle until disease progression.
Overall Number of Participants Analyzed	61	18
Median (Full Range); Unit of Measure: weeks
	8.1; (4.1 to 44.1)	4.3; (4.1 to 33.7)

16. Secondary Outcome

Title	Change From Baseline in the European Organization for Research and Treatment of Cancer Cancer Quality of Life Questionnaire for Patients With Cancer (EORTC QLQ-C30) Global Health Status Domain
Description	The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Global Health Status/QOL scale is scored between 0 and 100, with a high score indicating better Global Health Status/QOL. Negative change from Baseline values indicate deterioration in QOL or functioning and positive values indicate improvement.
Time Frame	Day 1 of Cycle 1 (Baseline), and Day 1 of Cycles 2, 3, 4, 5 and 6

Outcome Measure Data

Analysis Population Description

The Patient Reported Outcomes (PRO) study population includes any intent-to-treat study participants with 1 active treatment and 1 PRO measurement item completed. Only participants with available data at Baseline and each time point are included.

Arm/Group Title		Pomalidomide Plus Low-Dose Dexamethasone	High-Dose Dexamethasone
Arm/Group Description:		Participants received 4 mg pomalidomide administered by mouth on Days 1-21 of each 28-day treatment cycle and 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1, 8, 15, and 22 of a 28-day cycle until disease progression.	Participants received 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1 through 4, 9 through 12, and 17 through 20 of a 28-day cycle until disease progression.
Overall Number of Participants Analyzed		289	144
Mean (Standard Deviation); Unit of Measure: units on a scale
Cycle 2, Day 1	Number Analyzed	209 participants	91 participants
		0.52 (23.01)	-3.75 (24.10)
Cycle 3, Day 1	Number Analyzed	175 participants	53 participants
		2.67 (24.97)	-2.36 (21.08)
Cycle 4, Day 1	Number Analyzed	157 participants	33 participants
		0.80 (24.62)	-3.03 (22.42)
Cycle 5 Day 1	Number Analyzed	130 participants	27 participants
		0.51 (26.81)	0.00 (27.44)
Cycle 6, Day 1	Number Analyzed	116 participants	18 participants
		-2.51 (25.57)	-0.93 (17.59)

17. Secondary Outcome

Title	Change From Baseline in the EORTC QLQ-C30 Physical Functioning Domain
Description	The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used in clinical research to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Physical Functioning Scale is scored between 0 and 100, with a high score indicating better functioning/support. Negative change from Baseline values indicate deterioration in functioning and positive values indicate improvement.
Time Frame	Day 1 of Cycle 1 (Baseline), and Day 1 of Cycles 2, 3, 4, 5 and 6

Outcome Measure Data

Analysis Population Description

PRO population with available data at Baseline and each time point.

Arm/Group Title		Pomalidomide Plus Low-Dose Dexamethasone	High-Dose Dexamethasone
Arm/Group Description:		Participants received 4 mg pomalidomide administered by mouth on Days 1-21 of each 28-day treatment cycle and 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1, 8, 15, and 22 of a 28-day cycle until disease progression.	Participants received 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1 through 4, 9 through 12, and 17 through 20 of a 28-day cycle until disease progression.
Overall Number of Participants Analyzed		289	144
Mean (Standard Deviation); Unit of Measure: units on a scale
Cycle 2, Day 1	Number Analyzed	210 participants	91 participants
		-2.32 (18.25)	-3.96 (18.35)
Cycle 3, Day 1	Number Analyzed	177 participants	53 participants
		-0.56 (19.86)	-9.69 (16.67)
Cycle 4, Day 1	Number Analyzed	159 participants	33 participants
		0.17 (20.25)	-8.08 (13.31)
Cycle 5 Day 1	Number Analyzed	132 participants	27 participants
		0.91 (19.92)	-5.43 (19.31)
Cycle 6, Day 1	Number Analyzed	118 participants	18 participants
		0.54 (21.30)	-4.81 (14.24)

18. Secondary Outcome

Title	Change From Baseline in the EORTC QLQ-C30 Emotional Functioning Domain
Description	The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used in clinical research to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Emotional Functioning Scale is scored between 0 and 100, with a high score indicating better functioning/support. Negative change from Baseline values indicate deterioration in functioning and positive values indicate improvement.
Time Frame	Day 1 of Cycle 1 (Baseline), and Day 1 of Cycles 2, 3, 4, 5 and 6

Outcome Measure Data

Analysis Population Description

PRO population with available data at Baseline and each time point.

Arm/Group Title		Pomalidomide Plus Low-Dose Dexamethasone	High-Dose Dexamethasone
Arm/Group Description:		Participants received 4 mg pomalidomide administered by mouth on Days 1-21 of each 28-day treatment cycle and 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1, 8, 15, and 22 of a 28-day cycle until disease progression.	Participants received 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1 through 4, 9 through 12, and 17 through 20 of a 28-day cycle until disease progression.
Overall Number of Participants Analyzed		289	144
Mean (Standard Deviation); Unit of Measure: units on a scale
Cycle 2, Day 1	Number Analyzed	210 participants	91 participants
		1.22 (21.44)	-2.87 (21.57)
Cycle 3, Day 1	Number Analyzed	176 participants	53 participants
		2.40 (20.36)	-5.66 (25.36)
Cycle 4, Day 1	Number Analyzed	158 participants	33 participants
		2.44 (21.05)	-6.31 (23.48)
Cycle 5 Day 1	Number Analyzed	131 participants	27 participants
		1.91 (21.97)	-8.64 (23.17)
Cycle 6, Day 1	Number Analyzed	117 participants	18 participants
		0.19 (22.30)	-4.17 (13.18)

19. Secondary Outcome

Title	Change From Baseline in the EORTC QLQ-C30 Fatigue Domain
Description	The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used in clinical research to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Fatigue Scale is scored between 0 and 100, with a high score indicating a higher level of symptoms. Negative change from Baseline values indicate reduction in fatigue (i.e. improvement in symptom) and positive values indicate increases in fatigue (i.e. worsening of symptom).
Time Frame	Day 1 of Cycle 1 (Baseline), and Day 1 of Cycles 2, 3, 4, 5 and 6

Outcome Measure Data

Analysis Population Description

PRO population with available data at Baseline and each time point.

Arm/Group Title		Pomalidomide Plus Low-Dose Dexamethasone	High-Dose Dexamethasone
Arm/Group Description:		Participants received 4 mg pomalidomide administered by mouth on Days 1-21 of each 28-day treatment cycle and 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1, 8, 15, and 22 of a 28-day cycle until disease progression.	Participants received 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1 through 4, 9 through 12, and 17 through 20 of a 28-day cycle until disease progression.
Overall Number of Participants Analyzed		289	144
Mean (Standard Deviation); Unit of Measure: units on a scale
Cycle 2, Day 1	Number Analyzed	210 participants	91 participants
		2.43 (27.39)	4.03 (25.37)
Cycle 3, Day 1	Number Analyzed	177 participants	53 participants
		3.26 (27.66)	7.76 (23.73)
Cycle 4, Day 1	Number Analyzed	159 participants	33 participants
		1.71 (26.21)	9.43 (28.88)
Cycle 5 Day 1	Number Analyzed	132 participants	27 participants
		0.21 (28.41)	9.47 (23.00)
Cycle 6, Day 1	Number Analyzed	118 participants	18 participants
		0.99 (31.13)	10.49 (16.38)

20. Secondary Outcome

Title	Change From Baseline in the EORTC QLQ-C30 Pain Domain
Description	The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used in clinical research to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Pain Scale is scored between 0 and 100, with a high score indicating a higher level of symptoms. Negative change from Baseline values indicate reductions in pain (i.e. improvement in symptom) and positive values indicate increases in pain (i.e. worsening of symptom).
Time Frame	Day 1 of Cycle 1 (Baseline), and Day 1 of Cycles 2, 3, 4, 5 and 6

Outcome Measure Data

Analysis Population Description

PRO population with available data at Baseline and each time point

Arm/Group Title		Pomalidomide Plus Low-Dose Dexamethasone	High-Dose Dexamethasone
Arm/Group Description:		Participants received 4 mg pomalidomide administered by mouth on Days 1-21 of each 28-day treatment cycle and 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1, 8, 15, and 22 of a 28-day cycle until disease progression.	Participants received 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1 through 4, 9 through 12, and 17 through 20 of a 28-day cycle until disease progression.
Overall Number of Participants Analyzed		289	144
Mean (Standard Deviation); Unit of Measure: units on a scale
Cycle 2, Day 1	Number Analyzed	210 participants	92 participants
		-2.70 (25.74)	0.36 (25.32)
Cycle 3, Day 1	Number Analyzed	177 participants	53 participants
		-3.58 (29.62)	2.83 (25.47)
Cycle 4, Day 1	Number Analyzed	159 participants	33 participants
		-2.41 (30.52)	3.03 (25.84)
Cycle 5 Day 1	Number Analyzed	132 participants	27 participants
		-1.64 (28.00)	2.47 (31.59)
Cycle 6, Day 1	Number Analyzed	118 participants	18 participants
		-2.40 (30.99)	10.19 (23.67)

21. Secondary Outcome

Title	Change From Baseline in the European Organization for Research and Treatment of Cancer QoL Questionnaire for Patients With Multiple Myeloma (EORTC QLQ-MY20) Disease Symptoms
Description	The European Organization for Research and Treatment of Cancer QoL Questionnaire for Patients with Multiple Myeloma (EORTC QLQ-MY20) is a 20-question tool used in clinical research to assess health-related quality of life in multiple myeloma patients. The QLQ-MY20 includes four domains (Disease Symptoms, Side-Effects of Treatment, Body Image and Future Perspective). The EORTC QLQ-MY20 Disease Symptoms Scale is scored between 0 and 100, with a high score reflecting a higher level of symptoms. Negative change from Baseline values indicate reduction (i.e. improvement) in symptoms and positive values indicate increase (i.e. worsening) of symptoms.
Time Frame	Day 1 of Cycle 1 (Baseline), and Day 1 of Cycles 2, 3, 4, 5 and 6

Outcome Measure Data

Analysis Population Description

PRO population with available data at Baseline and each time point.

Arm/Group Title		Pomalidomide Plus Low-Dose Dexamethasone	High-Dose Dexamethasone
Arm/Group Description:		Participants received 4 mg pomalidomide administered by mouth on Days 1-21 of each 28-day treatment cycle and 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1, 8, 15, and 22 of a 28-day cycle until disease progression.	Participants received 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1 through 4, 9 through 12, and 17 through 20 of a 28-day cycle until disease progression.
Overall Number of Participants Analyzed		289	144
Mean (Standard Deviation); Unit of Measure: units on a scale
Cycle 2, Day 1	Number Analyzed	218 participants	99 participants
		-0.50 (16.51)	-1.07 (17.78)
Cycle 3, Day 1	Number Analyzed	180 participants	56 participants
		-1.36 (19.51)	0.97 (19.93)
Cycle 4, Day 1	Number Analyzed	161 participants	37 participants
		-1.15 (19.54)	1.35 (16.94)
Cycle 5 Day 1	Number Analyzed	135 participants	30 participants
		-0.53 (17.39)	1.48 (17.56)
Cycle 6, Day 1	Number Analyzed	115 participants	21 participants
		0.60 (19.64)	2.12 (13.43)

22. Secondary Outcome

Title	Change From Baseline in the EORTC QLQ-MY20 Side Effects Domain
Description	The European Organization for Research and Treatment of Cancer QoL Questionnaire for Patients with Multiple Myeloma (EORTC QLQ-MY20) is a 20-question tool used in clinical research to assess health-related quality of life in multiple myeloma patients. The QLQ-MY20 includes four domains (Disease Symptoms, Side-Effects of Treatment, Body Image and Future Perspective). The EORTC QLQ-MY20 Side Effects Scale is scored between 0 and 100, with a high score reflecting a higher level of symptoms. Negative change from Baseline values indicate reduction in side effects (i.e.improvement in symptom) and positive values indicate increase in side effects (i.e. worsening of symptom).
Time Frame	Day 1 of Cycle 1 (Baseline), and Day 1 of Cycles 2, 3, 4, 5 and 6

Outcome Measure Data

Analysis Population Description

PRO population with available data at Baseline and each time point.

Arm/Group Title		Pomalidomide Plus Low-Dose Dexamethasone	High-Dose Dexamethasone
Arm/Group Description:		Participants received 4 mg pomalidomide administered by mouth on Days 1-21 of each 28-day treatment cycle and 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1, 8, 15, and 22 of a 28-day cycle until disease progression.	Participants received 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1 through 4, 9 through 12, and 17 through 20 of a 28-day cycle until disease progression.
Overall Number of Participants Analyzed		289	144
Mean (Standard Deviation); Unit of Measure: units on a scale
Cycle 2, Day 1	Number Analyzed	218 participants	99 participants
		2.71 (13.90)	2.61 (13.34)
Cycle 3, Day 1	Number Analyzed	180 participants	55 participants
		3.26 (13.72)	5.35 (12.27)
Cycle 4, Day 1	Number Analyzed	161 participants	37 participants
		3.73 (14.47)	7.46 (11.61)
Cycle 5 Day 1	Number Analyzed	135 participants	30 participants
		4.74 (14.45)	6.89 (10.32)
Cycle 6, Day 1	Number Analyzed	115 participants	21 participants
		4.55 (15.76)	7.30 (9.35)

23. Secondary Outcome

Title	Change From Baseline in the European Quality of Life-5 Dimensions (EQ-5D) Utility Index Score
Description	EQ-5D is a self-administered questionnaire that assesses health-related quality of life (QOL). The EQ-5D descriptive health profile comprises five dimensions of health (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). Each dimension has 3 levels of response: No problem (1), some problems (2), and extreme problems (3). A unique EQ-5D health state is defined by combining one level from each of the five dimensions into a single utility index score. EQ-5D index values range from -0.59 to 1.00 where an EQ-5D score of 1.00 equals "perfect health", a score of 0 equals "death" and a score of -0.59 equals worst imaginable health state. A positive change from Baseline score indicates improvement in health status. A negative change from Baseline score indicates worsening in health status. Negative scores represent the possible though unlikely situation that a patient's QOL is worse than death, i.e. they would rather be dead than living with that QOL
Time Frame	Day 1 of Cycle 1 (Baseline), and Day 1 of Cycles 2, 3, 4, 5 and 6

Outcome Measure Data

Analysis Population Description

PRO population with available data at Baseline and each time point.

Arm/Group Title		Pomalidomide Plus Low-Dose Dexamethasone	High-Dose Dexamethasone
Arm/Group Description:		Participants received 4 mg pomalidomide administered by mouth on Days 1-21 of each 28-day treatment cycle and 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1, 8, 15, and 22 of a 28-day cycle until disease progression.	Participants received 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1 through 4, 9 through 12, and 17 through 20 of a 28-day cycle until disease progression.
Overall Number of Participants Analyzed		289	144
Mean (Standard Deviation); Unit of Measure: units on a scale
Cycle 2, Day 1	Number Analyzed	198 participants	89 participants
		-0.03 (0.28)	-0.02 (0.23)
Cycle 3, Day 1	Number Analyzed	167 participants	52 participants
		0.01 (0.29)	-0.06 (0.27)
Cycle 4, Day 1	Number Analyzed	146 participants	33 participants
		0.04 (0.31)	-0.07 (0.29)
Cycle 5, Day 1	Number Analyzed	125 participants	25 participants
		0.01 (0.32)	-0.04 (0.26)
Cycle 6, Day 1	Number Analyzed	108 participants	18 participants
		0.03 (0.31)	-0.12 (0.19)

24. Secondary Outcome

Title	Time to First Worsening of Quality of Life (QOL) Domains
Description	Time to worsening in quality of life domains was calculated as the time from Baseline to the first worsened minimally important difference (MID), defined as the smallest change in a QOL score considered important to patients that would lead the patient or clinician to consider a change in therapy. MID thresholds were calculated in Standard Error of Measurement (SEM) units using the Baseline QOL data. Based on the MID, participants were classified as worsened according to the following: For the EORTC QLQ-C30 global health status and functional scales and the EQ-5D health utility score, participants were classified as worsened if their change from Baseline score was less than -1 SEM. For the EORTC QLQ-C30 symptom scores (fatigue and pain) and EORTC QLQ-MY20 disease symptoms and side effects scales, participants were classified as worsened if their change from Baseline score was greater than 1 SEM. See previous outcome measures for definitions of each scale.
Time Frame	Assessed on Day 1 of the first 6 treatment cycles.

Outcome Measure Data

Analysis Population Description

PRO population

Arm/Group Title	Pomalidomide Plus Low-Dose Dexamethasone	High-Dose Dexamethasone
Arm/Group Description:	Participants received 4 mg pomalidomide administered by mouth on Days 1-21 of each 28-day treatment cycle and 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1, 8, 15, and 22 of a 28-day cycle until disease progression.	Participants received 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1 through 4, 9 through 12, and 17 through 20 of a 28-day cycle until disease progression.
Overall Number of Participants Analyzed	289	144
Median (95% Confidence Interval); Unit of Measure: days
Global Health Status	71; (60 to 92)	57; (36 to 91)
Physical Functioning	128; (92 to 225)	67; (57 to 106)
Emotional Functioning	146; (120 to 259)	85; (57 to 124)
Fatigue	58; (57 to 85)	57; (46 to 67)
Pain	92; (86 to 147)	85; (62 to 337)
Disease Symptoms	127; (92 to 155)	106; (67 to 141)
Side Effects of Treatment	90; (78 to 123)	85; (58 to 113)
Health Utility	225; (123 to 338)	162 [1]; (85 to NA)

[1]

Could not be calculated due to a low number of events

Adverse Events

Time Frame	From first dose of study drug through to 30 days after the last dose as of the end of the study (29 August 2017); maximum time on treatment was 297, 269, and 239 weeks in the Pomalidomide + LD-Dex, HD-Dex, and cross-over groups respectively.
Adverse Event Reporting Description	[Not Specified]
Arm/Group Title	Pomalidomide Plus Low-Dose Dexamethasone	High-Dose Dexamethasone	HD-Dex / Pomalidomide
Arm/Group Description	Participants received 4 mg pomalidomide administered by mouth on Days 1-21 of each 28-day treatment cycle and 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1, 8, 15, and 22 of a 28-day cycle until disease progression.	Participants received 40 mg dexamethasone (participants > 75 years of age received 20 mg dexamethasone) administered by mouth once per day on Days 1 through 4, 9 through 12, and 17 through 20 of a 28-day cycle until disease progression, or until cross-over to pomalidomide. Data are up to the time of cross-over.	Participants initially randomized to high-dose dexamethasone (HD-Dex) crossed over to receive 4 mg pomalidomide administered by mouth on Days 1-21 of each 28-day treatment cycle, with or without low-dose dexamethasone (40 mg for participants ≤ 75 years or 20 mg for participants > 75 years of age, administered orally once per day on Days 1, 8, 15, and 22 of each 28-day cycle) at the discretion of the investigator. Data include AEs that occurred after cross-over to pomalidomide.
All-Cause Mortality
	Pomalidomide Plus Low-Dose Dexamethasone	High-Dose Dexamethasone	HD-Dex / Pomalidomide
	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)
Total	252/300 (84.00%)	124/150 (82.67%)	8/11 (72.73%)
Serious Adverse Events
	Pomalidomide Plus Low-Dose Dexamethasone	High-Dose Dexamethasone	HD-Dex / Pomalidomide
	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)
Total	195/300 (65.00%)	80/150 (53.33%)	4/11 (36.36%)
Blood and lymphatic system disorders
Anaemia † 1	10/300 (3.33%)	7/150 (4.67%)	0/11 (0.00%)
Blood disorder † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Febrile neutropenia † 1	19/300 (6.33%)	0/150 (0.00%)	0/11 (0.00%)
Haemorrhagic anaemia † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Hyperviscosity syndrome † 1	1/300 (0.33%)	2/150 (1.33%)	0/11 (0.00%)
Neutropenia † 1	10/300 (3.33%)	1/150 (0.67%)	0/11 (0.00%)
Pancytopenia † 1	2/300 (0.67%)	2/150 (1.33%)	0/11 (0.00%)
Thrombocytopenia † 1	6/300 (2.00%)	4/150 (2.67%)	0/11 (0.00%)
Cardiac disorders
Angina pectoris † 1	0/300 (0.00%)	1/150 (0.67%)	0/11 (0.00%)
Atrial fibrillation † 1	7/300 (2.33%)	1/150 (0.67%)	0/11 (0.00%)
Atrial flutter † 1	1/300 (0.33%)	1/150 (0.67%)	0/11 (0.00%)
Atrial tachycardia † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Cardiac amyloidosis † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Cardiac arrest † 1	1/300 (0.33%)	1/150 (0.67%)	1/11 (9.09%)
Cardiac failure † 1	4/300 (1.33%)	2/150 (1.33%)	0/11 (0.00%)
Cardiac failure acute † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Cardiac failure congestive † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Cardio-respiratory arrest † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Ischaemic cardiomyopathy † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Myocardial infarction † 1	2/300 (0.67%)	0/150 (0.00%)	0/11 (0.00%)
Myocardial ischaemia † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Sinus bradycardia † 1	0/300 (0.00%)	1/150 (0.67%)	0/11 (0.00%)
Sinus node dysfunction † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Tachycardia † 1	0/300 (0.00%)	1/150 (0.67%)	0/11 (0.00%)
Ventricular tachycardia † 1	2/300 (0.67%)	0/150 (0.00%)	0/11 (0.00%)
Ear and labyrinth disorders
Otorrhoea † 1	0/300 (0.00%)	1/150 (0.67%)	0/11 (0.00%)
Eye disorders
Blepharitis † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Diplopia † 1	2/300 (0.67%)	0/150 (0.00%)	0/11 (0.00%)
Gastrointestinal disorders
Abdominal pain † 1	2/300 (0.67%)	0/150 (0.00%)	0/11 (0.00%)
Constipation † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Dental caries † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Diarrhoea † 1	2/300 (0.67%)	2/150 (1.33%)	0/11 (0.00%)
Enteritis † 1	0/300 (0.00%)	1/150 (0.67%)	0/11 (0.00%)
Gastrointestinal haemorrhage † 1	1/300 (0.33%)	1/150 (0.67%)	0/11 (0.00%)
Haemorrhoids † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Incarcerated inguinal hernia † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Large intestine perforation † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Melaena † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Necrotising colitis † 1	0/300 (0.00%)	1/150 (0.67%)	0/11 (0.00%)
Oesophagitis † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Papilla of Vater stenosis † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Retroperitoneal haemorrhage † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Stomatitis † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Vomiting † 1	2/300 (0.67%)	0/150 (0.00%)	0/11 (0.00%)
General disorders
Adverse drug reaction † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Asthenia † 1	1/300 (0.33%)	2/150 (1.33%)	0/11 (0.00%)
Catheter site haemorrhage † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Fatigue † 1	3/300 (1.00%)	0/150 (0.00%)	0/11 (0.00%)
General physical health deterioration † 1	26/300 (8.67%)	12/150 (8.00%)	0/11 (0.00%)
Hyperthermia † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Malaise † 1	2/300 (0.67%)	0/150 (0.00%)	0/11 (0.00%)
Multiple organ dysfunction syndrome † 1	4/300 (1.33%)	0/150 (0.00%)	0/11 (0.00%)
Non-cardiac chest pain † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Oedema peripheral † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Pain † 1	2/300 (0.67%)	0/150 (0.00%)	0/11 (0.00%)
Performance status decreased † 1	0/300 (0.00%)	1/150 (0.67%)	0/11 (0.00%)
Pyrexia † 1	26/300 (8.67%)	7/150 (4.67%)	0/11 (0.00%)
Sudden death † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Hepatobiliary disorders
Cholestasis † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Gallbladder perforation † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Hepatic mass † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Infections and infestations
Abscess limb † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Acute sinusitis † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Anal abscess † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Aspergillus infection † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Bacteraemia † 1	2/300 (0.67%)	1/150 (0.67%)	0/11 (0.00%)
Bacterial diarrhoea † 1	0/300 (0.00%)	1/150 (0.67%)	0/11 (0.00%)
Bacterial infection † 1	0/300 (0.00%)	1/150 (0.67%)	0/11 (0.00%)
Bacterial sepsis † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Bronchitis † 1	8/300 (2.67%)	1/150 (0.67%)	0/11 (0.00%)
Bronchopulmonary aspergillosis † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Cellulitis † 1	2/300 (0.67%)	0/150 (0.00%)	1/11 (9.09%)
Conjunctivitis † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Cytomegalovirus chorioretinitis † 1	2/300 (0.67%)	0/150 (0.00%)	0/11 (0.00%)
Device related infection † 1	3/300 (1.00%)	1/150 (0.67%)	0/11 (0.00%)
Device related sepsis † 1	0/300 (0.00%)	1/150 (0.67%)	0/11 (0.00%)
Diverticulitis † 1	0/300 (0.00%)	1/150 (0.67%)	0/11 (0.00%)
Enterobacter infection † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Enterobacter sepsis † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Erysipelas † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Escherichia infection † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Escherichia sepsis † 1	3/300 (1.00%)	0/150 (0.00%)	0/11 (0.00%)
Escherichia urinary tract infection † 1	1/300 (0.33%)	1/150 (0.67%)	0/11 (0.00%)
Gastroenteritis † 1	2/300 (0.67%)	0/150 (0.00%)	0/11 (0.00%)
Gastroenteritis salmonella † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Herpes zoster † 1	2/300 (0.67%)	1/150 (0.67%)	0/11 (0.00%)
Infection † 1	3/300 (1.00%)	1/150 (0.67%)	0/11 (0.00%)
Infective exacerbation of chronic obstructive airways disease † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Influenza † 1	2/300 (0.67%)	0/150 (0.00%)	0/11 (0.00%)
Intervertebral discitis † 1	0/300 (0.00%)	1/150 (0.67%)	0/11 (0.00%)
Klebsiella sepsis † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Listeria sepsis † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Lower respiratory tract infection † 1	6/300 (2.00%)	4/150 (2.67%)	1/11 (9.09%)
Lung infection † 1	5/300 (1.67%)	1/150 (0.67%)	0/11 (0.00%)
Meningitis † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Meningitis cryptococcal † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Neutropenic sepsis † 1	3/300 (1.00%)	0/150 (0.00%)	0/11 (0.00%)
Ophthalmic herpes simplex † 1	0/300 (0.00%)	1/150 (0.67%)	0/11 (0.00%)
Ophthalmic herpes zoster † 1	0/300 (0.00%)	1/150 (0.67%)	0/11 (0.00%)
Pneumococcal bacteraemia † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Pneumocystis jirovecii pneumonia † 1	3/300 (1.00%)	1/150 (0.67%)	0/11 (0.00%)
Pneumonia † 1	57/300 (19.00%)	14/150 (9.33%)	2/11 (18.18%)
Pneumonia bacterial † 1	2/300 (0.67%)	0/150 (0.00%)	0/11 (0.00%)
Pneumonia pneumococcal † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Pneumonia pseudomonal † 1	0/300 (0.00%)	1/150 (0.67%)	0/11 (0.00%)
Pneumonia respiratory syncytial viral † 1	0/300 (0.00%)	0/150 (0.00%)	1/11 (9.09%)
Pneumonia staphylococcal † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Pneumonia streptococcal † 1	1/300 (0.33%)	1/150 (0.67%)	0/11 (0.00%)
Progressive multifocal leukoencephalopathy † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Pseudomonal bacteraemia † 1	0/300 (0.00%)	1/150 (0.67%)	0/11 (0.00%)
Pseudomonal sepsis † 1	0/300 (0.00%)	1/150 (0.67%)	0/11 (0.00%)
Respiratory syncytial virus infection † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Respiratory tract infection † 1	4/300 (1.33%)	0/150 (0.00%)	1/11 (9.09%)
Salmonella sepsis † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Sepsis † 1	7/300 (2.33%)	3/150 (2.00%)	0/11 (0.00%)
Sepsis syndrome † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Septic arthritis streptococcal † 1	0/300 (0.00%)	1/150 (0.67%)	0/11 (0.00%)
Septic shock † 1	4/300 (1.33%)	6/150 (4.00%)	0/11 (0.00%)
Sialoadenitis † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Sinusitis † 1	2/300 (0.67%)	0/150 (0.00%)	0/11 (0.00%)
Skin infection † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Spinal cord infection † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Streptococcal infection † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Streptococcal sepsis † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Subcutaneous abscess † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Subdural empyema † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Tooth infection † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Upper respiratory tract infection † 1	7/300 (2.33%)	1/150 (0.67%)	0/11 (0.00%)
Urinary tract infection † 1	0/300 (0.00%)	5/150 (3.33%)	0/11 (0.00%)
Urosepsis † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Injury, poisoning and procedural complications
Femoral neck fracture † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Femur fracture † 1	5/300 (1.67%)	1/150 (0.67%)	0/11 (0.00%)
Hip fracture † 1	2/300 (0.67%)	0/150 (0.00%)	0/11 (0.00%)
Humerus fracture † 1	2/300 (0.67%)	1/150 (0.67%)	0/11 (0.00%)
Lumbar vertebral fracture † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Overdose † 1	1/300 (0.33%)	1/150 (0.67%)	0/11 (0.00%)
Pelvic fracture † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Post procedural haematoma † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Rib fracture † 1	1/300 (0.33%)	1/150 (0.67%)	0/11 (0.00%)
Spinal compression fracture † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Subdural haematoma † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Investigations
Blood creatinine increased † 1	1/300 (0.33%)	2/150 (1.33%)	0/11 (0.00%)
Blood immunoglobulin M increased † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
C-reactive protein increased † 1	0/300 (0.00%)	1/150 (0.67%)	0/11 (0.00%)
Platelet count decreased † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Metabolism and nutrition disorders
Cachexia † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Decreased appetite † 1	0/300 (0.00%)	1/150 (0.67%)	0/11 (0.00%)
Dehydration † 1	4/300 (1.33%)	2/150 (1.33%)	0/11 (0.00%)
Diabetic ketoacidosis † 1	0/300 (0.00%)	1/150 (0.67%)	0/11 (0.00%)
Hypercalcaemia † 1	13/300 (4.33%)	5/150 (3.33%)	0/11 (0.00%)
Hyperglycaemia † 1	2/300 (0.67%)	3/150 (2.00%)	0/11 (0.00%)
Hyperuricaemia † 1	0/300 (0.00%)	1/150 (0.67%)	0/11 (0.00%)
Hypokalaemia † 1	2/300 (0.67%)	1/150 (0.67%)	0/11 (0.00%)
Hyponatraemia † 1	4/300 (1.33%)	0/150 (0.00%)	0/11 (0.00%)
Musculoskeletal and connective tissue disorders
Arthritis reactive † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Back pain † 1	8/300 (2.67%)	2/150 (1.33%)	0/11 (0.00%)
Bone pain † 1	10/300 (3.33%)	1/150 (0.67%)	0/11 (0.00%)
Groin pain † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Intervertebral disc compression † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Joint swelling † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Muscular weakness † 1	0/300 (0.00%)	2/150 (1.33%)	0/11 (0.00%)
Musculoskeletal chest pain † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Musculoskeletal pain † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Myalgia † 1	0/300 (0.00%)	1/150 (0.67%)	0/11 (0.00%)
Myopathy † 1	0/300 (0.00%)	1/150 (0.67%)	0/11 (0.00%)
Neck pain † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Osteoarthritis † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Osteonecrosis of jaw † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Pain in extremity † 1	2/300 (0.67%)	0/150 (0.00%)	0/11 (0.00%)
Pain in jaw † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Pathological fracture † 1	2/300 (0.67%)	1/150 (0.67%)	0/11 (0.00%)
Spinal pain † 1	2/300 (0.67%)	0/150 (0.00%)	0/11 (0.00%)
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Basal cell carcinoma † 1	3/300 (1.00%)	0/150 (0.00%)	0/11 (0.00%)
Plasma cell leukaemia † 1	1/300 (0.33%)	2/150 (1.33%)	0/11 (0.00%)
Plasma cell myeloma † 1	1/300 (0.33%)	1/150 (0.67%)	0/11 (0.00%)
Plasmacytoma † 1	3/300 (1.00%)	1/150 (0.67%)	0/11 (0.00%)
Prostate cancer stage IV † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Squamous cell carcinoma of skin † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Tumour pain † 1	0/300 (0.00%)	1/150 (0.67%)	0/11 (0.00%)
Nervous system disorders
Cerebral haemorrhage † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Cerebrovascular accident † 1	2/300 (0.67%)	1/150 (0.67%)	0/11 (0.00%)
Cognitive disorder † 1	1/300 (0.33%)	1/150 (0.67%)	0/11 (0.00%)
Coma † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Depressed level of consciousness † 1	2/300 (0.67%)	0/150 (0.00%)	0/11 (0.00%)
Dizziness † 1	1/300 (0.33%)	1/150 (0.67%)	0/11 (0.00%)
Dyspraxia † 1	0/300 (0.00%)	1/150 (0.67%)	0/11 (0.00%)
Encephalopathy † 1	0/300 (0.00%)	1/150 (0.67%)	0/11 (0.00%)
Epilepsy † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Headache † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Hemiparesis † 1	0/300 (0.00%)	1/150 (0.67%)	0/11 (0.00%)
Ischaemic cerebral infarction † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Loss of consciousness † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Neurological decompensation † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Parkinson's disease † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Partial seizures † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Post herpetic neuralgia † 1	0/300 (0.00%)	1/150 (0.67%)	0/11 (0.00%)
Presyncope † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Radiculopathy † 1	0/300 (0.00%)	1/150 (0.67%)	0/11 (0.00%)
Seizure † 1	0/300 (0.00%)	1/150 (0.67%)	0/11 (0.00%)
Spinal cord compression † 1	2/300 (0.67%)	1/150 (0.67%)	0/11 (0.00%)
Subarachnoid haemorrhage † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Syncope † 1	2/300 (0.67%)	0/150 (0.00%)	1/11 (9.09%)
Transient ischaemic attack † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Vertigo CNS origin † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Psychiatric disorders
Aggression † 1	0/300 (0.00%)	1/150 (0.67%)	0/11 (0.00%)
Bradyphrenia † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Confusional state † 1	3/300 (1.00%)	3/150 (2.00%)	0/11 (0.00%)
Delirium † 1	0/300 (0.00%)	1/150 (0.67%)	0/11 (0.00%)
Renal and urinary disorders
Acute kidney injury † 1	11/300 (3.67%)	7/150 (4.67%)	1/11 (9.09%)
Crush syndrome † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Renal failure † 1	8/300 (2.67%)	1/150 (0.67%)	0/11 (0.00%)
Renal impairment † 1	4/300 (1.33%)	2/150 (1.33%)	0/11 (0.00%)
Urinary retention † 1	3/300 (1.00%)	0/150 (0.00%)	0/11 (0.00%)
Respiratory, thoracic and mediastinal disorders
Acute pulmonary oedema † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Atelectasis † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Chronic obstructive pulmonary disease † 1	2/300 (0.67%)	0/150 (0.00%)	0/11 (0.00%)
Cough † 1	2/300 (0.67%)	0/150 (0.00%)	0/11 (0.00%)
Dyspnoea † 1	9/300 (3.00%)	1/150 (0.67%)	0/11 (0.00%)
Epistaxis † 1	3/300 (1.00%)	2/150 (1.33%)	0/11 (0.00%)
Lung disorder † 1	2/300 (0.67%)	1/150 (0.67%)	0/11 (0.00%)
Lung infiltration † 1	0/300 (0.00%)	1/150 (0.67%)	0/11 (0.00%)
Oropharyngeal pain † 1	0/300 (0.00%)	1/150 (0.67%)	0/11 (0.00%)
Pneumonitis † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Productive cough † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Pulmonary embolism † 1	5/300 (1.67%)	0/150 (0.00%)	0/11 (0.00%)
Pulmonary oedema † 1	0/300 (0.00%)	2/150 (1.33%)	0/11 (0.00%)
Respiratory failure † 1	3/300 (1.00%)	0/150 (0.00%)	0/11 (0.00%)
Skin and subcutaneous tissue disorders
Acute febrile neutrophilic dermatosis † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Vascular disorders
Axillary vein thrombosis † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Circulatory collapse † 1	0/300 (0.00%)	1/150 (0.67%)	0/11 (0.00%)
Deep vein thrombosis † 1	2/300 (0.67%)	0/150 (0.00%)	0/11 (0.00%)
Hypertension † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Hypertensive crisis † 1	1/300 (0.33%)	0/150 (0.00%)	0/11 (0.00%)
Hypotension † 1	2/300 (0.67%)	0/150 (0.00%)	0/11 (0.00%)
1 Term from vocabulary, MedDRA 19; † Indicates events were collected by systematic assessment
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	5%
	Pomalidomide Plus Low-Dose Dexamethasone	High-Dose Dexamethasone	HD-Dex / Pomalidomide
	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)
Total	291/300 (97.00%)	143/150 (95.33%)	11/11 (100.00%)
Blood and lymphatic system disorders
Anaemia † 1	159/300 (53.00%)	77/150 (51.33%)	4/11 (36.36%)
Leukopenia † 1	40/300 (13.33%)	8/150 (5.33%)	0/11 (0.00%)
Lymphopenia † 1	13/300 (4.33%)	8/150 (5.33%)	0/11 (0.00%)
Neutropenia † 1	155/300 (51.67%)	30/150 (20.00%)	5/11 (45.45%)
Thrombocytopenia † 1	87/300 (29.00%)	42/150 (28.00%)	1/11 (9.09%)
Cardiac disorders
Palpitations † 1	7/300 (2.33%)	4/150 (2.67%)	1/11 (9.09%)
Ear and labyrinth disorders
Ear pain † 1	0/300 (0.00%)	0/150 (0.00%)	1/11 (9.09%)
Endocrine disorders
Adrenal insufficiency † 1	2/300 (0.67%)	1/150 (0.67%)	1/11 (9.09%)
Eye disorders
Cataract † 1	8/300 (2.67%)	4/150 (2.67%)	1/11 (9.09%)
Macular pigmentation † 1	0/300 (0.00%)	0/150 (0.00%)	1/11 (9.09%)
Gastrointestinal disorders
Abdominal pain upper † 1	8/300 (2.67%)	3/150 (2.00%)	1/11 (9.09%)
Constipation † 1	72/300 (24.00%)	22/150 (14.67%)	1/11 (9.09%)
Diarrhoea † 1	73/300 (24.33%)	26/150 (17.33%)	3/11 (27.27%)
Epigastric discomfort † 1	0/300 (0.00%)	0/150 (0.00%)	1/11 (9.09%)
Mouth ulceration † 1	3/300 (1.00%)	2/150 (1.33%)	1/11 (9.09%)
Nausea † 1	57/300 (19.00%)	16/150 (10.67%)	3/11 (27.27%)
Parotid gland enlargement † 1	0/300 (0.00%)	0/150 (0.00%)	1/11 (9.09%)
Vomiting † 1	25/300 (8.33%)	6/150 (4.00%)	1/11 (9.09%)
General disorders
Asthenia † 1	53/300 (17.67%)	25/150 (16.67%)	1/11 (9.09%)
Chest pain † 1	11/300 (3.67%)	4/150 (2.67%)	1/11 (9.09%)
Chills † 1	18/300 (6.00%)	2/150 (1.33%)	0/11 (0.00%)
Fatigue † 1	103/300 (34.33%)	40/150 (26.67%)	3/11 (27.27%)
General physical health deterioration † 1	17/300 (5.67%)	5/150 (3.33%)	0/11 (0.00%)
Influenza like illness † 1	3/300 (1.00%)	1/150 (0.67%)	2/11 (18.18%)
Malaise † 1	10/300 (3.33%)	1/150 (0.67%)	1/11 (9.09%)
Oedema † 1	9/300 (3.00%)	7/150 (4.67%)	1/11 (9.09%)
Oedema peripheral † 1	51/300 (17.00%)	15/150 (10.00%)	3/11 (27.27%)
Pyrexia † 1	74/300 (24.67%)	31/150 (20.67%)	3/11 (27.27%)
Hepatobiliary disorders
Cholelithiasis † 1	1/300 (0.33%)	0/150 (0.00%)	1/11 (9.09%)
Infections and infestations
Bronchitis † 1	34/300 (11.33%)	7/150 (4.67%)	1/11 (9.09%)
Cellulitis † 1	6/300 (2.00%)	2/150 (1.33%)	1/11 (9.09%)
Herpes zoster † 1	6/300 (2.00%)	1/150 (0.67%)	1/11 (9.09%)
Nasopharyngitis † 1	30/300 (10.00%)	1/150 (0.67%)	1/11 (9.09%)
Pneumonia † 1	19/300 (6.33%)	5/150 (3.33%)	0/11 (0.00%)
Respiratory tract infection † 1	15/300 (5.00%)	5/150 (3.33%)	0/11 (0.00%)
Sinusitis † 1	10/300 (3.33%)	4/150 (2.67%)	1/11 (9.09%)
Tooth abscess † 1	1/300 (0.33%)	1/150 (0.67%)	1/11 (9.09%)
Upper respiratory tract infection † 1	47/300 (15.67%)	10/150 (6.67%)	2/11 (18.18%)
Urinary tract infection † 1	18/300 (6.00%)	6/150 (4.00%)	2/11 (18.18%)
Wound infection † 1	0/300 (0.00%)	0/150 (0.00%)	1/11 (9.09%)
Injury, poisoning and procedural complications
Chillblains † 1	0/300 (0.00%)	0/150 (0.00%)	1/11 (9.09%)
Contusion † 1	5/300 (1.67%)	2/150 (1.33%)	1/11 (9.09%)
Fall † 1	10/300 (3.33%)	4/150 (2.67%)	1/11 (9.09%)
Jaw fracture † 1	0/300 (0.00%)	0/150 (0.00%)	1/11 (9.09%)
Ligament sprain † 1	0/300 (0.00%)	0/150 (0.00%)	1/11 (9.09%)
Post-traumatic pain † 1	0/300 (0.00%)	0/150 (0.00%)	1/11 (9.09%)
Rib fracture † 1	7/300 (2.33%)	2/150 (1.33%)	1/11 (9.09%)
Skin abrasion † 1	0/300 (0.00%)	1/150 (0.67%)	1/11 (9.09%)
Investigations
Blood creatinine increased † 1	18/300 (6.00%)	6/150 (4.00%)	2/11 (18.18%)
Blood thyroid stimulating hormone increased † 1	0/300 (0.00%)	1/150 (0.67%)	1/11 (9.09%)
Neutrophil count decreased † 1	19/300 (6.33%)	1/150 (0.67%)	1/11 (9.09%)
Weight decreased † 1	15/300 (5.00%)	5/150 (3.33%)	2/11 (18.18%)
White blood cell count decreased † 1	10/300 (3.33%)	1/150 (0.67%)	1/11 (9.09%)
Metabolism and nutrition disorders
Decreased appetite † 1	40/300 (13.33%)	10/150 (6.67%)	3/11 (27.27%)
Dehydration † 1	14/300 (4.67%)	8/150 (5.33%)	0/11 (0.00%)
Hypercalcaemia † 1	11/300 (3.67%)	11/150 (7.33%)	0/11 (0.00%)
Hyperglycaemia † 1	18/300 (6.00%)	10/150 (6.67%)	0/11 (0.00%)
Hypocalcaemia † 1	14/300 (4.67%)	9/150 (6.00%)	0/11 (0.00%)
Hypokalaemia † 1	31/300 (10.33%)	12/150 (8.00%)	0/11 (0.00%)
Musculoskeletal and connective tissue disorders
Arthralgia † 1	28/300 (9.33%)	7/150 (4.67%)	0/11 (0.00%)
Back pain † 1	59/300 (19.67%)	23/150 (15.33%)	2/11 (18.18%)
Bone pain † 1	54/300 (18.00%)	20/150 (13.33%)	2/11 (18.18%)
Coccydynia † 1	0/300 (0.00%)	0/150 (0.00%)	1/11 (9.09%)
Muscle spasms † 1	47/300 (15.67%)	11/150 (7.33%)	1/11 (9.09%)
Muscular weakness † 1	11/300 (3.67%)	18/150 (12.00%)	2/11 (18.18%)
Musculoskeletal chest pain † 1	12/300 (4.00%)	3/150 (2.00%)	1/11 (9.09%)
Musculoskeletal pain † 1	18/300 (6.00%)	5/150 (3.33%)	0/11 (0.00%)
Myalgia † 1	12/300 (4.00%)	4/150 (2.67%)	1/11 (9.09%)
Myopathy † 1	4/300 (1.33%)	11/150 (7.33%)	0/11 (0.00%)
Osteolysis † 1	2/300 (0.67%)	0/150 (0.00%)	1/11 (9.09%)
Pain in extremity † 1	21/300 (7.00%)	9/150 (6.00%)	0/11 (0.00%)
Nervous system disorders
Amnesia † 1	2/300 (0.67%)	0/150 (0.00%)	1/11 (9.09%)
Burning sensation † 1	0/300 (0.00%)	1/150 (0.67%)	1/11 (9.09%)
Carpal tunnel syndrome † 1	0/300 (0.00%)	0/150 (0.00%)	1/11 (9.09%)
Dizziness † 1	40/300 (13.33%)	13/150 (8.67%)	0/11 (0.00%)
Headache † 1	28/300 (9.33%)	9/150 (6.00%)	1/11 (9.09%)
Lethargy † 1	9/300 (3.00%)	4/150 (2.67%)	1/11 (9.09%)
Neuropathy peripheral † 1	9/300 (3.00%)	1/150 (0.67%)	1/11 (9.09%)
Paraesthesia † 1	12/300 (4.00%)	6/150 (4.00%)	1/11 (9.09%)
Peripheral sensory neuropathy † 1	26/300 (8.67%)	4/150 (2.67%)	2/11 (18.18%)
Tremor † 1	19/300 (6.33%)	2/150 (1.33%)	0/11 (0.00%)
Psychiatric disorders
Agitation † 1	17/300 (5.67%)	7/150 (4.67%)	0/11 (0.00%)
Anxiety † 1	15/300 (5.00%)	9/150 (6.00%)	0/11 (0.00%)
Confusional state † 1	10/300 (3.33%)	7/150 (4.67%)	1/11 (9.09%)
Delirium † 1	1/300 (0.33%)	0/150 (0.00%)	1/11 (9.09%)
Insomnia † 1	36/300 (12.00%)	32/150 (21.33%)	0/11 (0.00%)
Renal and urinary disorders
Acute kidney injury † 1	5/300 (1.67%)	3/150 (2.00%)	1/11 (9.09%)
Nocturia † 1	1/300 (0.33%)	0/150 (0.00%)	1/11 (9.09%)
Reproductive system and breast disorders
Gynaecomastia † 1	0/300 (0.00%)	0/150 (0.00%)	1/11 (9.09%)
Respiratory, thoracic and mediastinal disorders
Cough † 1	64/300 (21.33%)	15/150 (10.00%)	1/11 (9.09%)
Dyspnoea † 1	60/300 (20.00%)	21/150 (14.00%)	2/11 (18.18%)
Dyspnoea exertional † 1	18/300 (6.00%)	3/150 (2.00%)	0/11 (0.00%)
Epistaxis † 1	26/300 (8.67%)	13/150 (8.67%)	1/11 (9.09%)
Haemoptysis † 1	0/300 (0.00%)	2/150 (1.33%)	1/11 (9.09%)
Productive cough † 1	5/300 (1.67%)	1/150 (0.67%)	1/11 (9.09%)
Skin and subcutaneous tissue disorders
Blister † 1	1/300 (0.33%)	0/150 (0.00%)	1/11 (9.09%)
Dry skin † 1	8/300 (2.67%)	3/150 (2.00%)	1/11 (9.09%)
Erythema † 1	8/300 (2.67%)	2/150 (1.33%)	1/11 (9.09%)
Hyperhidrosis † 1	18/300 (6.00%)	1/150 (0.67%)	0/11 (0.00%)
Hyperkeratosis † 1	0/300 (0.00%)	0/150 (0.00%)	1/11 (9.09%)
Pruritus † 1	22/300 (7.33%)	4/150 (2.67%)	1/11 (9.09%)
Rash † 1	25/300 (8.33%)	1/150 (0.67%)	1/11 (9.09%)
Rash papular † 1	1/300 (0.33%)	0/150 (0.00%)	1/11 (9.09%)
Swelling face † 1	2/300 (0.67%)	4/150 (2.67%)	1/11 (9.09%)
Vascular disorders
Haematoma † 1	9/300 (3.00%)	2/150 (1.33%)	1/11 (9.09%)
Hot flush † 1	2/300 (0.67%)	2/150 (1.33%)	1/11 (9.09%)
Hypotension † 1	13/300 (4.33%)	4/150 (2.67%)	1/11 (9.09%)
Thrombophlebitis † 1	1/300 (0.33%)	0/150 (0.00%)	1/11 (9.09%)
1 Term from vocabulary, MedDRA 19; † Indicates events were collected by systematic assessment

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

Institution may publish the results of its findings if a multicenter publication is not forthcoming within 18 months after study completion. Institution shall provide Celgene with a copy of the papers prior to submission; Celgene shall complete its review within 60 days after receipt. Upon Celgene's request, proposed publication or presentation will be delayed up to 60 additional days to enable Celgene to secure adequate intellectual property protection of patentable material.

Results Point of Contact

• Name/Title: Associate Director, Clinical Trials Disclosure
• Organization: Celgene Corporation
• Phone: 1-888-260-1599

Publications of Results:

Moreau P, Weisel KC, Song KW, Gibson CJ, Saunders O, Sternas LA, Hong K, Zaki MH, Dimopoulos MA. Relationship of response and survival in patients with relapsed and refractory multiple myeloma treated with pomalidomide plus low-dose dexamethasone in the MM-003 trial randomized phase III trial (NIMBUS). Leuk Lymphoma. 2016 Dec;57(12):2839-2847. doi: 10.1080/10428194.2016.1180685. Epub 2016 May 13.

Miguel JS, Weisel K, Moreau P, Lacy M, Song K, Delforge M, Karlin L, Goldschmidt H, Banos A, Oriol A, Alegre A, Chen C, Cavo M, Garderet L, Ivanova V, Martinez-Lopez J, Belch A, Palumbo A, Schey S, Sonneveld P, Yu X, Sternas L, Jacques C, Zaki M, Dimopoulos M. Pomalidomide plus low-dose dexamethasone versus high-dose dexamethasone alone for patients with relapsed and refractory multiple myeloma (MM-003): a randomised, open-label, phase 3 trial. Lancet Oncol. 2013 Oct;14(11):1055-1066. doi: 10.1016/S1470-2045(13)70380-2. Epub 2013 Sep 3.

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Siegel DS, Weisel KC, Dimopoulos MA, Baz R, Richardson P, Delforge M, Song KW, San Miguel JF, Moreau P, Goldschmidt H, Cavo M, Jagannath S, Yu X, Hong K, Sternas L, Zaki M, Palumbo A. Pomalidomide plus low-dose dexamethasone in patients with relapsed/refractory multiple myeloma and moderate renal impairment: a pooled analysis of three clinical trials. Leuk Lymphoma. 2016 Dec;57(12):2833-2838. doi: 10.1080/10428194.2016.1177181. Epub 2016 Jun 7.

Weisel KC, Dimopoulos MA, Moreau P, Lacy MQ, Song KW, Delforge M, Karlin L, Goldschmidt H, Banos A, Oriol A, Alegre A, Chen C, Cavo M, Garderet L, Ivanova V, Martinez-Lopez J, Knop S, Yu X, Hong K, Sternas L, Jacques C, Zaki MH, San Miguel J. Analysis of renal impairment in MM-003, a phase III study of pomalidomide + low - dose dexamethasone versus high - dose dexamethasone in refractory or relapsed and refractory multiple myeloma. Haematologica. 2016 Jul;101(7):872-8. doi: 10.3324/haematol.2015.137083. Epub 2016 Apr 14.

Dimopoulos MA, Weisel KC, Song KW, Delforge M, Karlin L, Goldschmidt H, Moreau P, Banos A, Oriol A, Garderet L, Cavo M, Ivanova V, Alegre A, Martinez-Lopez J, Chen C, Spencer A, Knop S, Bahlis NJ, Renner C, Yu X, Hong K, Sternas L, Jacques C, Zaki MH, San Miguel JF. Cytogenetics and long-term survival of patients with refractory or relapsed and refractory multiple myeloma treated with pomalidomide and low-dose dexamethasone. Haematologica. 2015 Oct;100(10):1327-33. doi: 10.3324/haematol.2014.117077. Epub 2015 Aug 6.

San Miguel JF, Weisel KC, Song KW, Delforge M, Karlin L, Goldschmidt H, Moreau P, Banos A, Oriol A, Garderet L, Cavo M, Ivanova V, Alegre A, Martinez-Lopez J, Chen C, Renner C, Bahlis NJ, Yu X, Teasdale T, Sternas L, Jacques C, Zaki MH, Dimopoulos MA. Impact of prior treatment and depth of response on survival in MM-003, a randomized phase 3 study comparing pomalidomide plus low-dose dexamethasone versus high-dose dexamethasone in relapsed/refractory multiple myeloma. Haematologica. 2015 Oct;100(10):1334-9. doi: 10.3324/haematol.2015.125864. Epub 2015 Jul 9.

Weisel K, Dimopoulos M, Song KW, Moreau P, Palumbo A, Belch A, Schey S, Sonneveld P, Sternas L, Yu X, Amatya R, Gibson CJ, Zaki M, Jacques C, San Miguel J. Pomalidomide and Low-Dose Dexamethasone Improves Health-Related Quality of Life and Prolongs Time to Worsening in Relapsed/Refractory Patients With Multiple Myeloma Enrolled in the MM-003 Randomized Phase III Trial. Clin Lymphoma Myeloma Leuk. 2015 Sep;15(9):519-30. doi: 10.1016/j.clml.2015.05.007. Epub 2015 Jun 6.

Song KW, Dimopoulos MA, Weisel KC, Moreau P, Palumbo A, Belch A, Schey S, Sonneveld P, Sternas L, Yu X, Amatya R, Monzini MS, Zaki M, Jacques C, San Miguel J. Health-related quality of life from the MM-003 trial of pomalidomide plus low-dose dexamethasone versus high-dose dexamethasone in relapsed and/or refractory multiple myeloma. Haematologica. 2015 Feb;100(2):e63-7. doi: 10.3324/haematol.2014.112557. Epub 2014 Nov 25. No abstract available.

• Responsible Party: Celgene
• ClinicalTrials.gov Identifier: NCT01311687
• Other Study ID Numbers: CC-4047-MM-003; 2010-019820-30 ( EudraCT Number )
• First Submitted: March 8, 2011
• First Posted: March 9, 2011
• Results First Submitted: March 28, 2014
• Results First Posted: April 30, 2014
• Last Update Posted: October 24, 2018