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A Survival Study in Patients With High Risk Myelodysplastic Syndromes Comparing Azacitidine Versus Conventional Care

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00071799
Recruitment Status : Completed
First Posted : November 5, 2003
Results First Posted : July 5, 2010
Last Update Posted : October 29, 2019
Sponsor:
Information provided by (Responsible Party):
Celgene

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Other
Condition Myelodysplastic Syndromes
Interventions Drug: Azacitidine
Other: Physician Choice
Enrollment 358
Recruitment Details  
Pre-assignment Details 738 patients screened from 98 investigator sites. Randomized patients contributed by 79 investigator sites.
Arm/Group Title Azacitidine Conventional Care
Hide Arm/Group Description Azacitidine, 75 mg/m^2/day given by subcutaneous injection for 7 days of every 28 day cycle, plus best supportive care. Physician choice of low dose cytarabine, standard chemotherapy, or best supportive care (consisting of blood products, growth factors, antibiotics)
Period Title: Overall Study
Started 179 179 [1]
Completed 109 [2] 81 [3]
Not Completed 70 98
Reason Not Completed
Adverse Event             19             10
Protocol Violation             1             7
Lost to Follow-up             1             1
Withdrawal by Subject             15             37
Physician Decision             0             2
Lack of Efficacy             11             18
Disease Progression             23             20
Different Central+Local Pathology Review             0             1
Not Documented             0             2
[1]
Best supportive care: 105, Low-dose cytarabine: 49, Standard chemotherapy: 25 patients
[2]
Includes those who died or transformed to AML, or receiving drug + eligible for extension period.
[3]
Includes patients who died or transformed to AML during treatment, or were still receiving drug.
Arm/Group Title Azacitidine Conventional Care Total
Hide Arm/Group Description Azacitidine, 75 mg/m^2/day given by subcutaneous injection for 7 days of every 28 day cycle, plus best supportive care. Physician choice of low dose cytarabine, standard chemotherapy, or best supportive care (consisting of blood products, growth factors, antibiotics) Total of all reporting groups
Overall Number of Baseline Participants 179 179 358
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 179 participants 179 participants 358 participants
68.0  (7.57) 69.2  (7.87) 68.6  (7.73)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 179 participants 179 participants 358 participants
Female
47
  26.3%
60
  33.5%
107
  29.9%
Male
132
  73.7%
119
  66.5%
251
  70.1%
Race/Ethnicity, Customized  
Measure Type: Number
Unit of measure:  Participants
 Number Analyzed 179 participants 179 participants 358 participants
Caucasian 177 175 352
Black or African American 0 0 0
Asian/Oriental 2 3 5
Hispanic 0 1 1
Other 0 0 0
French-American-British (FAB) Classification   [1] 
Measure Type: Number
Unit of measure:  Participants
 Number Analyzed 179 participants 179 participants 358 participants
Refractory anemia with excess blasts (RAEB) 104 103 207
RAEB in transformation 61 62 123
Modified chronic myelomonocytic leukemia 6 5 11
Acute myeloid leukemia 1 1 2
Myeloproliferative disease 4 2 6
Indeterminate 3 6 9
[1]
Measure Description: FAB classifications were determined by the Independent Review Committee
International Prognostic Scoring System (IPSS)   [1] 
Measure Type: Number
Unit of measure:  Participants
 Number Analyzed 179 participants 179 participants 358 participants
Intermediate risk level 1 (0.5-1.0) 5 13 18
Intermediate risk level 2 (1.5-2.0) 76 70 146
High risk (2.5-3.5) 82 85 167
Not applicable 5 3 8
Indeterminate 11 8 19
[1]
Measure Description: The international prognostic scoring system (IPSS) is a standard for risk assessment in primary myelodysplastic syndromes (MDS) that categorizes prognoses taking into account cytogenetics, cytopenias, blasts and blood counts. The scale is 0-3.5 at .5 increments. Scores of 2.5-3.5 represent high risk which corresponds to poorer prognosis. The assessment was performed by the Independent Review Committee.
World Health Organization (WHO) Classification   [1] 
Measure Type: Number
Unit of measure:  Participants
 Number Analyzed 179 participants 179 participants 358 participants
Refractory anemia with excess blasts - 1 14 17 31
Refractory anemia with excess blasts - 2 98 95 193
Chronic myelomonocytic leukemia - 1 (CMMoL-1) 1 0 1
Chronic myelomonocytic leukemia - 2 (CMMoL-2) 10 5 15
Acute myeloid leukemia 55 58 113
Indeterminate 1 4 5
[1]
Measure Description: WHO classifications were determined by the Independent Review Committee
Body Surface Area  
Mean (Standard Deviation)
Unit of measure:  Meters squared
Number Analyzed 179 participants 179 participants 358 participants
1.9  (0.19) 1.8  (0.19) 1.9  (0.19)
Weight  
Mean (Standard Deviation)
Unit of measure:  Kilograms
Number Analyzed 179 participants 179 participants 358 participants
76.5  (14.08) 74.6  (13.58) 75.6  (13.85)
1.Primary Outcome
Title Kaplan-Meier Estimates for Median Time to Death From Any Cause
Hide Description Kaplan-Meier estimates for the median months until death from any cause within the intent-to-treat population. Patients surviving at the end of the follow-up period were censored at the date of last contact. If a patient withdrew consent to follow-up or was lost to follow-up, the patient was censored as of the last date of contact.
Time Frame Day 1 (randomization) to 42 months
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to treat population. Includes participants who died and participants who were censored.
Arm/Group Title Azacitidine Conventional Care
Hide Arm/Group Description:
Azacitidine, 75 mg/m^2/day given by subcutaneous injection for 7 days of every 28 day cycle, plus best supportive care.
Physician choice of low dose cytarabine, standard chemotherapy, or best supportive care (consisting of blood products, growth factors, antibiotics)
Overall Number of Participants Analyzed 179 179
Measure Type: Number
Unit of Measure: months
24.46 15.02
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments

A 95% CI range value of 'does not exist' is not accommodated in the results table, so all the 95% CI range values are offered here.

Azacitidine: low range of 17.9 months and high range of 'does not exist'.

Conventional Care: low range of 9.8 and high range of 17.0 months.
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0001
Comments [Not Specified]
Method Log Rank
Comments The p value is two-sided from the log rank test stratified by the randomization stratification factors of IPSS classification and FAB classification.
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0002
Comments [Not Specified]
Method Regression, Cox
Comments [Not Specified]
Method of Estimation Estimation Parameter Hazard Ratio (HR)
Estimated Value 0.58
Confidence Interval (2-Sided) 95%
0.43 to 0.77
Estimation Comments Cox proportional hazards model stratified on the randomization factors of FAB and IPSS with model term of treatment.
2.Primary Outcome
Title Summary of Subgroup Analyses for Kaplan-Meier Estimates for Time to Death From Any Cause
Hide Description

Kaplan-Meier estimates for the median months until death from any cause within the intent-to-treat population. Patients surviving at the end of the follow-up period were censored at the date of last contact. If a patient withdrew consent to follow-up or was lost to follow-up, the patient was censored as of the last date of contact.

Subgroups that were analyzed are age, gender, French-American-British (FAB) classification, World Health Organization (WHO) classification and International Prognostic Scoring System (IPSS) classification.
Time Frame Day 1 (randomization) to 42 months
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to treat population. Includes participants who died and those who were censored.
Arm/Group Title Azacitidine Conventional Care
Hide Arm/Group Description:
Azacitidine, 75 mg/m^2/day given by subcutaneous injection for 7 days of every 28 day cycle, plus best supportive care.
Physician choice of low dose cytarabine, standard chemotherapy, or best supportive care (consisting of blood products, growth factors, antibiotics)
Overall Number of Participants Analyzed 179 179
Measure Type: Number
Unit of Measure: months
Age <65 years 11.31 7.87
Age >= 65 years 24.46 13.87
Age >= 75 years 8.92 6.20
Gender: Male 24.46 15.02
Gender: Female 25.11 14.85
FAB: Refractory anemia with excess blasts (RAEB) 34.66 15.21
FAB: RAEB in transformation 17.25 15.25
WHO: RAEB 1 11.54 6.72
WHO: RAEB 2 21.11 15.02
WHO: Other (AML, CMMoL-1 and 2, indeterminate) 20.46 15.25
IPSS: Intermediate 2 34.66 16.89
IPSS: High 19.21 14.52
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments

Age < 65 years The Kaplan-Meier median time to death was not reached due to a small number of events, so the KM 25th percentile survival time is presented.

Some of the values in outcome table #2 do not have 95% CI values so no 95% CI ranges are contained in the table. Those 95% CI ranges are added here.

Azacitidine: low range of 7.1 months and high range of 15.6 months.

Conventional Care: low range of 4.4 and high range of 12.4 months.
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3973
Comments The p value is two-sided from the log rank test stratified by the randomization stratification factors of IPSS classification and FAB classification
Method Log Rank
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments

Age >= 65 years

Some of the values in outcome table #2 do not have 95% CI values so no 95% CI ranges are contained in the table. Those 95% CI ranges are added here.

Azacitidine: low range of 17.1 months and high range of 34.7 months.

Conventional Care: low range of 8.8 and high range of 16.4 months.
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments The p value is two-sided from the log rank test stratified by the randomization stratification factors of IPSS classification and FAB classification
Method Log Rank
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments

Age >= 75 years. The Kaplan-Meier median time to death was not reached due to a small number of events, so the KM 25th percentile survival time is presented.

Some of the values in outcome table #2 do not have 95% CI values so no 95% CI ranges are contained in the table. Those 95% CI ranges are added here.

Azacitidine: low range of 1.7 months and high range of 15.0 months.

Conventional Care: low range of 4.1 and high range of 7.6 months.
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0707
Comments The p value is two-sided from the log rank test stratified by the randomization stratification factors of IPSS classification and FAB classification
Method Log Rank
Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments

Gender: Male

Some of the values in outcome table #2 do not have 95% CI values so no 95% CI ranges are contained in the table. Those 95% CI ranges are added here.

Azacitidine: low range of 17.9 months and high range of 'does not exist'.

Conventional Care: low range of 10.8 and high range of 17.2 months.
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0042
Comments The p value is two-sided from the log rank test stratified by the randomization stratification factors of IPSS classification and FAB classification
Method Log Rank
Comments [Not Specified]
Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments

Gender: Female

Some of the values in outcome table #2 do not have 95% CI values so no 95% CI ranges are contained in the table. Those 95% CI ranges are added here.

Azacitidine: low range of 13.0 months and high range of 'does not exist'.

Conventional Care: low range of 8.2 and high range of 17.6 months.
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0469
Comments The p value is two-sided from the log rank test stratified by the randomization stratification factors of IPSS classification and FAB classification
Method Log Rank
Comments [Not Specified]
Hide Statistical Analysis 6
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments FAB: Refractory anemia with excess blasts (RAEB). All patients were stratified at randomization by FAB classification and IPSS score as determined by the investigator using centrally read bone marrow and cytogenetic data. Subsequently, the FAB classifications and IPSS scores were reviewed by an Independent Review Committee (IRC). The subgroup analyses presented by FAB and IPSS represent the FAB classification and IPSS scores as determined by the IRC.
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0056
Comments

95% CI ranges are added here.

Azacitidine: low range of 21.1 months and high range of 'does not exist'.

Conventional Care: low range of 9.3 and high range of 21.9 months.
Method Log Rank
Comments The p value is two-sided from the log rank test stratified by the randomization stratification factors of IPSS classification and FAB classification.
Hide Statistical Analysis 7
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments

FAB: RAEB in transformation

Some of the values in outcome table #2 do not have 95% CI values so no 95% CI ranges are contained in the table. Those 95% CI ranges are added here.

Azacitidine: low range of 11.7 months and high range of 'does not exist'.

Conventional Care: low range of 9.4 and high range of 17.0 months.
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0322
Comments The p value is two-sided from the log rank test stratified by the randomization stratification factors of IPSS classification and FAB classification
Method Log Rank
Comments [Not Specified]
Hide Statistical Analysis 8
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments

WHO: RAEB 1. The Kaplan-Meier median time to death was not reached due to a small number of events, so the KM 25th percentile survival time is presented.

Some of the values in outcome table #2 do not have 95% CI values so no 95% CI ranges are contained in the table. Those 95% CI ranges are added here.

Azacitidine: low range of 6.6 months and high range of 'does not exist'.

Conventional Care: low range of 1.8 and high range of 9.8 months.
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1679
Comments The p value is two-sided from the log rank test stratified by the randomization stratification factors of IPSS classification and FAB classification
Method Log Rank
Comments [Not Specified]
Hide Statistical Analysis 9
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments

WHO: RAEB-2

Some of the values in outcome table #2 do not have 95% CI values so no 95% CI ranges are contained in the table. Those 95% CI ranges are added here.

Azacitidine: low range of 15.9 months and high range of 'does not exist'.

Conventional Care: low range of 8.8 and high range of 19.4 months.
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0692
Comments The p value is two-sided from the log rank test stratified by the randomization stratification factors of IPSS classification and FAB classification
Method Log Rank
Comments [Not Specified]
Hide Statistical Analysis 10
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments

WHO: Other

Some of the values in outcome table #2 do not have 95% CI values so no 95% CI ranges are contained in the table. Those 95% CI ranges are added here.

Azacitidine: low range of 15.6 months and high range of 'does not exist'.

Conventional Care: low range of 11.1 and high range of 17.5 months.
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0017
Comments The p value is two-sided from the log rank test stratified by the randomization stratification factors of IPSS classification and FAB classification
Method Log Rank
Comments [Not Specified]
Hide Statistical Analysis 11
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments IPSS: Intermediate 2 All patients were stratified at randomization by FAB classification and IPSS score as determined by the investigator using centrally read bone marrow and cytogenetic data. Subsequently, the FAB classifications and IPSS scores were reviewed by an Independent Review Committee (IRC). The subgroup analyses presented by FAB and IPSS represent the FAB classification and IPSS scores as determined by the IRC.
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1030
Comments

95% CI ranges are added here.

Azacitidine: low range of 17.1 months and high range of 'does not exist'.

Conventional Care: low range of 8.7 and high range of 24.1 months.
Method Log Rank
Comments The p value is two-sided from the log rank test stratified by the randomization stratification factors of IPSS classification and FAB classification.
Hide Statistical Analysis 12
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments IPSS: High All patients were stratified at randomization by FAB classification and IPSS score as determined by the investigator using centrally read bone marrow and cytogenetic data. Subsequently, the FAB classifications and IPSS scores were reviewed by an Independent Review Committee (IRC). The subgroup analyses presented by FAB and IPSS represent the FAB classification and IPSS scores as determined by the IRC.
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0020
Comments

95% CI ranges are added here.

Azacitidine: low range of 15.0 months and high range of 'does not exist'.

Conventional Care: low range of 9.0 and high range of 17.0 months.
Method Log Rank
Comments The p value is two-sided from the log rank test stratified by the randomization stratification factors of IPSS classification and FAB classification
3.Primary Outcome
Title Number of Participants Who Died
Hide Description Count of participants who died during the study
Time Frame 42 months
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to treat population
Arm/Group Title Azacitidine Conventional Care
Hide Arm/Group Description:
Azacitidine, 75 mg/m^2/day given by subcutaneous injection for 7 days of every 28 day cycle, plus best supportive care.
Physician choice of low dose cytarabine, standard chemotherapy, or best supportive care (consisting of blood products, growth factors, antibiotics)
Overall Number of Participants Analyzed 179 179
Measure Type: Number
Unit of Measure: participants
82 113
4.Secondary Outcome
Title Kaplan-Meier Estimate for Median Time to Transformation to Acute Myeloid Leukemia (AML) or Death From Any Cause, Whichever Occurred First
Hide Description The time to transformation to AML or death from any cause (whichever occurred first) was defined as the number of days from the date of randomization until the date of documented AML transformation or death from any cause. Patients who did not transform to AML or die were censored at the date of last follow-up.
Time Frame Day 1 (randomization) to 42 months
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to treat population. Participants who either transformed to AML or died are 120 for azacitidine and 132 for conventional care. Remaining participants were censored.
Arm/Group Title Azacitidine Conventional Care
Hide Arm/Group Description:
Azacitidine, 75 mg/m^2/day given by subcutaneous injection for 7 days of every 28 day cycle, plus best supportive care.
Physician choice of low dose cytarabine, standard chemotherapy, or best supportive care (consisting of blood products, growth factors, antibiotics)
Overall Number of Participants Analyzed 179 179
Median (95% Confidence Interval)
Unit of Measure: months
13.02
(9.9 to 15.0)
7.61
(5.4 to 9.8)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0025
Comments The p value is two-sided from the log rank test stratified by the randomization stratification factors of IPSS classification and FAB classification.
Method Log Rank
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0027
Comments [Not Specified]
Method Regression, Cox
Comments [Not Specified]
Method of Estimation Estimation Parameter Hazard Ratio (HR)
Estimated Value 0.68
Confidence Interval (2-Sided) 95%
0.53 to 0.87
Estimation Comments Cox proportional hazards model stratified on the randomization factors of FAB and IPSS with model term of treatment.
5.Secondary Outcome
Title Kaplan-Meier Estimates for Median Time to Transformation to Acute Myeloid Leukemia (AML)
Hide Description The time to transformation to AML was defined as the number of days from the date of randomization until the date of documented AML transformation, defined as a bone marrow blast count ≥ 30% independent of baseline bone marrow count. Patients who did not transform to AML were censored at the date of last follow-up or date of death.
Time Frame Day 1 (randomization) to 42 months
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to treat population. Participants who were transformed to AML are 78 for azacitidine and 71 for conventional care. Remaining participants were censored based on the last bone marrow assessment.
Arm/Group Title Azacitidine Conventional Care
Hide Arm/Group Description:
Azacitidine, 75 mg/m^2/day given by subcutaneous injection for 7 days of every 28 day cycle, plus best supportive care.
Physician choice of low dose cytarabine, standard chemotherapy, or best supportive care (consisting of blood products, growth factors, antibiotics)
Overall Number of Participants Analyzed 179 179
Median (95% Confidence Interval)
Unit of Measure: months
20.66
(14.9 to 25.5)
15.44
(12.4 to 22.5)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.2555
Comments The p value is two-sided from the log rank test stratified by the randomization stratification factors of IPSS classification and FAB classification
Method Log Rank
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.2562
Comments [Not Specified]
Method Regression, Cox
Comments [Not Specified]
Method of Estimation Estimation Parameter Hazard Ratio (HR)
Estimated Value 0.83
Confidence Interval (2-Sided) 95%
0.60 to 1.15
Estimation Comments Cox proportional hazards model stratified on the randomization factors of FAB and IPSS with model term of treatment.
6.Secondary Outcome
Title Summary of Participants' Red Blood Cell (RBC) Transfusion Status for Participants Who Were Transfusion Dependent at Baseline
Hide Description Summary of dependence and independence from red blood cell (RBC) transfusion at baseline and during treatment, for patients who were dependent at baseline. A patient was considered transfusion independent at baseline if the patient had no transfusions during the 56 days prior to randomization. During study, a patient was considered transfusion independent during the on-treatment period if the patient had no transfusions during any 56 consecutive days or more. Otherwise, the patient was considered transfusion dependent.
Time Frame Day 1 (randomization) to 42 months
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to treat population
Arm/Group Title Azacitidine Conventional Care
Hide Arm/Group Description:
Azacitidine, 75 mg/m^2/day given by subcutaneous injection for 7 days of every 28 day cycle, plus best supportive care.
Physician choice of low dose cytarabine, standard chemotherapy, or best supportive care (consisting of blood products, growth factors, antibiotics)
Overall Number of Participants Analyzed 111 114
Measure Type: Number
Unit of Measure: participants
Baseline Dependent; On-Treatment Independent 50 13
Baseline Dependent; On-Treatment Dependent 61 101
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments The p value is from Fisher's exact test comparing the difference in the azacitidine group and the combined group of CCR regimens among patients who were transfusion dependent at baseline and transfusion independent during the on-treatment period.
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
7.Secondary Outcome
Title Summary of Participants' Red Blood Cell (RBC) Transfusion Status for Participants Who Were Transfusion Independent at Baseline
Hide Description Summary of dependence and independence from red blood cell (RBC) transfusion at baseline and during treatment, for patients who were independent at baseline. A patient was considered transfusion independent at baseline if the patient had no transfusions during the 56 days prior to randomization. During study, a patient was considered transfusion independent during the on-treatment period if the patient had no transfusions during any 56 consecutive days or more. Otherwise, the patient was considered transfusion dependent.
Time Frame Day 1 (randomization) to 42 months
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to treat population
Arm/Group Title Azacitidine Conventional Care
Hide Arm/Group Description:
Azacitidine, 75 mg/m^2/day given by subcutaneous injection for 7 days of every 28 day cycle, plus best supportive care.
Physician choice of low dose cytarabine, standard chemotherapy, or best supportive care (consisting of blood products, growth factors, antibiotics)
Overall Number of Participants Analyzed 68 65
Measure Type: Number
Unit of Measure: participants
Baseline Independent; On-Treatment Independent 58 37
Baseline Independent; On-Treatment Dependent 10 28
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments The p value is from Fisher's exact test comparing the difference in the azacitidine group and the combined group of CCR regimens among patients who were transfusion independent at baseline and remained transfusion independent during the on-treatment period.
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0005
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
8.Secondary Outcome
Title Summary of Participants' Platelet Transfusion Status for Participants Who Were Transfusion Dependent at Baseline
Hide Description Summary of dependence and independence from platelet transfusion at baseline and during treatment for patients who were dependent at baseline. A patient was considered transfusion independent at baseline if the patient had no transfusions during the 56 days prior to randomization. During study, a patient was considered transfusion independent during the on-treatment period if the patient had no transfusions during any 56 consecutive days or more. Otherwise, the patient was considered transfusion dependent.
Time Frame Day 1 (randomization) to 42 months
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to treat population
Arm/Group Title Azacitidine Conventional Care
Hide Arm/Group Description:
Azacitidine, 75 mg/m^2/day given by subcutaneous injection for 7 days of every 28 day cycle, plus best supportive care.
Physician choice of low dose cytarabine, standard chemotherapy, or best supportive care (consisting of blood products, growth factors, antibiotics)
Overall Number of Participants Analyzed 38 27
Measure Type: Number
Unit of Measure: participants
Baseline Dependent; On-Treatment Independent 16 11
Baseline Dependent; On-Treatment Dependent 22 16
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments The p value is from Fisher's exact test comparing the difference in the azacitidine group and the combined group of CCR regimens among patients who were transfusion dependent at baseline and transfusion independent during the on-treatment period
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 1.000
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
9.Secondary Outcome
Title Summary of Participants' Platelet Transfusion Status for Participants Who Were Transfusion Independent at Baseline
Hide Description Summary of dependence and independence from platelet transfusion at baseline and during treatment for patients who were independent at baseline. A patient was considered transfusion independent at baseline if the patient had no transfusions during the 56 days prior to randomization. During study, a patient was considered transfusion independent during the on-treatment period if the patient had no transfusions during any 56 consecutive days or more. Otherwise, the patient was considered transfusion dependent.
Time Frame Day 1 (randomization) to 42 months
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to treat population
Arm/Group Title Azacitidine Conventional Care
Hide Arm/Group Description:
Azacitidine, 75 mg/m^2/day given by subcutaneous injection for 7 days of every 28 day cycle, plus best supportive care.
Physician choice of low dose cytarabine, standard chemotherapy, or best supportive care (consisting of blood products, growth factors, antibiotics)
Overall Number of Participants Analyzed 141 152
Measure Type: Number
Unit of Measure: participants
Baseline Independent; On-Treatment Independent 126 102
Baseline Independent; On-Treatment Dependent 15 50
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments The p value is from Fisher's exact test comparing the difference in the azacitidine group and the combined group of CCR regimens among patients who were transfusion independent at baseline and remained transfusion independent during the on-treatment period.
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
10.Secondary Outcome
Title Number of Participants Considered Hematologic Responders by Investigator Determinations Using International Working Group (IWG 2000) Criteria for Myelodysplastic Syndrome (MDS)
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Investigator determined responses followed IWG criteria for

  • complete remission(CR): repeat bone marrow show <5% myeloblasts, and peripheral blood evaluations lasting >=2 months of hemoglobin(>110 g/L), neutrophils(>=1.5x10^9/L), platelets(>=100x10^9/L), blasts (0%) and no dysplasia
  • partial remission(PR) is the same as CR for peripheral blood: bone marrow shows blasts decrease by >=50% or a less advanced FAB classification from pretreatment
  • stable disease(SD) is a failure to achieve at least a partial remission, but with no evidence of progression for at least 2 months.
Time Frame Day 1 to 42 months
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Hide Analysis Population Description
Intent to treat population.
Arm/Group Title Azacitidine Conventional Care
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Azacitidine, 75 mg/m^2/day given by subcutaneous injection for 7 days of every 28 day cycle, plus best supportive care.
Physician choice of low dose cytarabine, standard chemotherapy, or best supportive care (consisting of blood products, growth factors, antibiotics)
Overall Number of Participants Analyzed 179 179
Measure Type: Number
Unit of Measure: participants
Overall (Complete + Partial Remission) 51 21
Complete Remission 30 14
Partial Remission 21 7
Stable Disease 75 65
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments Overall (Complete + Partial Remission)
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0001
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments Complete remission
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0150
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments Partial Remission
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0094
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments Stable Disease
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3297
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
11.Secondary Outcome
Title Number of Participants Showing Hematologic Improvement Using International Working Group (IWG 2000) Criteria for Myelodysplastic Syndrome (MDS) Assessed by Independent Review Committee
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IWG 2000 Criteria: Pretreatment=hemoglobin <100g/L or RBC transfusion-dependent, platelet count <100x10^9/L or platelet transfusion dependent, absolute neutrophil count <1.5x10^9/L.

Erythroid response: Major->20g/L increase or transfusion independent. Minor- 10-20g/L increase or >=50% decrease in transfusion requirements.

Platelet response: Major-absolute increase of >=30x10^9/L or platelet transfusion independence. Minor->=50% increase.

Neutrophil response: Major->=100% increase or an absolute increase of >0.5x10^9/L. Minor->=100% increase and absolute increase of <0.5x10^9/L.
Time Frame Day 1 to 42 months
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Hide Analysis Population Description
Intent to treat population.
Arm/Group Title Azacitidine Conventional Care
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Azacitidine, 75 mg/m^2/day given by subcutaneous injection for 7 days of every 28 day cycle, plus best supportive care.
Physician choice of low dose cytarabine, standard chemotherapy, or best supportive care (consisting of blood products, growth factors, antibiotics)
Overall Number of Participants Analyzed 179 179
Measure Type: Number
Unit of Measure: participants
Any Improvement n=177, 178 87 51
Erythroid Response - Major n=157, 160 62 17
Erythroid Response - Minor n=157, 160 2 1
Platelet Response - Major n=141, 129 46 18
Platelet Response - Minor n=138, 127 6 4
Neutrophil Response - Major n=131, 111 25 20
Neutrophil Response - Minor n=131, 111 5 9
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments Any Improvement
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments Erythroid Response - Major
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments Erythroid Response - Minor
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.6203
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments Platelet Response - Major
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0003
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments Platelet Response - Minor
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.7514
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
Hide Statistical Analysis 6
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments Neutrophil Response - Major
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.8695
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
Hide Statistical Analysis 7
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments Neutrophil Response - Minor
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1760
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
12.Secondary Outcome
Title Time to Disease Progression, Relapse After Complete or Partial Remission, or Death From Any Cause
Hide Description The time to disease progression, relapse after complete or partial remission (CR, PR), or death from any cause was defined as the time from the date of randomization until the first date of documented disease progression, relapse after CR or PR, or death from any cause.
Time Frame Day 1 (randomization) to 42 months
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Hide Analysis Population Description
Intent to treat population. The number of participants with disease progression, relapse after remission or death from any cause is 84 for azacitidine and 79 for conventional care. Remaining participants were censored.
Arm/Group Title Azacitidine Conventional Care
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Azacitidine, 75 mg/m^2/day given by subcutaneous injection for 7 days of every 28 day cycle, plus best supportive care.
Physician choice of low dose cytarabine, standard chemotherapy, or best supportive care (consisting of blood products, growth factors, antibiotics)
Overall Number of Participants Analyzed 179 179
Median (95% Confidence Interval)
Unit of Measure: months
14.13
(9.5 to 27.6)
8.82
(6.4 to 12.4)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0466
Comments The p value is two-sided from the log rank test which compares whether the azacitidine and control group follow the same duration curve.
Method Log Rank
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0474
Comments [Not Specified]
Method Regression, Cox
Comments [Not Specified]
Method of Estimation Estimation Parameter Hazard Ratio (HR)
Estimated Value 0.73
Confidence Interval (2-Sided) 95%
0.53 to 1.00
Estimation Comments [Not Specified]
13.Secondary Outcome
Title Duration of Any Hematologic Improvement
Hide Description The duration of improvement was defined as the time from the date of hematologic improvement until the date of first documented progression or relapse after hematologic improvement or death from any cause.
Time Frame Day 1 (randomization) to 42 months
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Hide Analysis Population Description
Intent to treat population. Participants showing hematologic improvement were 48 in azacitidine and 31 in conventional care.
Arm/Group Title Azacitidine Conventional Care
Hide Arm/Group Description:
Azacitidine, 75 mg/m^2/day given by subcutaneous injection for 7 days of every 28 day cycle, plus best supportive care.
Physician choice of low dose cytarabine, standard chemotherapy, or best supportive care (consisting of blood products, growth factors, antibiotics)
Overall Number of Participants Analyzed 87 51
Median (95% Confidence Interval)
Unit of Measure: months
13.57
(10.1 to 16.3)
5.18
(4.1 to 9.7)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0002
Comments The p value is two-sided from the log rank test which compares whether the azacitidine and control group follow the same duration curve.
Method Log Rank
Comments [Not Specified]
14.Secondary Outcome
Title Number of Infections Per Treatment Year Requiring Intravenous Antibiotics, Antifungals or Antivirals
Hide Description The on-treatment adverse event rate of infection requiring IV antibiotics, antifungals, or antivirals per patient-years. The on-treatment period was considered the period from the date of randomization to the last treatment study visit.
Time Frame Day 1 (randomization) to 42 months
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to treat population
Arm/Group Title Azacitidine Conventional Care
Hide Arm/Group Description:
Azacitidine, 75 mg/m^2/day given by subcutaneous injection for 7 days of every 28 day cycle, plus best supportive care.
Physician choice of low dose cytarabine, standard chemotherapy, or best supportive care (consisting of blood products, growth factors, antibiotics)
Overall Number of Participants Analyzed 179 179
Measure Type: Number
Unit of Measure: infections per treatment year
0.16 0.24
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1327
Comments [Not Specified]
Method exact binomial
Comments [Not Specified]
Method of Estimation Estimation Parameter Risk Ratio (RR)
Estimated Value 0.67
Confidence Interval (2-Sided) 95%
0.35 to 1.20
Estimation Comments [Not Specified]
15.Secondary Outcome
Title Number of Participants in Different Categories of Adverse Experiences During Core Study Period
Hide Description Patient counts for a variety of subsets of adverse experiences for the core study period (day 1 to 42 months). The individual options for Conventional Care Regimens (Best Supportive Care Only, Low-Dose Cytarabine, and Standard Chemotherapy) are presented as separate treatments.
Time Frame Day 1 (randomization) to 42 months
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Hide Analysis Population Description
Safety population excludes 4 Azacitidine patients, 3 Best Supportive Care Only patients, 5 Low-dose Cytarabine patients, and 6 Standard Chemotherapy patients who were randomized/assigned to those regimens but did not receive treatment.
Arm/Group Title Azacitidine Best Supportive Care Only Low-dose Cytarabine Standard Chemotherapy
Hide Arm/Group Description:
Azacitidine, 75 mg/m2/day given by subcutaneous injection for 7 days of every 28 day cycle, plus best supportive care.
Care can include transfusions, antibiotics, myeloid growth factors [G-CSF and GM CSF] for neutropenic infections until the end of the study.
Cytarabine, 20 mg/m2/day subcutaneously on Days 1-14, with 28-42 days between cycles. Plus best supportive care.
Induction cycle: Cytarabine infusion 100-200 mg/m2/day on days 1-7 + anthracycline on days 1, 2, 3 Consolidation cycle: Cytarabine infusion 100-200 mg/m2/day for 3-7 days + anthracycline on days 1 and 2 There are 28-70 days between cycles - 1 induction cycle and maximum of 2 consolidation cycles; best supportive care follows the final consolidation cycle.
Overall Number of Participants Analyzed 175 102 44 19
Measure Type: Number
Unit of Measure: participants
Patients with >=1 treatment emergent AE (TEAE) 175 97 44 19
Patients with >=1 treatment related TEAE 169 1 34 19
Patients with >=1 serious TEAE 114 71 27 14
Patients with >=1 serious treatment related TEAE 43 0 13 13
Patients w TEAE leading to discontinued treatment 22 4 6 2
Patients w TEAE leading to dose reduction 20 0 2 0
Patients w TEAE leading to dose interruption 82 0 12 0
16.Post-Hoc Outcome
Title Kaplan-Meier Estimates for Median Time to Transformation to Acute Myeloid Leukemia (AML) Based on the Last Bone Marrow Assessment
Hide Description A sensitivity analysis of time to transformation to AML during the entire study was performed based on the last bone marrow assessment. Patients were censored based on the last bone marrow assessment.
Time Frame Day 1 (randomization) to 42 months
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to treat population. Participants who were transformed to AML are 78 for azacitidine and 71 for conventional care. Remaining participants were censored based on the last bone marrow assessment.
Arm/Group Title Azacitidine Conventional Care
Hide Arm/Group Description:
Azacitidine, 75 mg/m^2/day given by subcutaneous injection for 7 days of every 28 day cycle, plus best supportive care.
Physician choice of low dose cytarabine, standard chemotherapy, or best supportive care (consisting of blood products, growth factors, antibiotics)
Overall Number of Participants Analyzed 179 179
Median (95% Confidence Interval)
Unit of Measure: months
17.80
(13.6 to 23.6)
11.48
(8.3 to 14.5)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments The p value is two-sided from the log rank test stratified by the randomization stratification factors of IPSS classification and FAB classification
Method Log Rank
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Azacitidine, Conventional Care
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Regression, Cox
Comments [Not Specified]
Method of Estimation Estimation Parameter Hazard Ratio (HR)
Estimated Value 0.50
Confidence Interval (2-Sided) 95%
0.35 to 0.70
Estimation Comments Cox proportional hazards model stratified on the randomization factors of FAB and IPSS with model term of treatment.
Time Frame Treatment-emergent AEs for the 'core study' from Day 1 to 42 months.
Adverse Event Reporting Description Safety population excludes 4 Azacitidine patients, 3 Best Supportive Care Only patients, 5 Low-dose Cytarabine patients, and 6 Standard Chemotherapy patients who were randomized/assigned to those regimens but did not receive treatment.
 
Arm/Group Title Azacitidine Best Supportive Care Only Low-dose Cytarabine Standard Chemotherapy
Hide Arm/Group Description Azacitidine, 75 mg/m2/day given by subcutaneous injection for 7 days of every 28 day cycle, plus best supportive care. Care can include transfusions, antibiotics, myeloid growth factors [G-CSF and GM CSF] for neutropenic infections until the end of the study. Cytarabine, 20 mg/m2/day subcutaneously on Days 1-14, with 28-42 days between cycles. Plus best supportive care. Induction cycle: Cytarabine infusion 100-200 mg/m2/day on days 1-7 + anthracycline on days 1, 2, 3 Consolidation cycle: Cytarabine infusion 100-200 mg/m2/day for 3-7 days + anthracycline on days 1 and 2 There are 28-70 days between cycles - 1 induction cycle and maximum of 2 consolidation cycles; best supportive care follows the final consolidation cycle.
All-Cause Mortality
Azacitidine Best Supportive Care Only Low-dose Cytarabine Standard Chemotherapy
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/--   --/--   --/-- 
Hide Serious Adverse Events
Azacitidine Best Supportive Care Only Low-dose Cytarabine Standard Chemotherapy
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   114/175 (65.14%)   71/102 (69.61%)   27/44 (61.36%)   14/19 (73.68%) 
Blood and lymphatic system disorders         
Anaemia † 1  12/175 (6.86%)  3/102 (2.94%)  2/44 (4.55%)  0/19 (0.00%) 
Bone marrow failure † 1  2/175 (1.14%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Febrile bone marrow aplasia † 1  0/175 (0.00%)  0/102 (0.00%)  2/44 (4.55%)  0/19 (0.00%) 
Febrile neutropenia † 1  19/175 (10.86%)  3/102 (2.94%)  1/44 (2.27%)  5/19 (26.32%) 
Haemolysis † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Haemorrhagic diathesis † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Leukocytosis † 1  0/175 (0.00%)  0/102 (0.00%)  1/44 (2.27%)  0/19 (0.00%) 
Leukopenia † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Neutropenia † 1  5/175 (2.86%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Pancytopenia † 1  2/175 (1.14%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Thrombocytopenia † 1  8/175 (4.57%)  2/102 (1.96%)  2/44 (4.55%)  1/19 (5.26%) 
Cardiac disorders         
Acute myocardial infarction † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Angina pectoris † 1  2/175 (1.14%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Atrial fibrillation † 1  1/175 (0.57%)  1/102 (0.98%)  0/44 (0.00%)  1/19 (5.26%) 
Cardiac failure † 1  3/175 (1.71%)  2/102 (1.96%)  1/44 (2.27%)  0/19 (0.00%) 
Cardiac failure acute † 1  2/175 (1.14%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Cardiac failure congestive † 1  0/175 (0.00%)  2/102 (1.96%)  0/44 (0.00%)  0/19 (0.00%) 
Cardiomyopathy † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Coronary artery disease † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Coronary artery occlusion † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Left ventricular failure † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Myocardial infarction † 1  2/175 (1.14%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Pericardial effusion † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Ventricular tachycardia † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Ear and labyrinth disorders         
Vertigo † 1  1/175 (0.57%)  2/102 (1.96%)  0/44 (0.00%)  0/19 (0.00%) 
Vestibular disorder † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Endocrine disorders         
Hypothyroidism † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Eye disorders         
Angle closure glaucoma † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Endophthalmitis † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Eye Haemorrhage † 1  2/175 (1.14%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Myopia † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Retinal artery occlusion † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Retinal Haemorrhage † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Retinal tear † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Strabismus † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Gastrointestinal disorders         
Abdominal pain † 1  2/175 (1.14%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Anal fissure † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Anal fistula † 1  0/175 (0.00%)  0/102 (0.00%)  1/44 (2.27%)  0/19 (0.00%) 
Caecitis † 1  0/175 (0.00%)  0/102 (0.00%)  1/44 (2.27%)  0/19 (0.00%) 
Colitis ulcerative † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Constipation † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Diarrhoea † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Dysphagia † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Food poisoning † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Gastritis † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Gastritis haemorrhagic † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Gastrointestinal haemorrhage † 1  1/175 (0.57%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Gingival bleeding † 1  2/175 (1.14%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Haematemesis † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Haemorrhoidal haemorrhage † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Ileitis † 1  0/175 (0.00%)  0/102 (0.00%)  1/44 (2.27%)  0/19 (0.00%) 
Intestinal haemorrhage † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Large intestinal haemorrhage † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Melaena † 1  0/175 (0.00%)  2/102 (1.96%)  0/44 (0.00%)  0/19 (0.00%) 
Mouth haemorrhage † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Neutropenic colitis † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  2/19 (10.53%) 
Rectal haemorrhage † 1  2/175 (1.14%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Stomatitis † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Subileus † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Tooth disorder † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
General disorders         
Asthenia † 1  1/175 (0.57%)  0/102 (0.00%)  1/44 (2.27%)  0/19 (0.00%) 
Catheter site inflammation † 1  0/175 (0.00%)  0/102 (0.00%)  1/44 (2.27%)  0/19 (0.00%) 
Chest pain † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Death † 1  0/175 (0.00%)  2/102 (1.96%)  0/44 (0.00%)  0/19 (0.00%) 
Facial pain † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Fatigue † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
General physical health deterioration † 1  2/175 (1.14%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Injection site nodule † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Injection site pain † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Mucosal inflammation † 1  0/175 (0.00%)  0/102 (0.00%)  1/44 (2.27%)  1/19 (5.26%) 
Pain † 1  0/175 (0.00%)  0/102 (0.00%)  1/44 (2.27%)  0/19 (0.00%) 
Pyrexia † 1  10/175 (5.71%)  3/102 (2.94%)  5/44 (11.36%)  0/19 (0.00%) 
Hepatobiliary disorders         
Bile duct stone † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Hepatic function abnormal † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Immune system disorders         
Drug hypersensitivity † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Infections and infestations         
Abdominal wall abscess † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Abscess neck † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Arthritis infection † 1  0/175 (0.00%)  0/102 (0.00%)  1/44 (2.27%)  0/19 (0.00%) 
Aspergillosis † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Bacteraemia † 1  2/175 (1.14%)  0/102 (0.00%)  0/44 (0.00%)  2/19 (10.53%) 
Bronchitis † 1  1/175 (0.57%)  0/102 (0.00%)  1/44 (2.27%)  0/19 (0.00%) 
Bronchopneumonia † 1  3/175 (1.71%)  3/102 (2.94%)  0/44 (0.00%)  0/19 (0.00%) 
Candidiasis † 1  0/175 (0.00%)  0/102 (0.00%)  1/44 (2.27%)  0/19 (0.00%) 
Cellulitis † 1  2/175 (1.14%)  2/102 (1.96%)  2/44 (4.55%)  0/19 (0.00%) 
Clostridium difficile colitis † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Corynebacterium infection † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Disseminated tuberculosis † 1  0/175 (0.00%)  0/102 (0.00%)  1/44 (2.27%)  0/19 (0.00%) 
Diverticulitis † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Ear infection † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Enterobacter bacteraemia † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Escherichia sepsis † 1  0/175 (0.00%)  2/102 (1.96%)  0/44 (0.00%)  0/19 (0.00%) 
Escherichia urinary tract infection † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Fungal sepsis † 1  1/175 (0.57%)  0/102 (0.00%)  1/44 (2.27%)  0/19 (0.00%) 
Gasteroenteritis † 1  1/175 (0.57%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Gasteroenteritis salmonella † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Gingival infection † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Herpes zoster † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Infection † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  1/19 (5.26%) 
Klebsiella bacteraemia † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Laryngopharyngitis † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Lobar pneumonia † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Lower respiratory tract infection † 1  2/175 (1.14%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Lung infection † 1  2/175 (1.14%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Meningitis † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Mucormycosis † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Neutropenic infection † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Neutropenic sepsis † 1  4/175 (2.29%)  1/102 (0.98%)  1/44 (2.27%)  2/19 (10.53%) 
Oral herpes † 1  1/175 (0.57%)  0/102 (0.00%)  1/44 (2.27%)  0/19 (0.00%) 
Parotitis † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Perianal abscess † 1  2/175 (1.14%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Pneumonia † 1  20/175 (11.43%)  12/102 (11.76%)  2/44 (4.55%)  3/19 (15.79%) 
Pneumonia bacterial † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Pneumonia fungal † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  2/19 (10.53%) 
Pseudomembranous colitis † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Pseudomonal sepsis † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Pulmonary tuberculosis † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Renal abscess † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Respiratory tract infection † 1  1/175 (0.57%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Salmonella sepsis † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Sepsis † 1  6/175 (3.43%)  3/102 (2.94%)  2/44 (4.55%)  0/19 (0.00%) 
Septic shock † 1  3/175 (1.71%)  1/102 (0.98%)  2/44 (4.55%)  0/19 (0.00%) 
Sialoadenitis † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Splenic abscess † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Staphylococcal bacteraemia † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Subcutaneous abscess † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Subdiaphragmatic abscess † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Tooth abscess † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Tuberculosis † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Upper respiratory tract infection † 1  2/175 (1.14%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Urinary tract infection † 1  5/175 (2.86%)  0/102 (0.00%)  2/44 (4.55%)  1/19 (5.26%) 
Bronchopulmonary aspergillosis † 1  2/175 (1.14%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Injury, poisoning and procedural complications         
Clavicle fracture † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Hip fracture † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Subdural haematoma † 1  0/175 (0.00%)  0/102 (0.00%)  1/44 (2.27%)  0/19 (0.00%) 
Synovial rupture † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Transfusion reaction † 1  3/175 (1.71%)  0/102 (0.00%)  1/44 (2.27%)  0/19 (0.00%) 
Traumatic intracranial haemorrhage † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Upper limb fracture † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Metabolism and nutrition disorders         
Diabetes mellitus † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Haemosiderosis † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Hyperkalaemia † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Hypokalaemia † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Musculoskeletal and connective tissue disorders         
Arthralgia † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Arthritis † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Back pain † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Bursitis † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Chondrocalcinosis pyrophosphate † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Osteoporosis † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Pain in extremity † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Pathological fracture † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)         
Acute myeloid leukaemia † 1  30/175 (17.14%)  35/102 (34.31%)  6/44 (13.64%)  1/19 (5.26%) 
Colon cancer † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Lung adenocarcinoma † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Myelodysplastic syndrome † 1  4/175 (2.29%)  2/102 (1.96%)  1/44 (2.27%)  0/19 (0.00%) 
Myelofibrosis † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Neoplasm prostate † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Oesophageal carcinoma † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Squamous cell carcinoma of skin † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Urinary tract neoplasm † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Nervous system disorders         
Cerebral haemorrhage † 1  4/175 (2.29%)  3/102 (2.94%)  0/44 (0.00%)  0/19 (0.00%) 
Cerebral ischaemia † 1  0/175 (0.00%)  0/102 (0.00%)  1/44 (2.27%)  0/19 (0.00%) 
Coma † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Convulsion † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Dizziness † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Grand mal convulsion † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Haemorrhage intracranial † 1  1/175 (0.57%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Headache † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Intracranial haematoma † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Loss of consciousness † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Somnolence † 1  0/175 (0.00%)  0/102 (0.00%)  1/44 (2.27%)  0/19 (0.00%) 
Syncope † 1  2/175 (1.14%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Transient ischaemic attack † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Psychiatric disorders         
Confusional state † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Delirium † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Depression † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Psychotic disorder † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Renal and urinary disorders         
Haematuria † 1  2/175 (1.14%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Nephrolithiasis † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Renal colic † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Renal failure † 1  1/175 (0.57%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Renal failure acute † 1  0/175 (0.00%)  2/102 (1.96%)  0/44 (0.00%)  0/19 (0.00%) 
Renal impairment † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Urethral stenosis † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Urinary retention † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Reproductive system and breast disorders         
Benign prostatic hyperplasia † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Metrorrhagia † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Prostatitis † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Vaginal haemorrhage † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Respiratory, thoracic and mediastinal disorders         
Acute pulmonary oedema † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Acute respiratory failure † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Bronchitis chronic † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Chronic obstructive pulmonary disease † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Epistaxis † 1  4/175 (2.29%)  3/102 (2.94%)  2/44 (4.55%)  0/19 (0.00%) 
Haemoptysis † 1  1/175 (0.57%)  0/102 (0.00%)  1/44 (2.27%)  0/19 (0.00%) 
Lung infiltration † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Pleural effusion † 1  2/175 (1.14%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Pleuritic pain † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Pneumonitis † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Pulmonary embolism † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Pulmonary fibrosis † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Pulmonary hypertension † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Respiratory failure † 1  1/175 (0.57%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Skin and subcutaneous tissue disorders         
Actinic keratosis † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Pruritus † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Purpura † 1  0/175 (0.00%)  0/102 (0.00%)  1/44 (2.27%)  0/19 (0.00%) 
Vascular disorders         
Aortic aneurysm † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Circulatory collapse † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Hypotension † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
Shock † 1  0/175 (0.00%)  1/102 (0.98%)  0/44 (0.00%)  0/19 (0.00%) 
†
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 10.0
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Azacitidine Best Supportive Care Only Low-dose Cytarabine Standard Chemotherapy
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   175/175 (100.00%)   86/102 (84.31%)   44/44 (100.00%)   19/19 (100.00%) 
Blood and lymphatic system disorders         
Anaemia † 1  84/175 (48.00%)  42/102 (41.18%)  19/44 (43.18%)  11/19 (57.89%) 
Coagulopathy † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  2/19 (10.53%) 
Febrile neutropenia † 1  6/175 (3.43%)  8/102 (7.84%)  0/44 (0.00%)  4/19 (21.05%) 
Leukopenia † 1  31/175 (17.71%)  2/102 (1.96%)  3/44 (6.82%)  6/19 (31.58%) 
Lymphadenopathy † 1  2/175 (1.14%)  0/102 (0.00%)  4/44 (9.09%)  0/19 (0.00%) 
Lymphopenia † 1  3/175 (1.71%)  0/102 (0.00%)  0/44 (0.00%)  3/19 (15.79%) 
Neutropenia † 1  114/175 (65.14%)  29/102 (28.43%)  15/44 (34.09%)  10/19 (52.63%) 
Thrombocytopenia † 1  118/175 (67.43%)  34/102 (33.33%)  21/44 (47.73%)  13/19 (68.42%) 
Cardiac disorders         
Bradycardia † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Bundle branch block right † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Cardiomegaly † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Sinus bradycardia † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Tachycardia † 1  6/175 (3.43%)  0/102 (0.00%)  1/44 (2.27%)  1/19 (5.26%) 
Eye disorders         
Chalazion † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Conjunctival haemorrhage † 1  6/175 (3.43%)  1/102 (0.98%)  0/44 (0.00%)  1/19 (5.26%) 
Conjunctivitis † 1  2/175 (1.14%)  2/102 (1.96%)  2/44 (4.55%)  1/19 (5.26%) 
Ocular hyperaemia † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Vitreous floaters † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Gastrointestinal disorders         
Abdominal discomfort † 1  2/175 (1.14%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Abdominal pain † 1  21/175 (12.00%)  7/102 (6.86%)  1/44 (2.27%)  5/19 (26.32%) 
Abdominal pain upper † 1  10/175 (5.71%)  3/102 (2.94%)  0/44 (0.00%)  2/19 (10.53%) 
Anal haemorrhage † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Aphthous stomatitis † 1  2/175 (1.14%)  0/102 (0.00%)  2/44 (4.55%)  1/19 (5.26%) 
Constipation † 1  88/175 (50.29%)  8/102 (7.84%)  11/44 (25.00%)  8/19 (42.11%) 
Diarrhoea † 1  38/175 (21.71%)  18/102 (17.65%)  10/44 (22.73%)  12/19 (63.16%) 
Dry mouth † 1  5/175 (2.86%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Dyspepsia † 1  10/175 (5.71%)  2/102 (1.96%)  0/44 (0.00%)  2/19 (10.53%) 
Gastritis † 1  3/175 (1.71%)  3/102 (2.94%)  0/44 (0.00%)  1/19 (5.26%) 
Gastrointestinal pain † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Gastrooesophageal reflux disease † 1  4/175 (2.29%)  0/102 (0.00%)  0/44 (0.00%)  2/19 (10.53%) 
Gingival bleeding † 1  10/175 (5.71%)  5/102 (4.90%)  2/44 (4.55%)  2/19 (10.53%) 
Gingival pain † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Gingivitis † 1  6/175 (3.43%)  4/102 (3.92%)  0/44 (0.00%)  1/19 (5.26%) 
Haemorrhoids † 1  12/175 (6.86%)  5/102 (4.90%)  2/44 (4.55%)  1/19 (5.26%) 
Lip haemorrhage † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  2/19 (10.53%) 
Mouth ulceration † 1  9/175 (5.14%)  6/102 (5.88%)  0/44 (0.00%)  0/19 (0.00%) 
Nausea † 1  84/175 (48.00%)  12/102 (11.76%)  16/44 (36.36%)  9/19 (47.37%) 
Oral discomfort † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Oral soft tissue disorder † 1  2/175 (1.14%)  1/102 (0.98%)  0/44 (0.00%)  1/19 (5.26%) 
Rectal haemorrhage † 1  4/175 (2.29%)  3/102 (2.94%)  2/44 (4.55%)  1/19 (5.26%) 
Stomatitis † 1  5/175 (2.86%)  1/102 (0.98%)  0/44 (0.00%)  2/19 (10.53%) 
Vomiting † 1  47/175 (26.86%)  7/102 (6.86%)  5/44 (11.36%)  6/19 (31.58%) 
General disorders         
Asthenia † 1  27/175 (15.43%)  15/102 (14.71%)  12/44 (27.27%)  2/19 (10.53%) 
Catheter site erythema † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Catheter site haematoma † 1  3/175 (1.71%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Catheter site haemorrhage † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  2/19 (10.53%) 
Catheter site pain † 1  2/175 (1.14%)  1/102 (0.98%)  1/44 (2.27%)  1/19 (5.26%) 
Chest pain † 1  9/175 (5.14%)  3/102 (2.94%)  2/44 (4.55%)  4/19 (21.05%) 
Chills † 1  5/175 (2.86%)  2/102 (1.96%)  4/44 (9.09%)  1/19 (5.26%) 
Fatigue † 1  42/175 (24.00%)  11/102 (10.78%)  10/44 (22.73%)  4/19 (21.05%) 
Gait disturbance † 1  0/175 (0.00%)  0/102 (0.00%)  1/44 (2.27%)  1/19 (5.26%) 
General physical health deterioration † 1  3/175 (1.71%)  2/102 (1.96%)  0/44 (0.00%)  1/19 (5.26%) 
Generalised oedema † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Injection site bruising † 1  9/175 (5.14%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Injection site erythema † 1  75/175 (42.86%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Injection site haematoma † 1  11/175 (6.29%)  0/102 (0.00%)  3/44 (6.82%)  0/19 (0.00%) 
Injection site induration † 1  9/175 (5.14%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Injection site pain † 1  32/175 (18.29%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Injection site rash † 1  10/175 (5.71%)  0/102 (0.00%)  0/44 (0.00%)  0/19 (0.00%) 
Injection site reaction † 1  51/175 (29.14%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Mucosal inflammation † 1  2/175 (1.14%)  0/102 (0.00%)  2/44 (4.55%)  4/19 (21.05%) 
Oedema † 1  9/175 (5.14%)  5/102 (4.90%)  1/44 (2.27%)  3/19 (15.79%) 
Oedema peripheral † 1  23/175 (13.14%)  13/102 (12.75%)  8/44 (18.18%)  3/19 (15.79%) 
Pain † 1  7/175 (4.00%)  2/102 (1.96%)  1/44 (2.27%)  5/19 (26.32%) 
Pitting oedema † 1  3/175 (1.71%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Pyrexia † 1  46/175 (26.29%)  16/102 (15.69%)  17/44 (38.64%)  11/19 (57.89%) 
Hepatobiliary disorders         
Cholestasis † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Infections and infestations         
Aspergillosis † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  2/19 (10.53%) 
Bacteraemia † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  2/19 (10.53%) 
Bacteriuria † 1  0/175 (0.00%)  0/102 (0.00%)  1/44 (2.27%)  1/19 (5.26%) 
Bronchitis † 1  16/175 (9.14%)  8/102 (7.84%)  3/44 (6.82%)  0/19 (0.00%) 
Catheter related infection † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  2/19 (10.53%) 
Catheter site infection † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  2/19 (10.53%) 
Clostridial infection † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Enterobacter infection † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Escherichia bacteraemia † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Fungal skin infection † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Influenza † 1  10/175 (5.71%)  5/102 (4.90%)  1/44 (2.27%)  0/19 (0.00%) 
Klebsiella bacteraemia † 1  0/175 (0.00%)  0/102 (0.00%)  1/44 (2.27%)  1/19 (5.26%) 
Nail infection † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Nasopharyngitis † 1  33/175 (18.86%)  13/102 (12.75%)  3/44 (6.82%)  0/19 (0.00%) 
Oral candidiasis † 1  11/175 (6.29%)  5/102 (4.90%)  2/44 (4.55%)  1/19 (5.26%) 
Oral herpes † 1  17/175 (9.71%)  5/102 (4.90%)  1/44 (2.27%)  3/19 (15.79%) 
Pharyngeal candidiasis † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Pharyngitis † 1  8/175 (4.57%)  2/102 (1.96%)  1/44 (2.27%)  1/19 (5.26%) 
Pneumonia † 1  2/175 (1.14%)  0/102 (0.00%)  3/44 (6.82%)  1/19 (5.26%) 
Pseudomonas infection † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Respiratory tract infection † 1  5/175 (2.86%)  3/102 (2.94%)  0/44 (0.00%)  1/19 (5.26%) 
Rhinitis † 1  10/175 (5.71%)  1/102 (0.98%)  1/44 (2.27%)  1/19 (5.26%) 
Sinusitis † 1  6/175 (3.43%)  1/102 (0.98%)  0/44 (0.00%)  1/19 (5.26%) 
Staphylococcal infection † 1  1/175 (0.57%)  0/102 (0.00%)  1/44 (2.27%)  1/19 (5.26%) 
Upper respiratory tract infection † 1  15/175 (8.57%)  4/102 (3.92%)  0/44 (0.00%)  0/19 (0.00%) 
Urinary tract infection † 1  12/175 (6.86%)  3/102 (2.94%)  1/44 (2.27%)  0/19 (0.00%) 
Viral upper respiratory tract infection † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Injury, poisoning and procedural complications         
Contusion † 1  14/175 (8.00%)  5/102 (4.90%)  1/44 (2.27%)  0/19 (0.00%) 
Fall † 1  5/175 (2.86%)  0/102 (0.00%)  3/44 (6.82%)  0/19 (0.00%) 
Overdose † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Procedural pain † 1  4/175 (2.29%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Scratch † 1  1/175 (0.57%)  1/102 (0.98%)  0/44 (0.00%)  1/19 (5.26%) 
Thermal burn † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Transfusion reaction † 1  19/175 (10.86%)  5/102 (4.90%)  3/44 (6.82%)  1/19 (5.26%) 
Investigations         
Alanine aminotransferase increased † 1  5/175 (2.86%)  0/102 (0.00%)  1/44 (2.27%)  1/19 (5.26%) 
Aspartate aminotransferase increased † 1  2/175 (1.14%)  0/102 (0.00%)  1/44 (2.27%)  1/19 (5.26%) 
Blood lactate dehydrogenase increased † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Blood potassium decreased † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  2/19 (10.53%) 
International normalised ratio increased † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Weight decreased † 1  14/175 (8.00%)  0/102 (0.00%)  2/44 (4.55%)  4/19 (21.05%) 
Metabolism and nutrition disorders         
Anorexia † 1  25/175 (14.29%)  9/102 (8.82%)  5/44 (11.36%)  5/19 (26.32%) 
Hypermagnesaemia † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Hyperuricaemia † 1  4/175 (2.29%)  2/102 (1.96%)  0/44 (0.00%)  1/19 (5.26%) 
Hypoalbuminaemia † 1  1/175 (0.57%)  1/102 (0.98%)  0/44 (0.00%)  1/19 (5.26%) 
Hypocalcaemia † 1  2/175 (1.14%)  0/102 (0.00%)  0/44 (0.00%)  2/19 (10.53%) 
Hypokalaemia † 1  11/175 (6.29%)  2/102 (1.96%)  1/44 (2.27%)  8/19 (42.11%) 
Hypomagnesaemia † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  2/19 (10.53%) 
Hyponatraemia † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Hypophosphataemia † 1  1/175 (0.57%)  0/102 (0.00%)  1/44 (2.27%)  1/19 (5.26%) 
Musculoskeletal and connective tissue disorders         
Arthralgia † 1  15/175 (8.57%)  8/102 (7.84%)  2/44 (4.55%)  1/19 (5.26%) 
Back pain † 1  15/175 (8.57%)  7/102 (6.86%)  4/44 (9.09%)  5/19 (26.32%) 
Bone pain † 1  7/175 (4.00%)  5/102 (4.90%)  3/44 (6.82%)  0/19 (0.00%) 
Bursitis † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  2/19 (10.53%) 
Chondrocalcinosis pyrophosphate † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Joint swelling † 1  1/175 (0.57%)  2/102 (1.96%)  1/44 (2.27%)  1/19 (5.26%) 
Muscle spasms † 1  10/175 (5.71%)  5/102 (4.90%)  3/44 (6.82%)  0/19 (0.00%) 
Muscular weakness † 1  3/175 (1.71%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Musculoskeletal chest pain † 1  1/175 (0.57%)  2/102 (1.96%)  1/44 (2.27%)  1/19 (5.26%) 
Musculoskeletal pain † 1  9/175 (5.14%)  3/102 (2.94%)  1/44 (2.27%)  3/19 (15.79%) 
Neck pain † 1  2/175 (1.14%)  1/102 (0.98%)  0/44 (0.00%)  2/19 (10.53%) 
Pain in extremity † 1  11/175 (6.29%)  4/102 (3.92%)  4/44 (9.09%)  4/19 (21.05%) 
Nervous system disorders         
Dizziness † 1  17/175 (9.71%)  7/102 (6.86%)  2/44 (4.55%)  2/19 (10.53%) 
Headache † 1  25/175 (14.29%)  8/102 (7.84%)  6/44 (13.64%)  6/19 (31.58%) 
Lethargy † 1  13/175 (7.43%)  2/102 (1.96%)  0/44 (0.00%)  0/19 (0.00%) 
Sinus headache † 1  2/175 (1.14%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Syncope † 1  4/175 (2.29%)  1/102 (0.98%)  1/44 (2.27%)  2/19 (10.53%) 
Psychiatric disorders         
Anxiety † 1  9/175 (5.14%)  1/102 (0.98%)  2/44 (4.55%)  3/19 (15.79%) 
Depression † 1  9/175 (5.14%)  3/102 (2.94%)  2/44 (4.55%)  1/19 (5.26%) 
Hallucination † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Insomnia † 1  15/175 (8.57%)  3/102 (2.94%)  5/44 (11.36%)  4/19 (21.05%) 
Sleep disorder † 1  6/175 (3.43%)  0/102 (0.00%)  0/44 (0.00%)  3/19 (15.79%) 
Renal and urinary disorders         
Chromaturia † 1  1/175 (0.57%)  0/102 (0.00%)  1/44 (2.27%)  1/19 (5.26%) 
Haematuria † 1  10/175 (5.71%)  1/102 (0.98%)  0/44 (0.00%)  2/19 (10.53%) 
Haemoglobinuria † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Nephropathy toxic † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Proteinuria † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Urethral obstruction † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Reproductive system and breast disorders         
Prostatitis † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Testicular swelling † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Vaginal inflammation † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Respiratory, thoracic and mediastinal disorders         
Cough † 1  34/175 (19.43%)  15/102 (14.71%)  5/44 (11.36%)  6/19 (31.58%) 
Dyspnoea † 1  26/175 (14.86%)  5/102 (4.90%)  7/44 (15.91%)  3/19 (15.79%) 
Dyspnoea exertional † 1  9/175 (5.14%)  1/102 (0.98%)  2/44 (4.55%)  0/19 (0.00%) 
Epistaxis † 1  26/175 (14.86%)  14/102 (13.73%)  6/44 (13.64%)  3/19 (15.79%) 
Hypoxia † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Nasal congestion † 1  3/175 (1.71%)  0/102 (0.00%)  0/44 (0.00%)  3/19 (15.79%) 
Pharyngolaryngeal pain † 1  11/175 (6.29%)  3/102 (2.94%)  2/44 (4.55%)  1/19 (5.26%) 
Pleural effusion † 1  1/175 (0.57%)  2/102 (1.96%)  0/44 (0.00%)  1/19 (5.26%) 
Pleurisy † 1  1/175 (0.57%)  1/102 (0.98%)  0/44 (0.00%)  1/19 (5.26%) 
Pneumonitis † 1  1/175 (0.57%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Productive cough † 1  5/175 (2.86%)  2/102 (1.96%)  1/44 (2.27%)  1/19 (5.26%) 
Pulmonary oedema † 1  1/175 (0.57%)  0/102 (0.00%)  1/44 (2.27%)  2/19 (10.53%) 
Skin and subcutaneous tissue disorders         
Alopecia † 1  7/175 (4.00%)  0/102 (0.00%)  0/44 (0.00%)  3/19 (15.79%) 
Dermatitis allergic † 1  1/175 (0.57%)  1/102 (0.98%)  0/44 (0.00%)  2/19 (10.53%) 
Ecchymosis † 1  8/175 (4.57%)  7/102 (6.86%)  2/44 (4.55%)  0/19 (0.00%) 
Erythema † 1  13/175 (7.43%)  3/102 (2.94%)  1/44 (2.27%)  2/19 (10.53%) 
Erythema nodosum † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Petechiae † 1  20/175 (11.43%)  4/102 (3.92%)  7/44 (15.91%)  3/19 (15.79%) 
Pruritus † 1  20/175 (11.43%)  2/102 (1.96%)  2/44 (4.55%)  3/19 (15.79%) 
Purpura † 1  4/175 (2.29%)  1/102 (0.98%)  2/44 (4.55%)  1/19 (5.26%) 
Rash † 1  18/175 (10.29%)  1/102 (0.98%)  1/44 (2.27%)  5/19 (26.32%) 
Rash macular † 1  1/175 (0.57%)  1/102 (0.98%)  1/44 (2.27%)  1/19 (5.26%) 
Rash papular † 1  1/175 (0.57%)  1/102 (0.98%)  0/44 (0.00%)  1/19 (5.26%) 
Rash pruritic † 1  2/175 (1.14%)  0/102 (0.00%)  1/44 (2.27%)  1/19 (5.26%) 
Skin lesion † 1  7/175 (4.00%)  3/102 (2.94%)  0/44 (0.00%)  2/19 (10.53%) 
Urticaria † 1  2/175 (1.14%)  1/102 (0.98%)  1/44 (2.27%)  1/19 (5.26%) 
Vascular disorders         
Arteriosclerosis † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Haematoma † 1  21/175 (12.00%)  10/102 (9.80%)  6/44 (13.64%)  1/19 (5.26%) 
Hypertension † 1  15/175 (8.57%)  4/102 (3.92%)  6/44 (13.64%)  3/19 (15.79%) 
Hypotension † 1  10/175 (5.71%)  2/102 (1.96%)  0/44 (0.00%)  4/19 (21.05%) 
Jugular vein thrombosis † 1  0/175 (0.00%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
Pallor † 1  4/175 (2.29%)  1/102 (0.98%)  0/44 (0.00%)  1/19 (5.26%) 
Peripheral vascular disorder † 1  2/175 (1.14%)  0/102 (0.00%)  0/44 (0.00%)  1/19 (5.26%) 
†
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 10.0
[Not Specified]
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.
The investigator shall have the right to publish and/or present study data provided that the investigator shall (i) furnish the sponsor a copy of any proposed publication or presentation generally thirty (30) days in advance of the submission, (ii) delete any confidential information of the sponsor, and (iii) delay submission for generally up to ninety (90) days to permit the preparation and filing of intellectual property applications or until sponsor gives its consent in a timely manner.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: CL Beach
Organization: Celgene Corporation
EMail: CLBeach@celgene.com
Publications of Results:
Fenaux P, Mufti GJ, Hellstrom-Lindberg E, Santini V, Gattermann N, Germing U, Sanz G, List AF, Gore S, Seymour JF, Dombret H, Backstrom J, Zimmerman L, McKenzie D, Beach CL, Silverman LR. Azacitidine prolongs overall survival compared with conventional care regimens in elderly patients with low bone marrow blast count acute myeloid leukemia. J Clin Oncol. 2010 Feb 1;28(4):562-9. doi: 10.1200/JCO.2009.23.8329. Epub 2009 Dec 21.
Fenaux P, Gattermann N, Seymour JF, Hellstrom-Lindberg E, Mufti GJ, Duehrsen U, Gore SD, Ramos F, Beyne-Rauzy O, List A, McKenzie D, Backstrom J, Beach CL. Prolonged survival with improved tolerability in higher-risk myelodysplastic syndromes: azacitidine compared with low dose ara-C. Br J Haematol. 2010 Apr;149(2):244-9. doi: 10.1111/j.1365-2141.2010.08082.x. Epub 2010 Feb 5. Erratum In: Br J Haematol. 2010 Jun;149(6):919.
Santini V, Fenaux P, Mufti GJ, Hellstrom-Lindberg E, Silverman LR, List A, Gore SD, Seymour JF, Backstrom J, Beach CL. Management and supportive care measures for adverse events in patients with myelodysplastic syndromes treated with azacitidine*. Eur J Haematol. 2010 Aug;85(2):130-8. doi: 10.1111/j.1600-0609.2010.01456.x. Epub 2010 Apr 12.
Seymour JF, Fenaux P, Silverman LR, Mufti GJ, Hellstrom-Lindberg E, Santini V, List AF, Gore SD, Backstrom J, McKenzie D, Beach CL. Effects of azacitidine compared with conventional care regimens in elderly (>/= 75 years) patients with higher-risk myelodysplastic syndromes. Crit Rev Oncol Hematol. 2010 Dec;76(3):218-27. doi: 10.1016/j.critrevonc.2010.04.005. Epub 2010 May 6.
Fenaux P, Mufti GJ, Hellstrom-Lindberg E, Santini V, Finelli C, Giagounidis A, Schoch R, Gattermann N, Sanz G, List A, Gore SD, Seymour JF, Bennett JM, Byrd J, Backstrom J, Zimmerman L, McKenzie D, Beach C, Silverman LR; International Vidaza High-Risk MDS Survival Study Group. Efficacy of azacitidine compared with that of conventional care regimens in the treatment of higher-risk myelodysplastic syndromes: a randomised, open-label, phase III study. Lancet Oncol. 2009 Mar;10(3):223-32. doi: 10.1016/S1470-2045(09)70003-8. Epub 2009 Feb 21.
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Layout table for additonal information
Responsible Party: Celgene
ClinicalTrials.gov Identifier: NCT00071799    
Other Study ID Numbers: AZA PH GL 2003 CL001
First Submitted: October 31, 2003
First Posted: November 5, 2003
Results First Submitted: March 2, 2010
Results First Posted: July 5, 2010
Last Update Posted: October 29, 2019