Decitabine Versus Azacitidine in Myelodysplastic Syndrome Patients With Low and Intermediate-1 Risk

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ClinicalTrials.gov Identifier: NCT01720225

Recruitment Status : Completed

First Posted : November 2, 2012

Results First Posted : December 24, 2020

Last Update Posted : December 24, 2020

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Sponsor:

Information provided by (Responsible Party):

M.D. Anderson Cancer Center

Study Type	Interventional
Study Design	Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: None (Open Label); Primary Purpose: Treatment
Condition	Leukemia
Interventions	Drug: Decitabine Drug: Azacitidine
Enrollment	113

Participant Flow

Recruitment Details	Recruitment Period: November 2012 to February 2016
Pre-assignment Details


Arm/Group Title	Decitabine	Azacitidine
Arm/Group Description	Patients randomized to receive Deci...	Patients randomized to receive Azac...
Arm/Group Description	Patients randomized to receive Decitabine 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days. Decitabine: 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days.	Patients randomized to receive Azacitidine 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days. Azacitidine: 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days.
Period Title: Overall Study
Started	73	40
Completed	70	39
Not Completed	3	1
Reason Not Completed
Withdrawal by Subject	0	1
Did not complete 2 cycles of therapy	3	0

Baseline Characteristics

Arm/Group Title		Decitabine	Azacitidine	Total
Arm/Group Description		Patients randomized to receive Deci...	Patients randomized to receive Azac...	Total of all reporting groups
Arm/Group Description		Patients randomized to receive Decitabine 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days. Decitabine: 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days.	Patients randomized to receive Azacitidine 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days. Azacitidine: 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days.	Total of all reporting groups
Overall Number of Baseline Participants		73	40	113
Baseline Analysis Population Description		[Not Specified]
Baseline Analysis Population Description		[Not Specified]
Age, Categorical Measure Type: Count of Participants Unit of measure: Participants
	Number Analyzed	73 participants	40 participants	113 participants
	<=18 years	0 0.0%	0 0.0%	0 0.0%
	Between 18 and 65 years	26 35.6%	12 30.0%	38 33.6%
	>=65 years	47 64.4%	28 70.0%	75 66.4%
Age, Continuous Median (Full Range) Unit of measure: Years
	Number Analyzed	73 participants	40 participants	113 participants
		70 (44 to 88)	70 (53 to 84)	70 (44 to 88)
Sex: Female, Male Measure Type: Count of Participants Unit of measure: Participants
	Number Analyzed	73 participants	40 participants	113 participants
	Female	24 32.9%	16 40.0%	40 35.4%
	Male	49 67.1%	24 60.0%	73 64.6%
Race (NIH/OMB) Measure Type: Count of Participants Unit of measure: Participants
	Number Analyzed	73 participants	40 participants	113 participants
	American Indian or Alaska Native	0 0.0%	0 0.0%	0 0.0%
	Asian	0 0.0%	0 0.0%	0 0.0%
	Native Hawaiian or Other Pacific Islander	0 0.0%	0 0.0%	0 0.0%
	Black or African American	2 2.7%	1 2.5%	3 2.7%
	White	71 97.3%	39 97.5%	110 97.3%
	More than one race	0 0.0%	0 0.0%	0 0.0%
	Unknown or Not Reported	0 0.0%	0 0.0%	0 0.0%
Region of Enrollment Measure Type: Number Unit of measure: Participants
United States	Number Analyzed	73 participants	40 participants	113 participants
United States		73	40	113

Outcome Measures

1.Primary Outcome


Title	Participants With a Response
Description	Overall Response = complete remission (CR) + partial remission (PR) + ...
Description	Overall Response = complete remission (CR) + partial remission (PR) + marrow CR (mCR) + hematologic improvement (HI). CR is normalization of peripheral blood and bone marrow with <5% bone marrow blasts, a peripheral blood granulocyte count > (1.0 x 10^9/L, and platelet count > 100 x 10^9/L). PR is same as CR except for the presence of 6-15% marrow blasts, or 50% reduction if <15% at start of treatment. Marrow CR is blasts </= 5% and decreased by >/=50% from baseline. HI is platelets increase by 50% and to above 30 x 10^9/L untransfused (if lower than that pre-therapy; or hemoglobin increase by 2 g/dl; or transfusion independent; or splenomegaly reduction by > 50%; or monocytosis reduction by > 50% if pretreatment > 5 X1 0^9/L.
Time Frame	56 days

Outcome Measure Data

Analysis Population Description

[Not Specified]


Arm/Group Title	Decitabine	Azacitidine
Arm/Group Description:	Patients randomized to receive Deci...	Patients randomized to receive Azac...
Arm/Group Description:	Patients randomized to receive Decitabine 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days. Decitabine: 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days.	Patients randomized to receive Azacitidine 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days. Azacitidine: 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days.
Overall Number of Participants Analyzed	70	39
Measure Type: Count of Participants Unit of Measure: Participants
	49 70.0%	19 48.7%

2.Secondary Outcome


Title	Number of Participants Who Became Transfusion Independent
Description	Participants who were transfusion dependent at baseline prior to start...
Description	Participants who were transfusion dependent at baseline prior to starting therapy on the Decitabine or Azacitidine arm will be analyzed for transfusion independence. Transfusion independence defined as being transfusion-free for ≥8 consecutive weeks between the first dose and study treatment discontinuation.
Time Frame	8 weeks

Outcome Measure Data

Analysis Population Description

Participants analyzed for Transfusion Independence must have been transfusion dependent at baseline. Thirty-seven participants in the Decitabine arm and nineteen participants in the Azacitidine arm where transfusion dependent prior to treatment on this study.


Arm/Group Title	Decitabine	Azacitidine
Arm/Group Description:	Patients randomized to receive Deci...	Patients randomized to receive Azac...
Arm/Group Description:	Patients randomized to receive Decitabine 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days. Decitabine: 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days.	Patients randomized to receive Azacitidine 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days. Azacitidine: 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days.
Overall Number of Participants Analyzed	37	19
Measure Type: Count of Participants Unit of Measure: Participants
	12 32.4%	3 15.8%

Adverse Events

Time Frame	3 years, 3 months
Adverse Event Reporting Description	[Not Specified]

Arm/Group Title	Decitabine		Azacitidine
Arm/Group Description	Patients randomized to receive Deci...		Patients randomized to receive Azac...
Arm/Group Description	Patients randomized to receive Decitabine 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days. Decitabine: 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days.		Patients randomized to receive Azacitidine 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days. Azacitidine: 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days.
All-Cause Mortality
	Decitabine		Azacitidine
	Affected / at Risk (%)		Affected / at Risk (%)
Total	7/73 (9.59%)		2/40 (5.00%)
Serious Adverse Events Serious Adverse Events
	Decitabine		Azacitidine
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	32/73 (43.84%)		14/40 (35.00%)
Blood and lymphatic system disorders
Neutropenic Fever ^† 1	9/73 (12.33%)	17	2/40 (5.00%)	2
Cardiac disorders
Heart Failure ^† 1	2/73 (2.74%)	3	0/40 (0.00%)	0
Myocardial Infarction ^† 1	1/73 (1.37%)	1	0/40 (0.00%)	0
Pericarditis ^† 1	1/73 (1.37%)	1	0/40 (0.00%)	0
Ear and labyrinth disorders
Otitis Externa ^† 1	1/73 (1.37%)	1	0/40 (0.00%)	0
Gastrointestinal disorders
Ascites ^† 1	1/73 (1.37%)	1	0/40 (0.00%)	0
Diverticulitis ^† 1	0/73 (0.00%)	0	1/40 (2.50%)	1
Gastroenteritis ^† 1	1/73 (1.37%)	1	0/40 (0.00%)	0
Gastrointestinal Bleed ^† 1	1/73 (1.37%)	1	0/40 (0.00%)	0
Nausea ^† 1	1/73 (1.37%)	1	0/40 (0.00%)	0
Rectal Hermorrhage ^† 1	1/73 (1.37%)	1	0/40 (0.00%)	0
Sialadenitis ^† 1	0/73 (0.00%)	0	1/40 (2.50%)	1
Small Intestine Obstruction ^† 1	0/73 (0.00%)	0	1/40 (2.50%)	1
General disorders
Back Pain ^† 1	1/73 (1.37%)	1	0/40 (0.00%)	0
Chest Pain ^† 1	1/73 (1.37%)	1	1/40 (2.50%)	1
Edema Limbs ^† 1	0/73 (0.00%)	0	1/40 (2.50%)	1
Fatigue ^† 1	1/73 (1.37%)	1	0/40 (0.00%)	0
Fever ^† 1	2/73 (2.74%)	3	0/40 (0.00%)	0
Headache ^† 1	1/73 (1.37%)	1	0/40 (0.00%)	0
Pain ^† 1	1/73 (1.37%)	1	0/40 (0.00%)	0
Pain Abdominal Abcess ^† 1	1/73 (1.37%)	1	0/40 (0.00%)	0
Pain Right Extremity ^† 1	0/73 (0.00%)	0	1/40 (2.50%)	1
Right Hip Pain ^† 1	0/73 (0.00%)	0	1/40 (2.50%)	1
Infections and infestations
Appendicitis ^† 1	1/73 (1.37%)	1	0/40 (0.00%)	0
Cellulitis ^† 1	0/73 (0.00%)	0	1/40 (2.50%)	1
Ear Infection ^† 1	1/73 (1.37%)	1	0/40 (0.00%)	0
Tooth Infection ^† 1	1/73 (1.37%)	1	0/40 (0.00%)	0
Joint Infection ^† 1	1/73 (1.37%)	1	0/40 (0.00%)	0
Leg Infection ^† 1	1/73 (1.37%)	1	0/40 (0.00%)	0
Lung Infection ^† 1	3/73 (4.11%)	3	0/40 (0.00%)	0
Pneumonia ^† 1	6/73 (8.22%)	7	1/40 (2.50%)	4
Salmonella Bactremia ^† 1	0/73 (0.00%)	0	1/40 (2.50%)	1
Sepsis ^† 1	4/73 (5.48%)	4	0/40 (0.00%)	0
Submandibular Cellulitis ^† 1	1/73 (1.37%)	1	1/40 (2.50%)	1
Urinary Tract Infection ^† 1	2/73 (2.74%)	2	1/40 (2.50%)	1
Wound Infection ^† 1	1/73 (1.37%)	1	0/40 (0.00%)	0
Injury, poisoning and procedural complications
Fall ^† 1	0/73 (0.00%)	0	1/40 (2.50%)	1
Food Poisoning ^† 1	0/73 (0.00%)	0	1/40 (2.50%)	1
Nervous system disorders
Altered Mental Status ^† 1	0/73 (0.00%)	0	1/40 (2.50%)	1
Intracranial Hemorrhage ^† 1	1/73 (1.37%)	1	0/40 (0.00%)	0
Syncopal episode ^† 1	1/73 (1.37%)	1	0/40 (0.00%)	0
Renal and urinary disorders
Acute Kidney Injury ^† 1	1/73 (1.37%)	1	0/40 (0.00%)	0
Renal Failure ^† 1	1/73 (1.37%)	1	0/40 (0.00%)	0
Reproductive system and breast disorders
Vaginal Fistual ^† 1	0/73 (0.00%)	0	1/40 (2.50%)	1
Respiratory, thoracic and mediastinal disorders
Acute Respiratory Distress Syndrome ^† 1	1/73 (1.37%)	1	0/40 (0.00%)	0
Bronchopulmonary Hemorrhage ^† 1	0/73 (0.00%)	0	1/40 (2.50%)	1
Hemoptysis ^† 1	1/73 (1.37%)	1	0/40 (0.00%)	0
Left plerual effusion ^† 1	0/73 (0.00%)	0	1/40 (2.50%)	1
Right atelectasis ^† 1	1/73 (1.37%)	1	0/40 (0.00%)	0
Right Pleural Effusion ^† 1	1/73 (1.37%)	1	0/40 (0.00%)	0
Respiratory Failure ^† 1	3/73 (4.11%)	3	0/40 (0.00%)	0
Shortness of Breath ^† 1	1/73 (1.37%)	1	0/40 (0.00%)	0
Skin and subcutaneous tissue disorders
Groin Abcess ^† 1	1/73 (1.37%)	1	0/40 (0.00%)	0
Skin Ulceration ^† 1	1/73 (1.37%)	1	0/40 (0.00%)	0
Surgical and medical procedures
Cholecystectomy ^† 1	1/73 (1.37%)	1	0/40 (0.00%)	0
Vascular disorders
Hypertension ^† 1	1/73 (1.37%)	1	0/40 (0.00%)	0
1 Term from vocabulary, CTCAE (4.0) † Indicates events were collected by systematic assessment
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	5%
	Decitabine		Azacitidine
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	20/73 (27.40%)		16/40 (40.00%)
Gastrointestinal disorders
Nausea ^† 1	11/73 (15.07%)	11	6/40 (15.00%)	6
Constipation ^† 1	3/73 (4.11%)	3	6/40 (15.00%)	6
Diarrhea ^† 1	2/73 (2.74%)	2	3/40 (7.50%)	3
General disorders
Fatigue ^† 1	6/73 (8.22%)	6	4/40 (10.00%)	4
1 Term from vocabulary, CTCAE (4.0) † 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 NOT 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.

Results Point of Contact

Layout table for Results Point of Contact information

Name/Title:	Elias Joseph MD./Professor
Organization:	The University of Texas MD Anderson Cancer Center
Phone:	713-792-4764
EMail:	ejabbour@mdanderson.org

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

Jabbour E, Short NJ, Montalban-Bravo G, Huang X, Bueso-Ramos C, Qiao W, Yang H, Zhao C, Kadia T, Borthakur G, Pemmaraju N, Sasaki K, Estrov Z, Cortes J, Ravandi F, Alvarado Y, Komrokji R, Sekeres MA, Steensma DP, DeZern A, Roboz G, Kantarjian H, Garcia-Manero G. Randomized phase 2 study of low-dose decitabine vs low-dose azacitidine in lower-risk MDS and MDS/MPN. Blood. 2017 Sep 28;130(13):1514-1522. doi: 10.1182/blood-2017-06-788497. Epub 2017 Aug 3.

Layout table for additonal information

Responsible Party:	M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier:	NCT01720225
Other Study ID Numbers:	2012-0507 NCI-2012-02215 ( Registry Identifier: NCI CTRP )
First Submitted:	October 31, 2012
First Posted:	November 2, 2012
Results First Submitted:	December 1, 2020
Results First Posted:	December 24, 2020
Last Update Posted:	December 24, 2020

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