Trial Outcomes & Findings for A Phase II Study of Intravenous Azacitidine Alone in Patients With Myelodysplastic Syndromes (NCT NCT00384956)

Last Updated: 2016-12-12

Results Overview

Defined according to the modified International Working Group (IWG) (2006) response criteria for myelodysplasia: CR=bone marrow with \<5% myeloblasts and 0% peripheral blasts, hemoglobin ≥11g/dL, platelets ≥ 100 x 10\^9/L, and neutrophils ≥1.0 x 10\^9/L. Residual dysplasia was allowed. PR= All of the CR criteria if abnormal before treatment except: bone marrow blasts decreased by ≥50% over pretreatment but still \>5%.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

25 participants

Primary outcome timeframe

After 4 cycles of therapy (up to 112 days after start of treatment)

Results posted on

2016-12-12

Participant Flow

Enrollment to the study started on 08/17/2006 and enrollment to the study closed on 06/10/2008.

Participant milestones

Participant milestones
Measure	Azacitidine Azacitidine 75 mg/m2 IV on days 1-5 of each 28 day cycle. Patients that do not respond after two cycles will have the dose increased to 100 mg/m2. Patients who achieve a CR will receive 3 additional 28 day cycles and then begin treatment on days 1-5 of a 56 day cycle. Individuals who demonstrate a loss of response will resume 28 day cycles.
Overall Study STARTED	25
Overall Study COMPLETED	22
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Reasons for withdrawal
Measure	Azacitidine Azacitidine 75 mg/m2 IV on days 1-5 of each 28 day cycle. Patients that do not respond after two cycles will have the dose increased to 100 mg/m2. Patients who achieve a CR will receive 3 additional 28 day cycles and then begin treatment on days 1-5 of a 56 day cycle. Individuals who demonstrate a loss of response will resume 28 day cycles.
Overall Study Ineligible (acute myeloid leukemia)	3

Baseline Characteristics

A Phase II Study of Intravenous Azacitidine Alone in Patients With Myelodysplastic Syndromes

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Azacitidine n=22 Participants Azacitidine 75 mg/m2 IV on days 1-5 of each 28 day cycle. Patients that do not respond after two cycles will have the dose increased to 100 mg/m2. Patients who achieve a CR will receive 3 additional 28 day cycles and then begin treatment on days 1-5 of a 56 day cycle. Individuals who demonstrate a loss of response will resume 28 day cycles.
Age, Continuous	69.5 years n=93 Participants
Gender Female	9 Participants n=93 Participants
Gender Male	13 Participants n=93 Participants
Region of Enrollment United States	22 participants n=93 Participants
Eastern Cooperative Oncology Group (ECOG) Performance Status 0	4 participants n=93 Participants
Eastern Cooperative Oncology Group (ECOG) Performance Status 1	15 participants n=93 Participants
Eastern Cooperative Oncology Group (ECOG) Performance Status 2	2 participants n=93 Participants
Eastern Cooperative Oncology Group (ECOG) Performance Status Unknown	1 participants n=93 Participants
Duration of myelodysplastic syndrome (MDS) at study entry	15.5 days n=93 Participants
French, American, British (FAB) classification RA	6 participants n=93 Participants
French, American, British (FAB) classification CMML	2 participants n=93 Participants
French, American, British (FAB) classification RAEB	12 participants n=93 Participants
French, American, British (FAB) classification RAEB-t	2 participants n=93 Participants
World Health Organization (WHO) classification RA	3 participants n=93 Participants
World Health Organization (WHO) classification RCMD	3 participants n=93 Participants
World Health Organization (WHO) classification CMML-1	2 participants n=93 Participants
World Health Organization (WHO) classification RAEB-1	3 participants n=93 Participants
World Health Organization (WHO) classification RAEB-2	9 participants n=93 Participants
World Health Organization (WHO) classification AML	2 participants n=93 Participants
Cytogenetics Normal	7 participants n=93 Participants
Cytogenetics Intermediate	4 participants n=93 Participants
Cytogenetics Complex	11 participants n=93 Participants
International Prognostic Scoring System (IPSS) Low	1 participants n=93 Participants
International Prognostic Scoring System (IPSS) Int-1	8 participants n=93 Participants
International Prognostic Scoring System (IPSS) Int-2	7 participants n=93 Participants
International Prognostic Scoring System (IPSS) High	6 participants n=93 Participants
Bone marrow cellularity at baseline	68 percentage of bone marrow cellularity n=93 Participants
Transfusion dependence Red blood cell	4 participants n=93 Participants
Transfusion dependence Platelet	3 participants n=93 Participants
Transfusion dependence Red blood cell and platelet	3 participants n=93 Participants

PRIMARY outcome

Timeframe: After 4 cycles of therapy (up to 112 days after start of treatment)

Outcome measures

Outcome measures
Measure	Azacitidine n=22 Participants Azacitidine 75 mg/m2 IV on days 1-5 of each 28 day cycle. Patients that do not respond after two cycles will have the dose increased to 100 mg/m2. Patients who achieve a CR will receive 3 additional 28 day cycles and then begin treatment on days 1-5 of a 56 day cycle. Individuals who demonstrate a loss of response will resume 28 day cycles.
Rate of Complete Remission (CR) and Partial Remission (PR) Complete remission (CR)	5 participants
Rate of Complete Remission (CR) and Partial Remission (PR) Partial remission (PR)	1 participants

SECONDARY outcome

Timeframe: 4 weeks following last azacitidine dose [median number of cycles 4.5 (1-20)]

International Working Group (IWG) for Myelodysplasia (MDS).

Outcome measures

Outcome measures
Measure	Azacitidine n=22 Participants Azacitidine 75 mg/m2 IV on days 1-5 of each 28 day cycle. Patients that do not respond after two cycles will have the dose increased to 100 mg/m2. Patients who achieve a CR will receive 3 additional 28 day cycles and then begin treatment on days 1-5 of a 56 day cycle. Individuals who demonstrate a loss of response will resume 28 day cycles.
Rate of Hematologic Improvement	0 participants

SECONDARY outcome

Timeframe: 4 weeks following last azacitidine dose [median number of cycles 4.5 (1-20)]

Population: Only participants with baseline transfusion dependence were assessed for this outcome measure.

Outcome measures

Outcome measures
Measure	Azacitidine n=10 Participants Azacitidine 75 mg/m2 IV on days 1-5 of each 28 day cycle. Patients that do not respond after two cycles will have the dose increased to 100 mg/m2. Patients who achieve a CR will receive 3 additional 28 day cycles and then begin treatment on days 1-5 of a 56 day cycle. Individuals who demonstrate a loss of response will resume 28 day cycles.
Rate of Transfusion Independence Red blood cell	2 participants
Rate of Transfusion Independence Platelet	1 participants
Rate of Transfusion Independence Red blood cell and platelet	0 participants

SECONDARY outcome

Timeframe: 2 years after first dose of study drug or until participant is lost to follow-up or dies

Population: This secondary outcome was not analyzed.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 2 years after first dose of study drug or until participant is lost to follow-up or dies

Overall survival is defined as the date of first dose of study drug to the date of death from any cause.

Outcome measures

Outcome measures
Measure	Azacitidine n=22 Participants Azacitidine 75 mg/m2 IV on days 1-5 of each 28 day cycle. Patients that do not respond after two cycles will have the dose increased to 100 mg/m2. Patients who achieve a CR will receive 3 additional 28 day cycles and then begin treatment on days 1-5 of a 56 day cycle. Individuals who demonstrate a loss of response will resume 28 day cycles.
Rate of Overall Survival	444 days Interval 23.0 to At the time the data was analyzed, the upper range of overall survival was not reached.

SECONDARY outcome

Timeframe: 2 years after first dose of study drug or until participant is lost to follow-up or dies

Population: This secondary outcome was not analyzed.

Outcome measures

Outcome data not reported

POST_HOC outcome

Timeframe: 4 weeks following last dose of azacitidine [median number of cycles 4.5 (1-20)]

Outcome measures

Outcome measures
Measure	Azacitidine n=22 Participants Azacitidine 75 mg/m2 IV on days 1-5 of each 28 day cycle. Patients that do not respond after two cycles will have the dose increased to 100 mg/m2. Patients who achieve a CR will receive 3 additional 28 day cycles and then begin treatment on days 1-5 of a 56 day cycle. Individuals who demonstrate a loss of response will resume 28 day cycles.
Time to Best Response	108 days Interval 27.0 to 192.0

POST_HOC outcome

Timeframe: 2 years after first dose of study drug or until participant is lost to follow-up or dies

Outcome measures

Outcome measures
Measure	Azacitidine n=22 Participants Azacitidine 75 mg/m2 IV on days 1-5 of each 28 day cycle. Patients that do not respond after two cycles will have the dose increased to 100 mg/m2. Patients who achieve a CR will receive 3 additional 28 day cycles and then begin treatment on days 1-5 of a 56 day cycle. Individuals who demonstrate a loss of response will resume 28 day cycles.
Duration of Response (DOR)	450 days Interval 169.0 to At the time that the data was analyzed, the upper range for DOR had not been reached.

POST_HOC outcome

Timeframe: 2 years after first dose of study drug or until participant is lost to follow-up or dies

PFS is defined as the interval from the date of first dose of study drug to date of treatment failure, recurrence, or death due to any cause. Disease progression * for patients w/ \<5% blasts; a ≥50% increase in blasts to \>5% blasts * for patients w/ 5% to 10% blasts; a ≥50 increase to \>10% blasts * for patients w/ 10% to 20% blasts; a ≥50% increase to \>20% blasts * for patients w/ 20% to 30% blasts; a ≥50% increase to \>30% blasts * One or more of the following ≥50% decrement from maximum remission/response levels in granulocytes or platelets, reduction in hemoglobin concentration by ≥2 g/dL or transfusion dependence

Outcome measures

Outcome measures
Measure	Azacitidine n=22 Participants Azacitidine 75 mg/m2 IV on days 1-5 of each 28 day cycle. Patients that do not respond after two cycles will have the dose increased to 100 mg/m2. Patients who achieve a CR will receive 3 additional 28 day cycles and then begin treatment on days 1-5 of a 56 day cycle. Individuals who demonstrate a loss of response will resume 28 day cycles.
Progression-free Survival (PFS)	339 days Interval 32.0 to At the time the data was analyzed, the upper range of PFS was not reached.

Adverse Events

Azacitidine

Serious events: 12 serious events

Other events: 24 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Azacitidine n=24 participants at risk Azacitidine 75 mg/m2 IV on days 1-5 of each 28 day cycle. Patients that do not respond after two cycles will have the dose increased to 100 mg/m2. Patients who achieve a CR will receive 3 additional 28 day cycles and then begin treatment on days 1-5 of a 56 day cycle. Individuals who demonstrate a loss of response will resume 28 day cycles.
Respiratory, thoracic and mediastinal disorders Pneumonia	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Infections and infestations Infection with unknown neutrophils (sepsis)	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Gastrointestinal disorders Rectal hemorrhage	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Nervous system disorders Dizziness	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Respiratory, thoracic and mediastinal disorders Dyspnea	8.3% 2/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Respiratory, thoracic and mediastinal disorders Cough	8.3% 2/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
General disorders Fever	8.3% 2/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Cardiac disorders Myocardial infarction	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Infections and infestations Infection (sepsis)	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Renal and urinary disorders Renal failure	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
General disorders Chills	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Respiratory, thoracic and mediastinal disorders Atelectasis	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Respiratory, thoracic and mediastinal disorders Pleural effusion	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Cardiac disorders Hypotension	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
General disorders Fatigue	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Musculoskeletal and connective tissue disorders Arthralgia	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Infections and infestations Neutropenic fever	20.8% 5/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
General disorders Death due to progressive disease	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Gastrointestinal disorders Constipation	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Infections and infestations Infection without neutropenia	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.

Other adverse events

Other adverse events
Measure	Azacitidine n=24 participants at risk Azacitidine 75 mg/m2 IV on days 1-5 of each 28 day cycle. Patients that do not respond after two cycles will have the dose increased to 100 mg/m2. Patients who achieve a CR will receive 3 additional 28 day cycles and then begin treatment on days 1-5 of a 56 day cycle. Individuals who demonstrate a loss of response will resume 28 day cycles.
Gastrointestinal disorders Abdominal pain	12.5% 3/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Investigations Alkaline phosphatase	16.7% 4/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Metabolism and nutrition disorders Anorexia	37.5% 9/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Musculoskeletal and connective tissue disorders Arthralgia	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Respiratory, thoracic and mediastinal disorders Atelectasis	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Cardiac disorders Atrial fibrillation	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Musculoskeletal and connective tissue disorders Back pain	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
General disorders Bilateral lower extremity edema	20.8% 5/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
General disorders Bilateral upper extremity edema	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Injury, poisoning and procedural complications Bruising	16.7% 4/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Musculoskeletal and connective tissue disorders Chest/ribs pain	20.8% 5/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Gastrointestinal disorders Colitis	12.5% 3/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Psychiatric disorders Confusion	8.3% 2/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Gastrointestinal disorders Constipation	25.0% 6/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Respiratory, thoracic and mediastinal disorders Cough	16.7% 4/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Gastrointestinal disorders Diarrhea	29.2% 7/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Nervous system disorders Dizziness	25.0% 6/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Gastrointestinal disorders Dysgeusia	25.0% 6/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Gastrointestinal disorders Dyspepsia/heartburn	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Respiratory, thoracic and mediastinal disorders Dyspnea	20.8% 5/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Investigations Elevated creatinine	20.8% 5/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Respiratory, thoracic and mediastinal disorders Epistaxis	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Musculoskeletal and connective tissue disorders Face pain	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
General disorders Fatigue	33.3% 8/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
General disorders Fever - no infection	16.7% 4/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Nervous system disorders Headache	12.5% 3/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Blood and lymphatic system disorders Hemoglobin	58.3% 14/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Musculoskeletal and connective tissue disorders Hip pain	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Investigations Hyperbilirubinemia	33.3% 8/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Investigations Hypercalcemia	8.3% 2/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Investigations Hyperglycemia	37.5% 9/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Investigations Hyperkalemia	20.8% 5/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Investigations Hypermagnesemia	16.7% 4/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Investigations Hypernatremia	16.7% 4/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Cardiac disorders Hypertension	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Investigations Hypoalbuminemia	45.8% 11/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Investigations Hypocalcemia	37.5% 9/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Investigations Hypoglycemia	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Investigations Hypokalemia	20.8% 5/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Investigations Hyponatremia	33.3% 8/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Respiratory, thoracic and mediastinal disorders Hypoxia	20.8% 5/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Infections and infestations Infection with neutropenia	37.5% 9/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Psychiatric disorders Insomnia	8.3% 2/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
General disorders Left apical mass	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Musculoskeletal and connective tissue disorders Leg pain	8.3% 2/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Skin and subcutaneous tissue disorders Lesion	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Investigations Leukocytes (WBC)	45.8% 11/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Gastrointestinal disorders Lower gastrointestinal hemorrhage	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Blood and lymphatic system disorders Lymphadenopathy	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Investigations Lymphopenia	16.7% 4/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Psychiatric disorders Mental status change	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Psychiatric disorders Mood alteration - depression	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Gastrointestinal disorders Mucositis	25.0% 6/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Musculoskeletal and connective tissue disorders Myalgia	8.3% 2/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Respiratory, thoracic and mediastinal disorders Nasal drainage/congestion	8.3% 2/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Gastrointestinal disorders Nausea	70.8% 17/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Infections and infestations Neutropenic fever	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Investigations Neutrophils (ANC)	54.2% 13/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Gastrointestinal disorders Oral cavity hemorrhage	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Cardiac disorders Palpitations	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Blood and lymphatic system disorders Petechiae	8.3% 2/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Investigations Platelets	33.3% 8/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Respiratory, thoracic and mediastinal disorders Pleural effusion	12.5% 3/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Respiratory, thoracic and mediastinal disorders Pneumonitis/pulmonary infiltrates	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Skin and subcutaneous tissue disorders Rash/desquamation	8.3% 2/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Gastrointestinal disorders Right abdominal infection	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
General disorders Right lower quadrant mass	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
General disorders Rigors/chills	16.7% 4/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Investigations SGOT (AST)	29.2% 7/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Investigations SGPT (ALT)	37.5% 9/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Nervous system disorders Sensory neuropathy	8.3% 2/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Psychiatric disorders Somnolence	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
General disorders Subclavian nodularity	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
General disorders Sweating	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Cardiac disorders Tachycardia	12.5% 3/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Ear and labyrinth disorders Tinnitus	8.3% 2/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Musculoskeletal and connective tissue disorders Upper extremity pain	8.3% 2/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Investigations Uric acid	12.5% 3/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Renal and urinary disorders Urinary urgency	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Reproductive system and breast disorders Vaginal hemorrhage	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Respiratory, thoracic and mediastinal disorders Voice changes	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Gastrointestinal disorders Vomiting	33.3% 8/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.
Investigations Weight loss	4.2% 1/24 • Adverse events were collected from start of treatment until 4 weeks following last dose of azacitidine. 24 patients out of 25 patients received at least the first dose of azacitidine. One patient was enrolled in the study but did not receive treatment due to a catheter port infection.

Additional Information

Ravi Vij, M.D.

Washington University School of Medicine

Phone: 314-454-8304

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place