Low Dose Decitabine, Low Dose Azacitidine, or Standard Dose Azacitidine in Treating Patients with Transfusion-Dependent Myelodysplastic Syndrome or Best Supportive Care in Patients with Transfusion-Independent Myelodysplastic Syndrome
- Sign an Institutional Review Board (IRB)-approved informed consent document
- International Prognostic Scoring System (IPSS) low- or intermediate-1-risk MDS, including chronic myelomonocytic leukemia (CMML)-1
- Eastern Cooperative Oncology Group (ECOG) performance status of =< 3 at study entry
- Serum creatinine =< 2 mg/dL
- Total bilirubin =< 2 x upper limit of normal (ULN)
- Alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase [SGPT]) =< 2 x ULN; aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase [SGOT]) =< 2 x ULN
- Women of childbearing potential must have a negative serum or urine pregnancy test within 7 days and will also need to use contraceptives; men must agree not to father a child and agree to use a condom if his partner is of child bearing potential
- Breast feeding females
- Prior therapy with decitabine or azacitidine
I. Compare the event-free survival rates of two different drugs: decitabine (DAC) versus azacitidine (AZA) on an abbreviated schedule to a standard arm of AZA given over 5 days in patients with low-risk MDS transfusion-dependent and to best supportive care (BSC) in patients with low-risk MDS transfusion-independent.
I. Compare the response rates for the transfusion independent and the transfusion dependent patients. For example the response rate of two different drugs DAC versus AZA on abbreviated schedule to a standard arm of AZA given over 5 days.
II. Evaluate the durability of response, the overall and transformation-free survival rates, and the safety profile of 2 different drugs.
III. The quality of life protocol (2014-0636) titled “Interventional Validation of an MDS-Specific Measure of Quality of Life: Assessing the Responsiveness of the QUALMS-1 to Different Hypomethylating Agent Regimens for Low and Intermediate Risk Disease” was written specifically as a companion study to protocol 2014-0112 and may be offered as an optional assessment to patients enrolled onto this protocol.
OUTLINE: Transfusion-dependent patients are randomized to 1 of 3 treatment arms (Arm I, Arm II, or Arm III). Transfusion-independent patients are assigned to Arm IV.
ARM I (LOW DOSE DECITABINE): Patients receive decitabine intravenously (IV) over 1 hour on days 1-3. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.
ARM II (LOW DOSE AZACITIDINE): Patients receive azacitidine IV or subcutaneously (SC) on days 1-3. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.
ARM III (STANDARD DOSE AZACITIDINE): Patients receive azacitidine IV or SC on days 1-5. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.
ARM IV (SUPPORTIVE CARE): Patients receive best supportive care.
After completion of study treatment, patients are followed up every 6-12 months for up to 5 years.
Trial Phase Phase II
Trial Type Treatment
M D Anderson Cancer Center
- Primary ID 2014-0112
- Secondary IDs NCI-2014-02339
- Clinicaltrials.gov ID NCT02269280