Evaluation of Azacitidine in Transfusion Dependent Patients With Low-risk Myelodysplastic Syndrome (MDS) or Chronic Myelomonocytic Leukemia (CMML)

Inclusion criteria

• Must be 18 years of age at the time of signing the informed consent form
• MDS at IPSS Low or Int-1, or mixed MDS/MPD; either CMML with < 10% marrow blasts or RARS-T
• Patients with high or intermediate probability for response according to the predictive model (see Hellstrom-Lindberg et al, Br J Haematol 99:344-51 1997)should be refractory to EPO / darbepoetin (equivalent to > 60 000 U of EPO / week for > 8 weeks) followed by EPO + G-CSF for > 8 weeks, or biosimilar drugs in equipotent doses, or EPO + G-CSF upfront for 8 weeks. Patients with low probability for response according to the predictive model, could be included without prior EPO/G-CSF treatment
• Transfusion need >4 units over the last 8 weeks, or >8 units over the last 26 weeks.
• Subject has signed the informed consent document.
• Men and women of childbearing potential must use effective contraception during, and for up to 3 months after treatment.

Exclusion criteria

• Pregnant or lactating females.
• Patients who are eligible for curative treatment
• Expected survival less than 24 weeks.
• Symptomatic thrombocytopenia / active bleeding
• Patients with JAK-2 positive RARS-T if eligible for new investigational drugs
• Serum biochemical values as follows

• Serum creatinine >2.0 mg/dL (177 micromol/L)
• Serum aminotransferase (AST)/serum glutamic-oxaloacetic transaminase (SGOT) or alanine transaminase (ALT)/serum glutamate pyruvate transaminase (SGPT) >3.0 x upper limit of normal (ULN)
• Serum total bilirubin >1.5 mg/dL (26 micromol/L)
• Uncontrolled systemic infection
• Considered not capable of following the study protocol