Clinical and Biological Evaluation of Azacitidine in Transfusion-dependent Patients With Low and Intermediate-1 Risk MDS, and Low-risk CMML, Who Are Either Refractory to or Not Eligible for Treatment With Erythropoietin +/- G-CSF
- 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
- 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
- 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.
- 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
1. Serum creatinine >2.0 mg/dL (177 micromol/L)
2. 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)
3. Serum total bilirubin >1.5 mg/dL (26 micromol/L)
- Uncontrolled systemic infection
- Considered not capable of following the study protocol