5-azacytidine Treatment Versus 5-azacytidine Followed by Allogeneic Stem Cell Transplantation in Elderly Patients With Myelodysplastic Syndrome (MDS)

Comparison Between 5-azacytidine Treatment and 5-azacytidine Followed by Allogeneic Stem Cell Transplantation in Elderly Patients With Advanced MDS According to Donor Availability

5-azacytidine treatment prolongs survival in patients with myelodysplastic syndrome (MDS), but does not cure the disease. Allogeneic stem cell transplantation is a curative treatment option but is associated with a high risk treatment-related morbidity and mortality. In the current trial allogeneic stem cell transplantation will be compared to 5-azacytidine only treatment according to donor availability in elderly patients with MDS (55-70 years).

5-azacytidine treatment prolongs survival in patients with myelodysplastic syndrome (MDS), but does not cure the disease. Allogeneic stem cell transplantation is a curative treatment option but is associated with a high risk treatment-related morbidity and mortality. Dose-reduced conditioning prior transplantation allows also treatment of elderly patients with MDS. In the current trial allogeneic stem cell transplantation will be compared to 5-azacytidine only treatment according to donor availability in elderly patients with MDS (55-70 years).

Inclusion Criteria:

• Patients with proven de novo or therapy-related MDS / CMML (WBC <13 GPT/l)according to FAB and risk profile according to IPSS: intermediate II- risk or high-risk or intermediate I with high-risk cytogenetic (according to IPSS, taking into account that IPSS, however, was not validated for t- MDS), patients with secondary AML (according to WHO) and blasts ≤ 30 % (= RAEB-t according to FAB)
• Previously untreated or maximal 1 cycle of 5-azacytidine (Vidaza®)
• Male or Female; Age 55 - 70 years
• Understand and voluntarily sign an informed consent form
• ECOG performance status of ≤ 2 at study entry
• Adequate renal and liver function: creatinine and bilirubin < 3 x the upper limit of normal
• Sufficient cardiac function (ejection fraction > 30 %)

Exclusion Criteria:

• Blasts > 30 % in bone marrow at time of diagnosis
• Central nervous involvement
• Severe irreversible renal, hepatic, pulmonary or cardiac disease, such as
• Total bilirubin, SGPT or SGOT ≥ 3 times upper the normal level
• Left ventricular ejection fraction < 30 %
• Creatinine clearance < 30 ml/min
• DLCO < 35 % and/or receiving supplementary continuous oxygen
• Pregnant or breastfeeding female subject
• Patients with a life-expectancy of less than six months because of another debilitating disease
• Serious psychiatric or psychological disorders
• Uncontrolled invasive fungal infection at time of registration
• Known positive for HIV or acute infectious hepatitis, type A, B or C
• Participation in another study with ongoing use of unlicensed investigational product from 28 days before study enrollment until the end of the study