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

Universitätsklinikum Hamburg-Eppendorf

Status and phase

Completed

Phase 2

Conditions

Myelodysplastic Syndrome

Chronic Myelomonocytic Leukemia

Treatments

Procedure: 5-azacytidine until progress

Procedure: allogeneic stem cell transplantation

Study type

Interventional

Funder types

Other

Identifiers

NCT01404741

VidazaAlloStudy

Details and patient eligibility

About

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).

Full description

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).

Enrollment

191 patients

Sex

All

Ages

55 to 70 years old

Volunteers

No Healthy Volunteers

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