This is a multicenter, multinational, non-randomized, non-controlled open-label phase II trial to evaluate the safety and efficacy of treosulfan in a combination regimen with fludarabine as conditioning therapy prior to allogeneic stem cell transplantation (SCT) in patients with MDS. The aim is to demonstrate a clinical benefit compared to historical data with intravenous busulfan.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
45
14 g/m2/d, day -6 to -4
Helsinki University Central Hospital
Helsinki, Finland
Efficacy: Evaluation of engraftment
Time frame: 4 years
Safety: Evaluation of CTC grade 3 and 4 adverse events between Day -6 and Day +28: hyperbilirubinemia and mucositis/stomatitis, veno-occlusive disease, seizures
Time frame: 4 years
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