MDS
Conditions
Keywords
Low risk MDS, Transplantation
Brief summary
Comparison of survival in patients with or without a matched donor at 36 months
Detailed description
Patients with a matched donor (8/8 at molecular level unrelated donor or matched sibling) received an allogeneic hematopoietic stem cell transplantation. Patients without a matched donor received the best available treatment. All patients will be followed at least 36 months or until the end of the study.
Interventions
OTHERtransplantation
allogeneic hematopoietic stem cell transplantation in patients with donor
Sponsors
Novartis
Neovii Biotech
Groupe Francophone des Myelodysplasies
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 69 Years
Healthy volunteers
No
Inclusion criteria
1. Signed Informed consent 2. Classical IPSS intermediate 1 or low myelodysplastic syndrome associated with at least one poor prognosis feature: 1. Intermediate or higher risk revised IPSS 2. RBC transfusion dependent anemia and failure to 2 or more lines or therapy (including EPO, Lenalidomide or demethylating agent…) 3. thrombocytopenia \< 20 G/L requiring transfusion 4. neutropenia \< 0.5 G/L associated with severe infection (defined as requiring hospitalization) 3. Patient aged ≥ 18 and \< 70 years For young patients, 18-45 years, Fanconi disease and dyskeratosis should be ruled out 4. Patient for whom a transplantation from a matched donor, (8/8 (HLA A, B, C, DRB1) identical at molecular level)unrelated donor or matched sibling), is considered irrespective of donor availability 5. Performance status 0-2 on the Eastern Cooperative Oncology Group (ECOG) Scale (At time of screening) 6. Negative pregnancy and adequate contraception (including in male patients wishing to father), if relevant. 7. Wash-out of at least 30 days since a previous treatment with Vidaza, Lenalidomide, EPO or any other treatment inducing cytopenias.
Exclusion criteria
1. MDS classified according to classical IPSS as intermediate 2 or High risk 2. Transformation in Acute myeloid Leukemia (AML) 3. Severe active infection or any other uncontrolled severe condition. 4. Organ dysfunctions including the following * Hepatic : total bilirubin \> 2 times upper limit of normal (ULN) (except moderate unconjugated hyperbilirubinemia due to intra medullary hemolysis or Gilbert syndrome) , alanine transaminase (ALT) and aspartate transaminase (AST) \> 3xULN * Symptomatic respiratory chronic failure * Symptomatic cardiac failure * Renal clearance \< 60ml/min 5. Prior malignancy (except in situ cervix carcinoma, limited basal cell carcinoma, or other tumors if not active during the last 3 years) 6. MDS with the following causal germline disease : Fanconi anemia, GATA2 related syndromes and telomere disorders
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| overall survival | 36 months | comparison of overall survival in patients with or without a matched donor (8/8 unrelated donor or matched sibling) at 36 months |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| number of patients with complete response at 36 month | 36 months | comparison between patients with or without a donor for cumulative incidence of complete response at 36 month |
| number of patients with transformation in AML at 36 month | 36 months | comparison between patients with or without a donor for cumulative incidence of transformation in AML at 36 month |
| proportion of patients with iron overload | 16 months | proportion of patients with iron overload (Serum Ferritin (SF)\>1000 ng/mL or Red Blood Cells transfusion\>20) at time of inclusion and at 16 month after inclusion for non-transplanted patients and 12 months post-transplant for transplanted patients |
| quality of life | 12, 24 and 36 months | comparison of quality of life in patients with or without a matched donor, quality of life assessed by questionnaire (EORTC version 3) at inclusion, 12, 24 and 36 months |
| efficiency of chelation | 3 and 16 months | the effect of chelation will be assessed at 3 month after inclusion for all patient and post transplant by measuring Serum ferritin level |
| number of patients with adverse events grade III and IV as assessed by CTCAE v4.0 | 36 months | comparison between patients with or without a donor for number of Grade III and IV toxicities (hematological and non-hematological) recorded according to NCI CTCAE criteria versions 4.0 during the 36 months |
| evolution of innovative iron markers including Non-transferrin binding iron (NTBI), labile plasmatic Iron (LPI) and Hepcidine | 3 and 16 months | evolution of innovative iron markers including Non-transferrin binding iron (NTBI), labile plasmatic Iron (LPI) and Hepcidine measured at time of inclusion, at 3 month and 16 month post-inclusion for all patients; In transplanted patients these markers will be measured just before conditioning regimen (J-5), Just before the transplantation (J0), at D7, 30, 100 and 12 month after transplant. |
Countries
France
Outcome results
None listed