Description:
Comparison of survival in patients with or without a matched donor at 36 months
Title
- Brief Title: Allogeneic Hematopoietic Stem Cell Transplantation in Patients With Myelodysplastic Syndrome Low Risk
- Official Title: Allogeneic Hematopoietic Stem Cell Transplantation in Patients With Low or Intermediate-1 Myelodysplastic Syndrome: A Prospective Multicenter Phase II Study Based on Donor Availability on Behalf of the GFM & SFGM-TC
Clinical Trial IDs
- ORG STUDY ID:
MDS-ALLO-RISK
- SECONDARY ID:
2015-A00292-47
- NCT ID:
NCT02757989
Conditions
Purpose
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.
Trial Arms
Name | Type | Description | Interventions |
---|
Patients with donor | Experimental | Patients with a matched donor (8/8 at molecular level unrelated donor or matched sibling) | |
Patients without donor | No Intervention | Patients without a matched donor | |
Eligibility Criteria
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
Maximum Eligible Age: | 69 Years |
Minimum Eligible Age: | 18 Years |
Eligible Gender: | All |
Healthy Volunteers: | No |
Primary Outcome Measures
Measure: | overall survival |
Time Frame: | 36 months |
Safety Issue: | |
Description: | comparison of overall survival in patients with or without a matched donor (8/8 unrelated donor or matched sibling) at 36 months |
Secondary Outcome Measures
Measure: | quality of life |
Time Frame: | 12, 24 and 36 months |
Safety Issue: | |
Description: | 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 |
Measure: | number of patients with complete response at 36 month |
Time Frame: | 36 months |
Safety Issue: | |
Description: | comparison between patients with or without a donor for cumulative incidence of complete response at 36 month |
Measure: | number of patients with transformation in AML at 36 month |
Time Frame: | 36 months |
Safety Issue: | |
Description: | comparison between patients with or without a donor for cumulative incidence of transformation in AML at 36 month |
Measure: | proportion of patients with iron overload |
Time Frame: | 16 months |
Safety Issue: | |
Description: | 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 |
Measure: | evolution of innovative iron markers including Non-transferrin binding iron (NTBI), labile plasmatic Iron (LPI) and Hepcidine |
Time Frame: | 3 and 16 months |
Safety Issue: | |
Description: | 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. |
Measure: | efficiency of chelation |
Time Frame: | 3 and 16 months |
Safety Issue: | |
Description: | the effect of chelation will be assessed at 3 month after inclusion for all patient and post transplant by measuring Serum ferritin level |
Measure: | number of patients with adverse events grade III and IV as assessed by CTCAE v4.0 |
Time Frame: | 36 months |
Safety Issue: | |
Description: | 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 |
Details
Phase: | N/A |
Primary Purpose: | Interventional |
Overall Status: | Active, not recruiting |
Lead Sponsor: | Groupe Francophone des Myelodysplasies |
Trial Keywords
- Low risk MDS
- Transplantation
Last Updated
July 14, 2021