Myelodysplastic Syndromes
Conditions
Keywords
myelodysplastic syndrome, erythropoetin
Brief summary
This is an open-label, randomized, multicenter, phase III study Patients with baseline Hb comprised between 9 and 10.5g/dl will be randomized to receive EPO Alfa 60000 UI/week for at least 12 weeks: * Either at diagnosis Or * at the Hb threshold chosen for RBC transfusions (must be \< 9g/dl)
Detailed description
in this trial we will compare the early introduction of EPO alfa to the delayed introduction in lower risk MDS with non RBC transfusion dependent anemia. At enrollment patients will be randomised in the 2 arms (early and delayed start of EPO alfa). Treatment Regimen Epoetin alfa 60000 UI/week for at least 12 weeks 1. Early onset arm: early onset of EPO ALFA 60000 IU/week , at patient inclusion 2. Delayed onset arm: late introduction of EPO ALFA 60000 IU/week, whenever the patient reaches the level chosen RBC transfusions (based on age, comorbidities, anticipated tolerance of anemia).
Interventions
DRUGEPREX
60 000 U/week for at least 12 weeks
Sponsors
Groupe Francophone des Myelodysplasies
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
randomazed study
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Age ≥ 18 years 2. MDS according to WHO 2016 criteria, with low or int 1 classical IPSS 3. Non-RBC transfusion dependent anemia 4. Hb level between 9 and 10.5g/dl (at the center's lab) 5. Hb level should be at least 1g/dl higher than the Hb threshold chosen to start RBC transfusions based on age, comorbidities and predicted clinical tolerance of anemia (this transfusion threshold should be chosen between 8 and 9g/dl) 6. Serum EPO level \<500U/l 7. No other cause of anemia (including iron deficiency, vitamin B12 or B9 deficiency, hemolysis, hypothyroidism….) 8. Performance status \<=2
Exclusion criteria
1. Higher risk MDS (IPSS intermediate-2 or high) 2. Del 5q 3. Baseline Hemoglobin level \> 10.5 g/dl or \<9g/dl 4. Transfusion threshold (based on age , comorbidities…) \>9g/dl 5. Transfusion threshold less than 1 g/dl below baseline Hb level 6. RBC transfusion dependence. Patients may have received only one transfusion series for MDS prior to inclusion 7. CMML , if \>10 % BM blasts or WBC\>13.000/mm3 8. Uncontrolled hypertension 9. Uncontrolled cardiovascular disease including angina pectoris or cardiac failure 10. Renal failure: Creatinine clearance\<40ml/min (using MDRD formula) 11. Pregnancy (positive bettaHCG) or nursing
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Time to RBC transfusion dependence in non RBC transfusion dependent lower risk MDS patients with anemia with early (at inclusion of the patient) versus delayed onset,( at the threshold chosen for RBC transfusion) of EPO ALFA | 12 weeks | RBC transfusion dependence will be defined by requirement of at least transfusions of 2 PRBC within an interval of less than 8 weeks, given for Hb \<8g/dl or \<9g/dl according to comorbidities and in the absence of other cause of anemia (bleeding, surgery…), taking into account only transfusions given at least 12 weeks after onset of treatment with EPO ALFA. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Erythroid response (according to IWG 2006 criteria) | 12 weeks | Erythroid response (according to IWG 2006 criteria) after 12 weeks of EPO ALFA treatment |
| response duration to EPO ALFA | 4 years | response duration to EPO ALFA measured from the date of enrollment until failure |
| Overall survival | 4 years | Overall survival measured from the date of enrollment to death or the date of last contact |
Countries
France
Contacts
PRINCIPAL_INVESTIGATORSophie Park, Prof
University Hospital, Grenoble
Outcome results
None listed