Title
Thalidomide at Low Dose for the Treatment of Patient With Myelodysplastic Syndromes - THAL-SMD-200
Thalidomide for the Treatment of Cytopenias of Patients With Low Risk Myelodysplastic Syndromes
Phase
Phase 2Lead Sponsor
Groupe Francophone des MyelodysplasiesStudy Type
InterventionalStatus
Completed No Results PostedIndication/Condition
Myelodysplastic SyndromesIntervention/Treatment
thalidomide ...Study Participants
112The GFM previously conducted a dose-escalating phase II trial of thalidomide in MDS with a minimum dose of 200mg/d and a maximum dose 800mg/d. Responses were evaluated according to IWG criteria at week 16 and thalidomide continued up to week 56 in responders. 82% patients received at least 8 weeks of treatment and were evaluable. 59% had hematological improvement, mainly on the erythroid lineage (Increase of Hemoglobin). Most responses were observed at low doses and between 4 and 8 weeks.
The objectives of this trial (Thal-SMD-20) are to evaluate the efficacy and tolerance of lower doses thalidomide in low risk MDS patients with transfusion-dependant anemia.
Thalidomide:
First part of the trial: 82 patients at 200mg/day given at bedtime x 12 weeks, decreased to 100mg/day if grade 1 or 2 side. Stopped temporally for 1 week if grade 3 or 4 side effects. Then reintroduced at the same dose. If side effects again, definitively stopped.
Responses evaluated at 12 weeks according to IWG criteria for the erythroid lineage
At week 12:
If no Hematological improvement (HI): increased to 300mg/day for 8 weeks and then eventually to 400mg/day for 8 weeks more, if no HI.
If Hematological improvement (HI): continued at the same dose.
Second part of the trial: 30 patients treated at 50mg/day x 12 weeks. Responses evaluated at 12 weeks according to IWG criteria for the erythroid lineage
At week 12:
If no Hematological improvement (HI): increased to 100mg/day for 8 weeks and then eventually to 200mg/day for 8 weeks more, if no HI.
If Hematological improvement (HI): continued at the same dose.
Inclusion Criteria: Patients ≥18 years, with IPSS Low or Int-1 MDS Transfusion dependant anemia above 2 packed red blood cells (PRBC)/month ECOG index = 0, 1, 2 No peripheral neurological disease Exclusion Criteria: MDS patients with IPSS Int-2 or High Patients with less than 2 packed red blood cells (PRBC)/month Patients with previous history of venous thrombosis Patient treated with EPO +/- G-CSF in the 2 months before inclusion in the protocol Patient having received intensive chemotherapy in the 3 months before inclusion in the protocol Patient having received Thalidomide in a previous protocol Patient presenting an iron, B12 vitamin or folic acid uncorrected deficiency Patient with peripheral neurological disease Patient not being able to subject itself to a regular clinical and biological follow-up Pregnant patient or patient in a period of lactation Patient refusing to take a contraceptive treatment through out all the study Patient receiving drugs able to interfere with the mechanism of action of Thalidomide Patient refusing to sign the informed consent.
