Myelodysplastic Syndromes
Conditions
Keywords
Low risk myelodysplastic syndromes, MDS, Bone marrow diseases, Thalidomide, Cytopenias, Anemia
Brief summary
The 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.
Detailed description
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.
Interventions
DRUGThalidomide
Sponsors
Groupe Francophone des Myelodysplasies
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Efficacy evaluated at week 12 according to the IWG criterias | — |
Secondary
| Measure | Time frame |
|---|---|
| Safety | — |
Countries
France
Outcome results
None listed