Low Doses of Thalidomide (Thal) in Low Risk MDS with Transfusion-Dependant Anemia: The GFM THAL-SMD-200 Trial

Autor: Caroline Elie, Francois Dreyfus, Achille Aouba, Pierre Fenaux, Agnès Guerci, Frédéric Bauduer, Martine Gardembas, Jerome Tamburini, D. Vassilief, Christian Berthou, Didier Bouscary, M.C. Quarre, S. Vaultier, Laurence Legros, Aspasia Stamatoullas, Laurence Sanhes, Jean-Jacques Sotto, Odile Beyne-Rauzy
Rok vydání: 2006
Předmět:
Zdroj: Blood. 108:2673-2673
ISSN: 1528-0020
0006-4971
DOI: 10.1182/blood.v108.11.2673.2673
Popis: Background: Thal has shown some efficacy in low risk MDS, but with dose-limiting toxicity requiring discontinuation in > 50% pts (Bouscary D, BJH, 2005, 131, 609 and other groups). Thus we assessed Thal at lower dose in such MDS. Methods: From Jan 2003 to Oct 2005, we performed a prospective study of low dose Thal in 87 MDS pts with transfusion-dependant anemia (THAL-SMD-200 trial). The first 59 pts received Thal at 200 mg/d, whereas the last 28 pts received 50 mg/d. Responses (IWG) were evaluated at week 12. Results: 48 M and 39 F pts were included. Median age 73.6 years. IPSS Low 34 pts, INT-1 44 pts, INT-2 9 pts. Mean number of RBC units transfused within 2 months of inclusion 6 (2–16). Median ANC, plts, Hb were 1960/μl (0–24160), 164 000/μL (1 000–789 000) and 8,2 (5,1–14,7) resp. 14 (16%) patients had del 5q. Thirty two (37%) pts stopped Thal before week 12, 19 (32%) and 13 (46%) patients in the 200mg/d and the 50mg/d groups resp. Reasons for interruption were: important toxicity in 23 (72%) (mainly dizziness, fatigue, sleepiness), patient’s decision in 6 (19%), progression to AML in 3 (9%) and death in 1 (3%). 3 pts developed deep venous thrombosis in the 200mg/d group (none in the 50mg/d group). 55 patients completed 12 weeks of treatment, 22 of them (40%, ie 25% of pts included) had an erythroid response, including 12 major responses (HI-EM) and 10 minor responses (HI-Em). We observed 17 (43.5% of pts who completed 12 weeks, 28% of all pts) and 5 (29.4% of pts who completed 12 weeks, 18% of all pts) erythroid responses in the 200mg/d and the 50mg/d group respectively, not statistically different (p = 0,27). Erythroid response was seen in 15 (44%) patients with IPSS low, versus 6 (14%) in INT-1 and 1 (11%) in INT-2 resp (p = 0.005), whereas no other prognostic factor of response was found. No neutrophil or platelet response was seen. Conclusion: Thal at 200mg/d improved erythropoiesis in 40% of the pts who reached week 12 (28% of included pts). Side effects leading to treatment interruption < week 12 occurred in 32% of pts, ie a similar efficacy and somewhat better tolerance than in our experience with higher doses. Most erythroid responses occurred in IPSS Low pts. On the other hand, Thal at 50mg/d gave a similar incidence of side effects as 200mg/d, with slightly lower but not statistically different erythroid response rate, and thus brought no obvious benefit over 200mg/d. Longer follow up of pts who continued Thal will be presented.
Databáze: OpenAIRE
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