These two authors equally contributed to this work.
A non-randomised dose-escalating phase II study of thalidomide for the treatment of patients with low-risk myelodysplastic syndromes: the Thal-SMD-2000 trial of the Groupe Français des Myélodysplasies
Article first published online: 8 NOV 2005
British Journal of Haematology
Volume 131, Issue 5, pages 609–618, December 2005
How to Cite
Bouscary, D., Legros, L., Tulliez, M., Dubois, S., Mahe, B., Beyne-Rauzy, O., Quarre, M. C., Vassilief, D., Varet, B., Aouba, A., Gardembas, M., Giraudier, S., Guerci, A., Rousselot, P., Gaillard, F., Moreau, A., Rousselet, M. C., Ifrah, N., Fenaux, P., Dreyfus, F. and For The Groupe Français des Myélodysplasies (GFM) (2005), A non-randomised dose-escalating phase II study of thalidomide for the treatment of patients with low-risk myelodysplastic syndromes: the Thal-SMD-2000 trial of the Groupe Français des Myélodysplasies. British Journal of Haematology, 131: 609–618. doi: 10.1111/j.1365-2141.2005.05817.x
- Issue published online: 8 NOV 2005
- Article first published online: 8 NOV 2005
- Received 21 July 2005; accepted for publication 22 August 2005
- myelodysplastic syndromes;
- vascular endothelial growth factor;
Patients (n = 47) with low-risk myelodysplastic syndrome were treated with thalidomide [200 mg/d, increased by 200 mg/d/4 weeks up to week 16]. Responses were evaluated according to the International Working Group criteria at week 16 for 39 patients who received at least 8 weeks of treatment. Twenty-three (59%) patients showed haematological improvement (HI): four major erythroid response (HI-EM), 15 minor erythroid response, six major neutrophil response, two major platelet response. Side effects caused 22/39 to stop thalidomide before week 16. Nine of 23 responders continued thalidomide after week 16 [19% of trial patients] with sustained response in eight of nine. Six reached week 56, including the four HI-EM patients [13% of trial patients]. Nineteen of 36 red blood cell transfusion-dependent patients (53%) showed erythroid response, but only four became transfusion-independent. Among the 23 responders, the median duration of response was 260 d (range 30–650). Responses were sustained in all patients except one, and were observed between week 4 and week 8 in 85% of patients, at doses ranging from 200 to 400 mg. Only two patients responded at 600 mg/d and none at 800 mg/d. No clinical characteristics of responding versus non-responding patients were identified. The erythroid response rate was identical in all cytogenetic subgroups, including 5q31.1 deletions. Pretreatment vascular endothelial growth factor levels were lower in responders compared with non-responders (P = 0·004). Microvessel density (MVD) increased and apoptosis decreased in four of six and in all six responders studied respectively whereas MVD and apoptosis were unchanged in three non-responders.