@article{JF Abgrall_I Guibaud_JN Bastie_M Flesch_JF Rossi_L Lacotte-Thierry_F Boyer_P Casassus_B Slama_C Berthou_P Rodon_M Leporrier_B Villemagne_C Himberlin_K Ghomari_F Larosa_F Rollot_J Dugay_C Allard_M Maigre_F Isnard_R Zerbib_JM Cauvin_Groupe Ouest-Est Leucemies et Maladies du Sang (GOELAMS)_2006, place={Pavia, Italy}, title={Thalidomide versus placebo in myeloid metaplasia with myelofibrosis: a prospective, randomized, double-blind, multicenter study}, volume={91}, url={https://haematologica.org/article/view/4088}, DOI={10.3324/%x}, abstractNote={BACKGROUND AND OBJECTIVES: In non-randomized studies, thalidomide appeared to be effective in myeloid metaplasia with myelofibrosis (MMM). We compared thalidomide to placebo for treatment of anemia in MMM. DESIGN AND METHODS: A prospective phase II B, randomized double-blind multicenter trial comparing thalidomide 400 mg/d with placebo for 180 days was conducted in 52 anemic patients (hemoglobin pounds Sterling 9 g/dL or transfused). The main outcome measure was a 2 g/L increase in hemoglobin or 20% reduction in transfusions. RESULTS: In the thalidomide group only 10 patients completed 6 months of treatment. At 180 days, in an intention-to-treat analysis, no difference was observed between the thalidomide and placebo groups as regards improvement of hemoglobin levels (one patient in each group) or reduction of red blood cell transfusions (three vs five patients, respectively). The spleen size, determined by ultrasonography, increased significantly less in the thalidomide group than in the placebo group (p < 0.05). Thalidomide had no apparent benefit on the Dupriez score, the severity score, survival, death, or any other clinical or biological parameter. Somnolence, gastro-intestinal signs, weight gain, and edema were significantly more frequent in the thalidomide group. Outpatient discontinuation of thalidomide was significantly correlated with a high severity score > 4 (odds ratio, OR = 16; p < 0.01), and g-glutamyl transferase levels > 40 IU/L (OR = 12; p &lt; 0.05). INTERPRETATION AND CONCLUSIONS: Thalidomide (200-400 mg/d) does not demonstrate substantial efficacy in anemic MMM patients. The natural history of disease in the placebo group revealed spontaneous periods of remission of anemia. Tolerance of thalidomide was significantly correlated wih the severity and liver involvement of the disease.}, number={8}, journal={Haematologica}, author={JF Abgrall and I Guibaud and JN Bastie and M Flesch and JF Rossi and L Lacotte-Thierry and F Boyer and P Casassus and B Slama and C Berthou and P Rodon and M Leporrier and B Villemagne and C Himberlin and K Ghomari and F Larosa and F Rollot and J Dugay and C Allard and M Maigre and F Isnard and R Zerbib and JM Cauvin and Groupe Ouest-Est Leucemies et Maladies du Sang (GOELAMS)}, year={2006}, month={Aug.}, pages={1027-1032} }