Is there a role for all-trans retinoic acid in combination with recombinant erythropoetin in myelodysplastic syndromes? A report on 59 cases.

Abstract	Erythropoiesis-stimulating agents (ESAs) remain the first-line treatment of anemia in lower risk myelodysplastic syndromes (MDS) without 5q deletion. A preliminary report suggested that adding all-trans retinoic acid (ATRA) to ESAs may improve their erythroid response, particularly in patients with high endogenous erythropoietin (EPO) level, and may improve other cytopenias. We conducted a prospective multicenter study of EPO-beta and ATRA in anemic MDS patients with marrow blasts <10% and either previous ESA failure or relapse, endogenous EPO >500 U/l or other cytopenia(s) (absolute neutrophilic count <1.0 G/l or platelets <50 G/l). A total of 59 patients were evaluable after 12 weeks of treatment. The erythroid response rates according to IWG 2000 and 2006 criteria, respectively, were as follows: overall: 49 and 36%; patients with previous ESA failure (n=28): 43 and 32%; patients with endogenous EPO >500 U/l (n=18): 11 and 19%; patients transfused >2 red blood cells units/month (n=28) 43 and 39%. Only one neutrophil, but no platelet response, and no major side effect were observed. EPO-beta-ATRA combination appears a possible therapeutic option in anemia of MDS having failed an ESA alone, but not in patients with high endogenous EPO level, and does not improve neutropenia and thrombocytopenia.
Authors	R Itzykson, S Ayari, D Vassilief, E Berger, B Slama, N Vey, F Suarez, O Beyne-Rauzy, A Guerci, S Cheze, X Thomas, A Stamatoullas, M Gardembas, F Bauduer, A Kolb, M C Chaury, L Legros, G Damaj, F Chermat, F Dreyfus, P Fenaux, L Ades, Groupe Francophone des Myelodysplasies (GFM)
Journal	Leukemia (Leukemia) Vol. 23 Issue 4 Pg. 673-8 (Apr 2009) ISSN: 1476-5551 [Electronic] England
PMID	19151787 (Publication Type: Clinical Trial, Journal Article, Multicenter Study)
Chemical References	Recombinant Proteins Erythropoietin Tretinoin
Topics	Adult Aged Aged, 80 and over Drug Therapy, Combination Erythropoietin (therapeutic use) Female Humans Leukocyte Count Male Middle Aged Myelodysplastic Syndromes (drug therapy) Neutropenia (drug therapy) Neutrophils Platelet Count Recombinant Proteins Thrombocytopenia (drug therapy) Treatment Outcome Tretinoin (therapeutic use) Young Adult

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