1. ¹Service d'hématologie clinique, Assistance Publique-Hôpitaux de Paris (AP-HP), Hôpital Avicenne—Paris 13 University, Bobigny, France
2. ²Groupe Francophone des Myelodysplasies (GFM), Bobigny, France
3. ³Service d'Hématologie Clinique, CHU de Nantes, Nantes, France
4. ⁴Service d'Hématologie, Hôpital Cochin, AP-HP, Paris, France
5. ⁵Service d'Hématologie, CHU de Dijon, Dijon, France
6. ⁶Service d'Hématologie, Hôpital d'Avignon, Avignon, France
7. ⁷Département d'Hématologie, Institut Paoli-Calmette, Marseille, France
8. ⁸Service d'Hématologie adulte, Hôpital Necker–Enfants-Malades, Paris, France
9. ⁹Service d'Hématologie, Hôpital Purpan, Toulouse, France
10. ¹⁰Service d'Hématologie, CHU de Nancy, Nancy, France
11. ¹¹Service d'Hématologie Clinique, Hôpital Clemenceau, Caen, France
12. ¹²Service d'Hématologie, Hôpital Edouard Herriot, Lyon, France
13. ¹³Service d'Hématologie, CHU de Rouen, Rouen, France
14. ¹⁴Service d'hématologie, CHU d'Angers, Angers, France
15. ¹⁵Service d'Hématologie, Centre Hospitalier de la Côte Basque, Bayonne, France
16. ¹⁶Service d'Hématologie, CHU de Reims, Reims, France
17. ¹⁷Service d'Hématologie, CHU de Limoges, Limoges, France
18. ¹⁸Fédération de Médecine Interne Hématologie, Hôpital Archet, Nice, France
19. ¹⁹Service d'hématologie, CHU d'Amiens, Amiens, France
20. ²⁰Inserm Unit 848, Institut Gustave Roussy, Villejuif, France

Correspondence: Dr L Adès, Hematology Department, Assistance Publique-Hôpitaux de Paris (AP-HP), Hôpital Avicenne, Paris 13 University, 125 rue de Stalingrad, Hopital Avicenne, Bobigny 93009, France. E-mail: lionel.ades@avc.aphp.fr

Received 5 September 2008; Revised 23 October 2008; Accepted 19 November 2008; Published online 8 January 2009.

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- 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-–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.