Is there a role for all-trans retinoic acid in combination with recombinant erythropoetin in myelodysplastic syndromes? A report on 59 cases.
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.
Itzykson
R
R
Service d'Hématologie Clinique, Assistance Publique-Hôpitaux de Paris (AP-HP), Hôpital Avicenne-Paris 13 University, Bobigny, France.
Ayari
S
S
Vassilief
D
D
Berger
E
E
Slama
B
B
Vey
N
N
Suarez
F
F
Beyne-Rauzy
O
O
Guerci
A
A
Cheze
S
S
Thomas
X
X
Stamatoullas
A
A
Gardembas
M
M
Bauduer
F
F
Kolb
A
A
Chaury
M C
MC
Legros
L
L
Damaj
G
G
Chermat
F
F
Dreyfus
F
F
Fenaux
P
P
Ades
L
L
D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D004359:Drug Therapy, Combination; D004921:Erythropoietin / Q000627:therapeutic use*; D005260:Female; D006801:Humans; D007958:Leukocyte Count; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000188:drug therapy*; D009503:Neutropenia / Q000188:drug therapy; D009504:Neutrophils; D010976:Platelet Count; D011994:Recombinant Proteins; D013921:Thrombocytopenia / Q000188:drug therapy; D016896:Treatment Outcome; D014212:Tretinoin / Q000627:therapeutic use*; D055815:Young Adult
D011994:Recombinant Proteins; D004921:Erythropoietin; D014212:Tretinoin