Efficacy and safety of lenalidomide in intermediate-2 or high-risk myelodysplastic syndromes with 5q deletion: results of a phase 2 study.
Higher-risk MDS with del5q carry a poor prognosis. In this phase 2 trial, 47 patients with higher-risk MDS received lenalidomide 10 mg/day. International Prognostic Scoring System was high in 60%, intermediate-2 risk in 40%. del 5q was isolated, with one additional and more than one additional abnormality in 19%, 23%, and 58% patients, respectively. Thirteen (27%) patients achieved hematologic response, including 7 hematologic complete remission (CR) (with complete [4] or partial [3] cytogenetic response), 2 marrow CR and 4 hematologic improvement erythroid, and 12 became red blood cell (RBC) transfusion independent, for a median duration of 6.5 months. Median CR duration was 11.5 months. Six of 9 (67%) patients with isolated del 5q achieved CR, versus 1 of 11 and none of 27 patients with one or more than one additional abnormality, respectively (P < .001). Seven of 20 (35%) with initial platelets more than 100,000/mm(3) obtained CR, compared with none of the 27 with lower platelet counts less than 100,000/mm(3) (P = .001). Our data support a potential role of lenalidomide in higher-risk MDS with isolated del 5q.
Adès
Lionel
L
Hôpital Avicenne, Université Paris, Paris, France.
Boehrer
Simone
S
Prebet
Thomas
T
Beyne-Rauzy
Odile
O
Legros
Laurence
L
Ravoet
Christophe
C
Dreyfus
François
F
Stamatoullas
Aspasia
A
Chaury
Marie Pierre
MP
Delaunay
Jacques
J
Laurent
Guy
G
Vey
Norbert
N
Burcheri
Sara
S
Mbida
Rose-Marie
RM
Hoarau
Natacha
N
Gardin
Claude
C
Fenaux
Pierre
P
D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D002872:Chromosome Deletion; D002877:Chromosomes, Human / Q000235:genetics*; D005260:Female; D006801:Humans; D000077269:Lenalidomide; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000188:drug therapy* / Q000235:genetics* / Q000473:pathology; D011379:Prognosis; D012307:Risk Factors; D015996:Survival Rate; D013792:Thalidomide / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000494:pharmacology / Q000627:therapeutic use; D016896:Treatment Outcome
D013792:Thalidomide; D000077269:Lenalidomide