Premium
Outcomes in RBC transfusion‐dependent patients with L ow‐/ I ntermediate‐1‐risk myelodysplastic syndromes with isolated deletion 5q treated with lenalidomide: a subset analysis from the MDS ‐004 study
Author(s) -
Giagounidis Aristoteles,
Mufti Ghulam J.,
Mittelman Moshe,
Sanz Guillermo,
Platzbecker Uwe,
Muus Petra,
Selleslag Dominik,
BeyneRauzy Odile,
Boekhorst Peter,
Cañizo Consuelo,
GuerciBresler Agnès,
Nilsson Lars,
Lübbert Michael,
Quesnel Bruno,
Ganser Arnold,
Bowen David,
Schlegelberger Brigitte,
Göhring Gudrun,
Fu Tommy,
Benettaib Bouchra,
HellströmLindberg Eva,
Fenaux Pierre
Publication year - 2014
Publication title -
european journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.904
H-Index - 84
eISSN - 1600-0609
pISSN - 0902-4441
DOI - 10.1111/ejh.12380
Subject(s) - lenalidomide , myelodysplastic syndromes , medicine , placebo , gastroenterology , adverse effect , international prognostic scoring system , anemia , multiple myeloma , bone marrow , pathology , alternative medicine
Objective A subset analysis of the randomised, phase 3, MDS ‐004 study to evaluate outcomes in patients with I nternational P rognostic S coring S ystem ( IPSS )‐defined L ow‐/ I ntermediate ( I nt)‐1‐risk myelodysplastic syndromes ( MDS ) with isolated del(5q). Methods Patients received lenalidomide 10 mg/d (days 1–21; n = 47) or 5 mg/d (days 1–28; n = 43) on 28‐d cycles or placebo ( n = 45). From the placebo and lenalidomide 5 mg groups, 84% and 58% of patients, respectively, crossed over to lenalidomide 5 or 10 mg at 16 wk, respectively. Results Rates of red blood cell‐transfusion independence ( RBC ‐ TI ) ≥182 d were higher in the lenalidomide 10 mg (57.4%; P < 0.0001) and 5 mg (37.2%; P = 0.0001) groups vs. placebo (2.2%). Cytogenetic response rates (major + minor responses) were 56.8% ( P < 0.0001), 23.1% ( P = 0.0299) and 0%, respectively. Two‐year cumulative risk of acute myeloid leukaemia progression was 12.6%, 17.4% and 16.7% in the lenalidomide 10 mg, 5 mg, and placebo groups, respectively. In a 6‐month landmark analysis, overall survival was longer in lenalidomide‐treated patients with RBC ‐ TI ≥182 d vs. non‐responders ( P = 0.0072). The most common grade 3–4 adverse event was myelosuppression. Conclusions These data support the clinical benefits and acceptable safety profile of lenalidomide in transfusion‐dependent patients with IPSS ‐defined L ow‐/ I nt‐1‐risk MDS with isolated del(5q).