TY - JOUR
T1 - Outcomes in RBC transfusion-dependent patients with Low-/Intermediate-1-risk myelodysplastic syndromes with isolated deletion 5q treated with lenalidomide
T2 - A subset analysis from the MDS-004 study
AU - Giagounidis, Aristoteles
AU - Mufti, Ghulam J.
AU - Mittelman, Moshe
AU - Sanz, Guillermo
AU - Platzbecker, Uwe
AU - Muus, Petra
AU - Selleslag, Dominik
AU - Beyne-Rauzy, Odile
AU - te Boekhorst, Peter
AU - del Cañizo, Consuelo
AU - Guerci-Bresler, Agnès
AU - Nilsson, Lars
AU - Lübbert, Michael
AU - Quesnel, Bruno
AU - Ganser, Arnold
AU - Bowen, David
AU - Schlegelberger, Brigitte
AU - Göhring, Gudrun
AU - Fu, Tommy
AU - Benettaib, Bouchra
AU - Hellström-Lindberg, Eva
AU - Fenaux, Pierre
N1 - Publisher Copyright:
© 2014 The Authors.
PY - 2014/11/1
Y1 - 2014/11/1
N2 - Objective: A subset analysis of the randomised, phase 3, MDS-004 study to evaluate outcomes in patients with International Prognostic Scoring System (IPSS)-defined Low-/Intermediate (Int)-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 Low-/Int-1-risk MDS with isolated del(5q).
AB - Objective: A subset analysis of the randomised, phase 3, MDS-004 study to evaluate outcomes in patients with International Prognostic Scoring System (IPSS)-defined Low-/Intermediate (Int)-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 Low-/Int-1-risk MDS with isolated del(5q).
KW - Acute myeloid leukaemia
KW - Del(5q)
KW - Lenalidomide
KW - Myelodysplastic syndromes
KW - Transfusion independence
UR - http://www.scopus.com/inward/record.url?scp=84911955301&partnerID=8YFLogxK
U2 - 10.1111/ejh.12380
DO - 10.1111/ejh.12380
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C2 - 24813620
AN - SCOPUS:84911955301
SN - 0902-4441
VL - 93
SP - 429
EP - 438
JO - European Journal of Haematology
JF - European Journal of Haematology
IS - 5
ER -