Outcomes in RBC transfusion-dependent patients with Low-/Intermediate-1-risk myelodysplastic syndromes with isolated deletion 5q treated with lenalidomide: a subset analysis from the MDS-004 study | Aplastic Anemia and MDS International Foundation

Outcomes in RBC transfusion-dependent patients with Low-/Intermediate-1-risk myelodysplastic syndromes with isolated deletion 5q treated with lenalidomide: a subset analysis from the MDS-004 study

Journal Title: 
Eur J Haematol
Author(s): 
Giagounidis A, Mufti GJ, Mittelman M, Sanz G, Platzbecker U, Muus P, Selleslag D, Beyne-Rauzy O, Te Boekhorst P, Del Cañizo C, Guerci-Bresler A, Nilsson L, Lübbert M, Quesnel B, Ganser A, Bowen D, Schlegelberger B, Göhring G, Fu T, Benettaib B
Primary Author: 
Giagounidis A
Original Publication Date: 
Saturday, May 10, 2014

OBJECTIVE:

A subset analysis of the randomized, 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/day (days 1-21; n = 47) or 5 mg/day (days 1-28; n = 43) on 28-day cycles, or placebo (n = 45). From the placebo and lenalidomide 5 mg groups, 84% and 58% of patients, respectively, crossed over to lenalidomide 5 mg or 10 mg at 16 weeks, respectively.

RESULTS:

Rates of red blood cell-transfusion independence (RBC-TI) ≥182 days were higher in the lenalidomide 10 mg (57.4%; P < 0.0001) and 5 mg (37.2%; P = 0.0001) groups versus 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 days versus 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). The study is registered at www.ClinicalTrials.gov, identifier NCT00179621.

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