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.

Abstract

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).

• Authors: Aristoteles Giagounidis, Ghulam J Mufti, Moshe Mittelman, Guillermo Sanz, Uwe Platzbecker, Petra Muus, Dominik Selleslag, Odile Beyne-Rauzy, Peter te Boekhorst, Consuelo del Cañizo, Agnès Guerci-Bresler, Lars Nilsson, Michael Lübbert, Bruno Quesnel, Arnold Ganser, David Bowen, Brigitte Schlegelberger, Gudrun Göhring, Tommy Fu, Bouchra Benettaib, Eva Hellström-Lindberg, Pierre Fenaux
• Journal: European journal of haematology (Eur J Haematol) Vol. 93 Issue 5 Pg. 429-38 (Nov 2014) ISSN: 1600-0609 [Electronic] England
• PMID: 24813620 (Publication Type: Clinical Trial, Phase III, Journal Article, Randomized Controlled Trial, Research Support, Non-U.S. Gov't)
• Copyright: © 2014 The Authors. European Journal of Haematology Published by John Wiley & Sons Ltd.

Chemical References

• Antineoplastic Agents
• Thalidomide
• Lenalidomide

Topics

• Adult
• Aged
• Aged, 80 and over
• Antineoplastic Agents (therapeutic use)
• Chromosome Deletion
• Chromosomes, Human, Pair 5
• Cytogenetic Analysis
• Disease Progression
• Drug Administration Schedule
• Erythrocyte Transfusion
• Female
• Humans
• Lenalidomide
• Leukemia, Myeloid, Acute (genetics, mortality, pathology, therapy)
• Male
• Middle Aged
• Myelodysplastic Syndromes (genetics, mortality, pathology, therapy)
• Retrospective Studies
• Risk
• Survival Analysis
• Thalidomide (analogs & derivatives, therapeutic use)
• Treatment Outcome