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

Research output: Contribution to journalArticle


ObjectiveA 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). MethodsPatients received lenalidomide 10mg/d (days 1-21; n=47) or 5mg/d (days 1-28; n=43) on 28-d cycles or placebo (n=45). From the placebo and lenalidomide 5mg groups, 84% and 58% of patients, respectively, crossed over to lenalidomide 5 or 10mg at 16wk, respectively. ResultsRates of red blood cell-transfusion independence (RBC-TI) 182d were higher in the lenalidomide 10mg (57.4%; P<0.0001) and 5mg (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 10mg, 5mg, and placebo groups, respectively. In a 6-month landmark analysis, overall survival was longer in lenalidomide-treated patients with RBC-TI 182d vs. non-responders (P=0.0072). The most common grade 3-4 adverse event was myelosuppression. ConclusionsThese 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).


  • Aristoteles Giagounidis
  • Ghulam J. Mufti
  • Moshe Mittelman
  • Guillermo Sanz
  • Uwe Platzbecker
  • Petra Muus
  • Dominik Selleslag
  • Odile Beyne-Rauzy
  • Peter te Boekhorst
  • Consuelo del Canizo
  • Agnes Guerci-Bresler
  • Lars Nilsson
  • Michael Luebbert
  • Bruno Quesnel
  • Arnold Ganser
  • David Bowen
  • Brigitte Schlegelberger
  • Gudrun Goehring
  • Tommy Fu
  • Bouchra Benettaib
  • And 2 others
  • Eva Hellstrom-Lindberg
  • Pierre Fenaux
Research areas and keywords

Subject classification (UKÄ) – MANDATORY

  • Hematology


  • acute myeloid leukaemia, del(5q), lenalidomide, myelodysplastic, syndromes, transfusion independence
Original languageEnglish
Pages (from-to)429-438
JournalEuropean Journal of Haematology
Issue number5
Publication statusPublished - 2014
Publication categoryResearch