A randomized phase 3 study of lenalidomide versus placebo in RBC transfusion-dependent patients with Low-/Intermediate-1-risk myelodysplastic syndromes with del5q

Research output: Contribution to journalArticle


This phase 3, randomized, double-blind study assessed the efficacy and safety of lenalidomide in 205 red blood cell (RBC) transfusion-dependent patients with International Prognostic Scoring System Low-/Intermediate-1-risk del5q31 myelodysplastic syndromes. Patients received lenalidomide 10 mg/day on days 1-21 (n = 69) or 5 mg/day on days 1-28 (n = 69) of 28-day cycles; or placebo (n = 67). Crossover to lenalidomide or higher dose was allowed after 16 weeks. More patients in the lenalidomide 10- and 5-mg groups achieved RBC-transfusion independence (TI) for >= 26 weeks (primary endpoint) versus placebo (56.1% and 42.6% vs 5.9%; both P < .001). Median duration of RBC-TI was not reached (median follow-up, 1.55 years), with 60% to 67% of responses ongoing in patients without progression to acute myeloid leukemia (AML). Cytogenetic response rates were 50.0% (10 mg) versus 25.0% (5 mg; P = .066). For the lenalidomide groups combined, 3-year overall survival and AML risk were 56.5% and 25.1%, respectively. RBC-TI for >= 8 weeks was associated with 47% and 42% reductions in the relative risks of death and AML progression or death, respectively (P = .021 and .048). The safety profile was consistent with previous reports. Lenalidomide is beneficial and has an acceptable safety profile in transfusion-dependent patients with Low-/Intermediate-1-risk del5q myelodysplastic syndrome. This trial was registered at www.clinicaltrials.gov as #NCT00179621. (Blood. 2011; 118(14):3765-3776)


  • Pierre Fenaux
  • Aristoteles Giagounidis
  • Dominik Selleslag
  • Odile Beyne-Rauzy
  • Ghulam Mufti
  • Moshe Mittelman
  • Petra Muus
  • Peter Te Boekhorst
  • Guillermo Sanz
  • Consuelo del Canizo
  • Agnes Guerci-Bresler
  • Lars Nilsson
  • Uwe Platzbecker
  • Michael Luebbert
  • Bruno Quesnel
  • Mario Cazzola
  • Arnold Ganser
  • David Bowen
  • Brigitte Schlegelberger
  • Carlo Aul
  • And 4 others
  • Robert Knight
  • John Francis
  • Tommy Fu
  • Eva Hellstrom-Lindberg
Research areas and keywords

Subject classification (UKÄ) – MANDATORY

  • Hematology
Original languageEnglish
Pages (from-to)3765-3776
Issue number14
Publication statusPublished - 2011
Publication categoryResearch

Bibliographic note

The information about affiliations in this record was updated in December 2015. The record was previously connected to the following departments: Hematopoietic Stem Cell Laboratory (013022012)