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

Pierre Fenaux, Aristoteles Giagounidis, Dominik Selleslag, Odile Beyne-Rauzy, Ghulam Mufti, Moshe Mittelman, Petra Muus, Peter Te Boekhorst, Guillermo Sanz, Consuelo Del Cañizo, Agnes Guerci-Bresler, Lars Nilsson, Uwe Platzbecker, Michael Lübbert, Bruno Quesnel, Mario Cazzola, Arnold Ganser, David Bowen, Brigitte Schlegelberger, Carlo AulRobert Knight, John Francis, Tommy Fu, Eva Hellström-Lindberg

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Abstract

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.

Original languageEnglish
Pages (from-to)3765-3776
Number of pages12
JournalBlood
Volume118
Issue number14
DOIs
Publication statusPublished - Oct 6 2011

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ASJC Scopus subject areas

  • Hematology
  • Biochemistry
  • Cell Biology
  • Immunology

Cite this

Fenaux, P., Giagounidis, A., Selleslag, D., Beyne-Rauzy, O., Mufti, G., Mittelman, M., Muus, P., Te Boekhorst, P., Sanz, G., Del Cañizo, C., Guerci-Bresler, A., Nilsson, L., Platzbecker, U., Lübbert, M., Quesnel, B., Cazzola, M., Ganser, A., Bowen, D., Schlegelberger, B., ... Hellström-Lindberg, E. (2011). A randomized phase 3 study of lenalidomide versus placebo in RBC transfusion-dependent patients with Low-/Intermediate-1-risk myelodysplastic syndromes with del5q. Blood, 118(14), 3765-3776. https://doi.org/10.1182/blood-2011-01-330126