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 Aul & 4 others Robert 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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Erythrocyte Transfusion
Myelodysplastic Syndromes
Blood
Placebos
Acute Myeloid Leukemia
Safety
Double-Blind Method
Cytogenetics
lenalidomide
Cells
Survival

ASJC Scopus subject areas

  • Hematology
  • Biochemistry
  • Cell Biology
  • Immunology

Cite this

Fenaux, P., Giagounidis, A., Selleslag, D., Beyne-Rauzy, O., Mufti, G., Mittelman, M., ... 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

A randomized phase 3 study of lenalidomide versus placebo in RBC transfusion-dependent patients with Low-/Intermediate-1-risk myelodysplastic syndromes with del5q. / Fenaux, Pierre; Giagounidis, Aristoteles; Selleslag, Dominik; Beyne-Rauzy, Odile; Mufti, Ghulam; Mittelman, Moshe; Muus, Petra; Te Boekhorst, Peter; Sanz, Guillermo; Del Cañizo, Consuelo; Guerci-Bresler, Agnes; Nilsson, Lars; Platzbecker, Uwe; Lübbert, Michael; Quesnel, Bruno; Cazzola, Mario; Ganser, Arnold; Bowen, David; Schlegelberger, Brigitte; Aul, Carlo; Knight, Robert; Francis, John; Fu, Tommy; Hellström-Lindberg, Eva.

In: Blood, Vol. 118, No. 14, 06.10.2011, p. 3765-3776.

Research output: Contribution to journalArticle

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, Aul, C, Knight, R, Francis, J, Fu, T & 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, vol. 118, no. 14, pp. 3765-3776. https://doi.org/10.1182/blood-2011-01-330126
Fenaux, Pierre ; Giagounidis, Aristoteles ; Selleslag, Dominik ; Beyne-Rauzy, Odile ; Mufti, Ghulam ; Mittelman, Moshe ; Muus, Petra ; Te Boekhorst, Peter ; Sanz, Guillermo ; Del Cañizo, Consuelo ; Guerci-Bresler, Agnes ; Nilsson, Lars ; Platzbecker, Uwe ; Lübbert, Michael ; Quesnel, Bruno ; Cazzola, Mario ; Ganser, Arnold ; Bowen, David ; Schlegelberger, Brigitte ; Aul, Carlo ; Knight, Robert ; Francis, John ; Fu, Tommy ; Hellström-Lindberg, Eva. / A randomized phase 3 study of lenalidomide versus placebo in RBC transfusion-dependent patients with Low-/Intermediate-1-risk myelodysplastic syndromes with del5q. In: Blood. 2011 ; Vol. 118, No. 14. pp. 3765-3776.
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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.",
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AU - Fenaux, Pierre

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AU - Selleslag, Dominik

AU - Beyne-Rauzy, Odile

AU - Mufti, Ghulam

AU - Mittelman, Moshe

AU - Muus, Petra

AU - Te Boekhorst, Peter

AU - Sanz, Guillermo

AU - Del Cañizo, Consuelo

AU - Guerci-Bresler, Agnes

AU - Nilsson, Lars

AU - Platzbecker, Uwe

AU - Lübbert, Michael

AU - Quesnel, Bruno

AU - Cazzola, Mario

AU - Ganser, Arnold

AU - Bowen, David

AU - Schlegelberger, Brigitte

AU - Aul, Carlo

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AU - Francis, John

AU - Fu, Tommy

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N2 - 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.

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