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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; Boekhorst, Peter Te; Sanz, Guillermo and del Canizo, Consuelo, et al. (2011) In Blood 118(14). p.3765-3776
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%... (More)
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) (Less)
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published
subject
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Blood
volume
118
issue
14
pages
3765 - 3776
publisher
American Society of Hematology
external identifiers
  • wos:000295807700007
  • scopus:80053621748
ISSN
1528-0020
DOI
10.1182/blood-2011-01-330126
language
English
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yes
id
3348df58-c58c-4b03-b170-fd87119b1d69 (old id 2212942)
date added to LUP
2011-12-01 08:36:00
date last changed
2017-11-05 03:09:11
@article{3348df58-c58c-4b03-b170-fd87119b1d69,
  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 &gt;= 26 weeks (primary endpoint) versus placebo (56.1% and 42.6% vs 5.9%; both P &lt; .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 &gt;= 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)},
  author       = {Fenaux, Pierre and Giagounidis, Aristoteles and Selleslag, Dominik and Beyne-Rauzy, Odile and Mufti, Ghulam and Mittelman, Moshe and Muus, Petra and Boekhorst, Peter Te and Sanz, Guillermo and del Canizo, Consuelo and Guerci-Bresler, Agnes and Nilsson, Lars and Platzbecker, Uwe and Luebbert, Michael and Quesnel, Bruno and Cazzola, Mario and Ganser, Arnold and Bowen, David and Schlegelberger, Brigitte and Aul, Carlo and Knight, Robert and Francis, John and Fu, Tommy and Hellstrom-Lindberg, Eva},
  issn         = {1528-0020},
  language     = {eng},
  number       = {14},
  pages        = {3765--3776},
  publisher    = {American Society of Hematology},
  series       = {Blood},
  title        = {A randomized phase 3 study of lenalidomide versus placebo in RBC transfusion-dependent patients with Low-/Intermediate-1-risk myelodysplastic syndromes with del5q},
  url          = {http://dx.doi.org/10.1182/blood-2011-01-330126},
  volume       = {118},
  year         = {2011},
}