A randomized phase 3 study of lenalidomide versus placebo in RBC transfusion-dependent patients with Low-/Intermediate-1-risk myelodysplastic syndromes with del5q
| Autor: | Lennart Nilsson, Agnes Guerci-Bresler, Tommy Fu, Carlo Aul, Guillermo Sanz, Dominik Selleslag, Bruno Quesnel, Michael Lübbert, Peter A. W. te Boekhorst, Mario Cazzola, Consuelo del Cañizo, John Francis, Moshe Mittelman, Eva Hellström-Lindberg, Aristoteles Giagounidis, Arnold Ganser, David G. Bowen, Brigitte Schlegelberger, Odile Beyne-Rauzy, Robert Knight, Uwe Platzbecker, Petra Muus, Ghulam J. Mufti, Pierre Fenaux |
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| Přispěvatelé: | Hematology |
| Rok vydání: | 2011 |
| Předmět: |
Adult
Male medicine.medical_specialty Anemia Immunology Phases of clinical research Antineoplastic Agents Placebo Biochemistry Gastroenterology Double-Blind Method Translational research [ONCOL 3] Internal medicine medicine Clinical endpoint Humans Lenalidomide Survival analysis Aged Aged 80 and over business.industry Myelodysplastic syndromes Cell Biology Hematology Middle Aged medicine.disease Prognosis Survival Analysis Surgery Thalidomide International Prognostic Scoring System Myelodysplastic Syndromes Cytogenetic Analysis Chromosomes Human Pair 5 Female Chromosome Deletion business Erythrocyte Transfusion medicine.drug |
| Zdroj: | Blood r-IIS La Fe. Repositorio Institucional de Producción Científica del Instituto de Investigación Sanitaria La Fe instname Blood, 118(14), 3765-3776. American Society of Hematology Blood, 118, 14, pp. 3765-76 Blood, 118, 3765-76 |
| ISSN: | 0006-4971 |
| Popis: | 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. |
| Databáze: | OpenAIRE |
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