Lenalidomide in International Prognostic Scoring System Low and Intermediate-1 risk myelodysplastic syndromes with del(5q): an Italian phase II trial of health-related quality of life, safety and efficacy

• Published in: Leukemia & lymphoma 2013-11, Vol.54 (11), p.2458-2465
• Main Authors: Oliva, Esther N., Latagliata, Roberto, Laganà, Carmelo, Breccia, Massimo, Galimberti, Sara, Morabito, Fortunato, Poloni, Antonella, Balleari, Enrico, Cortelezzi, Agostino, Palumbo, Giuseppe, Sanpaolo, Grazia, Volpe, Antonio, Specchia, Giorgina, Finelli, Carlo, D'Errigo, Maria Grazia, Rodà, Filippo, Alati, Caterina, Alimena, Giuliana, Nobile, Francesco, Aloe Spiriti, Maria A.
• Format: Article
• Language: English
• Subjects: Aged; Aged, 80 and over; Angiogenesis Inhibitors - adverse effects; Angiogenesis Inhibitors - therapeutic use; Chromosome Deletion; Chromosomes, Human, Pair 5; del(5q); Disease Progression; Female; health-related quality of life; Humans; Lenalidomide; Male; Middle Aged; myelodysplastic syndromes; Myelodysplastic Syndromes - drug therapy; Myelodysplastic Syndromes - genetics; Myelodysplastic Syndromes - mortality; Quality of Life; safety; Thalidomide - adverse effects; Thalidomide - analogs & derivatives; Thalidomide - therapeutic use; Treatment Outcome
• ISSN: 1042-8194; 1029-2403
• DOI: 10.3109/10428194.2013.778406

Abstract In lower-risk myelodysplastic syndromes (MDS) with del(5q), lenalidomide induces erythroid responses associated with better survival. In a phase II, single-arm trial, 45 patients with anemia and lower-risk del(5q) MDS received lenalidomide 10 mg/day to evaluate quality of life (QoL) changes, measured by QOL-E, safety, responses and survival. Lenalidomide was well tolerated, with 80% completing ≥ 24 weeks of treatment. Earlier study discontinuation was related to disease progression (n = 5), death (n = 1) and withdrawal of consent (n = 3). Within 24 weeks, 82% obtained erythroid responses, durable in 69% at 52 weeks. Cytogenetic responses occurred in 29 patients (64%), with 10 patients achieving a complete cytogenetic response. QoL-E scores correlated with hemoglobin levels and improved in erythroid responders. Erythroid responders had an 86% reduced risk of disease progression and an 80% reduction in mortality risk compared with non-responders. These findings corroborate earlier studies and give further support to the use of lenalidomide in lower-risk MDS and del(5q).