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

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).

Keywords::

• Lenalidomide
• myelodysplastic syndromes
• del(5q)
• health-related quality of life
• safety

Acknowledgements

The study was supported by Associazione QOL-ONE, Reggio Calabria, Italy. The study was also supported by Celgene Corporation. The authors received editorial and writing support in the preparation of this manuscript from Anna Keller, PhD, of Excerpta Medica, sponsored by Celgene Corporation. The authors are fully responsible for the content and editorial decisions for this manuscript.

Potential conflict of interest:

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