Journal
LEUKEMIA
Volume 32, Issue 7, Pages 1493-1499Publisher
NATURE PUBLISHING GROUP
DOI: 10.1038/s41375-018-0029-9
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Funding
- NATIONAL CANCER INSTITUTE [P01CA066996] Funding Source: NIH RePORTER
- NCI NIH HHS [P01 CA066996] Funding Source: Medline
- NHLBI NIH HHS [R01 HL082945] Funding Source: Medline
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y There are few instances in oncology where reciprocal clinical and laboratory translation studies have accelerated the understanding of disease biology and treatment more so than the decade following the Food and Drug Administration (FDA) approval of lenalidomide (Revlimid (TM); Celgene Corporation, Summit, NJ, USA) for the treatment of patients with myelodysplastic syndrome (MDS) and chromosome 5q deletion (del(5q)). Lenalidomide was approved by the FDA in December 2005 on the merits of a multicenter phase 2 study, which demonstrated sustained and prolonged transfusion independence in the majority of participants. Since then, del(5q) MDS has emerged as one of the best characterized bone marrow malignancies and, in particular, has raised our understanding as to how allelic haplodeficiency underlies both its hematological phenotype and the selective sensitivity to lenalidomide by virtue of synthetic lethality. Herein, we review the clinical and biological discoveries that have advanced our understanding of del(5q) MDS and its treatment since its approval by United States and European regulatory agencies.
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