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PLoS medicine DOI:10.1371/journal.pmed.0050035

An erythroid differentiation signature predicts response to lenalidomide in myelodysplastic syndrome.

Publication TypeJournal Article
Year of Publication2008
AuthorsEbert, BL, Galili, N, Tamayo, P, Bosco, J, Mak, R, Pretz, J, Tanguturi, S, Ladd-Acosta, C, Stone, R, Golub, TR, Raza, A
JournalPLoS medicine
Date Published2008/02/01

Lenalidomide is an effective new agent for the treatment of patients with myelodysplastic syndrome (MDS), an acquired hematopoietic disorder characterized by ineffective blood cell production and a predisposition to the development of leukemia. Patients with an interstitial deletion of Chromosome 5q have a high rate of response to lenalidomide, but most MDS patients lack this deletion. Approximately 25% of patients without 5q deletions also benefit from lenalidomide therapy, but response in these patients cannot be predicted by any currently available diagnostic assays. The aim of this study was to develop a method to predict lenalidomide response in order to avoid unnecessary toxicity in patients unlikely to benefit from treatment.