Journal article

Targeting BCL2 with venetoclax in relapsed chronic lymphocytic leukemia

AW Roberts, MS Davids, JM Pagel, BS Kahl, SD Puvvada, JF Gerecitano, TJ Kipps, MA Anderson, JR Brown, L Gressick, S Wong, M Dunbar, M Zhu, MB Desai, E Cerri, SH Enschede, RA Humerickhouse, WG Wierda, JF Seymour

New England Journal of Medicine | MASSACHUSETTS MEDICAL SOC | Published : 2016

Abstract

BACKGROUND: New treatments have improved outcomes for patients with relapsed chronic lymphocytic leukemia (CLL), but complete remissions remain uncommon. Venetoclax has a distinct mechanism of action; it targets BCL2, a protein central to the survival of CLL cells. METHODS: We conducted a phase 1 dose-escalation study of daily oral venetoclax in patients with relapsed or refractory CLL or small lymphocytic lymphoma (SLL) to assess safety, pharmacokinetic profile, and efficacy. In the dose-escalation phase, 56 patients received active treatment in one of eight dose groups that ranged from 150 to 1200 mg per day. In an expansion cohort, 60 additional patients were treated with a weekly stepwis..

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