Nilotinib in patients with systemic mastocytosis: analysis of the phase 2, open-label, single-arm nilotinib registration study.

ABSTRACT:

Purpose

Activating KIT mutations are part of the pathogenesis of systemic mastocytosis (SM). Nilotinib is a tyrosine kinase inhibitor that potently inhibits activated forms of KIT. This phase 2, open-label, single-arm study (CAMN107A2101; www.clinicaltrials.gov NCT00109707) evaluated nilotinib in patients with SM.

Methods

Patients with SM [aggressive SM (ASM), indolent SM, or other] received nilotinib 400 mg twice daily. C-findings were collected retrospectively to assess response using criteria proposed after trial initiation. Response was evaluated using improvements in laboratory findings (for all patients) and ASM response criteria (for the ASM subgroup).

Results

In 61 patients enrolled, the median nilotinib exposure was 232 days (range 3-1274 days) with a median

SUBMITTER: Hochhaus A 

PROVIDER: S-EPMC4768228 | biostudies-literature | 2015 Nov

REPOSITORIES: biostudies-literature