Phase I trial of pegylated interferon-alpha-2b in young patients with plexiform neurofibromas.

ABSTRACT:

Objective

Interferon has antiproliferative and antiangiogenic properties. We sought to evaluate preliminary efficacy and determine the recommended phase II dose (RP2D) for pegylated interferon-α-2b (PI) in patients with unresectable progressive or symptomatic plexiform neurofibromas (PN).

Methods

PI was administered weekly in cohorts of 3-6 patients during the dose-finding phase and continued for up to 2 years. Twelve patients were treated at the RP2D to further evaluate toxicity and activity.

Results

Thirty patients (median age 9.3 years, range 1.9-34.7 years) were enrolled. No dose-limiting toxicity (DLT) was seen in patients treated at the 3 μg/kg dose level (DL) during the first 4 weeks. All 5 patients treated at the 4.5 μg/kg DL came off study or required dose re

SUBMITTER: Jakacki RI 

PROVIDER: S-EPMC3034394 | biostudies-literature | 2011 Jan

REPOSITORIES: biostudies-literature