Open-label bendamustine monotherapy for pediatric patients with relapsed or refractory acute leukemia: efficacy and tolerability.

TitleOpen-label bendamustine monotherapy for pediatric patients with relapsed or refractory acute leukemia: efficacy and tolerability.
Publication TypeJournal Article
Year of Publication2014
AuthorsFraser C, Brown P, Megason G, Ahn HSeop, Cho B, Kirov I, Frankel L, Aplenc R, Bensen-Kennedy D, Munteanu M, Weaver J, Harker-Murray P
JournalJ Pediatr Hematol Oncol
Volume36
Issue4
Paginatione212-8
Date Published2014 May
ISSN1536-3678
KeywordsAdolescent, Adult, Antineoplastic Agents, Alkylating, Bendamustine Hydrochloride, Child, Child, Preschool, Female, Humans, Infant, Leukemia, Myeloid, Acute, Male, Nitrogen Mustard Compounds, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Recurrence
Abstract

This open-label, single-arm, phase I/II, dose-escalation study was designed to determine the recommended phase II dose (RP2D), pharmacokinetics, tolerability, and efficacy of bendamustine in pediatric patients (age ranging from 1 to 20 y) with histologically proven relapsed/refractory acute lymphoblastic leukemia (ALL) or acute myeloid leukemia (AML). Patients (27 with ALL, 16 with AML) received intravenous bendamustine on days 1 and 2 of each treatment cycle. Phase I involved planned dose escalation of bendamustine to establish the RP2D for phase II. Objectives included overall response rate, duration of response, and tolerability. Eleven patients were treated in phase I, and the RP2D was 120 mg/m. In phase II, 32 patients received bendamustine 120 mg/m. Two patients with ALL (bendamustine 90 mg/m) experienced complete response (CR). Among patients who received bendamustine 120 mg/m, 2 experienced partial response (PR); 7 had stable disease. The overall response rate (CR+CR without platelet recovery [CRp]) was 4.7% and biological activity rate (CR+CRp+PR) was 9.3%. No AML patients responded. The most common adverse events were anemia, neutropenia, thrombocytopenia, pyrexia, nausea, vomiting, and diarrhea. Bendamustine monotherapy has acceptable tolerability in heavily pretreated children with relapsed/refractory ALL or AML and appears to have some activity in ALL, warranting further studies in combination trials.

DOI10.1097/MPH.0000000000000021
Alternate JournalJ. Pediatr. Hematol. Oncol.
PubMed ID24072240
PubMed Central IDPMC4020582
Grant ListP30 CA016520 / CA / NCI NIH HHS / United States