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

Chris Fraser, Patrick Brown, Gail Megason, Hyo Seop Ahn, Bin Cho, Ivan Kirov, Lawrence Frankel, Richard Aplenc, Debra Bensen-Kennedy, Mihaela Munteanu, Jennifer Weaver, Paul Harker-Murray

Research output: Contribution to journalArticlepeer-review

11 Scopus citations


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/m2. In phase II, 32 patients received bendamustine 120 mg/m2. Two patients with ALL (bendamustine 90 mg/m2) experienced complete response (CR). Among patients who received bendamustine 120 mg/m2, 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.

Original languageEnglish (US)
Pages (from-to)e212-e218
JournalJournal of Pediatric Hematology/Oncology
Issue number4
StatePublished - May 2014


  • acute lymphoblastic leukemia
  • acute myeloid leukemia
  • bendamustine
  • dose-ranging study
  • overall response rate

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Hematology
  • Oncology


Dive into the research topics of 'Open-label bendamustine monotherapy for pediatric patients with relapsed or refractory acute leukemia: Efficacy and tolerability'. Together they form a unique fingerprint.

Cite this