Cyclophosphamide for the treatment of progressive low-grade astrocytoma: a Pediatric Oncology Group phase II Study

R P Kadota1, L E Kun, J W Langston

  • 1Pediatric Oncology Group, Chicago, Illinois, USA.

Insights

Cyclophosphamide (CPM) showed insufficient activity against pediatric low-grade astrocytoma. The phase II trial was closed early due to excessive progressive disease in children treated with CPM.

Area of Science:

  • Pediatric Oncology
  • Neuro-Oncology

Background:

  • Low-grade astrocytomas are the most common brain tumors in children.
  • Treatment options for progressive pediatric low-grade astrocytomas are limited.

Purpose of the Study:

  • To evaluate the efficacy and safety of cyclophosphamide (CPM) in children with progressive low-grade astrocytoma.
  • To report the results of a phase II clinical trial.

Main Methods:

  • Fifteen pediatric patients with a median age of 39 months were enrolled.
  • Four courses of intravenous CPM (1.2 g/m2) were administered every 3 weeks.
  • Tumor locations included optic pathway, hypothalamus, and thalamus in 11 patients.

Main Results:

  • Toxicity was primarily hematologic.
  • One patient achieved a complete response, 8 had stable disease, and 5 had progressive disease (PD).
  • The high rate of PD led to early study closure.

Conclusions:

  • Cyclophosphamide (CPM) demonstrated insufficient anti-tumor activity for pediatric low-grade astrocytoma in this trial.
  • Further patient accrual was not justified due to lack of efficacy.
Abstract

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