Phase I clinical trial of mafosfamide in infants and children aged 3 years or younger with newly diagnosed embryonal tumors: A Pediatric Brain Tumor Consortium Study (PBTC-001)

Susan M. Blaney, James Boyett, Henry Friedman, Amar Gajjar, Russ Geyer, Marc Horowtiz, Daniel Hunt, Mark Kieran, Larry Kun, Roger Packer, Peter Phillips, Ian F. Pollack, Michael Prados, Richard Heideman

Research output: Contribution to journalArticle

34 Scopus citations

Abstract

Purpose: A phase I trial of intrathecal (IT) mafosfamide was performed to determine the optimal dose, dose-limiting toxicities, and incidence and severity of other toxicities when administered in association with concomitant multiagent systemic chemotherapy to children younger than 3 years with newly diagnosed embryonal tumors. Patients and Methods: Twenty-five assessable patients received IT mafosfamide at one of six dose levels ranging from 5 mg to 17 mg. Patients were premedicated with dexamethasone (0.15 mg/kg) and morphine (0.1 mg/kg) before receiving IT mafosfamide. Serial samples of CSF for pharmacokinetic studies were obtained in a subset of patients with Ommaya reservoirs. Results: Irritability, presumably secondary to pain or headache during mafosfamide administration, was dose limiting in two of three patients at the 17-mg dose level. The maximum-tolerated dose of IT mafosfamide following premedication with dexamethasone and morphine was 14 mg. Conclusion: The maximum tolerated dose and recommended phase II dose of IT mafosfamide in patients younger than 3 years with newly diagnosed embryonal CNS tumors is 14 mg. A trial to assess the efficacy of regional therapy with IT mafosfamide administered with intensive systemic chemotherapy in children younger than 3 years with primary intracranial embryonal tumors is now in progress.

Original languageEnglish (US)
Pages (from-to)525-531
Number of pages7
JournalJournal of Clinical Oncology
Volume23
Issue number3
DOIs
StatePublished - Dec 1 2005

ASJC Scopus subject areas

  • Oncology
  • Cancer Research

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    Blaney, S. M., Boyett, J., Friedman, H., Gajjar, A., Geyer, R., Horowtiz, M., Hunt, D., Kieran, M., Kun, L., Packer, R., Phillips, P., Pollack, I. F., Prados, M., & Heideman, R. (2005). Phase I clinical trial of mafosfamide in infants and children aged 3 years or younger with newly diagnosed embryonal tumors: A Pediatric Brain Tumor Consortium Study (PBTC-001). Journal of Clinical Oncology, 23(3), 525-531. https://doi.org/10.1200/JCO.2005.06.544