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IV melphalan in children.

J B Belasco1, C D Mitchell, T Rohrbaugh

  • 1Division of Oncology, Children's Hospital of Philadelphia, PA 19104.

Cancer Treatment Reports
|December 1, 1987
PubMed
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Intravenous melphalan in children with relapsed cancers showed severe hematologic toxicity across all doses. While some responses were observed, the narrow therapeutic window suggests careful consideration for future pediatric cancer treatment strategies.

Area of Science:

  • Pediatric Oncology
  • Pharmacology
  • Hematology

Background:

  • Multiply relapsed cancers in children present significant treatment challenges.
  • Melphalan is an alkylating agent used in cancer chemotherapy.
  • Understanding melphalan's toxicity and efficacy in pediatric populations is crucial.

Purpose of the Study:

  • To evaluate the safety and efficacy of intravenous melphalan in children with multiply relapsed cancers.
  • To determine the dose-limiting toxicities of melphalan in this patient group.
  • To identify potential roles for melphalan in future pediatric cancer therapy.

Main Methods:

  • Forty children with multiply relapsed cancers were enrolled.
  • Intravenous melphalan was administered at three dose levels: 30, 45, and 60 mg/m2.

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  • Hematologic and nonhematologic toxicities were assessed, along with treatment response.

    • Main Results:

    • Severe and protracted hematologic toxicity (grade 3 or 4) was observed in 37 of 39 evaluable patients.
    • Nonhematologic toxic effects were infrequent and mild.
    • Two complete responses (Hodgkin's disease, rhabdomyosarcoma) and eight partial responses were noted, with 30 treatment failures.

    Conclusions:

    • Intravenous melphalan demonstrated a narrow therapeutic index in children with relapsed cancers, characterized by significant hematologic toxicity.
    • Further investigation of melphalan may be warranted at higher doses (e.g., >100 mg/m2) with marrow rescue for cytoreduction, or in combination therapy for specific pediatric cancers (lymphoma, sarcoma).
    • Lower doses (20-30 mg/m2 every 4 weeks) might be considered as part of combination regimens.