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Updated: Jul 8, 2025

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Optimizing high dose melphalan.

Gunjan Shah1, Sergio Giralt1, Parastoo Dahi1

  • 1Adult BMT Service Memorial Sloan Kettering Cancer Center, 530 East 74th Street, New York, NY 10021, United States of America.

Blood Reviews
|December 14, 2023
PubMed
Summary

High-dose melphalan (HDM) is key for multiple myeloma HCT. Strategies are explored to improve HDM efficacy and reduce toxicities like myelosuppression and mucositis for better patient outcomes.

Keywords:
Autologous stem cell transplantationHigh dose therapyMelphalanMyeloma

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Area of Science:

  • Hematology
  • Oncology
  • Pharmacology

Background:

  • High-dose melphalan (HDM) is a cornerstone of conditioning regimens for autologous hematopoietic cell transplantation (HCT) in multiple myeloma.
  • Despite its efficacy, HDM is associated with significant toxicities, including myelosuppression, gastrointestinal issues, and mucositis.

Purpose of the Study:

  • To review current strategies for enhancing the efficacy of HDM in multiple myeloma treatment.
  • To explore methods for mitigating HDM-related toxicities in patients undergoing HCT.

Main Methods:

  • Review of established and emerging approaches to manage HDM toxicity, such as dose modification, antiemetics, cryotherapy, amifostine, and growth factors.
  • Discussion of novel strategies including personalized dosing, combination therapies (e.g., with busulfan, bendamustine), and new formulations like propylene glycol-free melphalan (Evomela).

Main Results:

  • Established methods effectively manage common HDM toxicities.
  • Personalized dosing, combination therapies, and novel formulations show promise for optimizing melphalan exposure and reducing adverse effects.

Conclusions:

  • Multiple strategies exist to improve the therapeutic index of HDM in multiple myeloma HCT.
  • Further clinical trials are needed to validate these strategies, aiming to enhance safety and efficacy for improved patient outcomes.