High Melphalan Exposure Increases the Risk of Graft-Versus-Host Disease in Pediatric Patients Undergoing Alpha-Beta T-Cell Depleted Haploidentical Transplantation
High melphalan exposure predicted by a validated population PK model is associated with an increased likelihood of developing ES and subsequently acute and chronic GVHD. Given that a substantial number of patients already require adjustment of standard BSA-based dosing for young age or obesity, a prospective trial of model-based dosing to individualize melphalan exposure is warranted to confirm these results.
Authors
- Michael A. Pulsipher (ORCID: https://orcid.org/0000-0003-3030-8420)
- William C. Temple
- Geoffrey Cheng
- James N. Huang (ORCID: https://orcid.org/0000-0002-1808-0358)
- Gabriel Salinas Cisneros (ORCID: https://orcid.org/0000-0002-4593-3221)
- Lena E. Winestone (ORCID: https://orcid.org/0000-0001-9982-1594)
- Christopher C. Dvorak (ORCID: https://orcid.org/0000-0002-6146-3952)
- Serine Avagyan (ORCID: https://orcid.org/0000-0001-9522-6237)
- Soohee Cho
- Kristin A. Shimano (ORCID: https://orcid.org/0000-0001-8371-1040)
- Paibel Aguayo‐Hiraldo (ORCID: https://orcid.org/0000-0002-0196-806X)
- Sandhya Kharbanda (ORCID: https://orcid.org/0000-0002-6014-7659)
- Ron J. Keizer (ORCID: https://orcid.org/0000-0002-2460-7056)
- Janel Long-Boyle
- Nahal Lalefar (ORCID: https://orcid.org/0000-0002-5651-7426)
- Christine S. Higham (ORCID: https://orcid.org/0000-0002-4618-3813)
- Philip T. Pauerstein (ORCID: https://orcid.org/0000-0001-5157-204X)
- Julia Chu (ORCID: https://orcid.org/0000-0003-4956-2189)
Institutions
- Primary Children's Hospital (US)
- Children's Hospital of Los Angeles (US)
- University of California, San Francisco (US)
- Huntsman Cancer Institute (US)
- IDinsight (US)
- International Neuromodulation Society (US)
Publication Details
- Journal
- Transplantation and Cellular Therapy
- Published
- 2025-04-05
- DOI
- https://doi.org/10.1016/j.jtct.2025.03.020
- Citations
- 2
- Primary Topic
- Hematopoietic Stem Cell Transplantation
- Type
- article
- Field-Weighted Citation Impact
- 3.54
Funders
- National Institutes of Health
- National Heart, Lung, and Blood Institute
- National Cancer Institute