Alternative Donor Hematopoietic Cell Transplantation Conditioned With Myeloablative Busulfan, Fludarabine, and Melphalan is Well Tolerated and Effective Against High-risk Myeloid Malignancies

J Pediatr Hematol Oncol. 2016 Nov;38(8):e315-e318. doi: 10.1097/MPH.0000000000000621.

Abstract

Busulfan, fludarabine, and melphalan as hematopoietic cell transplant conditioning, was used in 6 patients aged 1 to 19 years with very high-risk myeloid malignancies. This dose regimen had an acceptable toxicity profile resulting in complete donor engraftment even following transplantation of small 2/6 antigen disparate umbilical cord blood grafts. It provided excellent disease control as all patients had high-risk features in terms of cytogenetics, therapy-related leukemia, and/or significant measurable disease before transplant. All patients remain in remission, without acute or chronic graft-versus-host disease with a median follow-up of 24 months. A larger study is indicated to confirm the efficacy and safety of this regimen.

MeSH terms

  • Adolescent
  • Busulfan / therapeutic use
  • Child
  • Child, Preschool
  • Female
  • Hematopoietic Stem Cell Transplantation / methods*
  • Humans
  • Infant
  • Leukemia, Myeloid / therapy*
  • Male
  • Melphalan / therapeutic use
  • Myeloablative Agonists / therapeutic use*
  • Myeloproliferative Disorders / therapy*
  • Remission Induction
  • Tissue Donors
  • Transplantation Conditioning / methods*
  • Treatment Outcome
  • Vidarabine / analogs & derivatives
  • Vidarabine / therapeutic use
  • Young Adult

Substances

  • Myeloablative Agonists
  • Vidarabine
  • Busulfan
  • fludarabine
  • Melphalan