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

Emmanuel Katsanis, Lauren N. Sapp, Luz Pelayo-Katsanis, Katherine Whitney, Yi - Zeng, Lisa M Kopp

Research output: Contribution to journalArticle

2 Citations (Scopus)

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.

Original languageEnglish (US)
Pages (from-to)e315-e318
JournalJournal of Pediatric Hematology/Oncology
Volume38
Issue number8
DOIs
StatePublished - Nov 1 2016

Fingerprint

Busulfan
Melphalan
Cell Transplantation
Tissue Donors
Transplants
Neoplasms
Graft vs Host Disease
Fetal Blood
Cytogenetics
Leukemia
Transplantation
Safety
Antigens
fludarabine
Therapeutics

Keywords

  • conditioning regimens
  • hematopoietic cell transplantation
  • myeloid leukemia

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Hematology
  • Oncology

Cite this

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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.",
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AU - Sapp, Lauren N.

AU - Pelayo-Katsanis, Luz

AU - Whitney, Katherine

AU - Zeng, Yi -

AU - Kopp, Lisa M

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