title: Alternative Donor Hematopoietic Cell Transplantation Conditioned With Myeloablative Busulfan, Fludarabine, and Melphalan is Well Tolerated and Effective Against High-risk Myeloid Malignancies.
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.
authors.affiliation: Departments of *Pediatrics †Immunobiology ‡Medicine §Pathology ∥University of Arizona Cancer Center, University of Arizona ¶Banner University Medical Center, Tucson, AZ.
authors.forename: Emmanuel
authors.initials: E
authors.lastname: Katsanis
authors.forename: Lauren N
authors.initials: LN
authors.lastname: Sapp
authors.forename: Luz
authors.initials: L
authors.lastname: Pelayo-Katsanis
authors.forename: Katherine
authors.initials: K
authors.lastname: Whitney
authors.forename: Yi
authors.initials: Y
authors.lastname: Zeng
authors.forename: Lisa M
authors.initials: LM
authors.lastname: Kopp
mesh_terms: D000293:Adolescent; D002066:Busulfan / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D007223:Infant; D007951:Leukemia, Myeloid / Q000628:therapy*; D008297:Male; D008558:Melphalan / Q000627:therapeutic use; D019653:Myeloablative Agonists / Q000627:therapeutic use*; D009196:Myeloproliferative Disorders / Q000628:therapy*; D012074:Remission Induction; D014019:Tissue Donors; D019172:Transplantation Conditioning / Q000379:methods*; D016896:Treatment Outcome; D014740:Vidarabine / Q000031:analogs & derivatives / Q000627:therapeutic use; D055815:Young Adult
chemical_list: D019653:Myeloablative Agonists; D014740:Vidarabine; D002066:Busulfan; C024352:fludarabine; D008558:Melphalan