Reduced-intensity conditioning therapy with busulfan, fludarabine, and antithymocyte globulin for HLA-haploidentical hematopoietic cell transplantation in acute leukemia and myelodysplastic syndrome

Blood. 2011 Sep 1;118(9):2609-17. doi: 10.1182/blood-2011-02-339838. Epub 2011 Jun 28.

Abstract

Any role for reduced-intensity conditioning (RIC) before hematopoietic cell transplantation (HCT) from a human leukocyte antigen (HLA)-haploidentical donor remains to be defined. We therefore assessed 83 patients (age, 16-70 years): 68 with acute leukemia (including 34 in remission and 34 with refractory disease) and 15 patients with myelodysplastic syndrome, in HCT trials using RIC with busulfan, fludarabine, and antithymocyte globulin. The HLA-haploidentical donors, offspring (n = 38), mothers (n = 24), or siblings (n = 21) of patients, underwent leukapheresis after receiving granulocyte colony-stimulating factor, and donated cells were transplanted without further manipulation. Cyclosporine and methotrexate were given for GVHD prophylaxis. The cumulative incidences of neutrophil engraftment, grade 2 to 4 acute GVHD, chronic GVHD, and transplantation-related mortality after HCT, were 92%, 20%, 34%, and 18%, respectively. After a median follow-up time of 26.6 months (range, 16.8-78.8 months), the event-free and overall survival rates were 56% and 45%, respectively, for patients with acute leukemia in remission; 9% and 9%, respectively, for patients with refractory acute leukemia; and 53% and 53%, respectively, for patients with myelodysplastic syndrome. HCT from an HLA-haploidentical family member resulted in favorable outcomes when RIC containing antithymocyte globulin was performed. This study is registered at www.clinicaltrials.gov as #NCT00521430 and #NCT00732316.

Publication types

  • Evaluation Study

MeSH terms

  • Acute Disease
  • Adolescent
  • Adult
  • Aged
  • Antilymphocyte Serum / administration & dosage*
  • Busulfan / administration & dosage*
  • Disease-Free Survival
  • Female
  • Graft Survival
  • Graft vs Host Disease / prevention & control
  • Granulocyte Colony-Stimulating Factor / pharmacology
  • HLA Antigens / analysis*
  • Hematopoietic Stem Cell Transplantation / methods*
  • Histocompatibility
  • Humans
  • Infections
  • Kaplan-Meier Estimate
  • Leukemia / mortality
  • Leukemia / surgery*
  • Male
  • Middle Aged
  • Myelodysplastic Syndromes / mortality
  • Myelodysplastic Syndromes / surgery*
  • Peripheral Blood Stem Cell Transplantation / methods*
  • Retrospective Studies
  • T-Lymphocytes / immunology
  • Tissue Donors
  • Transplantation Conditioning / methods*
  • Treatment Outcome
  • Vidarabine / administration & dosage
  • Vidarabine / analogs & derivatives*
  • Young Adult

Substances

  • Antilymphocyte Serum
  • HLA Antigens
  • Granulocyte Colony-Stimulating Factor
  • Vidarabine
  • Busulfan
  • fludarabine

Associated data

  • ClinicalTrials.gov/NCT00521430
  • ClinicalTrials.gov/NCT00732316