Long-term outcomes of unmanipulated haploidentical HSCT for paediatric patients with acute leukaemia

Bone Marrow Transplant. 2013 Nov;48(12):1519-24. doi: 10.1038/bmt.2013.99. Epub 2013 Jul 29.

Abstract

Allogeneic hematopoietic SCT is indicated for children whose disease demonstrates dismal prognosis with chemotherapy. This study aims to analyse the most recent outcomes of unmanipulated haploidentical (HID) HSCT for paediatric patients with acute leukaemia. Those from matched sibling donors (MSD) HSCT provided a parallel cohort to illustrate the benefits of HID. Conditioning regimen was modified BuCy2. Anti-thymoglobulin was used for HID. Mobilised marrow and blood stem cells were used as the grafts. All patients in HID achieved neutrophil recovery and 96.7% platelet recovery. In HID, the incidences of acute GVHD 3-4 and extensive chronic GVHD were 14.3 and 26.6%. Play-performance score 90-100% was recorded in 79.7% of all survivors. The 5-year leukaemia-free survival (LFS) in CR1, CR2, beyond CR2 or non-remission were 68.9%, 56.6%, 22.2% and 82.5%, 59.4%, 42.9% for ALL and AML, respectively. In MSD group, LFS for ALL and AML in CR1 were 62.5 and 71.7%. Outcomes of the HID HSCT for paediatric patients with acute leukaemia showed benefits that were similar to those of the parallel cohort of MSD HSCT.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Antineoplastic Combined Chemotherapy Protocols / administration & dosage
  • Busulfan / administration & dosage
  • Child
  • Child, Preschool
  • Cohort Studies
  • Cyclophosphamide / administration & dosage
  • Disease-Free Survival
  • Female
  • Hematopoietic Stem Cell Transplantation / methods*
  • Humans
  • Leukemia, Myeloid, Acute / drug therapy
  • Leukemia, Myeloid, Acute / surgery
  • Leukemia, Myeloid, Acute / therapy*
  • Male
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma / drug therapy
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma / surgery
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma / therapy*
  • Survival Analysis
  • Transplantation Conditioning / methods*
  • Transplantation, Autologous
  • Treatment Outcome

Substances

  • Cyclophosphamide
  • Busulfan

Supplementary concepts

  • BUCY-2 protocol