Paediatric reduced intensity conditioning: analysis of centre strategies on regimens and definitions by the EBMT Paediatric Diseases and Complications and Quality of Life WP

Bone Marrow Transplant. 2015 Apr;50(4):592-7. doi: 10.1038/bmt.2014.306. Epub 2015 Jan 26.

Abstract

The aim of this analysis was to explore the diversity of reduced intensity conditioning (RIC) in paediatric allo-SCT in daily practice across Europe. Data from the European Group for Blood and Marrow Transplantation (EBMT) Promise database from 1994 to 2008 were supplemented by a survey of EBMT centres performing paediatric allo-SCT on the current policy asking for the underlying diseases and for the drug combinations. Records from 161 centres from 30 countries were analysed and 139 various RIC regimens were reported. More centres applied RIC for malignant rather than for non-malignant diseases. In general, fludarabine (FLU)-based regimens predominated except for BU-based regimens in myeloid malignancies and haemoglobinopathies. Treosulfan (TREO) was mainly applied for unspecified malignant diseases and for haemophagocytic diseases. FLU-based regimens revealed the greatest number of different combinations. Correlating the number of regimens with the number of treating centres revealed the lowest variety in FLU and the highest variety in TBI and TREO. FLU/melphalane and FLU/CY were the most frequent combinations. This extreme heterogeneity in RIC may influence both the efficacy and the safety of the procedures, which requires further investigation. Optimization and standardization of RIC is the final goal to provide a platform for future prospective studies.

Publication types

  • Multicenter Study

MeSH terms

  • Adolescent
  • Allografts
  • Antineoplastic Agents, Alkylating / administration & dosage
  • Busulfan / administration & dosage
  • Busulfan / analogs & derivatives
  • Child
  • Child, Preschool
  • Databases, Factual*
  • Female
  • Hematologic Diseases / therapy*
  • Hematopoietic Stem Cell Transplantation*
  • Humans
  • Infant
  • Male
  • Myeloablative Agonists / administration & dosage
  • Quality of Health Care
  • Quality of Life*
  • Retrospective Studies
  • Transplantation Conditioning / methods*
  • Vidarabine / administration & dosage
  • Vidarabine / analogs & derivatives

Substances

  • Antineoplastic Agents, Alkylating
  • Myeloablative Agonists
  • treosulfan
  • Vidarabine
  • Busulfan
  • fludarabine