Clinical comparison of weight- and age-based strategy of dose administration in children receiving intravenous busulfan for hematopoietic stem cell transplantation

Pediatr Transplant. 2015 May;19(3):307-15. doi: 10.1111/petr.12430. Epub 2015 Feb 9.

Abstract

Bu, combined with TDM-guided dosing, is associated with fewer graft failures/relapses and lower toxicity in pediatric HSCT. We aimed this retrospective study for comparison of weight- and age-based dosing in terms of clinical outcomes such as time to engraftment, early complications, EFS, OS, and toxicity profiles in children receiving iv Bu. Sixty-one children who underwent HSCT from April 2010 to February 2013 by means of a Bu-based conditioning regimen and completed 100 days after transplantation at Ankara Children?s Hematology and Oncology Hospital Bone Marrow Transplantation Unit were enrolled in this study. SOS and neutropenic fever occurred more frequently in the weight-based dosing group. We found a statistically significant correlation between Bu dose and the incidence of SOS (r = 0.26, p = 0.04). Multivariate analysis showed only weight-based dosing of Bu was a significant predictor of SOS (HR = 9.46; p = 0.009). However, no relationship was found between two groups in terms of hemorrhagic cystitis, engraftment syndrome, acute or chronic GvHD, time to engraftment, chimerism, TRM, OS, and EFS rates. Weight-based dosing of Bu may cause higher incidence of SOS and early infectious complications at the places where TDM of Bu cannot be performed.

Keywords: age-based dosing strategy; busulfan; hematopoietic stem cell transplantation; weight-based dosing strategy.

Publication types

  • Comparative Study

MeSH terms

  • Adolescent
  • Age Factors
  • Body Weight
  • Busulfan / administration & dosage*
  • Child
  • Child, Preschool
  • Cyclophosphamide / therapeutic use
  • Female
  • Fever / etiology
  • Graft Survival
  • Graft vs Host Disease / etiology
  • Hematologic Diseases / therapy
  • Hematologic Neoplasms / therapy
  • Hematopoietic Stem Cell Transplantation*
  • Humans
  • Infant
  • Infusions, Intravenous*
  • Male
  • Multivariate Analysis
  • Neutropenia / etiology
  • Recurrence
  • Retrospective Studies
  • Transplantation Conditioning
  • Treatment Outcome

Substances

  • Cyclophosphamide
  • Busulfan