Fludarabine-based reduced-intensity conditioning regimen for hematopoietic stem cell transplantation in pediatric patient with IL10 receptor deficiency

Pediatr Transplant. 2021 Mar;25(2):e13819. doi: 10.1111/petr.13819. Epub 2020 Aug 26.

Abstract

IL-10R deficiency results in severe immune dysregulation. Herein, we describe the successful treatment of a girl aged 6.8 years with IL10R deficiency by using RIC prior to HSCT from a matched unrelated donor. The regimen was well tolerated, the engraftment was completely attained. On a follow-up of 7 months, the patient remained in good medical conditions with full donor chimerism. All complications before HSCT were completely resolved and her growth was accelerated. RIC regimen might be adequate to induce permanent engraftment and avoid severe organ toxicity in IL-10R deficiency patients.

Keywords: IL-10R deficiency; hematopoietic stem cell transplantation; reduced-intensity conditioning.

Publication types

  • Case Reports

MeSH terms

  • Child
  • Drug Administration Schedule
  • Drug Therapy, Combination
  • Female
  • Genetic Markers
  • Hematopoietic Stem Cell Transplantation*
  • Humans
  • Injections, Intravenous
  • Myeloablative Agonists / therapeutic use*
  • Primary Immunodeficiency Diseases / genetics
  • Primary Immunodeficiency Diseases / therapy*
  • Receptors, Interleukin-10 / deficiency*
  • Receptors, Interleukin-10 / genetics
  • Transplantation Conditioning / methods*
  • Vidarabine / analogs & derivatives*
  • Vidarabine / therapeutic use

Substances

  • Genetic Markers
  • Myeloablative Agonists
  • Receptors, Interleukin-10
  • Vidarabine
  • fludarabine