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Related Experiment Video

Updated: Nov 3, 2025

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Treosulfan-based conditioning for inborn errors of immunity.

Mary A Slatter1, Andrew R Gennery2

  • 1Great North Children's Hospital, Clinical Resource Building, Floor 4, Block 2, Queen Victoria Road, Newcastle Upon Tyne NE1 4LP, UK.

Therapeutic Advances in Hematology
|June 7, 2021
PubMed
Summary

Treosulfan offers a safer, effective conditioning regimen for children with inborn errors of immunity (IEI) undergoing haematopoietic stem-cell transplantation (HSCT). Individualized dosing of treosulfan may further improve outcomes, especially in infants.

Keywords:
haematopoietic stem cell transplantationinborn errors of immunitytreosulfan

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Area of Science:

  • Immunology
  • Hematology
  • Pediatric Medicine

Background:

  • Inborn errors of immunity (IEI) are genetic disorders affecting immune system development and function.
  • Haematopoietic stem-cell transplantation (HSCT) is a key treatment for many IEI, with outcomes improving due to advances in conditioning regimens.
  • Reduced-toxicity conditioning has significantly enhanced HSCT success rates in children.

Purpose of the Study:

  • To review the establishment and efficacy of treosulfan as a conditioning agent for pediatric HSCT in IEI.
  • To highlight treosulfan's advantages over traditional agents like busulfan.
  • To discuss future directions in individualized conditioning for improved HSCT outcomes.

Main Methods:

  • Review of clinical data and guidelines regarding treosulfan use in pediatric HSCT for IEI.
  • Comparison of treosulfan's properties with busulfan, focusing on toxicity and efficacy.
  • Analysis of emerging data on treosulfan pharmacokinetics, particularly in infants.

Main Results:

  • Treosulfan demonstrates myeloablative and immunosuppressive properties with reduced toxicity compared to busulfan.
  • It is widely adopted in European guidelines for conditioning children with IEI undergoing HSCT.
  • Emerging data suggest potential for improved outcomes with pharmacokinetic-guided dosing, especially in infants.

Conclusions:

  • Treosulfan is a safe and effective conditioning agent for children with IEI undergoing HSCT worldwide.
  • Individualized dosing strategies for conditioning agents, including treosulfan, are anticipated to further optimize HSCT outcomes.
  • Future research may explore optimizing graft composition and post-transplant immunotherapy based on these conditioning advancements.