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Area of Science:

  • Clinical Immunology
  • Transplantation Medicine
  • Hematology

Background:

  • Primary immunodeficiencies, or inborn errors of immunity (IEI), stem from defects in blood cells originating from hematopoietic stem cells.
  • Hematopoietic stem cell transplantation (HSCT) has been a definitive therapy for IEI for five decades.
  • Advances in immunology and transplantation medicine enable better diagnosis and successful HSCT for IEI.

Purpose of the Study:

  • To offer practical guidance for allergist-immunologists managing IEI patients undergoing HSCT.
  • To cover HSCT basics, outcomes in specific IEI types, and pre-transplant care.
  • To emphasize lifelong post-HSCT immunologic and medical monitoring.

Main Methods:

  • Review of current literature and clinical practices in IEI and HSCT.
  • Synthesis of information on diagnostic advancements and therapeutic outcomes.
  • Guideline development for pre- and post-HSCT management.

Main Results:

  • Increasing identification of monogenic causes for IEI.
  • Improved success rates and broader application of HSCT for IEI treatment.
  • Recognition of the critical need for comprehensive pre- and post-HSCT care.

Conclusions:

  • Allergist-immunologists play a key role in diagnosing and managing IEI patients before and after HSCT.
  • Successful HSCT requires meticulous pre-transplant support and long-term monitoring.
  • This review provides essential guidance for optimizing IEI patient care through HSCT.