Update on Advances in Hematopoietic Cell Transplantation for Primary Immunodeficiency Disorders

Immunol Allergy Clin North Am. 2019 Feb;39(1):113-128. doi: 10.1016/j.iac.2018.08.003. Epub 2018 Nov 1.

Abstract

Hematopoietic stem cell transplantation (HSCT) in patients with primary immunodeficiency disorders (PIDDs) is being increasingly used as a curative option. Understanding the critical components, such as disease's nature and activity and pre-HSCT and post-HSCT patient care is key to a successful outcome. HSCT should be tailored to the underlying PIDD, as different PIDDs, such as severe combined immune deficiency, Treg dysfunction, and phagocytic disorders, have different transplant approaches. Therefore, successful HSCT in patients with PIDDs requires teamwork between immunologists and transplant physicians. In this article, the authors elaborate on various aspects of PIDD-HSCT and highlight recent advances.

Keywords: Bone marrow transplantation; Conditioning; Hematopoietic stem cell transplantation; Immune dysregulation; Primary immune deficiency.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Bone Marrow Transplantation
  • Disease Management
  • Disease Susceptibility
  • Graft vs Host Disease / etiology
  • Hematopoietic Stem Cell Transplantation* / adverse effects
  • Hematopoietic Stem Cell Transplantation* / methods
  • Humans
  • Immunologic Deficiency Syndromes / diagnosis
  • Immunologic Deficiency Syndromes / etiology
  • Immunologic Deficiency Syndromes / therapy*
  • Infections / etiology
  • Severe Combined Immunodeficiency / diagnosis
  • Severe Combined Immunodeficiency / etiology
  • Severe Combined Immunodeficiency / therapy
  • Transplantation Conditioning
  • Treatment Outcome