Lookup NU author(s): Professor Andrew Gennery,
Dr Mary Slatter
This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).
© 2019, The Author(s).Purpose of Review: Hematopoietic cell transplantation (HCT) is an established curative treatment for children with primary immunodeficiencies. This article reviews the latest developments in conditioning regimens for primary immunodeficiency (PID). It focuses on data regarding transplant outcomes according to newer reduced toxicity conditioning regimens used in HCT for PID. Recent Findings: Conventional myeloablative conditioning regimens are associated with significant acute toxicities, transplant-related mortality, and late effects such as infertility. Reduced toxicity conditioning regimens have had significant positive impacts on HCT outcome, and there are now well-established strategies in children with PID. Treosulfan has emerged as a promising preparative agent. Use of a peripheral stem cell source has been shown to be associated with better donor chimerism in patients receiving reduced toxicity conditioning. Minimal conditioning regimens using monoclonal antibodies are in clinical trials with promising results thus far. Summary: Reduced toxicity conditioning has emerged as standard of care for PID and has resulted in improved transplant survival for patients with significant comorbidities.
Author(s): Lum SH, Hoenig M, Gennery AR, Slatter MA
Publication type: Review
Publication status: Published
Journal: Current Allergy and Asthma Reports
Online publication date: 18/11/2019
Acceptance date: 02/04/2018
ISSN (print): 1529-7322
ISSN (electronic): 1534-6315
Publisher: Current Medicine Group LLC 1
PubMed id: 31741098