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The challenges presented by haematopoietic stem cell transplantation in children with primary immunodeficiency

Lookup NU author(s): Professor Andrew GenneryORCiD

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Abstract

© The Author(s) 2020. Published by Oxford University Press. All rights reserved. For Permissions, please email: journals.permissions@oup.com.INTRODUCTION OR BACKGROUND: For many primary immunodeficiencies (PIDs), haematopoietic stem cell transplantation (HSCT) offers treatment to cure disease. However, patients with PID present a unique set of challenges when considering HSCT. SOURCES OF DATA: Review of recent literature. AREAS OF AGREEMENT: The most significant recent impact on successful outcome is introduction of newborn screening programmes for diagnosis of severe combined immunodeficiency-wider adoption of screening in an increasing number of countries will see further improvements. Other PIDs have better outcomes when treated earlier, before development of co-morbidities-early referral for consideration of HSCT is important. Evolution of conditioning regimens is improving short- and long-term toxicities-targeted busulfan and low-toxicity myeloablative treosulfan regimens deliver good survival with reduced short-term toxicities. AREAS OF CONTROVERSY: The most radical development, still in clinical trials, is the use of mono-antibody-based conditioning, which eliminates the requirement for chemotherapy and is likely to become much more important in HSCT for non-malignant disease in the future. GROWING POINTS: Multidisciplinary working for optimum care is essential. AREAS TIMELY FOR DEVELOPING RESEARCH: International collaborations are important to learn about rare presentations and complications, and to formulate the most effective and safe treatment strategies.

Publication metadata

Author(s): Gennery AR

Publication type: Article

Publication status: Published

Journal: British Medical Bulletin

Year: 2020

Volume: 135

Issue: 1

Pages: 4-15

Online publication date: 15/07/2020

Acceptance date: 21/05/2020

ISSN (print): 0007-1420

ISSN (electronic): 1471-8391

Publisher: Oxford University Press

URL: https://doi.org/10.1093/bmb/ldaa017

DOI: 10.1093/bmb/ldaa017

PubMed id: 32676650

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