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Lookup NU author(s): Dr Mario Abinun,
Dr Mary Slatter
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Copyright © 2021 Wolters Kluwer Health, Inc. All rights reserved. PURPOSE OF REVIEW: A small proportion of children affected by rheumatic diseases suffer from severe, progressive disease, resistant to conventional antirheumatic therapies and to biologic agents interfering with inflammatory cytokines, costimulatory molecules expressed on immune system cells and intracellular signalling pathways. Adding to the poor prognosis is a high risk from significant morbidity and mortality associated with long-term treatment with multiple, often combined anti-inflammatory and immunosuppressive agents. Carefully selected patients from this unfortunate group may benefit from treatment with haematopoietic stem cell transplantation. RECENT FINDINGS: The majority of patients with severe paediatric rheumatic and autoinflammatory diseases treated with autologous and/or allogeneic haematopoietic stem cell transplantation achieved long-term remission. However, the incidence of disease relapse and transplant related morbidity and mortality is still significant. SUMMARY: Careful patient and donor selection, timing of the transplant earlier in the course of disease rather than the 'last resort' and choosing the most suitable conditioning regimen for each individual patient are the major factors favouring successful outcome. Close co-operation between the patients, their family, and involved medical teams is essential.
Author(s): Abinun M, Slatter MA
Publication type: Article
Publication status: Published
Journal: Current Opinion in Rheumatology
Print publication date: 01/09/2021
Acceptance date: 02/04/2018
ISSN (print): 1040-8711
ISSN (electronic): 1531-6963
Publisher: Lippincott Williams & Wilkins, Ltd
PubMed id: 34261117
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