2022
DOI: 10.1111/bjh.18176
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Haematopoietic stem cell transplantation for severe autoimmune diseases in children: A review of current literature, registry activity and future directions on behalf of the autoimmune diseases and paediatric diseases working parties of the European Society for Blood and Marrow Transplantation

Abstract: Although modern clinical management strategies have improved the outcome of paediatric patients with severe autoimmune and inflammatory diseases over recent decades, a proportion will experience ongoing or recurrent/relapsing disease activity despite multiple therapies often leading to irreversible organ damage, and compromised quality of life, growth/development and long-term survival. Autologous and allogeneic haematopoietic stem cell transplantation (HSCT) have been used successfully to induce disease contr… Show more

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Cited by 9 publications
(2 citation statements)
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References 155 publications
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“…The EBMT registry has documented 16 paediatric cases of progressive, treatment-resistant SSc since 1999 including limited disease course and outcome data. 22 In 2004, a European study of 57 patients with SSc included five children. Overall, most patients had a favourable response to AHSCT.…”
Section: Discussionmentioning
confidence: 99%
“…The EBMT registry has documented 16 paediatric cases of progressive, treatment-resistant SSc since 1999 including limited disease course and outcome data. 22 In 2004, a European study of 57 patients with SSc included five children. Overall, most patients had a favourable response to AHSCT.…”
Section: Discussionmentioning
confidence: 99%
“… 1 In addition, with the increasing recognition of genetic risk variants contributing to the development of certain AIDs, it has become evident that allogeneic rather than autologous HSCT could be a promising approach for long-term disease control, particularly when there is overlap with autoinflammatory and immunodeficiency diseases in the pediatric setting. 7 , 8 A careful and multidisciplinary appraisal of potential benefits and risks by disease and transplant specialists, working closely together in a multidisciplinary team with patients and carers, is strongly recommended to determine individual suitability for HSCT. 9 …”
Section: Introductionmentioning
confidence: 99%