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. (27th April 2022)

• Record Type: Journal Article
• Authors: Achini‐Gutzwiller, Federica R.; Snowden, John A.; Corbacioglu, Selim; Greco, Raffaella
• Other Names: Greco Raffaella investigator.; Alexander Tobias investigator.; Snowden John investigator.; Badoglio Manuela investigator.; Labopin Myriam investigator.; Abinun Mario investigator.; Apte Shashikant investigator.; Arnold Renate investigator.; Domenech Ariadna investigator.; Brierley Charlotte investigator.; Burman Joachim investigator.; Castilla‐Llorente Cristina investigator.; Cooper Nichola investigator.; Daghia Giulia investigator.; Daikeler Thomas investigator.; del Papa Nicoletta investigator.; de Vries‐Bouwstra Jeska investigator.; Farge Dominique investigator.; Finke Jurgen investigator.; Hagglund Hans investigator.; Hawkey Chris investigator.; Henes Jörg investigator.; Hiepe Falk investigator.; Jessop Helen investigator.; Kiely David investigator.; Kazmi Majid investigator.; Kirgizov Kirill investigator.; Kramer Ellen investigator.; Mancardi Gianluigi investigator.; Marjanovic Zora investigator.; Martin Roland investigator.; Martin Thierry investigator.; Ma David investigator.; Moore John investigator.; Miller Paul investigator.; Muraro Paolo investigator.; Oliveira Maria‐Carolina investigator.; Polushin Alexey investigator.; Onida Francesco investigator.; Simoes Belinda investigator.; Puyade Mathieu investigator.; Resnick Igor investigator.; Ricart Elena investigator.; Rovira Montserrat investigator.; Saccardi Riccardo investigator.; Saif Muhammad investigator.; Sakellari Ioanna investigator.; Sharrack Basil investigator.; Snarski Emilian investigator.; Scherer Hans Ulrich investigator.; Sossa Claudia investigator.; Withers Barbara investigator.; Wulffraat Nico investigator.; Zaccara Eleanora investigator.; Amrolia Persis investigator.; Ansari Marc investigator.; Balduzzi Adriana investigator.; Corbacioglu Selim investigator.; Dalassier Arnaud investigator.; Dalle Jean‐Hugues investigator.; Diaz Cristina Hereda investigator.; Feuchtinger Tobias investigator.; Locatelli Franco investigator.; Lucchini Giovanna investigator.; Galimard Jaques‐Emmanuel investigator.; Vincent Marta Gonzalez investigator.; Handgretinger Rupert investigator.; Kleinschmidt Katharina investigator.; Lawitschka Anita investigator.; Martinez Antonio Perez investigator.; Peters Christina investigator.; Rocha Vanderson investigator.; Ruggeri Annalisa investigator.; Sedlacek Petr investigator.; Svec Peter investigator.; Toporski Jacek investigator.; Yesilipek Akif investigator.;
• Is Part Of: British journal of haematology. Volume 198:Number 1(2022)
• Page Start: 24
• Page End: 45
• Publication Date: 2022-04-27
• Subjects: autoimmune diseases -- haematopoietic stem cell transplantation -- paediatric; Hematology -- Periodicals; Blood -- Diseases -- Periodicals; 616.15
• DOI: 10.1111/bjh.18176
• Languages: English
• ISSNs: 0007-1048

Abstract

Summary: 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 control and often apparent cure of severe treatment‐refractory autoimmune diseases (ADs) in children. However, transplant‐related outcomes are disease‐dependent and long‐term outcome data are limited in respect to efficacy and safety. Moreover, balancing risks of HSCT against AD prognosis with continually evolving non‐transplant options is challenging. This review appraises published literature on HSCT strategies and outcomes in individual paediatric ADs. We also provide a summary of the European Society for Blood and Marrow Transplantation (EBMT) Registry, where 343 HSCT procedures (176 autologous and 167 allogeneic) have been reported in 326 children (<18 years) for a range of AD indications. HSCT is a promising treatment modality, with potential long‐term disease control or cure, but therapy‐related morbidity and mortality need to be reduced. Further research is warranted to establish the position of HSCT in paediatric ADs via registries and prospective clinical studies to support evidence‐based interspeciality guidelines and recommendations.