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
Creators: name: Achini‐Gutzwiller, Federica R.
name: Snowden, John A.
name: Corbacioglu, Selim
name: Greco, Raffaella
name: Alexander, Tobias
name: Snowden, John
name: Badoglio, Manuela
name: Labopin, Myriam
name: Abinun, Mario
name: Apte, Shashikant
name: Arnold, Renate
name: Domenech, Ariadna
name: Brierley, Charlotte
name: Burman, Joachim
name: Castilla‐Llorente, Cristina
name: Cooper, Nichola
name: Daghia, Giulia
name: Daikeler, Thomas
name: Del Papa, Nicoletta
name: de Vries‐Bouwstra, Jeska
name: Farge, Dominique
name: Finke, Jürgen
name: Hagglund, Hans
name: Hawkey, Chris
name: Henes, Jörg
name: Hiepe, Falk
name: Jessop, Helen
name: Kiely, David
name: Kazmi, Majid
name: Kirgizov, Kirill
name: Kramer, Ellen
name: Mancardi, Gianluigi
name: Marjanovic, Zora
name: Martin, Roland
name: Martin, Thierry
name: Ma, David
name: Moore, John
name: Miller, Paul
name: Muraro, Paolo
name: Oliveira, Maria‐Carolina
name: Polushin, Alexey
name: Onida, Francesco
name: Simoes, Belinda
name: Puyade, Mathieu
name: Resnick, Igor
name: Ricart, Elena
name: Rovira, Montserrat
name: Saccardi, Riccardo
name: Saif, Muhammad
name: Sakellari, Ioanna
name: Sharrack, Basil
name: Snarski, Emilian
name: Scherer, Hans Ulrich
name: Sossa, Claudia
name: Withers, Barbara
name: Wulffraat, Nico
name: Zaccara, Eleanora
name: Amrolia, Persis
name: Ansari, Marc
name: Balduzzi, Adriana
name: Corbacioglu, Selim
name: Dalassier, Arnaud
name: Dalle, Jean‐Hugues
name: Diaz, Cristina Hereda
name: Feuchtinger, Tobias
name: Locatelli, Franco
name: Lucchini, Giovanna
name: Galimard, Jacques-Emmanuel
name: Vincent, Marta Gonzalez
name: Handgretinger, Rupert
name: Kleinschmidt, Katharina
name: Lawitschka, Anita
name: Martinez, Antonio Perez
name: Peters, Christina
name: Rocha, Vanderson
name: Ruggeri, Annalisa
name: Sedlacek, Petr
name: Svec, Peter
name: Toporski, Jacek
name: Yesilipek, Akif
Publisher: name: Universität Regensburg
Year: 2022
Subjects: subject: autoimmune diseases, haematopoietic stem cell transplantation, paediatric
subject: 610 Medizin

description: 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 (&lt;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.