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

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

Research output: Contribution to journalArticle

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 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.
Original languageEnglish
Pages (from-to)24-45
Number of pages22
JournalBritish Journal of Haematology
Volume198
DOIs
Publication statusPublished - 2022

Keywords

  • autoimmune diseases
  • paediatric
  • haematopoietic stem cell transplantation

Fingerprint

Dive into the research topics of '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'. Together they form a unique fingerprint.

Cite this