TY - JOUR
T1 - Haematopoietic stem cell transplantation for severe autoimmune diseases in children
T2 - 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
AU - The EBMT Autoimmune Diseases (ADWP) and Paediatric Diseases (PDWP) Working Parties
AU - Achini-Gutzwiller, Federica R.
AU - Snowden, John A.
AU - Corbacioglu, Selim
AU - Greco, Raffaella
AU - Alexander, Tobias
AU - Snowden, John
AU - Badoglio, Manuela
AU - Labopin, Myriam
AU - Abinun, Mario
AU - Apte, Shashikant
AU - Arnold, Renate
AU - Domenech, Ariadna
AU - Brierley, Charlotte
AU - Burman, Joachim
AU - Castilla-Llorente, Cristina
AU - Cooper, Nichola
AU - Daghia, Giulia
AU - Daikeler, Thomas
AU - del Papa, Nicoletta
AU - de Vries-Bouwstra, Jeska
AU - Farge, Dominique
AU - Finke, Jurgen
AU - Hagglund, Hans
AU - Hawkey, Chris
AU - Henes, Jörg
AU - Hiepe, Falk
AU - Jessop, Helen
AU - Kiely, David
AU - Kazmi, Majid
AU - Kirgizov, Kirill
AU - Kramer, Ellen
AU - Mancardi, Gianluigi
AU - Marjanovic, Zora
AU - Martin, Roland
AU - Martin, Thierry
AU - Ma, David
AU - Moore, John
AU - Miller, Paul
AU - Muraro, Paolo
AU - Oliveira, Maria Carolina
AU - Polushin, Alexey
AU - Onida, Francesco
AU - Simoes, Belinda
AU - Puyade, Mathieu
AU - Resnick, Igor
AU - Ricart, Elena
AU - Rovira, Montserrat
AU - Saccardi, Riccardo
AU - Saif, Muhammad
AU - Sossa, Claudia
N1 - Publisher Copyright:
© 2022 The Authors. British Journal of Haematology published by British Society for Haematology and John Wiley & Sons Ltd.
PY - 2022/7/1
Y1 - 2022/7/1
N2 - 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.
AB - 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.
KW - autoimmune diseases
KW - haematopoietic stem cell transplantation
KW - paediatric
UR - http://www.scopus.com/inward/record.url?scp=85133894266&partnerID=8YFLogxK
U2 - 10.1111/bjh.18176
DO - 10.1111/bjh.18176
M3 - Articulo en revista no especializada
AN - SCOPUS:85133894266
SN - 0007-1048
VL - 198
SP - 24
EP - 45
JO - British Journal of Haematology
JF - British Journal of Haematology
IS - 1
ER -