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
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 irrev...
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| Veröffentlicht in: | BRITISH JOURNAL OF HAEMATOLOGY 2022-07, Vol.198 (1), p.24-45 |
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| creator | Achini‐Gutzwiller, Federica R. Snowden, John A. Corbacioglu, Selim Greco, Raffaella Greco, Raffaella Alexander, Tobias Snowden, John Badoglio, Manuela Labopin, Myriam Abinun, Mario Apte, Shashikant Arnold, Renate Domenech, Ariadna Brierley, Charlotte Burman, Joachim Castilla‐Llorente, Cristina Cooper, Nichola Daghia, Giulia Daikeler, Thomas Papa, Nicoletta Vries‐Bouwstra, Jeska Farge, Dominique Finke, Jurgen Hagglund, Hans Hawkey, Chris Henes, Jörg Hiepe, Falk Jessop, Helen Kiely, David Kazmi, Majid Kirgizov, Kirill Kramer, Ellen Mancardi, Gianluigi Marjanovic, Zora Martin, Roland Martin, Thierry Ma, David Moore, John Miller, Paul Muraro, Paolo Oliveira, Maria‐Carolina Polushin, Alexey Onida, Francesco Simoes, Belinda Puyade, Mathieu Resnick, Igor Ricart, Elena Rovira, Montserrat Saccardi, Riccardo Saif, Muhammad Sakellari, Ioanna Sharrack, Basil Snarski, Emilian Scherer, Hans Ulrich Sossa, Claudia Withers, Barbara Wulffraat, Nico Zaccara, Eleanora Amrolia, Persis Ansari, Marc Balduzzi, Adriana Corbacioglu, Selim Dalassier, Arnaud Dalle, Jean‐Hugues Diaz, Cristina Hereda Feuchtinger, Tobias Locatelli, Franco Lucchini, Giovanna Galimard, Jaques‐Emmanuel Vincent, Marta Gonzalez Handgretinger, Rupert Kleinschmidt, Katharina Lawitschka, Anita Martinez, Antonio Perez Peters, Christina Rocha, Vanderson Ruggeri, Annalisa Sedlacek, Petr Svec, Peter Toporski, Jacek Yesilipek, Akif |
| description | 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 ( |
| doi_str_mv | 10.1111/bjh.18176 |
| format | Article |
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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.</description><identifier>ISSN: 0007-1048</identifier><identifier>EISSN: 1365-2141</identifier><identifier>DOI: 10.1111/bjh.18176</identifier><language>eng</language><publisher>Oxford: Blackwell Publishing Ltd</publisher><subject>Autografts ; Autoimmune diseases ; Children ; Disease control ; haematopoietic stem cell transplantation ; Hematology ; Hematopoietic stem cells ; Inflammatory diseases ; Medical prognosis ; Morbidity ; paediatric ; Pediatrics ; Quality of life ; Stem cell transplantation</subject><ispartof>BRITISH JOURNAL OF HAEMATOLOGY, 2022-07, Vol.198 (1), p.24-45</ispartof><rights>2022 The Authors. published by British Society for Haematology and John Wiley & Sons Ltd.</rights><rights>2022. 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Jörg</creatorcontrib><creatorcontrib>Hiepe, Falk</creatorcontrib><creatorcontrib>Jessop, Helen</creatorcontrib><creatorcontrib>Kiely, David</creatorcontrib><creatorcontrib>Kazmi, Majid</creatorcontrib><creatorcontrib>Kirgizov, Kirill</creatorcontrib><creatorcontrib>Kramer, Ellen</creatorcontrib><creatorcontrib>Mancardi, Gianluigi</creatorcontrib><creatorcontrib>Marjanovic, Zora</creatorcontrib><creatorcontrib>Martin, Roland</creatorcontrib><creatorcontrib>Martin, Thierry</creatorcontrib><creatorcontrib>Ma, David</creatorcontrib><creatorcontrib>Moore, John</creatorcontrib><creatorcontrib>Miller, Paul</creatorcontrib><creatorcontrib>Muraro, Paolo</creatorcontrib><creatorcontrib>Oliveira, Maria‐Carolina</creatorcontrib><creatorcontrib>Polushin, Alexey</creatorcontrib><creatorcontrib>Onida, Francesco</creatorcontrib><creatorcontrib>Simoes, Belinda</creatorcontrib><creatorcontrib>Puyade, Mathieu</creatorcontrib><creatorcontrib>Resnick, Igor</creatorcontrib><creatorcontrib>Ricart, Elena</creatorcontrib><creatorcontrib>Rovira, Montserrat</creatorcontrib><creatorcontrib>Saccardi, Riccardo</creatorcontrib><creatorcontrib>Saif, Muhammad</creatorcontrib><creatorcontrib>Sakellari, Ioanna</creatorcontrib><creatorcontrib>Sharrack, Basil</creatorcontrib><creatorcontrib>Snarski, Emilian</creatorcontrib><creatorcontrib>Scherer, Hans Ulrich</creatorcontrib><creatorcontrib>Sossa, Claudia</creatorcontrib><creatorcontrib>Withers, Barbara</creatorcontrib><creatorcontrib>Wulffraat, Nico</creatorcontrib><creatorcontrib>Zaccara, Eleanora</creatorcontrib><creatorcontrib>Amrolia, Persis</creatorcontrib><creatorcontrib>Ansari, Marc</creatorcontrib><creatorcontrib>Balduzzi, Adriana</creatorcontrib><creatorcontrib>Corbacioglu, Selim</creatorcontrib><creatorcontrib>Dalassier, Arnaud</creatorcontrib><creatorcontrib>Dalle, Jean‐Hugues</creatorcontrib><creatorcontrib>Diaz, Cristina Hereda</creatorcontrib><creatorcontrib>Feuchtinger, Tobias</creatorcontrib><creatorcontrib>Locatelli, Franco</creatorcontrib><creatorcontrib>Lucchini, Giovanna</creatorcontrib><creatorcontrib>Galimard, Jaques‐Emmanuel</creatorcontrib><creatorcontrib>Vincent, Marta Gonzalez</creatorcontrib><creatorcontrib>Handgretinger, Rupert</creatorcontrib><creatorcontrib>Kleinschmidt, Katharina</creatorcontrib><creatorcontrib>Lawitschka, Anita</creatorcontrib><creatorcontrib>Martinez, Antonio Perez</creatorcontrib><creatorcontrib>Peters, Christina</creatorcontrib><creatorcontrib>Rocha, Vanderson</creatorcontrib><creatorcontrib>Ruggeri, Annalisa</creatorcontrib><creatorcontrib>Sedlacek, Petr</creatorcontrib><creatorcontrib>Svec, Peter</creatorcontrib><creatorcontrib>Toporski, Jacek</creatorcontrib><creatorcontrib>Yesilipek, Akif</creatorcontrib><creatorcontrib>The EBMT Autoimmune Diseases (ADWP) and Paediatric Diseases (PDWP) Working Parties</creatorcontrib><title>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</title><title>BRITISH JOURNAL OF HAEMATOLOGY</title><description>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.</description><subject>Autografts</subject><subject>Autoimmune diseases</subject><subject>Children</subject><subject>Disease control</subject><subject>haematopoietic stem cell transplantation</subject><subject>Hematology</subject><subject>Hematopoietic stem cells</subject><subject>Inflammatory diseases</subject><subject>Medical prognosis</subject><subject>Morbidity</subject><subject>paediatric</subject><subject>Pediatrics</subject><subject>Quality of life</subject><subject>Stem cell 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Ltd</general><scope>24P</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>7T5</scope><scope>H94</scope><scope>7X8</scope><scope>ADTPV</scope><scope>AOWAS</scope><scope>D8T</scope><scope>ZZAVC</scope><orcidid>https://orcid.org/0000-0001-6819-3476</orcidid><orcidid>https://orcid.org/0000-0003-1070-8486</orcidid><orcidid>https://orcid.org/0000-0002-0797-8146</orcidid><orcidid>https://orcid.org/0000-0002-6117-5328</orcidid></search><sort><creationdate>202207</creationdate><title>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</title><author>Achini‐Gutzwiller, Federica R. ; Snowden, John A. ; Corbacioglu, Selim ; Greco, Raffaella ; Greco, Raffaella ; Alexander, Tobias ; Snowden, John ; Badoglio, Manuela ; Labopin, Myriam ; Abinun, Mario ; Apte, Shashikant ; Arnold, Renate ; Domenech, Ariadna ; Brierley, Charlotte ; Burman, Joachim ; Castilla‐Llorente, Cristina ; Cooper, Nichola ; Daghia, Giulia ; Daikeler, Thomas ; Papa, Nicoletta ; Vries‐Bouwstra, Jeska ; Farge, Dominique ; Finke, Jurgen ; Hagglund, Hans ; Hawkey, Chris ; Henes, Jörg ; Hiepe, Falk ; Jessop, Helen ; Kiely, David ; Kazmi, Majid ; Kirgizov, Kirill ; Kramer, Ellen ; Mancardi, Gianluigi ; Marjanovic, Zora ; Martin, Roland ; Martin, Thierry ; Ma, David ; Moore, John ; Miller, Paul ; Muraro, Paolo ; Oliveira, Maria‐Carolina ; Polushin, Alexey ; Onida, Francesco ; Simoes, Belinda ; Puyade, Mathieu ; Resnick, Igor ; Ricart, Elena ; Rovira, Montserrat ; Saccardi, Riccardo ; Saif, Muhammad ; Sakellari, Ioanna ; Sharrack, Basil ; Snarski, Emilian ; Scherer, Hans Ulrich ; Sossa, Claudia ; Withers, Barbara ; Wulffraat, Nico ; Zaccara, Eleanora ; Amrolia, Persis ; Ansari, Marc ; Balduzzi, Adriana ; Corbacioglu, Selim ; Dalassier, Arnaud ; Dalle, Jean‐Hugues ; Diaz, Cristina Hereda ; Feuchtinger, Tobias ; Locatelli, Franco ; Lucchini, Giovanna ; Galimard, Jaques‐Emmanuel ; Vincent, Marta Gonzalez ; Handgretinger, Rupert ; Kleinschmidt, Katharina ; Lawitschka, Anita ; Martinez, Antonio Perez ; Peters, Christina ; Rocha, Vanderson ; Ruggeri, Annalisa ; Sedlacek, Petr ; Svec, Peter ; Toporski, Jacek ; Yesilipek, Akif</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c4036-42f8d87d37d92045dc257bf8fdea90c82c28068bfb4c146e09563defc7ed91ba3</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2022</creationdate><topic>Autografts</topic><topic>Autoimmune diseases</topic><topic>Children</topic><topic>Disease control</topic><topic>haematopoietic stem cell transplantation</topic><topic>Hematology</topic><topic>Hematopoietic stem cells</topic><topic>Inflammatory diseases</topic><topic>Medical prognosis</topic><topic>Morbidity</topic><topic>paediatric</topic><topic>Pediatrics</topic><topic>Quality of life</topic><topic>Stem cell transplantation</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Achini‐Gutzwiller, Federica R.</creatorcontrib><creatorcontrib>Snowden, John A.</creatorcontrib><creatorcontrib>Corbacioglu, Selim</creatorcontrib><creatorcontrib>Greco, Raffaella</creatorcontrib><creatorcontrib>Greco, Raffaella</creatorcontrib><creatorcontrib>Alexander, Tobias</creatorcontrib><creatorcontrib>Snowden, John</creatorcontrib><creatorcontrib>Badoglio, Manuela</creatorcontrib><creatorcontrib>Labopin, Myriam</creatorcontrib><creatorcontrib>Abinun, Mario</creatorcontrib><creatorcontrib>Apte, Shashikant</creatorcontrib><creatorcontrib>Arnold, Renate</creatorcontrib><creatorcontrib>Domenech, Ariadna</creatorcontrib><creatorcontrib>Brierley, Charlotte</creatorcontrib><creatorcontrib>Burman, Joachim</creatorcontrib><creatorcontrib>Castilla‐Llorente, Cristina</creatorcontrib><creatorcontrib>Cooper, Nichola</creatorcontrib><creatorcontrib>Daghia, Giulia</creatorcontrib><creatorcontrib>Daikeler, Thomas</creatorcontrib><creatorcontrib>Papa, Nicoletta</creatorcontrib><creatorcontrib>Vries‐Bouwstra, Jeska</creatorcontrib><creatorcontrib>Farge, Dominique</creatorcontrib><creatorcontrib>Finke, Jurgen</creatorcontrib><creatorcontrib>Hagglund, Hans</creatorcontrib><creatorcontrib>Hawkey, Chris</creatorcontrib><creatorcontrib>Henes, Jörg</creatorcontrib><creatorcontrib>Hiepe, Falk</creatorcontrib><creatorcontrib>Jessop, Helen</creatorcontrib><creatorcontrib>Kiely, David</creatorcontrib><creatorcontrib>Kazmi, Majid</creatorcontrib><creatorcontrib>Kirgizov, Kirill</creatorcontrib><creatorcontrib>Kramer, Ellen</creatorcontrib><creatorcontrib>Mancardi, Gianluigi</creatorcontrib><creatorcontrib>Marjanovic, Zora</creatorcontrib><creatorcontrib>Martin, Roland</creatorcontrib><creatorcontrib>Martin, Thierry</creatorcontrib><creatorcontrib>Ma, David</creatorcontrib><creatorcontrib>Moore, John</creatorcontrib><creatorcontrib>Miller, Paul</creatorcontrib><creatorcontrib>Muraro, Paolo</creatorcontrib><creatorcontrib>Oliveira, Maria‐Carolina</creatorcontrib><creatorcontrib>Polushin, Alexey</creatorcontrib><creatorcontrib>Onida, Francesco</creatorcontrib><creatorcontrib>Simoes, Belinda</creatorcontrib><creatorcontrib>Puyade, Mathieu</creatorcontrib><creatorcontrib>Resnick, Igor</creatorcontrib><creatorcontrib>Ricart, Elena</creatorcontrib><creatorcontrib>Rovira, Montserrat</creatorcontrib><creatorcontrib>Saccardi, Riccardo</creatorcontrib><creatorcontrib>Saif, Muhammad</creatorcontrib><creatorcontrib>Sakellari, Ioanna</creatorcontrib><creatorcontrib>Sharrack, Basil</creatorcontrib><creatorcontrib>Snarski, Emilian</creatorcontrib><creatorcontrib>Scherer, Hans Ulrich</creatorcontrib><creatorcontrib>Sossa, Claudia</creatorcontrib><creatorcontrib>Withers, Barbara</creatorcontrib><creatorcontrib>Wulffraat, Nico</creatorcontrib><creatorcontrib>Zaccara, Eleanora</creatorcontrib><creatorcontrib>Amrolia, Persis</creatorcontrib><creatorcontrib>Ansari, Marc</creatorcontrib><creatorcontrib>Balduzzi, Adriana</creatorcontrib><creatorcontrib>Corbacioglu, Selim</creatorcontrib><creatorcontrib>Dalassier, Arnaud</creatorcontrib><creatorcontrib>Dalle, Jean‐Hugues</creatorcontrib><creatorcontrib>Diaz, Cristina Hereda</creatorcontrib><creatorcontrib>Feuchtinger, Tobias</creatorcontrib><creatorcontrib>Locatelli, Franco</creatorcontrib><creatorcontrib>Lucchini, Giovanna</creatorcontrib><creatorcontrib>Galimard, Jaques‐Emmanuel</creatorcontrib><creatorcontrib>Vincent, Marta Gonzalez</creatorcontrib><creatorcontrib>Handgretinger, Rupert</creatorcontrib><creatorcontrib>Kleinschmidt, Katharina</creatorcontrib><creatorcontrib>Lawitschka, Anita</creatorcontrib><creatorcontrib>Martinez, Antonio Perez</creatorcontrib><creatorcontrib>Peters, Christina</creatorcontrib><creatorcontrib>Rocha, Vanderson</creatorcontrib><creatorcontrib>Ruggeri, Annalisa</creatorcontrib><creatorcontrib>Sedlacek, Petr</creatorcontrib><creatorcontrib>Svec, Peter</creatorcontrib><creatorcontrib>Toporski, Jacek</creatorcontrib><creatorcontrib>Yesilipek, Akif</creatorcontrib><creatorcontrib>The EBMT Autoimmune Diseases (ADWP) and Paediatric Diseases (PDWP) Working Parties</creatorcontrib><collection>Wiley Online Library Open Access</collection><collection>CrossRef</collection><collection>Immunology Abstracts</collection><collection>AIDS and Cancer Research Abstracts</collection><collection>MEDLINE - Academic</collection><collection>SwePub</collection><collection>SwePub Articles</collection><collection>SWEPUB Freely available online</collection><collection>SwePub Articles full text</collection><jtitle>BRITISH JOURNAL OF HAEMATOLOGY</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Achini‐Gutzwiller, Federica R.</au><au>Snowden, John A.</au><au>Corbacioglu, Selim</au><au>Greco, Raffaella</au><au>Greco, Raffaella</au><au>Alexander, Tobias</au><au>Snowden, John</au><au>Badoglio, Manuela</au><au>Labopin, Myriam</au><au>Abinun, Mario</au><au>Apte, Shashikant</au><au>Arnold, Renate</au><au>Domenech, Ariadna</au><au>Brierley, Charlotte</au><au>Burman, Joachim</au><au>Castilla‐Llorente, Cristina</au><au>Cooper, Nichola</au><au>Daghia, Giulia</au><au>Daikeler, Thomas</au><au>Papa, Nicoletta</au><au>Vries‐Bouwstra, Jeska</au><au>Farge, Dominique</au><au>Finke, Jurgen</au><au>Hagglund, Hans</au><au>Hawkey, Chris</au><au>Henes, Jörg</au><au>Hiepe, Falk</au><au>Jessop, Helen</au><au>Kiely, David</au><au>Kazmi, Majid</au><au>Kirgizov, Kirill</au><au>Kramer, Ellen</au><au>Mancardi, Gianluigi</au><au>Marjanovic, Zora</au><au>Martin, Roland</au><au>Martin, Thierry</au><au>Ma, David</au><au>Moore, John</au><au>Miller, Paul</au><au>Muraro, Paolo</au><au>Oliveira, Maria‐Carolina</au><au>Polushin, Alexey</au><au>Onida, Francesco</au><au>Simoes, Belinda</au><au>Puyade, Mathieu</au><au>Resnick, Igor</au><au>Ricart, Elena</au><au>Rovira, Montserrat</au><au>Saccardi, Riccardo</au><au>Saif, Muhammad</au><au>Sakellari, Ioanna</au><au>Sharrack, Basil</au><au>Snarski, Emilian</au><au>Scherer, Hans Ulrich</au><au>Sossa, Claudia</au><au>Withers, Barbara</au><au>Wulffraat, Nico</au><au>Zaccara, Eleanora</au><au>Amrolia, Persis</au><au>Ansari, Marc</au><au>Balduzzi, Adriana</au><au>Corbacioglu, Selim</au><au>Dalassier, Arnaud</au><au>Dalle, Jean‐Hugues</au><au>Diaz, Cristina Hereda</au><au>Feuchtinger, Tobias</au><au>Locatelli, Franco</au><au>Lucchini, Giovanna</au><au>Galimard, Jaques‐Emmanuel</au><au>Vincent, Marta Gonzalez</au><au>Handgretinger, Rupert</au><au>Kleinschmidt, Katharina</au><au>Lawitschka, Anita</au><au>Martinez, Antonio Perez</au><au>Peters, Christina</au><au>Rocha, Vanderson</au><au>Ruggeri, Annalisa</au><au>Sedlacek, Petr</au><au>Svec, Peter</au><au>Toporski, Jacek</au><au>Yesilipek, Akif</au><aucorp>The EBMT Autoimmune Diseases (ADWP) and Paediatric Diseases (PDWP) Working Parties</aucorp><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>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</atitle><jtitle>BRITISH JOURNAL OF HAEMATOLOGY</jtitle><date>2022-07</date><risdate>2022</risdate><volume>198</volume><issue>1</issue><spage>24</spage><epage>45</epage><pages>24-45</pages><issn>0007-1048</issn><eissn>1365-2141</eissn><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.</abstract><cop>Oxford</cop><pub>Blackwell Publishing Ltd</pub><doi>10.1111/bjh.18176</doi><tpages>22</tpages><orcidid>https://orcid.org/0000-0001-6819-3476</orcidid><orcidid>https://orcid.org/0000-0003-1070-8486</orcidid><orcidid>https://orcid.org/0000-0002-0797-8146</orcidid><orcidid>https://orcid.org/0000-0002-6117-5328</orcidid><oa>free_for_read</oa></addata></record> |
| fulltext | fulltext |
| identifier | ISSN: 0007-1048 |
| ispartof | BRITISH JOURNAL OF HAEMATOLOGY, 2022-07, Vol.198 (1), p.24-45 |
| issn | 0007-1048 1365-2141 |
| language | eng |
| recordid | cdi_swepub_primary_oai_swepub_ki_se_858422 |
| source | Wiley Free Content; Wiley Online Library Journals Frontfile Complete; SWEPUB Freely available online |
| subjects | Autografts Autoimmune diseases Children Disease control haematopoietic stem cell transplantation Hematology Hematopoietic stem cells Inflammatory diseases Medical prognosis Morbidity paediatric Pediatrics Quality of life Stem cell transplantation |
| title | 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 |
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