Long-Term Outcomes of Hematopoietic Stem Cell Transplantation for Severe Treatment-Resistant Autoimmune Cytopenia in Children

Marco Rabusin; J. A. Snowden; Paul Veys; Pierre Quartier; Jean Hughes Dalle; Catharina Dhooge; Paolo Di Bartolomeo; Maria Gonzalez-Vicent; Brenda Gibson; Arturo Iriondo; Herbert Juergens; Igor Lisukov; Chiara Messina; Valerie Mialou; Colin Graham Steward; Christian Urban; Marleen Renard; Nagua Giurici; Christina Peters; Manuela Badoglio; Luca Ronfani; Giorgio Dini; Dominique Farge; Riccardo Saccardi

doi:10.1016/j.bbmt.2012.12.008

Long-Term Outcomes of Hematopoietic Stem Cell Transplantation for Severe Treatment-Resistant Autoimmune Cytopenia in Children

Marco Rabusin, J. A. Snowden, Paul Veys, Pierre Quartier, Jean Hughes Dalle, Catharina Dhooge, Paolo Di Bartolomeo, Maria Gonzalez-Vicent, Brenda Gibson, Arturo Iriondo, Herbert Juergens, Igor Lisukov, Chiara Messina, Valerie Mialou, Colin Graham Steward, Christian Urban, Marleen Renard, Nagua Giurici, Christina Peters, Manuela BadoglioLuca Ronfani, Giorgio Dini, Dominique Farge, Riccardo Saccardi

IRCCS pediatrico Burlo Garofolo

Research output: Contribution to journal › Article › peer-review

Abstract

We analyzed the long-term outcomes of pediatric patients registered in the European Group for Blood and Marrow Transplantation database who underwent hematopoietic stem cell transplantation (HSCT) for severe treatment refractory autoimmune cytopenia. With a median follow-up of 100 months, event-free survival was 54% overall, with no significant difference between allogeneic HSCT (n = 15) and autologous HSCT (n = 7) recipients (58% versus 42%; P = .50). Despite a trend toward failure of response or relapse after autologous HSCT compared with allogeneic HSCT, the difference was not significant (43% versus 13%; P = .30). Treatment-related mortality was high in both HSCT groups (29% and 16%; P = .09). Based on the limited numbers of subjects in this retrospective analysis, both allogeneic and autologous HSCT may induce complete and persistent responses in approximately one-half of pediatric patients with severe refractory autoimmune cytopenia, although treatment-related toxicity is high.

Original language	English
Pages (from-to)	666-669
Number of pages	4
Journal	Biology of Blood and Marrow Transplantation
Volume	19
Issue number	4
DOIs	https://doi.org/10.1016/j.bbmt.2012.12.008
Publication status	Published - Apr 2013

Keywords

Allogeneic transplant
Childhood
Evans syndrome
HSCT
Refractory cytopenia

ASJC Scopus subject areas

Transplantation
Hematology

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10.1016/j.bbmt.2012.12.008

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Rabusin, M., Snowden, J. A., Veys, P., Quartier, P., Dalle, J. H., Dhooge, C., Di Bartolomeo, P., Gonzalez-Vicent, M., Gibson, B., Iriondo, A., Juergens, H., Lisukov, I., Messina, C., Mialou, V., Steward, C. G., Urban, C., Renard, M., Giurici, N., Peters, C., ... Saccardi, R. (2013). Long-Term Outcomes of Hematopoietic Stem Cell Transplantation for Severe Treatment-Resistant Autoimmune Cytopenia in Children. Biology of Blood and Marrow Transplantation, 19(4), 666-669. https://doi.org/10.1016/j.bbmt.2012.12.008

Rabusin, M, Snowden, JA, Veys, P, Quartier, P, Dalle, JH, Dhooge, C, Di Bartolomeo, P, Gonzalez-Vicent, M, Gibson, B, Iriondo, A, Juergens, H, Lisukov, I, Messina, C, Mialou, V, Steward, CG, Urban, C, Renard, M, Giurici, N, Peters, C, Badoglio, M, Ronfani, L, Dini, G, Farge, D & Saccardi, R 2013, 'Long-Term Outcomes of Hematopoietic Stem Cell Transplantation for Severe Treatment-Resistant Autoimmune Cytopenia in Children', Biology of Blood and Marrow Transplantation, vol. 19, no. 4, pp. 666-669. https://doi.org/10.1016/j.bbmt.2012.12.008

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abstract = "We analyzed the long-term outcomes of pediatric patients registered in the European Group for Blood and Marrow Transplantation database who underwent hematopoietic stem cell transplantation (HSCT) for severe treatment refractory autoimmune cytopenia. With a median follow-up of 100 months, event-free survival was 54% overall, with no significant difference between allogeneic HSCT (n = 15) and autologous HSCT (n = 7) recipients (58% versus 42%; P = .50). Despite a trend toward failure of response or relapse after autologous HSCT compared with allogeneic HSCT, the difference was not significant (43% versus 13%; P = .30). Treatment-related mortality was high in both HSCT groups (29% and 16%; P = .09). Based on the limited numbers of subjects in this retrospective analysis, both allogeneic and autologous HSCT may induce complete and persistent responses in approximately one-half of pediatric patients with severe refractory autoimmune cytopenia, although treatment-related toxicity is high.",

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author = "Marco Rabusin and Snowden, {J. A.} and Paul Veys and Pierre Quartier and Dalle, {Jean Hughes} and Catharina Dhooge and {Di Bartolomeo}, Paolo and Maria Gonzalez-Vicent and Brenda Gibson and Arturo Iriondo and Herbert Juergens and Igor Lisukov and Chiara Messina and Valerie Mialou and Steward, {Colin Graham} and Christian Urban and Marleen Renard and Nagua Giurici and Christina Peters and Manuela Badoglio and Luca Ronfani and Giorgio Dini and Dominique Farge and Riccardo Saccardi",

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AU - Rabusin, Marco

AU - Snowden, J. A.

AU - Veys, Paul

AU - Quartier, Pierre

AU - Dalle, Jean Hughes

AU - Dhooge, Catharina

AU - Di Bartolomeo, Paolo

AU - Gonzalez-Vicent, Maria

AU - Gibson, Brenda

AU - Iriondo, Arturo

AU - Juergens, Herbert

AU - Lisukov, Igor

AU - Messina, Chiara

AU - Mialou, Valerie

AU - Steward, Colin Graham

AU - Urban, Christian

AU - Renard, Marleen

AU - Giurici, Nagua

AU - Peters, Christina

AU - Badoglio, Manuela

AU - Ronfani, Luca

AU - Dini, Giorgio

AU - Farge, Dominique

AU - Saccardi, Riccardo

PY - 2013/4

Y1 - 2013/4

N2 - We analyzed the long-term outcomes of pediatric patients registered in the European Group for Blood and Marrow Transplantation database who underwent hematopoietic stem cell transplantation (HSCT) for severe treatment refractory autoimmune cytopenia. With a median follow-up of 100 months, event-free survival was 54% overall, with no significant difference between allogeneic HSCT (n = 15) and autologous HSCT (n = 7) recipients (58% versus 42%; P = .50). Despite a trend toward failure of response or relapse after autologous HSCT compared with allogeneic HSCT, the difference was not significant (43% versus 13%; P = .30). Treatment-related mortality was high in both HSCT groups (29% and 16%; P = .09). Based on the limited numbers of subjects in this retrospective analysis, both allogeneic and autologous HSCT may induce complete and persistent responses in approximately one-half of pediatric patients with severe refractory autoimmune cytopenia, although treatment-related toxicity is high.

AB - We analyzed the long-term outcomes of pediatric patients registered in the European Group for Blood and Marrow Transplantation database who underwent hematopoietic stem cell transplantation (HSCT) for severe treatment refractory autoimmune cytopenia. With a median follow-up of 100 months, event-free survival was 54% overall, with no significant difference between allogeneic HSCT (n = 15) and autologous HSCT (n = 7) recipients (58% versus 42%; P = .50). Despite a trend toward failure of response or relapse after autologous HSCT compared with allogeneic HSCT, the difference was not significant (43% versus 13%; P = .30). Treatment-related mortality was high in both HSCT groups (29% and 16%; P = .09). Based on the limited numbers of subjects in this retrospective analysis, both allogeneic and autologous HSCT may induce complete and persistent responses in approximately one-half of pediatric patients with severe refractory autoimmune cytopenia, although treatment-related toxicity is high.

KW - Allogeneic transplant

KW - Childhood

KW - Evans syndrome

KW - HSCT

KW - Refractory cytopenia

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Long-Term Outcomes of Hematopoietic Stem Cell Transplantation for Severe Treatment-Resistant Autoimmune Cytopenia in Children

Abstract

Keywords

ASJC Scopus subject areas

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