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

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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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

Long-Term Outcomes of Hematopoietic Stem Cell Transplantation for Severe Treatment-Resistant Autoimmune Cytopenia in Children. / Rabusin, Marco; Snowden, J. A.; Veys, Paul; Quartier, Pierre; Dalle, Jean Hughes; Dhooge, Catharina; Di Bartolomeo, Paolo; Gonzalez-Vicent, Maria; Gibson, Brenda; Iriondo, Arturo; Juergens, Herbert; Lisukov, Igor; Messina, Chiara; Mialou, Valerie; Steward, Colin Graham; Urban, Christian; Renard, Marleen; Giurici, Nagua; Peters, Christina; Badoglio, Manuela; Ronfani, Luca; Dini, Giorgio; Farge, Dominique; Saccardi, Riccardo.

In: Biology of Blood and Marrow Transplantation, Vol. 19, No. 4, 04.2013, p. 666-669.

Research output: Contribution to journal › Article

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

Rabusin, Marco ; Snowden, J. A. ; Veys, Paul ; Quartier, Pierre ; Dalle, Jean Hughes ; Dhooge, Catharina ; Di Bartolomeo, Paolo ; Gonzalez-Vicent, Maria ; Gibson, Brenda ; Iriondo, Arturo ; Juergens, Herbert ; Lisukov, Igor ; Messina, Chiara ; Mialou, Valerie ; Steward, Colin Graham ; Urban, Christian ; Renard, Marleen ; Giurici, Nagua ; Peters, Christina ; Badoglio, Manuela ; Ronfani, Luca ; Dini, Giorgio ; Farge, Dominique ; Saccardi, Riccardo. / Long-Term Outcomes of Hematopoietic Stem Cell Transplantation for Severe Treatment-Resistant Autoimmune Cytopenia in Children. In: Biology of Blood and Marrow Transplantation. 2013 ; Vol. 19, No. 4. pp. 666-669.

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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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AU - Di Bartolomeo, Paolo

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AU - Peters, Christina

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AU - Ronfani, Luca

AU - Dini, Giorgio

AU - Farge, Dominique

AU - Saccardi, Riccardo

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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.

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