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

Research output: Contribution to journalArticle

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 languageEnglish
Pages (from-to)666-669
Number of pages4
JournalBiology of Blood and Marrow Transplantation
Volume19
Issue number4
DOIs
Publication statusPublished - 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