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dc.contributor.authorFernandes, Juliana Follonipt_BR
dc.contributor.authorBonfim, Carmem Maria Salespt_BR
dc.contributor.authorDaudt, Liane Estevespt_BR
dc.date.accessioned2020-01-16T04:08:36Zpt_BR
dc.date.issued2018pt_BR
dc.identifier.issn1573-2592pt_BR
dc.identifier.urihttp://hdl.handle.net/10183/204336pt_BR
dc.description.abstractThe results of hematopoietic stem cell transplant (HSCT) for primary immunodeficiency diseases (PID) have been improving over time. Unfortunately, developing countries do not experience the same results. This first report of Brazilian experience of HSCT for PID describes the development and results in the field. We included data from transplants in 221 patients, performed at 11 centers which participated in the Brazilian collaborative group, from July 1990 to December 2015. The majority of transplants were concentrated in one center (n = 123). The median age at HSCT was 22 months, and the most common diseases were severe combined immunodeficiency (SCID) (n = 67) and Wiskott-Aldrich syndrome (WAS) (n = 67). Only 15 patients received unconditioned transplants. Cumulative incidence of GVHD grades II to IV was 23%, and GVHD grades III to IV was 10%. The 5-year overall survival was 71.6%. WAS patients had better survival compared to other diseases. Most deaths (n = 53) occurred in the first year after transplantation mainly due to infection (55%) and GVHD (13%). Although transplant for PID patients in Brazil has evolved since its beginning, we still face some challenges like delayed diagnosis and referral, severe infections before transplant, a limited number of transplant centers with expertise, and resources for more advanced techniques. Measures like newborn screening for SCID may hasten the diagnosis and ameliorate patients’ conditions at the moment of transplant.en
dc.format.mimetypeapplication/pdfpt_BR
dc.language.isoengpt_BR
dc.relation.ispartofJournal of clinical immunology. Amsterdam. vol. 38, no. 8 (Nov. 2018), p. 917-926pt_BR
dc.rightsOpen Accessen
dc.subjectPrimary immunodeficienciesen
dc.subjectSíndromes de imunodeficiênciapt_BR
dc.subjectStem cell transplantationen
dc.subjectTransplante de células-troncopt_BR
dc.subjectSevere combined immunodeficiencyen
dc.subjectSíndrome de Wiskott-Aldrichpt_BR
dc.subjectWiskott-Aldrich syndromeen
dc.subjectLatin Americaen
dc.titleTransplantation of hematopoietic stem cells for primary immunodeficiencies in Brazil : challenges in treating rare diseases in developing countriespt_BR
dc.typeArtigo de periódicopt_BR
dc.identifier.nrb001107016pt_BR
dc.type.originEstrangeiropt_BR


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