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Transplantation of Hematopoietic Stem Cells for Primary Immunodeficiencies in Brazil: Challenges in Treating Rare Diseases in Developing Countries.
Fernandes, Juliana Folloni; Nichele, Samantha; Daudt, Liane E; Tavares, Rita B; Seber, Adriana; Kerbauy, Fábio R; Koliski, Adriana; Loth, Gisele; Vieira, Ana K; Darrigo-Junior, Luiz G; Rocha, Vanderson; Gomes, Alessandra A; Colturato, Vergílio; Mantovani, Luiz F; Ribeiro, Andreza F; Ribeiro, Lisandro L; Kuwahara, Cilmara; Rodrigues, Ana L M; Zecchin, Victor G; Costa-Carvalho, Beatriz T; Carneiro-Sampaio, Magda; Condino-Neto, Antonio; Fasth, Anders; Gennery, Andrew; Pasquini, Ricardo; Hamerschlak, Nelson; Bonfim, Carmem.
Afiliación
  • Fernandes JF; Instituto da Criança - Hospital das Clínicas da Universidade de São Paulo, São Paulo, Brazil. j.fernandes@hc.fm.usp.br.
  • Nichele S; Hospital Israelita Albert Einstein, São Paulo, SP, Brazil. j.fernandes@hc.fm.usp.br.
  • Daudt LE; Hospital de Clínicas da Universidade Federal do Paraná, Curitiba, Brazil.
  • Tavares RB; Hospital de Clínicas de Porto Alegre - Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil.
  • Seber A; Instituto Nacional do Cancer, Rio de Janeiro, Brazil.
  • Kerbauy FR; Instituto de Oncologia Pediátrica, Universidade Federal de São Paulo, São Paulo, Brazil.
  • Koliski A; Hospital Israelita Albert Einstein, São Paulo, SP, Brazil.
  • Loth G; Hospital de Clínicas da Universidade Federal do Paraná, Curitiba, Brazil.
  • Vieira AK; Hospital de Clínicas da Universidade Federal do Paraná, Curitiba, Brazil.
  • Darrigo-Junior LG; Hospital de Clínicas da Universidade Federal de Minas Gerais, Belo Horizonte, Brazil.
  • Rocha V; Hospital das Clínicas da Universidade de São Paulo, Ribeirão Preto, Brazil.
  • Gomes AA; Hospital Sírio Libanês, São Paulo, Brazil.
  • Colturato V; Churchill Hospital, University of Oxford, NHS-BT, Oxford, UK.
  • Mantovani LF; Hospital Sírio Libanês, São Paulo, Brazil.
  • Ribeiro AF; Hospital Amaral Carvalho, Jaú, São Paulo, Brazil.
  • Ribeiro LL; Instituto da Criança - Hospital das Clínicas da Universidade de São Paulo, São Paulo, Brazil.
  • Kuwahara C; Hospital Israelita Albert Einstein, São Paulo, SP, Brazil.
  • Rodrigues ALM; Hospital Israelita Albert Einstein, São Paulo, SP, Brazil.
  • Zecchin VG; Hospital de Clínicas da Universidade Federal do Paraná, Curitiba, Brazil.
  • Costa-Carvalho BT; Hospital Nossa Senhora das Graças, Curitiba, Brazil.
  • Carneiro-Sampaio M; Hospital Infantil Pequeno Príncipe, Curitiba, Brazil.
  • Condino-Neto A; Hospital Infantil Pequeno Príncipe, Curitiba, Brazil.
  • Fasth A; Instituto de Oncologia Pediátrica, Universidade Federal de São Paulo, São Paulo, Brazil.
  • Gennery A; Grupo Brasileiro de Imunodeficiências, BRAGID, São Paulo, Brazil.
  • Pasquini R; Instituto da Criança - Hospital das Clínicas da Universidade de São Paulo, São Paulo, Brazil.
  • Hamerschlak N; Consórcio Brasileiro de Centros de Referência em Imunodeficiências Primárias, CoBID, São Paulo, Brazil.
  • Bonfim C; Department of Immunology, Institute of Biomedical Sciences, and Institute of Tropical Medicine, University of São Paulo, São Paulo, SP, Brazil.
J Clin Immunol ; 38(8): 917-926, 2018 11.
Article en En | MEDLINE | ID: mdl-30470982
The 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.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Trasplante de Células Madre Hematopoyéticas / Enfermedades Raras / Enfermedad Injerto contra Huésped / Síndromes de Inmunodeficiencia Tipo de estudio: Clinical_trials / Diagnostic_studies Límite: Female / Humans / Infant / Male / Newborn País/Región como asunto: America do sul / Brasil Idioma: En Revista: J Clin Immunol Año: 2018 Tipo del documento: Article País de afiliación: Brasil Pais de publicación: Países Bajos
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Trasplante de Células Madre Hematopoyéticas / Enfermedades Raras / Enfermedad Injerto contra Huésped / Síndromes de Inmunodeficiencia Tipo de estudio: Clinical_trials / Diagnostic_studies Límite: Female / Humans / Infant / Male / Newborn País/Región como asunto: America do sul / Brasil Idioma: En Revista: J Clin Immunol Año: 2018 Tipo del documento: Article País de afiliación: Brasil Pais de publicación: Países Bajos