Transplantation of Hematopoietic Stem Cells for Primary Immunodeficiencies in Brazil: Challenges in Treating Rare Diseases in Developing Countries

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dc.contributorSistema FMUSP-HC: Faculdade de Medicina da Universidade de São Paulo (FMUSP) e Hospital das Clínicas da FMUSP
dc.contributor.authorFERNANDES, Juliana Folloni
dc.contributor.authorNICHELE, Samantha
dc.contributor.authorDAUDT, Liane E.
dc.contributor.authorTAVARES, Rita B.
dc.contributor.authorSEBER, Adriana
dc.contributor.authorKERBAUY, Fabio R.
dc.contributor.authorKOLISKI, Adriana
dc.contributor.authorLOTH, Gisele
dc.contributor.authorVIEIRA, Ana K.
dc.contributor.authorDARRIGO-JUNIOR, Luiz G.
dc.contributor.authorROCHA, Vanderson
dc.contributor.authorGOMES, Alessandra A.
dc.contributor.authorCOLTURATO, Vergilio
dc.contributor.authorMANTOVANI, Luiz F.
dc.contributor.authorRIBEIRO, Andreza F.
dc.contributor.authorRIBEIRO, Lisandro L.
dc.contributor.authorKUWAHARA, Cilmara
dc.contributor.authorRODRIGUES, Ana L. M.
dc.contributor.authorZECCHIN, Victor G.
dc.contributor.authorCOSTA-CARVALHO, Beatriz T.
dc.contributor.authorCARNEIRO-SAMPAIO, Magda
dc.contributor.authorCONDINO-NETO, Antonio
dc.contributor.authorFASTH, Anders
dc.contributor.authorGENNERY, Andrew
dc.contributor.authorPASQUINI, Ricardo
dc.contributor.authorHAMERSCHLAK, Nelson
dc.contributor.authorBONFIM, Carmem
dc.date.accessioned2019-01-17T13:35:19Z
dc.date.available2019-01-17T13:35:19Z
dc.date.issued2018
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 22months, 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.eng
dc.description.indexMEDLINEeng
dc.identifier.citationJOURNAL OF CLINICAL IMMUNOLOGY, v.38, n.8, p.917-926, 2018
dc.identifier.doi10.1007/s10875-018-0564-1
dc.identifier.eissn1573-2592
dc.identifier.issn0271-9142
dc.identifier.urihttps://observatorio.fm.usp.br/handle/OPI/30014
dc.language.isoeng
dc.publisherSPRINGER/PLENUM PUBLISHERSeng
dc.relation.ispartofJournal of Clinical Immunology
dc.rightsrestrictedAccesseng
dc.rights.holderCopyright SPRINGER/PLENUM PUBLISHERSeng
dc.subjectPrimary immunodeficiencieseng
dc.subjectstem cell transplantationeng
dc.subjectsevere combined immunodeficiencyeng
dc.subjectWiskott-Aldrich syndromeeng
dc.subjectLatin Americaeng
dc.subject.otherchronic granulomatous-diseaseeng
dc.subject.otherbone-marrow-transplantationeng
dc.subject.otherwiskott-aldrich-syndromeeng
dc.subject.otherlatin-american societyeng
dc.subject.otherversus-host-diseaseeng
dc.subject.otherlong-term survivaleng
dc.subject.otherposttransplant cyclophosphamideeng
dc.subject.otherhaploidentical bmteng
dc.subject.otheraplastic-anemiaeng
dc.subject.otherinborn-errorseng
dc.subject.wosImmunologyeng
dc.titleTransplantation of Hematopoietic Stem Cells for Primary Immunodeficiencies in Brazil: Challenges in Treating Rare Diseases in Developing Countrieseng
dc.typearticleeng
dc.type.categoryoriginal articleeng
dc.type.versionpublishedVersioneng
dspace.entity.typePublication
hcfmusp.affiliation.countrySuécia
hcfmusp.affiliation.countryInglaterra
hcfmusp.affiliation.countryisogb
hcfmusp.affiliation.countryisose
hcfmusp.author.externalNICHELE, Samantha:Univ Fed Parana, Hosp Clin, Curitiba, Parana, Brazil
hcfmusp.author.externalDAUDT, Liane E.:Univ Fed Rio Grande do Sul, Hosp Clin Porto Alegre, Porto Alegre, RS, Brazil
hcfmusp.author.externalTAVARES, Rita B.:Inst Nacl Canc, Rio De Janeiro, Brazil
hcfmusp.author.externalSEBER, Adriana:Univ Fed Sao Paulo, Inst Oncol Pediat, Sao Paulo, Brazil
hcfmusp.author.externalKERBAUY, Fabio R.:Hosp Israelita Albert Einstein, Sao Paulo, SP, Brazil
hcfmusp.author.externalKOLISKI, Adriana:Univ Fed Parana, Hosp Clin, Curitiba, Parana, Brazil
hcfmusp.author.externalVIEIRA, Ana K.:Univ Fed Minas Gerais, Hosp Clin, Belo Horizonte, MG, Brazil
hcfmusp.author.externalDARRIGO-JUNIOR, Luiz G.:Univ Sao Paulo, Hosp Clin, Ribeirao Preto, Brazil
hcfmusp.author.externalROCHA, Vanderson:Hosp Sirio Libanes, Sao Paulo, Brazil; Univ Oxford, Churchill Hosp, NHS BT, Oxford, England
hcfmusp.author.externalGOMES, Alessandra A.:Hosp Sirio Libanes, Sao Paulo, Brazil
hcfmusp.author.externalCOLTURATO, Vergilio:Hosp Amaral Carvalho, Jau, SP, Brazil
hcfmusp.author.externalRIBEIRO, Andreza F.:Hosp Israelita Albert Einstein, Sao Paulo, SP, Brazil
hcfmusp.author.externalRIBEIRO, Lisandro L.:Univ Fed Parana, Hosp Clin, Curitiba, Parana, Brazil; Hosp Nossa Senhora Gracas, Curitiba, Parana, Brazil
hcfmusp.author.externalKUWAHARA, Cilmara:Hosp Infantil Pequeno Principe, Curitiba, Parana, Brazil
hcfmusp.author.externalRODRIGUES, Ana L. M.:Hosp Infantil Pequeno Principe, Curitiba, Parana, Brazil
hcfmusp.author.externalZECCHIN, Victor G.:Univ Fed Sao Paulo, Inst Oncol Pediat, Sao Paulo, Brazil
hcfmusp.author.externalCOSTA-CARVALHO, Beatriz T.:BRAGID, Grp Brasileiro Imunodeficiencias, Sao Paulo, Brazil
hcfmusp.author.externalFASTH, Anders:Univ Gothenburg, Dept Pediat, Gothenburg, Sweden
hcfmusp.author.externalGENNERY, Andrew:Great North Childrens Hosp, Newcastle Upon Tyne, Tyne & Wear, England
hcfmusp.author.externalPASQUINI, Ricardo:Univ Fed Parana, Hosp Clin, Curitiba, Parana, Brazil; Hosp Nossa Senhora Gracas, Curitiba, Parana, Brazil
hcfmusp.author.externalHAMERSCHLAK, Nelson:Hosp Israelita Albert Einstein, Sao Paulo, SP, Brazil
hcfmusp.author.externalBONFIM, Carmem:Univ Fed Parana, Hosp Clin, Curitiba, Parana, Brazil; Hosp Nossa Senhora Gracas, Curitiba, Parana, Brazil; Hosp Infantil Pequeno Principe, Curitiba, Parana, Brazil
hcfmusp.citation.scopus14
hcfmusp.contributor.author-fmusphcJULIANA FOLLONI FERNANDES
hcfmusp.contributor.author-fmusphcLUIZ FERNANDO ALVES LIMA MANTOVANI
hcfmusp.contributor.author-fmusphcMAGDA MARIA SALES CARNEIRO SAMPAIO
hcfmusp.contributor.author-fmusphcANTONIO CONDINO NETO
hcfmusp.description.beginpage917
hcfmusp.description.endpage926
hcfmusp.description.issue8
hcfmusp.description.volume38
hcfmusp.origemWOS
hcfmusp.origem.pubmed30470982
hcfmusp.origem.scopus2-s2.0-85057133423
hcfmusp.origem.wosWOS:000453283000010
hcfmusp.publisher.cityNEW YORKeng
hcfmusp.publisher.countryUSAeng
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