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

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Fältnamn	Indikatorer	Metadata
000		04072naa a2200601 4500
001		oai:gup.ub.gu.se/274255
003		SwePub
008		240528s2018 \| \|\|\|\|\|\|\|\|\|\|\|000 \|\|eng\|
024	7	a https://gup.ub.gu.se/publication/2742552 URI
024	7	a https://doi.org/10.1007/s10875-018-0564-12 DOI
040		a (SwePub)gu
041		a eng
042		9 SwePub
072	7	a ref2 swepub-contenttype
072	7	a art2 swepub-publicationtype
100	1	a Fernandes, Juliana Folloni4 aut
245	1 0	a Transplantation of Hematopoietic Stem Cells for Primary Immunodeficiencies in Brazil: Challenges in Treating Rare Diseases in Developing Countries.
264		c 2018-11-24
264	1	b Springer Science and Business Media LLC,c 2018
520		a 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 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.
650	7	a MEDICIN OCH HÄLSOVETENSKAPx Klinisk medicinx Pediatrik0 (SwePub)302212 hsv//swe
650	7	a MEDICAL AND HEALTH SCIENCESx Clinical Medicinex Pediatrics0 (SwePub)302212 hsv//eng
700	1	a Nichele, Samantha4 aut
700	1	a Daudt, Liane E4 aut
700	1	a Tavares, Rita B4 aut
700	1	a Seber, Adriana4 aut
700	1	a Kerbauy, Fábio R4 aut
700	1	a Koliski, Adriana4 aut
700	1	a Loth, Gisele4 aut
700	1	a Vieira, Ana K4 aut
700	1	a Darrigo-Junior, Luiz G4 aut
700	1	a Rocha, Vanderson4 aut
700	1	a Gomes, Alessandra A4 aut
700	1	a Colturato, Vergílio4 aut
700	1	a Mantovani, Luiz F4 aut
700	1	a Ribeiro, Andreza F4 aut
700	1	a Ribeiro, Lisandro L4 aut
700	1	a Kuwahara, Cilmara4 aut
700	1	a Rodrigues, Ana L M4 aut
700	1	a Zecchin, Victor G4 aut
700	1	a Costa-Carvalho, Beatriz T4 aut
700	1	a Carneiro-Sampaio, Magda4 aut
700	1	a Condino-Neto, Antonio4 aut
700	1	a Fasth, Anders,d 1945u Gothenburg University,Göteborgs universitet,Institutionen för kliniska vetenskaper, Avdelningen för pediatrik,Institute of Clinical Sciences, Department of Pediatrics4 aut0 (Swepub:gu)xfasan
700	1	a Gennery, Andrew4 aut
700	1	a Pasquini, Ricardo4 aut
700	1	a Hamerschlak, Nelson4 aut
700	1	a Bonfim, Carmem4 aut
710	2	a Göteborgs universitetb Institutionen för kliniska vetenskaper, Avdelningen för pediatrik4 org
773	0	t Journal of clinical immunologyd : Springer Science and Business Media LLCg 38:8, s. 917-926q 38:8<917-926x 1573-2592x 0271-9142
856	4	u https://lume.ufrgs.br/bitstream/10183/204336/1/001107016.pdf
856	4 8	u https://gup.ub.gu.se/publication/274255
856	4 8	u https://doi.org/10.1007/s10875-018-0564-1

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