Hematopoietic cell transplantation in severe combined immunodeficiency: The SCETIDE 2006-2014 European cohort.

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Fältnamn	Indikatorer	Metadata
000		05887naa a2201021 4500
001		oai:gup.ub.gu.se/310492
003		SwePub
008		240528s2022 \| \|\|\|\|\|\|\|\|\|\|\|000 \|\|eng\|
009		oai:prod.swepub.kib.ki.se:150362734
024	7	a https://gup.ub.gu.se/publication/3104922 URI
024	7	a https://doi.org/10.1016/j.jaci.2021.10.0172 DOI
024	7	a http://kipublications.ki.se/Default.aspx?queryparsed=id:1503627342 URI
040		a (SwePub)gud (SwePub)ki
041		a eng
042		9 SwePub
072	7	a ref2 swepub-contenttype
072	7	a art2 swepub-publicationtype
100	1	a Lankester, Arjan C4 aut
245	1 0	a Hematopoietic cell transplantation in severe combined immunodeficiency: The SCETIDE 2006-2014 European cohort.
264	1	b Elsevier BV,c 2022
520		a Hematopoietic stem cell transplantation (HSCT) represents a curative treatment for patients with severe combined immunodeficiency (SCID), a group of monogenic immune disorders with an otherwise fatal outcome.We performed a comprehensive multicenter analysis of genotype-specific HSCT outcome, including detailed analysis of immune reconstitution (IR) and the predictive value for clinical outcome.HSCT outcome was studied in 338 patients with genetically confirmed SCID who underwent transplantation in 2006-2014 and who were registered in the SCETIDE registry. In a representative subgroup of 152 patients, data on IR and long-term clinical outcome were analyzed.Two-year OS was similar with matched family and unrelated donors and better than mismatched donor HSCT (P<.001). The 2-year event-free survival (EFS) was similar in matched and mismatched unrelated donor and less favorable in mismatched related donor (MMRD) HSCT (P< .001). Genetic subgroups did not differ in 2-year OS (P= .1) and EFS (P=.073). In multivariate analysis, pretransplantation infections and use of MMRDs were associated with less favorable OS and EFS. With a median follow-up of 6.2 years (range, 2.0-11.8 years), 73 of 152 patients in the IR cohort were alive and well without Ig dependency. IL-2 receptor gamma chain/Janus kinase 3/IL-7 receptor-deficient SCID, myeloablative conditioning, matched donor HSCT, and naive CD4 T lymphocytes >0.5×10e3/μL at+1 year were identified as independent predictors of favorable clinical and immunologic outcome.Recent advances in HSCT in SCID patients have resulted in improved OS and EFS in all genotypes and donor types. To achieve a favorable long-term outcome, treatment strategies should aim for optimal naive CD4 T lymphocyte regeneration.
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
650	7	a MEDICIN OCH HÄLSOVETENSKAPx Medicinska och farmaceutiska grundvetenskaperx Immunologi inom det medicinska området0 (SwePub)301102 hsv//swe
650	7	a MEDICAL AND HEALTH SCIENCESx Basic Medicinex Immunology in the medical area0 (SwePub)301102 hsv//eng
700	1	a Neven, Benedicte4 aut
700	1	a Mahlaoui, Nizar4 aut
700	1	a von Asmuth, Erik G J4 aut
700	1	a Courteille, Virginie4 aut
700	1	a Alligon, Mikael4 aut
700	1	a Albert, Michael H4 aut
700	1	a Serra, Isabelle Badell4 aut
700	1	a Bader, Peter4 aut
700	1	a Balashov, Dmitry4 aut
700	1	a Beier, Rita4 aut
700	1	a Bertrand, Yves4 aut
700	1	a Blanche, Stephane4 aut
700	1	a Bordon, Victoria4 aut
700	1	a Bredius, Robbert G4 aut
700	1	a Cant, Andrew4 aut
700	1	a Cavazzana, Marina4 aut
700	1	a Diaz-de-Heredia, Cristina4 aut
700	1	a Dogu, Figen4 aut
700	1	a Ehlert, Karoline4 aut
700	1	a Entz-Werle, Natacha4 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 Ferrua, Francesca4 aut
700	1	a Ferster, Alina4 aut
700	1	a Formankova, Renata4 aut
700	1	a Friedrich, Wilhelm4 aut
700	1	a Gonzalez-Vicent, Marta4 aut
700	1	a Gozdzik, Jolanta4 aut
700	1	a Güngör, Tayfun4 aut
700	1	a Hoenig, Manfred4 aut
700	1	a Ikinciogullari, Aydan4 aut
700	1	a Kalwak, Krzysztof4 aut
700	1	a Kansoy, Savas4 aut
700	1	a Kupesiz, Alphan4 aut
700	1	a Lanfranchi, Arnalda4 aut
700	1	a Lindemans, Caroline A4 aut
700	1	a Meisel, Roland4 aut
700	1	a Michel, Gerard4 aut
700	1	a Miranda, Nuno A A4 aut
700	1	a Moraleda, Jose4 aut
700	1	a Moshous, Despina4 aut
700	1	a Pichler, Herbert4 aut
700	1	a Rao, Kanchan4 aut
700	1	a Sedlacek, Petr4 aut
700	1	a Slatter, Mary4 aut
700	1	a Soncini, Elena4 aut
700	1	a Speckmann, Carsten4 aut
700	1	a Sundin, Mikaelu Karolinska Institutet4 aut
700	1	a Toren, Amos4 aut
700	1	a Vettenranta, Kim4 aut
700	1	a Worth, Austen4 aut
700	1	a Yeşilipek, Mehmet A4 aut
700	1	a Zecca, Marco4 aut
700	1	a Porta, Fulvio4 aut
700	1	a Schulz, Ansgar4 aut
700	1	a Veys, Paul4 aut
700	1	a Fischer, Alain4 aut
700	1	a Gennery, Andrew R4 aut
710	2	a Göteborgs universitetb Institutionen för kliniska vetenskaper, Avdelningen för pediatrik4 org
773	0	t The Journal of allergy and clinical immunologyd : Elsevier BVg 149:5q 149:5x 1097-6825x 0091-6749
856	4	u http://www.jacionline.org/article/S0091674921016298/pdf
856	4 8	u https://gup.ub.gu.se/publication/310492
856	4 8	u https://doi.org/10.1016/j.jaci.2021.10.017
856	4 8	u http://kipublications.ki.se/Default.aspx?queryparsed=id:150362734

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Av författaren/redakt...: Lankester, Arjan ...; Neven, Benedicte; Mahlaoui, Nizar; von Asmuth, Erik ...; Courteille, Virg ...; Alligon, Mikael; visa fler...; Albert, Michael ...; Serra, Isabelle ...; Bader, Peter; Balashov, Dmitry; Beier, Rita; Bertrand, Yves; Blanche, Stephan ...; Bordon, Victoria; Bredius, Robbert ...; Cant, Andrew; Cavazzana, Marin ...; Diaz-de-Heredia, ...; Dogu, Figen; Ehlert, Karoline; Entz-Werle, Nata ...; Fasth, Anders, 1 ...; Ferrua, Francesc ...; Ferster, Alina; Formankova, Rena ...; Friedrich, Wilhe ...; Gonzalez-Vicent, ...; Gozdzik, Jolanta; Güngör, Tayfun; Hoenig, Manfred; Ikinciogullari, ...; Kalwak, Krzyszto ...; Kansoy, Savas; Kupesiz, Alphan; Lanfranchi, Arna ...; Lindemans, Carol ...; Meisel, Roland; Michel, Gerard; Miranda, Nuno A ...; Moraleda, Jose; Moshous, Despina; Pichler, Herbert; Rao, Kanchan; Sedlacek, Petr; Slatter, Mary; Soncini, Elena; Speckmann, Carst ...; Sundin, Mikael; Toren, Amos; Vettenranta, Kim; Worth, Austen; Yeşilipek, Mehme ...; Zecca, Marco; Porta, Fulvio; Schulz, Ansgar; Veys, Paul; Fischer, Alain; Gennery, Andrew ...; visa färre...

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