Long-term outcomes of 176 patients with X-linked hyper-IgM syndrome treated with or without hematopoietic cell transplantation

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dc.contributor.authorde la Morena, M. Teresa
dc.contributor.authorLeonard, David
dc.contributor.authorTorgerson, Troy R.
dc.contributor.authorCabral-Marques, Otavio
dc.contributor.authorSlatter, Mary
dc.contributor.authorAghamohammadi, Asghar
dc.contributor.authorChandra, Sharat
dc.contributor.authorMurguia-Favela, Luis
dc.contributor.authorBonilla, Francisco A.
dc.contributor.authorKanariou, Maria
dc.contributor.authorDamrongwatanasuk, Rongras
dc.contributor.authorKuo, Caroline Y.
dc.contributor.authorDvorak, Christopher C.
dc.contributor.authorMeyts, Isabelle
dc.contributor.authorChen, Karin
dc.contributor.authorKobrynski, Lisa
dc.contributor.authorKapoor, Neena
dc.contributor.authorRichter, Darko
dc.contributor.authorDiGiovanni, Daniela
dc.contributor.authorDhalla, Fatima
dc.contributor.authorFarmaki, Evangelia
dc.contributor.authorSpeckmann, Carsten
dc.contributor.authorEspanol, Teresa
dc.contributor.authorShcherbina, Anna
dc.contributor.authorHanson, Imelda Celine
dc.contributor.authorLitzman, Jiri
dc.contributor.authorRoutes, John M.
dc.contributor.authorWong, Melanie
dc.contributor.authorFuleihan, Ramsay
dc.contributor.authorSeneviratne, Suranjith L.
dc.contributor.authorSmall, Trudy N.
dc.contributor.authorJanda, Ales
dc.contributor.authorBezrodnik, Liliana
dc.contributor.authorSeger, Reinhard
dc.contributor.authorRaccio, Andrea Gomez
dc.contributor.authorEdgar, J. David M.
dc.contributor.authorChou, Janet
dc.contributor.authorAbbott, Jordan K.
dc.contributor.authorvan Montfrans, Joris
dc.contributor.authorGonzalez-Granado, Luis Ignacio
dc.contributor.authorBunin, Nancy
dc.contributor.authorKutukculer, Necil
dc.contributor.authorGray, Paul
dc.contributor.authorSeminario, Gisela
dc.contributor.authorPasic, Srdjan
dc.contributor.authorAquino, Victor
dc.contributor.authorWysocki, Christian
dc.contributor.authorAbolhassani, Hassan
dc.contributor.authorDorsey, Morna
dc.contributor.authorCunningham-Rundles, Charlotte
dc.contributor.authorKnutsen, Alan P.
dc.contributor.authorSleasman, John
dc.contributor.authorCarvalho, Beatriz Tavares Costa
dc.contributor.authorCondino-Neto, Antonio
dc.contributor.authorGrunebaum, Eyal
dc.contributor.authorChapel, Helen
dc.contributor.authorOchs, Hans D.
dc.contributor.authorFilipovich, Alexandra
dc.contributor.authorCowan, Mort
dc.contributor.authorGennery, Andrew
dc.contributor.authorCant, Andrew
dc.contributor.authorNotarangelo, Luigi D.
dc.contributor.authorRoifman, Chaim M.
dc.date.accessioned2019-10-27T11:09:31Z
dc.date.available2019-10-27T11:09:31Z
dc.date.issued2017
dc.departmentEge Üniversitesien_US
dc.description.abstractBackground: X-linked hyper-IgM syndrome (XHIGM) is a primary immunodeficiency with high morbidity and mortality compared with those seen in healthy subjects. Hematopoietic cell transplantation (HCT) has been considered a curative therapy, but the procedure has inherent complications and might not be available for all patients. Objectives: We sought to collect data on the clinical presentation, treatment, and follow-up of a large sample of patients with XHIGM to (1) compare long-term overall survival and general well-being of patients treated with or without HCT along with clinical factors associated with mortality and (2) summarize clinical practice and risk factors in the subgroup of patients treated with HCT. Methods: Physicians caring for patients with primary immunodeficiency diseases were identified through the Jeffrey Modell Foundation, United States Immunodeficiency Network, Latin American Society for Immunodeficiency, and Primary Immune Deficiency Treatment Consortium. Data were collected with a Research Electronic Data Capture Web application. Survival from time of diagnosis or transplantation was estimated by using the Kaplan-Meier method compared with log-rank tests and modeled by using proportional hazards regression. Results: Twenty-eight clinical sites provided data on 189 patients given a diagnosis of XHIGM between 1964 and 2013; 176 had valid follow-up and vital status information. Sixty-seven (38%) patients received HCT. The average follow-up time was 8.5 +/- 7.2 years (range, 0.1-36.2 years). No difference in overall survival was observed between patients treated with or without HCT (P = .671). However, risk associated with HCT decreased for diagnosis years 1987-1995; the hazard ratio was significantly less than 1 for diagnosis years 1995-1999. Liver disease was a significant predictor of overall survival (hazard ratio, 4.9; 95% confidence limits, 2.2-10.8; P < .001). Among survivors, those treated with HCT had higher median Karnofsky/Lansky scores than those treated without HCT (P < .001). Among patients receiving HCT, 27 (40%) had graft-versus-host disease, and most deaths occurred within 1 year of transplantation. Conclusion: No difference in survival was observed between patients treated with or without HCT across all diagnosis years (1964-2013). However, survivors treated with HCT experienced somewhat greater well-being, and hazards associated with HCT decreased, reaching levels of significantly less risk in the late 1990s. Among patients treated with HCT, treatment at an early age is associated with improved survival. Optimism remains guarded as additional evidence accumulates.en_US
dc.description.sponsorshipJeffrey Modell Foundation; National Institutes of Health Office of Rare Diseases, National Center for Advancing Translational Sciences and National, Institute of Allergy and Infectious Disease [U54 AI 082973, R13AI094943]en_US
dc.description.sponsorshipSupported by a grant from Jeffrey Modell Foundation (to M.d.l.M.). The Primary Immune Deficiency Treatment Consortium (PIDTC) is supported by the National Institutes of Health Office of Rare Diseases, National Center for Advancing Translational Sciences and National, Institute of Allergy and Infectious Disease grants U54 AI 082973 and R13AI094943.en_US
dc.identifier.doi10.1016/j.jaci.2016.07.039en_US
dc.identifier.endpage1292en_US
dc.identifier.issn0091-6749
dc.identifier.issn1097-6825
dc.identifier.issue4en_US
dc.identifier.pmid27697500en_US
dc.identifier.scopusqualityQ1en_US
dc.identifier.startpage1282en_US
dc.identifier.urihttps://doi.org/10.1016/j.jaci.2016.07.039
dc.identifier.urihttps://hdl.handle.net/11454/32295
dc.identifier.volume139en_US
dc.identifier.wosWOS:000398771800023en_US
dc.identifier.wosqualityQ1en_US
dc.indekslendigikaynakWeb of Scienceen_US
dc.indekslendigikaynakScopusen_US
dc.indekslendigikaynakPubMeden_US
dc.language.isoenen_US
dc.publisherMosby-Elsevieren_US
dc.relation.ispartofJournal of Allergy and Clinical Immunologyen_US
dc.relation.publicationcategoryMakale - Uluslararası Hakemli Dergi - Kurum Öğretim Elemanıen_US
dc.rightsinfo:eu-repo/semantics/openAccessen_US
dc.subjectX-linked hyper-IgM syndromeen_US
dc.subjectCD40 liganden_US
dc.subjecthematopoietic cell transplantationen_US
dc.subjectdefects in class-switch recombinationen_US
dc.subjectlong-term outcomesen_US
dc.subjectprimary immunodeficiencyen_US
dc.subjectKarnofsky/Lansky scoresen_US
dc.titleLong-term outcomes of 176 patients with X-linked hyper-IgM syndrome treated with or without hematopoietic cell transplantationen_US
dc.typeArticleen_US

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