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

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dc.citation.issue4
dc.citation.volume139
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 [UNIFESP]
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.coverageNew York
dc.date.accessioned2020-07-17T14:02:34Z
dc.date.available2020-07-17T14:02:34Z
dc.date.issued2017
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 2013en
dc.description.abstract176 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 1987-1995en
dc.description.abstractthe hazard ratio was significantly less than 1 for diagnosis years 1995-1999. Liver disease was a significant predictor of overall survival (hazard ratio, 4.9en
dc.description.abstract95% confidence limits, 2.2-10.8en
dc.description.abstractP < .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
dc.description.affiliationUniv Texas Southwestern Med Ctr Dallas, 5323 Harry Hines Blvd, Dallas, TX 75390 USA
dc.description.affiliationChildrens Hlth, Childrens Med Ctr, Dallas, TX USA
dc.description.affiliationUniv Washington, Seattle, WA 98195 USA
dc.description.affiliationSeattle Childrens Res Inst, Seattle, WA USA
dc.description.affiliationUniv Lubeck, Dept Rheumatol, Lubeck, Germany
dc.description.affiliationRoyal Victoria Infirm, Newcastle Upon Tyne, Tyne & Wear, England
dc.description.affiliationUniv Tehran Med Sci, Childrens Med Ctr, Res Ctr Immunodeficiencies, Pediat Ctr Excellence, Tehran, Iran
dc.description.affiliationCincinnati Childrens Hosp Med Ctr, Cincinnati, OH 45229 USA
dc.description.affiliationHosp Sick Children, 555 Univ Ave, Toronto, ON M5G 1X8, Canada
dc.description.affiliationBoston Childrens Hosp, Boston, MA USA
dc.description.affiliationSophia Childrens Hosp Athens, Athens, AL USA
dc.description.affiliationUniv S Florida, All Childrens FL, St Petersburg, FL 33701 USA
dc.description.affiliationUniv Calif Los Angeles, David Geffen Sch Med, Geffen SOM, Los Angeles, CA 90095 USA
dc.description.affiliationUC San Francisco, San Francisco, CA USA
dc.description.affiliationUniv Hosp Leuven, Leuven, Germany
dc.description.affiliationUniv Utah, Sch Med, Salt Lake City, UT USA
dc.description.affiliationEmory Univ, Atlanta, GA 30322 USA
dc.description.affiliationChildrens Hosp Los Angeles, Keck Sch Med, Los Angeles, CA 90027 USA
dc.description.affiliationUniv Hosp Ctr, Zagreb, Croatia
dc.description.affiliationHosp Ninos Dr Ricardo Gutierrez, Buenos Aires, DF, Argentina
dc.description.affiliationUniv Oxford, Oxford OX1 2JD, England
dc.description.affiliationIppokration Gen Hosp, Thessaloniki, Greece
dc.description.affiliationCtr Chron Immunodeficiency Univ Med Ctr, Dept Pediat & Adolescent Med, Freiburg, Germany
dc.description.affiliationHosp Valle De Hebron, Barcelona, Spain
dc.description.affiliationRes & Clin Ctr Pediat Hematol Oncol & Immunol, Moscow, Russia
dc.description.affiliationBaylor Texas Childrens Hosp, Houston, TX USA
dc.description.affiliationMasaryk Univ, Dept Clin Immunol & Allergol, St Annes Univ Hosp Brno, Fac Med, Brno, Czech Republic
dc.description.affiliationChildrens Hosp Wisconsin, Milwaukee, WI 53201 USA
dc.description.affiliationChildrens Hosp Westmead, Sydney, NSW, Australia
dc.description.affiliationAnn & Robert H Lurie Childrens Hosp Chicago, Chicago, IL USA
dc.description.affiliationRoyal Free Hosp, London, England
dc.description.affiliationMem Sloan Kettering Canc Ctr, 1275 York Ave, New York, NY 10021 USA
dc.description.affiliationUniv Hosp Motol, Prague, Czech Republic
dc.description.affiliationReg Immunol Serv, Belfast, Antrim, North Ireland
dc.description.affiliationChildrens Hosp Boston, Boston, MA USA
dc.description.affiliationNatl Jewish Hlth, Denver, CO USA
dc.description.affiliationUMC Utrecht, Div Pediat Pediat Immunol & Infectieziekten, Wilhelmina Childrens Hosp, Utrecht, Netherlands
dc.description.affiliationInst Invest Hosp 12 Octubre, Unidad Immunodeficiencias Primarias, Madrid, Spain
dc.description.affiliationInst Invest Hosp 12 Octubre, Unidad Hemato & Oncol Pediat, Madrid, Spain
dc.description.affiliationChildrens Hosp Philadelphia, Philadelphia, PA 19104 USA
dc.description.affiliationEge Univ, Fac Med, Izmir, Turkey
dc.description.affiliationSydney Childrens Hosp, Randwick, NSW, Australia
dc.description.affiliationMother & Child Hlth Inst Serbia, Belgrade, Serbia
dc.description.affiliationUC San Francisco, San Francisco, CA USA
dc.description.affiliationMt Sinai Hosp, New York, NY 10029 USA
dc.description.affiliationSt Louis Univ, St Louis, MO 63103 USA
dc.description.affiliationDuke Univ, Durham, NC USA
dc.description.affiliationUniv Fed Sao Paulo, Dept Pediat, Div Allergy Immunol & Rheumatol, Sao Paulo, Brazil
dc.description.affiliationUniv Sao Paulo, Inst Biomed Sci, Dept Immunol, BR-05508 Sao Paulo, Brazil
dc.description.affiliationNIAID, Lab Host Def, NIH, 9000 Rockville Pike, Bethesda, MD 20892 USA
dc.description.affiliationUnifespUniv Fed Sao Paulo, Dept Pediat, Div Allergy Immunol & Rheumatol, Sao Paulo, Brazil
dc.description.sourceWeb of Science
dc.description.sponsorshipJeffrey Modell Foundation
dc.description.sponsorshipNational Institutes of Health Office of Rare Diseases, National Center for Advancing Translational Sciences and National, Institute of Allergy and Infectious Disease
dc.description.sponsorshipIDNational Institutes of Health Office of Rare Diseases, National Center for Advancing Translational Sciences and National, Institute of Allergy and Infectious Disease: U54 AI 082973
dc.description.sponsorshipIDNational Institutes of Health Office of Rare Diseases, National Center for Advancing Translational Sciences and National, Institute of Allergy and Infectious Disease: R13AI094943
dc.format.extent1282-1292
dc.identifierhttp://dx.doi.org/10.1016/j.jaci.2016.07.039
dc.identifier.citationJournal Of Allergy And Clinical Immunology. New York, v. 139, n. 4, p. 1282-1292, 2017.
dc.identifier.doi10.1016/j.jaci.2016.07.039
dc.identifier.issn0091-6749
dc.identifier.urihttps://repositorio.unifesp.br/handle/11600/54866
dc.identifier.wosWOS:000398771800023
dc.language.isoeng
dc.publisherMosby-Elsevier
dc.relation.ispartofJournal Of Allergy And Clinical Immunology
dc.rightsinfo:eu-repo/semantics/openAccess
dc.subjectX-linked hyper-IgM syndromeen
dc.subjectCD40 liganden
dc.subjecthematopoietic cell transplantationen
dc.subjectdefects in class-switch recombinationen
dc.subjectlong-term outcomesen
dc.subjectprimary immunodeficiencyen
dc.subjectKarnofsky/Lansky scoresen
dc.titleLong-term outcomes of 176 patients with X-linked hyper-IgM syndrome treated with or without hematopoietic cell transplantationen
dc.typeinfo:eu-repo/semantics/article
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