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Publication Details
AFRICAN RESEARCH NEXUS
SHINING A SPOTLIGHT ON AFRICAN RESEARCH
immunology and microbiology
Long-term outcomes of 176 patients with X-linked hyper-IgM syndrome treated with or without hematopoietic cell transplantation
Journal of Allergy and Clinical Immunology, Volume 139, No. 4, Year 2017
Notification
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Description
Background 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. © 2016 American Academy of Allergy, Asthma & Immunology
Authors & Co-Authors
de la Morena, Maite Teresa
United States, Dallas
Ut Southwestern Medical Center
Torgerson, Troy Robert
United States, Seattle
University of Washington
Cabral‑Marques, Otavio
Germany, Lubeck
Universität zu Lübeck
Slatter, Mary Anne
United Kingdom, Newcastle
Royal Victoria Infirmary
Aghamohammadi, Asgahr
Iran, Tehran
Research Center for Immunodeficiencies
Chandra, Sharat
United States, Cincinnati
Cincinnati Children's Hospital Medical Center
Murguía-Favela, Luis
Canada, Toronto
Hospital for Sick Children University of Toronto
Bonilla, Francisco A.
United States, Boston
Boston Children's Hospital
Kanariou, Maria G.
Unknown Affiliation
Kuo, Caroline Y.
United States, Los Angeles
University of California, Los Angeles
Dvorak, Christopher C.
United States, San Francisco
University of California, San Francisco
Meyts, Isabelle
Belgium, Leuven
Ku Leuven– University Hospital Leuven
Chen, Karin
United States, Salt Lake City
University of Utah School of Medicine
Kobrynski, Lisa J.
United States, Atlanta
Emory University
Kapoor, Neena
United States, Los Angeles
Keck School of Medicine of Usc
Richter, Darko
Croatia, Zagreb
Kbc Zagreb
Farmaki, Evangelia
Greece, Thessaloniki
Ippokration Hospital Thessaloniki
Speckmann, Carsten
Germany, Freiburg Im Breisgau
Universitätsklinikum Freiburg
Español, Theresa
Spain, Barcelona
Hospital Universitari Vall D'hebron
Shcherbina, Anna Yu
Unknown Affiliation
Hanson, Imelda Celine Guerra
United States, Houston
Texas Children's Hospital
Litzman, Jiří
Czech Republic, Brno
Masaryk University
Routes, John M.
United States, Wauwatosa
Children's Hospital of Wisconsin Wauwatosa
Wong, Melanie
Australia, Sydney
The Children's Hospital at Westmead
Fuleihan, Ramsay L.
United States, Chicago
Children's Memorial Hospital
Seneviratne, Suranjith Luke
United Kingdom, London
The Royal Free Hospital
Janda, Aleś
Czech Republic, Prague
Fakultní Nemocnice V Motole
Bezrodnik, Liliana
Argentina, Buenos Aires
Hospital de Ninos Ricardo Gutierrez
Seger, Reinhard A.
Unknown Affiliation
Edgar, J. David M.
Unknown Affiliation
Chou, Janet S.
United States, Boston
Boston Children's Hospital
Abbott, Jordan Keith
United States, Denver
National Jewish Health
Montfrans, Jorisvan M.(Joris)
Netherlands, Utrecht
University Medical Center Utrecht
González-Granado, Luis Ignacio
Spain, Madrid
Instituto de Investigación Hospital 12 de Octubre
Kütükçüler, Necil
Turkey, Izmir
Ege University Medical School
Gray, Paul Edgar A.
Australia, Sydney
Sydney Children's Hospital, Randwick
Seminario, Gisela Analia
Argentina, Buenos Aires
Hospital de Ninos Ricardo Gutierrez
Pašić, Srdjan S.
Serbia, Belgrade
Mother and Child Health Institute, Belgrade, rs
Aquino, Victor M.
United States, Dallas
Ut Southwestern Medical Center
Abolhassani, Hassan
Iran, Tehran
Research Center for Immunodeficiencies
Dorsey, Morna J.
United States, San Francisco
University of California, San Francisco
Cunningham Rundles, Charlotte H.
United States, New York
The Mount Sinai Hospital
Sleasman, John William
United States, Durham
Duke University
Costa-Carvalho, Beatriz Tavares
Brazil, Sao Paulo
Universidade Federal de São Paulo
Condino‑Neto, Antonio A.
Brazil, Sao Paulo
Universidade de São Paulo
Grunebaum, Eyal
Canada, Toronto
Hospital for Sick Children University of Toronto
Chapel, Helen M.
United Kingdom, Oxford
University of Oxford
Ochs, Hans Dietrich
United States, Seattle
University of Washington
Filipovich, Alexandra Hult
United States, Cincinnati
Cincinnati Children's Hospital Medical Center
Cowan, Morton J.
United States, San Francisco
University of California, San Francisco
Gennery, Andrew R.
United Kingdom, Newcastle
Royal Victoria Infirmary
Cant, Andrew James
United Kingdom, Newcastle
Royal Victoria Infirmary
Notarangelo, Luigi Daniele
United States, Bethesda
National Institutes of Health Nih
Roifman, Chaim M.
Canada, Toronto
Hospital for Sick Children University of Toronto
Statistics
Citations: 95
Authors: 54
Affiliations: 42
Identifiers
Doi:
10.1016/j.jaci.2016.07.039
ISSN:
00916749
Research Areas
Environmental
Study Design
Cohort Study