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Long-term outcomes of 176 patients with X-linked hyper-IgM syndrome treated with or without hematopoietic cell transplantation
MT. de la Morena, D. Leonard, TR. Torgerson, O. Cabral-Marques, M. Slatter, A. Aghamohammadi, S. Chandra, L. Murguia-Favela, FA. Bonilla, M. Kanariou, R. Damrongwatanasuk, CY. Kuo, CC. Dvorak, I. Meyts, K. Chen, L. Kobrynski, N. Kapoor, D....
Jazyk angličtina Země Spojené státy americké
Typ dokumentu časopisecké články, multicentrická studie, pozorovací studie
- MeSH
- čas MeSH
- dítě MeSH
- dospělí MeSH
- imunodeficience s hyper-IgM mortalita terapie MeSH
- Kaplanův-Meierův odhad MeSH
- kohortové studie MeSH
- kojenec MeSH
- lidé středního věku MeSH
- lidé MeSH
- mladiství MeSH
- mladý dospělý MeSH
- následné studie MeSH
- předškolní dítě MeSH
- proporcionální rizikové modely MeSH
- retrospektivní studie MeSH
- transplantace hematopoetických kmenových buněk mortalita MeSH
- Check Tag
- dítě MeSH
- dospělí MeSH
- kojenec MeSH
- lidé středního věku MeSH
- lidé MeSH
- mladiství MeSH
- mladý dospělý MeSH
- mužské pohlaví MeSH
- předškolní dítě MeSH
- ženské pohlaví MeSH
- Publikační typ
- časopisecké články MeSH
- multicentrická studie MeSH
- pozorovací studie MeSH
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.
Ann and Robert H Lurie Children's Hospital of Chicago Chicago Ill
Baylor Texas Children's Hospital Houston Tex
Boston Children's Hospital Boston Mass
Children's Hospital at Westmead Sydney Australia
Children's Hospital Boston Boston Mass
Children's Hospital Los Angeles Keck School of Medicine Los Angeles Calif
Children's Hospital of Philadelphia Philadelphia Pa
Children's Hospital of Wisconsin Milwaukee Wis
Cincinnati Children's Hospital Medical Center Cincinnati Ohio
Department of Immunology Institute of Biomedical Sciences University of São Paulo São Paulo Brazil
Department of Rheumatology University of Lübeck Lübeck Germany
Ege University Faculty of Medicine Izmir Turkey
Geffen SOM at David Geffen School of Medicine at UCLA Los Angeles Calif
Hospital de Niños Dr Ricardo Gutierrez Buenos Aires Argentina
Hospital for Sick Children Toronto Ontario Canada
Hospital Vall d'Hebron Barcelona Spain
Ippokration General Hospital Thessaloniki Greece
Laboratory of Host Defenses NIAID National Institutes of Health Bethesda Md
Memorial Sloan Kettering Cancer Center New York NY
Mother and Child Health Institute Belgrade Serbia
Mount Sinai Hospital New York NY
National Jewish Health Denver Colo
Regional Immunology Service Belfast United Kingdom
Research and Clinical Center for Pediatric Hematology Oncology and Immunology Moscow Russia
Royal Free Hospital London United Kingdom
Royal Victoria Infirmary Newcastle upon Tyne United Kingdom
Saint Louis University St Louis Mo
Sophia Children's Hospital Athens Athens Greece
Sydney Children's Hospital Randwick Australia
UC San Francisco San Francisco Calif
University Hospital Center Zagreb Croatia
University Hospital Motol Prague Czech Republic
University Hospitals Leuven Leuven Belgium
University of Oxford Oxford United Kingdom
University of South Florida All Childrens FL St Petersburg Fla
University of Utah School of Medicine Salt Lake City Utah
University of Washington and Seattle Children's Research Institute Seattle Wash
Citace poskytuje Crossref.org
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- $a de la Morena, M Teresa $u University of Texas Southwestern Medical Center and Children's Medical Center, Children's Health, Dallas, Tex. Electronic address: maite.delamorena@utsouthwestern.edu.
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- $a Long-term outcomes of 176 patients with X-linked hyper-IgM syndrome treated with or without hematopoietic cell transplantation / $c MT. de la Morena, D. Leonard, TR. Torgerson, O. Cabral-Marques, M. Slatter, A. Aghamohammadi, S. Chandra, L. Murguia-Favela, FA. Bonilla, M. Kanariou, R. Damrongwatanasuk, CY. Kuo, CC. Dvorak, I. Meyts, K. Chen, L. Kobrynski, N. Kapoor, D. Richter, D. DiGiovanni, F. Dhalla, E. Farmaki, C. Speckmann, T. Español, A. Shcherbina, IC. Hanson, J. Litzman, JM. Routes, M. Wong, R. Fuleihan, SL. Seneviratne, TN. Small, A. Janda, L. Bezrodnik, R. Seger, AG. Raccio, JD. Edgar, J. Chou, JK. Abbott, J. van Montfrans, LI. González-Granado, N. Bunin, N. Kutukculer, P. Gray, G. Seminario, S. Pasic, V. Aquino, C. Wysocki, H. Abolhassani, M. Dorsey, C. Cunningham-Rundles, AP. Knutsen, J. Sleasman, BT. Costa Carvalho, A. Condino-Neto, E. Grunebaum, H. Chapel, HD. Ochs, A. Filipovich, M. Cowan, A. Gennery, A. Cant, LD. Notarangelo, CM. Roifman,
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- $a 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.
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