MORENA, M. Teresa de la, David LEONARD, Troy R. TORGERSON, Otavio CABRAL-MARQUES, Mary SLATTER, Asghar AGHAMOHAMMADI, Sharat CHANDRA, Luis MURGUIA-FAVELA, Francisco A. BONILLA, Maria KANARIOU, Rongras DAMRONGWATANASUK, Caroline Y. KUO, Chistopher C. DVORAK, Isabelle MEYTS, Karin CHEN, Lisa KOBRYNSKI, Neena KAPOOR, Darko RICHTER, Daniela DIGIOVANNI, Fatima DHALLA, Evangelia FARMAKI, Carsten SPECKMANN, Teresa ESPANOL, Anna SHCHERBINA, Imelda HANSON, Jiří LITZMAN, John M. ROUTES, Melanie WONG, Ramsay FULEIHAN, Suranjith SENEVIRATNE, Trudy N. SMALL, Ales JANDA, Liliana BEZRODNIK, Reinhard SEGER, Andrea Gomez RACCIO, J. David M. EDGAR, Janet CHOU, Jordan K. ABBOTT, Joris van MONTFRANS, Luis Ignacio GONZALEZ-GRANADO, Nancy BUNIN, Necil KUTUKCULER, Paul GRAY, Gisela SEMINARIO, Srdjan PASIC, Victor AQUINO, Christian WYSOCKI, Hassan ABOLHASSANI, Morna DORSEY, Charlotte CUNNINGHAM-RUNDLES, Alan KNUTSEN, John SLEASMAN, Beatriz Tavares Costa CARVALHO, Antonio CONDINO-NETO, Eyal GRUNEBAUM, Helen CHAPEL, Hans D. OCHS, Alexandra FILIPOVICH, Mort COWAN, Andrew GENNERY, Andrew CANT, Luigi D. NOTARANGELO a Chaim M. ROIFMAN. 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. New York: Mosby-Elsevier, 2017, roč. 139, č. 4, s. 1282-1292. ISSN 0091-6749. Dostupné z: https://dx.doi.org/10.1016/j.jaci.2016.07.039. |
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@article{1379720, author = {Morena, M. Teresa de la and Leonard, David and Torgerson, Troy R. and CabralandMarques, Otavio and Slatter, Mary and Aghamohammadi, Asghar and Chandra, Sharat and MurguiaandFavela, Luis and Bonilla, Francisco A. and Kanariou, Maria and Damrongwatanasuk, Rongras and Kuo, Caroline Y. and Dvorak, Chistopher C. and Meyts, Isabelle and Chen, Karin and Kobrynski, Lisa and Kapoor, Neena and Richter, Darko and DiGiovanni, Daniela and Dhalla, Fatima and Farmaki, Evangelia and Speckmann, Carsten and Espanol, Teresa and Shcherbina, Anna and Hanson, Imelda and Litzman, Jiří and Routes, John M. and Wong, Melanie and Fuleihan, Ramsay and Seneviratne, Suranjith and Small, Trudy N. and Janda, Ales and Bezrodnik, Liliana and Seger, Reinhard and Raccio, Andrea Gomez and Edgar, J. David M. and Chou, Janet and Abbott, Jordan K. and Montfrans, Joris van and GonzalezandGranado, Luis Ignacio and Bunin, Nancy and Kutukculer, Necil and Gray, Paul and Seminario, Gisela and Pasic, Srdjan and Aquino, Victor and Wysocki, Christian and Abolhassani, Hassan and Dorsey, Morna and CunninghamandRundles, Charlotte and Knutsen, Alan and Sleasman, John and Carvalho, Beatriz Tavares Costa and CondinoandNeto, Antonio and Grunebaum, Eyal and Chapel, Helen and Ochs, Hans D. and Filipovich, Alexandra and Cowan, Mort and Gennery, Andrew and Cant, Andrew and Notarangelo, Luigi D. and Roifman, Chaim M.}, article_location = {New York}, article_number = {4}, doi = {http://dx.doi.org/10.1016/j.jaci.2016.07.039}, keywords = {X-linked hyper-IgM syndrome; CD40 ligand; hematopoietic cell transplantation; defects in class-switch recombination; long-term outcomes; primary immunodeficiency; Karnofsky/Lansky scores}, language = {eng}, issn = {0091-6749}, journal = {Journal of allergy and clinical immunology}, title = {Long-term outcomes of 176 patients with X-linked hyper-IgM syndrome treated with or without hematopoietic cell transplantation}, volume = {139}, year = {2017} }
TY - JOUR ID - 1379720 AU - Morena, M. Teresa de la - Leonard, David - Torgerson, Troy R. - Cabral-Marques, Otavio - Slatter, Mary - Aghamohammadi, Asghar - Chandra, Sharat - Murguia-Favela, Luis - Bonilla, Francisco A. - Kanariou, Maria - Damrongwatanasuk, Rongras - Kuo, Caroline Y. - Dvorak, Chistopher C. - Meyts, Isabelle - Chen, Karin - Kobrynski, Lisa - Kapoor, Neena - Richter, Darko - DiGiovanni, Daniela - Dhalla, Fatima - Farmaki, Evangelia - Speckmann, Carsten - Espanol, Teresa - Shcherbina, Anna - Hanson, Imelda - Litzman, Jiří - Routes, John M. - Wong, Melanie - Fuleihan, Ramsay - Seneviratne, Suranjith - Small, Trudy N. - Janda, Ales - Bezrodnik, Liliana - Seger, Reinhard - Raccio, Andrea Gomez - Edgar, J. David M. - Chou, Janet - Abbott, Jordan K. - Montfrans, Joris van - Gonzalez-Granado, Luis Ignacio - Bunin, Nancy - Kutukculer, Necil - Gray, Paul - Seminario, Gisela - Pasic, Srdjan - Aquino, Victor - Wysocki, Christian - Abolhassani, Hassan - Dorsey, Morna - Cunningham-Rundles, Charlotte - Knutsen, Alan - Sleasman, John - Carvalho, Beatriz Tavares Costa - Condino-Neto, Antonio - Grunebaum, Eyal - Chapel, Helen - Ochs, Hans D. - Filipovich, Alexandra - Cowan, Mort - Gennery, Andrew - Cant, Andrew - Notarangelo, Luigi D. - Roifman, Chaim M. PY - 2017 TI - Long-term outcomes of 176 patients with X-linked hyper-IgM syndrome treated with or without hematopoietic cell transplantation JF - Journal of allergy and clinical immunology VL - 139 IS - 4 SP - 1282-1292 EP - 1282-1292 PB - Mosby-Elsevier SN - 00916749 KW - X-linked hyper-IgM syndrome KW - CD40 ligand KW - hematopoietic cell transplantation KW - defects in class-switch recombination KW - long-term outcomes KW - primary immunodeficiency KW - Karnofsky/Lansky scores N2 - 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. ER -
MORENA, M. Teresa de la, David LEONARD, Troy R. TORGERSON, Otavio CABRAL-MARQUES, Mary SLATTER, Asghar AGHAMOHAMMADI, Sharat CHANDRA, Luis MURGUIA-FAVELA, Francisco A. BONILLA, Maria KANARIOU, Rongras DAMRONGWATANASUK, Caroline Y. KUO, Chistopher C. DVORAK, Isabelle MEYTS, Karin CHEN, Lisa KOBRYNSKI, Neena KAPOOR, Darko RICHTER, Daniela DIGIOVANNI, Fatima DHALLA, Evangelia FARMAKI, Carsten SPECKMANN, Teresa ESPANOL, Anna SHCHERBINA, Imelda HANSON, Jiří LITZMAN, John M. ROUTES, Melanie WONG, Ramsay FULEIHAN, Suranjith SENEVIRATNE, Trudy N. SMALL, Ales JANDA, Liliana BEZRODNIK, Reinhard SEGER, Andrea Gomez RACCIO, J. David M. EDGAR, Janet CHOU, Jordan K. ABBOTT, Joris van MONTFRANS, Luis Ignacio GONZALEZ-GRANADO, Nancy BUNIN, Necil KUTUKCULER, Paul GRAY, Gisela SEMINARIO, Srdjan PASIC, Victor AQUINO, Christian WYSOCKI, Hassan ABOLHASSANI, Morna DORSEY, Charlotte CUNNINGHAM-RUNDLES, Alan KNUTSEN, John SLEASMAN, Beatriz Tavares Costa CARVALHO, Antonio CONDINO-NETO, Eyal GRUNEBAUM, Helen CHAPEL, Hans D. OCHS, Alexandra FILIPOVICH, Mort COWAN, Andrew GENNERY, Andrew CANT, Luigi D. NOTARANGELO a Chaim M. ROIFMAN. Long-term outcomes of 176 patients with X-linked hyper-IgM syndrome treated with or without hematopoietic cell transplantation. \textit{Journal of allergy and clinical immunology}. New York: Mosby-Elsevier, 2017, roč.~139, č.~4, s.~1282-1292. ISSN~0091-6749. Dostupné z: https://dx.doi.org/10.1016/j.jaci.2016.07.039.
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