Clinical features and hematopoietic stem cell transplantations for CD40 ligand deficiency in Japan
Copyright © 2015 American Academy of Allergy, Asthma & Immunology. Published by Elsevier Inc. All rights reserved..
BACKGROUND: The long-term outcome of X-linked hyper-IgM syndrome (XHIM) caused by mutations in CD40LG is poor, and the only curative treatment is hematopoietic stem cell transplantation (HSCT).
OBJECTIVE: We sought to determine the clinical features and factors affecting outcomes in patients with XHIM.
METHODS: We enrolled and retrospectively analyzed data from 56 Japanese patients with XHIM, including 29 patients who received HSCT.
RESULTS: The long-term survival rate was poor in those not undergoing HSCT (overall survival rate at 40 years of age, 28.2%). The overall survival rate of patients undergoing HSCT (n = 29) was significantly higher than that of those not undergoing HSCT (n = 27, P = .0231). Moreover, event-free and disease-free survival rates were significantly greater in patients 5 years old or younger at the time of transplantation (n = 14) than in older patients (n = 15).
CONCLUSION: On the basis of these results, we concluded that HSCT improved the outcomes of patients with XHIM and that an age of 5 years or younger was optimal for the timing of HSCT because persistent infections and severe organ damage were frequently observed in patients older than 6 years.
| Medienart: |
E-Artikel |
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| Erscheinungsjahr: |
2015 |
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| Erschienen: |
2015 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:136 |
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| Enthalten in: |
The Journal of allergy and clinical immunology - 136(2015), 4 vom: 01. Okt., Seite 1018-24 |
| Sprache: |
Englisch |
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| Beteiligte Personen: |
Mitsui-Sekinaka, Kanako [VerfasserIn] |
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| Links: |
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| Anmerkungen: |
Date Completed 22.01.2016 Date Revised 08.04.2022 published: Print-Electronic Citation Status MEDLINE |
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| doi: |
10.1016/j.jaci.2015.02.020 |
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| funding: |
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| Förderinstitution / Projekttitel: |
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| PPN (Katalog-ID): |
NLM247730556 |
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| 100 | 1 | |a Mitsui-Sekinaka, Kanako |e verfasserin |4 aut | |
| 245 | 1 | 0 | |a Clinical features and hematopoietic stem cell transplantations for CD40 ligand deficiency in Japan |
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| 500 | |a Date Revised 08.04.2022 | ||
| 500 | |a published: Print-Electronic | ||
| 500 | |a Citation Status MEDLINE | ||
| 520 | |a Copyright © 2015 American Academy of Allergy, Asthma & Immunology. Published by Elsevier Inc. All rights reserved. | ||
| 520 | |a BACKGROUND: The long-term outcome of X-linked hyper-IgM syndrome (XHIM) caused by mutations in CD40LG is poor, and the only curative treatment is hematopoietic stem cell transplantation (HSCT) | ||
| 520 | |a OBJECTIVE: We sought to determine the clinical features and factors affecting outcomes in patients with XHIM | ||
| 520 | |a METHODS: We enrolled and retrospectively analyzed data from 56 Japanese patients with XHIM, including 29 patients who received HSCT | ||
| 520 | |a RESULTS: The long-term survival rate was poor in those not undergoing HSCT (overall survival rate at 40 years of age, 28.2%). The overall survival rate of patients undergoing HSCT (n = 29) was significantly higher than that of those not undergoing HSCT (n = 27, P = .0231). Moreover, event-free and disease-free survival rates were significantly greater in patients 5 years old or younger at the time of transplantation (n = 14) than in older patients (n = 15) | ||
| 520 | |a CONCLUSION: On the basis of these results, we concluded that HSCT improved the outcomes of patients with XHIM and that an age of 5 years or younger was optimal for the timing of HSCT because persistent infections and severe organ damage were frequently observed in patients older than 6 years | ||
| 650 | 4 | |a Journal Article | |
| 650 | 4 | |a Research Support, Non-U.S. Gov't | |
| 650 | 4 | |a CD40 ligand | |
| 650 | 4 | |a Cryptococcus neoformans | |
| 650 | 4 | |a Cryptosporidium parvum | |
| 650 | 4 | |a Pneumocystis jirovecii | |
| 650 | 4 | |a class-switch recombination | |
| 650 | 4 | |a combined immunodeficiency | |
| 650 | 4 | |a hematopoietic stem cell transplantation | |
| 650 | 4 | |a hyper-IgM syndrome | |
| 650 | 4 | |a primary immunodeficiency | |
| 650 | 7 | |a CD40 Ligand |2 NLM | |
| 650 | 7 | |a 147205-72-9 |2 NLM | |
| 700 | 1 | |a Imai, Kohsuke |e verfasserin |4 aut | |
| 700 | 1 | |a Sato, Hiroki |e verfasserin |4 aut | |
| 700 | 1 | |a Tomizawa, Daisuke |e verfasserin |4 aut | |
| 700 | 1 | |a Kajiwara, Michiko |e verfasserin |4 aut | |
| 700 | 1 | |a Nagasawa, Masayuki |e verfasserin |4 aut | |
| 700 | 1 | |a Morio, Tomohiro |e verfasserin |4 aut | |
| 700 | 1 | |a Nonoyama, Shigeaki |e verfasserin |4 aut | |
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