Outcomes in hematopoetic cell transplantation in the setting of mold infections in patients with chronic granulomatous disease.

IF 4.5 2区 医学 Q1 HEMATOLOGY
Ahnika Kline, Mark Parta, Jennifer Cuellar-Rodriguez, Juan Gea-Banacloche, Corin Kelly, Stefania Pittaluga, Christa S Zerbe, Steven M Holland, Harry L Malech, Elizabeth M Kang
{"title":"Outcomes in hematopoetic cell transplantation in the setting of mold infections in patients with chronic granulomatous disease.","authors":"Ahnika Kline, Mark Parta, Jennifer Cuellar-Rodriguez, Juan Gea-Banacloche, Corin Kelly, Stefania Pittaluga, Christa S Zerbe, Steven M Holland, Harry L Malech, Elizabeth M Kang","doi":"10.1038/s41409-024-02389-x","DOIUrl":null,"url":null,"abstract":"<p><p>Chronic granulomatous disease (CGD) is a disorder of immunity characterized by phagocyte dysfunction. Mold infections in patients with CGD are often severe and disseminated. We present patient characteristics, microbiological data, and outcomes for 26 patients with CGD who received hematopoietic cell transplantation (HCT) or gene therapy-modified cells (GT) between 2008 and 2019, with proven fungal infection either before or during their transplant. All patients engrafted, and all but one GT recipient had neutrophil recovery and evidence of functional correction. Eighteen patients (69%) are currently alive and 19 patients (73% of total, 90% of patients with repeat imaging performed) had evidence of radiographic improvement. With 3 exceptions, deaths were not principally related to the fungal infection and duration of antecedent infection did not correlate with death. Aspergillus species accounted for the majority of disease (50%), followed by Phellinus species (18%). Osteomyelitis and disseminated disease were common, as only 11 patients (42%) had disease restricted to pneumonia. Triazole therapy was used in all 26 patients, with combination therapy used in 25 (96%). HCT or gene therapy, with appropriate antifungal therapy, are viable therapies for refractory fungal infections in patients with CGD.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5000,"publicationDate":"2024-11-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Bone Marrow Transplantation","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1038/s41409-024-02389-x","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"HEMATOLOGY","Score":null,"Total":0}
引用次数: 0

Abstract

Chronic granulomatous disease (CGD) is a disorder of immunity characterized by phagocyte dysfunction. Mold infections in patients with CGD are often severe and disseminated. We present patient characteristics, microbiological data, and outcomes for 26 patients with CGD who received hematopoietic cell transplantation (HCT) or gene therapy-modified cells (GT) between 2008 and 2019, with proven fungal infection either before or during their transplant. All patients engrafted, and all but one GT recipient had neutrophil recovery and evidence of functional correction. Eighteen patients (69%) are currently alive and 19 patients (73% of total, 90% of patients with repeat imaging performed) had evidence of radiographic improvement. With 3 exceptions, deaths were not principally related to the fungal infection and duration of antecedent infection did not correlate with death. Aspergillus species accounted for the majority of disease (50%), followed by Phellinus species (18%). Osteomyelitis and disseminated disease were common, as only 11 patients (42%) had disease restricted to pneumonia. Triazole therapy was used in all 26 patients, with combination therapy used in 25 (96%). HCT or gene therapy, with appropriate antifungal therapy, are viable therapies for refractory fungal infections in patients with CGD.

慢性肉芽肿病患者在霉菌感染情况下进行造血细胞移植的结果。
慢性肉芽肿病(CGD)是一种以吞噬细胞功能障碍为特征的免疫紊乱疾病。CGD患者的霉菌感染通常很严重,并呈播散性。我们介绍了2008年至2019年期间接受造血细胞移植(HCT)或基因治疗改造细胞(GT)的26名CGD患者的患者特征、微生物学数据和治疗结果,这些患者在移植前或移植期间均已证实感染了真菌。所有患者都进行了移植,除一名GT受者外,其他所有患者的中性粒细胞均已恢复,并有功能矫正的迹象。目前有18名患者(69%)存活,19名患者(占总数的73%,90%的患者进行了重复成像)有放射学改善的证据。除 3 名患者外,其他患者的死亡均与真菌感染无关,且前驱感染的持续时间与死亡无关。曲霉菌感染占大多数(50%),其次是黄柏菌(18%)。骨髓炎和播散性疾病很常见,只有11名患者(42%)的疾病仅限于肺炎。所有 26 名患者都使用了三唑疗法,其中 25 人(96%)使用了联合疗法。HCT或基因疗法配合适当的抗真菌治疗,是治疗CGD患者难治性真菌感染的可行疗法。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
求助全文
约1分钟内获得全文 求助全文
来源期刊
Bone Marrow Transplantation
Bone Marrow Transplantation 医学-免疫学
CiteScore
8.40
自引率
8.30%
发文量
337
审稿时长
6 months
期刊介绍: Bone Marrow Transplantation publishes high quality, peer reviewed original research that addresses all aspects of basic biology and clinical use of haemopoietic stem cell transplantation. The broad scope of the journal thus encompasses topics such as stem cell biology, e.g., kinetics and cytokine control, transplantation immunology e.g., HLA and matching techniques, translational research, and clinical results of specific transplant protocols. Bone Marrow Transplantation publishes 24 issues a year.
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
确定
请完成安全验证×
copy
已复制链接
快去分享给好友吧!
我知道了
右上角分享
点击右上角分享
0
联系我们:info@booksci.cn Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术官方微信