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囊性纤维化患儿中非结核分枝杆菌的治疗:一项多中心回顾性研究。

Treating nontuberculous mycobacteria in children with cystic fibrosis: a multicentre retrospective study.

机构信息

Respiratory Unit, Alder Hey Children's NHS Foundation Trust, Liverpool, Merseyside, UK.

Department of Child Health (University of Liverpool), Institute in the Park, Alder Hey Children's Hospital, Liverpool, Merseyside, UK.

出版信息

Arch Dis Child. 2022 May;107(5):479-485. doi: 10.1136/archdischild-2021-322177. Epub 2021 Nov 5.

DOI:10.1136/archdischild-2021-322177
PMID:34740877
Abstract

BACKGROUND

Respiratory infection with nontuberculous mycobacteria (NTM) in children with cystic fibrosis (CF) has increased in prevalence. The condition is difficult to diagnose and treatments are complex with limited evidence to guide practice. This study describes the approaches to diagnosis, management and consequences of treatment in a multicentre cohort of children with CF in the UK.

METHODS

Retrospective data were collected from 11 CF specialist centres from patients less than 17 years old, treated for NTM infection between 2006 and 2017. Descriptive statistics were used to describe the clinical characteristics of children treated. Treatment regimens, adverse events and success of treatment, with respect to lung function and culture conversion, were evaluated.

RESULTS

Data from 70 patients treated for NTM pulmonary disease were collated (60 complex (MABSC); 10 . complex (MAC)). Older age and previous diagnosis of allergic bronchopulmonary aspergillosis were all significantly associated with NTM. There was a wide variance in drug choice and side effects were reported with all agents. NTM eradication occurred in 80% of patients with MAC and 48% with MABSC, with variable outcomes on lung function.

CONCLUSIONS

Diagnosis and treatment of NTM infection in children with CF is challenging. Treatment success is not guaranteed, particularly for MABSC. Large clinical trials are urgently required to evaluate treatment regimes and their suitability and efficacy in children.

摘要

背景

囊性纤维化(CF)患儿中分枝杆菌(NTM)呼吸道感染的患病率有所增加。这种疾病很难诊断,治疗方法复杂,而且缺乏指导实践的有限证据。本研究描述了英国 11 家 CF 专科中心对一组儿童患者的诊断、管理和治疗后果的方法。

方法

从 2006 年至 2017 年期间接受 NTM 感染治疗的 11 家 CF 专科中心的 11 岁以下患者中,收集回顾性数据。采用描述性统计方法描述了接受治疗的儿童的临床特征。评估了治疗方案、不良反应以及治疗的成功率,具体涉及肺功能和培养物转化。

结果

共整理了 70 例接受 NTM 肺病治疗患者的数据(60 例为复合(MABSC);10 例为简单(MAC))。年龄较大和先前诊断为变应性支气管肺曲霉病与 NTM 均有显著相关性。药物选择差异很大,所有药物均有报道出现副作用。MAC 患者中有 80%的患者根除了 NTM,MABSC 患者中有 48%的患者根除了 NTM,肺功能的结果各不相同。

结论

CF 患儿 NTM 感染的诊断和治疗具有挑战性。并不能保证治疗成功,特别是对于 MABSC。迫切需要开展大型临床试验,以评估治疗方案及其在儿童中的适用性和疗效。

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