Department of Pediatrics, Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO 80045, USA.
Department of Medicine, National Jewish Health, Denver, CO 80206, USA.
J Cyst Fibros. 2024 Jan;23(1):50-57. doi: 10.1016/j.jcf.2023.08.007. Epub 2023 Sep 4.
Nontuberculous mycobacteria (NTM) are an important cause of airway infections in people with cystic fibrosis (pwCF). Isolation of NTM from respiratory specimens of pwCF do not mandate treatment in the absence of clinical and radiologic features of NTM pulmonary disease (NTM-PD), as some pwCF clear the infection without treatment and others do not appear to progress to NTM-PD despite persistent infection. An evidence-based protocol to standardize diagnosis of NTM-PD is needed to systematically identify pwCF who may benefit from treatment.
In this multicenter observational study, eligible pwCF who are 6 years of age and older and who have had a recent positive NTM culture are systematically evaluated for NTM-PD. Participants are identified based on positive NTM culture results obtained during routine clinical care and following enrollment are evaluated for NTM-PD and CF-related comorbidities. Participants are followed in PREDICT until they meet NTM-PD diagnostic criteria and are ready to initiate NTM treatment, or until study termination. Active participants who have not met these criteria are re-consented every 5 years to enable long-term participation.
The primary endpoint will summarize the proportion of participants who meet the NTM-PD diagnosis definition. The time from enrollment to NTM-PD diagnosis will be derived from Kaplan-Meier estimates.
A prospective protocol to identify NTM-PD in pwCF will test if this standardized approach defines a cohort with signs and symptoms associated with NTM-PD, to assist with clinical decision making and to build a framework for future therapeutic trials.
ClinicalTrials.gov Identifier: NCT02073409.
非结核分枝杆菌（NTM）是囊性纤维化（CF）患者气道感染的重要原因。CF 患者呼吸道标本中分离出 NTM 并不一定需要治疗，除非有 NTM 肺病（NTM-PD）的临床和影像学特征，因为一些 CF 患者在未经治疗的情况下清除了感染，而另一些患者尽管持续感染但似乎并未进展为 NTM-PD。需要制定一个基于证据的方案来规范 NTM-PD 的诊断，以便系统地识别可能受益于治疗的 CF 患者。
在这项多中心观察性研究中，年龄在 6 岁及以上且最近有阳性 NTM 培养结果的合格 CF 患者，将对其进行 NTM-PD 的系统评估。参与者是根据在常规临床护理中获得的阳性 NTM 培养结果确定的，在登记后对其进行 NTM-PD 和 CF 相关合并症的评估。在 PREDICT 中对参与者进行随访，直到他们符合 NTM-PD 诊断标准并准备开始 NTM 治疗，或研究终止。未达到这些标准的活跃参与者将每 5 年重新同意参与，以实现长期参与。
主要终点将总结符合 NTM-PD 诊断定义的参与者比例。从登记到 NTM-PD 诊断的时间将从 Kaplan-Meier 估计中得出。
一项前瞻性方案旨在确定 CF 患者中的 NTM-PD，将测试这种标准化方法是否可以确定一个具有与 NTM-PD 相关的症状和体征的队列，以协助临床决策并为未来的治疗试验建立框架。
ClinicalTrials.gov 标识符：NCT02073409。
