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利用索赔数据为慢性阻塞性肺疾病患者开发一种预测算法,以确定潜在未诊断的非结核分枝杆菌肺病。

Identifying potentially undiagnosed nontuberculous mycobacterial lung disease among patients with chronic obstructive pulmonary disease: Development of a predictive algorithm using claims data.

机构信息

Insmed Incorporated, Bridgewater, NJ.

Department of Medicine, University of Toronto, ON, Canada.

出版信息

J Manag Care Spec Pharm. 2023 Aug;29(8):927-937. doi: 10.18553/jmcp.2023.22417. Epub 2023 May 27.

Abstract

Nontuberculous mycobacterial lung disease (NTMLD) is a debilitating disease. Chronic obstructive pulmonary disease (COPD) is the leading comorbidity associated with NTMLD in the United States. Their similarities in symptoms and overlapping radiological findings may delay NTMLD diagnosis in patients with COPD. To develop a predictive model that identifies potentially undiagnosed NTMLD among patients with COPD. This retrospective cohort study developed a predictive model of NTMLD using US Medicare beneficiary claims data (2006 - 2017). Patients with COPD with NTMLD were matched 1:3 to patients with COPD without NTMLD by age, sex, and year of COPD diagnosis. The predictive model was developed using logistic regression modeling risk factors such as pulmonary symptoms, comorbidities, and health care resource utilization. The final model was based on model fit statistics and clinical inputs. Model performance was evaluated for both discrimination and generalizability with c-statistics and receiver operating characteristic curves. There were 3,756 patients with COPD with NTMLD identified and matched to 11,268 patients with COPD without NTMLD. A higher proportion of patients with COPD with NTMLD, compared with those with COPD without NTMLD, had claims for pulmonary symptoms and conditions, including hemoptysis (12.6% vs 1.4%), cough (63.4% vs 24.7%), dyspnea (72.5% vs 38.2%), pneumonia (59.2% vs 13.4%), chronic bronchitis (40.5% vs 16.3%), emphysema, (36.7% vs 11.1%), and lung cancer (15.7% vs 3.5%). A higher proportion of patients with COPD with NTMLD had pulmonologist and infectious disease (ID) specialist visits than patients with COPD without NTMLD (≥ 1 pulmonologist visit: 81.3% vs 23.6%, respectively; ≥ 1 ID visit: 28.3% vs 4.1%, respectively, < 0.0001). The final model consists of 10 risk factors (≥ 2 ID specialist visits; ≥ 4 pulmonologist visits; the presence of hemoptysis, cough, emphysema, pneumonia, tuberculosis, lung cancer, or idiopathic interstitial lung disease; and being underweight during a 1-year pre-NTMLD period) predicting NTMLD with high sensitivity and specificity (c-statistic, 0.9). The validation of the model on new testing data demonstrated similar discrimination and showed the model was able to predict NTMLD earlier than the receipt of the first diagnostic claim for NTMLD. This predictive algorithm uses a set of criteria comprising patterns of health care use, respiratory symptoms, and comorbidities to identify patients with COPD and possibly undiagnosed NTMLD with high sensitivity and specificity. It has potential application in raising timely clinical suspicion of patients with possibly undiagnosed NTMLD, thereby reducing the period of undiagnosed NTMLD. Dr Wang and Dr Hassan are employees of Insmed, Inc. Dr Chatterjee was an employee of Insmed, Inc, at the time of this study. Dr Marras is participating in multicenter clinical trials sponsored by Insmed, Inc, has consulted for RedHill Biopharma, and has received a speaker's honorarium from AstraZeneca. Dr Allison is an employee of Statistical Horizons, LLC. This study was funded by Insmed Inc.

摘要

非结核分枝杆菌肺病(NTMLD)是一种使人虚弱的疾病。慢性阻塞性肺疾病(COPD)是与美国 NTMLD 相关的主要合并症。它们在症状和重叠的影像学发现方面的相似性可能会延迟 COPD 患者的 NTMLD 诊断。为了开发一种可以识别 COPD 患者中潜在未诊断的 NTMLD 的预测模型。这项回顾性队列研究使用美国医疗保险受益索赔数据(2006-2017 年)开发了一种 NTMLD 预测模型。将 NTMLD 合并 COPD 的患者与无 NTMLD 的 COPD 患者按年龄、性别和 COPD 诊断年份进行 1:3 匹配。使用逻辑回归模型对预测模型进行建模,风险因素包括肺部症状、合并症和医疗保健资源利用情况。最终模型基于模型拟合统计数据和临床输入。使用 c 统计量和接收者操作特征曲线评估模型的区分度和泛化能力。共确定了 3756 例 NTMLD 合并 COPD 的患者,并与 11268 例无 NTMLD 的 COPD 患者匹配。与无 NTMLD 的 COPD 患者相比,NTMLD 合并 COPD 的患者有更多的肺部症状和疾病索赔,包括咯血(12.6%比 1.4%)、咳嗽(63.4%比 24.7%)、呼吸困难(72.5%比 38.2%)、肺炎(59.2%比 13.4%)、慢性支气管炎(40.5%比 16.3%)、肺气肿(36.7%比 11.1%)和肺癌(15.7%比 3.5%)。NTMLD 合并 COPD 的患者与无 NTMLD 的 COPD 患者相比,有更多的肺病专家和传染病(ID)专家就诊(≥1 次肺病专家就诊:81.3%比 23.6%;≥1 次 ID 专家就诊:28.3%比 4.1%,均 < 0.0001)。最终模型包含 10 个风险因素(≥2 次 ID 专家就诊;≥4 次肺病专家就诊;咯血、咳嗽、肺气肿、肺炎、结核病、肺癌或特发性间质性肺病的存在;以及在 NTMLD 前 1 年期间体重不足),对 NTMLD 具有较高的敏感性和特异性(c 统计量为 0.9)。在新的测试数据上对模型进行验证表明,其具有相似的区分度,并表明该模型能够更早地预测 NTMLD,而不是在收到 NTMLD 的第一个诊断索赔后。该预测算法使用一组标准,包括医疗保健使用模式、呼吸症状和合并症,以高敏感性和特异性识别 COPD 患者和可能未确诊的 NTMLD。它具有提高可能未确诊 NTMLD 患者及时临床怀疑的潜力,从而减少未确诊 NTMLD 的时间。Wang 博士和 Hassan 博士是 Insmed, Inc. 的员工。Chatterjee 博士在这项研究期间是 Insmed, Inc. 的员工。Marras 博士参与了 Insmed, Inc. 赞助的多中心临床试验,曾为 RedHill Biopharma 咨询过,并收到了 AstraZeneca 的演讲酬金。Allison 博士是 Statistical Horizons, LLC 的员工。这项研究由 Insmed Inc. 资助。

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