Kerr Kim M, Elliott C Greg, Benza Raymond L, Channick Richard N, Chin Kelly M, Davis R Duane, Jain Sonia, LaCroix Andrea Z, Madani Michael M, McLaughlin Vallerie V, Park Myung H, Tapson Victor F, Auger William R
Division of Pulmonary, Critical Care & Sleep Medicine, University of California San Diego, La Jolla, CA, United States.
Department of Medicine, Intermountain Medical Center, Murray, UT, United States.
JMIR Res Protoc. 2021 May 25;10(5):e25397. doi: 10.2196/25397.
Chronic thromboembolic pulmonary hypertension (CTEPH) is a rare sequela of acute pulmonary embolism that is treatable when recognized. Awareness of this disease has increased with recent advancements in therapeutic options, but delays in diagnosis remain common, and diagnostic and treatment guidelines are often not followed. Data gathered from international registries have improved our understanding of CTEPH, but these data may not be applicable to the US population owing to differences in demographics and medical practice patterns.
The US CTEPH Registry (US-CTEPH-R) was developed to provide essential information to better understand the demographics, risk factors, evaluation, and treatment of CTEPH in the United States, as well as the short- and long-term outcomes of surgical and nonsurgical therapies in the modern treatment era.
Thirty sites throughout the United States enrolled 750 subjects in this prospective, longitudinal, observational registry of patients newly diagnosed with CTEPH. Enrollment criteria included a mean pulmonary artery pressure ≥25 mmHg by right heart catheterization and radiologic confirmation of CTEPH by a multidisciplinary adjudication committee. Following enrollment, subjects were followed biannually until the conclusion of the study. Quality of life surveys were administered at enrollment and biannually, and all other testing was at the discretion of the treating clinician. Details regarding surgical therapy, balloon pulmonary angioplasty, and medical therapy were collected at enrollment and at follow-up, as well as information related to health care utilization and survival.
Data from this registry will improve understanding of the demographics, risk factors, and treatment patterns of patients with CTEPH, and the longitudinal impact of therapies on quality of life, health care utilization, and survival.
This manuscript details the methodology and design of the first large, prospective, longitudinal registry of patients with CTEPH in the United States.
ClinicalTrials.gov NCT02429284; https://www.clinicaltrials.gov/ct2/show/NCT02429284.
INTERNATIONAL REGISTERED REPORT IDENTIFIER (IRRID): DERR1-10.2196/25397.
慢性血栓栓塞性肺动脉高压（CTEPH）是急性肺栓塞的一种罕见后遗症，若能得到诊断则可治疗。随着治疗选择方面的最新进展，对这种疾病的认识有所提高，但诊断延误仍然很常见，而且诊断和治疗指南常常未得到遵循。从国际登记处收集的数据增进了我们对CTEPH的了解，但由于人口统计学和医疗实践模式的差异，这些数据可能不适用于美国人群。
建立美国CTEPH登记处（US-CTEPH-R），以提供重要信息，从而更好地了解美国CTEPH患者的人口统计学特征、危险因素、评估和治疗情况，以及现代治疗时代手术和非手术治疗的短期和长期结果。
美国各地的30个地点在这个针对新诊断为CTEPH患者的前瞻性、纵向、观察性登记处纳入了750名受试者。纳入标准包括右心导管检查显示平均肺动脉压≥25 mmHg，以及多学科判定委员会通过影像学确认CTEPH。纳入后，每半年对受试者进行随访，直至研究结束。在入组时和每半年进行一次生活质量调查，所有其他检查由主治医生自行决定。在入组时和随访时收集有关手术治疗、球囊肺动脉成形术和药物治疗的详细信息，以及与医疗保健利用和生存相关的信息。
该登记处的数据将增进对CTEPH患者的人口统计学特征、危险因素和治疗模式的了解，以及治疗对生活质量、医疗保健利用和生存的纵向影响。
本手稿详细介绍了美国首个大型、前瞻性、纵向CTEPH患者登记处的方法和设计。
ClinicalTrials.gov NCT02429284；https://www.clinicaltrials.gov/ct2/show/NCT02429284。
国际注册报告识别码（IRRID）：DERR1-10.2196/25397。
