Development of a Comprehensive, Integrated Patient Data Repository of US Pulmonary Arterial Hypertension (PAH) Patients to Assess the Impact of Clinical Care on Patient Outcomes

ABSTRACT

RATIONALE Advances in PAH management and well-established treatment guidelines have improved the prognosis for patients. However, the extent to which guidelines are implemented in real-world practice and the relationship between guidelines and real-world patient outcomes remain in question. To assess real-world treatment and outcomes, a new type of comprehensive, integrated patient data repository (CIPDR) was created. Here, we describe the process to create this repository to enable interpretation of the collected data. METHODS: The TRIO CIPDR was created with guidance from six pulmonologists who have experience in design of and/or participation in PH registries (e.g. REVEAL). The CIPDR includes data elements of demographics, disease, comorbidities, laboratory data, pulmonary function testing, functional status, PAH treatment, reasons for treatment discontinuation/switch, hospitalizations, and death, which are collected through HIPAA-secure online forms. To minimize entry errors, participating sites received form training and ongoing support, and each form contained logic to identify improbable entries. All data were deidentified prior to storage in secure, redundant servers. The site engagement, data collection forms, data storage, and data output processes were all designed to allow both retrospective and prospective data collection and for near-immediate repository expansion through addition of other PAH-treating centers. Eleven Pulmonary Hypertension Association-certified care centers initially contributed to the CIPDR though two centers were unable to continue participation due to COVID19 impact. Central IRB approval was obtained though many sites independently received approval for the repository protocol by their IRBs. To facilitate enrollment, specialty pharmacy data corresponding to each site were used to identify potential patients and pre-populate qualification forms. Each site reviewed and qualified patients who met repository criteria age >18 years, prescribed PAH-specific medications, and confirmation of PAH diagnosis by right heart catheterization (mean Pulmonary Arterial Pressure ≥25mmHg, Pulmonary Capillary Wedge Pressure ≤15 mmHg, and Pulmonary Vascular Resistance ≥3.0 Wood Units at rest). The initial data collection included care encounters between Jan 2019 and Dec 2020 and data concerning diagnosis, onset of symptoms, procedures, and laboratory values closest to enrollment. After completion of data collection, all data were reviewed by Trio Health and adjudicated with each site. RESULTS: Of 3200 patients identified as potentially qualified, 1009 were initially enrolled and their retrospective data encompassing 4489 visits collected. Descriptive measures of the repository are presented in the TABLE. CONCLUSION: The Trio CIPDR is an important step forward to uniquely characterize the patient journey ,treatment patterns, and outcomes for patients with PAH.