Enrichment Benefits of Risk Algorithms for Pulmonary Arterial Hypertension Clinical Trials.

ABSTRACT: Rationale: Event-driven primary endpoints are increasingly used in pulmonary arterial hypertension clinical trials, substantially increasing required sample sizes and trial lengths. The U.S. Food and Drug Administration advocates the use of prognostic enrichment of clinical trials by preselecting a patient population with increased likelihood of experiencing the trial's primary endpoint.Objectives: This study compares validated clinical scales of risk (Comparative, Prospective Registry of Newly Initiated Therapies for Pulmonary Hypertension, the French score, and Registry to Evaluate Early and Long-Term Pulmonary Arterial Hypertension Disease Management [REVEAL] 2.0) to identify patients who are likely to experience a clinical worsening event for trial enrichment.Methods:<

SUBMITTER: Scott JV 

PROVIDER: S-EPMC7958501 | biostudies-literature | 2021 Mar

REPOSITORIES: biostudies-literature