Frantz Robert P, Chin Kelly M, Zhao Carol, Flynn Megan, Badesch David
Department of Cardiovascular Disease, Mayo Clinic College of Medicine, Rochester, MN, USA.
Internal Medicine, Pulmonary Hypertension Program, University of Texas Southwestern Medical Center, Dallas, TX, USA.
Pulm Circ. 2020 May 14;10(2):2045894020923957. doi: 10.1177/2045894020923957. eCollection 2020 Apr-Jun.
Patient-reported outcomes are important measures to include in pulmonary arterial hypertension clinical trials but are not widely utilized in clinical practice. Pulmonary Arterial Hypertension-Symptoms and Impact Questionnaire (PAH-SYMPACT) is the only pulmonary arterial hypertension-specific patient-reported outcomes instrument developed and validated in accordance with the US Food and Drug Administration guidance on patient-reported outcomes development. The PAH-SYMPACT tool measures pulmonary arterial hypertension-related symptoms and impact of pulmonary arterial hypertension on daily life. Symptoms are reported each day for seven consecutive days, and the impact of pulmonary arterial hypertension over one week is recalled and reported on day 7; however, daily symptom reporting may overburden patients and healthcare resources, limiting the practicality of PAH-SYMPACT outside of clinical trials. To determine the practicability of an abridged version of PAH-SYMPACT for which all reporting is completed on one day, symptom data from the SYMPHONY trial (NCT01841762; PAH-SYMPACT validation study) were retrospectively analyzed to assess whether symptoms reported on each day correlated with the weekly average and whether one-day symptom scores were sensitive to disease severity. Correlation coefficients comparing the weekly average and individual day symptom scores were mostly high or very high regardless of the day they were measured. Findings were similar when using either Spearman's rank correlation or weighted kappa method. One-day symptom scores differentiated well between World Health Organization functional classes II and III/IV pulmonary arterial hypertension and were sensitive to change in disease severity as measured by the Patient Global Assessment of Disease Severity. These data suggest that the one-day PAH-SYMPACT is feasible and appropriate for routine implementation in clinical practice.
患者报告结局是肺动脉高压临床试验中应纳入的重要指标，但在临床实践中并未得到广泛应用。《肺动脉高压症状与影响问卷》（PAH-SYMPACT）是唯一一款根据美国食品药品监督管理局关于患者报告结局制定的指南开发并验证的肺动脉高压特异性患者报告结局工具。PAH-SYMPACT工具用于测量肺动脉高压相关症状以及肺动脉高压对日常生活的影响。连续七天每天报告症状，并在第7天回顾并报告肺动脉高压在一周内的影响；然而，每日症状报告可能给患者和医疗资源带来负担，限制了PAH-SYMPACT在临床试验之外的实用性。为了确定在一天内完成所有报告的PAH-SYMPACT简化版的实用性，对SYMPHONY试验（NCT01841762；PAH-SYMPACT验证研究）的症状数据进行回顾性分析，以评估每天报告的症状是否与每周平均值相关，以及单日症状评分对疾病严重程度是否敏感。比较每周平均值和单日症状评分的相关系数大多较高或非常高，无论测量的是哪一天。使用Spearman等级相关或加权kappa方法时结果相似。单日症状评分能够很好地区分世界卫生组织功能分级II级和III/IV级肺动脉高压，并且对通过患者整体疾病严重程度评估所测量的疾病严重程度变化敏感。这些数据表明，单日PAH-SYMPACT在临床实践中常规实施是可行且合适的。
