Abstract
Several patient-reported outcome measures have been developed to assess health status in pulmonary arterial hypertension. The required change in instrument scores needed, to be seen as meaningful to the individual, however remain unknown. We sought to identify minimal clinically important differences in the Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR) and to validate these against objective markers of functional capacity. Minimal clinically important differences were established from a discovery cohort (n = 129) of consecutive incident cases of idiopathic pulmonary arterial hypertension with CAMPHOR scores recorded at treatment-naïve baseline and 4–12 months following pulmonary arterial hypertension therapy. An independent validation cohort (n = 87) was used to verify minimal clinically important differences. Concurrent measures of functional capacity relative to CAMPHOR scores were collected. Minimal clinically important differences were derived using anchor- and distributional-based approaches. In the discovery cohort, mean (SD) was 54.4 (16.4) years and 64% were female. Most patients (63%) were treated with sequential pulmonary arterial hypertension therapy. Baseline CAMPHOR scores were: Symptoms, 12 (7); Activity, 12 (7) and quality of life, 10 (7). Pulmonary arterial hypertension treatment resulted in significant improvements in CAMPHOR scores (p < 0.05). CAMPHOR minimal clinically important differences averaged across methods for health status improvement were: Symptoms, –4 points; Activity, –4 points and quality of life –3 points. CAMPHOR Activity score change ≥minimal clinically important difference was associated with significantly greater improvement in six-minute walk distance, in both discovery and validation populations. In conclusion, CAMPHOR scores are responsive to pulmonary arterial hypertension treatment. Minimal clinically important differences in pulmonary hypertension-specific scales may provide useful insights into treatment response in future clinical trials.
Highlights
Pulmonary arterial hypertension (PAH) is a rare disorder characterised by a progressive rise in mean pulmonary artery pressure and pulmonary vascular resistance, resulting in right heart failure and death.[1]
We provide the first estimation of a Minimal Clinically Important Difference (MCID) in a pulmonary hypertension-specific patientreported outcomes (PROs) measure (CAMPHOR) using both distributional- and anchor-based methods
Changes in health-related quality of life (HRQoL) in response to PAH treatment in the ‘real world’ setting, remains poorly understood. This is the first systematic study to directly assess the impact of PAH therapy on HRQoL outside of the clinical trial setting using a PH-specific PRO measure; Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR)
Summary
Pulmonary arterial hypertension (PAH) is a rare disorder characterised by a progressive rise in mean pulmonary artery pressure and pulmonary vascular resistance, resulting in right heart failure and death.[1]. 2 | Establishing a MCID for CAMPHOR in IPAH Bunclark et al. Conventionally, establishing drug efficacy in PAH clinical trials has relied upon observed changes in functional status and capacity.[4] It has not until relatively recently that composite morbidity and mortality end-points have been employed in event-driven trials, as highlighted by Sitbon et al.[9] Whether selected trial end-points are relevant to the individual, remains unknown. There is an increasing awareness of the need for patientreported outcomes (PROs) to be incorporated as secondary end-points in PAH clinical trials.[10]
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