Because health-related quality of life (HRQOL) factors into prognosis for individuals with pulmonary hypertension (PH), HRQOL questionnaires specific to PH were developed by researchers conducting the EmPHasis-10 and CAMPHOR trials. New research findings published in Respiratory Medicine now suggest that these questionnaires are valid methods for assessing longitudinal HRQOL in patients with PH and may thus be useful for clinicians in daily practice.

The recent study, which aimed to investigate the 2 questionnaires’ long-term validity and minimal clinically important differences, involved 61 consecutive patients with newly diagnosed, treatment-naive PH, including 31 patients with pulmonary arterial hypertension (PAH; mean age, 62.3±16.4 years; 67.7% women) and 30 patients with chronic thromboembolic pulmonary hypertension (CTEPH; mean age, 64.8±14.9 years; 50% women).

After diagnosis, patients received guideline-based treatment and were followed over the course of 1 year, with EmPHasis-10 and CAMPHOR scores taken at baseline, 6 months, and 12 months. The investigators then correlated those scores with measures that included a 5-scale symptom severity score, a 5-scale overall health score (based on patients’ health perceptions), the New York Heart Association classification score, the 6-minute walking distance (6MWD) score, and N-terminal pro b-type natriuretic peptide (NT-pro-BNP) and echocardiographic parameters.


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After 1 year of treatment, the investigators noted a significant reduction in EmPHasis-10 score (P =.044) and CAMPHOR QOL (P =.040) and symptoms (P =.002) domain score. The questionnaires also showed moderate to good correlations with the overall health and symptom severity score and 6MWD. Relevant correlations were not noted between the questionnaires and NT-pro-BNP and echocardiographic parameters.

The baseline 6MWD was 346 meters, but this increased significantly to 386 meters by 1 year (P <.001). The NT-pro-BNP significantly decreased from baseline to 1 year (62 pmol/L vs 36 pmol/L, respectively; P <.001), while the noninvasive risk score improved significantly during the study period (P <.001 for all domains).

Outcomes of the EmPHasis-10 questionnaire demonstrated strong correlations with the CAMPHOR questionnaire at baseline (Activity: r=0.631; QOL: r=0.707; Symptoms: r=0.832; all P <.001), 6 months (Activity: r=0.614; QOL: r=0.637; Symptoms: r=0.755; all P <.001), and 1 year (Activity: r=0.715; QOL: r=0.772; Symptoms: r=0.784; all P <.001). Additionally, the EmPHasis-10 score change after 1-year follow-up demonstrated good correlation with the change in total CAMPHOR score (r=0.686; P <.001).

The minimal clinically important difference (MCID) for the EmPHasis-10 questionnaire was -8, while the MCIDs for the CAMPHOR domains were −3 for activity, -4 for symptoms, and -3 for QOL. The CAMPHOR MCIDs were observed in 32.1% of patients for activity, 34.5% of patients for the QOL domain, and 32.7% of patients for the symptoms domain. Approximately 12.7% of patients reached the MCID of all domains on the CAMPHOR scale. In contrast, 30.9% of patients reached the EmPHasis-10 MCID.

The researchers said the study was limited by disruption caused by the COVID-19 pandemic, which resulted in some follow-up being conducted by phone or virtually instead of in person.

The investigators concluded that the EmPHasis-10 and CAMPHOR questionnaires are both suitable for longitudinal HRQOL follow-up for patients with PAH and CTEPH, and that “the much shorter EmPHasis-10…may be easier to use in daily practice.”

Disclosure: This clinical trial was supported by Actelion Pharmaceuticals. Please refer to the original article for a full list of disclosures.

Reference

Hendriks PM, van Thor MCJ, Wapenaar M, et al. The longitudinal use of EmPHasis-10 and CAMPHOR questionnaire health-related quality of life scores in patients with pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension. Respir Med. Published online June 29, 2021. doi:10.1016/j.rmed.2021.106525