Psychometric Validation of the Pulmonary Arterial Hypertension-Symptoms and Impact (PAH-SYMPACT) Questionnaire

Chin, Kelly; Gomberg‐Maitland, Mardi; Channick, Richard N.; Cuttica, Michael J.; Fischer, Aryeh; Frantz, Robert P.; Hunsche, Elke; Kleinman, Leah; McConnell, John W.; McLaughlin, Vallerie V.; Miller, Chad E.; Zamanian, Roham T.; Zastrow, Michael; Badesch, David B.

doi:10.1016/j.chest.2018.04.027

Cited by 48 publications

(82 citation statements)

References 27 publications

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“…The CAMPHOR (Cambridge Pulmonary Hypertension Outcome Review) questionnaire, the first PAH/CTEPH-specific questionnaire, is quite lengthy and does not track with other clinical measures over time [27]. The 10-question survey proposed by the Pulmonary Hypertension Association UK (emPHasis-10) is more efficient, but needs further study [28], and the recently reported SYMPACT study appears to be the more efficient and inclusive, but also needs additional validation [29].…”

Section: Current State Of Clinical Trial Design and Therapeutics In Pmentioning

confidence: 99%

Clinical trial design and new therapies for pulmonary arterial hypertension

et al. 2019

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Until 20 years ago the treatment of pulmonary arterial hypertension (PAH) was based on case reports and small series, and was largely ineffectual. As a deeper understanding of the pathogenesis and pathophysiology of PAH evolved over the subsequent two decades, coupled with epidemiological studies defining the clinical and demographic characteristics of the condition, a renewed interest in treatment development emerged through collaborations between international experts, industry and regulatory agencies. These efforts led to the performance of robust, high-quality clinical trials of novel therapies that targeted putative pathogenic pathways, leading to the approval of more than 10 novel therapies that have beneficially impacted both the quality and duration of life. However, our understanding of PAH remains incomplete and there is no cure. Accordingly, efforts are now focused on identifying novel pathogenic pathways that may be targeted, and applying more rigorous clinical trial designs to better define the efficacy of these new potential treatments and their role in the management scheme. This article, prepared by a Task Force comprised of expert clinicians, trialists and regulators, summarises the current state of the art, and provides insight into the opportunities and challenges for identifying and assessing the efficacy and safety of new treatments for this challenging condition.

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Section: Current State Of Clinical Trial Design and Therapeutics In Pmentioning

confidence: 99%

Clinical trial design and new therapies for pulmonary arterial hypertension

et al. 2019

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“…Of note, a disease-specific patient-reported outcome (PRO) instrument, the Pulmonary Arterial Hypertension-Symptoms and Impact (PAH-SYMPACT ® ) questionnaire, has been recently finalized and validated using data from the SYMPHONY trial which included 278 US patients with PAH treated with macitentan. 43…”

Section: Macitentan: Clinical Evidencementioning

confidence: 99%

Treatment of pulmonary arterial hypertension with the dual endothelin receptor antagonist macitentan: clinical evidence and experience

Belge

Delcroix

2019

Ther Adv Respir Dis

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Macitentan (10 mg once daily orally), a dual endothelin receptor antagonist (ERA) developed by modifying the structure of bosentan to increase the efficacity and safety, is approved for the treatment of pulmonary arterial hypertension (PAH). The pivotal SERAPHIN trial, (a landmark trial in the history of PAH trials because of the large number of included patients, the long-term follow up and the first trial with morbidity/mortality as the primary endpoint) showed a reduction of the risk of a morbidity or mortality event by 45% over the treatment time compared with placebo. The positive effect on the primary endpoint was observed whether or not the patient was already on PAH therapy. There has been no direct comparison between macitentan and other ERAs, which were approved based on improved exercise capacity, but preclinical and clinical data suggest better pharmacological and safety profiles. Further analyses of the SERAPHIN trial investigated the predictive value of different indices and events on long-term outcome and mortality. The efficacy in children, the long-term effects and safety of macitentan and its place in combination therapy compared with other ERAs are still under investigation. This review presents the preclinical evidence of superiority of macitentan compared with other ERAs, and the available clinical trial data. The place of macitentan in the therapeutic algorithm for PAH treatment, post-marketing experience and future perspectives are discussed.

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“…As reported previously, 284 patients were enrolled in SYMPHONY between April 2013 and October 2015 at 71 centers in the United States. 21 Correlation between the weekly average and one-day symptom scores…”

Section: Resultsmentioning

confidence: 99%

Pulmonary Arterial Hypertension—Symptoms and Impact Questionnaire: feasibility of utilizing one‐day versus seven‐day symptom reporting

et al. 2020

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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.

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Psychometric Validation of the Pulmonary Arterial Hypertension-Symptoms and Impact (PAH-SYMPACT) Questionnaire

Abstract: ClinicalTrials.gov; No.: NCT01841762; URL: www.clinicaltrials.gov.

Cited by 48 publications

References 27 publications

Clinical trial design and new therapies for pulmonary arterial hypertension

Clinical trial design and new therapies for pulmonary arterial hypertension

Treatment of pulmonary arterial hypertension with the dual endothelin receptor antagonist macitentan: clinical evidence and experience

Pulmonary Arterial Hypertension—Symptoms and Impact Questionnaire: feasibility of utilizing one‐day versus seven‐day symptom reporting

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