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Psychometric Validation of the PAH Symptoms and Impact (PAH-SYMPACT) Questionnaire: Results From the SYMPHONY Study
Pulmonary Vascular Disease SESSION TITLE: Pulmonary Hypertension SESSION TYPE: Original Investigation Slide PRESENTED ON: Monday, October 24, 2016 at 04:30 PM - 05:30 PM
Psychometric Validation of the PAH Symptoms and Impact (PAH-SYMPACT) Questionnaire: Results From the SYMPHONY Study
PULMONARY VASCULAR DISEASE
Robert Frantz MD* Richard Channick MD Kelly Chin MD Aryeh Fischer MD Mardi Gomberg-Maitland MD Leah Kleinman DrPH Chad Miller MD Elke Hunsche PhD Roham Zamanian MD Michael Zastrow PhD; and David Badesch MD Mayo Clinic, Rochester, MN PURPOSE: The PAH-SYMPACT is a patient reported outcome (PRO) instrument developed based on interviews with PAH patients and following the process outlined in the FDA’s PRO guidance. Its psychometric characteristics are being evaluated using data from SYMPHONY - a phase IIIb PAH study with macitentan. After item reduction, the final instrument consists of 11 symptom and 11 impact items across 4 domains (cardiopulmonary symptoms [CP], cardiovascular symptoms [CV], physical impact and cognitive/emotional impacts). The objective of the current analyses was to evaluate the psychometric properties of the PAH-SYMPACT domain scores. METHODS: Patients enrolled in SYMPHONY completed generic and disease-specific PROs, while clinicians provided assessment of severity and changes in symptomatology. Internal consistency reliability, test-retest and construct, and known-group validity as well as sensitivity to change were evaluated. In addition, performance of the instrument based on oxygen use was assessed. RESULTS: The analyses included data from 278 patients (79% female, mean age 60 years), mainly in WHO functional class II/III (40/ 59%). 41% were on background therapy with a PDE-5 inhibitor and about a third of patients used oxygen therapy at any given time. Initial analysis demonstrated that individual item scores do not differ based on oxygen use. Internal consistency reliability was very good for all 4 domains (Cronbach’s alpha > 0.80); test-retest reliability (assessed in stable patients) was highly reproducible for all 4 domains (intra-class correlation coefficient [ICC] 0.84-0.94). Correlations with SF-36 and CAMPHOR were moderate-to-high, with slightly lower correlations occurring in the CV domain than the CP domain. The instrument was shown to differentiate well between patients with different disease severity levels. Domain scores were sensitive to changes in clinician- and patient-reported disease severity levels. CONCLUSIONS: The PAH-SYMPACT domain scores were shown to be reproducible, to differentiate between patients of different severity, to correlate with other instruments measuring similar constructs, and to be sensitive to change. CLINICAL IMPLICATIONS: In this study, the PAH-SYMPACT performed well with good psychometric properties. The instrument has been found to be relevant to PAH patients and clinicians; it has the potential to become an important outcome measure for PAH in both clinical trials and clinical practice. DISCLOSURE: Robert Frantz: Consultant fee, speaker bureau, advisory committee, etc.: Robert Frantz sits on a Steering Committee and advisory board for Actelion, without personal financial gain aside from coverage of travel expenses Richard Channick: Consultant fee, speaker bureau, advisory committee, etc.: Richard Channick sits on a Steering Committee for Actelion Pharmaceuticals Ltd Kelly Chin: Consultant fee, speaker bureau, advisory committee, etc.: Kelly Chin sits on a Steering Committee for Actelion Pharmaceuticals Ltd Aryeh Fischer: Consultant fee, speaker bureau, advisory committee, etc.: Aryeh Fischer serves as a consultant for Actelion Pharmaceuticals Ltd Mardi Gomberg-Maitland: Grant monies (from industry related sources): Mardi Gomberg-Maitland has reveived research grant money for clinical trials from Actelion Pharmaceuticals Ltd, Consultant fee, speaker bureau, advisory committee, etc.: Mardi Gomberg-Maitland has served as a consultant for Actelion Pharmaceuticals Ltd Leah Kleinman: Employee: Leah Kleinman is a salaried employee of Evidera Chad Miller: Grant monies (from industry related sources): Chad Miller has received research grant money for active clinical trials from Actelion Pharmaceuticals Ltd, Consultant fee, speaker bureau, advisory committee, etc.: Chad Miller has received compensation for speaker’s bureau Elke Hunsche: Shareholder: Elke Hunsche is a shareholder of Actelion Pharmaceuticals Ltd, Employee: Elke Hunsche is an employee of Actelion Pharmaceuticals Ltd Roham Zamanian: Grant monies (from industry related sources): Roham Zamanian has received grant money from Actelion Pharmaceuticals Ltd, Consultant fee, speaker bureau, advisory committee, etc.: Roham Zamanian has acted as a consultant for Actelion Pharmaceuticals Ltd, Consultant fee, speaker bureau, advisory committee, etc.: Consultant to Actelion, United Therapeutics, Bayer Michael Zastrow: Shareholder: Michael Zastrow is a shareholder of Actelion Pharmaceuticals Ltd, Employee: Micheal Zastrow is an employee of Actelion Pharmaceuticals Ltd David Badesch: Grant monies (from industry related sources): David Badesch has received research grant money for clinical trials including this study from Actelion Pharmaceuticals Ltd, Consultant fee, speaker bureau, advisory committee, etc.: David Badesch sits on a Steering Committee and advisory boards for Actelion Pharmaceuticals Ltd No Product/Research Disclosure Information DOI:
http://dx.doi.org/10.1016/j.chest.2016.08.1269
Copyright ª 2016 American College of Chest Physicians. Published by Elsevier Inc. All rights reserved.
1160A
[
150#4S CHEST OCTOBER 2016
]
Psychometric Validation of the PAH Symptoms and Impact (PAH-SYMPACT) Questionnaire: Results From the SYMPHONY Study
PULMONARY VASCULAR DISEASE
Robert Frantz MD* Richard Channick MD Kelly Chin MD Aryeh Fischer MD Mardi Gomberg-Maitland MD Leah Kleinman DrPH Chad Miller MD Elke Hunsche PhD Roham Zamanian MD Michael Zastrow PhD; and David Badesch MD Mayo Clinic, Rochester, MN PURPOSE: The PAH-SYMPACT is a patient reported outcome (PRO) instrument developed based on interviews with PAH patients and following the process outlined in the FDA’s PRO guidance. Its psychometric characteristics are being evaluated using data from SYMPHONY - a phase IIIb PAH study with macitentan. After item reduction, the final instrument consists of 11 symptom and 11 impact items across 4 domains (cardiopulmonary symptoms [CP], cardiovascular symptoms [CV], physical impact and cognitive/emotional impacts). The objective of the current analyses was to evaluate the psychometric properties of the PAH-SYMPACT domain scores. METHODS: Patients enrolled in SYMPHONY completed generic and disease-specific PROs, while clinicians provided assessment of severity and changes in symptomatology. Internal consistency reliability, test-retest and construct, and known-group validity as well as sensitivity to change were evaluated. In addition, performance of the instrument based on oxygen use was assessed. RESULTS: The analyses included data from 278 patients (79% female, mean age 60 years), mainly in WHO functional class II/III (40/ 59%). 41% were on background therapy with a PDE-5 inhibitor and about a third of patients used oxygen therapy at any given time. Initial analysis demonstrated that individual item scores do not differ based on oxygen use. Internal consistency reliability was very good for all 4 domains (Cronbach’s alpha > 0.80); test-retest reliability (assessed in stable patients) was highly reproducible for all 4 domains (intra-class correlation coefficient [ICC] 0.84-0.94). Correlations with SF-36 and CAMPHOR were moderate-to-high, with slightly lower correlations occurring in the CV domain than the CP domain. The instrument was shown to differentiate well between patients with different disease severity levels. Domain scores were sensitive to changes in clinician- and patient-reported disease severity levels. CONCLUSIONS: The PAH-SYMPACT domain scores were shown to be reproducible, to differentiate between patients of different severity, to correlate with other instruments measuring similar constructs, and to be sensitive to change. CLINICAL IMPLICATIONS: In this study, the PAH-SYMPACT performed well with good psychometric properties. The instrument has been found to be relevant to PAH patients and clinicians; it has the potential to become an important outcome measure for PAH in both clinical trials and clinical practice. DISCLOSURE: Robert Frantz: Consultant fee, speaker bureau, advisory committee, etc.: Robert Frantz sits on a Steering Committee and advisory board for Actelion, without personal financial gain aside from coverage of travel expenses Richard Channick: Consultant fee, speaker bureau, advisory committee, etc.: Richard Channick sits on a Steering Committee for Actelion Pharmaceuticals Ltd Kelly Chin: Consultant fee, speaker bureau, advisory committee, etc.: Kelly Chin sits on a Steering Committee for Actelion Pharmaceuticals Ltd Aryeh Fischer: Consultant fee, speaker bureau, advisory committee, etc.: Aryeh Fischer serves as a consultant for Actelion Pharmaceuticals Ltd Mardi Gomberg-Maitland: Grant monies (from industry related sources): Mardi Gomberg-Maitland has reveived research grant money for clinical trials from Actelion Pharmaceuticals Ltd, Consultant fee, speaker bureau, advisory committee, etc.: Mardi Gomberg-Maitland has served as a consultant for Actelion Pharmaceuticals Ltd Leah Kleinman: Employee: Leah Kleinman is a salaried employee of Evidera Chad Miller: Grant monies (from industry related sources): Chad Miller has received research grant money for active clinical trials from Actelion Pharmaceuticals Ltd, Consultant fee, speaker bureau, advisory committee, etc.: Chad Miller has received compensation for speaker’s bureau Elke Hunsche: Shareholder: Elke Hunsche is a shareholder of Actelion Pharmaceuticals Ltd, Employee: Elke Hunsche is an employee of Actelion Pharmaceuticals Ltd Roham Zamanian: Grant monies (from industry related sources): Roham Zamanian has received grant money from Actelion Pharmaceuticals Ltd, Consultant fee, speaker bureau, advisory committee, etc.: Roham Zamanian has acted as a consultant for Actelion Pharmaceuticals Ltd, Consultant fee, speaker bureau, advisory committee, etc.: Consultant to Actelion, United Therapeutics, Bayer Michael Zastrow: Shareholder: Michael Zastrow is a shareholder of Actelion Pharmaceuticals Ltd, Employee: Micheal Zastrow is an employee of Actelion Pharmaceuticals Ltd David Badesch: Grant monies (from industry related sources): David Badesch has received research grant money for clinical trials including this study from Actelion Pharmaceuticals Ltd, Consultant fee, speaker bureau, advisory committee, etc.: David Badesch sits on a Steering Committee and advisory boards for Actelion Pharmaceuticals Ltd No Product/Research Disclosure Information DOI:
http://dx.doi.org/10.1016/j.chest.2016.08.1269
Copyright ª 2016 American College of Chest Physicians. Published by Elsevier Inc. All rights reserved.
1160A
[
150#4S CHEST OCTOBER 2016
]