Poster Presentations: Wednesday, October 26, 2011 |

Establishing the Need for Development of a Patient Reported Outcome Instrument in Pulmonary Arterial Hypertension FREE TO VIEW

Matthew Murawski, PhD; Robert Frantz, MD; Mardi Gomberg-Maitland, MD; Hubert Chen, MD; Joel Fain, PhD; James Cwengros, PharmD; Kafi Sanders, MPH
Chest. 2011;140(4_MeetingAbstracts):711A. doi:10.1378/chest.1119980
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PURPOSE: Review literature and existing evidence to identify PRO needs in PAH. Delineate characteristics of an instrument for use to optimize treatment and clinical outcomes. Determine the optimal method for instrument development.

METHODS: Systematic review of the PAH literature and solicitation of expert opinions from clinicians in the field. Results were synthesized and recurring themes identified.

RESULTS: Changes in reported mortality indicate prolongation of survival time in PAH. Currently instruments developed for related disease states are used. A single 75-item PAH-specific instrument has been validated. Expert opinion suggests that clinicians perceive the need for a brief instrument suitable for use as a standard screening tool in ongoing patient management. Traditional psychometric instrument development requires large numbers of patients with the disease of interest, imposes significant respondent burden, and typically produces instruments with a high level of measurement precision. Instrument responsiveness to change may not be optimal. The Clinical Impact Method permits instrument development using substantially fewer patients, and is conducive to the development of shorter instruments. The CIM consistently develops instruments responsive to change and with a higher level of clinical sensibility (face/content validity) than psychometrically developed instruments.

CONCLUSIONS: Quality of life in PAH patients is an increasingly important outcome measure. Clinician consultation and literature evidence indicate the need for a PRO instrument for general use that emphasizes physical functioning and symptoms. Given the limited number of PAH patients and the debilitating nature of the disease, the Clinical Impact Method for instrument development will impose the lowest respondent burden on the fewest number of patients.

CLINICAL IMPLICATIONS: A short screening instrument would enhance patient/clinician communication, facilitate discernment of patient distress or response, and potentially optimize patient outcomes. The Clinical Impact Method is a preferred approach to produce a short, clinically sensible, PAH-specific PRO tool for use in day-to-day screening of patients.

DISCLOSURE: Matthew Murawski: Consultant fee, speaker bureau, advisory committee, etc.: serves as a consultant to Pfizer,Inc

Robert Frantz: Grant monies (from industry related sources): Pfizer: Consulting fees,United Therapeutics: Served on advisory boards without personal financial gain. but with coverage of travel expense. Received research support. Received educational grants.;, Consultant fee, speaker bureau, advisory committee, etc.: Actelion: Served on advisory boards without personal financial gain but with coverage of travel expense.. Received educational grants.Gilead: Served on advisory boards without personal financial gain, but with coverage of travel expense. , Other: Bayer: Consulting to develop educational program with them; my institution received the fees in keeping with Mayo COI policy for investigators.

Mardi Gomberg-Maitland: Grant monies (from industry related sources): Actelion, Gilead, Lilly/Icos, Pfizer, Novartis, and United Therapeutics have provided funding to the University of Chicago to support Dr. Gomberg-Maitland’s conduct of clinical trials., Consultant fee, speaker bureau, advisory committee, etc.: She has served as a consultant/participant on data safety monitoring board/steering committee for clinical trials for Actelion, Drais, Gilead, Medtronic, and Pfizer.

Hubert Chen: Consultant fee, speaker bureau, advisory committee, etc.: Pfizer,Inc, Consultant fee, speaker bureau, advisory committee, etc.: United Therapeutics, Consultant fee, speaker bureau, advisory committee, etc.: Genentech

Joel Fain: Employee: Pfizer,Inc

James Cwengros: Employee: Pfizer,Inc

Kafi Sanders: Employee: Pfizer,Inc

No Product/Research Disclosure Information

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