Development of the Alopecia Areata Patient Priority Outcomes Instrument: A Qualitative Study

Abstract

Introduction: Although alopecia areata (AA) profoundly impacts patients' physical appearance, emotional state, and daily activities, no treatment approved for AA currently exists. Patient-reported outcome (PRO) instruments currently used to capture patients' AA experiences do not meet the requirements to support claims of treatment benefit as described in the US Food and Drug Administration's 2009 PRO guidance. Our objective was to explore the consequences and priority treatment outcomes among individuals with AA and develop a PRO measure consistent with regulatory requirements that assesses these priorities and represents clinical benefit from the AA patient perspective. Methods: Targeted literature and instrument reviews informed an initial concept set. Concept elicitation interviews with 20 adults with AA confirmed the relevance and importance of the initial concepts, identified additional relevant concepts, and informed an AA consequence model. Thematic analysis yielded a draft item pool, which was evaluated through two iterative rounds of cognitive debriefing interviews with 16 patients with AA (9 adults; 7 adolescents). Results: Hair loss was the primary consequence of importance to patients with AA. Patients emphasized the need to differentiate hair loss by location: scalp, eyebrows, eyelashes, and body. Consequences of AA include difficulty conducting daily activities, particularly outdoor activities and exercise, and emotional impacts such as sadness, frustration, and negative selfimage. Following cognitive debriefing interviews, 11 items were included to form the Alopecia Areata Patient Priority Outcome (AAPPO), assessing AA-related symptoms and impacts over the past week. Conclusions: The AAPPO is a novel, contentvalid PRO that captures the consequences of AA of the highest priority to patients.

Validation of the Alopecia Areata Patient Priority Outcomes (AAPPO) Questionnaire in Adults and Adolescents with Alopecia Areata

“…Although the CFA results provided support for consideration of an overall hair loss subscale (Items 1-4), patients with AA present clinically with hair loss on the scalp and/or any hair-bearing area on the body [38]. Moreover, the qualitative evidence obtained during the AAPPO development process demonstrated that not all patients experienced hair loss in each measured location (i.e., scalp, eyebrows, eyelashes, and the body) or prioritized hair loss from each area equally [7]. Therefore, the decision was made to score the four individual Hair Loss items separately and not as a four-item summed domain score.…”

“…AAPPO The 11-item AAPPO [7] contains four items, categorized as ''Hair Loss'' from (1) the scalp, (2) eyebrows, (3) eyelashes, and (4) body, and asks the patient to describe the current amount of hair loss using a five-point response scale that ranges from 0 (no hair loss) to 4 (complete hair loss: ''I do not have any hair on my [insert hair loss area]''). The remaining seven items ask the patient to rate Emotional Symptoms (4 items) and Activity Limitations (3 items) of AA over the past week on a 5-point scale ranging from ''Never'' to ''Always.''…”

“…The Medical Outcomes Study (MOS) SF-36v2 Acute is a generic health status instrument that measures concepts of healthrelated quality of life over the past week for 8 general health domains: (1) physical functioning, (2) role limitations due to physical health, (3) bodily pain, (4) general health perceptions, (5) vitality, (6) social functioning, (7) role limitations due to emotional problems, and (8) mental health [15,16]. These domains can also be summarized as Physical and Mental Component Summary (PCS and MCS) scores.…”

“…Prior content validity work in the development of the AAPPO with adults and adolescents indicated that the AAPPO appropriately assesses disease status in both age groups [7]. Therefore, analyses planned to establish the AAPPO scoring algorithm (i.e., response distributions, interitem correlations, and factor analyses) and assess reliability and construct validity were conducted with data pooled across both age groups using SAS v9.4 for Windows statistical software [17], with sensitivity analyses conducted in the separate adult and adolescent samples.…”

“…Existing AA-specific patient-reported outcome (PRO) measures are missing concepts that are a high priority to individuals with AA or employ response options and recall periods that may not sufficiently capture the impacts of AA. Thus, a novel AA-specific PRO measure, the Alopecia Areata Patient Priority Outcomes (AAPPO) tool, was developed to assess hair loss signs as well as the emotional symptoms and activity limitations from the patient's perspective [7]. Development of the AAPPO met the requirements described in the Food and Drug Administration (FDA) patient-focused guidance and was adherent to the principles of the FDA Patient-Focused Drug Development initiative [6,8].…”