Warm autoimmune hemolytic anemia (wAIHA) is a rare autoimmune disease in which red blood cells are targeted by autoantibodies and prematurely destroyed (NORD. Warm Autoimmune Hemolytic Anemia, 2022), causing a range of signs and symptoms (S/S) that can impact an individual's health-related quality of life (HRQoL). Fatigue is a key aspect of the wAIHA patient experience and an important symptom to assess in a clinical trial setting via a patient-reported outcome (PRO) questionnaire (Love, et. al EHA, 2024). Little research has been published on the patient experience of wAIHA specifically, including the evaluation of PRO questionnaires in this population.

This study used semi-structured qualitative interviews with individuals with wAIHA to 1) characterize the patient experience with S/S and HRQoL impacts of wAIHA via concept elicitation (CE), and 2) review the Patient-Reported Outcomes Measurement Information System Fatigue Short Form 13a (PROMIS Fatigue 13a) via cognitive debriefing (CD) to evaluate the questionnaire's comprehensibility and relevance for assessing fatigue in this population. All interview materials and processes underwent ethics review and approval prior to participant enrollment.

A total of 15 CE and 20 CD interviews were conducted virtually between January and October of 2023 with 20 adults in the United States who had a confirmed diagnosis of wAIHA. Participants were identified and recruited through patient advocacy groups and non-profit organizations. Verbatim interview transcripts were qualitatively coded, and coded text was organized for analysis. CE interview data were analyzed for concept frequency and description. CD interview data were analyzed for concept relevance and participant interpretation of the instructions, items, and response options of the PROMIS Fatigue 13a (a 13-item PRO questionnaire assessing fatigue and its impact on daily activities and function).

Thirty unique wAIHA-related S/S and 76 HRQoL impacts were elicited from the CE interviews.Fatigue was the only S/S concept reported by all participants (n=15/15, 100%), with a majority of participants reporting it as their most bothersome S/S (n=13/15, 87%) and the S/S most important to improve with treatment (n=9/15, 60%). The most frequently reported HRQoL impact concept was reduced social interaction (n=11/15, 73%).

All 20 CD participants who reviewed the PROMIS Fatigue 13a interpreted the instructions as intended, although one participant did not correctly interpret the recall period of the “past 7 days”, referring instead to only a five-day period of Monday through Friday. Three participants (n=3/20, 15%) provided suggestions to revise the instructions, two of which suggested the recall period be shorter, and one of which suggested the recall period be longer. Nearly all participants (>93%) interpreted each item as intended, and most (>70%) reported that the way each individual item was worded was clear. The most notable issue identified for items was related to the item “I feel listless (‘washed out‘)”, which five participants reported was redundant with other items or that the term could be further clarified. Nearly all participants (>93%) interpreted each of the five response options as intended. Although eight participants (n=8/20, 40%) indicated that the response options could be clarified, the only issue reported by more than one participant was that the reversed directionality of the response scale for items “I have energy” and “I am able to do my usual activities” may be confusing for respondents. Most participants (72%) reported that all items in the questionnaire were relevant to their experience with wAIHA, except item “I am too tired to eat” which five participants (25%) stated was not relevant.

Results demonstrate the varied S/S experience of wAIHA and the substantive ways life can be impacted by this condition. Fatigue was shown to be a key symptom of wAIHA from the patient perspective. Results of the PROMIS Fatigue 13a CD indicate that participants were able to read and interpret the questionnaire as intended and that the concepts contained in the questionnaire (i.e., fatigue and fatigue-related impacts) are relevant to patients' experience with wAIHA. These results indicate the PROMIS Fatigue 13a is suitable for use in the evaluation of wAIHA in adults, as it assesses key concepts relevant to the wAIHA experience in ways that are easily understood and relevant to wAIHA patients.

Disclosures

D'Alessio:Novartis Pharmaceuticals: Current Employment, Current holder of stock options in a privately-held company. Schulte:Novartis Pharmaceuticals: Current Employment, Current holder of stock options in a privately-held company. Mathias:Disc Medicine: Consultancy. Murakhovskaya:Apellis: Consultancy; Alexion: Consultancy; Novartis: Consultancy; Sanofi: Consultancy; Janssen: Consultancy; Janssen: Other: Steering committee; Alpine: Membership on an entity's Board of Directors or advisory committees.

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2024
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