Exploring patients' experiences with wAIHA and the content validity of the FACIT-fatigue: a qualitative interview study.
| Title: | Exploring patients' experiences with wAIHA and the content validity of the FACIT-fatigue: a qualitative interview study. |
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| Authors: | Shukla A; IQVIA, Bangalore, Karnataka, India.; Cooper O; IQVIA, London, UK.; Jones K; wAIHA Warriors, Marion, OH, USA.; Hill QA; Leeds Teaching Hospitals NHS Trust, Leeds, UK.; McCrae K; The Cleveland Clinic, Cleveland, OH, USA.; Fattizzo B; Università Degli Studi Di Milano-Bicocca, Milan, Italy.; Kostikas M; IQVIA, Herikerbergweg 314, 1101 CT, Amsterdam, The Netherlands.; Krol M; IQVIA, Herikerbergweg 314, 1101 CT, Amsterdam, The Netherlands. marieke.krol@iqvia.com.; Bascle S; Sanofi, Gentilly, France.; Bozzi S; Sanofi, Gentilly, France.; Daak A; Sanofi, Cambridge, MA, USA.; Maher J; Sanofi, Reading, UK. |
| Source: | Orphanet journal of rare diseases [Orphanet J Rare Dis] 2025 Aug 06; Vol. 20 (1), pp. 403. Date of Electronic Publication: 2025 Aug 06. |
| Publication Type: | Journal Article |
| Language: | English |
| Journal Info: | Publisher: BioMed Central Country of Publication: England NLM ID: 101266602 Publication Model: Electronic Cited Medium: Internet ISSN: 1750-1172 (Electronic) Linking ISSN: 17501172 NLM ISO Abbreviation: Orphanet J Rare Dis Subsets: MEDLINE |
| Imprint Name(s): | Original Publication: [London] : BioMed Central, 2006- |
| MeSH Terms: | Fatigue*/physiopathology ; Anemia, Hemolytic, Autoimmune*/physiopathology; Humans ; Middle Aged ; Female ; Male ; Adult ; Aged ; Qualitative Research ; Interviews as Topic |
| Abstract: | Background: Warm Autoimmune Hemolytic Anemia (wAIHA) is a rare form of anemia caused by premature destruction of red cells. This qualitative study aimed to understand the patient experience with wAIHA and provide evidence of FACIT-Fatigue scale validity by interviewing adult wAIHA patients along with examining patients' definitions of meaningful change for item 1 of the FACIT Fatigue.; Methods: Hybrid Concept Elicitation (CE) and Cognitive Debriefing (CD) interviews, lasting approximately 75 min, were conducted with US-based adults diagnosed with wAIHA. The CE part of the interview focused on understanding the patient's experiences with wAIHA, particularly the signs/symptoms, and impacts. The CD part evaluated patient's feedback on clarity, relevance, and comprehensibility of the FACIT-Fatigue, and examined their interpretation of meaningful change for item 1.; Results: Nineteen adult wAIHA patients (mean age 49.7 years) from the US were interviewed, 31 signs/symptoms were identified with fatigue being the most common (reported by all patients, 100%) and bothersome. In addition, many patients experienced shortness of breath (95%) and skin/eye color change (74%). Other common symptoms included headaches (63%), dark/brown urine (63%), heart palpitations (63%), and dizziness (53%). The study also found that wAIHA can have negative impact on patients' emotional functioning, physical functioning, daily activities, social and professional life. The most frequently reported impacts included feeling anxious or scared (79%), difficulty climbing stairs (74%), needing help from others (74%), and reduced physical strength/mobility (74%). Patients also frequently reported feelings of anger/frustration (68%), needing frequent rests (68%), difficulty walking (68%), feeling worrisome (53%), and impacts on social activities (53%). A novel conceptual disease model was developed, highlighting the associations between symptoms and their impacts on patients' lives. Patients indicated the FACIT-Fatigue was clear, relevant, and appropriate for measuring their wAIHA related fatigue. A 1-point score change in the item 1 was considered meaningful for both improvement and worsening of their fatigue by most patients.; Conclusions: The study offers insight into the patient experience with wAIHA, detailing common symptoms and impacts on daily life as part of wAIHA conceptual disease model. It also supports the content validity of the FACIT-Fatigue instrument for assessing fatigue in wAIHA patients.; (© 2025. The Author(s).) |
| Competing Interests: | Declarations. Ethics approval and consent to participate: This study was conducted in accordance with the ethical principles that have their origin in the Declaration of Helsinki and that are consistent with Good Clinical Practice and applicable regulatory requirements. The research protocol, interview guide, and all patient communication templates were reviewed and approved by WIRB-Copernicus Group (WCG) Institutional Review Board (IRB) prior to the study commencing (#20225959) and all patients gave written informed consent to participate. Consent for publication: Not applicable. Competing interests: Joshua Maher, Stephanie Bascle, Ahmed Daak, and Sylvie Bozzi are employees and may hold stock and/or stock options in Sanofi. Anubha Shukla, Owen Cooper, Michael Kostikas and Marieke Krol are employees of IQVIA who provided research services for Sanofi for this study. Karen Jones is an employee of wAIHA Warriors. wAIHA Warriors received remuneration from IQVIA for assisting in identifying and recruiting patients with wAIHA to participate in this study. Quentin Hill provides consultancy to Alpine, Amgen, Argenx, Gliknik, Incyte, Immunovant, Janssen, Novartis, ReAlta, Sanofi and Sobi, and is speaker honoraria at Grifols and Novartis. |
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| Contributed Indexing: | Keywords: Anaemia; Concept elicitation; Content validity; FACIT-Fatigue; Patient experience; Patient interviews; WAIHA |
| Entry Date(s): | Date Created: 20250807 Date Completed: 20250807 Latest Revision: 20250810 |
| Update Code: | 20260130 |
| PubMed Central ID: | PMC12326857 |
| DOI: | 10.1186/s13023-025-03767-4 |
| PMID: | 40770380 |
| Database: | MEDLINE |
Journal Article