Qualitative interviews to inform development of a patient reported outcome (PRO) strategy in RLBP1 retinitis pigmentosa (RLBP1 RP)

• Objectives: RLBP1 RP is a rare autosomal recessive form of retinitis pigmentosa (RP), characterized by night blindness, prolonged dark adaptation, constricted visual fields and reduced macular function. This study aimed to better understand the patient experience of RLBP1 RP and to evaluate the content validity of existing patient reported outcome (PRO) instruments in this condition. Methods: This qualitative study involved 90 minute, semi-structured, concept elicitation and cognitive debriefing interviews with patients with RLBP1 RP in Canada (n=10) and Sweden (n=11). Qualitative analysis of anonymized, verbatim transcripts was performed using Atlas.Ti software and thematic analysis methods. Participants were cognitively debriefed on The National Eye Institute Visual Functioning Questionnaire (NEI VFQ-25), Low Luminance Questionnaire (LLQ) and four items of the Visual Activities Questionnaire (VAQ). Results: Fourteen visual symptoms were reported. The symptoms most frequently reported were night blindness (n=21), difficulty adapting to changes in lighting (n=21) and difficulties seeing in bright lighting (n=18). Impacts on daily activities (n=21) and physical functioning (n=17) were important to participants. Other domains of quality of life affected included social functioning (n=21), emotional functioning (n=19), work and education (n=18), and psychological functioning (n=17). Participant understanding and interpretation of the NEI VFQ-25 and LLQ was mixed. Patients reported that examples in single items represented different levels of functional impairment. In addition, some items did not specify what lighting conditions should be considered when responding. LLQ items were more relevant to RLBP1 RP than NEI VFQ-25 items. The four VAQ items assessing light/dark adaptation were well understood and relevant to participants. There were both gaps and overlaps in conceptual coverage of the instruments. Conclusions: The symptoms of RLBP1 RP have a substantial impact on patients’ daily lives and physical functioning. Issues have been identified with conceptual coverage, rel- evance and patient understanding of the NEI VFQ-25, LLQ and VAQ in RLBP1 RP.

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MEDICIN OCH HÄLSOVETENSKAP  -- Hälsovetenskap -- Hälso- och sjukvårdsorganisation, hälsopolitik och hälsoekonomi (hsv//swe)

MEDICAL AND HEALTH SCIENCES  -- Health Sciences -- Health Care Service and Management, Health Policy and Services and Health Economy (hsv//eng)

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