Prophylactic use of treatment is important for good outcomes in haemophilia, yet adherence can be suboptimal. To better understand the relationship between treatment adherence and patients' beliefs about treatment there is a need to quantify patients' treatment attitudes. The aim of this study was to develop a brief, clinically relevant, patient-reported outcome (PRO) to measure ease of use and patients' preference for haemophilia treatment. A 40-item questionnaire was completed by male adults with haemophilia A from Austria, Germany, Italy, Spain and the UK. Robust statistical methods for item evaluation including item-level statistics, dimensionality analyses and input from clinical and outcomes experts were used to inform item reduction. Retained items were subjected to psychometric evaluation including exploratory factor analysis (EFA), known-groups validity and internal consistency reliability. 273 patients completed the questionnaire. Of the 40 items, 28 items were flagged for possible deletion based on item-level statistics, three of which were retained due to clinical relevance. Two items had acceptable statistical performance but were deleted based on low clinical relevance. A total of 13 items were retained. EFA produced a conceptually defined 5-factor solution. The survey had acceptable known-groups validity and internal consistency. Refinements were made to wording and scoring, and one new item was added to assess general ease of use, resulting in a 14-item questionnaire - the HaemoPREF. Preliminary measurement properties of the HaemoPREF support the instrument to evaluate patient perception and preference for haemophilia treatment. Further psychometric evaluation is required to examine and confirm the measurement properties of the scale.
A597Objectives: Haemophilia A treatment involves replacement of missing clotting factor (FVIII) by intravenous infusion prophylactically or on-demand. Treatment adherence is influenced by patients' beliefs about their condition, treatment and side effects; mode of administration can also be a significant barrier. This study aimed to assess the psychometric properties of a survey evaluating patient perception and preference for haemophilia A treatment. MethOds: A 40-item survey (derived from existing literature) was developed to assess patient perception and preference for haemophilia A treatment and delivery systems in five European countries. Item refinement and analyses involved: 1) item response and dimensionality analyses using classical test theory; 2) finalisation of items based on data analysis and clinical relevance; 3) scoring development; 4) psychometric testing of the resulting scores including 4a) Rasch analysis, factor analysis, item-level discriminant validity tests and item response distributions; 4b) internal consistency reliability; and 4c) known-groups validity. Results: A total of 273 male patients with haemophilia A completed the survey. The results support the survey's construct, known-groups; item-level convergent and divergent validity; and internal consistency reliability. A five-factor solution was observed with the following subscales: 1) Ease of using clotting factor treatment; 2) Impact of clotting factor treatment; 3) Risk associated with clotting factor treatment; 4) Burden of clotting factor treatment; and 5) Influence of others on treatment choices. Twenty-seven items were deleted based on redundancy identified through poor psychometric performance and low clinical relevance, and one item was added (to provide a rating on ease of use of treatment which was considered missing from a content validity perspective) resulting in a 14-item scale. cOnclusiOns: A sequential process of item evaluation and reduction resulted in a short, patient-completed 'Perceptions of clotting factor treatment' survey -a brief, psychometrically tested method assessing patient perceptions and preference for clotting factor treatment in haemophilia A.
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