Development and psychometric evaluation of a novel tool for assessing patient perception and preference for haemophilia treatment (HaemoPREF)

Abstract

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.

Adherence to prophylactic treatment in patients with haemophilia in Germany

“…Several new methods have evolved for the assessment of patient perception and preference for haemophilia treatment, e.g. the HaemoPREF and an adherence measurement implementing the Morisky Medication Adherence Scale (MMAS‐4) . The VERITAS‐Pro has greater utility than a global or informal rating of adherence because it represents a quantified and validated measure of adherence from the patient's perspective, and it divides adherence into specific areas, allowing insight into particular issues underlying non‐adherence .…”

HaemoPREF: Further evaluation of patient perception and preference for treatment in a real world setting

“…16,17 A novel patient-reported outcome (PRO) questionnaire, the HaemoPREF (Table 1) has recently been developed to evaluate patient perception and preference for haemophilia A treatment. 18,19 The HaemoPREF was developed based on a cross-sectional survey to identify factors associated with haemophilia A patients' preferences for different FVIII delivery systems. This prior survey also explored patient attributes that drive prophylactic use and tested the ease of use and patient preference for one treatment regimen and delivery system (ReFacto AF® with FuseNGo®).…”

“…20,21 Some of the measurement characteristics of the HaemoPREF have previously been evaluated during development including item/scale convergent validity, item discriminant validity, known-groups validity and internal consistency reliability. 18,19 However, other measurement characteristics such as scale convergent validity and test-retest reliability remain to be determined.…”

“…Some of the barriers to adherence relate specifically to this reconstitution step and include perceived negative consequences of reconstitution and infusion devices (eg pain caused by infusions) and their associated burden (eg, time taken to prepare treatment) . A novel patient‐reported outcome (PRO) questionnaire, the HaemoPREF (Table ) has recently been developed to evaluate patient perception and preference for haemophilia A treatment …”

“…This prior survey also explored patient attributes that drive prophylactic use and tested the ease of use and patient preference for one treatment regimen and delivery system (ReFacto AF® with FuseNGo®) . Some of the measurement characteristics of the HaemoPREF have previously been evaluated during development including item/scale convergent validity, item discriminant validity, known‐groups validity and internal consistency reliability . However, other measurement characteristics such as scale convergent validity and test‐retest reliability remain to be determined.…”