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The objective of this study was to describe the development and initial psychometric analysis of the UK English version of the Duchenne muscular dystrophy Functional Ability Self-Assessment Tool (DMDSAT), a patient-reported outcome (PRO) scale designed to measure functional ability in patients with Duchenne muscular dystrophy (DMD). Item selection was made by neuromuscular specialists and a Rasch analysis was performed to understand the psychometric properties of the DMDSAT. Instrument scores were also linked to cost of illness and health-related quality of life data. The administered version, completed by 186 UK patient–caregivers pairs, included eight items in four domains: Arm function, Mobility, Transfers, and Ventilation status. These items together successfully operationalized functional ability in DMD, with excellent targeting and reliability (Person Separation Index: 0.95; Cronbach's α: 0.93), stable item locations, and good fit to the Rasch model (mean person/item fit residual: −0.21/−0.44, SD: 0.32/1.28). Estimated item difficulty was in excellent agreement with clinical opinion (Spearman's ρ: 0.95) and instrument scores mapped well onto health economic outcomes. We show that the DMDSAT is a PRO instrument fit for purpose to measure functional ability in ambulant and non-ambulant patients with DMD. Rasch analysis augments clinical expertise in the development of robust rating scales. [ABSTRACT FROM AUTHOR]

Abstract: Introduction: In the treatment of bleeds in haemophilia patients with inhibitors, a high initial dose of recombinant Factor VIIa (rFVIIa) provides at least equal efficacy and a similar safety profile to a standard initial dose. However, no pharmacoeconomic comparison between these dosing regimens has previously been performed. Here, we assess the pharmacoeconomics of high (>120μg/kg) versus standard (≤120μg/kg) initial rFVIIa dose in inhibitor patients and the impact of time to treatment initiation on costs and outcomes. Methods: In a retrospective analysis, observational data on bleed characteristics, rFVIIa treatment, hospitalizations and outcomes were extracted from the Czech Republic HemoRec registry. Crude comparisons and generalized linear regression modelling (GLM; correcting for patient differences) were performed to compare costs and outcomes between the high and standard initial dosing groups. Results: Of 314 rFVIIa-treated bleeding episodes (12 inhibitor patients), most were spontaneous joint bleeds and 67.5% were treated with a high initial dose. In the crude comparison, high initial rFVIIa dosing was associated with a lower mean number of doses needed to achieve haemostasis compared with standard dosing (p<0.001), but higher total dose and costs (p≤0.008). However, regression analyses revealed that high initial dose was associated with similar costs (p=0.891) and a shorter time to bleeding resolution (p=0.014). Increasing time to treatment initiation increased both time to bleeding resolution and total costs. Conclusion: Compared with a standard dose, a high initial rFVIIa dose may improve treatment outcomes without increasing costs. Early treatment initiation may reduce treatment costs. [Copyright &y& Elsevier]