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Introduction: In recent years, there has been increased focus on individualizing treatment for persons with hemophilia including pharmacokinetic‐guided (PK) dosing. Aims: In this retrospective study clinical outcomes before and after PK‐guided prophylaxis were examined. Materials and methods: Eight Haemophilia Treatment Centres from the United States participated in the study and included 132 patients classified into two cohorts: those undergoing a PK‐assessment for product switch (switchers) or to optimize treatment (non‐switchers). Subset analyses for the two most common products and patients with dosing per prescription label were included for annual bleeding rates (ABR), mean weekly consumption outcomes, and annualized cost of prophylaxis. Results: The most common products before and after index date were octocog alfa, rurioctocog alfa pegol, and efmoroctocog alfa. Seventy‐four (56%) patients were identified as switchers and 58 (44%) patients were classified as non‐switchers. The majority of patients (78.0%) experienced either a decrease in ABR post‐index or maintained 0 ABR during pre‐ and post‐index time periods, with similar proportions identified in both switchers (77.0%) and non‐switchers (79.3%) populations. Non‐switchers were identified as having no significant change in cost of therapy, while switchers experienced increased cost of therapy driven by higher price of extended half‐life products. Within subset analyses, patients receiving rurioctocog alfa pegol and efmoroctocog alfa had mean ABR under 1 after index date. Conclusion: PK‐guided prophylaxis has the potential to improve clinical outcomes without increase in cost of therapy for patients maintaining product and can aid in maintaining effective protection against bleeds in those switching product. [ABSTRACT FROM AUTHOR]

Introduction: People with haemophilia rely on specialists for their care, yet the specific dosing regimens of treatments prescribed by these specialists have not been widely studied. Aim: The objective of this study is to describe trends in clinician prescribing practices for the management of haemophilia in the United States (US). Methods: We administered surveys to members of the Hemostasis and Thrombosis Research Society via paper surveys at its in‐person annual symposia in 1999 and 2015, and an online survey in 2021. The surveys collected information on haemophilia treatments including factor dosing, inhibitor therapy and gene therapy. Results: Clinicians treating haemophilia for more than 50% of their practice time have increased from 37.5% of respondents in 1999 to 46.3% in 2021. Clinicians prescribing factor concentrates at >40 units/kg for routine bleeding events increased from 0% in 1999 to 29.3% in 2021 in haemophilia A (HA) and from 22.5% to 87.8% in haemophilia B (HB). In 2021, the clinicians reported prescribing emicizumab to treat HA patients (>89.5% paediatric, >85.7% adult) with or without inhibitors at least some of the time. Approximately 78.0% of respondents reported that they expected to recommend gene therapy at least some of time. Conclusion: These data indicate changing trends in prescribing practices among US haemophilia specialists during the past 22 years. Preference for high doses of factor (>40 units/kg) has increased during this period. Emicizumab prophylaxis has been prescribed for patients with and without HA inhibitors. Clinicians expect gene therapy to have value for some haemophilia patients. [ABSTRACT FROM AUTHOR]

Purpose: Guidelines for the use of goal attainment scaling (GAS) recommend that the patient specify at least three goals. Even so, this may not always be feasible or align with patient preferences. Investigations into the psychometric properties of GAS using three or more goals largely support its reliability, validity, and responsiveness compared with standard measures. As evaluations of responsiveness rely on variability estimates, this metric may be impacted when GAS is based on fewer than three goals. For this reason, we investigated the responsiveness of one- and two-goal GAS. Methods: Secondary analyses were conducted on data from a mixed sample of pediatric, adolescent and adult subjects with hemophilia A. The standardized response mean (SRM) and its 95% confidence intervals (CI) were used to assess responsiveness of one- and two-goal GAS at six and twelve weeks. Results: Both one-goal and two-goal GAS demonstrated similar responsiveness to change at 6-week (Patient-Rated GAS: one-goal SRM [95% CI] = 0.70 [0.45–1.08], two-goal = 0.96 [0.68–1.30]; Clinician-Rated GAS: one-goal = 1.26 [0.81–1.77], two-goal = 1.01 [0.73–1.32]) and 12-week follow-up (Patient-Rated GAS: one-goal SRM [95% CI] = 1.14 [0.53–1.71], two-goal = 1.35 [0.92–1.82]; Clinician-Rated GAS: one-goal = 1.71 [1.12–2.30], two-goal = 1.48 [1.02–2.02]). Larger SRMs were observed for clinician-rated GAS, but all were within the rubric of a large effect size. Conclusions: One-goal GAS is responsive to change in a clinical population. Further research is recommended in a larger sample where responsiveness of one- and multiple-goal GAS can be compared [ABSTRACT FROM AUTHOR]