Background: Goal Attainment Scaling for Hemophilia (GOAL-Hēm) is a novel, hemophilia-specific, validated patient engagement tool and patient-reported outcome instrument.Objective:We evaluated the degree to which the language of GOAL-Hēm was patientcentric and the content valuable and relevant for people with hemophilia (PWH) and/ or their caregivers.Patients/Methods: Patients and caregivers participated in one of three investigations: an online survey, one-on-one patient interviews, or a focus group. The survey and interviews assessed the clarity and relevance of the GOAL-Hēm menu items.Interviews were semistructured, audio recorded, and transcribed verbatim. Feedback from interviews was coded as "clear," "unclear," "remove," or "add." The focus group explored participants' experience of GOAL-Hēm and elicited recommendations for implementation. Quotations from focus group and interview transcripts were indexed and charted to emergent themes for analysis.Results:Participants comprised 19 adults with hemophilia and 19 caregivers of children with hemophilia (survey, n = 20; interview, n = 12; focus group, n = 6). After their feedback, 32% (15/48) of goals were retained unchanged. Further feedback resulted in the removal of 45% (286/635) of the goal descriptors, and 30% (193/635) of the retained descriptors were modified. Three new (total = 38) goals and 42 descriptors (total = 368) were added to the menu. Thematic analysis indicated that participants were enthusiastic about patient-centric language, empowered through the goal-setting process, and recognized GOAL-Hēm could measure clinically meaningful change.
Conclusion:By listening closely to patients and caregivers, we refined GOAL-Hēm to better capture the experiences of PWH, enhance content validity, and augment implementation strategies. Incorporating the patient voice is integral to developing patient-centered outcome measures.
Introduction: GOAL-H ēm is a novel, haemophilia-specific, patient-centred outcome measure (PCOM) based on goal attainment scaling, allowing people with haemophilia (PwH) to set and monitor the attainment of individualized goals for treatment.
Aim:To provide a thorough overview of the creation, validation, and development of GOAL-H ēm.Methods: Clinician workshops were held to develop a haemophilia-specific goal menu.Qualitative data from semistructured interviews with PwH and their caregivers guided further revisions to the goal menu (i.e., goal domains and descriptors). A feasibility study was performed including a 12-week, prospective, noninterventional evaluation involving clinicians and PwH at four US haemophilia treatment centres. Finally, the Patient Voice Study gathered feedback from PwH and their caregivers via an online survey, interviews, and a focus group.
Results:The feasibility study validated GOAL-H ēm with successful outcomes in construct/content validity and responsiveness, including a large effect in patient-and clinician-rated goal attainments. The Patient Voice Study led to significant refinement of GOAL-H ēm goals and descriptors, resulting in a more straightforward and relatable menu for PwH and their caregivers. Overall, GOAL-H ēm captured qualitative data in areas important to PwH and employed quantitative methods to evaluate meaningful changes in those areas. The individualized tool was well equipped to handle the complex and chronic nature of haemophilia and was endorsed by PwH, their caregivers, and clinicians.
Conclusion:The GOAL-H ēm development journey may serve as a roadmap for other PCOMs in a variety of settings, including clinical studies, haemophilia treatment centres for care planning, and as a tool to gather real-world evidence.
IntroductionIn recent years, there has been increased focus on individualizing treatment for persons with hemophilia including pharmacokinetic‐guided (PK) dosing.AimsIn this retrospective study clinical outcomes before and after PK‐guided prophylaxis were examined.Materials and methodsEight 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.ResultsThe 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.ConclusionPK‐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.
Introduction: Females with haemophilia A (HA [FHAs]) and HA carriers (HACs) have an increased risk of bleeding and complications compared to the general population. Aim: To examine the characteristics, billed annualised bleed rates (ABR b ), costs and healthcare resource utilisation for males with HA (MHAs), FHAs and HACs in the United States. Methods: Data were extracted from the IBM® MarketScan® Research Databases (Commercial and Medicaid) for claims during the index period (July 2016 to September 2018) and analysed across MHAs, FHAs and HACs. Results: Dual diagnosis females (DDFs; both HA and HAC claims) were grouped as a separate cohort. MHAs were generally younger than females (all cohorts) by up to 19 years (Commercial) and 23 years (Medicaid). ABR b >0 was more frequent in females.Factor VIII claims were higher for MHAs versus female cohorts. Joint-related health issues were reported for 24.4 and 25.6% (Commercial) and 29.3 and 26.6% (Medicaid) of MHAs and FHAs, respectively; lower rates were reported in the other two cohorts.Heavy menstrual bleeding claims occurred for approximately a fifth (Commercial) to a quarter (Medicaid) of female cohorts. All-cause emergency department and inpatient visits in FHAs and DDFs were similar to, or more frequent than, those in MHAs; bleed-related inpatient visits were infrequent. In MHAs (Commercial), mean all-cause
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