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Objectives: To describe clinical characteristics, factor consumption, and events of interest in patients with haemophilia A without inhibitors receiving prophylaxis in France, and the clinical impact of switching to Elocta® in this population. Methods: This retrospective, observational study using the Système National des Données de Santé database, analysed data from patients with haemophilia A without inhibitors using prophylactic factor VIII (FVIII) replacement therapy during 2016–2019. Clinical characteristics, treatment patterns and switches, factor consumption, and rate of events of interest were determined. In a sub-cohort of patients treated with Elocta®, clinical characteristics, factor consumption, and rate of events of interest before and after switching to Elocta® were compared. Results: For 545 patients, with mean age (standard deviation [SD]) 25.4 (17.8) years, Elocta® was the most used treatment. Bleeding events and articular non-bleeding events leading to hospitalization occurred in 15.4% and 13.9% of patients, respectively, and 9.9% of patients had surgeries or procedures related to haemophilic arthropathy. The mean (SD) FVIII product consumption was 344 (93) IU/kg/month for extended half-life treatment, and 331 (98) IU/kg/month for standard half-life products. For the sub-cohort of 146 patients, bleeding events (SD) decreased from 0.32 (2.2) to 0.09 (0.42) events/patient/year (p = 0.227) after switching to Elocta®. There was no statistically significant difference in rates of factor consumption or articular non-bleeding events before and after initiation of Elocta®. Conclusion: This study provides real-world insights that advance the understanding of treatment patterns and events of interest in patients with haemophilia A on prophylactic regimens in France. [ABSTRACT FROM AUTHOR]

Abstract Background The use of prescription anti-obesity medication (AOM) is becoming increasingly common as treatment options grow and become more accessible. However, AOM may not be without a wide range of potentially negative impacts on patient functioning and well being. The Treatment Related Impact Measure (TRIM-Weight) is an obesity treatment-specific patient reported outcomes (PRO) measure designed to assess the key impacts of prescription anti-obesity medication. This paper will present the validation findings for the TRIM-Weight. Methods The online validation battery survey was administered in four countries (the U.S., U.K., Australia, and Canada). Eligible subjects were over age eighteen, currently taking a prescription AOM and were currently or had been obese during their life. Validation analyses were conducted according to an a priori statistical analysis plan. Item level psychometric and conceptual criteria were used to refine and reduce the preliminary item pool and factor analysis to identify structural domains was performed. Reliability and validity testing was then performed and the minimally importance difference (MID) explored. Results Two hundred and eight subjects completed the survey. Twenty-one of the 43 items were dropped and a five-factor structure was achieved: Daily Life, Weight Management, Treatment Burden, Experience of Side Effects, and Psychological Health. A-priori criteria for internal consistency and test-retest coefficients for the total score and all five subscales were met. All pre-specified hypotheses for convergent and known group validity were also met with the exception of the domain of Daily Life (proven in an ad hoc analysis) as well as the 1/2 standard deviation threshold for the MID. Conclusion The development and validation of the TRIM-Weight has been conducted according to well-defined principles for the creation of a PRO measure. Based on the evidence to date, the TRIM-Weight can be considered a brief, conceptually sound, valid and reliable PRO measure.

Objective: Acute postprandial hyperglycemia (aPPHG) is often symptomatic and can be associated with behavioral changes such as impaired working memory and attention. However, there is little evidence of the impact of aPPHG on the daily lives of patients. The aim of this study was to explore the frequency and severity of aPPHG episodes and their impact on daily functioning in people with insulin-treated diabetes.Methods: Adults (n = 1200) with insulin-treated diabetes mellitus type 1 (T1DM) or 2 (T2DM), most of whom experienced aPPHG, were recruited to complete an online cross-sectional survey in the USA and UK. The survey captured self-reported severity and frequency of aPPHG episodes and included a newly developed questionnaire (aPPHG-Q) assessing the impact of aPPHG episodes on patients' daily lives. Data was analyzed separately according to diabetes type and country. Regression analyses were used to assess the relationship between severity or frequency and scores on the aPPHG-Q.Results: Between 70% and 86% of USA, and 87% and 88% of UK participants reported experiencing aPPHG episodes. Increasing frequency and severity of aPPHG episodes were associated with worse scores on the aPPHG-Q in patients with both T1DM and T2DM in both countries (p < .014) on all subscale scores (excluding the worry and concerns scores for T1DM in the UK), although the magnitude of the association was smaller for aPPHG frequency.Conclusions: Increased severity and frequency of aPPHG episodes in patients with insulin-treated diabetes is associated with greater burden and experience of symptoms, and can negatively impact daily functioning. [ABSTRACT FROM AUTHOR]

Background:  The Hemophilia Functional Ability Scoring Tool (Hemo-FAST), consisting of a patient-reported outcome (PRO) part and a clinician-reported outcome (ClinRO) part, was developed as a rapid and effective tool to assess functional mobility in clinical practice. This study (NCT04731701) aimed to validate the psychometric properties of Hemo-FAST for assessment of joint health in people with haemophilia (PwH)., Methods:  PwH A or B aged ≥18 years completed questionnaires including the PRO part of Hemo-FAST and the short-form 36 health survey (SF-36) during one study visit. Clinicians completed the Haemophilia Joint Health Score (HJHS) and the ClinRO part of Hemo-FAST at the same visit. Validation was performed using reliability, construct validity, and structure validity assessments., Results:  The study enrolled 180 PwH A or B from 14 centres across France. Estimated time to complete the PRO part was mean (standard deviation) 4.6 (5.4) minutes. PRO items showed good test-retest reliability (intraclass correlation coefficient value ≥0.70). Inter-rater values were >0.70 for 7/9 ClinRO items, indicating good reliability. All items (15 PRO; 9 ClinRO) had high internal consistency (Cronbach's coefficient alpha: 0.97). Hemo-FAST demonstrated convergent construct validity with HJHS and the SF-36 physical component and discriminant construct validity with the SF-36 mental health component. Hemo-FAST scores distinguished between subgroups of people with expected differences in joint health status, including by haemophilia severity ( p  < 0.0001)., Conclusion:  This study successfully validated Hemo-FAST as a rapid and reliable tool for the functional assessment of joint health in adults with haemophilia, both in clinical practice and clinical research settings., Competing Interests: V.B. declares support for the present manuscript from Sobi to the medical writer for medical writing; grants or contracts from CSL Behring paid to their institution; consulting fees from Sobi and LFB; and support for attending meetings and/or travel from Sobi. Y.R. declares payment or honoraria from Sobi, LFB, and Roche, and support for attending meetings and/or travel from Sobi, CSL Behring, Roche, and Novo Nordisk. D.D. and B.F. declare no conflicts of interest. N.D. declares support for the present manuscript from Sobi to their institution; consulting fees from Sobi and Octapharma; participation on a Data Safety Monitoring Board or Advisory Board for Sobi; and support for attending meetings and/or travel from Novo Nordisk, LFB, and Roche. M.-L.P.-J. declares payment or honoraria from Novo Nordisk, and support for attending meetings and/or travel from Amgen, Novo Nordisk, Octapharma, LFB, CSL Behring, and Sobi. S.Cas. declares consulting fees from Sobi; payment for expert testimony from Sobi, Novo Nordisk, LFB, and Takeda; and support for attending meetings and/or travel from Novo Nordisk, Sobi, and Roche. F.G.-V. declares payment or honoraria for oral presentations from Sobi, Roche, Takeda, and CSL Behring; support for attending meetings and/or travel from Sobi, Roche, LFB, CSL Behring, and Pfizer; and participation on a Data Safety Monitoring Board or Advisory Board for Sobi, Pfizer, and LFB. B.T. declares grants or contracts from Sobi, Novo Nordisk, Roche, Takeda, CSL Behring, and Octapharma paid to their institution; payment or honoraria from Novo Nordisk, Sobi, and CSL Behring; and support for attending meetings and/or travel from Novo Nordisk, Roche, and CSL Behring. A.H. declares payment or honoraria for oral presentations from Sobi, Roche, LFB, Takeda, Novo Nordisk, CSL Behring, and Octapharma; support for attending meetings and/or travel from Sobi, Roche, LFB, Takeda, Novo Nordisk, CSL Behring, and Octapharma; and participation on a Data Safety Monitoring Board or Advisory Board for Sobi, Roche, LFB, Takeda, Novo Nordisk, CSL Behring, and Octapharma. C.G. and N.K. are shareholders of stock or stock options of Sobi, are employees of Sobi, and declare support for the present manuscript from Sobi. M.Z. is an employee of Sobi, and declares support for the present manuscript from Sobi. S.Car. is a shareholder of stock or stock options of Sobi, is an employee of Sobi, and a deputy member of the Sobi board of directors (employee representative). E.B. is a shareholder of stock or stock options of Sobi and is an employee of Sobi. C.N. declares support for the present manuscript from Sobi; grants or contracts from Novo Nordisk and Sobi; consulting fees from Sobi; and payment or honoraria from Novo Nordisk and Sobi. A.L. declares support for the present manuscript from Sobi; grants or contracts from Sobi, Takeda, Bayer, CSL Behring, LFB, Novo Nordisk, Octopharma, Pfizer, and Roche; consulting fees from LFB, Pfizer, Roche, and Sobi; payment or honoraria from LFB, Pfizer, Roche, Sobi, and Takeda; and support for attending meetings and/or travel from CSL Behring, Novo Nordisk, Octapharma, Roche, and Sobi., (The Author(s). This is an open access article published by Thieme under the terms of the Creative Commons Attribution-NonDerivative-NonCommercial License, permitting copying and reproduction so long as the original work is given appropriate credit. Contents may not be used for commercial purposes, or adapted, remixed, transformed or built upon. (https://creativecommons.org/licenses/by-nc-nd/4.0/).)

Background: People living with hemophilia A face challenges impacting their daily lives despite treatment innovations. Previous studies have explored perceptions and treatment experiences; however, there is a lack of an evidence-based, comprehensive model to identify concepts (clinical, physical, and psychological functioning) relevant for people with hemophilia A (PwHA). Objectives: The aim of this qualitative study was to address the question: What is the humanistic and symptomatic experience of adolescents, adults, and children living with hemophilia A and what is the impact of hemophilia A on their quality of life? Methods: Participants, identified through patient associations in the UK, were male PwHA and caregivers of male PwHA receiving prophylactic treatment. Qualitative research was conducted involving semistructured telephone interviews with PwHA and caregivers between April 2020 and September 2020 in the UK. Standard analytical techniques of conceptual model development were used. Results: Of 30 participants, 23 were PwHA and 7 were caregivers. A conceptual model was produced describing patient experience of symptoms, physical functioning, treatment experiences, and the impact of symptoms and treatment on daily lives. Participants reported hemophilia-related symptoms, including bleeding, pain, and joint stiffness, as well as difficulties engaging with social and leisure activities. They also reported protection from bleeds provided by their treatment, relief from symptoms, and the resultant sense of normality. Concepts were broadly relevant across all age groups; however, psychological impacts were reported only by adult PwHA, and caregivers reported impacts related to outdoor activities, play, and education. Participants indicated that their ideal treatment would be delivered orally. Discussion: This study highlights the range of symptoms experienced by PwHA across a broad range of age groups, thus enabling the evaluation of relevant concepts across different stages of life. The research supports development of a conceptual model documenting symptoms, impacts, and treatment experience relevant to PwHA. Conclusion: Insights gathered through the interviews and resulting conceptual model support development of new therapies to address the physical and social challenges identified by PwHA and highlight a need for novel hemophilia A treatments that can ease treatment administration, provide adequate level of protection, and enable life to be lived normally., Competing Interests: Nadine McGale and Patrick Marquis are employees of Modus Outcomes, a consultancy company that received funding from Sobi for this research. Rakhee Ghelani was also an employee of Modus Outcomes at the time that the trial was conducted. Linda Bystrická, Nana Kragh, Jameel Nazir and Zalmai Hakimi are employees of Sobi.

Objectives: To evaluate joint health, pain and health-related quality of life (HRQoL) in patients with moderate/severe haemophilia A in Europe., Design: Multinational, cross-sectional survey, with retrospective data collection. Data were taken from the Adelphi Real World Haemophilia Disease Specific Programme Wave II, using surveys completed by physicians and patients between February 2020 and May 2021., Setting: Haematologists/haemato-oncologists and their patients in France, Germany, Italy, Spain and the UK., Participants: Males aged ≥18 years with moderate or severe haemophilia A (baseline clotting factor level ≤5%), without existing inhibitors and currently receiving prophylaxis. Patients were grouped into those with or without haemophilia-affected joints (HAJs) based on bleeding, radiographic, surgical, mobility and joint pain data., Primary Outcome Measure: Characterisation of humanistic and clinical outcomes in patients with or without HAJs., Results: A total of 120 physicians provided data for 351 eligible patients; 209 (59.5%) patients had HAJs and 142 (40.5%) had no HAJs. Pain/discomfort was significantly different (p=0.01) and reported more frequently in the HAJ (85.7%) vs non-HAJ group (53.3%). Pain medication use was significantly higher in the HAJ versus non-HAJ group (73.2% vs 60.6%; p=0.01). Up to half of the patients with HAJs had synovitis (49.8%) or arthropathy (48.4%), and one-third had undergone joint surgery (35.4%). Overall health status was significantly worse in the HAJ versus non-HAJ group (mean (SD) EuroQol Visual Analogue Scale score: 65.5 (19.3) vs 81.1 (14.6); p=0.01)., Conclusions: In this multinational real-world study, nearly two-thirds of adults with moderate/severe haemophilia A without inhibitors experienced HAJs despite prophylaxis. Individuals with HAJs had higher rates of pain and pain medication use, and lower HRQoL compared with those without HAJs. These data indicate that HAJs represent a clinically relevant burden and early identification/monitoring and management of affected joints should be an important consideration to help prevent long-term joint morbidity., Competing Interests: Competing interests: HDlC-R reports consultancy/advisory, honoraria, expert testimony fees and support for meeting attendance from Bayer, CSL-Behring, Novo Nordisk, Pfizer, Roche, Sobi and Takeda. LB, ZH and NK are employees of and shareholders in Sobi. NB, SO and KG are employees of Adelphi Real World., (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)

Introduction: The economic and clinical burden of haemophilia A is high. Primary prophylaxis with factor VIII replacement therapy is the recognised standard of care, but the emergence of non-factor therapies, such as emicizumab, is extending treatment options for people with haemophilia A., Aim: There are currently no direct comparisons of efficacy or cost between recombinant factor FVIII Fc-fusion protein efmoroctocog alfa (a recombinant factor FVIII Fc-fusion protein referred to herein as rFVIIIFc) and emicizumab; therefore, a cost-effectiveness model was developed to compare prophylactic treatment with rFVIIIFc versus emicizumab in patients with haemophilia A without inhibitors in the UK., Methods: The cost-effectiveness model was based on a matching-adjusted indirect comparison and included male patients, aged ≥12 years, with haemophilia A without inhibitors. The model was designed as a Markov process with a flexible lifelong time horizon, and cost-effectiveness was presented as an incremental cost-effectiveness ratio. Base-case analysis and sensitivity analyses (including scenario analyses, one-way deterministic sensitivity analysis [DSA] and probability sensitivity analysis [PSA]) were performed using the following treatment strategies: individualised prophylaxis with rFVIIIFc and prophylaxis with emicizumab administered once weekly (scenario analyses used regimens of once every 2 weeks or once every 4 weeks)., Results: Base-case analysis, DSA and PSA indicated that, compared with emicizumab administered once weekly, rFVIIIFc individualised prophylaxis was the dominant treatment strategy, with lower costs, a greater number of quality-adjusted life years, and a lower number of bleeds., Conclusions: rFVIIIFc has proven efficacy and is cost-effective compared with emicizumab, providing clinicians with a viable treatment option to improve the health outcomes for adults and adolescents with haemophilia A in the UK., (© 2022 The Authors. European Journal of Haematology published by John Wiley & Sons Ltd.)