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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: Health literacy (HL) and health numeracy (HN) are underestimated barriers to treatment adherence in patients with haemophilia. Aim: To test the ability of an educational intervention to improve knowledge, HL, HN, adherence and joint health in adolescent and young adult (AYA) males with haemophilia. Methods: We performed a longitudinal pilot study of 41 participants aged 12–21 years with haemophilia A or B during two clinic visits 6–12 months apart. The first visit included a comprehensive pre‐intervention assessment: demographics, knowledge survey, Montreal Cognitive Assessment testing, 5‐question tool to assess baseline HN, assessment of HL with the Rapid Estimate of Adolescent Literacy in Medicine tool, history of adherence and Haemophilia Joint Health Score (HJHS). An educational intervention using a visual aid explained basic pharmacokinetic (PK) concepts and personal teaching regarding haemophilia treatment regimens was used during this visit. The second visit included a post‐intervention assessment: a reassessment of knowledge, HL, HN, HJHS, adherence to prescribed therapy and number of joint bleeds since the pre‐intervention visit. Results: Forty‐one males with haemophilia A or B were enrolled in the study. Of these, 33 completed the post‐intervention assessment. Knowledge (p =.002) and HN (p =.05) were significantly improved post‐intervention, although the HL, number of joint bleeds, adherence to prescribed therapy and HJHS were not. Conclusions: Participants with low HL and/or HN may benefit from alternate methods of education such as audiovisual material. Education using audiovisual materials improved knowledge and HN in this study; however, this did not affect adherence to prescribed therapy. [ABSTRACT FROM AUTHOR]

Introduction: BAY 94-9027 is a newly developed extended half-life product to treat haemophilia, allowing for fewer injections than with standard products. This post hoc analysis aimed to compare physicians' and patients' opinions on BAY 94-9027 prophylaxis, and explore how qualitative interview data is aligned with the data from the Haemophilia-specific Quality of Life questionnaire for Adults (Haemo-QoL-A).Methods: Exploratory qualitative interviews were conducted with physicians and patients by phone upon the exit of patients from the PROTECT VIII extension phase following a semi-directed guide. In this post hoc analysis, all transcripts were reviewed and reported concepts were compared to assess the level of concordance between physicians and patients. These qualitative data were compared with the Haemo-QoL-A mean global and subscale scores at baseline and end of main phase (36 weeks later).Results: Ten physicians and 16 patients (mean age 47 years) from Israel, the Netherlands and the USA were interviewed. Significant improvements were reported by all physicians from baseline [e.g. lower frequency of bleeds (80%), improvement in emotional functioning (90%)], which is in concordance with patients' reports. The improved confidence reported by physicians cascaded to greater participation in various activities, resulting in a better perceived emotional state and a significant improvement on the Haemo-QoL-A emotional impact subscale score (p = 0.04) between baseline and end of main phase. Most physicians (80%) reported improvement in bleed frequency, as patients did (88%). Improvement in physical functioning or mobility was not consistently reported in this 8-month study.Conclusion: Interviewed physicians and patients generally agreed on the beneficial impact of BAY 94-9027, specifically regarding the increased level of self-confidence in patients and its subsequent positive impact on patients' lives. These findings supported the observed improvement on the Haemo-QoL-A emotional impact subscale. Overall, this study highlights the concordance between physician and patient perspective on the positive experience with BAY 94-9027. [ABSTRACT FROM AUTHOR]

Aim/objective: Understanding pharmacokinetic (PK) differences between standard and extended half-life (EHL) products is important, particularly for factor IX (FIX), where differences are more significant than for factor VIII. Two single-dose PK trials showed N9-GP achieves higher FIX levels and greater area-under-the-curve than pdFIX, rFIX, and rFIXFc through higher recovery and longer terminal half-life. In paradigm 7, N9-GP demonstrated consistently favorable PK characteristics compared with rFIXFc. Collins et al explored population PK differences between N9-GP and pdFIX/rFIX based upon paradigm 1 data. This analysis uses population PK models based upon the paradigm 7 trial. Methods: 15 patients (21–65 years) with hemophilia B received single 50-IU/kg doses of N9-GP and rFIXFc ≥21 days apart. A population PK model developed from single-dose PK profiles simulated plasma FIX activity following dosing for surgery and on-demand treatment of bleeds. Simulations explored doses and frequencies required to sustain target World Federation of Hemophilia (WFH) factor activity levels. Results: PK profiles of N9-GP and rFIXFc were described by one- and three-compartment models, respectively. Simulations predicted significantly reduced dosing frequency and consumption for N9-GP than rFIXFc. For severe bleeds, a single N9-GP dose (80 IU/kg) is sufficient to maintain WFH-recommended FIX levels, whereas multiple rFIXFc doses are required. For surgery, redosing in the first week with N9-GP is modeled at day 6 vs rFIXFc dosing at 6, 30, 54, 78, and 126 hrs. For life-threatening bleeds, N9-GP is required at days 0, 3, 6, 13, and 18 vs rFIXFc redosing after 6 hrs with 10 additional doses at 24-, 48-, and 72 hr intervals. Conclusion: PK modeling approaches based upon direct comparative studies offer insights into PK differences between EHL FIX products. Model simulations show N9-GP may allow on-demand treatment and perioperative management with 55–75% fewer injections and 65–74% lower overall factor concentrate consumption than rFIXFc. [ABSTRACT FROM AUTHOR]

Hemophilia is a congenital bleeding disorder characterized by episodic hemorrhages that can be spontaneous or trauma related and can pose challenges in the emergency department both in diagnosis and management. Timely infusions of hemostatic clotting factors can control bleeding in most circumstances and significantly minimize sequelae. Hemorrhages in some anatomical areas such as the central nervous system, within the abdomen, or into restricted spaces in extremities require a high index of suspicion for early detection. Patients with hemophilia with inhibitors against specific clotting factors can be particularly challenging to treat. Patients with acquired hemophilia can present with severe spontaneous bleeding. Prompt correction of factor deficiencies by infusing an appropriately dosed replacement product is fundamental to effective emergency hemophilia management. [Copyright &y& Elsevier]