Pregnancy loss in individuals with von Willebrand disease and unspecified mucocutaneous bleeding disorders: a multicenter cohort study.

Background: While bleeding around pregnancy is well described in von Willebrand disease (VWD), the risk of pregnancy loss is less certain.

Objectives: We aimed to describe the frequency of pregnancy loss in females with VWD compared with those with a similar mucocutaneous bleeding phenotype and no VWD or compared with nonbleeding disorder controls.

Methods: Female patients were consecutively approached in 8 specialty bleeding disorder clinics between 2014 and 2023.

Factor V haemostatic diathesis impairing thrombin activation, membrane binding and circulating antigen level due to a novel compound heterozygous mutation, Leu1821Ser and Gly2192Cys.

Introduction: Congenital factor V (FV) deficiency is a rare clotting disorder affecting ∼1 in 1,000,000, with bleeding severity that ranges broadly for poorly understood reasons.

Aim: To help understand the molecular basis of the observed phenotype in FV deficient patients, the genetics and biochemistry causing a patient's FV deficiency were evaluated.

Methods And Results: A 71-year-old female, who had serious life-long bleeding upon provocation and profound menorrhagia that lead to hysterectomy, was found to have 3% of normal plasma FV antigen with normal electrophoretic mobility.

Physician Assistant School Applicants' Perceptions of Virtual Interviews.

Purpose: To evaluate the attitudes of applicants of virtual physician assistant (PA) school interviews during the 2021-2022 cycle which was impacted by the COVID-19 pandemic.

Methods: This quasi-experimental design studied applicants to PA programs in the United States. The study recruited applicants who interviewed virtually between March 2020 and January 2022 via an anonymous online survey.

Treatment changes in hemophilia A with chromogenic factor VIII assay implementation.

Background: The one-stage assay (OSA) and the chromogenic assay (CSA) are 2 factor VIII (FVIII) assays used for the diagnosis and classification of hemophilia A. Discrepancies between the 2 assays exist in approximately one-third of patients with mild hemophilia A.

Objectives: The objectives of this study were to report the proportion of patients with mild or moderate hemophilia A and OSA-CSA discrepancies and to report the observed changes in treatment approach prompted by the presence of assay discrepancy.

Little discrepancy between one-stage and chromogenic factor VIII (FVIII)/IX assays in a large international cohort of persons with nonsevere hemophilia A and B.

Background: Accurate measurements of coagulation factor activity form an essential part of hemophilia management and are performed by the one-stage or chromogenic assay. Current literature suggests that approximately one-third of persons with nonsevere hemophilia A exhibit assay discrepancy, albeit with a high variability between studies. Such data are scarce in nonsevere hemophilia B.

Diabetes, hepatitis C and human immunodeficiency virus influence hypertension risk differently in cohorts of haemophilia patients, veterans and the general population.

Introduction: The reasons for the high prevalence of hypertension in persons with haemophilia (PWH) are poorly understood.

Aim: To examine the roles of diabetes, Hepatitis C Virus (HCV) and Human Immunodeficiency Virus (HIV) in the etiology of hypertension for PWH.

Methods: Retrospective cross-sectional design.

Blood pressure in persons with haemophilia with a focus on haemophilia-specific risk factors.

Introduction: Persons with haemophilia (PWH) have a higher prevalence of hypertension compared to the general population, which cannot be explained entirely by the usual cardiovascular risk factors. Neutralizing antibodies (inhibitors) against clotting factors might have some relation to cardiovascular disease in PWH. However, whether inhibitors facilitate hypertension is unknown.

Risk of diabetes in haemophilia patients compared to clinic and non-clinic control cohorts.

Introduction: Ageing patients with haemophilia (PWH) develop cardiovascular risk factors impacting care. Little is known about the prevalence of diabetes in PWH and its relation to other comorbidities.

Aim: To examine the risk of diabetes for adult PWH compared to men from the general United States population (National Health and Nutrition Examination Surveys [NHANES]) and outpatients attending a Veterans Affairs Medical Center (VAMC) clinic.

Trends of outcomes and healthcare utilization following orthopaedic procedures in adults with haemophilia: A 3-decade retrospective review.

Introduction: Haemophilic arthropathy is a serious complication of haemophilia often requiring surgical intervention. It is unclear whether advances in comprehensive care are associated with a reduction in orthopaedic interventions and peri-procedural resource utilization.

Aim: To determine temporal patterns of orthopaedic interventions in persons with haemophilia (PWH), and evaluate changes in healthcare utilization and outcomes.

The impact of extended half-life factor concentrates on patient reported health outcome measures in persons with hemophilia A and hemophilia B.

Background: Recombinant factors VIII and IX Fc (rFVIIIFc/rFIXFc) were the only available extended half-life (EHL) products in Canada during 2016 to 2018.

Objectives: To evaluate if patient-reported outcome measures (PROMs) improved in Canadian persons with hemophilia who switched from standard half-life (SHL) to EHL products (rFVIIIFc/rFIXFc).

Patients/methods: This prospective cohort study enrolled persons with moderate or severe hemophilia aged ≥6 years who switched to rFVIIIFc/rFIXFc (2016-2018) and those who remained on SHL.

Factor product utilization and health outcomes in patients with haemophilia A and B on extended half-life concentrates: A Canadian observational study of real-world outcomes.

Introduction: Recombinant factors VIII and IX Fc (rFVIIIFc/rFIXFc) became available in Canada in 2016 and were the only extended half-life (EHL) factor concentrates available in Canada until 2018.

Objectives: We aim to describe the change in product utilization in Canadians who switched to rFVIIIFc/rFIXFc.

Methods: This prospective and retrospective cohort study enrolled males aged ≥6 years with moderate or severe haemophilia who switched to rFVIIIFc/rFIXFc and those who remained on standard half-life (SHL) between 2016 and 2018.

Patient-relevant health outcomes for hemophilia care: Development of an international standard outcomes set.

Background: Patient-relevant health outcomes for persons with hemophilia should be identified and prioritized to optimize and individualize care for persons with hemophilia. Therefore, an international group of persons with hemophilia and multidisciplinary health care providers set out to identify a globally applicable standard set of health outcomes relevant to all individuals with hemophilia.

Methods: A systematic literature search was performed to identify possible health outcomes and risk adjustment variables.

A Practical, One-Clinic Visit Protocol for Pharmacokinetic Profile Generation with the ADVATE myPKFiT Dosing Tool in Severe Hemophilia A Subjects.

Standard pharmacokinetic (PK) assessments are demanding for persons with hemophilia A, requiring a 72-hour washout and 5 to 11 timed blood samples. A no-washout, single-clinic visit, sparse sampling population PK (PPK) protocol is an attractive alternative. Here, we compared PK parameters obtained with a traditional washout, 6-sampling time point PPK protocol with a no-washout, single-clinic visit, reverse 2-sampling time point PPK protocol in persons with severe hemophilia A (SHA) receiving ADVATE.

Patient-centred care in haemophilia: Patient perspectives on visualization and participation in decision-making.

Introduction And Aim: The British Columbia Adult Haemophilia Team recently adopted a patient-centred care approach. The team presented visual information on an individual's pharmacokinetic profile and bleed history and encouraged patients to participate in treatment decisions. This qualitative study explored how this approach changed patients' understanding of haemophilia and how it facilitated them to make treatment decisions.

Clinical practice of personalized prophylaxis in hemophilia: Illustrations of experiences and benefits from two continents.

These cases of people with hemophilia (PWH) illustrate the importance of understanding the patient's expectations and desires and adapting treatment to meet these needs, in addition to traditional clinical targets. Population PK modeling and FVIII products with improved PK profiles provide the opportunity to individualize care and improve long-term outcomes.

Erectile dysfunction in patients with haemophilia.

Introduction: Patients with haemophilia (PWH) are experiencing a nearly normal life span with safe factor replacement therapy and effective antiviral treatments for co-infections. As a result, many ageing-related health issues are starting to emerge. One rarely discussed health issue is erectile dysfunction (ED).

Outcomes indicators and processes in transitional care in adolescents with haemophilia: A Delphi survey of Canadian haemophilia care providers.

Introduction: It is unclear which outcome indicators should be used to measure the success of haemophilia transition programs, and what are key elements of a haemophilia transition program to ensure success.

Aim: To establish by expert consensus a list of important and feasible outcome indicators of successful haemophilia transition, and a list of key elements of transition planning.

Methods: A modified two-stage Delphi survey was developed and disseminated among a panel of Canadian interdisciplinary haemophilia care providers.

Vocational experiences and career support opportunities among Canadian men with moderate and severe haemophilia.

Aim: The purpose of this research was to provide haemophilia treatment centres (HTCs) with guidance for the potential development of appropriate and timely interventions related to employment and vocational counselling and supports.

Methods: A multi-method approach was employed, where initial focus groups (n = 13) and review of the literature were used to construct a structured survey instrument (n = 75).

Results: Focus group participants made choices about employment with keen awareness of how their bleeding disorder might limit them physically; they described the role of social networks in career choices; and they wrestled with issues of disclosure.

Desmopressin in moderate hemophilia A patients: a treatment worth considering.

Desmopressin increases endogenous factor VIII levels in hemophilia A. Large inter-individual variation in the response to desmopressin is observed. Patients with a lower baseline factor VIII activity tend to show a reduced response, therefore, desmopressin is less frequently used in moderate hemophilia A patients (baseline factor VIII activity 1-5 international units/deciliter), even though factor VIII levels may rise substantially in some of them.

Inhibitor Risk Stratification and Individualized Treatment in Patients With Nonsevere Hemophilia A: A Single-Institution Practice Audit.

Inhibitor risk in nonsevere hemophilia A increases with cumulative factor VIII (FVIII) exposure days and high-risk mutations. A standardized approach to minimize inhibitor risk is warranted. Following establishment of a systematic approach to reduce inhibitor risk in nonsevere hemophilia, we evaluated the uptake of these strategies into clinical practice.

Patient powered prophylaxis: A 12-month study of individualized prophylaxis in adults with severe haemophilia A.

Introduction: Adults with severe haemophilia A (SHA) may experience breakthrough bleeds despite standard weight-based FVIII prophylaxis three times weekly. Individualized prophylaxis has evolved to optimize patient outcomes.

Aims: This study aimed to evaluate the impact of a standardized approach to individualized prophylaxis on annualized bleeding rates (ABR), factor utilization, physical activity and quality of life in adults with SHA.

Joining the patient on the path to customized prophylaxis: one hemophilia team explores the tools of engagement.

Background: The relationship between hemophilia team interventions and achievement of optimal clinical outcomes remains to be elucidated. The British Columbia Hemophilia Adult Team has previously reported results of a comprehensive approach to individualize prophylaxis that has resulted in substantially reduced bleeding rates. In order to facilitate knowledge exchange and potential replication, it was important to gain a thorough understanding of the team's approach.

Men, Masculinities, and Hemophilia.

Hemophilia is a congenital bleeding disorder that predominantly affects men. Home intravenous replacement of missing clotting factor is the most effective treatment; however, the uptake of preventative treatment (also known as prophylaxis) varies among men with hemophilia. The purpose of the current qualitative study was to describe the connections between masculinities and men's (n = 11) experiences of hemophilia across varying age groups.