Concizumab prophylaxis in persons with hemophilia A or B with inhibitors: patient-reported outcome results from the phase 3 explorer7 study.

Background: Patient-reported outcomes (PROs) reflect patient perceptions of disease and treatment and are important for evaluating new therapies.

Objectives: Evaluate the effects of once-daily concizumab prophylaxis on health-related quality of life (HRQoL), treatment burden, and treatment preference in males aged ≥12 years with hemophilia A/B with inhibitors.

Methods: Patients enrolled in the multicenter, open-label explorer7 phase 3 study (ClinicalTrials.

Cross-cultural assessment of the influence of the COVID-19 pandemic on the perceived mental health and medical experiences of persons with inherited bleeding disorders and their parents/guardians.

Background: Objectives were to 1) assess COVID-19-associated medical and psychological challenges facing persons with inherited bleeding disorders (PIBD) and their parents/guardians (PG) in Germany, the US, and the UK; 2) describe similarities and differences among these countries; 3) identify needs and opportunities for intervention by patient advocacy organizations (PAGs).

Research Design & Methods: A cross-sectional, international survey was conducted in three countries using validated psychometric instruments and investigator-developed items.

Results: Five hundred and four surveys were included.

Association of patient, treatment and disease characteristics with patient-reported outcomes: Results of the ECHO Registry.

Introduction: Patient-reported outcomes (PROs) in people living with haemophilia A (PLWHA) are often under-reported. Investigating PROs from a single study with a diverse population of PLWHA is valuable, irrespective of FVIII product or regimen.

Aim: To report available data from the Expanding Communications on Haemophilia A Outcomes (ECHO) registry investigating the associations of patient, treatment and disease characteristics with PROs and clinical outcomes in PLWHA.

Gene Therapy in Hemophilia: A Transformational Patient Experience.

Hemophilia is a bleeding disorder caused by a single absent/defective gene and characterized by a lack of functional clotting factors. People with hemophilia may experience joint damage, pain, and psychological impairments, all of which could contribute to reduced health-related quality of life (HRQoL). The current standard of care is clotting factor replacement, which is associated with regular infusions; therefore, alternative treatments such as gene therapy (GT) are in development.

The effect of a virtual child disease management programme on burden and social adjustment of caregivers of children with coagulation factor deficiencies.

Introduction: The use of virtual interventions is of interest to patients with chronic disease and healthcare professionals. This study aimed to determine the effect of virtual child disease management programme on burden and social adjustment of caregivers of children with coagulation factor deficiencies. Moreover, the effect of this intervention on children's acute pain and bleeds was assessed.

Efficacy and safety in patients with haemophilia A switching to octocog alfa (BAY 81-8973): Final results of the global real-world study, TAURUS.

Objectives: To report the final results of the 2-year TAURUS study, assessing weekly prophylaxis dosing regimens of octocog alfa (Kovaltry®/BAY 81-8973) used in standard clinical practice in patients with moderate-to-severe haemophilia A.

Methods: TAURUS (NCT02830477) is a phase 4, multinational, prospective, non-interventional, single-arm study in patients of any age with moderate or severe haemophilia A (≤5% factor [F]VIII activity). TAURUS was designed to primarily investigate weekly prophylaxis dosing regimens used in standard clinical practice.

The effect of emicizumab prophylaxis on long-term, self-reported physical health in persons with haemophilia A without factor VIII inhibitors in the HAVEN 3 and HAVEN 4 studies.

Introduction: Severe haemophilia A (HA) has a major impact on health-related quality of life (HRQoL).

Aim: Assess the impact of emicizumab on HRQoL in persons with severe HA (PwHA) without factor VIII (FVIII) inhibitors in the phase 3 HAVEN 3 and 4 studies.

Methods: This pooled analysis examines the HRQoL of PwHA aged ≥ 18 years treated with emicizumab prophylaxis via Haemophilia-Specific Quality of Life Questionnaire for Adults (Haem-A-QoL) and EuroQoL 5-Dimensions 5-levels (EQ-5D-5L).

[Impact of COVID-19 Pandemic on Medical Care of Patients with Inherited Bleeding Disorders].

Background And Objectives: Chronic diseases, such as inherited bleeding disorders (IBD) are often associated with high costs of medical care. COVID-19 containment measures, including isolation and triage, led to restrictions in the health care of chronically ill patients. The aim of the present study was to investigate the effects of the COVID-19 pandemic on the health care of IBD patients.

Health-related quality of life and health status in adolescent and adult people with haemophilia A without factor VIII inhibitors-A non-interventional study.

Introduction: Real-world data on health-related outcomes in persons with haemophilia A (PwHA) can provide useful information for improving patient care. The global, non-interventional study (NIS; NCT02476942) prospectively collected high-quality data in PwHA, including those without factor VIII (FVIII) inhibitors treated according to local routine clinical practice.

Aim: To report health-related quality of life (HRQoL) and health status of adult/adolescent PwHA without FVIII inhibitors.

Clinical phenotype, fibrinogen supplementation, and health-related quality of life in patients with afibrinogenemia.

Due to the low prevalence of afibrinogenemia, epidemiologic data on afibrinogenemia are limited, and no data are available on health-related quality of life (HRQoL). We conducted a cross-sectional international study to characterize the clinical features, the fibrinogen supplementation modalities, and their impact on HRQoL in patients with afibrinogenemia. A total of 204 patients (119 adults and 85 children) from 25 countries were included.

Challenges and key lessons from the design and implementation of an international haemophilia registry supported by a pharmaceutical company.

Introduction: Real-world data are lacking regarding the relationship between prospectively collected patient-reported outcomes (PROs), clinical outcomes and treatment in people with haemophilia (PWH). The Expanding Communications on Hemophilia A Outcomes (ECHO) registry was designed to address this data gap, but a range of difficulties led to early study closure.

Aim: To describe the challenges faced and lessons learned from implementing a multinational haemophilia registry.

Evaluation of the U.S. Adherence Questionnaires VERITAS-PRO and VERITAS-PRN for Use in Patients with Hemophilia in the German Healthcare System.

Aim:  Since the U.S. adherence instruments VERITAS-PRO and VERITAS-PRN were developed in another healthcare system, we assumed that they are not appropriate for the German solidarity healthcare system.

Health-related quality of life and caregiver burden of emicizumab in children with haemophilia A and factor VIII inhibitors-Results from the HAVEN 2 study.

Introduction: Persons with haemophilia A (PwHA) with factor (F)VIII inhibitors, including children, have impaired health-related quality of life (HRQoL). The HAVEN 2 study (NCT027955767) of paediatric PwHA with FVIII inhibitors demonstrated that subcutaneous emicizumab prophylaxis resulted in low annualizedbleed rates.

Aim: We assessed the impact of emicizumab prophylaxis on the HRQoL of children and their caregivers participating in HAVEN 2.

Determining meaningful health-related quality-of-life improvement in persons with haemophilia A using the Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL).

Introduction: The Haem-A-QoL is frequently utilized in haemophilia clinical trials and captures relevant aspects of disease impact. Thresholds for some domains 'Physical Health' (PH), 'Sports & Leisure' (S&L) and 'Total Score' (TS) have previously been identified to benchmark the amount of change that is meaningful to patients, but not been independently confirmed.

Aim: The objective of this analysis was to determine the clinically important responder (CIR) thresholds for these three domains.

Impact of COVID-19 pandemic on mental health of patients with inherited bleeding disorders in Germany.

Background: The worldwide pandemic spread of SARS-CoV-2 can lead to either respiratory infection or containment-associated isolation with possible higher impact on chronic diseases such as inherited bleeding disorders (IBD). The aim of the study was to evaluate the impact of COVID-19 on patients and caregivers of IBD patients regarding their concerns and worries related to own health, access to treatment and availability of factor concentrates and their experiences related to medical care.

Methods: Multicentre, cross-sectional study evaluating the impact of COVID-19 on mental health of IBD patients.

Outcomes in children with hemophilia A with inhibitors: Results from a noninterventional study.

Background: Data regarding management of pediatric persons with hemophilia A (PwHA) with factor VIII (FVIII) inhibitors are limited. This prospective noninterventional study (NCT02476942) evaluated annualized bleeding rates (ABRs), safety, and health-related quality of life (HRQoL) in pediatric PwHA with FVIII inhibitors.

Procedure: PwHA aged <12 years with current FVIII inhibitors and high-titer inhibitor history were enrolled.

Cross-Sectional and Longitudinal Construct Validity of the Generic KINDL-A(dult)B(rief) Questionnaire in Adults with Thrombophilia or with Hereditary and Acquired Bleeding Disorders.

Background/aims: The newly adapted generic KINDL-A(dult)B(rief) questionnaire showed satisfactory cross-sectional psychometric properties in adults with bleeding disorders or thrombophilia. This investigation aimed to evaluate its cross-sectional and longitudinal construct validity.

Methods: After ethical committee approval and written informed consent, 335 patients (mean age 51.

Cross-cultural adaptation and validation of Haem-A-QoL in Côte d'Ivoire.

Introduction: Health-related quality (HRQoL) evaluations are considered essential outcomes in the assessment of people with haemophilia. In developing countries, reliable HRQoL data are even more critical whilst enabling government agencies to develop national haemophilia care programmes. However, validated tools are not yet available in sub-Saharan African countries.

Health-Related Quality of Life, Treatment Satisfaction and Adherence Outcomes of Haemophilia Patients Living in a German Rural Region.

In the context of the 'Mobile Haemophilia Outpatient Care (MHOC)' project we aimed to gather insights into the health-related quality of life (HRQoL), treatment satisfaction (TS) and adherence of persons with haemophilia (PWHs) who get treated at the Saarland University Hospital Haemophilia Treatment Centre (HTC). PWHs were visited at home at least twice (baseline, follow-up) by trained medical staff. Individual interviews were performed to measure patients' HRQoL and TS with validated questionnaires (Haem-A-QoL/Haemo-QoL and Hemo-Sat/Hemo-Sat).

Heavy menstrual bleeding in women with inherited bleeding disorders.

Heavy menstrual bleeding (HMB) is the commonest bleeding symptom among women with inherited bleeding disorders (IBD). Since HMB starts at the very onset of menarche and continues throughout the reproductive life, the health related quality of life of these women is affected and they are at an increased risk of developing iron-deficiency anemia. Because of the entrenched stigma and taboos, women and girls are often reluctant to discuss the problem of HMB within their families and do not seek medical advice.

BAY 81-8973 prophylaxis and pharmacokinetics in haemophilia A: Interim results from the TAURUS study.

Objectives: To report interim data from TAURUS, a study assessing real-world prophylactic treatment with unmodified, full-length recombinant FVIII BAY 81-8973 (Kovaltry ; Bayer) indicated for haemophilia A.

Methods: TAURUS (NCT02830477) is an international, open-label, prospective, non-interventional, single-arm study with a one-year observation period (target N = 350). Patients have moderate or severe haemophilia A (FVIII ≤5% or ≤1%) and ≥50 exposure days to any FVIII product.

Burden on parents of children with haemophilia: The impact of sociodemographic and child's medical condition.

Aims And Objectives: To describe the perceived burden on parents of children with severe or moderate haemophilia and the impact of sociodemographic aspects and the child's medical condition on this.

Background: Parents of children with haemophilia face a multitude of demands. The child needs frequent intravenous injections, hospital visits, extra supervision and care.

Physical activity improved by adherence to prophylaxis in an Italian population of children, adolescents and adults with severe haemophilia A: the SHAPE Study.

Background: Physical activity in people with haemophilia (PWH) reduces the development of severe arthropathy, but it must be performed after regular, proper prophylaxis. Strict adherence to treatment is crucial to achieving effectiveness and established outcomes. The primary aim of this study was to collect prospective data on adherence to prophylaxis for over 36 months.

Health-related quality-of-life and treatment satisfaction of individuals with hemophilia A treated with turoctocog alfa pegol (N8-GP): a new recombinant extended half-life FVIII.

Background: Prophylactic treatment regimens lead to improvements in health-related quality-of-life (HRQoL) among individuals with hemophilia. Turoctocog alfa pegol (N8-GP) provides the benefit of extending the duration of protection from bleeding and reducing the number of injections, which is expected to impact HRQoL and treatment satisfaction (TS).

Aim: To investigate the HRQoL and TS of patients with severe hemophilia A from two phase III trials evaluating the safety and efficacy of N8-GP.

Health-related quality of life and health status in persons with haemophilia A with inhibitors: A prospective, multicentre, non-interventional study (NIS).

Introduction: Real-world data (RWD) on health-related outcomes in persons with haemophilia A (PwHA) provide insights into patient needs and can guide clinical study design. A global, prospective, non-interventional study (NIS; NCT02476942) collected detailed RWD on bleeding outcomes, health-related quality of life (HRQoL) and health status in PwHA treated per local routine clinical practice.

Aim: To report HRQoL and health status in the adult/adolescent PwHA with inhibitors cohort in the NIS.