Severe hemophilia and subsequent hemophilic arthropathy result in joint pain impaired health-related quality of life (HRQoL). Assessment HRQoL persons with (PWH), including underlying factors that drive differences, is important determining health care resource allocation making individualized clinical decisions.To examine potential associations between HRQoL, interference, self-reported arthritis age, employment, activity, bleed frequency, treatment center professional utilization.PWH (age...

Haemophilia is characterized by frequent haemarthrosis, leading to acute/chronic joint pain.To assess self-reported prevalence, description and management of pain in adult males with mild-to-severe haemophilia history pain/bleeding.Participants completed a survey five patient-reported outcome instruments assessing pain, functional impairment health-related quality life (HRQoL).Of 381 participants enrolled, median age was 34 years; 77% had A, 71% severe disease 65% were overweight/obese. Many...

Background Gene therapy trial results show potential to cure haemophilia A and B. Securing broad access a for lifelong chronic disease is anticipated face barriers at the individual healthcare system levels, which can be partly mitigated by harmonized planning of clinical research studies. The aim core HEM project was determine set outcome measures required evaluate efficacy, safety, comparative effectiveness value gene haemophilia. Methods Modified Delphi consensus process, based on methods...

Management of insulin administration for intake carbohydrates and physical activity can be burdensome people with type 1 diabetes on hybrid closed-loop systems. Bihormonal fully (FCL) systems could help reduce this burden. In trial, we assessed the long-term performance safety a bihormonal FCL system.

Summary. The National Pain Study was a prospective, computer‐based, descriptive survey of the pain experience persons with bleeding disorder conducted in United States over 28 month period from 2007 to 2009. aim this study (i) determine language used by patients describe and differentiate acute persistent pain, (ii) pharmacological non‐pharmacological strategies utilized control (iii) assess perceived effectiveness current management on quality life and, (iv) who provides population. One...

The Hemophilia Experiences, Results and Opportunities (HERO) initiative assessed psychosocial issues reported by people with moderate to severe hemophilia was led a multidisciplinary international advisory board. This analysis reports data from young adult respondents (aged 18–30 years), including both US overall global (including respondents) results, investigates treatment outcomes, quality of life, impacts on relationships. More adults in HERO received prophylaxis than on‐demand...

Abstract Introduction People with inherited and long‐term conditions such as haemophilia have been shown to adapt their levels of disability, often reporting better quality life (QoL) than expected from the general population (the disability paradox). Aim To investigate paradox in people United States by examining preference differences health state valuations versus population. Methods We conducted a discrete choice experiment including duration capture states based on patient‐reported...

Little data exist, especially for adolescent and young adult (AYA) persons with haemophilia (PWH), about the relationship between adherence to prescribed treatment regimen chronic pain. We examined this among PWH (moderate or severe) aged 13-25 via cross-sectional survey. Adherence was assessed using Validated Hemophilia Regimen Treatment Scale (VERITAS)-Pro VERITAS-PRN prophylactic on-demand participants respectively. VERITAS scores range from 24 (most adherent) 120 (least adherent)....

Standardized and disease-specific patient-reported outcome (PRO) instruments assessing pain, functional impairment health-related quality of life (HRQoL) in people with haemophilia (PWH) have been used studies, but infrequently comprehensive care settings for individual assessment or treatment planning.To assess the impact pain on HRQoL PWH.P-FiQ enrolled 381 adult PWH a history joint pain/bleeding included 5 PROs clinical evaluation (Hemophilia Joint Health Score v2.1 [HJHS]).Median age was...

Abstract Introduction Pain, functional impairment, anxiety, and depression associated with joint disease may affect health‐related quality of life (HRQoL) in people hemophilia. Objective To report detailed patient‐reported outcomes (PRO) assessments related to HRQoL participants the Functional Impairment, Quality Life (P‐FiQ) study. Methods Pain were assessed via PRO instruments US adult males hemophilia A or B a history pain bleeding. included EQ‐5D‐5L visual analog scale, Brief Inventory...

Depression, anxiety, pain, and treatment adherence have reciprocal effects not characterized extensively in hemophilia. This study explored the relationships between depression, chronic adults with hemophilia.Adults self-reported hemophilia A or B completed cross-sectional IMPACT QoL II survey. Depression (9-item Patient Health Questionnaire [PHQ-9]), anxiety (7-item Generalized Anxiety Disorder scale [GAD-7]), pain (Faces Pain Scale-Revised [FPS-R]), social support (Duke UNC Functional...

Introduction The National Hemophilia Foundation State of the Science Research Summit initiative sought to unify research efforts in US inherited bleeding disorders (BDs) community around key topics importance people living with BDs, lived experience experts.Areas covered This community-led and -informed project focused on six broad areas – hemophilia A or B; von Willebrand Disease (VWD), platelet dysfunctions other mucocutaneous BDs; ultra-rare unique challenges potential menstruate...

Health-related quality of life (HRQoL) is impaired in patients with hemophilia; however, the impact mild/moderate hemophilia B and affected women not well characterized.To evaluate factors that affect HRQoL adults caregivers children.US adult children completed distinct ~1-hour online surveys including patient-reported outcome instruments.In total, 299 150 participated. Adults moderate reported poorer health status (median EQ-5D-5L index score, 0.63) than those mild (0.73) or severe (0.74)...

Introduction Adherence to clotting‐factor treatment regimens, especially among adolescents and young adults ( AYA s), is under‐researched. Aim We determined factors associated with better adherence prophylaxis. Methods From April through December 2012, a convenience sample of (aged 13–25 years) persons haemophilia or von Willebrand disease VWD ) completed an online survey that assessed prescribed prophylactic regimens [Validated Haemophilia Regimen Treatment Scale VERITAS )‐Pro]. Logistic...

Gene therapy has shown promise in clinical trials for patients with haemophilia, but patient preference studies have focused on factor replacement treatments.We conducted a discrete choice experiment (DCE) to investigate the relative importance and differential preferences provide gene attributes.We surveyed male adults haemophilia United States recruited from panels including National Hemophilia Foundation Community Voices Research platform using an online survey over 4 months 2020/21....

Health-related quality of life (HRQoL) in adolescents and young adults with bleeding disorders is under-researched. We aimed to describe factors related HRQoL hemophilia A or B von Willebrand disease.A convenience sample volunteers aged 13 25 years disease completed a cross-sectional survey that assessed Physical (PCS) Mental (MCS) Component Summary scores on the SF-36 questionnaire. Quantile regression models were used assess associated HRQoL.Of 108 respondents, 79, 7, 14% had A, B,...

The Bridging Hemophilia B Experiences, Results and Opportunities Into Solutions (B-HERO-S) initiative was launched in an effort to address specific gaps the understanding of psychosocial impact mild-moderate-severe hemophilia B. original (HERO) qualitative study evaluated needs people with A or multiple countries; however, a majority participants had more common moderate-severe A. B-HERO-S designed collaboration community evaluate adults caregivers children B, including affected women girls...

Abstract The psychosocial impact of hemophilia on activities was recently investigated in the Hemophilia Experiences, Results and Opportunities ( HERO ) study (675 people with 561 caregivers children 10 countries). B may not be accurately reflected results, as ~75% respondents described issues affecting males A. To address needs this population, Bridging Into Solutions (B‐ ‐S) developed a pilot United States collaboration community. analysis reported here assessed engagement recreational...

Decades of inherited bleeding disorders (BD) research transformed severe haemophilia from a childhood killer to disorder managed across full lifespan for many in economically developed countries. Health equity, life unimpaired by disease complications, however, remains unimaginable most people with an BD (PWIBD).The National Hemophilia Foundation (NHF) and American Thrombosis Hemostasis Network (ATHN) undertook the development community-driven United States (US) Blueprint Inherited Bleeding...

Introduction Recurrent bleeding and associated pain are critical components in the management of disorders, yet scant data describe perceptions this patient population. Objective This study assessed adolescents young adults (AYAs) with haemophilia or von Willebrand disease (VWD) to determine agreement/disagreement between patients, caregivers health care providers. Methods Using an online questionnaire, AYA patients (N=89), their (N=77), providers (N=54) reported on perception, treatment...

We explored racial differences in adherence to recommended clotting factor treatment regimens, chronic pain, and quality of life (QoL) among adolescents young adults (AYAs) diagnosed with moderate or severe hemophilia. A convenience sample hemophilia patients aged 13–25 years completed an online cross-sectional survey 2012. Chronic pain was measured using the revised Faces Pain Scale (FPS-R) dichotomized as high (FPS-R ≥ 4) low < 4). QoL SF-36. Of 80 AYA participants (79 male), most had...

Abstract Introduction Epidemiologic studies suggest that joint bleeding occurs in patients with mild‐to‐moderate haemophilia, including women and girls. However, most previous on the impacts of haemophilia focus men severe disease. Aim To identify unmet needs haemophilia. Methods The Pain, Functional Impairment, Quality Life (P‐FiQ) study assessed impact pain functional impairment health‐related quality life A or B any severity. Bridging Hemophilia Experiences, Results Opportunities Into...