Evaluating the psychosocial impact of hemophilia B: The Bridging Hemophilia B Experiences, Results and Opportunities into Solutions (B-HERO-S) study.

The needs of individuals living with hemophilia B, especially those with mild or moderate hemophilia and affected females, are not well understood. The Bridging Hemophilia B Experiences, Results and Opportunities into Solutions (B-HERO-S) initiative was developed in an effort to obtain greater insights into the unique issues and challenges faced by those with hemophilia B. This study explored the impact of hemophilia B on education, employment, engagement in physical activities and other psychosocial aspects of the lives of affected individuals and their families. The B-HERO-S findings reveal a number of unmet needs in the hemophilia B population, and these results may be leveraged to inform patient outreach and education initiatives.

Impact of mild to severe hemophilia on education and work by US men, women, and caregivers of children with hemophilia B: The Bridging Hemophilia B Experiences, Results and Opportunities into Solutions (B-HERO-S) study.

The psychosocial impact of hemophilia on work was recently investigated in the Hemophilia Experiences, Results and Opportunities (HERO) study. The findings revealed that hemophilia had an impact for adults with moderate/severe hemophilia and caregivers of children with hemophilia. HERO did not specifically evaluate impact on education in adults/children with mild/moderate hemophilia or the impact on employment of spouses/partners of caregivers of affected children. The Bridging Hemophilia B Experiences, Results and Opportunities into Solutions (B-HERO-S) study evaluated the impact of hemophilia on the lives of adult men/women with mild-severe hemophilia B and caregivers of boys/girls with hemophilia B and their spouses/partners. Many adults with hemophilia B (94%) reported that hemophilia had a negative effect on their ability to complete a formal education, often attributed to the inability to attend or concentrate in school as a result of hemophilia-related bleeding or pain. Most adults with hemophilia B (95%) and caregivers/partners (89%/84%) indicated that hemophilia had a negative impact on employment. Most adults with hemophilia were employed (81%), with construction/manufacturing (35%) as the most frequently reported industry; many worked in jobs requiring manual labor (39%). Of those unemployed, 62% never worked, and those who stopped working reported that they left the workforce due to financial issues (59%), including insurance coverage/co-pays, or hemophilia-related issues (55%). Nearly one-third of caregivers voluntarily left the workforce to care for children with hemophilia. These results suggest a need to focus more effort on career counseling for adults with hemophilia B and caregivers of affected children, especially around mild/moderate hemophilia, as this population may not be as well informed regarding potential impact in school and the workplace.

Management of US men, women, and children with hemophilia and methods and demographics of the Bridging Hemophilia B Experiences, Results and Opportunities into Solutions (B-HERO-S) study.

The Bridging Hemophilia B Experiences, Results and Opportunities Into Solutions (B-HERO-S) initiative was launched in an effort to address specific gaps in the understanding of the psychosocial impact of mild-moderate-severe hemophilia B. The original Hemophilia Experiences, Results and Opportunities (HERO) qualitative study evaluated the needs of people with hemophilia A or B in multiple countries; however, a majority of participants had the more common moderate-severe hemophilia A. The B-HERO-S study was designed in collaboration with the hemophilia community to evaluate the needs of adults with hemophilia B and caregivers of children with hemophilia B, including affected women and caregivers of girls with hemophilia. The report presented here describes participant demographics and comorbidities, as well as treatment regimens and access to treatment. Bleeding symptoms were reported by 27% of mothers of children with hemophilia B who participated. Women were more likely than men to self-report arthritis and depression/anxiety as comorbidities associated with hemophilia B. More adults and children with hemophilia B were on routine treatment than on on-demand treatment, and a high percentage of adults with moderate hemophilia B received routine treatment (86%). Many adults with hemophilia B (78%) and caregivers (69%) expressed concern about access to factor in the next 5 years, and of adults with hemophilia B, women more commonly experienced issues with access to factor in the past than did men (72% vs 44%). The findings of the B-HERO-S study reveal potential unmet needs of some patients with mild-moderate hemophilia B, and the results may be leveraged to inform patient outreach by hemophilia treatment centers and education initiatives.

Unmet needs in the transition to adulthood: 18- to 30-year-old people with hemophilia.

Young adults with hemophilia face unique challenges during the transition to adulthood, including issues associated with switching from pediatric to adult hematology care, building mature interpersonal relationships, and establishing an independent career with an assurance of medical insurance coverage. A greater understanding of these challenges is essential for developing effective strategies to address the specific needs of this population. These challenges may be differentiated from those of older adults with hemophilia in large part because of more extensive childhood prophylaxis and safer factor products, resulting in fewer joint problems and lower rates of HIV and HCV infections. This analysis of the changing nature and unmet needs of today's young adults entering into adult hemophilia treatment centers, as well as potential strategies for optimally addressing these needs, was developed following roundtable discussions between patients, caregivers, hematologists, and other health care professionals participating in comprehensive care. Challenges identified among young adults with hemophilia include psychosocial issues related to maturity, personal responsibility, and increased independence, as well as concerns regarding when and with whom to share information about one's hemophilia, limited awareness of educational and financial resources, and a low perceived value of regular hematology care. The initiatives proposed herein highlight important opportunities for health care professionals at pediatric and adult hemophilia treatment centers, as well as national organizations, community groups, and career counselors, to address key unmet needs of this patient population.

Administration burden associated with recombinant human growth hormone treatment: perspectives of patients and caregivers.

Patients treated with recombinant human growth hormone (rhGH) for growth hormone disorders follow a challenging treatment schedule. This study assessed patient and caregiver experiences with rhGH therapy treatment regimens. Patients 13 years or older with growth hormone deficiency and caregivers completed Web-based surveys. A total of 61 patients and 239 caregivers participated. Storage of rhGH was considered burdensome by more than a third. More than 51% considered storage "somewhat more" to "much more of a burden" relative to the burden while not traveling. "Away from home or traveling" was the most frequently endorsed reason for missing a dose. rhGH treatment while traveling is challenging because of rhGH storage burden.

Growth hormone replacement in adults: Real-world data from two large studies in US and Europe.

OBJECTIVE: This report describes the effectiveness and safety of growth hormone replacement in 3180 adult patients with growth hormone deficiency followed-up for 0.0-12.2 years in two completed, complementary, non-interventional, multicentre studies, NordiNet® International Outcome Study (IOS) (NCT00960128) and the American Norditropin® Studies: Web-Enabled Research (ANSWER) Program (NCT01009905). DESIGN: In both studies, Norditropin® (somatropin; Novo Nordisk A/S, Denmark) was administered at the discretion of the treating physician and according to routine practice. We present data on baseline characteristics, growth hormone dose, safety data and change from baseline in waist circumference, body mass index and bioimpedance (NordiNet® IOS only). RESULTS: Mean (SD) baseline characteristics (effectiveness analysis set) in NordiNet® IOS (n = 971) and ANSWER (n = 304): females, 45%; 69%; mean growth hormone dose (mg/day) (female, 0.338 [0.177]; male, 0.289 [0.157]); (female, 0.501 [0.313]; male, 0.505 [0.351]). Most patients had BMI ≥25 kg/m2. Median (P10,P90) exposure (females, 3.5 [0.42,11.0]; 1.6 [3.2; 0.3,8.6]; males, 4.1 [0.33,10.8]; 2.3 [2.9; 0.0,7.5] years). Mean (SD) change from baseline for waist circumference (-0.46 [6.38] cm [n = 403], BMI (0.30 [3.30] kg/m2 [n = 857]) and bioimpedance (-17.4 (59.19) ohm [n = 239]) were associated with growth hormone dose (waist/bioimpedance) and duration of follow-up (BMI/bioimpedance). No new safety signals were observed among patients in the full analysis set (NordiNet® IOS, n = 2321; ANSWER, n = 859). CONCLUSIONS: Long-term growth hormone replacement is associated with an improvement in body composition. The accumulated data from >10 years of follow-up support the long-term effectiveness and safety of growth hormone replacement as prescribed in clinical practice.

Educational needs of hematologists and laboratory professionals regarding factor activity assays.

INTRODUCTION: Diagnosis and management of hemophilia require accurate and precise measurements of factor activity levels. Activity is traditionally measured via one-stage (OS) clot-based assay; however, chromogenic substrate (CS) assays may be needed for certain cases. A survey was performed to understand assay-related knowledge gaps among hematologists and laboratory professionals. METHODS: Separate web-based surveys were administered to hematologists who manage hemophilia and to laboratory professionals and queried practice patterns, knowledge of/attitudes toward CS assays, and interest in continuing education. RESULTS: A total of 51 hematologists participated in this study; 67% managed hemophilia patients for ≥10 years and 24% were affiliated with a hemophilia treatment center (HTC). Most (80%) stated familiarity with general assay interpretation. Majorities of non-HTC and HTC respondents agreed that CS assays are more accurate than OS assays (62%/67%), although non-HTC hematologists indicated less understanding of when to order a CS assay (49%/67%). Fewer non-HTC respondents expressed concerns regarding the reliability of OS assays for diagnosis (38%/67%) and monitoring (38%/75%). Most (80%) expressed an interest in factor assay education, especially on available assays, efficacy, and best practices (39%). A total of 57 laboratory professionals participated, averaging 10 years in their current position; most (88%) were hospital based. More performed OS (72%) than CS (10%) or both (17%) assays; only 11% reported confidence with the interpretation of CS results. Few expressed concerns regarding the reliability of OS for diagnosis (9%) or monitoring (12%). Reported barriers to CS use included infrequent need (68%), lack of US Food and Drug Administration (FDA) approval (61%), and need for validation work (56%). Most (70%) were interested in CS assay education; top interests included advantages over traditional assays, general information on CS assays, and indications for testing (each 18%). CONCLUSION: Future educational efforts may focus on limitations of OS assays, indications for CS assay diagnosis/monitoring, and support for clinic-laboratory dialog.

The Coags Uncomplicated App: Fulfilling Educational Gaps Around Diagnosis and Laboratory Testing of Coagulation Disorders.

BACKGROUND: Patients with coagulation disorders may present to a variety of physician specialties; however, accurate and efficient diagnosis can be challenging for physicians not specialized in hematology, due to identified gaps in knowledge around appropriate laboratory assays and interpretation of test results. Coags Uncomplicated was developed to fill this unmet educational need by increasing practical knowledge of coagulation disorders among nonexpert physicians and other health care professionals (HCPs) in a point-of-care (POC) setting. OBJECTIVE: The aim of this study was to assess patterns of use of the mobile app Coags Uncomplicated, a tool designed to support education regarding accurate and efficient diagnosis of bleeding disorders. METHODS: App metrics were obtained by tracking registered user data. Additionally, a survey was distributed to registered users, to assess circumstances and frequency of use. RESULTS: The most common specialties of 7596 registered US users were hematology-oncology (n=1534, 20.19%), hematology (n=1014, 13.35%), and emergency medicine (n=1222, 16.09%); most identified as physicians (n=4082, 53.74%). Specialties accounting for the greatest numbers of screen views were hematology-oncology (99,390 views), hematology (47,808 views), emergency medicine (23,121 views), and internal medicine (22,586 views). The most common diagnostic endpoints reached were disseminated intravascular coagulation (DIC; 2713 times), liver disease effect (2108 times), and vitamin K deficiency (1584 times). Of 3424 users asked to take the survey, 262 responded (7.65%); most were physicians in direct clinical care (71%) and specialized in hematology-oncology (39%) or emergency medicine (21%). Most frequent use was reported by hematologists (69%, ≥6 times) and hematologists-oncologists (38%, ≥6 times). Most physicians (89.2%) reported using the app for patient-case-related education around appropriate use of laboratory tests in diagnostic evaluation. Physicians rated Lab Value Analyzer (mean 4.43) and Lab Test Algorithm (mean 4.46) tools highly on a 5-point "how helpful" scale and were likely to recommend the app to colleagues. CONCLUSIONS: App use among physicians and other HCPs is consistent with value as a POC educational tool, which may facilitate differential diagnoses and appropriate early consultation with hematologists.