Introduction: Glanzmann thrombasthenia (GT) is a rare bleeding disorder (~1:1,000,000) caused by impaired function of platelet glycoprotein IIb/IIIa responsible for aggregation. This novel survey was designed to identify the burden of GT through better understanding of the management of the disorder and its psychosocial impact on patients and caregivers. Methods: Participants were recruited via a rare disease specialty recruiter from Comprehensive Health Education Services. Data were collected from January 31 through March 12, 2019, via a moderator-assisted online survey. On average, the survey was completed within 45 minutes. Information regarding demographics, diagnosis, treatment history, and quality of life was collected. Results: In total, 45 respondents (24 patients and 21 caregivers; 58% female) completed the survey. Many patients were born with significant bruising (76%) or bled extensively during circumcision (47%). As a result of their early symptoms, more than 50% of those surveyed were diagnosed prior to their first birthday. For others, the average time between experiencing initial symptoms and visiting a specialist was just over 1 year and to diagnosis was just over 2 years (average age, 2.6 years, range <1-38 years). Misdiagnosis with von Willebrand disease was common. Approximately 50% of patients experienced 1 bleed every day, and 13% reported over 500 bleeds per year; Most bleeds were skin bruises and mouth bleeds, but patients also reported joint/muscle and gastrointestinal bleeds. Only 24% of respondents reported being treated at a hemophilia treatment center; 71% reported visiting their hematologist regularly. The most common treatment for bleeds was antifibrinolytics (82%), followed by recombinant activated factor VII (rFVIIa; 42%). In addition, 73% reported receiving platelets and blood transfusions in the past year. Approximately 25% reported receiving more than 20 transfusions in their lifetime. Overall, 38% of patients reported having experienced refractoriness to platelets and 32% antibodies to platelets; notably, only these patients reported receiving treatment with rFVIIa. Many feared uncontrolled bleeding due to refractoriness or antibodies to platelets and stated that their health care providers (HCPs) were too quick to treat bleeds with platelets. Many female patients struggled to find a gynecologist with some knowledge of the management of menstruation, pregnancy, and childbirth in patients with GT; 11% of respondents reported menstrual bleeding that required hospitalization and/or emergency treatment. The majority (74%) of female patients reported taking hormonal contraceptives to prevent regular menstruation; some patients not taking hormones required monthly platelet transfusions. Fifty-eight percent of patients reported issues with excessive bleeding at school as children; 38% reported missing school days and 33% were bullied during childhood. In addition, 38% of adult patients and 24% of caregivers had missed work as a result of GT, and 21% of adult patients reported that their employer did not take their condition seriously. Many respondents (65%) were satisfied with the level of support they receive from their significant other, and 76% were satisfied with the level of support they receive from their friends. Conclusions: Many patients with GT are identified early in life owing to a severe phenotype, but diagnosis remains somewhat difficult. Patients with GT experience frequent bleeding episodes, commonly as bruises and nosebleeds, with 31% experiencing more than 100 bleeds per year. Additional support from outside the GT patient community is needed. Patients desire additional education for themselves and their HCPs, especially around menstruation, childbearing, and treatment options. Disclosures Duncan: Novo Nordisk Inc: Consultancy. Kellum:Novo Nordisk Inc: Membership on an entity's Board of Directors or advisory committees; Takeda: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau. Jain:Bioverativ/Sanofi: Consultancy, Membership on an entity's Board of Directors or advisory committees; Novo Nordisk Inc: Consultancy, Membership on an entity's Board of Directors or advisory committees; Bayer: Consultancy, Membership on an entity's Board of Directors or advisory committees; CSL Behring: Consultancy, Membership on an entity's Board of Directors or advisory committees; Octapharma: Consultancy, Membership on an entity's Board of Directors or advisory committees; Takeda/Shire: Consultancy, Membership on an entity's Board of Directors or advisory committees; BPL: Consultancy, Membership on an entity's Board of Directors or advisory committees. Peltier:Novo Nordisk Inc: Membership on an entity's Board of Directors or advisory committees. Cooper:Novo Nordisk Inc.: Employment. Saad:Novo Nordisk Inc: Employment.

Purpose Congenital factor VII (FVII) deficiency is a rare bleeding disorder of variable phenotype with predominantly mucocutaneous bleeding. The aim of this study was to identify the burden of FVII deficiency on patients and caregivers through a better understanding of the management and psychosocial impact of this disease. Materials and Methods A rare disease specialty recruiter from Comprehensive Health Education Services recruited participants for this online survey, which was conducted from January 31 to March 12, 2019. A moderator-assisted questionnaire was used to collect data on demographics, diagnosis, treatment, and psychosocial impact. Results Of the 45 respondents (25 patients and 20 caregivers), the majority were female (56%). Respondents reported a wide variety of initial bleeding symptoms, including bruising (58%), epistaxis (56%), and menorrhagia (36% of females). Because symptoms varied between individuals and were not always severe, diagnosis was often delayed. Mean time to obtain a diagnosis was 6.5 years and mean age at first diagnosis was 12.9 years. One-quarter (24%) of the respondents reported more than 100 bleeds of any severity over the previous year. When treating bleeds, 44% of patients reported using antifibrinolytics, and 42% reported using recombinant activated factor VII. Almost 31% of respondents reported missing schooldays as children, and 16% reported losing or resigning from a job in adulthood as a direct result of their disease. Notably, 29% of caregivers and 10% of their partners had also experienced issues with employment. Forty percent of respondents reported not participating in contact sports during childhood, and 22% continued to avoid contact sports in adulthood. Conclusion Overall, FVII deficiency has a substantial psychosocial impact, but most patients are satisfied with their disease management and are optimistic about their future. Patients desire additional educational, social, and financial support.