PB1291 - A Prospective, Observational Study of Bleeding and Quality of Life in Patients with Glanzmann Thrombasthenia

Background: Glanzmann Thrombasthenia (GT) is a severe bleeding disorder. In surveys and qualitative research patients indicate frequent bleeds associated with medical and psychosocial impacts. However, the true intensity of bleeding is likely underestimated in literature and clinical practice. Patients confirm under-reporting of bleeding and avoidance of healthcare utilization due to treatment and care access hurdles.

Aims: This study aimed to provide a uniquely detailed, prospective evaluation of all patient-reported bleeding events, with contemporaneous assessment of impacts of each reported event. An interim report was previously presented; the study is now complete.

Methods: Participants with GT completed daily electronic diaries, documenting bleeding events and resulting symptoms, treatments, social and economic impacts. The diary, designed collaboratively with clinicians, patients, caregivers, and patient representatives aimed to capture impacts they considered significant. Baseline ISTH-BAT scores and quality of life assessments were conducted.

Results: : 19 participants enrolled in the study; 12 female, 7 male, mean age 42 years (range 22-66). 18 participants returned reports. At baseline, most participants reported a history of bleeding symptoms; the most frequently reported were mucocutaneous and gynaecological (table 1). Previous gastrointestinal and joint bleeds were reported by 26.1% and 21.3% respectively. Analysis of the prospectively collected data revealed a high frequency of bleeding events (table 2). At least 1 bleed/day/patient was consistently recorded across the 16-week enrolment period. Ongoing analysis will characterize the nature of these bleeds and the directly associated patient reported impacts.

Conclusion(s): This study challenges the conventional view that severe GT symptoms are rare. Large volume potentially life-threatening bleeds can occur, but critically, the detailed bleed diaries reveal that intense, frequent bleeding episodes result in a severe disease phenotype for many patients, which impacts patients’ physical and psychosocial health. These findings highlight the necessity for both patients and healthcare providers to fully recognize the true burden of GT.