PB0228 - A Prospective, Observational Study of Bleeding and Quality of Life in Patients with Glanzmann Thrombasthenia in the United Kingdom: A First Report

Background: Surveys and registries document frequent bleed events in patients with Glanzmann Thrombasthenia (GT). However, no detailed, prospective diary study, characterising bleeds and associated patient-reported impacts, has previously been undertaken. Liaising with GT patients and representatives, an electronic bleed diary has been developed. Daily diaries from 30 patients will be captured over 3 months. 6 participants have been enrolled to date.

Aims: To accumulate prospective data regarding bleeding episodes, and the social and clinical impacts of bleeds in GT patients.

Methods: Patients record bleed events daily in a custom designed electronic bleed diary. They answer the question “Have you had a bleed?” at approximately the same time each day. If the answer is “yes”, they also include the anatomical site. Female patients are asked to record menstrual bleeding. For each bleed reported, patients can document symptoms, treatment(s) received, and the location where the treatment was received (e.g. in the home, at the hospital). They are also asked to record the socioeconomic impacts of individual bleeds (including missed social or leisure activities, missed work and education).

Results: Presented results in the first report will include up to date data from enrolled participants, collected over a minimum of 28 days. Key variables will include diary completion compliance, mean monthly bleed rate (excluding menstrual bleeding), as well as detailed recordings of types and anatomical locations of bleed events. Further details will include patient reported, bleed-associated symptoms and socioeconomic impacts. In addition, a thorough description of menstrual bleeds and the impacts reported by women with be presented.

Conclusion(s): This initial data review supports the previously reported high frequency bleed rates in patients with GT. It is of note that patients to date are reporting frequent impacts associated with all types of bleeds, particularly with regards to pain and the need for regular bleed treatment.