PB1393 - Living with Glanzmann’s Thrombasthenia: An interim report on 14 qualitative interviews from the Glanzmann’s 360 Study

Background: Glanzmann’s thrombasthenia (GT) is a rare platelet disorder caused by absent or reduced glycoprotein IIb/IIIa complex. Symptoms include bruising and abnormal bleeding, which may be severe and life-threatening.

Aims: The Glanzmanns 360 study is designed to explore the lived experience of people affected by GT and its impact on quality of life.

Methods: The study uses mixed methods, including an online survey and qualitative interviews. Recruitment is via social media and UK hospitals. Interviews are analysed using an inductive thematic methodology. The study is registered on ClinicalTrials.gov NCT05315232 and NIHR HAEM52408.

Results: 14 participants were interviewed between July 2022 and January 2023: 3 parents (2 mothers, 1 father) of children with GT (age range 2–6 years) and 10 people with GT (8 women, 3 men). Mean age of participants (children and adults with GT) was 36.7 years (range 2–78 years)

Bleeds
Reported bleeds included skin bleeds (13/14), epistaxis (11/14), mucosal bleeding (7/14), GI bleeds (6/14), scalp bleeds (2/13) and traumatic retinal bleeding (1/13). All women reported heavy menstrual bleeding; 4/8 reported prolonged and heavy post-partum bleeding.

Treatment
13/14 used tranexamic acid to treat bleeds. 12/14 had required platelet transfusions and 12/14 rFVIIa. Only 3/12 of those who had had rFVIIa were able to self-infuse; most were dependent on treatment centres to initiate and continue treatment.

Thematic Analysis
Six major themes have emerged: normalisation of abnormal bleeding; lack of understanding of GT among non-specialist healthcare providers; medical pessimism; reduced life choices; self-identification; and fatigue.

Conclusion(s): This study highlights a number of concerns and unmet needs for people with GT, including anaemia, fatigue and difficulty initiating prompt, effective treatment. There are also unrecognised psychological issues, particularly for women and their reproductive choices. Improved therapeutic and support options are required for people with GT.