OBJECTIVES: Glanzmann’s Thrombasthenia (GT) is a rare bleeding disorder with deficient fibrinogen receptor αIIbβ3 leading to impaired platelet aggregation. The inability to form effective clots leads to frequent bleeding episodes ranging from low volume epistaxis to potentially life-threatening bleeds, contributing to iron deficiency anemia (IDA) and deteriorating quality of life (QOL). This study aimed to review existing research on the burden, QoL, and economic impact of GT.

METHODS: A systematic literature review (SLR) of electronic databases, including MEDLINE® and Cochrane Library, was conducted from January 2000 to April 2024, along with relevant conference proceedings. Two reviewers independently double-screened studies according to pre-defined criteria. Data extraction included objectives, study population, study design, and the study results relating to burden, QoL and economic cost impact.

RESULTS: Of 237 titles and abstracts screened, 18 relevant studies (22%) & conference abstracts (78%) were identified for review. Of the 18 studies, 7 (39%) were conducted in the US with Saudi Arabia (17%) the second most common location. The most common study design were chart reviews (56%) and surveys (22%) with sample sizes ranging from 8 to 133. Research mainly focused on the burden of bleeding (89%) and healthcare (72%), with the burden of psychosocial & relationships (44%), and IDA (17%) also reported. Limited information was reported upon productivity & activity (39%), QoL (33%) and cost (6%).

CONCLUSIONS: Overall, there is a paucity of evidence elucidating the burden, QoL and economic cost impact on people with GT (PwGT) and caregivers. Recent literature suggests PwGT face significant burden from frequent bleeds which are often unreported to their healthcare professionals. Additional research focused on patients and caregivers is needed to characterize the burden of bleeding & IDA on QoL, direct healthcare costs, and indirect costs from productivity loss.