Clinical and economic impact of rFVIIa prophylaxis in Glanzmann thrombasthenia: cohort analysis in Saudi Arabia.

Glanzmann thrombasthenia (GT) is an ultrarare inherited platelet disorder associated with lifelong bleeding tendency and substantial health care burden. Evidence on the clinical and economic impact of prophylaxis in GT remains limited. We conducted a prospective cohort analysis of pediatric patients with severe GT receiving prophylactic recombinant activated factor VII (rFVIIa) at a specialist tertiary center in Saudi Arabia. Annualized clinical outcomes and direct medical costs during prophylaxis were compared with a historical preprophylaxis period of equal duration for each patient. Outcomes included treated bleeding episodes, hospital days, platelet transfusion use, and rFVIIa consumption. Costs were estimated by applying unit costs to captured resource use. Paired nonparametric analyses and deterministic sensitivity analyses were conducted. Eleven patients were included. Median annualized treated bleeding episodes decreased from 10 during the preprophylaxis period to 4 during prophylaxis. Median annualized hospital days decreased from 96 to 24, whereas platelet transfusion use decreased from 63 to 6 units. Although rFVIIa acquisition costs increased during prophylaxis, these were offset by lower hospitalization and platelet transfusion costs, resulting in a median net annual cost impact of -1305 Saudi riyal (SAR). Deterministic sensitivity analyses demonstrated robustness to plausible variation in costing assumptions, with median net annual cost impact ranging from -93 339 SAR to 90 729 SAR. These findings provide the first evidence indicating clinical and economic benefits associated with rFVIIa prophylaxis in GT and support further evaluation of prophylactic treatment strategies in this population.