Outcomes of recombinant activated factor VIIa (NovoSeven) therapy in glanzmann thrombasthenia: two case reports

Glanzmann thrombasthenia (GT) is a rare autosomal recessive platelet function disorder resulting from qualitative or quantitative defects in the GPIIb/IIIa (integrin αIIbβ3) complex. Patients typically present with mucocutaneous bleeding. Standard treatment involves antifibrinolytic agents and platelet transfusions; however, repeated transfusions may lead to alloimmunization and platelet refractoriness. Recombinant activated factor VIIa (rFVIIa; NovoSeven) offers an alternative hemostatic approach. We present two pediatric cases of GT managed with rFVIIa. The first case involves a 12-year-old female with a strong family history of GT, who initially responded well to rFVIIa prophylaxis but later experienced increased bleeding episodes during puberty. The second case is a 20-year-old male with a history of mucocutaneous bleeding, who achieved long-term bleeding control on regular rFVIIa prophylaxis. Both patients demonstrated reduced bleeding frequency and improved quality of life without thromboembolic complications. These cases underscore the therapeutic potential and safety of rFVIIa in managing bleeding episodes and preventing alloimmunization in GT, particularly when platelet transfusions are ineffective or contraindicated, and highlight the importance of individualized treatment approaches and the need for further research to refine prophylactic regimens in GT.