Weekly low-dose recombinant factor VIIa prophylaxis in Glanzmann thrombasthenia

Abstract

Glanzmann thrombasthenia is an inherited disease causing bleeding episodes due to platelet dysfunction. The standard treatment for moderate-severe bleeding is platelet transfusion. Recombinant factor VIIa (rFVIIa) is successfully used in bleeding episodes and invasive procedures. Here, we present a patient with Glanzmann thrombasthenia, whose bleeding episodes could only be controlled by rFVIIa. The patient is a 28 years old male, who has had frequent bleeding episodes unresponsive to local hemostatic agents and tranexamic acid and had an anaphylactoid reaction to platelet transfusion. We started the patient on a low-dose (20 μg/kg) rFVIIa once a week. The patient has no spontaneous bleeding since then. This is the first case report of a Glanzmann thrombasthenia patient on routine prophylaxis with low-dose rFVIIa.

Iron deficiency anemia and bleeding management in pediatric patients with Bernard‐Soulier syndrome and Glanzmann Thrombasthenia: A single‐institution analysis

“…In other inherited bleeding disorders such as hemophilia A, hemophilia B, and rare bleeding disorders due to clotting factor deficiencies, the benefits of prophylaxis with factor concentrates to prevent bleeding symptoms has been extensively described and advised as standard of care for individuals with severe bleeding 30,31 . In contrast, bleeding management in BSS or GT has been limited primarily to on‐demand platelet transfusions, on‐demand rFVIIa administration, or short‐term rFVIIa prophylaxis in the post‐surgical or severe bleed setting 26 . One study by Andıc et al.…”

“…One study by Andıc et al. reported a reduction in epistaxis and oral bleeding events corresponding to significantly improved quality of life in a 28‐year‐old male with GT after a 5‐month period of low dose rFVIIa prophylaxis at 20 μg/kg weekly 26 . In our patient cohort, we identified a subset of three patients with frequent SBE recalcitrant to on‐demand treatment and secondary chronic refractory IDA that ultimately prompted high dose prophylactic rFVIIa infusions at 90 μg/kg twice or thrice weekly.…”

“…24 The ideal frequency of monitoring and therapeutic approach to iron deficiency in this population is also unclear. 25 While limited case reports describe the use of short-term prophylactic rFVIIa as a method to prevent bleeding events not controlled by other therapies, 26 a standardized indication for long-term prophylactic rFVIIa has not been defined. This study aims to describe the impact of bleeding phenotype and IDA on disease management and outcomes of a pediatric cohort with BSS or GT, including the effect of long-term prophylactic rFVIIa infusions on associated bleeding complications in a subset of individuals.…”