Blood management strategies in congenital Glanzmann thrombasthenia at a hematology referral center

Abstract

Background Glanzmann thrombasthenia is associated with abnormalities in the glycoprotein IIb/IIIa receptor. This study, conducted at Ampang Hospital, Malaysia, aimed to assess outcomes of blood management strategies for Glanzmann thrombasthenia. Methods Ten patients with Glanzmann thrombasthenia aged 9 years (2009-2018) were examined. Data on clinical characteristics, transfusion practices, and patient blood management were obtained from medical records. Patient blood management methods included parenteral iron, erythropoietin, hormonal pills, intrauterine progesterone contraceptive devices, tranexamic acid, and recombinant factor VIIa. Primary outcomes were hemoglobin levels and the proportion of patients who received blood transfusion. Secondary outcomes were morbidity and mortality. Results The median age at diagnosis was 8.2 years (range, 1-15 yr). The female-to-male ratio was 9:1. Eight patients had type 2 disease (5-20% of normal GPIIb/IIIa), and two patients had type 1 disease (normal GPIIb/IIIa ＜5%). All patients had iron deficiency. All female patients presented with significant menorrhagia. Other bleeding symptoms included epistaxis, spontaneous skin bruising, hemoptysis, gingival bleeding, knee hemarthrosis, and pelvic hematoma. No patient experienced life-threatening bleeding. Our patients had a mean hemoglobin level of 5.6 g/dL at diagnosis. All patients were optimized using non-transfusion methods as described above. Our patient had a current mean hemoglobin level of 11 g/dL. Approximately 70% (7/10) of patients did not experience receiving blood transfusions in the last 5 years. No patient experienced non-transfusion-related morbidities such as sepsis, thromboembolism, or cardiorespiratory events. Conclusion High cost, transfusion-related adverse events, and immunomodulation could be effectively prevented by avoiding unnecessary blood transfusions.