RESULTS OF A RETROSPECTIVE COMPARATIVE ANALYSIS OF THE MANIFESTATIONS AND SEVERITY OF HEMORRHAGIC SYNDROME IN CHILDREN WITH GLANZMANN’S THROMBASTHENIA
Authors:   National Scientific and Practical Center for Pediatric Hematology, Oncology and Immunology named after Dmitry Rogachev, Moscow, Russia, Ustinova M.V. , Fedorova D.V. ,   National Scientific and Practical Center for Pediatric Hematology, Oncology and Immunology named after Dmitry Rogachev, Moscow, Russia, Pshonkin A.V. ,   National Scientific and Practical Center for Pediatric Hematology, Oncology and Immunology named after Dmitry Rogachev, Moscow, Russia, Poletaev A.V. ,   National Scientific and Practical Center for Pediatric Hematology, Oncology and Immunology named after Dmitry Rogachev, Moscow, Russia, Seregina E.A. ,   National Scientific and Practical Center for Pediatric Hematology, Oncology and Immunology named after Dmitry Rogachev, Moscow, Russia, Polokhov D.M. ,   National Scientific and Practical Center for Pediatric Hematology, Oncology and Immunology named after Dmitry Rogachev, Moscow, Russia, Ignatova A.A. ,   National Scientific and Practical Center for Pediatric Hematology, Oncology and Immunology named after Dmitry Rogachev, Moscow, Russia, Zharkov P.A. ,   National Scientific and Practical Center for Pediatric Hematology, Oncology and Immunology named after Dmitry Rogachev, Moscow, Russia
Published: September 2021

Glanzmann thrombastenia (GT) refers to a rare and severe pathology of platelets and causes frequent bleeding episodes in children and adults, miscarriage and intracranial hemorrhage in fetuses and newborns from mothers with GT. Objective of the study: to assess the severity of hemorrhagic syndrome in children diagnosed with GT and to conduct a comparative assessment of bleeding in patients with other hereditary thrombocytopathies and von Willebrand disease (VWD). Materials and methods of research: a retrospective analysis of 25 clinical observations of children diagnosed with GT (8 boys and 17 girls) was carried out; the median age at the time of treatment was 5 years, which were observed at the Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology in 2015–2020. Bleeding manifestations were assessed using the Pediatric Bleeding Questionnaire (PBQ) and the International Society on Thrombosis and Haemostasis Bleeding Assessment Tool (ISTH BAT) standard bleeding scales. The analysis of publications presented in the international databases SCOPUS and PUBMED over the past 10 years on modern approaches to GT therapy has been performed. Results: the diagnosis of GT was established in 25 children, the median PBQ score was 7 points compared with the group of children with thrombocytopathies 3 points [IQR 7 (5,5; 10,5) versus 3 (2; 5), p<0,00001] and VWD 4 points [IQR 7 (5,5; 10,5) versus 4 (2; 5,25), p<0,00001]. On the ISTH BAT scale in children with GT 8 points, in groups with thrombocytopathies 3 points [IQR 8 (6,5; 11,5) versus 3 (2; 5), p<0,00001] and VWD 4 points [IQR 8 (6,5; 11,5) versus 4 (2; 5,25), p<0,00001]. In 23 (92%) children, episodes of bleeding required periodic visits for emergency medical care, in 17 (68%) resulted in anemization and iron deficiency. Compared with the group of children with hereditary thrombocytopathies and VWD patients with GT had more severe and frequent bleeding symptoms, which required seeking medical attention. Conclusion: GT is a rare, severe disease in which bleeding is systemic, leads to anemization, causes frequent hospitalizations, which greatly reduces the quality of life of patients.