Hemophilia is a genetic disorder characterized by impaired blood coagulation, leading to increased bleeding risk. The severity of hemophilia varies significantly among individuals, influenced by genetic factors, family inheritance patterns, and the occurrence of complications such as hemarthrosis. Understanding these interrelationships is crucial for developing tailored management strategies for affected children. The purpose of this article is to explore the correlations between clinical severity and various factors, including modes of inheritance, hemarthrosis incidence, types of genetic mutations, and inhibitor presence in pediatric patients with hemophilia. By elucidating these relationships, the study aims to contribute to improved diagnostic and therapeutic approaches in this population.
Inhibitory hemophilia: contemporary treatment with emicizumab. Considerations for pediatric practice
This article provides an overview of current knowledge and global experience regarding the use of emicizumab, with a focus on its specific considerations in pediatric practice. Emicizumab, a monoclonal antibody, operates uniquely compared to other therapies. It has been approved in Moldova since 2019 for preventing bleeding in hemophilia patients. Significant data from clinical studies and accumulated clinical practice provide answers to most questions physicians have when prescribing emicizumab. The article presents recommendations based on current information and global experience to aid decision-making in emicizumab usage. The purpose of this article is to provide information on management tactics for pediatric patients with hemophilia A receiving emicizumab.