Hemophilia and the Impact of Treatment Regimens: Interlinking Quality of Life Measures
摘要
Hemophilia is a chronic disease characterized by deficiencies in coagulation factors VIII or IX, leading to high bleeding risks and life-threatening complications. Patients with hemophilia face limitations such as inability to practice contact sports, climb stairs frequently, and play freely with others. Currently, coagulation factor replacement therapy is a breakthrough in improving quality of life, but obstacles like high-titer inhibitors in severe hemophilia necessitate comprehensive research for anti-inhibitor therapies, bypass therapy, immunotherapy, and gene therapy to reduce the biopsychosocial impact of hemophilia on patients. This chapter presents research on clinical, pharmacological, economic, quality of life, and adherence to treatments, bringing the scientific community to the background of hemophilia physiopathology, treatment effectiveness, and quality of life questionnaires. This comprehensive approach ensures optimal, patient-centered care and guarantees their quality of life.