Activité du facteur IX substitues chez les hémophiles B

Abstract

Hemophilia B is a rare hereditary disease caused by a deficiency in coagulation factor IX, requiring appropriate management to reduce the risk of hemorrhagic complications. Improving patients’ quality of life largely depends on rigorous clinical and biological monitoring.Our study is a descriptive, retrospective analysis conducted in the pediatric department of the University Hospital of Sétif, during the period from April 30 to May 30, 2025. It included 12 patients with hemophilia B, aged between 1 and 20 years. The aim was to evaluate treatment modalities and biological monitoring, as well as their impact on patients' quality of life.Data were collected from medical records and a structured questionnaire divided into four sections: sociodemographic information, characteristics of hemophilia, biological monitoring, and quality of life.All patients were male. The majority (83.3%) had a severe form of the disease. In 91.6% of cases, transmission was familial, and 66.6% of diagnoses were made before the age of one. Treatment was based exclusively on the administration of plasma-derived factor IX. Ten patients (83.3%) were on a prophylactic protocol, mainly with a frequency of two injections per week. No cases of inhibitor development were observed.Biological tests (PT, aPTT, CBC, liver and kidney function tests) were generally within normal ranges, and no positive viral serologies (HBV, HCV, HIV) were detected.In terms of quality of life, 83.3% of the children participated in physical activity, but only 25% adhered strictly to their treatment. A need for psychological support was expressed by 58.3% of the patients.This study highlights the importance of early and regular treatment, appropriate biological monitoring, and psychological support to improve the overall management of patients with hemophilia B.