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<p>Haemophilia is a blood-clotting disorder caused by a deficiency in factor VIII or factor IX, which causes bleeding in the muscles and joints. The development of inhibitors impairs the ability of clotting factor concentrates to control bleeding. Although immune tolerance induction is the standard of care to reduce inhibitor titres, it is effective only in 70–80% of cases. Hence, there is a need for agents that could permanently eradicate inhibitors. To determine the efficacy and safety of rituximab as frontline therapy for the eradication of inhibitors in congenital haemophilia A. This was a prospective study done on 20 patients with people with haemophilia (PwH) with inhibitors over a one-year study period. All received rituximab at the dose of 375&#xa0;mg/m2 weekly for four doses and were followed up for 52 weeks. All 20 PwH had decreased annualized bleed rate at the end of the study period compared to the baseline. 50% of the PwH had a major response, and 50% of the of the PwH had a minor response. 90% PwH had decreased the monthly requirement of bypassing agents at the end of 24 weeks. PwH in the age groups 2–5 years, 6–18 years, and above 18 years all had improvements in quality-of-life scores. Rituximab can be an integral treatment option as frontline therapy in congenital haemophilia A patients with inhibitors with the aim of reducing inhibitor titre, annualized bleeding rate, and improving quality of life.</p>

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Efficacy and Safety of Rituximab as Frontline Therapy for Eradication of Inhibitors in Congenital Haemophilia A

  • 期刊论文

  • 发表日期：

    2025年3月7日

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  • Shipla Roy,
  • Prakas Kumar Mandal,
  • Kaustav Ghosh,
  • Tuphan Kanti Dolai

摘要

Haemophilia is a blood-clotting disorder caused by a deficiency in factor VIII or factor IX, which causes bleeding in the muscles and joints. The development of inhibitors impairs the ability of clotting factor concentrates to control bleeding. Although immune tolerance induction is the standard of care to reduce inhibitor titres, it is effective only in 70–80% of cases. Hence, there is a need for agents that could permanently eradicate inhibitors. To determine the efficacy and safety of rituximab as frontline therapy for the eradication of inhibitors in congenital haemophilia A. This was a prospective study done on 20 patients with people with haemophilia (PwH) with inhibitors over a one-year study period. All received rituximab at the dose of 375 mg/m2 weekly for four doses and were followed up for 52 weeks. All 20 PwH had decreased annualized bleed rate at the end of the study period compared to the baseline. 50% of the PwH had a major response, and 50% of the of the PwH had a minor response. 90% PwH had decreased the monthly requirement of bypassing agents at the end of 24 weeks. PwH in the age groups 2–5 years, 6–18 years, and above 18 years all had improvements in quality-of-life scores. Rituximab can be an integral treatment option as frontline therapy in congenital haemophilia A patients with inhibitors with the aim of reducing inhibitor titre, annualized bleeding rate, and improving quality of life.