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Prophylaxis Revolutionising Haemophilia Care in India

Why in the News ?

Prophylaxis, the gold standard treatment for severe haemophilia, is being promoted in India to prevent bleeds and disabilities. Experts stress awareness, early diagnosis, and policy support to expand access and improve patients’ quality of life. This prophylactic treatment approach aims to maintain adequate trough levels of coagulation factors to prevent bleeding episodes and improve the bleeding phenotype of patients with severe haemophilia.

Haemophilia and Diagnostic Challenges

● Definition: An inherited bleeding disorder caused by deficiency of clotting factors, commonly Factor VIII in Haemophilia A. This deficiency leads to impaired coagulation and increased risk of bleeding episodes, particularly in severe haemophilia cases.

● Risks: Even minor injuries can cause excessive bleeding; internal bleeds may damage joints, muscles, or the brain. Patients with severe haemophilia are particularly vulnerable to spontaneous bleeding episodes, which can significantly impact their quality of life and may require immune tolerance induction therapy in some cases.

● Prevalence gap: Expected 1–1.5 lakh cases in India; only ~29,000 diagnosed due to low awareness, poor diagnostics, and socio-economic barriers. This gap highlights the need for improved laboratory monitoring and clinical trials to enhance diagnosis and treatment of severe haemophilia.

● Impact of untreated bleeds: Each untreated bleed may reduce life expectancy by 16 days. Proper factor replacement therapy and prophylactic treatment can significantly reduce this impact by maintaining adequate trough levels of coagulation factors.

● Socio-economic cost: Leads to absenteeism, unemployment, and reduced productivity. Health economics studies have shown the long-term cost-effectiveness of prophylactic treatment in severe haemophilia, considering factors such as factor consumption and dosing frequency.

Benefits and Way Forward

● Joint protection: Prevents recurrent joint bleeding, preserving mobility and preventing disability. Prophylactic dosing helps maintain factor levels to prevent breakthrough bleeds and improve the bleeding phenotype of patients with severe haemophilia.

● Quality of life: Enables schooling, employment, and active lifestyles without fear of bleeding. Improved treatment adherence and reduced infusion frequency contribute to better outcomes for patients with severe haemophilia.

● Healthcare relief: Reduces emergency care needs and long-term treatment costs. The safety profile of modern factor concentrates has significantly improved treatment outcomes, including the development of extended half-life products through albumin fusion technology.

● Policy need: Promote awareness, early screening, and wider prophylaxis access through public health initiatives. This includes addressing the treatment burden and improving access to factor replacement therapy for patients with severe haemophilia.

● Vision: Achieve “zero bleeds” and disability-free life for all haemophilia patients in India. This goal requires ongoing clinical trials, improved prophylaxis regimens, and strategies to manage inhibitor development in patients with severe haemophilia.

About Prophylaxis:

● Prophylaxis: Regular infusion of clotting factor concentrates or non-factor products to prevent bleeds. This approach aims to maintain adequate trough levels of coagulation factors to prevent spontaneous bleeding episodes in severe haemophilia patients.

● On-demand therapy: Treats bleeds after onset, often too late to prevent joint damage. This approach is less effective in preventing long-term complications compared to prophylactic treatment for severe haemophilia.

● WHO Guidelines: Recognise prophylaxis as gold standard for haemophilia management, especially for severe haemophilia patients, emphasizing the importance of maintaining appropriate trough levels.

● Global scenario: ~90% of haemophilia patients in developed countries are on prophylaxis, enabling near-normal life expectancy. This includes the use of extended half-life products and albumin fusion technologies to reduce infusion frequency and improve factor consumption efficiency.

● India’s status: On-demand treatment still dominant; some States offer prophylaxis for children under 10. There’s a need to expand access to prophylactic treatment and factor replacement therapy across all age groups, particularly for those with severe haemophilia.