Context:

Haemophilia has long posed significant health and socio-economic challenges in India due to underdiagnoses, limited access to treatment, and reliance on reactive care. Recent advancements in treatment, including regular clotting factor replacement and newer non-factor therapies, have made it possible to prevent bleeds entirely, drastically improving patient outcomes.

About Hemophilia:

·         Hemophilia is a genetic bleeding disorder where blood doesn’t clot properly.

·         Caused by deficiency of clotting factors, leading to prolonged or spontaneous bleeding.

·         Primarily affects males; females are usually carriers.

·         Inherited, though some cases arise from new genetic mutations.

·         Clotting factor deficiency: Body lacks essential proteins for clotting.

Types of Hemophilia:

a)     Hemophilia A: Factor VIII deficiency (most common).

b)    Hemophilia B: Factor IX deficiency.

c)     Hemophilia C: Factor XI deficiency; rare and affects both genders.

·        Bleeding can occur in joints, muscles, or brain, requiring prompt treatment.

Shift from Reactive to Preventive Care:

• Traditionally, haemophilia management in India relied on on-demand therapy, where treatment is given after a bleed occurs. This approach often fails to prevent long-term joint damage and disability. In recent years, however, the focus has shifted toward prophylaxis, or regular replacement therapy, aimed at preventing bleeds before they happen.
• This marks a paradigm shift from merely treating haemophilia to envisioning a future of "zero bleeds". Newer therapies are also moving beyond simple replacement, aiming to rebalance or restore the body’s clotting system entirely, giving patients a chance at a near-normal life.
• However, despite an estimated 1 to 1.5 lakh haemophilia cases in India, only around 29,000 patients are officially diagnosed, highlighting a serious gap in awareness and healthcare access.

Benefits of Prophylactic Treatment:

Prophylaxis is globally regarded as the gold standard in haemophilia care. Its key benefits include:

• Preventing joint damage: Regular replacement keeps clotting factor levels stable, reducing spontaneous bleeds and long-term deformities.
• Improving quality of life: Children can attend school and play, adults can work and socialize, leading to better physical and mental well-being.
• Reducing healthcare burden: Fewer emergency hospital visits and long-term complications reduce overall treatment costs.

Developed nations report 90% of haemophilia patients on prophylaxis, achieving near-normal life expectancy. In India, however, the adoption remains low, though some states have begun offering prophylaxis to children under 10.

The Way Forward:

To fully realize the benefits of prophylaxis, India must:

• Expand diagnostic facilities at the grassroots.
• Ensure affordable access to clotting factor therapies.
• Promote policy-level support and public awareness.