Haemophilia and the growing shift toward prophylaxis as the preferred standard of care - Prof (Dr) Prakas Kumar Mandal

Haemophilia is a relatively rare but life-threatening hereditary bleeding disorder; it affects an estimated 850,000 individuals worldwide. Approximately 289,000 individuals live with a severe form of haemophilia where even minor injuries can lead to prolonged bleeding, joint damage, disability, and long-term complications.

After China, India has the second-largest population of people with hemophilia A globally, estimated at close to 135,000 individuals, primarily living with Hemophilia A. However, only around 20,000 cases have been reported, highlighting a significant gap in diagnosis and care — and underscoring how many people may be living with the condition without access to timely treatment, protection, or support, putting them at constant risk of bleeding episodes and irreversible harm.

In certain instances, Persons With Haemophilia A (PwHA) may have internal bleeding, which may be accompanied by recurrent episodes of haemorrhage within joints and soft tissues. However, this silent condition may ultimately lead to chronic pain, joint damage and permanent disability. Prophylaxis, regular replacement therapy, mitigates this risk through regular, monitored administration of therapeutic products to maintain haemostasis.

PwHA lack one of the most essential clotting proteins, Factor VIII, due to which even a minor injury can turn into a prolonged bleeding event, unlike in healthy individuals. In prophylaxis, a clotting-factor therapy is given regularly to maintain the protective Factor VIII levels in the bloodstream. The major goal is to prevent major bleeds before they start. Imagine a house with a tiny crack in the roof.

If you only fix it after every rainstorm, the water damage keeps returning and gets worse over time. Prophylaxis is like repairing and reinforcing the roof before the next rain — keeping the house safe and dry. This is a fundamental shift from managing disability to preserving Quality of Life. Many patients have long been treated with episodic replacement therapy, commonly referred to as on-demand treatment, usually given after bleeding has occurred. While it may provide symptomatic relief to patients, it does little to keep bleeding episodes under control, which remains the core challenge in battling haemophilia.

Prophylaxis and its role in achieving long-term clinical outcomes: Life

PwH must initiate prophylaxis early in life to secure better long-term clinical outcomes and improve their chances of restoring normal quality of life. Maintaining adequate clotting factor levels in children—especially infants—is critical.

Very young children are at the highest risk of intracranial bleeds (brain bleeds), which can be life-altering or fatal. Early initiation of prophylaxis is therefore essential.

Children metabolise clotting factors differently, and some may require higher doses to maintain protective trough levels of Factor VIII, even if symptoms appear mild. Ensuring adequate factor levels early helps prevent catastrophic bleeds, avoids long-term joint damage, and protects a child’s future quality of life.

Few states have already adopted prophylactic therapy as the standard of care for haemophilia A, demonstrating clear clinical and economic value. These state-led programs show that prophylaxis significantly reduces long-term healthcare costs by preventing hospitalisations, emergency interventions, and disability-related surgeries.

By preserving functional independence and continuity of care, prophylaxis enhances quality of life, sustains workforce productivity, and protects the nation’s human capital—contributing to long-term economic resilience.

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