Why doctors say prophylaxis should be standard care in haemophilia

From delayed diagnosis to new treatment options, experts outline why preventive treatment is replacing on-demand care and how early prophylaxis protects joints

For decades, the treatment for haemophilia focused largely on reacting to bleeding episodes. Today, that approach is changing. Across the world, and increasingly in India, doctors are advocating prophylaxis as the preferred standard of care, shifting the focus from managing bleeds to preventing them altogether. This transition is transforming long-term outcomes for people living with haemophilia, particularly children and young adults.

 

What is haemophilia and how does it affect blood clotting?

 

Haemophilia is a lifelong, inherited bleeding disorder in which the blood does not clot properly. Haemophilia A, the most common form, occurs due to a deficiency of clotting Factor VIII.

 

 

“Factor VIII plays a critical role in helping blood form a stable clot after injury,” explains Dr Rucha Patil, Department of Haemostasis and Thrombosis, ICMR-National Institute of Immunohematology (ICMR-NIIH), KEM Hospital, Mumbai. “When this factor is missing or present at very low levels, even minor injuries can lead to prolonged bleeding,” she says.

 

Common symptoms of haemophilia include prolonged bleeding after injuries or surgery, easy bruising, frequent nosebleeds, and painful swelling of joints or muscles due to internal bleeding.

In severe cases, bleeding can occur spontaneously, particularly into joints and muscles. These internal bleeds are painful and, if repeated, can cause long-term joint damage, deformity and reduced mobility. As haemophilia is genetic, people are born with the condition and require lifelong care. However, with timely treatment, individuals can now lead active and productive lives.

 

Prophylaxis versus on-demand treatment: what’s the difference?

 

Traditionally, many patients have relied on on-demand therapy or treatment given only after a bleed has already started. While this can stop the immediate bleeding episode, damage often begins before treatment is administered.

 

Prophylaxis takes a very different approach. “Prophylaxis, or regular replacement therapy, involves administering clotting-factor treatment at planned intervals to maintain protective levels in the blood,” says Dr Patil. “The aim is to prevent bleeds before they happen.”

 

By keeping the clotting system supported, prophylaxis dramatically reduces spontaneous bleeds, particularly into joints. This proactive strategy allows children and adults with haemophilia to live more confidently, attend school or work regularly, and avoid repeated hospital visits.

 

The challenges of on-demand care are especially pronounced in remote or underserved areas. Delays in accessing treatment can turn manageable bleeds into emergencies, increasing hospital stays, disability and financial stress for families. Prophylaxis helps reduce these risks and the indirect social and economic burden associated with repeated bleeding episodes.

 

Why guidelines now favour prophylaxis

 

"India is estimated to have the second-largest haemophilia population globally, with around 130,000–136,000 individuals affected. Yet only about 20,000–21,000 patients are officially registered," shares Dr Patil, highlighting significant under-diagnosis and gaps in care.

 

“Clinical evidence consistently shows that regular prophylaxis prevents joint bleeds, preserves musculoskeletal health and significantly improves quality of life,” says Dr Tulika Seth, Professor of Haematology at AIIMS, New Delhi, and Executive Committee member of the Haemophilia Federation of India.

 

This growing body of evidence has led both international and Indian guidelines to recommend prophylaxis as the standard of care. Some Indian states have begun funding prophylaxis programmes through public health systems. Kerala’s state-supported programme for eligible children is often cited as a model, demonstrating reduced bleeding rates and fewer hospital admissions.

 

From a health-economics perspective, preventing bleeds, hospitalisations and long-term disability can also make prophylaxis cost-effective when delivered through structured public programmes.

 

What is the importance of starting early

 

The benefits of prophylaxis are greatest when treatment begins early in life. According to Dr Seth, children who receive regular prophylaxis often achieve near-normal joint function, while those dependent on on-demand therapy are more likely to develop severe arthropathy.

 

“Once joint damage occurs, it cannot be reversed — only slowed,” she notes. “Starting prophylaxis before the age of three offers the best chance to protect joints and preserve mobility.”

 

Access challenges in India

 

While access to prophylactic therapy has improved, major barriers remain. Limited awareness, delayed diagnosis and uneven availability of factor concentrates mean many children present only after joint damage has already occurred.

 

Logistical challenges, especially the need for frequent intravenous infusions, have also affected adherence. However, recent policy shifts and the introduction of newer therapies are beginning to change this landscape.

 

"The recent highlight on the need for including lifesaving prophylaxis drug for haemophilia in the National Health Mission essential drug list (EDL) in the winter parliamentary session reinforces the need and positive direction towards enabling persons with haemophilia to lead a normal life," says Dr Seth.

 

What the future holds

 

Haemophilia care is rapidly evolving. Extended half-life factor therapies reduce the frequency of infusions, while newer non-factor treatments can be administered subcutaneously, in some cases just once a month, and are effective even for patients with inhibitors.

 

“These advances have made prophylaxis far more practical and acceptable for patients and caregivers,” Dr Seth explains. “Better adherence means fewer bleeds and better long-term outcomes.”

 

Gene therapy also offers the promise of long-term or potentially curative treatment, though widespread access is still several years away. However, experts agree that for now expanding access to prophylaxis is the key to ensuring patients can lead active, independent lives.    

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This report is for informational purposes only and is not a substitute for professional medical advice.