New Delhi

A reduced dose of emicizumab is just as effective as the standard dose in managing hemophilia A, potentially slashing treatment costs by more than half, a study has found.

Hemophilia A is a genetic disorder caused by a lack of factor VIII, a crucial protein required for blood clotting.

While only around 27,000 hemophilia A patients are officially registered in India, experts believe the actual number may be well over 1.4 lakh.

"Standard emicizumab therapy is prohibitively expensive for most of our patients, many of whom come from economically disadvantaged backgrounds," said Dr Manisha Madkaikar, Director of ICMR-National Institute of Immunohematology (NIIH).

Emicizumab is a monoclonal antibody that mimics the function of factor VIII, the missing clotting factor in hemophilia A. It helps prevent spontaneous and injury-related bleeding, particularly in joints.

The study was conducted by ICMR-NIIH and KEM Hospital, Mumbai, an internationally recognised hemophilia treatment centre.

The study led by Dr Rucha Patil, a scientist at NIIH, and Dr Chandrakala S, a professor at KEM Hospital, compared the efficacy and safety of low-dose emicizumab to standard low-dose factor VIII prophylaxis, which is the standard, through a retrospective chart review. The findings were published in the Journal of Thrombosis and Haemostasis.

"This study shows that the reduced dose emicizumab can be a viable and affordable alternative without compromising efficacy," NIIH director Madkaikar said.

According to the study, in India, the direct cost of low-dose emicizumab is around Rs 10 lakhs annually, less than half of the Rs 26 lakhs required for the standard dose.

In contrast, low-dose recombinant factor VIII prophylaxis ranged from Rs 6 to 13 lakhs, depending on the type of factor used.

"Using low-dose emicizumab could reduce the lifetime cost of treatment by more than Rs 7 crore per patient, assuming a 50-year lifespan and average weight of 50 kg," Madkaikar added.

Hemophilia A, a lifelong bleeding disorder, often causes spontaneous or injury-related bleeding, most commonly in large joints such as the knees, ankles, elbows, hips, and wrists.

Without timely and adequate treatment, these recurring bleeds can lead to irreversible joint damage and severe disability. Newer therapies like emicizumab offer more consistent protection against bleeding and require fewer infusions, making them especially valuable for patients who don't respond well to traditional factor replacement treatments.

Dr Rucha Patil noted that a randomised controlled trial supported by the Department of Health Research (DHR) of the Union Health Ministry, is currently in progress to validate the findings further.

"Early results are highly encouraging, not only in terms of cost savings and bleeding control but also in improving joint health and overall quality of life," she said.

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"The interim data has already garnered international recognition at the International Society on Thrombosis and Haemostasis (ISTH) 2025 in the US. We anticipate completing the trial by early 2026 and hope the evidence will help shape treatment guidelines, particularly in low- and middle-income countries where affordability is a critical factor," Patil added.