New Delhi, June 24 Low dose of emicizumab could be as effective as standard dose against Haemophilia A, according to a study led by the Indian Council of Medical Research (ICMR).
Emicizumab is a bispecific monoclonal antibody used to treat hemophilia A -- a genetic bleeding disorder.
The drug works by mimicking the function of the missing or deficient clotting factor VIII (FVIII), enabling blood clotting to occur and preventing or reducing bleeding episodes.
As per the official government data, there are 27,000 haemophilia patients registered and 1, 40,000 patients expected to be affected in India.
Low-dose emicizumab can also be potentially a cost-effective treatment option in persons with hemophilia A, especially in developing countries, the study revealed.
A standard dose of emicizumab is priced approximately at $15,000.
“As the standard dose of the drug is expensive making it difficult for people from lower economic strata to access it, the ICMR led the study to understand its effectiveness even with a low dose,” said Dr. Manisha Madkaikar, Director of ICMR- Centre for Research Management and Control of Haemoglobinopathies (CRHCM) in Nagpur.
“Low-dose emicizumab will reduce the cost of therapy by more than 50 per cent resulting in the saving of more than 7 crore per patient for lifetime treatment assuming a life span of 50 years and average weight of 50 kg,” she added.
In the study, the scientists compared the efficacy and safety of low-dose emicizumab with those on low-dose factor (F)VIII prophylaxis via chart review.
Published in the Journal of Thrombosis and Haemostasis, the ICMR team showed that the direct cost of low-dose emicizumab was approximately $6,000 and that for low-dose recombinant FVIII prophylaxis used in the study was $6,282 (the cost may range from $3432 to $7920 depending on the type of factor).
“Our study successfully demonstrated that low-dose emicizumab is equally effective in the treatment of Haemophilia,” Madkaikar said
Haemophilia can typically lead to preventable joint damage. In about 80 per cent of cases, haemophilia manifests as bleeding, most frequently involving the large joints like knees, ankles, elbows, hip, and wrist, followed by deep bleeds affecting large body muscles.
Each bleeding episode requires attention, and patients take a prolonged time for recovery and severe complications could occur if any bleeding is not attended. Treatment with monoclonal antibodies like emicizumab are offering better protection with fewer infusions, especially for patients with inhibitors.
The study showed that low-dose emicizumab also improves bleeding prevention, joint health, and quality of life in patients with haemophilia.
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