Is Low Dose Emicizumab Effective for Haemophilia A?
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Synopsis
A groundbreaking study from the ICMR suggests that a low dose of emicizumab could offer a cost-effective and equally effective treatment for Haemophilia A, potentially revolutionizing patient care and access to necessary therapies.
Key Takeaways
- Low-dose emicizumab may be as effective as the standard dose for treating Haemophilia A.
- The treatment can reduce costs by over 50%, making it more accessible.
- ICMR's research shows potential savings of more than 7 crore per patient over a lifetime.
- The study indicates improved bleeding prevention and better joint health.
- Monoclonal antibodies provide enhanced protection with fewer infusions.
New Delhi, June 24 (NationPress) A recent study by the Indian Council of Medical Research (ICMR) indicates that a low dose of emicizumab might be just as effective as the standard dose in treating Haemophilia A.
Emicizumab is a bispecific monoclonal antibody that serves as a treatment for this genetic bleeding disorder.
This medication functions by replicating the role of the missing or insufficient clotting factor VIII (FVIII), facilitating blood clotting and helping to prevent or diminish bleeding episodes.
Official government statistics show that there are 27,000 registered haemophilia patients in India, with an estimated 140,000 individuals expected to be affected.
The study suggests that low-dose emicizumab could be a cost-effective treatment for haemophilia A, particularly in developing nations.
The standard dose of emicizumab costs around $15,000.
Dr. Manisha Madkaikar, Director of ICMR's Centre for Research Management and Control of Haemoglobinopathies (CRHCM) in Nagpur, stated, “The high cost of the standard dose has created barriers for low-income individuals, prompting ICMR to investigate the drug's effectiveness at lower doses.”
“Using a low-dose regimen can potentially cut therapy costs by over 50 percent, saving more than 7 crore per patient over a lifetime, assuming a lifespan of 50 years and an average weight of 50 kg,” she added.
The study compared the efficacy and safety of low-dose emicizumab with low-dose factor (F)VIII prophylaxis through a thorough chart review.
Published in the Journal of Thrombosis and Haemostasis, the ICMR team found that the direct cost associated with low-dose emicizumab is about $6,000, while the cost for low-dose recombinant FVIII prophylaxis is around $6,282 (with costs varying from $3,432 to $7,920 based on the type of factor used).
“Our research successfully confirmed that low-dose emicizumab is equally effective in treating Haemophilia,” Madkaikar remarked.
Haemophilia can lead to avoidable joint damage, with bleeding episodes commonly affecting major joints such as knees, ankles, elbows, hips, and wrists. About 80 percent of cases present with bleeding, often leading to prolonged recovery times and severe complications if untreated.
Treatment options like monoclonal antibodies such as emicizumab provide enhanced protection with fewer infusions, which is especially beneficial for patients with inhibitors.
The study also highlighted that low-dose emicizumab aids in improving bleeding prevention, joint health, and overall quality of life for haemophilia patients.
Point of View
The findings from the ICMR study highlight a significant advancement in the treatment of Haemophilia A. The potential for low-dose emicizumab to provide effective therapy at reduced costs represents a crucial step towards making treatment more accessible for all, reflecting our commitment to improving healthcare outcomes across India.
NationPress
24/06/2025
Frequently Asked Questions
What is emicizumab?
Emicizumab is a bispecific monoclonal antibody used to treat Haemophilia A, mimicking the function of the missing clotting factor VIII.
How effective is low-dose emicizumab?
According to the ICMR study, low-dose emicizumab is shown to be equally effective as the standard dose in treating Haemophilia A.
How much can costs be reduced with low-dose emicizumab?
The study suggests that using low-dose emicizumab can reduce therapy costs by more than 50%, saving over 7 crore per patient over a lifetime.
What are the benefits of using monoclonal antibodies like emicizumab?
Monoclonal antibodies like emicizumab offer better protection with fewer infusions, which is particularly beneficial for patients with inhibitors.
What are the implications of this study for patients?
This study paves the way for more cost-effective treatment options and improved overall quality of life for haemophilia patients.
