Hemophilia A in India: What We Know Now About Emicizumab and Better Care

What You’ll Learn

This article summarizes a 2024 review of how Hemophilia A (HA) is managed in India, with a focus on a newer treatment called emicizumab. We’ll break down:

• What Hemophilia A is (in simple terms)
• The challenges of treating HA in India today
• How emicizumab works and why it’s changing care for many patients
• Real-world results from Indian studies
• What this means for you or your loved one living with HA

A Quick Look at Hemophilia A

Hemophilia A is a genetic bleeding disorder caused by a lack of factor VIII—a protein your body needs to form blood clots. Without enough factor VIII, even small injuries can lead to prolonged bleeding, and some people experience spontaneous bleeding (without an obvious cause) into joints, muscles, or organs.

HA is X-linked, meaning it’s passed from mothers to sons (males are most affected). It’s classified as:

• Mild: Factor VIII levels 6–40% of normal (bleeding after trauma)
• Moderate: 2–5% (bleeding after minor injuries)
• Severe: <1% (spontaneous bleeding, often into joints)

In India, HA is underdiagnosed: while an estimated 80,000–100,000 people have severe HA, only 19,000 are registered with the Hemophilia Federation of India. This gap leads to preventable complications like joint damage, disability, and even death.

Why Summarizing Research on HA in India Matters

For rare diseases like HA, information is often scattered across small studies or clinical trials. Reviews like this one bring together data from Indian patients, doctors, and global research to:

• Highlight unmet needs (e.g., access to treatment in rural areas)
• Share real-world results of new therapies (like emicizumab)
• Help patients and families make informed decisions about care
• Guide doctors in improving treatment for HA in India

The Core of the Review: Emicizumab and HA Management in India

The review focuses on emicizumab (brand name Hemlibra®), a breakthrough treatment approved in India in 2019. Let’s break down what it is, how it helps, and what Indian patients are experiencing.

Current Treatments and Their Challenges

Before emicizumab, the main treatment for HA was factor VIII replacement therapy—regular infusions of synthetic or plasma-derived factor VIII to prevent bleeding. While effective, it has big drawbacks:

• Frequent IV injections: Severe HA patients may need 2–3 infusions per week.
• Inhibitors: Up to 30% of severe HA patients develop antibodies (inhibitors) that block factor VIII, making treatment useless.
• Access issues: Many Indian patients live far from hemophilia centers (average 131 km travel time) or can’t afford regular infusions.

These challenges lead to:

• Joint damage: Repeated bleeding into knees, ankles, or elbows causes chronic pain and disability.
• School/work absenteeism: Children with HA miss an average of 15 days of school per year (dropping to 4 days with prophylaxis).
• Economic burden: Treatment costs and lost income push many families into poverty.

Emicizumab: A Game-Changer?

Emicizumab is a bispecific antibody—a lab-made protein that mimics factor VIII. Instead of replacing factor VIII, it helps your body form clots by bridging two other clotting proteins (factor IXa and factor X).

Key Benefits for Patients:

• Subcutaneous injections: Given under the skin (like an insulin shot) instead of IV—easier for kids and people with limited IV access.
• Less frequent dosing: Once weekly, every 2 weeks, or even monthly (vs. 2–3 times/week for factor VIII).
• No inhibitors: Emicizumab doesn’t trigger the same immune response as factor VIII, so inhibitors are rare.
• Reduced bleeding: Clinical trials show emicizumab cuts annualized bleed rates (ABRs) by 87–96% in patients with inhibitors and 96% in those without.

Real-World Results from India

Indian studies back up these benefits:

• A pilot study of 8 patients with severe HA found that low-dose emicizumab (given once monthly) led to zero bleeding events over 1 year.
• In children with inhibitors, emicizumab reduced joint bleeds from 1–12 per year to zero and improved mobility (measured by the Pediatric Hemophilia Activity List).
• Patients and caregivers report better quality of life: fewer hospital visits, less anxiety about bleeding, and more ability to participate in daily activities.

Challenges in Access and Affordability

While emicizumab is a breakthrough, it’s not yet accessible to all Indian patients:

• Cost: Emicizumab is expensive, though some government schemes (like Employees’ State Insurance Corporation) cover it.
• Awareness: Many doctors and patients don’t know about emicizumab or how to access it.
• Long-term data: Most Indian studies are small or short-term—more research is needed to confirm long-term safety and efficacy.

What This Means for Patients and Families

If you or a loved one has HA, emicizumab could be a life-changing option—especially if:

• You have inhibitors (factor VIII doesn’t work for you)
• You struggle with IV access (e.g., young children, people with frequent infections)
• You live far from a hemophilia center (less frequent dosing means fewer trips)
• You have a history of severe bleeding (e.g., intracranial hemorrhage)

Even if emicizumab isn’t an option yet, the review highlights the importance of:

• Early diagnosis: The sooner HA is diagnosed, the sooner prophylaxis (preventive treatment) can start—reducing joint damage and disability.
• Advocacy: Push for better access to emicizumab and other treatments through government schemes or patient organizations.
• Talking to your doctor: Ask about emicizumab, even if it’s new—your team can help you weigh the benefits and costs.

Gaps in Our Knowledge & Future Directions

The review also points out what we don’t know yet:

• Long-term efficacy: How well does emicizumab work over 5–10 years in Indian patients?
• Cost-effectiveness: Is emicizumab cheaper than factor VIII in the long run (when you consider fewer hospital visits and disability)?
• Access for rural patients: How can we get emicizumab to people living in remote areas with limited healthcare?

Future research will focus on answering these questions—and expanding access to emicizumab for all who need it.

Key Points to Remember

1. Emicizumab is a breakthrough: It’s the first HA treatment that’s subcutaneous, less frequent, and doesn’t cause inhibitors.
2. Real-world results are promising: Indian patients on emicizumab have fewer bleeds, better mobility, and improved quality of life.
3. Access is a challenge: Cost and awareness are barriers, but government schemes and advocacy can help.
4. Early diagnosis and prophylaxis save lives: The sooner HA is treated, the fewer complications you’ll face.

Talk to Your Doctor

This article is a starting point—not medical advice. If you’re interested in emicizumab or have questions about HA management, talk to your hemophilia specialist. They can help you:

• Understand if emicizumab is right for you
• Navigate access to treatment (e.g., government schemes)
• Create a care plan that fits your lifestyle

Remember: You’re not alone. Organizations like the Hemophilia Federation of India can connect you with support groups, resources, and advocacy efforts to help you get the care you need.

For more information, visit the Hemophilia Federation of India’s website or ask your doctor for resources tailored to your situation.