Haemophilia and Health Collective of North Introduces New Treatment Guidelines For Rare Blood Disorder In India

Haemophilia and Health Collective of North Introduces New Treatment Guidelines For Rare Blood Disorder In India

During a press briefing today, the doctors explained that people with severe haemophilia bleed frequently and often spontaneously, including repeated bleeds into joints and muscles.

In addition to disease severity, the frequency of bleeding also varies by bleed site. 

Depending on the area affected, these bleeds can be debilitating or even life-threatening Repeated bleeding into joints can cause hemophilic arthropathy, which can result in physical disability. 

As a result, most of the Haemophilia patients are physically challenged and need joint replacements at an early age.

Moreover, patients with haemophilia experience frequent absenteeism from school and work, as physical activity is limited due to the extreme pain and discomfort associated with the haemorrhagic episode.

“Haemophilia is an X chromosome linked congenital bleeding disorder that slows the blood clotting process due to lack of a protein called Factor 8 (FVIII) which is caused by gene mutations,” Dr. KK Kaul, President, HHCN explained.

As a result, people with Hemophilia A bleed for a longer time than normal, due to a deficiency of clotting factor VIII, he added.

Current treatment modalities in India primarily focus on on-demand therapy – Factor VIII replacement to address bleeding episodes as they occur. 

However, exposure to exogenous Factor VIII could lead to development of inhibitors. This renders treatment with Factor VIII ineffective.

The New Guidelines Recommend:

1. Use of prophylaxis as standard of care (SOC) in haemophilia patients to prevent them from bleeding,

2. Clearly articulates the need for comprehensive care including significance of timely diagnosis, physiotherapy and multidisciplinary care for PwH

3. First time a separate category for Non-factor replacement (Currently available as Emicizumab) is incorporated in the selection criteria. The real world experience bears out the rapid switchovers to Non-factor products and which remains the drug of choice

4. Scoring based patient selection criteria so that patients get maximum benefit from products. A relevant scoring pattern to identify the patients who qualify for prophylaxis with Non-factor replacement potentially leading to improved patient adherence with better treatment outcomes

5. Optimal deployment of resources for severe Hemophilia A patients including high bleeders, pediatric population, patients with inhibitors,

6. Budgetary calculation for payers and/or policymakers to understand the budgetary requirements of each state and central reimbursement bodies