Prophylaxis in Haemophilia Care

• Haemophilia: An inherited rare bleeding disorder due to deficiency/lack of clotting factors (most often Factor VIII in Haemophilia A).

• Impact:

  • Even Minor injuries → excessive bleeding.

  • Spontaneous internal bleeds → joints, muscles (painful, disabling), and brain (life-threatening).

Epidemiology in India

• Expected prevalence: ~1–1.5 lakh cases (1 in 10,000 population).

• Diagnosed cases: ~29,000 (~20% of expected).

• Gap due to: Lack of awareness, limited diagnostic facilities, socio-economic barriers.

• Public health impact:

  • Untreated bleed decrease life expectancy by ~16 days per episode.

  • Leads to disability, school absenteeism, unemployment, reduced productivity.

Treatment Approaches

1. On-demand therapy

• Treatment after a bleed occurs.

• Disadvantage: Damage already done (especially to joints/muscles) before intervention.

2. Prophylaxis (Gold Standard)

• Regular replacement of deficient clotting factors to prevent bleeds.

• Methods:

  • Frequent intravenous factor injections.

  • Non-factor products via subcutaneous injection.

Advantages of Prophylaxis

1. Prevents Joint Damage

   • Maintains clotting factor levels → prevents recurrent bleeds → protects joint mobility.

2. Enhances Quality of Life

   • Fewer bleeds, less pain, more independence.

   • Children attend school and play; adults can work and engage socially.

3. Reduces Healthcare Burden

   • Fewer hospitalisations/urgent care visits.

   • Lower long-term costs by avoiding complications.

Global vs. Indian Context

• Developed countries: ~90% of patients on prophylaxis → near-normal life expectancy.

• India: Predominantly on-demand therapy; some States have started prophylaxis in children less than 10 years.

Way Forward

• Policy advocacy for wider access to prophylaxis.

• Public education to improve diagnosis rates.

• Early intervention to prevent disability and enable haemophilia patients to live free from fear and pain.