What is Therapy for Paroxysmal Nocturnal Haemoglobinuria and Its Cost?

Therapies and Costs for Paroxysmal Nocturnal Haemoglobinuria (PNH) in India

Living with paroxysmal nocturnal Haemoglobinuria means battling fatigue, infections, and frequent transfusions. But a stable life is possible. Effective treatments now help protect red blood cells and platelets. In India, the cost of treatment starts at around Rs. 85,000, depending on the approach. Read on to explore the different treatment options and their associated costs across the country.

What are the Therapies Available for Paroxysmal Nocturnal Haemoglobinuria (PNH)? 

Before the availability of complement inhibitors, historical studies reported a median survival of approximately 10–15 years, with wide variability depending on complications such as thrombosis and bone marrow failure. With therapies like eculizumab, many patients now achieve near-normal life expectancy.

1. Medication

Oral formulations and infusion-based medication also promote better prescription fulfilment and adherence to this rare blood disorder; however, real‑world adherence varies depending on access, monitoring, and patient factors.

• Eculizumab:  Eculizumab treatment enhances the 5-year survival rate for around 95.5% of patients diagnosed with PNH. Apart from survival improvement, this therapy reduces hemolysis, transfusion and the number of thrombolytic events. However, due to availability issues and prohibitive cost, this treatment may have limited preference.  
  Note: Long‑term observational studies and registries show that patients treated with eculizumab have survival rates approaching those of the general population.
• Pegcetacoplan: Pegcetacoplan is an alternative complement inhibitor to eculizumab, with pricing and accessibility varying by formulation and provider. It involves a complement inhibitor, that attaches to the complement protein C3. It helps prevent complement-mediated destruction of red blood cells, thereby reducing hemolysis and transfusion needs. This treatment increases haemoglobin level and red blood cell count to reduce the requirement for transfusion.  
• Iptacopan: Iptacopan is an oral monotherapy and acts as a complement B inhibitor to manage patients with PNH. This medicine reduces hemolysis or destruction of red blood cells in patients struggling with Paroxysmal Nocturnal Haemoglobinuria. 
  Note: It is approved in some regions; availability and clinical use in India may be limited or evolving.

2. Surgical Treatment

Allogeneic hematopoietic stem cell transplantation is reserved for selected patients, mainly those with severe bone marrow failure syndromes or life‑threatening complications, due to its high morbidity and mortality.

• Allogeneic hematopoietic stem cell transplantation (HCT): Allogeneic hematopoietic stem cell transplantation is the only curative option for PNH but is typically reserved for patients with severe disease or associated bone marrow failure due to its risks. Patients of older age or without a matched sibling donor can be treated with antithymocyte globulin or cyclosporine as a standard treatment. However, younger, medically fit patients with severe aplastic anaemia and a suitable donor may be considered for HCT after careful risk–benefit assessment.

What is the Cost of PNH Therapy?

Paroxysmal Nocturnal Haemoglobinuria treatment in India will cost approximately between Rs. 85,000 and Rs. 35 lakhs at most. The cost may vary depending on the specific treatment requirements and the severity of the health condition. Below is the cost structure for various treatment options:

Important Notes: Overall treatment cost varies by hospitalisation, medication, and consultation. Hence, consult your doctor for accurate updated pricing details.

Which Factors Can Affect the Cost of Therapy for PNH?

Generally, treatments necessitate aftercare, including follow-up appointments and medications. These supplementary expenses can impact therapy costs for patients. Apart from that, keep the following in mind before treatment.  

• The intricacy and nature of the treatment required for PNH have a considerable impact on costs.
• Locations of clinics or hospitals across various cities in India can impact the overall treatment expenses.
• The utilisation of advanced equipment and innovative technology during treatment can increase overall expenditure for PNH patients.
• Severe cases of this rare blood disorder with low red blood cells and platelets lead to serious complications and increase financial outlay.  

Doctors utilise complement inhibitors, a specific treatment to restrict the complement system from destroying red blood cells. The cost of medications, such as eculizumab and pegcetacoplan, depends on treatment frequency and haemoglobin level.

Disclaimer: This article is intended for general informational and educational purposes only. Paroxysmal Nocturnal Haemoglobinuria (PNH) is a rare and complex condition, and treatment decisions must be made in consultation with a qualified haematologist or healthcare professional, based on individual clinical circumstances.