What is PNH?

What is PNH?

Paroxysmal nocturnal haemoglobinuria (PNH) is an acquired, rare and life-threatening blood disorder characterised by continuous intravascular haemolysis leading to thrombosis and end-organ damage.1-3

PNH is an ultra-rare debilitating and life-threatening blood disorder


PNH blood cells are deficient in glycosylphosphatidylinositol (GPI) anchors and, thus, lack a key, naturally occurring terminal complement inhibitor on the cell surface, rendering them vulnerable to lysis by the patient’s immune system.1,4,5

PNH epidemiology

  • Rare, acquired chronic disease of adults and children6
  • Affects male and female patients equally6
  • May be diagnosed at any age; median age is in the early 30s6
  • 35% of PNH patients die within 5 years of diagnosis7
  • Venous and arterial thromboses account for ~40–67% of PNH-related deaths; even the first thrombotic event might be fatal8
  • Diagnosis of PNH can take from 1 to >10 years9

Learn more

PNH may present de novo but often occurs in association with bone marrow failure syndromes such as aplastic anaemia and myelodysplasia.10

This chronic and progressive disorder has considerable impact on patient quality of life and significantly reduces life expectancy.7,10

  • References
    1. Rother RP et al. JAMA 2005; 293: 1653-1662.
    2. Brodsky RA. In: R. Hoffman et al, eds. Hematology: Basic Principles and Practice. 4th ed. Philadelphia, PA: Elsevier Churchill Livingstone, 2005: 419-427.
    3. Rother RP et al. Nat Biotechnol 2007; 25: 1256-1264.
    4. Rosse WF et al. Hematology Am Soc Hematol Educ Program 2004; 48-62.
    5. Wiedmer T et al. Blood 1993; 82: 1192-1196.
    6. Socié G et al. Lancet 1996; 348: 573-577.
    7. Hillmen P et al. N Engl J Med 1995; 333: 1253-1258.
    8. Hillmen P et al. Blood 2007; 110: 4123-4128.
    9. Dacie JV, Lewis SM. Ser Haematol 1972; 5: 3-23.
    10. Parker C et al. Blood 2005; 106: 3699-3709.

Netflow Portal


You are now leaving www.netflowconnect.com website

Alexion Pharma GmbH has no influence or control over these websites and shall not be held responsible in any way for any of the content held there.

Continue Cancel