PNH: A Life-Threatening Disease
PNH is a rare disease that affects approximately 8,000 to 10,000 people in North America and Western Europe.1 PNH is an acquired hematopoietic stem cell disorder that may be diagnosed at any age; median age is in the early 30s.2,3
Published reports document that 35% of PNH patients die within 5 years of diagnosis,4 which may be a result of missed diagnosis for many years prior. In a patient population in which half the patients have <30% clone, 1 in 7 patients died within 5 years.5
Early diagnosis and treatment is crucial for optimum management.
- PNH may be overlooked or misdiagnosed because the signs and symptoms are nonspecific, heterogeneous, and often similar to those of other diseases
The expected survival rate of an age- and sex-matched control group is shown for comparison.4
PNH clinical sequelae: pervasive, unpredictable from onset
PNH is progressive, life threatening, and can include fatal thrombosis and end organ damage or failure.2-4
- Renal insufficiency prevalence in PNH patients is 5x higher than reported for the general population6,7
- Venous or arterial thromboses account for approximately 40% to 67% of PNH-related deaths8
- Renal failure was identified as the cause of death in approximately 8% to 18% of PNH patients2
- Nearly 50% of PNH patients suffer from pulmonary hypertension9
References: 1. Richards SJ, Cullen MJ, Dickinson AJ, et al. The effect of eculizumab therapy on red cell response kinetics in patients with paroxysmal nocturnal hemoglobinuria [ASH abstract]. Blood. 2005;106: Abstract 1047.
2. Rosse WF, Hillmen P, Schreiber AD. Immune-mediated hemolytic anemia. Hematology (Am Soc Hematol Educ Program). 2004:48-62.
3. Socié G, Mary J-Y, de Gramont A, et al, for the French Society of Haematology. Paroxysmal nocturnal haemoglobinuria: long-term follow-up and prognostic factors. Lancet. 1996;348:573-577.
4. Hillmen P, Lewis SM, Bessler M, Luzzatto L, Dacie JV. Natural history of paroxysmal nocturnal hemoglobinuria. N Engl J Med. 1995;333:1253-1258.
5. Peffault de Latour R, Mary JY, Salanoubat C, et al. Paroxysmal nocturnal hemoglobinuria: natural history of disease subcategories. Blood. 2008;112:3099-3106.
6. Clark DA, Butler SA, Braren V, Hartmann RC, Jenkins DE Jr. The kidneys in paroxysmal nocturnal hemoglobinuria. Blood. 1981;57:83-89.
7. Stevens LA, Coresh J, Greene T, Levey AS. Assessing kidney function—measured and estimated glomerular filtration rate. N Engl J Med. 2006;354:2473-2483.
8. Hillmen P, Muus P, Duhrsen U, et al. Effect of the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria. Blood. 2007;110:4123-4128. doi:10.1182/blood-2007-06-095646.
9. Hill A, Rother RP, Wang X, et al. Eculizumab reduces pulmonary hypertension through inhibition of hemolysis-associated nitric oxide consumption in patients with paroxysmal nocturnal hemoglobinuria [ASH abstract]. Blood. 2008;112: Abstract 486.