PNH: Progressive, Destructive, and Life Threatening

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The expected survival rate of an age- and sex-matched control group is shown for comparison.1

Paroxysmal nocturnal hemoglobinuria (PNH) is a progressive and destructive disease that can cause thrombosis, end organ damage, and increased mortality.1-3

  • 35% of PNH patients die within 5 years of diagnosis1
  • In a patient population in which half the patients have <30% clone, 1 in 7 patients died within 5 years4

The Role of Hemolysis

Chronic hemolysis is the underlying cause of progressive morbidities and mortality in PNH. The systemic threats of hemolysis include thrombosis, chronic kidney disease, and pulmonary hypertension.5-7
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Clinical Sequelae

During chronic hemolysis, excess free hemoglobin depletes plasma nitric oxide (NO), which may play an important role in normal platelet function. It is believed that NO depletion may lead to platelet activation and aggregation. In addition, chronic consumption of nitric oxide by free hemoglobin can cause downstream sequelae to occur.8

CKD & PNH
64% of patients with PNH have chronic kidney disease (CKD).7

PNH Can Lead to Thrombosis and End Organ Damage
Thromboses (venous or arterial) account for approximately 40% to 67% of PNH-related deaths.7

Pulmonary Hypertension (PHT) in PNH
Nearly 50% of PNH patients suffer from pulmonary hypertension.7

Signs & Symptoms

PNH is a complex disease that presents uniquely in each patient.1-3 Its clinical sequelae are nonspecific, unpredictable, and often similar to those of other diseases. Common symptoms of hemolysis — such as abdominal pain, dyspnea, and fatigue — signal the underlying threat of serious consequences including pulmonary hypertension, cardiac dysfunction, and chronic kidney disease.

Hemolysis is the main cause of major health problems associated with PNH.9 Click here to view the signs and symptoms associated with PNH.

Want an easy way to assess PNH symptoms? Download this PNH symptoms assessment card.


References: 1. Hillmen P, Lewis SM, Bessler M, Luzzatto L, Dacie JV. Natural history of paroxysmal nocturnal hemoglobinuria. N Engl J Med. 1995;333:1253-1258. 2. 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. 3. Nishimura J-I, Kanakura Y, Ware RE, et al. Clinical course and flow cytometric analysis of paroxysmal nocturnal hemoglobinuria in the United States and Japan. Medicine. 2004;83:193-207. 4. Peffault de Latour R, Mary JY, Salanoubat C, et al. Paroxysmal nocturnal hemoglobinuria: natural history of disease subcategories. Blood. 2008;112:3099-3106. 5. Hillmen P, Young NS, Schubert J, et al. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2006;355:1233-1243. 6. Hillmen P, Elebute MO, Kelly R, et al. High incidence of progression to chronic renal insufficiency in patients with paroxysmal nocturnal hemoglobinuria (PNH) [ASH abstract]. Blood. 2007;110: Abstract 3678. 7. 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. 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.

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