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About PNH


PNH is a chronic, hemolytic disease with risk of unpredictable and life-threatening complications1

Paroxysmal nocturnal hemoglobinuria (PNH) is a hematopoietic stem cell disorder caused by an acquired mutation in the phosphatidylinositol glycan class A (PIG-A gene). All resulting cell lines from these PNH marrow clones lack key, naturally occurring terminal complement inhibitors on the cell surface. This renders them vulnerable to constant attack and lysis by the membrane attack complex (MAC), or C5b-9. The result is chronic, complement-mediated hemolysis, and is life-threatening.2-4

Hemolysis-induced complications increase the risk of premature mortality in patients with PNH5-7

PNH patient portrayal

  • Thrombosis and renal failure are leading causes of death1,9
  • PNH may be diagnosed at any age; median age is in the early 30s10
  • Diagnosis typically delayed from 1 to more than 10 years11

PNH is a chronic, catastrophic disease characterized by complement-mediated hemolysis2-4

PNH patient portrayal

PNH patient portrayal

Historical care for PNH

Historically, there have been few options for care of patients with PNH. Palliative therapies include blood transfusions, anticoagulants and bone marrow transplantation, but patients with PNH suffer high rates of mortality despite any of these supportive therapy options.10,15


Early diagnosis is essential for improved patient management and prognosis. Learn about ICCS and other expert recommendations for who should be tested for PNH.

PNH has been called “the most vicious acquired thrombophilic state known in medicine”.16 Learn which common symptoms are associated with an increased risk of thromboembolism in a patient diagnosed with PNH.

References: 1. Hillmen P, et al. Br J Haematol. 2013;162(1):62-73. 2. Borowitz MJ, et al. Cytometry B Clin Cytom. 2010;78(4):211-230. 3. Parker CJ. Hematology Am Soc Hematol Educ Program. 2011;2011:21-29. 4. Parker CJ. Hematology Am Soc Hematol Educ Program. 2016;2016(1):208-216. 5. Hillmen P, et al. N Engl J Med. 1995;333(19):1253-1258. 6. Kelly RJ, et al. Blood. 2011;117(25):6786-6792. 7. Peffault de Latour R, et al. Blood. 2008;112(8):3099-3106.8. Loschi M, et al. Am J Hematol. 2016;91(4):366-370. 9. Sharma VR. Clin Adv Hematol Oncol. 2013;11 suppl 13(9):2-8. 10. Socie G, et al. Lancet. 1996; 348(9027):573-577. 11. Dacie JV, Lewis SM. Ser Haematol. 1972;5(3):3-23. 12. Walport MJ. N Engl J Med. 2001;344(15):1140-1144. 13. Murphy K. Innate immunity: the first lines of defense. In: Scobie J, et al, eds. Janeway’s Immunobiology. 8th ed. New York, NY: Garland Science; 2012:37-73. 14. Kelly R, et al. Ther Clin Risk Manag. 2009;5:911-921. 15. Parker C, et al. Blood. 2005;106(12):3699-3709. 16. Hill A, et al. Blood. 2013;121(25):4985-4996.

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