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What is PNH?

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, potentially life-threatening hematologic disease that involves the destruction of red blood cells by the complement system. This hemolysis can lead to severe anemia and other symptoms such as1-3:

  • Low hemoglobin levels
  • Ongoing fatigue
  • Continued need for transfusions
  • Thrombotic events

In PNH, hemolysis occurs through 2 mechanisms4:

For untreated and newly diagnosed patients, ongoing hemolysis is a core manifestation of PNH, potentially causing multisystemic damage and symptoms that may not be adequately communicated between the patient and physician.5-7

Learn about a treatment option for your patients with PNH Learn how

References: 1. Risitano AM, Marotta S, Ricci P, et al. Anti-complement treatment for paroxysmal nocturnal hemoglobinuria: time for proximal complement inhibition? A position paper from the SAAWP of the EBMT. Front Immunol. 2019;10:1157. 2. Brodsky RA. Paroxysmal nocturnal hemoglobinuria. Blood. 2014;124(18):28‌04-28‌11. 3. Dingli D, Matos JE, Lehrhaupt K, et al. The burden of illness in patients with paroxysmal nocturnal hemoglobinuria receiving treatment with the C5-inhibitors eculizumab or ravulizumab: results from a US patient survey. Ann Hematol. 2022;101(2):251-263. 4. Hill A, DeZern AE, Kinoshita T, Brodsky RA. Paroxysmal nocturnal haemoglobinuria. Nat Rev Dis Primers. 2017;3:17028. 5. Parker CJ. Update on the diagnosis and management of paroxysmal nocturnal hemoglobinuria. Hematology Am Soc Hematol Educ Program. 2016;2016(1):208-216. 6. Sahin F, Ozkan MC, Mete NG, et al. Multidisciplinary clinical management of paroxysmal nocturnal hemoglobinuria. Am J Blood Res. 2015;5(1):1-9. 7. Sharma VR. Paroxysmal nocturnal hemoglobinuria: pathogenesis, testing, and diagnosis. Clin Adv Hematol Oncol. 2013;11(suppl 13)(9):2-8.

This site is intended for US healthcare professionals only.