What is PNH?
In PNH, uncontrolled terminal complement activation impairs the normal functioning of blood cells and leads to life-threatening consequences such as intravascular haemolysis (IVH), thromboembolic events, renal impairment and pulmonary hypertension.3 If PNH is left untreated, it can lead to early mortality.4,5
Thrombosis is a leading known cause of death,
occurring in ~40%–67% of patients, and can be
fatal at the first event.6-8*

PNH is caused by an acquired mutation in a specific gene called phosphatidylinositol glycan A (PIG-A) in one of the stem cells in the bone marrow.9-12

Explore the details of the underlying mechanism of PNH by visiting our Mechanism of Disease page below.

 Mechanism of PNH

*Data from a retrospective chart review of 301 patients with PNH, enrolled into the South Korean PNH registry, to assess the clinical signs and symptoms predictive of mortality using standard mortality ratio compared with an age- and sex-matched general South Korean population.8
Diagnosis of PNH

The most common symptoms include:

Thrombosis and renal failure are two severe and potentially life-threatening consequences of PNH.8,15-17

The image shows some common symptoms of PNH, but it is not an exhaustive list. For a thorough description of symptoms of PNH, please visit our dedicated Symptoms page.

 Symptoms of PNH

*Schrezenmeier 2014: N=856 patients (410 males) self-reporting symptoms via a baseline questionnaire.13

†Jang 2016: retrospective chart review of patients diagnosed with PNH in South Korea (N=301).8
Management of PNH
Living with PNH
Adverse Event Reporting

Please report any adverse events via your national reporting system. Adverse events can also be reported to Alexion, AstraZeneca Rare Disease by contacting: https://contactazmedical.astrazeneca.com/
Lee JW, et al. Expert Review of Clinical Pharmacology. 2022;15(7):851–861.  Barratt J. Complement Factor D as a Strategic Target of Regulating the Alternative Complement Pathway. Frontiers in Immunology 2021 1-15. Risitano AM, et al. Paroxysmal Nocturnal Hemoglobinuria: Pathophysiology, Natural History and Treatment Options in the Era of Biological Agents. Biologics.2008;2(2):205–222. Kulasekararaj AG, The Importance of Terminal Complement Inhibition in Paroxysmal Nocturnal Hemoglobinuria. Therapeutic Advances in Hematology 2022 1-16. Sharma VR. Paroxysmal Nocturnal Hemoglobinuria: Pathogenesis, Testing, and Diagnosis. Clinical Advances in Hematology Oncology. 2013;9(11 Suppl 13):2–8. Hill A, et al. Thrombosis in Paroxysmal Nocturnal Hemoglobinuria. Blood. 2013;25(121):4985–4996. Lee JW, et al. Clinical Signs and Symptoms Associated with Increased Risk for Thrombosis in Patients with Paroxysmal Nocturnal Hemoglobinuria from a Korean Registry. International Journal of Hematology. 2013;6(97):749–757. Jang JH, et al. Risk Factors of Mortality in Paroxysmal Nocturnal Hemoglobinuria. The Korean Academy of Medical Sciences. 2016;31(2):214–221. Szlendak U, Budziszewska B, Spychalska J, et al. Paroxysmal nocturnal hemoglobinuria: advances in the understanding of pathophysiology, diagnosis, and treatment. Pol Arch Intern Med 2022;132:16271.  Risitano AM, Peffault de Latour R. How We(‘ll) Treat Paroxysmal Nocturnal Haemoglobinuria: Diving into the Future. Br J Haematol 2022;196:288–303.  Gembillo G, Siligato R, Cernaro V, et al. Complement Inhibition Therapy and Dialytic Strategies in Paroxysmal Nocturnal Hemoglobinuria: the Nephrologist’s Opinion. J Clin Med 2020;9:1261. Hill A, et al. Paroxysmal nocturnal haemoglobinuria. Nature Review Disease Primers. 2017;3:17028. Schrezenmeier H, et al. Baseline Characteristics and Disease Burden in Patients in the International Paroxysmal Nocturnal Hemoglobinuria Registry. Paroxysmal Nocturnal. Haematologica. 2014;99(5):922–929. Brodsky R.A. Paroxysmal Nocturnal Hemoglobinuria. Best Principles and Practice. Chapter 31. Hematology. 2018:415-424. de Latour RP, et al. Paroxysmal Nocturnal Hemoglobinuria: Natural History of Disease Subcategories. Blood. 2008;112(8):3099–3106. Nishimura J, et al. Clinical Course and Flow Cytometric Anaylsis of Paroxysmal Nocturnal Hemoglobinuria in the United States and Japan. Medicine 2004. 193-207. Hillmen P. Long-term Safety and Efficacy of Sustained Eculizumab Treatment in Patients with Paroxysmal Nocturnal Haemoglobinuria. British Journal of Haemotology. 2013. Borowitz M, et al. Guidelines for the Diagnosis and Monitoring of Paroxysmal Nocturnal Hemoglobinuria and Related Disorders by Flow Cytometry Cytometry B Clin Cytom 2010 211-230. Richards SJ et al. Recent Advances in the Diagnosis, Monitoring, and Management of Patients with Paroxysmal Nocturnal Hemoglobinuria Cytometry B Clin Cytom 2007 291-298. advance I am a healthcare professional registered in the EU I am not a
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