Abstract
Eculizumab is a monoclonal antibody that binds with high affinity to the complement protein C5, preventing terminal complement-mediated intravascular haemolysis in patients with paroxysmal nocturnal haemoglobinuria (PNH). In three well designed clinical trials in patients with PNH, eculizumab blocked serum haemolytic activity and decreased transfusion rates. Pooled data from the three clinical trials demonstrated that eculizumab treatment decreased the overall thromboembolism rate in patients with PNH. Eculizumab carries a black box warning for the potential increased risk of meningococcal infections and requires patients to receive the meningococcal vaccine at least 2 weeks before starting treatment. Eculizumab is the first drug to be approved by the US FDA for the treatment of PNH and is a novel treatment that offers a new option for patients with PNH.
Similar content being viewed by others
Notes
The use of trade names is for product identification purposes only and does not imply endorsement.
References
Strubing P. Paroxysmale haemoglobinurie. Deutsche Med Wochenschrift 1882; 8: 1–16
Nishimura J, Murakami Y, Kinoshita T. Paroxysmal noctrnal hemoglobinuria: an acquired genetic disease. Am J Hematol 1999; 62: 175–82
Brodsky RA. New insights into paroxysmal nocturnal hemoglobinuria. Hematology Am Soc Hematol Educ Program 2006; 24-8: 516
Meletis J, Terpos E. Recent insights into pathophysiology of paroxysmal nocturnal hemoglobinuria. Med Sci Monit 2003; 9: 161–72
Parker CJ. The pathophysiology of paroxysmal nocturnal hemoglobinuria. Exp Hematol 2007; 35: 523–33
Hill A, Richards SJ, Hillmen P. Recent developments in the understanding and management of paroxysmal nocturnal haemoglobinuria. Br J Haematol 2007; 137: 181–92
Parham Peter, editor. The immune system. New York: Garland Publishing/Elsevier science Ltd., 2000: 185
Hillmen P, Lewis SM, Bessler M, et al. Natural history of paroxysmal nocturnal hemoglobinuria. N Engl J Med 1995; 333: 1253–8
Parker C, Omine M, Richards S, et al. Diagnosis and management of paroxysmal nocturnal hemoglobinuria. Blood 2005; 106: 3699–709
FDA News. FDA approves first-of-its-kind drug to treat rare blood disorder [online]. Available from URL: http://www.fda.gov/bbs/topics/NEWS/2007/NEW01589.html [Accessed 2007 Nov 1]
Soliris (eculizumab) prescribing information. Cheshire (CT): Alexion Pharmaceuticals, Inc.
Matis LA, Rollins SA. Complement-specific antibodies: designing novel anti-inflammatories. Nat Med 1995; 1: 839–42
Hillmen P, Hall C, Marsh J, et al. Effect of eculizumab on hemolysis and transfusion requirements in patients with paroxysmal nocturnal hemoglobinuria. N Engl J Med 2004; 350: 552–9
Hill A, Hillmen P, Richards SJ, et al. Sustained response and long-term safety of eculizumab in paroxysmal nocturnal hemoglobinuria. Blood 2005; 106: 2559–65
Hillmen P, Young NS, Schubert J, et al. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med 2006; 355: 1233–43
Brodsky RA, Young NS, Antonioli E, et al. Multicenter phase 3 study of the complement inhibitor eculizumab for the treatment of patients with paroxysmal nocturnal hemoglobinuria. Blood 2008; 111: 1840–7
Hillmen R, Muus P, Dührsen U, et al. Effect of the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria. Blood 2007; 110: 4123–8
Acknowledgements
No sources of funding were used to assist in the preparation of this review. The authors have no conflicts of interest that are directly relevant to the content of this review.
Author information
Authors and Affiliations
Corresponding author
Rights and permissions
About this article
Cite this article
Charneski, L., Patel, P.N. Eculizumab in Paroxysmal Nocturnal Haemoglobinuria. Drugs 68, 1341–1346 (2008). https://doi.org/10.2165/00003495-200868100-00001
Published:
Issue Date:
DOI: https://doi.org/10.2165/00003495-200868100-00001