Soliris in PNH

PNH is a progressive, destructive, and life-threatening disease caused by an acquired mutation in the phosphatidylinositol glycan class A (PIG-A gene).1 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 terminal complement complex (TCC), also known as C5b-9 or the membrane attack complex (MAC). The result is chronic, intravascular hemolysis.2

PNH may be diagnosed at any age, with the median age of diagnosis in the early 30s.3 Diagnosis is typically delayed from 1 to more than 10 years.4 Importantly, more than three-fourths of patients with PNH do not present with hemoglobinuria at time of diagnosis.5

Chronic complement-mediated hemolysis can lead to devastating consequences, including thrombosis and renal failure, the leading causes of death in patients with PNH.1,3,6,7

Historically, there have been few options for care of patients with PNH. Palliative therapies include blood transfusions, corticosteroids, anticoagulants, androgen therapy, supplements such as folic acid and iron, and bone marrow transplantation, but patients with PNH suffer high rates of mortality despite any of these supportive therapy options.

Treating Patients Who Have PNH with Soliris

Soliris® (eculizumab) is the first and only therapy specifically tested and approved for the treatment of patients with paroxysmal nocturnal haemoglobinuria (PNH), an ultra-rare, life-threatening blood disorder in which chronic, uncontrolled activation of complement, a component of the normal immune system, results in haemolysis (destruction of the patient’s red blood cells). Soliris has been approved for the treatment of patients with PNH in nearly 50 countries worldwide, including the United States (US), European Union (EU), Canada and Japan.

Data from numerous clinical trials show that Soliris significantly reduces haemolysis in patients with PNH, leading to an improvement in symptoms and a reduction in major health problems associated with the disease. With the availability of Soliris, the outlook for patients has changed dramatically: In the initial PNH registration trials, 100 percent of patients had an objective response to Soliris, and long-term data published by independent investigators from patients treated with Soliris for up to eight years showed improved survival rates similar to normal healthy individuals matched for their age and gender.

More Information

Click here for Soliris Product Monograph.  

Health Care Professional Information: Click here

PNH Registry

PNH Registry

The PNH Registry is an international, observational, and noninterventional study collecting safety, effectiveness, and QoL data on patients with PNH.

Unite with a global community of physicians in contributing to the largest, most comprehensive PNH patient registry. Those eligible for enrollment include all patients who have been newly or previously diagnosed with PNH or have evidence of positive PNH cells.

  • All physicians managing patients with PNH, regardless of treatment approach, are encouraged to participate

Contribute to treatment objectives, practice patterns, and best practices.

  • An invaluable opportunity to increase understanding of the natural history of PNH and the safety and efficacy of treatments
  • Physicians can enroll all patients with a PNH clone size ≥0.01%

Support scientific collaboration in the PNH community.

  • Offers the international community greater insight into an uncommon disease with potentially devastating consequences

Enroll your patients in the PNH Registry today.

Please contact the PNH Registry hotline:

E-mail: PNHregistry@iconplc.com
Telephone: 1.800.913.4893

References:
1. Hillmen P, Lewis SM, Bessler M, et al. N Engl J Med. 1995;333:1253-1258.
2. Risitano AM. Adv Exp Med Biol. 2013;735:155-172.
3. Soci├ę G, Mary J-Y, de Gramont A, et al; for the French Society of Haematology.Lancet. 1996;348:573-577.
4. Dacie JV, Lewis SM. Ser Haematol. 1972;5:3-23.
5. Parker C, Omine M, Richards S, et al; for International PNH Interest Group. Blood. 2005;106:3699-3709.
6. Rachidi S, Musallam KM, Taher AT. Eur J Intern Med. 2010;21:260-267.
7. Borowitz MJ, Craig FE, DiGiuseppe JA, et al; for Clinical Cytometry Society. Cytometry Part B. 2010;78B:211-230.