SOLIRIS (eculizumab): The first treatment specifically approved for PNH

For US Healthcare Professionals

PNH Morbidity and Mortality

Learn how SOLIRIS (eculizumab) treatment helps to benefit PNH patients.
Hemolysis. Disabling fatigue. Dysphagia. Is it PNH? Clinical assessment followed by flow cytometry can help confirm PNH diagnosis and optimize management decisions.

Chronic hemolysis is central to the morbidities and mortality of PNH1-6

Hemolysis is chronic in PNH, and is central to its characteristic signs and symptoms, illustrated in the diagram below.1,2,5,6,8-10

Hemolysis and PNH Morbidity and Mortality
click to enlarge

Thrombosis

  • Is the leading cause of death in PNH, occurring in approximately 40% of patients2
    • An estimated 50% of all PNH deaths may be attributed to thrombosis2
    • Thrombosis has been reported to increase the risk of death 10-fold in patients with PNH11

Anemia

  • Presents in a majority of PNH patients and is caused by chronic hemolysis and/or bone marrow failure1,8
  • Patients may become transfusion-dependent to maintain adequate levels of hemoglobin4,10

Smooth muscle dystonias1,9

  • Include dysphagia, abdominal pain, erectile dysfunction in men and pulmonary and systemic hypertension
  • Are hypothesized to be related to nitric oxide depletion during chronic hemolysis

Impaired quality of life

  • Quality of life is frequently impaired in patients with PNH, given the range of signs, symptoms and co-morbidities that often exist in these patients
  • Symptoms can include disabling fatigue, difficulty performing daily activities, pain and dyspnea

Next: Presentation of PNH

IMPORTANT SAFETY INFORMATION

WARNING: SERIOUS MENINGOCOCCAL INFECTION

SOLIRIS® increases the risk of meningococcal infections.

Vaccinate patients with a meningococcal vaccine at least 2 weeks prior to receiving the first dose of SOLIRIS; revaccinate according to current medication guidelines for vaccine use.

Monitor patients for early signs of meningococcal infections, evaluate immediately if infection is suspected, and treat with antibiotics if necessary.

The effect of withdrawal of anticoagulant therapy during SOLIRIS treatment has not been established. Therefore, treatment with SOLIRIS should not alter anticoagulant management.

SEE FULL PRESCRIBING INFORMATION FOR COMPLETE BOXED WARNING INCLUDING WARNINGS, PRECAUTIONS, AND ADVERSE REACTIONS.

The most frequent adverse events observed in clinical studies were headache, nasopharyngitis, back pain, nausea, fatigue, and cough.

Please see important safety information (including boxed warning) as well as the complete prescribing information.

References:
1. Parker C, Omine M, Richards S, et al. Diagnosis and management of paroxysmal nocturnal hemoglobinuria. Blood. 2005;106:3699-3709
2. Hillmen P, Lewis SM, Bessler M, Luzzatto L, Dacie JV. Natural history of paroxysmal nocturnal hemoglobinuria. N Engl J Med. 1995;333:1253-1258.
3. Johnson RJ, Hillmen P. Paroxysmal nocturnal hemoglobinuria: nature's gene therapy? J Clin Pathol Mol Pathol. 2992;55:145-152.
4. Brodsky RA. Paroxysmal nocturnal hemoglobinuria. In: Hoffman R, Benz EJ, Shattil SJ, et al. eds. Hematology. 4th ed. Philadelphia, PA: Elsevier Churchill Livingstone. 2005:419-427.
5. Luzzatto L, Gianfaldoni G. Recent advances in biological and clinical aspects of paroxysmal nocturnal hemoglobinuria. Int J Hematol. 2006;84:104-112.
6. Rosse WF, Hillmen P, Schrieber AD. Immune-mediated hemolytic anemia. Hematology. (Am Soc Hematol Educ Program) January 2004:48-62.
7. Hill A, Hillmen SJ, Elebute D, et al. Sustained response and long-term safety of eculizumab in paroxysmal nocturnal hemoglobinuria. Blood. 2005;106:2559-2565.
8. Besa EC. Paroxysmal nocturnal hemoglobinuria. eMedicine. August 2006;1-11. Available at: http://www.emedicine.com/med/topic2696.htm Accessed: 5/17/2007.
9. Rother RP, Bell L, Hillmen P, Gladwin MT. The clinical sequelae of intravascular hemolysis and extracellular plasma hemoglobin. JAMA. 2005;293:1653-1662.
10. Hillmen P, Young NS, Schubert J, et al. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2006;355:1233-1243.
11. Socié G, Mary J-Y, De Gramont A, et al. Paroxysmal nocturnal hemoglobinuria: long-term follow-up and prognostic factors. Lancet. 1996;348:573-577.