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

For US Healthcare Professionals

About PNH

The clinical sequelae of PNH can result in significant morbidity, severe complications and death.
Early diagnosis of PNH helps to optimize patient management. Know which patients to screen and how to screen for PNH.

PNH is a disabling and life-threatening disease1-3

Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired hematopoietic stem cell disease characterized by chronic hemolysis.4-5

  • PNH most often affects adults in the prime of their lives, with the median age at diagnosis in the early 30s1
     
  • Median survival following diagnosis is as low as 10 to 15 years1,4
     
  • Patients with PNH often experience anemia which may require transfusions, disabling fatigue and pain6,8,9
     
  • Approximately 40% of patients experience a thrombotic event during the course of their disease2
     
  • Thrombosis is the most common cause of death in PNH patients2

Learn more about the varied presentation of PNH and which of your patients should be screened for the disease.

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

Next: Pathophysiology

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. 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.
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. Rosse WF. Paroxysmal nocturnal hemoglobinuria. In: Hoffman R, Benz EJ, Shattil SJ, et al. eds. Hematology. 4th ed. Philadelphia, PA: Elsevier Churchill Livingstone. 2005:331-342.
4. Hillmen P, Lewis SM, Bessler M, Luzzatto L, Dacie JV. Natural history of paroxysmal nocturnal hemoglobinuria.N Engl J Med. 1995;333:1253-1258.
5. Hillmen P, Young NS, Schubert J, et al. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2006;355:1233-1242.
6. Johnson RJ, Hillmen P. Paroxysmal nocturnal hemoglobinuria: nature’s gene therapy? J Clin Pathol Mol Pathol. 2992;55:145-152.
7. Rother RP, Bell L, Hillmen P, Gladwin MT. The clinical sequelae of intravascular hemolysis and extracellular plasma hemoglobin. JAMA. 2005;293:1653-1662.
8. Rosse WF, Hillmen P, Schrieber AD. Immune-mediated hemolytic anemia. Hematology. (Am Soc Hematol Educ Program) January 2004:48-62.
9. 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.