All PNH Patients in Clinical Trials Showed a Benefit from Soliris®
Therapy
Many benefits occurred rapidly; others showed improvement over time
- The majority of patients (63%) received concomitant anticoagulant therapy 1,6
- The effect of anticoagulant withdrawal during Soliris treatment has not been studied 6
Factors to consider in evaluating Soliris therapeutic response
- All patients experience hemolysis; however, symptoms and their severity may vary
widely between patients7,8
- Time to and extent of therapeutic response in relation to fatigue, quality of life,
and transfusion frequency may vary1,2
- During infections and periods of stress, PNH patients may experience transient increased
episodes of anemia due to7,9:
- Reduced iron transport and erythropoiesis7
- Increased binding of C310
- Hypersensitivity to stress demonstrated by PNH red blood cells7
Patients with concomitant PNH and AA or MDS may require multiple therapeutic approaches 3
- In clinical trials, patients with bone marrow disorders in addition to PNH who were treated with concomitant IST medications also derived benefit from Soliris therapy similar to non-IST-treated PNH patients, without evident impact on safety or efficacy 1,9
- As Soliris primarily addresses RBC destruction, PNH patients with underlying bone marrow dysfunction related to RBC production (such as AA or MDS) may experience lower hemoglobin levels than patients with PNH only 11
Soliris is generally well tolerated. The most frequent adverse events observed in clinical studies were headache, nasopharyngitis, back pain, nausea, and fatigue.
IMPORTANT SAFETY INFORMATION
WARNING: SERIOUS MENINGOCOCCAL INFECTIONS
Soliris increases the risk of meningococcal infections. Meningococcal infection may become rapidly life-threatening or fatal if not recognized and treated early
- Vaccinate patients with a meningococcal vaccine at least 2 weeks prior to receiving the first dose of Soliris; revaccinate according to current medical 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.
The most frequent adverse events observed in clinical studies were headache, nasopharyngitis, back pain, nausea, and fatigue.
SEE FULL PRESCRIBING INFORMATION FOR COMPLETE BOXED WARNING INCLUDING WARNINGS, PRECAUTIONS, AND ADVERSE REACTIONS.
References: 1. Brodsky RA, Muus P, Dührsen U, et al. Effect of the terminal complement inhibitor eculizumab on patient reported outcomes in paroxysmal nocturnal hemoglobinuria (PNH): phase III TRIUMPH study results [ASH abstract]. Blood. 2006;108: Abstract 3770. 2. Hillmen P, Young NS, Schubert J, et al. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2006;355:1233-1243. 3. Socié G, Hillmen P, Muus P, et al. Sustained improvements in transfusion requirements, fatigue and thrombosis with eculizumab treatment in paroxysmal nocturnal hemoglobinuria [ASH abstract]. Blood. 2007;110: Abstract 3672. 4. Richards SJ, Cullen MJ, Dickinson AJ, et al. The effect of eculizumab therapy on red cell response kinetics in patients with paroxysmal nocturnal hemoglobinuria [ASH abstract]. Blood. 2005;106: Abstract 1047. 5. Hillmen P, Muus P, Dührsen U, et al. Effect of the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria. Blood. 2007;110:4123-4128. 6. Soliris® [package insert]. Cheshire, CT: Alexion Pharmaceuticals Inc; 2009. 7. Parker C, Omine M, Richards S, et al; for International PNH Interest Group. Diagnosis and management of paroxysmal nocturnal hemoglobinuria. Blood. 2005;106:3699-3709. 8. de Castro CM. Paroxysmal Nocturnal Hemoglobinuria (PNH) Basic Explanations. Annapolis, MD: Aplastic Anemia & MDS International Foundation; 2006. 9. Brodsky RA. Paroxysmal nocturnal hemoglobinuria. In: Hoffman R, Benz EJ Jr, Shattil SJ, et al, eds. Hematology: Basic Principles and Practice. 4th ed. Philadelphia, PA: Elsevier Churchill Livingstone; 2005:419-427. 10. Parker CJ, Wiedmer T, Sims PJ, Rosse WF. Characterization of the complement sensitivity of paroxysmal nocturnal hemoglobinuria erythrocytes. J Clin Invest. 1985;75:2074-2084. 11. Parker C. Ask the hematologist: how do I diagnose and treat PNH? The Hematologist: ASH News and Reports. Available at: http://www.hematology.org/Publications/Hematologist/2008/1252.aspx#top. Accessed August 13, 2009.