What Do Family and Friends Need to Know?
If a loved one has recently been diagnosed with PNH, one of the best ways you can help is by learning more about their disease. Some of the basic things you should know about PNH include:
- PNH is not an inherited disease. Just because you have it does not mean that another member of your family will have it or develop it.1
- PNH is not contagious. No one can get PNH from being in contact with you.1
- PNH is an acquired mutation that causes some or all of your body’s red blood cells (RBCs) to be destroyed by a process called hemolysis.2-4 PNH is a complex disease with signs and symptoms that are nonspecific, unpredictable, and often similar to those of other diseases. In addition, PNH presents uniquely in each person. If you have PNH, some or all of your RBCs may be missing an important protective protein. Without this protein, RBCs are prone to destruction by a part of your body’s defense system called complement.2-4
- The constant destruction of RBCs can cause, among other things, tiredness (fatigue) because the destroyed blood cells lose their ability to carry oxygen to parts of the body.1
- If you have PNH, chronic hemolysis is always taking place — whether you feel OK or whether you’re having a flare-up (paroxysm), such as during times of stress or infection.2,5-7
People with PNH may have other medical conditions that affect the function of their bone marrow such as aplastic anemia (AA) or myelodysplastic syndromes (MDS). Unlike PNH, which destroys RBCs, these diseases may reduce the production of blood cells and further complicate PNH.8 All of these contributing factors can make your symptoms inconsistent.
- PNH is a serious disease that should be addressed as quickly as possible. It can lead to life-threatening complications such as blood clots and damage to vital organs if not effectively treated.1,9 It is important for people with PNH to work closely with their physicians to manage their disease.
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 anticoagulant withdrawal during Soliris treatment has not been studied. Therefore, treatment with Soliris should not alter anticoagulant management.
Soliris is generally well tolerated. The most frequent adverse events observed in clinical studies were headache, a runny nose (nasopharyngitis), back pain, nausea, and tiredness (fatigue).
Please see full prescribing information for SOLIRIS, including boxed WARNING regarding serious meningitis.
References: 1. de Castro CM. Paroxysmal Nocturnal Hemoglobinuria (PNH) Basic Explanations. Annapolis, MD: Aplastic Anemia & MDS International Foundation; 2006.
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. Socié G, Mary J-Y, de Gramont A, et al, for the French Society of Haematology. Paroxysmal nocturnal haemoglobinuria: long-term follow-up and prognostic factors. Lancet. 1996;348:573-577.
4. Nishimura J-I, Kanakura Y, Ware RE, et al. Clinical course and flow cytometric analysis of paroxysmal nocturnal hemoglobinuria in the United States and Japan. Medicine. 2004;83:193-207.
5. Hillmen P, Young NS, Schubert J, et al. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2006;355:1233-1243.
6. Rother RP, Bell L, Hillmen P, Gladwin MT. The clinical sequelae of intravascular hemolysis and extracellular plasma hemoglobin: a novel mechanism of human disease. JAMA. 2005;293:1653-1662.
7. Hill A, Richards SJ, Hillmen P. Recent developments in the understanding and management of paroxysmal nocturnal haemoglobinuria. Br J Haematol. 2007;137:181-192.
8. Parker C, Omine M, Richards S, et al, for the International PNH Interest Group. Diagnosis and management of paroxysmal nocturnal hemoglobinuria. Blood. 2005;106:3699-3709.
9. 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.