The PNH Source - PNH: What You Need To Know

PNH: What You Need To Know

Facts and figures about PNH

Paroxysmal nocturnal hemoglobinuria (PNH) is currently estimated to affect approximately 8,000–10,000 people in North America and Western Europe [1]
PNH is a blood disorder that is caused by a mutation in your blood stem cells [2]
Although PNH was named after the dark urine (hemoglobinuria) many patients experience, only 26% of patients actually have this symptom at the time of their diagnosis [3]
Be sure to discuss all of your symptoms with your doctor. PNH may be overlooked or misdiagnosed because the signs and symptoms are nonspecific and vague
PNH can affect people of any age, but it’s most common for a patient to be diagnosed in his/her 3rd through 5th decade of life (20s through 40s), with a median age at the time of diagnosis of 42 years [3-5]
Although there are current treatment options to help ease the symptoms of PNH, to date, the only curative treatment option is a bone marrow transplant, which can be risky and is not recommended for all PNH patients
New therapies to help treat the underlying cause of PNH and its symptoms are being tested and may be available soon

References: 1. Richards SJ, Cullen MJ, Dickinson AJ, et al. Long term stability of red cell responses in patients with paroxysmal nocturnal haemoglobinuria (PNH) on eculizumab therapy: implications for the future treatment and management of PNH [abstract]. Br J Haematol. 2006;133(suppl 1):16. Abstract 169. 2. Rosse WF, Hillmen P, Schreiber AD. Immune-mediated hemolytic anemia. Hematology (Am Soc Hematol Educ Program). January 2004:48-62. 3. Dacie JV, Lewis SM. Paroxysmal nocturnal haemoglobinuria: clinical manifestations, haematology, and nature of the disease. Ser Haematol. 1972;5:3-23. 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. 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.