Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, life-threatening disease of the blood. The disease is characterized by destruction of red blood cells, blood clots, and impaired bone marrow function. PNH is closely related to aplastic anemia.
Experts at The Johns Hopkins Kimmel Cancer are nationally and internationally recognized as leaders in the research and treatment of paroxysmal nocturnal hemoglobinuria and other blood disorders.
At the Johns Hopkins Kimmel Cancer Center, PNH patients are managed by experts from the Bone Marrow Failures Disorders Program. The appropriate treatment for PNH depends on the severity of symptoms.
Find information about open paroxysmal nocturnal hemoglobinuria studies offered at the Johns Hopkins Kimmel Cancer Center.
The most widely used drug to treat PNH is eculizumab (Soliris). The drug binds to proteins in the blood that can destroy red blood cells. It reduces the risk of blood clotting and can improve quality of life in PNH patients. A newer drug called ravulizumab (Ultomiris) works in a similar way to eculizumab.
Bone Marrow Transplant
A bone marrow transplant gives a patient healthy stem cells – immature cells that grow into different types of blood cells. These stem cells come from marrow, the soft, fatty tissue inside the bones, replacing marrow that is not working properly.
In this therapy, the patient's non-functioning bone marrow is destroyed with drugs and/or radiation and replaced with donor bone marrow from a compatible donor, usually a sibling or other family member. Bone marrow transplant may be recommended for patients who do not respond to eculizumab or for those who have severe reductions in red blood cells, white blood cells and platelets. The transplant is usually recommended for young patients with very severe disease with significant marrow failure. (Learn more about the Johns Hopkins Bone Marrow Transplant Program.)
Clinical trials test new drugs and new ways of treating patients with PNH. Our experts conduct these studies to find better treatments. Many clinical trials become the next standard of care. To determine if a trial is right for you, make an appointment for a consultation at 410.955.8964.