Paroxysmal Nocturnal Hemoglobinuria: Current Management, Unmet Needs, and Recommendations

Paroxysmal nocturnal hemoglobinuria Paroxysmal nocturnal hemoglobinuria: (par-uk-SIZ-muhl nok-TURN-uhl hee-muh-gloe-buh-NYOOR-ee-uh) A rare and serious blood disease that causes red blood cells to break apart. Paroxysmal means sudden and irregular. Nocturnal means at night. Hemoglobinuria means hemoglobin in the urine. Hemoglobin is the red part of red blood cells. A… (PNH) is an ultra-rare, acquired clonal abnormality, which renders hematopoietic cells exquisitely sensitive to complement-mediated destruction. Classical features of PNH include intravascular hemolytic anemia hemolytic anemia: Anemia due primarily to the excessive hemolysis or destruction of red blood cells , increased thrombotic risk, and manifestations related to end-organ damage (eg fatigue, chest pain, dyspnea, renal failure, and pulmonary hypertension). With supportive care supportive care: Care given to improve the quality of life, or comfort, of a person with a chronic illness. Supportive care treats the symptoms rather than the underlying cause of a disease. The goal is to help the patient feel better. Patients with low blood counts may be given blood transfusions as supportive… alone, mortality rate of patients with PNH is approximately 35%. The anti-C5 monoclonal antibody monoclonal antibody: A type of protein called an antibody that is engineered to look for a specific substance in the body. There are many kinds of monocloncal antibodies. Each one looks for only one substance. Eculizumab (Soliris) is an monoclonal antibody that may be prescribed to treat patients with paroxysmal… , eculizumab eculizumab: Eculizumab (Soliris ®) is given as an IV into a vein at the doctor’s office or at a special center. The procedure usually takes about 35 minutes. You will probably get an IV once a week for the first 4 weeks. Starting in the 5th week, you will get a slightly higher dose of Soliris every 2 weeks. … , was the first targeted therapy approved for PNH, and led to improved hemoglobin hemoglobin: A protein in the red blood cells. Hemoglobin picks up oxygen in the lungs and brings it to cells in all parts of the body. , quality of life, reduced transfusion need, reduced thrombosis thrombosis: (throm-BOE-suss) A blood clot (thrombus) that develops and attaches to a blood vessel. , and greater overall survival. More recently, therapeutics such as longer acting anti-C5 (ravulizumab) and anti-C3 (pegcetacoplan) medications have been approved, along with other novel therapeutics in late-stage clinical trials clinical trials: Clinical research is at the heart of all medical advances, identifying new ways to prevent, detect or treat disease. If you have a bone marrow failure disease, you may want to consider taking part in a clinical trial, also called a research study. Understanding Clinical Trials Clinical… . Biosimilars Biosimilars: What is a Biosimilar? A biosimilar is a type of biologic medication. The term “biosimilar” means that the medication is highly similar to the already approved brand name biologic (“originator” medication). Biosimilars are not generic versions of the original biologic. Difference Between… of eculizumab are also now available. Proximal inhibitors (against C3, factor B, and factor D) have shown significant improvements in hemoglobin and transfusion-avoidance in patients who remain anemic despite C5 inhibition. Despite these novel therapies, some unmet challenges remain, including management of breakthrough hemolysis hemolysis: (hi-MOL-uh-suss) The destruction of red blood cells. , clinically significant iatrogenic extravascular hemolysis, optimal management in pregnancy, and infection risk mitigation as new targets in the complement system complement system: A group of proteins that move freely in the bloodstream. These proteins support (complement) the work of white blood cells by fighting infections. are blocked. In addition, the use of self-administered subcutaneous and oral therapies raises concerns around treatment adherence and the risks of uncontrolled terminal complement. Given the ultra-rare nature of PNH, development is underway of a centralized international registry to capture and analyze the data as they mature for various new therapies and characterize the clinical challenges related to PNH management.