How I Treat Paroxysmal nocturnal hemoglobinuria

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 a rare, clonal, complement-mediated hemolytic anemia hemolytic anemia: Anemia due primarily to the excessive hemolysis or destruction of red blood cells with protean manifestations. PNH can present as a hemolytic anemia, a form of bone marrow failure bone marrow failure: A condition that occurs when the bone marrow stops making enough healthy blood cells. The most common of these rare diseases are aplastic anemia, myelodysplastic syndromes (MDS) and paroxysmal nocturnal hemoglobinuria (PNH). Bone marrow failure can be acquired (begin any time in life) or can be… , a thrombophilia, or any combination of the above. Terminal complement inhibition is highly effective for treating intravascular hemolysis hemolysis: (hi-MOL-uh-suss) The destruction of red blood cells. from PNH and virtually eliminates the risk of thrombosis thrombosis: (throm-BOE-suss) A blood clot (thrombus) that develops and attaches to a blood vessel. , but is not effective for treating bone marrow failure. Here, we present a variety of clinical vignettes that highlight the clinical heterogeneity of PNH as well as the attributes and limitations of the two FDA-approved C5 inhibitors (eculizumab and ravulizumab) to treat PNH. We review the concept of pharmacokinetic and pharmacodynamic breakthrough hemolysis and briefly discuss new complement inhibitors upstream of C5 that are in clinical development. Lastly, we discuss the rare indications for bone marrow bone marrow: The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow. transplantation in PNH patients.