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It can cause intravascular hemolysis, thrombosis, and varying degrees of bone marrow failure. Some patients develop dark urine, anemia, fatigue, abdominal pain, or blood clots, while others may not have visible hemoglobinuria. ",[68,74,78,82,86],{"key":69,"title":70,"html":71,"icon":72,"updatedAt":73},"","Overview","\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">PNH is an acquired blood disorder caused by a genetic change in a hematopoietic stem cell.\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">\u003Cbr\u002F>\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">Despite the term “nocturnal” in its name, hemolysis does not occur only at night or after sleep. The name reflects early observations of the disease. Hemolysis can occur at any time, and not every patient develops visibly dark urine.\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">\u003Cbr\u002F>\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">The three major clinical problems in PNH are \u003Cstrong>intravascular hemolysis, thrombosis, and bone marrow failure\u003C\u002Fstrong>. The presentation varies widely. Some patients mainly have hemolysis and anemia, some first present with thrombosis, and others have PNH together with bone marrow failure disorders such as aplastic anemia.\u003C\u002Fspan>\u003C\u002Fp>","⚕️","2026-10-09",{"key":69,"title":75,"html":76,"icon":77,"updatedAt":73},"Causes","\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">PNH is associated with an acquired mutation in the \u003Cstrong>PIGA gene\u003C\u002Fstrong> in a hematopoietic stem cell.\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">\u003Cbr\u002F>\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">PIGA is involved in the production of glycosylphosphatidylinositol (GPI) anchors. When this pathway is disrupted, blood cells lose several GPI-anchored proteins, including the complement-regulating proteins \u003Cstrong>CD55 and CD59\u003C\u002Fstrong>.\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">\u003Cbr\u002F>\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">Without these protective proteins, red blood cells become unusually vulnerable to complement-mediated destruction, leading to intravascular hemolysis.\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">\u003Cbr\u002F>\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">The reason why some people develop a clinically significant PNH clone after acquiring a PIGA mutation is not fully understood.\u003C\u002Fspan>\u003C\u002Fp>","🧬",{"key":69,"title":79,"html":80,"icon":81,"updatedAt":73},"Signs and Symptoms","\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">Symptoms of PNH vary considerably, and not everyone develops the classic dark-colored urine.\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">\u003Cbr\u002F>\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">Possible signs and symptoms include:\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">\u003Cbr\u002F>\u003C\u002Fspan>\u003C\u002Fp>\u003Cul class=\" list-paddingleft-2\" style=\"list-style-type: disc;\">\u003Cli>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">Fatigue, weakness, pale skin, or shortness of breath from anemia\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Fli>\u003Cli>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">Red-brown, tea-colored, or very dark urine during significant hemolysis\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Fli>\u003Cli>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">Jaundice\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Fli>\u003Cli>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">Abdominal pain\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Fli>\u003Cli>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">Difficulty swallowing or esophageal spasm\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Fli>\u003Cli>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">Erectile dysfunction in men\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Fli>\u003Cli>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">Headache, chest pain, or shortness of breath\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Fli>\u003Cli>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">Bleeding related to low platelet counts\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Fli>\u003Cli>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">Increased infection risk when white blood cell counts are low\u003C\u002Fspan>\u003C\u002Fp>\u003C\u002Fli>\u003C\u002Ful>\u003Cp style=\"margin: 7px 0;text-align: justify;\">\u003Cspan style=\"font-size: 15px;\"> \u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">Thrombosis is one of the most serious complications of PNH. Blood clots can occur in common veins as well as unusual sites such as the hepatic, portal, mesenteric, or cerebral veins.\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">\u003Cbr\u002F>\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">Dark urine in PNH is caused by \u003Cstrong>free hemoglobin being filtered into the urine\u003C\u002Fstrong>, rather than intact destroyed blood cells being excreted.\u003C\u002Fspan>\u003C\u002Fp>","📋",{"key":69,"title":83,"html":84,"icon":85,"updatedAt":73},"Diagnosis","\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">Diagnosis of PNH usually combines blood counts, laboratory evidence of hemolysis, and peripheral blood flow cytometry.\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">\u003Cbr\u002F>\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">\u003Cstrong>Blood Tests\u003C\u002Fstrong>\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">\u003Cstrong>\u003Cbr\u002F>\u003C\u002Fstrong>\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">A complete blood count may show anemia, and some patients also have low white blood cell or platelet counts.\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">\u003Cbr\u002F>\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">Laboratory tests for hemolysis may include LDH, bilirubin, reticulocyte count, haptoglobin, and kidney function.\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">\u003Cbr\u002F>\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">\u003Cstrong>Flow Cytometry\u003C\u002Fstrong>\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">\u003Cstrong>\u003Cbr\u002F>\u003C\u002Fstrong>\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">High-sensitivity flow cytometry is the key test used to confirm PNH.\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">\u003Cbr\u002F>\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">Testing usually evaluates GPI-anchored proteins on granulocytes and monocytes and may use \u003Cstrong>FLAER\u003C\u002Fstrong> together with markers such as CD24, CD14, CD55, and CD59.\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">\u003Cbr\u002F>\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">Modern diagnosis does not rely on red blood cell CD55 and CD59 testing alone, because transfusions and ongoing hemolysis can affect red blood cell results.\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">\u003Cstrong>\u003Cbr\u002F>\u003C\u002Fstrong>\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">\u003Cstrong>Bone Marrow Testing\u003C\u002Fstrong>\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">\u003Cstrong>\u003Cbr\u002F>\u003C\u002Fstrong>\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">Bone marrow aspiration or biopsy does not directly diagnose PNH but may be important in patients with pancytopenia or suspected aplastic anemia, myelodysplastic neoplasms, or another bone marrow failure disorder.\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">\u003Cbr\u002F>\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">\u003Cstrong>Urine Tests\u003C\u002Fstrong>\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">\u003Cstrong>\u003Cbr\u002F>\u003C\u002Fstrong>\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">Urinalysis may show hemoglobin in the urine, sometimes without a corresponding increase in intact red blood cells.\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">\u003Cbr\u002F>\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">Older tests such as the Ham test and sucrose lysis test have largely been replaced by high-sensitivity flow cytometry and are no longer the primary diagnostic tests for PNH.\u003C\u002Fspan>\u003C\u002Fp>","🩺",{"key":69,"title":87,"html":88,"icon":89,"updatedAt":73},"Treatment","\u003Ch3 style=\"margin-top: 17px;margin-bottom: 17px;break-after: avoid;text-align: justify;\">\u003Cspan style=\"font-size: 15px;\">\u003Cstrong style=\"font-size: 15px;\">\u003C\u002Fstrong>\u003C\u002Fspan>\u003C\u002Fh3>\u003Cp style=\"margin: 7px 0;\">\u003Cstrong style=\"text-align: justify; font-size: 15px;\">Complement Inhibitors\u003C\u002Fstrong>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cbr\u002F>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">Complement inhibitors block different parts of the complement system and reduce complement-mediated destruction of red blood cells.\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">\u003Cbr\u002F>\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">C5 inhibitors include \u003Cstrong>eculizumab and ravulizumab\u003C\u002Fstrong>. Newer treatments target other parts of the complement pathway, including C3 and the alternative complement pathway.\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">\u003Cbr\u002F>\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">The choice of therapy depends on previous treatment, severity of anemia, ongoing hemolysis, and local approval and availability.\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cstrong style=\"font-size: 15px; text-align: justify;\">\u003Cbr\u002F>\u003C\u002Fstrong>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cstrong style=\"font-size: 15px; text-align: justify;\">Supportive Care\u003C\u002Fstrong>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cbr\u002F>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">Supportive care may include red blood cell transfusions, folic acid supplementation, and treatment of infections, kidney problems, or other complications.\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">\u003Cbr\u002F>\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">Iron replacement may be appropriate when true iron deficiency is confirmed. Long-term hemoglobin loss in the urine can itself lead to iron deficiency in some patients.\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">\u003Cbr\u002F>\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">Patients with significant bone marrow failure may need additional treatment directed at conditions such as aplastic anemia.\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cstrong style=\"font-size: 15px; text-align: justify;\">\u003Cbr\u002F>\u003C\u002Fstrong>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cstrong style=\"font-size: 15px; text-align: justify;\">Thrombosis Prevention and Treatment\u003C\u002Fstrong>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cbr\u002F>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">Patients who develop thrombosis generally need anticoagulation, together with evaluation for initiation or optimization of complement inhibitor therapy.\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">\u003Cbr\u002F>\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">Preventive anticoagulation is not a simple “yes or no” decision and should be individualized based on thrombosis risk, bleeding risk, previous clotting events, disease activity, and complement inhibitor treatment.\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cstrong style=\"font-size: 15px; text-align: justify;\">\u003Cbr\u002F>\u003C\u002Fstrong>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cstrong style=\"font-size: 15px; text-align: justify;\">Hematopoietic Stem Cell Transplantation\u003C\u002Fstrong>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cbr\u002F>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">\u003Cstrong>Allogeneic hematopoietic stem cell transplantation (allo-HSCT)\u003C\u002Fstrong> is the treatment with curative potential because it can replace the abnormal hematopoietic stem cell clone.\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">\u003Cbr\u002F>\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">However, complement inhibitors can now effectively control hemolysis and thrombosis in many patients with classical PNH, so transplantation is not routinely chosen simply because a patient has PNH.\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">\u003Cbr\u002F>\u003C\u002Fspan>\u003C\u002Fp>\u003Cp style=\"margin: 7px 0;\">\u003Cspan style=\"font-size: 15px;\">It is more often considered in selected patients with severe bone marrow failure, inadequate response to other treatment, or other specific high-risk features.\u003C\u002Fspan>\u003C\u002Fp>","💊",{"author":69,"reviewedBy":69,"lastReviewed":69,"datePublished":69,"citations":91,"disclaimer":69},[],false,1792294328441]