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Eculizumab
Eculizumab, sold under the brand name Soliris among others, is a medication used to treat paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), generalized myasthenia gravis, and neuromyelitis optica. In people with PNH, it reduces both the destruction of red blood cells and need for blood transfusion, but does not appear to affect the risk of death. Eculizumab was the first drug approved for each of its uses, and its approval was granted based on small trials. It is given in a clinic by intravenous (IV) infusion. Side effects include a risk for meningococcal infections and it is only prescribed to those who have enrolled in and follow a risk evaluation and mitigation strategy, which involves counseling people and ensuring that they are vaccinated. It is a humanized monoclonal antibody functioning as a terminal complement inhibitor. It has been developed, manufactured, and marketed by Alexion Pharmaceuticals, which had patent exclusivity un ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Paroxysmal Nocturnal Hemoglobinuria
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, life-threatening disease of the blood characterized by destruction of red blood cells by the complement system, a part of the body's innate immune system. This destructive process occurs due to deficiency of the red blood cell surface protein DAF, which normally inhibits such immune reactions. Since the complement cascade attacks the red blood cells within the blood vessels of the circulatory system, the red blood cell destruction (hemolysis) is considered an ''intravascular'' hemolytic anemia. Other key features of the disease, such as the high incidence of venous blood clot formation, are incompletely understood. PNH is the only hemolytic anemia caused by an ''acquired'' (rather than inherited) intrinsic defect in the cell membrane (deficiency of glycophosphatidylinositol or GPI) leading to the absence of protective exterior surface proteins that normally attach via a GPI anchor. It may develop on its own ("primary ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Atypical Hemolytic Uremic Syndrome
Atypical hemolytic uremic syndrome (aHUS) is an extremely rare, life-threatening, progressive disease that frequently has a genetic component. In most cases it can be effectively controlled by interruption of the complement cascade. Particular monoclonal antibodies, discussed later in the article, have proven efficacy in many cases. AHUS is usually caused by chronic, uncontrolled activation of the complement system, a branch of the body's immune system that destroys and removes foreign particles. The disease affects both children and adults and is characterized by systemic thrombotic microangiopathy (TMA), the formation of blood clots in small blood vessels throughout the body, which can lead to stroke, heart attack, kidney failure, and death. The complement system activation may be due to mutations in the complement regulatory proteins (factor H, factor I, or membrane cofactor protein), or is occasionally due to acquired neutralizing autoantibody inhibitors of these complement ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Alexion Pharmaceuticals
Alexion Pharmaceuticals is an American pharmaceutical company headquartered in Boston, Massachusetts that specializes in orphan drugs to treat rare diseases. It became an independent subsidiary of AstraZeneca in 2021. Its products include eculizumab (Soliris) with $4.064 billion in 2020 revenues and ravulizumab (Ultomiris) with $1.076 billion in 2020 revenues, both used to treat the rare disorders of atypical hemolytic uremic syndrome (aHUS) and paroxysmal nocturnal hemoglobinuria (PNH); asfotase alfa (Strensiq) with $731 million in 2020 revenues, used to treat hypophosphatasia; sebelipase alfa (Kanuma) with $117 million in 2020 revenues, used to treat lysosomal acid lipase deficiency, and andexanet alfa (Andexxa) with $78 million in 2020 revenues, used to stop life threatening or uncontrollable bleeding in people who are taking rivaroxaban or apixaban. With costs that can reach as much as $2 million per year, the drugs manufactured by Alexion are some of the most expensive dru ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Neuromyelitis Optica
Neuromyelitis optica spectrum disorders (NMOSD), including neuromyelitis optica (NMO), are autoimmune diseases characterized by acute inflammation of the optic nerve (optic neuritis, ON) and the spinal cord (myelitis). Episodes of ON and myelitis can be simultaneous or successive. A relapsing disease course is common, especially in untreated patients. In more than 80% of cases, NMO is caused by immunoglobulin G Autoantibody, autoantibodies to aquaporin 4 (anti-AQP4 diseases, anti-AQP4), the most abundant Aquaporin, water channel protein in the central nervous system. A subset of anti-AQP4-negative cases is associated with antibodies against myelin oligodendrocyte glycoprotein (Anti-MOG associated encephalomyelitis, anti-MOG). Rarely, NMO may occur in the context of other autoimmune diseases (e.g. Connective tissue disease, connective tissue disorders, paraneoplastic syndromes) or infectious diseases. In some cases, the etiology remains unknown (Idiopathic disease, idiopathic NMO). ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
CD55 Deficiency
CD55 deficiency, also called DAF deficiency or CHAPLE syndrome, is a rare genetic disorder of the immune system. CHAPLE stands for "CD55 deficiency with hyper-activation of complement, angiopathic thrombosis, and severe protein-losing enteropathy (PLE)." The disorder usually manifests in childhood and can be life-threatening. This condition was described by Özen, et al. in 2017. Signs and symptoms CHAPLE is characterized by severe protein-losing enteropathy leading to hypoproteinemia. Symptoms can include abdominal pain, nausea, vomiting, diarrhea, loss of appetite, weight loss, and edema. People also have chronic malabsorption, which causes deficiencies in iron, ferritin, calcium, magnesium, folate, vitamin D and vitamin B12. Some patients may have recurrent respiratory infections associated with hypogammaglobulinemia. Severe thrombotic vascular occlusions may also be found among these patients. Genetics CHAPLE syndrome is caused by mutations of the complement regulato ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Complement Component 5
Complement component 5 is a protein that in humans is encoded by the ''C5'' gene. Complement component 5 is involved in the complement system. It is cleaved into C5a and C5b: * C5a plays an important role in chemotaxis. * C5b forms the first part of the complement membrane attack complex. Deficiency is thought to cause Leiner's disease. Function Complement component 5 is the fifth component of complement, which plays an important role in inflammatory and cell killing processes. This protein is composed of alpha and beta polypeptide chains that are linked by a disulfide bridge. An activation peptide, C5a, which is an anaphylatoxin that possesses potent spasmogenic and chemotactic activity, is derived from the alpha polypeptide via cleavage with a C5-convertase. The C5b macromolecular cleavage product can form a complex with the C6 complement component, and this complex is the basis for formation of the membrane attack complex, which includes additional complement components. ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Complement System
The complement system, also known as complement cascade, is a part of the immune system that enhances (complements) the ability of antibodies and phagocytic cells to clear microbes and damaged cells from an organism, promote inflammation, and attack the pathogen's cell membrane. It is part of the innate immune system, which is not adaptable and does not change during an individual's lifetime. The complement system can, however, be recruited and brought into action by antibodies generated by the adaptive immune system. The complement system consists of a number of small proteins that are synthesized by the liver, and circulate in the blood as inactive precursors. When stimulated by one of several triggers, proteases in the system cleave specific proteins to release cytokines and initiate an amplifying cascade of further cleavages. The end result of this ''complement activation'' or ''complement fixation'' cascade is stimulation of phagocytes to clear foreign and damaged material ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Neisseria Meningitidis
''Neisseria meningitidis'', often referred to as meningococcus, is a Gram-negative bacterium that can cause meningitis and other forms of meningococcal disease such as meningococcemia, a life-threatening sepsis. The bacterium is referred to as a coccus because it is round, and more specifically a diplococcus because of its tendency to form pairs. About 10% of adults are carriers of the bacteria in their nasopharynx. As an exclusively human pathogen, it is the main cause of bacterial meningitis in children and young adults, causing developmental impairment and death in about 10% of cases. It causes the only form of bacterial meningitis known to occur epidemically, mainly in Africa and Asia. It occurs worldwide in both epidemic and endemic form. ''N. meningitidis'' is spread through saliva and respiratory secretions during coughing, sneezing, kissing, chewing on toys and through sharing a source of fresh water. It has also been reported to be transmitted through oral sex and cause ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Black Box Warning
In the United States, a boxed warning (sometimes "black box warning", colloquially) is a type of warning that appears on the package insert for certain prescription drugs, so called because the U.S. Food and Drug Administration specifies that it is formatted with a 'box' or border around the text. The FDA can require a pharmaceutical company to place a boxed warning on the labeling of a prescription drug, or in literature describing it. It is the strongest warning that the FDA requires, and signifies that medical studies indicate that the drug carries a significant risk of serious or even life-threatening adverse effects. Economists and physicians have thoroughly studied the effects of FDA boxed warnings on prescription patterns. It is not necessarily true that a physician and patient will have a conversation about a drug's boxed warning after it is issued. For instance, an FDA-mandated boxed warning decreased rosiglitazone use by 70%, but that still meant 3.8 million people wer ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Meningococcal Disease
Meningococcal disease describes infections caused by the bacterium ''Neisseria meningitidis'' (also termed meningococcus). It has a high mortality rate if untreated but is vaccine-preventable. While best known as a cause of meningitis, it can also result in sepsis, which is an even more damaging and dangerous condition. Meningitis and meningococcemia are major causes of illness, death, and disability in both developed and under-developed countries. There are approximately 2,600 cases of bacterial meningitis per year in the United States, and on average 333,000 cases in developing countries. The case fatality rate ranges between 10 and 20 percent. The incidence of endemic meningococcal disease during the last 13 years ranges from 1 to 5 per 100,000 in developed countries, and from 10 to 25 per 100,000 in developing countries. During epidemics the incidence of meningococcal disease approaches 100 per 100,000. Meningococcal vaccines have sharply reduced the incidence of the diseas ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Thrombotic Microangiopathy
Thrombotic microangiopathy (TMA) is a pathology that results in thrombosis in capillaries and arterioles, due to an endothelial injury. It may be seen in association with thrombocytopenia, anemia, purpura and kidney failure. The classic TMAs are hemolytic uremic syndrome and thrombotic thrombocytopenic purpura. Other conditions with TMA include atypical hemolytic uremic syndrome, disseminated intravascular coagulation, scleroderma renal crisis, malignant hypertension, antiphospholipid antibody syndrome, and drug toxicities, e.g. calcineurin inhibitor toxicity. Signs and symptoms The clinical presentation of TMA, although dependent on the type, typically includes: fever, microangiopathic hemolytic anemia (see schistocytes in a blood smear), kidney failure, thrombocytopenia and neurological manifestations. Generally, renal complications are particularly predominant with Shiga-toxin-associated hemolytic uremic syndrome (STx-HUS) and atypical HUS, whereas neurologic complicatio ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Anemia
Anemia or anaemia (British English) is a blood disorder in which the blood has a reduced ability to carry oxygen due to a lower than normal number of red blood cells, or a reduction in the amount of hemoglobin. When anemia comes on slowly, the symptoms are often vague, such as tiredness, weakness, shortness of breath, headaches, and a reduced ability to exercise. When anemia is acute, symptoms may include confusion, feeling like one is going to pass out, loss of consciousness, and increased thirst. Anemia must be significant before a person becomes noticeably pale. Symptoms of anemia depend on how quickly hemoglobin decreases. Additional symptoms may occur depending on the underlying cause. Preoperative anemia can increase the risk of needing a blood transfusion following surgery. Anemia can be temporary or long term and can range from mild to severe. Anemia can be caused by blood loss, decreased red blood cell production, and increased red blood cell breakdown. Causes o ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
