An error occurred while setting your user cookie. Please set your. browser to accept cookies to continue. NEJM. org uses cookies to improve performance by remembering your. ID when you navigate from page to page. This cookie stores just a. ID no other information is captured. Accepting the NEJM cookie is. Les fichiers tlchargs sont au format PDF. Pour les lire, utilisez la dernire version du logiciel Adobe Reader. Ce format est destin faciliter la. Case Records of the Massachusetts General Hospital. Case 292017 A 59YearOld Woman with Pain and Swelling in the Right Hand and Ankles. Presentation of Case. Dr. Studies have not been conducted to evaluate the pharmacokinetics of eculizumab in special patient populations identified by gender, race, age pediatric or geriatric. A broad spectrum of coagulation analyzers is available for purchase on the market today. Laboratories can choose from manual, semiautomated or fully automated. Journal Roundup, Part 2 New Studies on Phlebotomy This month, Phlebotomy TodaySTAT continues its roundup of recent publications appearing in the literature. Eculizumab SolirisNumber 0. Policy. Note Site of Care Utilization Management Policy applies. For information on site of service for Soliris infusions, see Utilization Management Policy on Site of Care for Specialty Drug Infusions. Aetna considers eculizumab Soliris medically necessary for the treatment of paroxysmal nocturnal hemoglobinuria PNH to reduce hemolysis when all of the following criteria are met Documented diagnosis of PNH, with flow cytometric confirmation of at least 1. PNH type III red cells or greater than 5. GPI AP deficient poly morphonuclear cells PMNs and Member has been vaccinated against meningococcal infection at least 2 weeks prior to eculizumab treatment, if not previously vaccinated and. Member does not meet diagnostic criteria for severe aplastic anemia see Appendix and. Member meets either of the following criteria. Member is transfusion dependent i. L in persons without anemic symptoms or less than 9 gd. L in persons with symptoms from anemia prior to initiation of eculizumab treatment or Member has a documented history of major adverse vascular events from thromboembolism see Appendix. Aetna considers continued use of eculizumab medically necessary for persons with paroxysmal nocturnal hemoglobunuria who met medical necessity criteria at the initiation of treatment and who have experienced a reduction in hemolysis as manifested by a stabilization of hemoglobin levels and reduction in transfusions from baseline at initiation. Aetna considers eculizumab medically necessary for the treatment of atypical hemolytic uremic syndrome without serious unresolved Neisseria meningitidis infection, and the member has been vaccinated against meningococcal infection at least 2 weeks prior to eculizumab treatment, if not previously vaccinated. Aetna considers continued use of eculizumab medically necessary for persons with atypical hemolytic uremic syndrome who met medical necessity criteria at the initiation of treatment and who have a reduction in signs of complement mediated thrombotic microangiopathy TMA including at least 2 of the following Platelet count increase from baseline at initiation. Hematologic normalization maintenance of normal platelet counts and LDH levels for at least 4 weeksComplete TMA response hematologic normalization plus at least 2. TMA even free status absence for at least 1. Daily TMA intervention rate defined as the number of plasma exchange or plasma infusion interventions and the number of new dialysis required per patient per dayNote While receiving eculizumab, the member should be revaccinated according to current medical guidelines for vaccine use while on eculizumab therapy REMS program. Aetna considers eculizumab experimental and investigational when criteria are not met and for all other indications including the following not an all inclusive list because its effectiveness for these indications has not been established Age related macular degeneration. D Cube R2 Software Engineering on this page. Antibody mediated rejection. Anti neutrophil cytoplasmic autoantibody ANCA vasculitis. Patient. A 45yearold male patient with thalassemia from the Department of Rare Diseases, National Research Center for Hematology, was enrolled in the study, and. Anti phospholipid antibody syndrome. Autoimmune hemolytic anemia. C3 glomerulopathyglomerulonephritisnephropathy. Dense deposit disease. Guillain Barre syndrome. Hemolytic cold agglutinin disease. Hyperhemolysis syndrome. Ig. A nephropathy. Inflammatory myositis e. Multi focal motor neuropathy. Myasthenia gravis. Neuromyelitis optica Devics diseaseNon exudative dry macular degeneration. Preeclampsia with hemolysis, elevated liver enzymes and low platelets HELLP syndrome. Prevention of intravascular hemolysis due to red blood cell alloantibodies. Shiga toxin E. coli related hemolytic uremic syndrome STEC HUSStem cell transplant associated thrombotic microangiopathy. Systemic lupus erythematosus. Thrombotic thrombocytopenic purpura TTPTransverse myelitis. For site of service for eculizumab infusions, see Utilization Management Policy on Site of Care for Drug Infusions. See CPB 0. 63. 4 Non myeloablative Bone MarrowPeripheral Stem Cell Transplantation Mini Allograft Reduced Intensity Conditioning Transplant. Background. Case Records of the Massachusetts General Hospital. Case 302017 A 65YearOld Woman with Altered Mental Status, Bacteremia, and Acute Liver Failure. Soliris eculizumab is a recombinant humanized monoclonal antibody Ig. G24k. Soliris eculizumab binds specifically to the complement protein C5 with high affinity, thereby inhibiting its cleavage to C5a and C5b and preventing the generation of the terminal complement complex C5b9. Eculizumab inhibits terminal complement mediated intravascular hemolysis in patients with paroxysmal nocturnal hemoglobinuria PNH and atypical hemolytic uremic syndrome a. HUS. Soliris eculizumab has been approved by the U. S. Food and Drug Administration FDA for the treatment of patients with PNH to reduce hemolysis and for the treatment of patients with atypical hemolytic uremic syndrome a. HUS to inhibit complementmediated thrombotic microangiopathy. Black Box Warnings Lifethreatening and fatal meningococcal infections have occurred in patients treated with Soliris eculizumab. Meningococcal infection may become rapidly lifethreatening or fatal if not recognized and treated early. Comply with the most current Advisory Committee on Immunization Practices ACIP recommendations for meningococcal vaccination in patients with complement deficiencies. Immunize patients with a meningococcal vaccine at least 2 weeks prior to administering the first dose of Soliris eculizumab, unless the risks of delaying Soliris eculizumab therapy outweigh the risk of developing a meningococcal infection. Monitor patients for early signs of meningococcal infections and evaluate immediately if infection is suspected. Risk Evaluation and Mitigation Strategy REMS Under the Soliris REMS, prescribers must enroll in the program. Enrollment in the Soliris REMS program and additional information are available by telephone 18. SOLIRIS 18. 887. ACIP recommendations for the meningococcal vaccine can be accessed via the following http www. Paroxysmal nocturnal hemoglobinuria PNH, a rare form of hemolytic anemia, is an acquired genetic blood disorder characterized by red blood cells RBC that develop abnormally and are destroyed by the bodys own complement system. A genetic mutation in PNH patients leads to the generation of populations of abnormal of RBC known as PNH cells that are deficient in the terminal complement inhibitors, rendering PNH RBCs sensitive to persistent terminal complementmediated destruction. PNH is caused by a somatic mutation of the X linked phosphatidylinositol glycan class A PIGA gene, which results in the absence of the glycosylphosphatidylinositol linked proteins necessary to protect cells from complement mediated lysis. The destruction and loss of these PNH cells intravascular hemolysis results in low RBC counts that causes the symptoms of PNH, and can lead to disability and premature death. The symptoms of PNH can include thrombosis, pulmonary hypertension, and damage to organs such as the brain, liver, gastrointestinal system, and kidneys. Patients may also experience a variety of symptoms that can interfere with quality of life including abdominal pain, difficulty swallowing, poor physical function, shortness of breath, erectile dysfunction, and debilitating fatigue. About one person out of a million people will be diagnosed with PNH. An estimated 8,0. North America and Europe are affected by PNH. Prior to 1. 99. 0, diagnosis of PNH was made by means of complement based tests. In the past 1. Preis et al, 2. Uncontrolled complement activity in PNH leads to systemic complications, principally through intravascular hemolysis and platelet activation Hill et al, 2.