In this site, the Molina Clinical Policy is made accessible to you to guide you in your medical decisions. In a prospective, double-masked, randomized clinical trial, Yehoshua et al (2014) evaluated the effect of eculizumab on the growth of geographic atrophy (GA) in patients with ARMD. Selective therapeutic depletion of B-cells became possible with the availability of the anti-CD20 antibody rituximab and anti-CD22 antibody epratuzumab. Transfusion threshold of 7 g per deciliter -- the new normal. Bomback AS, Smith RJ, Barile GR, et al. We’ll send you a link to a feedback form. Two to 10 % die and 1/3 progress to end-stage renal failure at first episode. Semin Thromb Hemost. Eculizumab in transplant-associated thrombotic microangiopathy. Based on medical status at last follow-up, patients were divided into 2 groups: favorable (nâ=â15) and unfavorable outcomes (nâ=â18). Clinical practice. Drugs Today (Barc). Overview. 2018;22(2). 2013;12(6):554-562. 2018;33(8):1385-1394. You can change your cookie settings at any time. Several studies pointed to a pathogenetic role of the immune system in this neuropathy, although this has not yet been proved. Madkaikar M, Gupta M, Jijina F, Ghosh K. Paroxysmal nocturnal haemoglobinuria: Diagnostic tests, advantages, & limitations. 2009;373:759-767. The authors concluded that given the complexity of disease pathogenesis in C3G, a patient-tailored approach including a comprehensive workup of complement abnormalities is necessary to evaluate the best treatment options. Concurrent use of therapeutic plasma exchange (TPE) would remove eculizumab from the blood as well as replenish C5 available for activation, and supplemental doses would be required after each TPE session. 2008;111(4):1840-1847. Both normal-luminance and low-luminance visual acuities were measured throughout the study, and the low-luminance deficits were calculated as the difference between the letter scores. Long-term eculizumab therapy in a child with refractory immune complex-mediated membranoproliferative glomerulonephritis. Six miscarriages (8%) occurred during the first trimester. Transfusion. 2018;33(8):1277-1281. Hughes PD, Cohney SJ. Myasthenia gravis: Recommendations for clinical research standards. Kidney Int Rep. 2017;3(2):482-485. Recurrent dense deposit disease after renal transplantation: An emerging role for complementary therapies. Boston, MA; Alexion … Clinical Practice Guidelines. Larger, multi-institutional, controlled studies are certainly needed to better evaluate the use of eculizumab in HSCT recipients, but the authorsâ pilot data support that patients with TA-TMA and high risk features, who historically have very poor outcome, should at least be considered as early candidates for eculizumab, as delaying therapy may prevent patients from achieving the best response and maximizing recovery of organ function. The authors concluded that this review has detected an absence of evidence for eculizumab compared with placebo for treating paroxysmal nocturnal hemoglobinuria (PNH), in terms of overall survival, nonfatal thrombotic events, transformation to myelodysplastic syndrome and acute myelogenous leukemia, and development and recurrence of aplastic anemia on treatment. 2015;85:177-189. Kelly RJ, Höchsmann B, Szer J, et al. The authors concluded that response rate and OS after eculizumab in this cohort compared favorably with previously published data in TMA after allogeneic HSCT. These investigators noted that the natural history of acute AMR after positive cross-match kidney transplantation involves an acute rise in donor-specific alloantibody in the first few weeks following transplantation. Pittock SJ, Lennon VA, McKeon A, et al. 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This case demonstrated several noteworthy features including hyperhemolysis in a patient without a Hb disorder, the development of an antibody to an unknown RBC antigen, and the failure of eculizumab to prevent intravascular hemolysis after transfusion. Sanders D, Wolfe G, Benatar M et al. Transplantation. These conclusions are based on one small trial with risk of attrition and selective reporting bias.The authors concluded, therefore, that the prescription of eculizumab for treating patients with PNH can neither be supported nor rejected, unless new evidence from a large high quality trial alters this conclusion. Three of the 4 studies comparing 2 immunosuppressant regimes (azathioprine with methotrexate, ciclosporin with methotrexate, and intra-muscular methotrexate with oral methotrexate plus azathioprine) showed no statistically significant difference in efficacy between the treatment regimes. A number of pharmacologic agents that have been explored for the treatment of TA-TMA include rituximab, vincristine, defibrotide, pravastatin, and eculizumab. Damico FM, Gasparin F, Scolari MR, et al. no criteria for thrombotic thrombocytopenic purpura (serum ADAMTS 13 activity greater than 10 %). Eculizumab in pregnant patients with paroxysmal nocturnal hemoglobinuria. 2018;102(4):557-568. Hilleman P, Muus P, Duhrsen U, et al. Firstly, the authors' current treatment protocol was introduced. Aetna Inc. and its subsidiary companies are not responsible or liable for the content, accuracy, or privacy practices of linked sites, or for products or services described on these sites. Moreover, they stated that prospective trials are needed to confirm these results.Jodele et al (2015) stated that hematopoietic stem cell transplantation (HSCT)-associated thrombotic microangiopathy (TA-TMA) is now a well-recognized and potentially severe complication of HSCT that carries a high risk of death. Due to refractory disease the patient was treated with eculizumab (anti-C5) for 3 months in an attempt to rescue renal function. The reported incidence of TA-TMA after HSCT is 0 % to 74 % and has shown to be associated with mortality rate of up to 100 %. Pediatr Nephrol. 2012;7(5):748-756. Blood. Loos S, Oh J, Kemper MJ. Walsh and Johnson (2019) noted that HUS remains a leading cause of pediatric AKI. Thrombotic micro-angiopathy after allogeneic HSCT shares similarities with aHUS in the underlying pathomechanisms. Policies and guidelines Tools and resources Claims and billing Patient management Become a provider Contact us Provider News Center . Orandi BJ, Zachary AA, Dagher NN, et al. Onset is from the neonatal period to the adult age. Pediatr Nephrol. No 436/07. Schrezenmeier H, Hochsmann B. Curr Drug Targets. Pediatr Nephrol. VI. Eculizumab compared with placebo showed a greater proportion of patients with transfusion independence: 51% (22/43) versus 0% (0/44); risk ratio (RR) 46.02, 95% CI 2.88 to 735.53; P = 0.007; moderate quality of evidence; and withdrawal for any reason: 4.7% (2/43) versus 22.72% (10/44); RR 0.20, 95% CI 0.05 to 0.88; P = 0.03; moderate quality of evidence. Active acute GVHD at TMA diagnosis was the only factor associated with worse OS (p = 0.009). Haematologica. In a pilot study of 6 children treated with eculizumab for multi-visceral TA-TMA, these investigator observed that patients after HSCT required higher doses or more frequent eculizumab infusions than currently recommended to achieve therapeutic drug levels and a clinical response in children with aHUS. Eculizumab in AQP4-IgG-positive relapsing neuromyelitis optica spectrum disorders: An open-label pilot study. Clin J Am Soc Nephrol. *Note: for Soliris switch therapy please refer to the package insert for appropriate switch dosing. Laboratory tests for paroxysmal nocturnal hemoglobinuria (PNH). An international consensus approach to the management of atypical hemolytic uremic syndrome in children. The primary outcomes were efficacy (the proportion of patients with restored ability to walk independently [functional grade less than or equal to 2] at week 4) in the eculizumab group and safety in the full analysis set. However, these researchers stated that this study was limited by absence of a control group and use of multiple therapeutic interventions in treatment groups; thus, prospective, controlled trials are needed. Ophthalmology. Scheiring J, Rosales A, Zimmerhackl LB. 2015;55(3):623-628. 2018;97(21):e10422. Nihon Rinsho Meneki Gakkai Kaishi. However, because this was a small study (n = 23 for the eculizumab-treated group) without statistical comparison with the placebo group, the safety and efficacy of eculizumab needs to be investigated in larger RCTs. Schrier SL. Pediatr Transplant. UpToDate [online serial]. Soliris eculizumab aHUS 300 mg vials 25682-0001-01 4 vials/120 ml MG/NMOSD 300 mg vials 25682-0001-01 4 vials/120ml PNH 300 mg vials 25682-0001-01 3 vials/90ml . The authors concluded that successful use of eculizumab in this case suggested that complement inhibition may be an effective treatment strategy for severe preeclampsia/HELLP syndrome. 2009;23(2):333-346. However, both patients died because of massive hemolysis after 85 and 106 days of intensive treatment, respectively. There were no maternal deaths and three fetal deaths (4%). Diagnosis and management of paroxysmal nocturnal hemoglobinuria. Chanchlani and colleagues (2017) stated that there had been recent developments in the understanding of the pathogenesis of membrano-proliferative glomerulo-nephritis (MPGN) supporting a prominent role for the complement alternative pathway (AP). A Cochrane review (Marti-Carvajal et al, 2014) assessed the clinical benefits and harms of eculizumab for treating patients with paroxysmal nocturnal hemoglobinuria (PNH). van Doorn (2009) noted that epidemiological studies have shown that the incidence of Guillain-Barre syndrome (GBS) remains stable at about 2/100,000 per year; but that there have been changes in hospitalization use, likely due to the widespread availability of intravenous immunoglobulin (IVIG). More recently, a number of novel therapies have been investigated in MMN, including interferon-β1a, rituximab, and eculizumab. This study was a 24-week, placebo-controlled study carried out in 13 hospitals in Japan. Pediatr Nephrol. Blocking of these cytokines by mAbs can be also a successful therapy for patients with SLE. Handy tools and resources for members ... Download guidelines that outline generally accepted minimum standards of care. Long-term efficacy and safety of eculizumab in Japanese patients with PNH: AEGIS trial. A rain sensor can also be connected. Molina has established Molina Clinical Policy that function as one of the sets of guidelines for coverage decisions or determinations. Here you will find information for assessing coverage options, guidelines for clinical utilization management, practice policies, the provider manual and support for delivering benefits to our members. These researchers presented the case of a 58-year old woman with treated human immunodeficiency virus and a normal Hb electrophoresis; she developed hyperhemolysis in the setting of a delayed hemolytic transfusion reaction (DHTR). McCaughan JA, O'Rourke DM, Courtney AE. The 4th study comparing pulsed oral dexamethasone with daily oral prednisolone and found that the dexamethasone regime had a shorter median time to relapse but fewer side effects. These researchers discussed recent advances in NMO diagnosis and treatment and the differential diagnosis in patients presenting with LETM. N Engl J Med. Burwick RM, Feinberg BB. Billing Code/Availability Information HCPCS code: J1300 – Injection, eculizumab, 10 mg; 1 billable unit = 10 mg NDC: Soliris 300 mg/30 mL single-use vials for injection: 25682-0001-xx VII. Brodsky RA, Young NS, Antonioli E, et al. Advice No: 0509 - April 2009. In 54% of pregnancies that progressed past the first trimester, the dose or the frequency of use of eculizumab had to be increased. While the kidney is most commonly affected, TA-TMA involving organs such as the lung, bowel, heart, and brain is now known to have specific clinical presentations. 600 mg weekly x 2. Parker CH, Kar S, Kirkpatrick P. Eculizumab. antibodies present in the recipient destroy donor cells. U.S. Food and Drug Administration (FDA). In the recent years, conceptual advances and the introduction of new therapies are yielding improvements in the management of this disease; clinical studies have been undertaken with selected monoclonal antibodies (mAbs) in the treatment of SLE. Cochrane Database Syst Rev. The kidneys in paroxysmal nocturnal hemoglobinuria. Furthermore, the intervention seems to reduce fatigue and withdrawals for any reason. Within 4 weeks of renal transplantation, deteriorating graft function and increasing proteinuria were evident. The authors concluded that hyperhemolysis is not restricted to patients with hemoglobinopathies. Atypical HUS (aHUS) defines non Shiga-toxin-HUS and even if some authors include secondary aHUS due to Streptococcus pneumoniae or other causes, aHUS designates a primary disease due to a disorder in complement alternative pathway regulation. Although nearly all patients reported at least one adverse event, discontinuation from treatment due to a nonfatal adverse event was seen in only five patients over the entire period of study. The placebo group was infused with saline. Medicine (Baltimore). The authors noted that more information is needed to clarify the effectiveness and role of eculizumab in dense deposit disease but the response in this patient was encouraging. N Engl J Med. 2010;15(1):8-10. Alexion Pharmaceuticals, Inc. A randomized controlled trial of eculizumab in AQP4 antibody-positive participants with NMO (PREVENT study). • Maintenance phase: 900 mg of Soliris administered via a 25 – 45 minute (35 minutes ± 10 minutes) For the efficacy end-point, these researchers pre-defined a response rate threshold of the lower 90 % CI boundary exceeding 50 %. 2000;70: 327-34. Kim and colleagues (2015) noted that transplant-associated thrombotic micro-angiopathy (TA-TMA) is a multi-factorial disorder, which occurs as a result of treatment-related endothelial injury and underlying disease process after hematopoietic stem cell transplantation (HSCT). Six studies compared an immunosuppressant or immunomodulator with placebo control, and 4 studies compared 2 immunosuppressant regimes with each other. Kim SS, Patel M, Yum K, Keyzner A. Hematopoietic stem cell transplant-associated thrombotic microangiopathy: Review of pharmacologic treatment options. Low-molecular-weight heparin was used in 88% of the pregnancies. These investigators retrospectively analyzed 12 patients who received eculizumab between 2010 and 2013 for severe post-HSCT TMA. Treatment with eculizumab resulted in a rapid and dramatic improvement of the graft function in all 3 patients and in improvement of the TMA lesions within the graft. Scottish Medicines Consortium. Birmingham, UK: NHSC; 2006. Long-term effect of the complement inhibitor eculizumab on kidney function in patients with paroxysmal nocturnal hemoglobinuria. Description of Services . One recent study suggested that combination therapy with plasmapheresis, high-dose IVIG and rituximab was more effective treatment for AMR than high-dose IVIG alone, but the role of anti-CD20 antibody is still unclear. 14th International Congress on Antiphospholipid Antibodies: Task force report on antiphospholipid syndrome treatment trends. Peripheral blood absolute neutrophil count (ANC) less than 500 per microL (<0.5 à 10â¹/L). These medical […] Ten hemorrhagic events and 2 thrombotic events were documented; both thrombotic events occurred during the postpartum period. Motamed-Gorji N, Matin N, Tabatabaie O, et al. 2017;23(2):175-180. Kavanagh D, Goodship TH. Eculizumab (Soliris) for paroxysmal nocturnal haemoglobinuria. Ottawa, ON: CADTH; February 19, 2010. Two authors independently selected papers, extracted data and assessed risk of bias in included studies. Eur J Haematol. They stated that this case showed that eculizumab could improve patientsâ CNS manifestations, although its long-term effectiveness and combined use with treprostinil require additional clinical examination. Today's understanding of the haemolytic uraemic syndrome. Defibrotide is an investigational agent in the United States; therefore, it is not readily available for use. The authors concluded that early initiation of eculizumab therapy in patients with progressive IgA nephropathy may have a beneficial effect by blocking complement-mediated renal inflammation. N Engl J Med. Pro-inflammatory cytokines such as tumor necrosis factor-alpha and interleukin-6 play an important role in propagating the inflammatory process responsible for tissue damage. Eculizumab (Soliris). Stegall MD, Gloor JM. Initial Phase. No therapeutic interventions have consistently improved outcomes for patients with primary or recurrent disease. At a median follow-up of 52 weeks (range of 3 to 113), the survivors (92 %) were doing well. The search strategy was applied to Ovid Medline, Embase, In-Process and Other Non-Indexed Citation, Cochrane Central Register of Controlled Trials and Scopus from 2006 to present. 2007;6:515-516. MAP has the following 2 variants: cutaneous MAP is manifested in the skin alone, whereas systemic MAP affects the gastro-intestinal (GI) tract, CNS, lungs, and other internal organs. Moreover, they stated that clinical trials assessing effectiveness of different complement blockers on the background of the individual complement profile are needed. The authors concluded that these findings may support the use of eculizumab in severe STEC-HUS patients, especially those presenting severe neurological symptoms. Alexion Pharma UK Ltd. Examination of sequential biopsies during eculizumab therapy showed that TMA lesions, C4d and apoptotic markers were rapidly cleared, but the C5b-9 deposits persisted for several months as a footprint of the TMA. View Information about Group Insurance Plans Identifies the procedures and services which require pre-authorization. New promising treatments are currently under evaluation in clinical trials, such as belimumab and eculizumab. Guidelines for the diagnosis and monitoring of paroxysmal nocturnal hemoglobinuria and related disorders by flow cytometry. This was the first reported case of recurrent dense deposit disease being managed with eculizumab. 2013;34(2):201-203. Jaretzki A, Barohn RJ, Ernstoff RM et al. Pregnancy was prolonged 17 days, likely resulting in a reduction of neonatal morbidity with its associated short- and long-term health care costs. 2019;23(1):65-75. Utilization Management Policy on Site of Care for Specialty Drug Infusions, CPB 0634 - Non-myeloablative Bone Marrow/Peripheral Stem Cell Transplantation (Mini-Allograft / Reduced Intensity Conditioning Transplant). Ochsner J. San Francisco, CA: Epocrates; 2019. It was sponsored by a pharmaceutical company. Eculizumab is also being examined in the treatment of various disorders/syndromes including antibody-mediated rejection, Guillain-Barre syndrome, and systemic lupus erythematosus. The complement pathway is thought to be active in this process. Eculizumab in STEC-HUS: Need for a proper randomized controlled trial. The assessment noted that, "although some private insurance companies in the United States give coverage to certain patients, most health systems in different countries do not cover it due to its high costs and because it provides marginal benefits when compared with standard care for PNH.". To achieve and to sustain therapeutic eculizumab level greater than 99 µg/ml, CH50 should be less than 10 % of the lower limit of normal, corresponding to 0 to 3 CAE if using a standard enzyme immunoassay or 0 to 15 CH50 units if the Diamedix hemolytic assay is used. Pediatr Nephrol. UnitedHealthcare reserves the right, in its sole discretion, to modify its Policies and Guidelines as necessary. 2012;6:195-208. Blood. In January last year, the National Institute for Health and Care Excellence approved use of Alexion's Soliris (eculizumab), the only known effective treatment for this condition, by the National Health Service. Immunosuppressants were associated with significant side effects. After 12 months, 2 subjects showed significantly reduced serum creatinine, 1 subject achieved marked reduction in proteinuria, and 1 subject had stable laboratory parameters but histopathologic improvements. The study population was 267 consecutive patients with donor-specific antibody undergoing desensitization. Diaz-Manera J, Rojas Garcia R, Illa I. Patients with C4d deposition in the renal arterioles and without documented complement gene abnormalities or detectable CFH autoantibodies also have a favorable response to eculizumab. 2004;350(6):552-559.
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