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Clinical, Journal: Bone Marrow of Contention: A Rare Case of Recurrent Acute Hepatitis. (Pubmed Central) - Apr 29, 2021 Treatment of PNH with the complement inhibitor eculizumab (Soliris) prevented both recurrences of episodes of intravascular hemolysis and liver enzyme alteration. This is the first known published case of PNH-associated hepatitis.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, Vaxzevria (ChAdOx1-S recombinant) / Oxford Biomedica, Emergent Biosolutions, Jenner Institute, Vaccitech, AstraZeneca, University of Oxford
Journal: Prothrombotic immune thrombocytopenia after COVID-19 vaccine. (Pubmed Central) - Apr 29, 2021 It is characterized by thrombocytopenia and anti-PF4 antibodies binding to platelets in AZD1222-dependent manner. Initial clinical experience suggests a risk of unusual and severe thromboembolic events.
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Review, Journal: Monoclonal Antibody-Based Therapies for Myasthenia Gravis. (Pubmed Central) - Apr 28, 2021 Thus far, only eculizumab, an antibody against C5, has reached the clinic. We review the present status of monoclonal antibody-based treatments for MG that have entered human testing and offer the promise to transform treatment of MG.
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Review, Journal: Thrombotic Microangiopathy After Kidney Transplantation: An Underdiagnosed and Potentially Reversible Entity. (Pubmed Central) - Apr 27, 2021 Plasma exchange typically resolves hematologic abnormalities but does not improve renal function. Complement blockade with eculizumab has been shown to be an effective therapy in post-transplant thrombotic microangiopathy, but it is necessary to define which patients can benefit from this therapy and when and how eculizumab should be used.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, Ultomiris IV (ravulizumab IV) / Alexion Pharma
Review, Journal: Interventions for atypical haemolytic uraemic syndrome. (Pubmed Central) - Apr 22, 2021 When compared with historical data, terminal complement inhibition appears to offer favourable outcomes in patients with aHUS, based upon very low-quality evidence drawn from five single-arm studies. It is unlikely that an RCT will be conducted in aHUS and therefore careful consideration of future single-arm data as well as longer term follow-up data will be required to better understand treatment duration, adverse outcomes and risk of disease recurrence associated with terminal complement inhibition.
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[VIRTUAL] HOMOZYGOUS MUTATION IN COMPLEMENT C3 GENE ASSOCIATED WITH FAMILIAL HEMOLYTIC UREMIC SYNDROME: CASE SERIES () - Apr 17, 2021 - Abstract #ISNWCN2021ISN-WCN_990; She was transferred to our center and immediately started on induction doses of anti-C5 monoclonal antibody (Eculizumab)...Conclusions Homozygous C3 gene mutation resulting in atypical hemolytic syndrome can have an early and devastating course, but fortunately its response to Anti-C monoclonal antibody is excellent. Larger sample size study with prospective design will definitely needed for better characterization of the course of the disease as well as potential complication of prolonged Complement C5 inhibition.
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[VIRTUAL] ECULIZUMAB IN ATYPICAL HEMOLYTIC UREMIC SYNDROME RELATED TO LUNG TRANSPLANT () - Apr 17, 2021 - Abstract #ISNWCN2021ISN-WCN_708; Induction and maintenance immunosuppressive therapy was based tacrolimus, mycophenolate and prednisone in all cases...Conversion from mycophenolate to everolimus had been carried out in 3 of the 4 patients...Conclusions aHUS is a critical complication in lung transplantation closely related to the immunosuppressive scheme, that can lead to end-stage kidney disease. Treatment with eculizumab appears promising.
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[VIRTUAL] COMPLEMENT IN THROMBOTIC MICROANGIOPATHIES () - Apr 14, 2021 - Abstract #ASPHO2021ASPHO_410; Patients with mild to moderate CM-TMA (with C5a, C5b-9 up to 1.2 times upper limit of normal (ULN) in cases 1-2) may improve with effective treatment of underlying disease. However, in more severe presentations (C5a, C5b-9 two to five x ULN in cases 3-4), early use of eculizumab may contribute to improved renal and other organ recovery, and patient outcomes.
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[VIRTUAL] Clinical and Economic Consequences of the Breakthrough Hemolysis () - Apr 12, 2021 - Abstract #ISPOR2021ISPOR_1151; Patients with BHE increase the cost for the target population to 3.4 bln rubles due to the need for therapy correction. The most frequently used approach in Russia for the correction of insufficiently effective eculizumab therapy by reducing the interval between injections is a more resource-intensive technique than the tactics of increasing the dosage.
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[VIRTUAL] Cost-of-Illness for Adults with Generalized Myasthenia Gravis in the US () - Apr 12, 2021 - Abstract #ISPOR2021ISPOR_1092; The 12-month period following a crisis event was also associated with high costs. CONCLUSIONS Beyond costs associated directly with crisis, use of immune-globulins, newly diagnosed status and the year prior to and following a crisis are a significant contributor to direct costs in adults with gMG in the US.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, Ultomiris IV (ravulizumab IV) / Alexion Pharma
[VIRTUAL] CLINICAL BURDEN OF PAROXYSMAL NOCTURNAL HEMOGLOBINURIA AMONG PATIENTS RECEIVING C5 INHIBITORS IN THE UNITED STATES () - Apr 11, 2021 - Abstract #AMCP2021AMCP_70; Treatment with the C5 inhibitors, eculizumab (ECU) or ravulizumab (RAV), can reduce intravascular hemolysis (IVH) and lead to improvements in morbidity and mortality. Findings from this burden of illness survey demonstrate that a majority of PNH patients remain anemic and report fatigue despite treatment with C5 inhibitors (ECU/RAV) for ≥3 months highlighting a significant unmet medical need.
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[VIRTUAL] Gastrointestinal Kohlmeier-Degos Disease: What a Dermatologist Needs to Know () - Apr 10, 2021 - Abstract #AADVMX2021AAD_VMX_353; An effective treatment regimen for gastrointestinal K-D, eculizumab and treprostinil, now exists [4]. Since skin lesions precede systemic disease and many patients initially present to and are diagnosed with cutaneous K-D by dermatologists, dermatologists have a critical role in promoting screening for systemic disease and in patient education.
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Review, Journal: Complement Inhibitor Therapy for Myasthenia Gravis. (Pubmed Central) - Apr 7, 2021 Second- and third-generation complement inhibitors are in development and approaching pivotal efficacy evaluations. This review will summarize the history and present the state of knowledge of this new therapeutic modality.
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