- |||||||||| Soliris (eculizumab) / Alexion Pharma
Biomarker, Journal: Neurology (Pubmed Central) - Jun 26, 2019 The use of levodopa-carbidopa intestinal gel and a subcutaneous apomorphine infusion allow a reduction of motor fluctuations in Parkinson's disease. Eculizumab appears as an alternative treatment in severe forms of myasthenia gravis.
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Journal: Antibody Inhibition of Properdin Prevents Complement-Mediated Intravascular and Extravascular Hemolysis. (Pubmed Central) - Jun 24, 2019 In a murine EVH model, complement-susceptible erythrocytes were completely eliminated within 3 d in control mAb-treated hP-Tg/P mice, whereas such cells were protected and persisted in hP-Tg/P mice treated with an anti-hP mAb. Collectively, these data suggest that anti-P mAbs can inhibit both IVH and EVH mediated by complement and may offer improved efficacy over eculizumab, the current standard therapy for PNH.
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Clinical, Journal: Fatal meningococcemia due to Neisseria meningitidis serogroup Y in a vaccinated child receiving eculizumab. (Pubmed Central) - Jun 20, 2019 Herein we describe an 11-year-old boy with atypical hemolytic uremic syndrome treated with eculizumab who developed fatal meningococcemia due to Neisseria meningitidis serogroup Y 16 months after receiving two doses of meningococcal conjugate vaccine (MenACWY-D) while on oral penicillin prophylaxis. Clinicians need to maintain a high index of suspicion for invasive meningococcal disease in patients taking eculizumab treatment, regardless of meningococcal vaccination or antimicrobial prophylaxis status.
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Journal: Medusa's Head: The Complement System in Traumatic Brain and Spinal Cord Injury. (Pubmed Central) - Jun 17, 2019 The role of upstream classical, alternative or extrinsic complement activation cascades remains unclear. Although several issues remain to be investigated, current evidence support the investigation of a number of complement-targeting agents targeting C3 or C5, such as eculizumab, for repurposing in TBI and SCI treatment.
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Journal: Disseminated cryptococcosis associated with administration of eculizumab. (Pubmed Central) - Jun 15, 2019 In our centre, first-line therapy is PE; however, patients are instantly switched to complement inhibitory therapy in case of treatment failure or intolerance. A 23-year-old man developed fatal disseminated cryptococcosis after treatment with eculizumab.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, Avastin (bevacizumab) / Roche
Journal: Bevacizumab-induced atypical hemolytic uremic syndrome and treatment with eculizumab. (Pubmed Central) - Jun 9, 2019 Eculizumab is a terminal complement inhibitor used in the treatment of atypical hemolytic uremic syndrome. Herein, we present three cases of bevacizumab-induced atypical hemolytic syndrome treated successfully with eculizumab.
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Journal: Deleterious Impact of a Novel CFH Splice Site Variant in Atypical Hemolytic Uremic Syndrome. (Pubmed Central) - Jun 4, 2019 Treatment with steroids, plasmapheresis, and the complement inhibitor eculizumab led to complete hematological and clinical remission after several months and stable renal function up to 6 years later. In conclusion, genetic investigation for pathogenic variants and evaluation of their functional impact, in particular in the case of splice site variants, is clinically relevant and enables not only better molecular understanding but helps to guide therapy with complement inhibitors.
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Clinical, Journal: Eculizumab in STEC-HUS: need for a proper randomized controlled trial. (Pubmed Central) - Jun 4, 2019 In a subgroup of patients, however, rapid neurological improvement was described. In the post-hoc-defined group of patients with favorable outcome, there was a trend towards more sustained complement inhibition, although this finding was not significant compared to patients with an unfavorable outcome.Because multiple interventions were used and the study did not include any control group, future controlled studies are urgently needed to resolve the debate as to whether eculizumab can be an effective treatment for both prevention and treatment of complications in STEC-HUS.
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Clinical, Journal: Clinical and genetic predictors of atypical hemolytic uremic syndrome phenotype and outcome. (Pubmed Central) - May 29, 2019 ...Here we characterize end-stage renal disease (ESRD)-free survival, the rate of thrombotic microangiopathy, organ involvement and the genetic background of 851 patients in the registry, prior to eculizumab treatment...Additionally extrarenal organ manifestations occur in 19%-38% of patients within six months of initial disease presentation (dependent on organ). Thus, our real-world results provide novel insights regarding phenotypic variables and genotypes on the clinical manifestation and progression of aHUS.
- |||||||||| Campath (alemtuzumab) / Sanofi, Soliris (eculizumab) / Alexion Pharma, Rituxan (rituximab) / Roche, Biogen
Journal: Current and emerging treatment options for autoimmune hemolytic anemia. (Pubmed Central) - May 22, 2019 The latter may give hints for targeted therapies (either B or T cell directed) and for new immunomodulatory drugs. Future studies on the genomic landscape in AIHA will further help in designing the best choice, sequence and/or combination of targeted therapies.
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Review, Journal: How we treat paroxysmal nocturnal hemoglobinuria: a consensus statement of the Canadian PNH Network and review of the national registry. (Pubmed Central) - May 22, 2019 ...Introduction of the terminal complement inhibitor eculizumab drastically improved outcomes in PNH patients; however, despite this improvement, there remain several challenges faced by PNH patients and physicians who care for them...Herein we address some of the common diagnostic and therapeutic challenges faced by PNH physicians and give our recommendations. Gaps in knowledge are also addressed and, where appropriate, consensus opinion is provided.
- |||||||||| Soliris (eculizumab) / AstraZeneca, Ultomiris IV (ravulizumab-cwvz IV) / AstraZeneca
Trial completion date: ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Na (clinicaltrials.gov) - May 16, 2019 P3, N=246, Active, not recruiting, Trial completion date: Dec 2019 --> Oct 2020 | Trial primary completion date: Jul 2019 --> Dec 2019 Trial completion date: Feb 2020 --> Jan 2023
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Journal: Steroid Responsive Atypical Hemolytic Uremic Syndrome Triggered by Influenza B Infection. (Pubmed Central) - May 15, 2019 Current accepted therapies for aHUS include plasma exchange and eculizumab. We describe a unique patient with aHUS with a rare membrane cofactor protein mutation triggered by influenza B infection, who achieved complete remission with treatment with high-dose corticosteroids after failure of plasmapheresis.
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Clinical, Journal: An Atypical Case of Atypical Hemolytic Uremic Syndrome. (Pubmed Central) - May 15, 2019 Atypical hemolytic uremic syndrome was suspected and eculizumab was administered resulting in rapid improvement. Genetic analysis revealed a mutation in the gene encoding complement factor H and atypical hemolytic uremic syndrome was confirmed.
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Journal: Paroxysmal nocturnal hemoglobinuria. (Pubmed Central) - May 11, 2019 Patients with severe hemo-lysis and/or thrombotic complications are indicated for administration of eculizumab - a monoclonal antibody against C5 part of complement. Key words: diagnosis - eculizumab - immunosuppression - paroxysmal nocturnal hemoglobinuria - pathogenesis - transplantation - treatment.
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Clinical, Journal: Meningococcal B Vaccine Immunogenicity in Children With Defects in Complement and Splenic Function. (Pubmed Central) - May 9, 2019 4CMenB administration is similarly immunogenic in healthy children and those with asplenia or splenic dysfunction. The significance of the trend to lower responses of SBA titers in complement-deficient children (especially those with terminal chain complement deficiency or those on eculizumab therapy) must be determined by ongoing surveillance for vaccine failures.
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Journal: Eculizumab is Efficacious But Often Not Accessible. (Pubmed Central) - May 8, 2019 The significance of the trend to lower responses of SBA titers in complement-deficient children (especially those with terminal chain complement deficiency or those on eculizumab therapy) must be determined by ongoing surveillance for vaccine failures. No abstract available
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