- |||||||||| Soliris (eculizumab) / Alexion Pharma, efgartigimod IV (ARGX-113 IV) / argenx, Broteio, Rituxan (rituximab) / Roche, Biogen
Review, Journal: Immunotherapy in myasthenia gravis in the era of biologics. (Pubmed Central) - Jan 30, 2020 Belimumab, an antibody against the B cell trophic factor BAFF, was ineffective in phase III trials, and efgartigimod, which depletes antibodies, was effective in a phase II study...Checkpoint inhibitors can trigger MG in some patients, necessitating early intervention. Increased availability of new biologics provides targeted immunotherapies and the opportunities to develop more specific therapies.
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Review, Journal: Compendium of current complement therapeutics. (Pubmed Central) - Jan 22, 2020 The driving role of complement in a single disease, paroxysmal nocturnal hemoglobinuria (PNH), provoked the development and eventual FDA (US Food and Drug Administration) approval of eculizumab (Soliris™), an anti-C5 antibody, for therapy...In this article, we do not set out to provide a text-heavy review of complement therapeutics but instead will simply highlight the targets, modalities and current status of the plethora of drugs approved or in clinical development. With such a fast-moving drug development landscape, such a compendium will inevitably become out-dated; however, we provide a snapshot of the current field and illustrate the increased choice that clinicians might enjoy in the future in selecting the best drug for their application, decisions based not only on efficacy but also cost, mechanistic target, modality and route of delivery.
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Survey to assess variability in management of myasthenic crisis among neurologists () - Jan 21, 2020 - Abstract #AAN2020AAN_2424; Our survey indicates that there is considerable variability in the acute treatment of myasthenic crisis, and escalated therapy continues into the post-discharge recovery period. We intend to collate data from a much larger community of clinicians and have this available for presentation at the annual meeting.
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Effect of Complement Inhibition on Lymphocytes in Treatment Refractory Myasthenia Gravis Patients () - Jan 21, 2020 - Abstract #AAN2020AAN_2373; Immunologically, eculizumab increased the frequency of selected CD8 T cells subsets and unbiased cluster analysis revealed that this increase stems from a rise in naïve and terminal effector CD8 T cells. Ongoing analyses will focus on less common immune subsets and qualitative immune signature changes in patients with a reduced clinical response to complement inhibition.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, Rituxan (rituximab) / Roche, Biogen, Zenyaku Kogyo
Benefit of Eculizumab for a Broad Range of Patients with Aquaporin-4 Antibody-positive Neuromyelitis Optica Spectrum Disorder: Findings from the Phase 3 PREVENT Study () - Jan 21, 2020 - Abstract #AAN2020AAN_1565; P3 Design/ In PREVENT, patients received eculizumab (maintenance dose, 1200 mg/2 weeks) or placebo, with stable-dose concomitant IST (except rituximab and mitoxantrone) permitted. The data from this post hoc subgroup analysis suggest that eculizumab reduced relapse risk in PREVENT compared with placebo, regardless of time since NMOSD diagnosis, relapse history, disability burden or prior IST use.
- |||||||||| Actemra IV (tocilizumab) / Roche, JW Pharma, Darzalex IV (daratumumab) / J&J, Soliris (eculizumab) / Alexion Pharma
Daratumumab as First Line Therapy in a POEMS Syndrome Patient Demonstrating Atypical Features () - Jan 21, 2020 - Abstract #AAN2020AAN_1106; Our use of daratumumab contrasts its success as salvage-therapy in a prior case. In addition, our patient developed liver dysfunction and abnormal blood counts either due to progression of POEMS or daratumumab usage, neither of which are reported in literature.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Biomarker, Phase classification, Enrollment change, Trial termination: Evaluation of Potential Predictors of Disease Progression in Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Including Genetics, Biomarkers, and Treatment (clinicaltrials.gov) - Jan 2, 2020 P=N/A, N=67, Terminated, No abstract available Phase classification: P4 --> P=N/A | N=330 --> 67 | Active, not recruiting --> Terminated; Terminated for administrative reasons before enrollment was complete.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Review, Journal, HEOR: Quality of life in refractory generalized myasthenia gravis: A rapid review of the literature. (Pubmed Central) - Jan 1, 2020 This outcome is of great importance for the choice of therapeutic options in patients with refractory GMG. Eculizumab generates improvements in the perception of patients' quality of life compared to placebo, making it a relevant therapeutic option in the management of refractory GMG.
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Clinical, Journal: Eculizumab Safety: Five-Year Experience From the Global Atypical Hemolytic Uremic Syndrome Registry. (Pubmed Central) - Jan 1, 2020 Deaths were more frequent in adult (4.7% and 9.9% of ever- and never-treated patients) compared with pediatric patients (1.8% of ever-treated patients; no deaths in never-treated patients).Three meningococcal infections were reported in ever-treated patients; 1 infection led to a fatal outcome. In this large observational dataset covering 5 years of registry enrollment, no new safety concerns were identified for adult or pediatric eculizumab-treated patients with aHUS, confirming a positive benefit-risk profile in a real-world setting.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Clinical, Journal: Use of Eculizumab During Pregnancy in Kidney Transplant Recipients With Atypical HUS. (Pubmed Central) - Jan 1, 2020 In this large observational dataset covering 5 years of registry enrollment, no new safety concerns were identified for adult or pediatric eculizumab-treated patients with aHUS, confirming a positive benefit-risk profile in a real-world setting. No abstract available
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Clinical, Journal: Kidney Transplantation in C3 Glomerulopathy: A Case Series. (Pubmed Central) - Dec 28, 2019 In a case series of C3G transplant recipients, the proportion of disease recurrence was high in both C3GN and DDD, although graft loss appeared to occur more frequently in DDD. In a small subset of study patients, eculizumab therapy was not consistently followed by salutary outcomes.
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Clinical, Journal, Adverse events: Adverse outcomes in obstetric-atypical haemolytic uraemic syndrome: a case series analysis. (Pubmed Central) - Dec 26, 2019 Presentation of obstetric-aHUS is preceded by various precipitating factors, suggesting pregnancy complications, and not the pregnancy per se, often induce aHUS in women with a genetic predisposition to its development. A delay in the correct diagnosis and initiation of the most effective treatment can have serious consequences, reinforcing the need to raise awareness of obstetric-aHUS.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, Rituxan (rituximab) / Roche, Biogen
Journal: Rituximab to Abbreviate Plasma Exchange in Anti-CFH (Complement Factor H) Antibody Mediated Atypical HUS. (Pubmed Central) - Dec 23, 2019 Rituximab might be a useful alternative in inducing haematological remission in children with poor or no response to plasma therapy. This abbreviates the duration of plasma exchange, which not only avoids complications due to prolonged plasma therapy but also helps reducing the cost of therapy.
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Complement Blockade for TA-TMA: Lessons Learned from Large Pediatric Cohort Treated with Eculizumab (World Center Marriott - Palms & Cypress Foyers) - Dec 18, 2019 - Abstract #TCTASTCTCIBMTR2020TCT_769; The best CystC GFR recovery in survivors was a median 20% lower (IQR 7.3-40.3%) than their pre-HSCT CystC GFR. Conclusion Complement blockade with eculizumab is an effective therapeutic strategy for high risk TA-TMA, but some patients with severe disease lack a complete response, prompting us to propose early intervention strategies and search for additional targetable endothelial injury pathways.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Review, Journal: Flow Cytometric Diagnosis of Paroxysmal Nocturnal Hemoglobinuria: Pearls and Pitfalls - A Critical Review Article. (Pubmed Central) - Dec 10, 2019 The recent advent of the therapeutic monoclonal antibody Eculizumab has dramatically improved both the quality of life and the life expectancy of the affected patients, further increasing the importance of an accurate FCM detection and monitoring of the clones. The technical features of the FCM diagnostic workup and the many critical aspects of the analytical process are discussed here.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Review, Journal: CHAPLE syndrome uncovers the primary role of complement in a familial form of Waldmann's disease. (Pubmed Central) - Dec 8, 2019 The observation that complement system is overly active in CHAPLE disease introduced a novel concept into the management of PLE; anti-complement therapy. While CD55 deficiency constitutes a treatable subgroup in the larger pool of patients with isolated PIL, the etiology remains to be identified in the remaining patients with intact CD55.
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Thinking Beyond HLH: Clinical Features of Patients with Concurrent Presentation of Hemophagocytic Lymphohistiocytosis and Thrombotic Microangiopathy (World Center Marriott - Palms: Sabal) - Dec 8, 2019 - Abstract #TCTASTCTCIBMTR2020TCT_586; Conclusion Our observations suggest co-activation of both interferon and complement pathways as a potential culprit in the evolution of thrombotic microangiopathy in patients with inflammatory disorders like HLH and may offer novel therapeutic approaches for these critically ill patients. TMA should be considered in children with HLH and multi-organ failure, as early institution of a brief course of complement blocking therapy in addition to HLH targeted therapy may improve clinical outcomes in these patients (Figure 3).
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Clinical, Journal: Eculizumab therapy in paroxysmal nocturnal haemoglobinuria patient undergoing aortic valve surgery. (Pubmed Central) - Dec 6, 2019 We report the successful management of a 69-year-old man undergoing aortic valve replacement with standard heparin-protamine protocol by using eculizumab, a monoclonal antibody of complement factor C5. The surgery was performed without triggering a haemolytic crisis, and the patient was discharged from the hospital without major complications.
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