- |||||||||| Soliris (eculizumab) / Alexion Pharma
[VIRTUAL] Eculizumab Use in Scleroderma Renal Crisis with Thrombotic Microangiopathy (On-Demand) - Oct 11, 2020 - Abstract #KIDNEYWEEK2020KIDNEY_WEEK_2314;
In addition, an increased FBb/FB ratio has been reported in a SRC with TMA, suggestive of subsequent recruitment of the alternative pathway through the C3b feedback cycle leading to further endothelial injury. These data support the potential role of complement blockade for the treatment of SRC with TMA.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
[VIRTUAL] Complement-Mediated Thrombotic Microangiopathy in a Patient with Anti-GBM Disease: A Case Report (On-Demand) - Oct 11, 2020 - Abstract #KIDNEYWEEK2020KIDNEY_WEEK_2312;
While hemolysis has been reported in the literature, this is the first report of complement-mediated TMA driving hemolysis in the setting of anti-GBM. Dysregulated activation of the alternative pathway in complement-mediated TMA and literature reports supporting a role for both classical and alternative pathways in anti-GBM disease suggests a possible link between the two diseases and a previously unrecognized role for complement dysregulation in cases of hemolysis observed in the setting of anti-GBM.
- |||||||||| bintrafusp alfa (M7824) / GSK, EMD Serono, Merck (MSD), Soliris (eculizumab) / Alexion Pharma
[VIRTUAL] Bintrafusp α-Associated Thrombotic Microangiopathy (On-Demand) - Oct 11, 2020 - Abstract #KIDNEYWEEK2020KIDNEY_WEEK_2307;
The patient received a dose of Soliris empirically, pending workup for atypical HUS...TMA was documented in 1 patient receiving Ipilimumab and was HD-dependent...Renal biopsy would expand our knowledge on the pathology of AKI post-ICPI treatment. We report the first case of TMA associated with the new bifunctional immunotherapy for solid cancers.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
[VIRTUAL] Atypical Hemolytic Uremic Syndrome Attributed to Complement Dysregulation in Setting of Metastatic Prostate Cancer Patient (On-Demand) - Oct 11, 2020 - Abstract #KIDNEYWEEK2020KIDNEY_WEEK_2306;
Patient last received chemotherapy with cabazitaxel five months prior...He was initiated on Eculizumab 900mg once weekly and began to show signs of renal recovery...He responded to eculuzimab, monoclonal antibody inhibiting activation of C5, with full recovery of his renal function. In cases of aHUS presenting in patients with malignancy, physicians should be aware that aHUS may still occur secondary to the primary hit of complement dysregulation and should consider treatments targeting this complement pathway.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
[VIRTUAL] An Unusual Case of Complement-Mediated Thrombotic Microangiopathy (On-Demand) - Oct 11, 2020 - Abstract #KIDNEYWEEK2020KIDNEY_WEEK_2304;
She endorsed not taking mesalamine and use of naproxen daily for two weeks...She received solumedrol 500mg/day IV x 3 and then prednisone 60mg/day...Prednisone, Eculizumab and PEX were continued...A genetic renal panel, which tested for CFH, CFI, CFI, MCP, and several other genes, confirmed she had a heterozygous deletion of a CFH related gene, CFHR1/CFHR3, as well as autoantibodies to CFH Discussion In our case, aHUS was likely predisposed by her heterozygous deletion of CFHR1/CFHR3 genes. What makes this case unusual is she did not have a homozygous deletion, but developed autoantibodies to CFH, to our knowledge has not been previously reported.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
[VIRTUAL] Development of Atypical Hemolytic Uremic Syndrome in a Patient with Complement 3 Glomerulonephritis (On-Demand) - Oct 11, 2020 - Abstract #KIDNEYWEEK2020KIDNEY_WEEK_2081;
Discussion In our growing understanding of alternative complement pathway, it is thought that dysregulation at fluid state is associated with C3GN, while solid state dysregulation is associated with widespread endothelial injury leading to aHUS. Our patient developed both pathologies, suggests further research is needed in understanding the details of complement system.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
[VIRTUAL] Drug-Induced Thrombotic Microangiopathy as a "Second-Hit" Phenomenon (On-Demand) - Oct 11, 2020 - Abstract #KIDNEYWEEK2020KIDNEY_WEEK_2039;
As such, he was diagnosed with presumed aHUS and treated with steroids, eculizumab, and plasmapheresis...However, in patients with persistent TMA and a suggestive family history, clinical suspicion for aHUS should be maintained. This distinction is important as drug discontinuation alone would be ineffective for aHUS whereas complement-blocking therapies could be potentially curative.
- |||||||||| Actemra IV (tocilizumab) / Roche, JW Pharma, Soliris (eculizumab) / Alexion Pharma
[VIRTUAL] Thrombotic Microangiopathy (TMA) in a Patient with COVID-19 (On-Demand) - Oct 11, 2020 - Abstract #KIDNEYWEEK2020KIDNEY_WEEK_1501;
Patient received anakinra and tocilizumab...Plasma exchange was not performed, but received a single dose of eculizumab on day 21...To the best of our knowledge, we report the first case of TMA associated with SARS-CoV-2 with presence of diffuse cortical necrosis and widespread microthrombi in kidney biopsy. It is not clear if the virus played a direct pathogenic role or unmasked a latent complement defect leading to widespread endothelial damage and micro thrombi
- |||||||||| Kyprolis (carfilzomib) / Amgen, Ono Pharma, Soliris (eculizumab) / Alexion Pharma
[VIRTUAL] Carfilzomib-Induced Thrombotic Microangiopathy Effectively Treated with Eculizumab (On-Demand) - Oct 11, 2020 - Abstract #KIDNEYWEEK2020KIDNEY_WEEK_838;
Given the effects of Carfilzomib on the alternate pathway, Eculizumab is a reasonable therapeutic option because it targets complement C5 and inhibits the assembly of the MAC.4,5,6,7. Since PIs are the mainstay of MM treatment, the nephrologist should be aware of the rare but serious consequences of PI- associated DITMA and should consider Eculizumab as a management strategy1,2,3.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
[VIRTUAL] Drug-Induced Thrombotic Microangiopathy from Trimethoprim-Sulfamethoxazole (On-Demand) - Oct 11, 2020 - Abstract #KIDNEYWEEK2020KIDNEY_WEEK_802;
Case Description A 62-year-old man with recent diagnosis of Sweet’s syndrome was started on high dose prednisone and TMP-SMX for prophylaxis...High suspicion of TTP requires urgent plasma exchange until ADAMTS-13 levels return, and if complement-mediated TMA is likely, the terminal-complement inhibitor eculizumab can be used...Trimethoprim-sulfamethoxazole is a rare cause of thrombotic microangiopathy, and the exact mechanism is not understood. Our case highlights the importance of considering TMP-SMX as a potential cause in patients presenting with TMA.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
[VIRTUAL] Catch 22: The Vicious Cycle of Malignant Hypertension and Worsening Renal Thrombotic Microangiopathy (On-Demand) - Oct 11, 2020 - Abstract #KIDNEYWEEK2020KIDNEY_WEEK_798;
The ‘domino effect’ created by this inflammatory cascade is difficult to reverse and may benefit from eculizumab therapy, whether they have a genetic predisposition or not. The detection of C5B-9 formation on the endothelial cells may reflect complement defects among patients with TMA and severe hypertension, which can be a valuable tool in sub-stratifying whether these patients may benefit from terminal complement inhibitor therapy.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, Rituxan (rituximab) / Biogen, Zenyaku Kogyo, Roche
[VIRTUAL] Infection Risk, Immunization Recommendations, and Antimicrobial Prophylaxis Needs when Treating Non-Malignant Hematologic Disorders - Wash Your Hands and What Else? - Live Q&A (Channel 18 (Virtual Meeting)) - Oct 11, 2020 - Abstract #ASH2020ASH_97;
Jolan Walter will discuss infection risk, immunization recommendations, and antimicrobial prophylaxis needs when treating patient with rituximab or eculizumab. She will address the infectious risks with these treatments, appropriate immunization strategies (vaccine choices, boosters, vaccine response monitoring), the management of the patient who needs rituximab who has positive hepatitis serologies and is at risk for viral hepatitis reactivation, the risk of infection with rituximab-induced neutropenia and whether GCSF has a benefit in this situation, and whether rituximab-associated hypogammaglobulinemia should be treated with IVIG.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Review, Journal: Update on neuromyelitis optica spectrum disorder. (Pubmed Central) - Oct 11, 2020
She will address the infectious risks with these treatments, appropriate immunization strategies (vaccine choices, boosters, vaccine response monitoring), the management of the patient who needs rituximab who has positive hepatitis serologies and is at risk for viral hepatitis reactivation, the risk of infection with rituximab-induced neutropenia and whether GCSF has a benefit in this situation, and whether rituximab-associated hypogammaglobulinemia should be treated with IVIG. Recent research in NMOSD has resulted in improved diagnosis and approved treatments.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, Rituxan (rituximab) / Biogen, Zenyaku Kogyo, Roche
[VIRTUAL] Internal Crowdsourcing To Achieve Cost-Savings During A Pandemic () - Oct 8, 2020 - Abstract #ASHP2020ASHP_433;
Empowering front-line staff with the tools to assess impact and feasibility, implement ideas, and track results is key to success. Future direction for this project includes increasing pharmacy technician participation through education on high-cost drug utilization and stepwise implementation through “Plan-Do-Check-Act” (PDCA) cycles with project owners.
- |||||||||| Actemra IV (tocilizumab) / Roche, JW Pharma, Soliris (eculizumab) / Alexion Pharma
Review, Journal: Current status of COVID-19 treatment: An opinion review. (Pubmed Central) - Oct 8, 2020
And (3) Are there specific clinical manifestations related to COVID-19 that can be treated with a customized and targeted therapy? By answering these questions, we wish to create a summary of current COVID-19 treatments and the anti-COVID-19 treatments proposed in the recent clinical trials developed in the last 3 mo, and to describe examples of clinical manifestations of the SARS-CoV-2 infection with a cause-related treatment.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, Rituxan (rituximab) / Biogen, Zenyaku Kogyo, Roche
Review, Journal: A Narrative Review on C3 Glomerulopathy: A Rare Renal Disease. (Pubmed Central) - Oct 8, 2020
Current treatment is based on corticosteroids and antiproliferative drugs (cyclophosphamide, mycophenolate mofetil), monoclonal antibodies (rituximab) or complement inhibitors (eculizumab). In some cases, it is suggested to include sessions of plasma exchange.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
[VIRTUAL] Review of Gastrointestinal Manifestations of Kohlmeier-Degos Disease () - Oct 8, 2020 - Abstract #ACRARHP2020ACR_ARHP_673;
All gastrointestinal cases were preceded by characteristic skin lesions and the presence or histology of these cutaneous lesions often aided diagnosis. All cases of bowel perforation were preceded by at least one gastrointestinal symptom.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Clinical, Review, Journal: Nephritic Factors: An Overview of Classification, Diagnostic Tools and Clinical Associations. (Pubmed Central) - Oct 3, 2020
This review will be focused in the description of the mechanism of action of the three known nephritic factors (C3NeF, C4NeF, and C5NeF), and their association with human diseases. Moreover, we present an overview regarding the diagnostic tools for its detection, and the main therapeutic approach for the patients with nephritic factors.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Journal: Semi-solid phase assay for the alternative complement pathway activity assessment (AP). (Pubmed Central) - Oct 3, 2020
AP has many advantages over the AH as it is more reliable for the Eculizumab monitoring and more practical with a purpose to be stored and transported for several weeks. AP is a portable and easy to use device both at the bedside and in the companion medical care.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, Rituxan (rituximab) / Biogen, Zenyaku Kogyo, Roche
Review, Journal, IO Biomarker: From Traditional to Targeted Immunotherapy in Myasthenia Gravis: Prospects for Research. (Pubmed Central) - Sep 30, 2020
Killing of B cells with Rituximab has been proposed and tested with positive results, particularly in patients with MuSK-associated MG...A differently targeted approach involved Eculizumab, a monoclonal antibody preventing the formation of C59b-induced MAC causing destruction of the neuromuscular junction...An even more recent approach is the interference with the neonatal Fc receptor leading to a rapid reduction of circulating IgGs and hence of specific autoantibodies, an approach suitable for both anti-acetylcholine- and MuSK-associated MG. The investigation of compounds that selectively target the immune system will stimulate the search for specific biomarkers of disease activity and response to treatment, setting the basis for personalized medicine in MG.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
[VIRTUAL] Alternatives to Apheresis: Emerging Therapies () - Sep 27, 2020 - Abstract #AABB2020AABB_103;
We will focus on the strengths and weaknesses of these approaches compared to TPE and speculate on the potential utility of combining these agents with TPE for the best care of patients in the future. Learning Objectives Explain the mechanism of FcRn inhibitors in reducing IgG levels and describe emerging data Indicate how IdeS enzymes work against IgG, and describe available clinical data Describe the mechanism of eculizumab and caplacizumab, and describe available evidence and indications
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Journal: Infections in the Era of Targeted Therapies: Mapping the Road Ahead. (Pubmed Central) - Sep 27, 2020
While these scenarios can potentially help us in terms of enhancing our predictive powers (higher vigilance, earlier detection, and risk mitigation), targeted therapies have also revealed unpredictable toxicities (i.e., natalizumab and progressive multifocal leukoencephalopathy)...The second step is to identify high risk patients (using risk calculators such as the RABBIT risk score; big data analyses; artificial intelligence). Finally, the most underutilized interventions to prevent severe infections in patients receiving targeted therapies across the spectrum of immune mediated inflammatory diseases are vaccinations.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Journal: Meningococcal sepsis without cerebrospinal fluid abnormalities under treatment with eculizumab (Pubmed Central) - Sep 26, 2020
While blood cultures were positive for Neisseria meningitidis, she showed no evidence for bacterial meningitis in the cerebrospinal fluid. This case shows that meningococcal sepsis without signs for bacterial meningitis despite repetitive vaccinations is possible in adults under eculizumab.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Biomarker, Clinical, Journal: Differential miRNA expression profile and proteome in plasma exosomes from patients with paroxysmal nocturnal hemoglobinuria. (Pubmed Central) - Sep 25, 2020
Treatment with eculizumab reduces intravascular hemolysis and thrombotic risk, but not in all cases...We found differential expression of miRNAs miR-148b-3p, miR-423-3p, miR29b-3p, miR15b-5p, let-7e-5p, miR126-3p, miR-125b-5p and miR-376c-3p as well as hemoglobin, haptoglobin, protein S and C4-binding protein in healthy controls vs PNH patients. Our results warrant further research and provide new information on the content of exosomes that could play a role in the hypercoagulable state in this disease.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, Ultomiris IV (ravulizumab IV) / Alexion Pharma
Review, Journal: Eculizumab and Beyond: The Past, Present, and Future of Complement Therapeutics. (Pubmed Central) - Sep 21, 2020
Several challenges still exist or will soon present themselves, including mitigation of infection risk, effective monitoring strategies, and how to choose between therapeutics when more than one is available. In this review, we will describe the lessons learned from the "eculizumab era," present many of the novel therapeutics currently or soon to be in trials, and highlight some of the challenges that will require attention as the field progresses.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Clinical, Journal: Atypical hemolytic uremic syndrome: a case report. (Pubmed Central) - Sep 21, 2020
Early diagnosis with prompt treatment will render a better outcome. The atypical hemolytic uremic syndrome needs to be considered in all patients with thrombotic microangiopathy.
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