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Journal: Neuromyelitis Optica Spectrum Disorders - Present Insights and Recent Developments (Pubmed Central) - Oct 22, 2021
The achievements of the last 15 years have essentially shaped the diagnostic methods and therapy of Neuromyelitis optica spectrum disorders (NMOSD): from discovery of aquaporin 4 antibodies and further development of diagnostic criteria the path has led to the approval of eculizumab and satralizumab as first disease modifying treatments in Europe. This article should give an overview on the present insights and future treatment options.
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[VIRTUAL] Eculizumab in the Treatment of Aquaporin-4 Seronegative Neuromyelitis Optica Spectrum Disorder: A Case Report () - Oct 21, 2021 - Abstract #ANA2021ANA_20;
She was treated with oral prednisone (initiated at 40 mg/day in 2014; the dose was halved in 2015 due to weight gain) and mycophenolate mofetil (MMF) 1 g twice daily (from June 2015), but between 2014 and 2019 experienced 4-5 relapses/year, requiring treatment with intravenous methylprednisolone, with added maintenance plasma exchange from 2018 onwards...Prednisone and MMF were discontinued mid-2018, and rituximab was prescribed from July 2018 (maintenance regimen two 1 g doses 2 weeks apart every 6 months) but discontinued in July 2019 owing to lack of significant improvement... Eculizumab shows promise as a treatment for AQP-4 IgGseronegative NMOSD and further studies are warranted.
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[VIRTUAL] A Case Report of Seropositive Neuromyelitis Optica Spectrum Disorder () - Oct 21, 2021 - Abstract #ANA2021ANA_1;
Patient started Rituximab, followed by four courses of plasma exchange and steroids...Patient’s AQP4 antibodies have remained negative while on Eculizumab...Relapses are common, with a quicker disability progression seen in comparison to MS. Recognizing and treating NMOSD early can slow progression of the disease and increase a patient’s quality of life.
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Journal: Consistent improvement with eculizumab across muscle groups in myasthenia gravis. (Pubmed Central) - Oct 20, 2021
P3
Recognizing and treating NMOSD early can slow progression of the disease and increase a patient’s quality of life. Eculizumab treatment elicits rapid and sustained improvements in muscle strength across ocular, bulbar, respiratory, and limb/gross motor muscle groups and in associated daily activities in patients with refractory anti-acetylcholine receptor antibody-positive generalized myasthenia gravis.
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[VIRTUAL] Recurrence of Scleroderma Renal Crisis After Kidney Transplantation () - Oct 17, 2021 - Abstract #KIDNEYWEEK2021KIDNEY_WEEK_3864;
Discussion SRC post KT is unusual but should be considered in the differential of AKI in the right clinical setting. Active dcSSc disease, use of steroids and calcineurin inhibitors may increase the risk of post transplant SRC
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[VIRTUAL] Complement Inhibition with a Short Course of Eculizumab for Refractory Vasculitis () - Oct 17, 2021 - Abstract #KIDNEYWEEK2021KIDNEY_WEEK_3803;
Conclusion The coadjuvant complement inhibition with eculizumab stabilized or increased renal function and decreased proteinuria significantly in refractory vasculitis. The short course of eculizumab seems to be highly effective in r-AGN, and also was associated with lower doses needed.
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[VIRTUAL] Atypical Haemolytic Uremic Syndrome in Lung Transplantation and Treatment with Eculizumab: Our Experience () - Oct 17, 2021 - Abstract #KIDNEYWEEK2021KIDNEY_WEEK_3796;
Induction and maintenance immunosuppressive therapy were based on tacrolimus, mycophenolate and prednisone...Previously of thrombotic microangiopathy, three patients were on treatment with everolimus instead of mycophenolate and two lung transplants have cytomegalovirus reactivation...Patients are at risk of end stage renal disease. Eculizumab treatment appears promising.
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[VIRTUAL] Utility of Genetic Testing in Kidney Transplant Evaluation () - Oct 17, 2021 - Abstract #KIDNEYWEEK2021KIDNEY_WEEK_3313;
Due to the potential increased risk of recurrence, simultaneous liver-kidney transplant and Eculizumab was considered...Genetic testing in pre-KT patients has potential clinical impact on post-KT management and selection of living-related donors. Further research is needed to describe the utility of genetic testing for kidney transplant candidates.
- |||||||||| Soliris (eculizumab) / AstraZeneca, Ultomiris IV (ravulizumab IV) / AstraZeneca
[VIRTUAL] Rare Case of Atypical Hemolytic Uremic Syndrome in a Child () - Oct 17, 2021 - Abstract #KIDNEYWEEK2021KIDNEY_WEEK_3009;
There are variable reports on the efficacy of other therapies, like plasmapheresis, on the course of this specific aHUS entity. Our case highlights the importance of considering these therapies in this population even after prolonged dialysis as this may alter the course of the disease and help kidney recovery.
- |||||||||| Thymoglobulin (anti-thymocyte globulin (rabbit)) / Sanofi, Soliris (eculizumab) / AstraZeneca
[VIRTUAL] Post-Transplant Thrombotic Microangiopathy due to a Pathogenic Mutation in Complement Factor I () - Oct 17, 2021 - Abstract #KIDNEYWEEK2021KIDNEY_WEEK_2628;
Eculizumab was administered, resulting in resolution of thrombocytopenia and hemolysis, and gradual renal recovery...Discussion This is a case of de novo post-transplant aHUS due to a pathogenic complement mutation, likely triggered by transplant surgery. Our strategy of recombinant protein production followed by detailed functional assessment defined the functional repertoire of the variant protein and provided critical guidance relative to the underlying pathophysiology and appropriate therapeutic regimen.
- |||||||||| Actemra IV (tocilizumab) / Roche, JW Pharma, Soliris (eculizumab) / AstraZeneca
[VIRTUAL] Cryoglobulinemic Vasculitis in a Patient with Known Thrombotic Microangiopathy () - Oct 17, 2021 - Abstract #KIDNEYWEEK2021KIDNEY_WEEK_2610;
It remains possible that the patient could have had TMA and cryoglobulinemia concurrently, with renal biopsy during this presentation only capturing the latter. Therefore, further research into the role of complement inhibitors for cryoglobulinemia is needed.
- |||||||||| Soliris (eculizumab) / AstraZeneca, Avastin (bevacizumab) / Roche
[VIRTUAL] Limited Therapeutic Arsenal for the Thrombotic Microangiopathy Spectrum () - Oct 17, 2021 - Abstract #KIDNEYWEEK2021KIDNEY_WEEK_2547;
Terminal complement inhibitors such as Eculizumab have been shown to improve renal function in C-TMA...Following her surgery she had been treated initially with paclitaxel and carboplatin and subsequently she had received bevacizumab and doxorubicin; the last doses of these latter agents were six months before admission...The indication for these medications becomes even more unclear when we factor in drug-induced TMA, cancer-associated TMA, malignant hypertension, and other TMAs without readily identifiable complement disorders. More research on the use of genetic testing and complement inhibition is warranted for the entire spectrum of TMA.
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[VIRTUAL] Diagnostic and Risk Factors for Deregulated Complement in Thrombotic Microangiopathy () - Oct 17, 2021 - Abstract #KIDNEYWEEK2021KIDNEY_WEEK_2537;
Eculizumab treatment was associated with clinical remission in C-TMA ( n / N =12/14; P <0.01 vs. n / N =3/16 untreated patients)...Conclusion Patients with TMA, low plasma C3, and glomerular thrombosis who present at younger age (i.e., <45 years) are at high risk for C-TMA. Although a kidney biopsy is often needed to detect the TMA, morphologic features of chronic damage cannot predict prognosis.
- |||||||||| Soliris (eculizumab) / AstraZeneca, Rituxan (rituximab) / Biogen, Zenyaku Kogyo, Roche
[VIRTUAL] C3 Glomerulonephritis with Nephritic Factor Treated with Rituximab () - Oct 17, 2021 - Abstract #KIDNEYWEEK2021KIDNEY_WEEK_2529;
Steroids, mycophenolate mofetil (MMF), rituximab, and eculizumab have been used with varying results...Patient was initiated on prednisone 60 mg/day with taper, MMF with a maximal dose 3 grams/day, and valsartan for 2 years with little improvement in clinical parameters...Reoccurrence of relapse is being monitored using serial C3NeF measurements. We believe that targeted B cell therapy should be considered in the treatment of C3GN cases which are C3NeF antibody mediated.
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[VIRTUAL] Myeloperoxidase Immunohistochemical Staining and Response to Eculizumab in a Pediatric Patient with Dense Deposit Disease () - Oct 17, 2021 - Abstract #KIDNEYWEEK2021KIDNEY_WEEK_2428;
He was treated with eculizumab (600 mg every 2 weeks) and azathioprine, and over the course of 6 months, his serum creatinine, proteinuria, and complement C3 levels returned to normal...Since then he has been maintained on eculizumab infusions (600 mg every 4 weeks) along with mycophenolate mofetil...To evaluate if eculizumab blocked complement mediated injury on glomerular endothelial cells, MPO staining of his initial and repeat biopsy was performed: his initial biopsy revealed diffuse endothelial staining for MPO along glomerular endothelium and the repeat biopsy showed either no MPO staining or weak MPO staining in the glomerular endothelium. Discussion In this case, we find that MPO immunohistochemical staining may be useful for monitoring the response to complement blockade in patients with DDD.
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[VIRTUAL] Early Recurrence of C3 Glomerulopathy (C3G) in the Allograft () - Oct 17, 2021 - Abstract #KIDNEYWEEK2021KIDNEY_WEEK_2420;
2 PTS received eculizumab- both were without clinical recurrence...Future study is needed to better understand if early detection of recurrence, coupled with anti-complement therapies, improves outcomes. Summary of Clinicopathologic Course After Histologic Recurrence
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[VIRTUAL] C3 Glomerulopathy in Children and Adolescents () - Oct 17, 2021 - Abstract #KIDNEYWEEK2021KIDNEY_WEEK_2411;
Our cohort presented a wide variability in disease course and presentation. Further understanding of the correlation between exact complement abnormality and C3G prognosis is warranted, especially now when new complement system blockers may become available.
- |||||||||| Soliris (eculizumab) / AstraZeneca, Kineret (anakinra) / SOBI
[VIRTUAL] TAFRO: A New Cause for Thrombotic Microangiopathy Mimicking Atypical Hemolytic Uremic Syndrome Successfully Treated with Anakinra and Eculizumab () - Oct 17, 2021 - Abstract #KIDNEYWEEK2021KIDNEY_WEEK_1418;
TAFRO can mimic aHUS & present with markedly elevated IL-2R levels rather than IL-6 levels which can fix complement by the classsic pathway leading to high membrane attack complexes adding to capillary leak, anasarca and TM. 2) An Anti IL-1 drug, Anakinra, & a drug inhibiting C5 cleavage to C5a & C5b , Eclizumab can be combined to successfully treat TAFRO 3) TAFRO must now be added to all reviews and textbooks as a new cause for TM & AKI with classic complement pathway activation.
- |||||||||| Soliris (eculizumab) / AstraZeneca, Ultomiris IV (ravulizumab IV) / AstraZeneca
[VIRTUAL] A Tricky Diagnosis of Complement-Mediated Thrombotic Microangiopathy () - Oct 17, 2021 - Abstract #KIDNEYWEEK2021KIDNEY_WEEK_1178;
Therapy was begun with eculizumab, an anti-C5 monoclonal antibody...He was later switched to ravulizumab for less frequent dosing...The associations between the genetic variants of C-TMA and specific clinical presentations warrants further investigation. The views expressed are those of the authors and do not reflect the official policy of the Department of the Army/Navy/Air Force, the Department of Defense or the United States Government.
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[VIRTUAL] Atypical Hemolytic Uremic Syndrome Secondary to Homozygous CFHR1-CFHR3 Mutation () - Oct 17, 2021 - Abstract #KIDNEYWEEK2021KIDNEY_WEEK_926;
Given high index of suspicion for aHUS and the absence of other causes of thrombotic microangiopathy, he was started on eculizumab...Delayed or inappropriately treated cases lead to increased morbidity and mortality. Early screening for aHUS related genetic mutations in patients with acute kidney injury and microangiopathic hemolytic anemia is essential for prompt diagnosis of aHUS and help guide treatment duration.
- |||||||||| Actemra IV (tocilizumab) / Roche, JW Pharma, Soliris (eculizumab) / AstraZeneca, Enspryng (satralizumab) / Roche
Clinical, Journal: Satralizumab in the treatment of neuromyelitis optica spectrum disorder. (Pubmed Central) - Oct 16, 2021
Here, we review the two randomized, double-blind, Phase III trials that investigated the subcutaneous administration of satralizumab as add-on treatment and monotherapy. Both studies revealed positive effects concerning the reduction of relapse risk for AQP4 seropositive NMOSD patients and generally good tolerability.
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Clinical, Journal: Ockham's razor defeated: about two atypical cases of hemolytic uremic syndrome. (Pubmed Central) - Oct 15, 2021
In case 2 however, it is the simultaneous occurrence of two infectious triggers that led to HUS. In dissent from Ockham's razor, an exceptional disease such as HUS may stem from the sequential occurrence or co-occurrence of several rare conditions.
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Review, Journal: Atypical Hemolytic Uremic Syndrome: New Challenges in the Complement Blockage Era. (Pubmed Central) - Oct 15, 2021
However, the high cost of therapy has led to make efforts to develop precise complement functional and genetic studies that help physicians to determine the appropriate duration of eculizumab therapy. Nowadays, more studies are needed to select candidates to adjustment of therapy.
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