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Purtscher-Like Retinopathy: A Cue for Underlying Thrombotic Microangiopathy () - Oct 23, 2019 - Abstract #KIDNEYWEEK2019KIDNEY_WEEK_5438;
She was treated with pulse dose steroids with slight improvement in her vision; and received 2 doses of Eculizumab...The presence of PLR should alert physicians to evaluate for an underlying TMA. Unfortunately, no effective treatment for PLR exists.
- |||||||||| Flagyl (metronidazole) / SLA, ciprofloxacin / Generic Mfg., Soliris (eculizumab) / Alexion Pharma
Escherichia coli-Associated Hemolytic Uremic Syndrome (HUS) Treated with Eculizumab () - Oct 23, 2019 - Abstract #KIDNEYWEEK2019KIDNEY_WEEK_5247;
Discussion We demonstrate here the favorable clinical course of Shiga toxin–associated hemolytic–uremic syndrome (STAHUS) following a single dose of the terminal complement inhibitor eculizumab in an otherwise healthy adult patient. Successful cases like this add to the base of evidence that eculizumab is useful in treating multiple diseases driven by complement system dysregulation.
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Maternal Pregnancy Outcomes in Women with Complement-Mediated TMA: Update of the Vienna TMA Cohort (145, Walter E. Washington Convention Center) - Oct 14, 2019 - Abstract #KIDNEYWEEK2019KIDNEY_WEEK_5172;
Four women received preventive plasma therapy for 6 pregnancies (2 KTX; 1 p-cTMA), 2 became pregnant during ongoing eculizumab therapy (3 pregnancies,1 KTX; 0 p-cTMA), 5 received therapeutic plasma therapy for p-cTMA (1 KTX), and 2 were switched to eculizumab (0 ESRD)...Conclusion In summary, VTC data demonstrates good maternal outcome (1) in the majority of untreated pregnancies in women with a diagnosis of cTMA and (2) in specifically treated cTMA patients in a specialized center. Figure 1.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, Rituxan (rituximab) / Roche, Biogen
Treatment of C3 Glomerulopathy in Kidney Transplant Recipients: A Meta-Analysis (Exhibit Hall, Walter E. Washington Convention Center) - Oct 14, 2019 - Abstract #KIDNEYWEEK2019KIDNEY_WEEK_4717;
Conclusion Our study suggests that KTX patients with C3G treated with eculizumab had the lowest incidence of allograft loss (33%) when compared to those treated with TPE or rituximab. Among those who received no treatment for C3G due to stable allograft function, there is an incidence of allograft loss of 33% in C3GN and 53% in DDD.
- |||||||||| tacrolimus / Generic Mfg., Soliris (eculizumab) / Alexion Pharma
A Unique Immunosuppression Strategy in a Patient with Atypical HUS Undergoing Kidney Transplant (Exhibit Hall, Walter E. Washington Convention Center) - Oct 14, 2019 - Abstract #KIDNEYWEEK2019KIDNEY_WEEK_4690;
We chose maintenance immunosuppression with belatacept instead of tacrolimus along with mycophenolate mofetil (MMF)...Belatacept is a selective T-cell co-stimulation blocker which has not been reported to induce TMA. Our case demonstrates the safety and efficacy of Thymoglobulin induction followed by belatacept/ MMF maintenance along with eculizumab in highly sensitized patient with aHUS.
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Massive Ex Vivo C5b9 Formation on Endothelium Indicates Complement Defects and a Clinical Response to Complement Inhibition in Patients with Thrombotic Microangiopathy (Exhibit Hall, Walter E. Washington Convention Center) - Oct 14, 2019 - Abstract #KIDNEYWEEK2019KIDNEY_WEEK_4607;
Seventeen patients with massive ex vivo C5b9 formation were treated with eculizumab (median number of treatment was 14); renal survival at 1 year was n/N=14/17 (82%) and n/N=9/23 (39%) for treated and untreated patients (P<0.01), respectively...Conclusion Massive ex vivo C5b9 formation indicates defects in complement regulation as the dominant cause of TMA, including TMAs that present with coexisting conditions. Patients with TMA and massive ex vivo C5b9 formation may benefit from complement inhibition.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, Rituxan (rituximab) / Roche, Biogen
Monoclonal Gammopathy-Associated Thrombotic Microangiopathy (Exhibit Hall, Walter E. Washington Convention Center) - Oct 14, 2019 - Abstract #KIDNEYWEEK2019KIDNEY_WEEK_4547;
Disease-directed therapy should be considered first-line treatment of TMA, in addition to PLEX if there is ADAMTS13 deficiency. Funding Commercial Support
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Successful Pregnancies During Ongoing Complement Blockade in Two Patients with Complement Mediated Thrombotic Microangiopathy (Exhibit Hall, Walter E. Washington Convention Center) - Oct 14, 2019 - Abstract #KIDNEYWEEK2019KIDNEY_WEEK_4120;
Data on pregnancies of cTMA patients during ongoing eculizumab (ECU) therapy, however, are scarce...Results of complement related tests in cord blood showed deficient complement activity, with low factor and regulator levels without overactivation, which most likely reflects the situation related to age and the presence of ECU in cord blood. Discussion Pregnancy and delivery outcomes with ongoing ECU therapy in two genetically high-risk cTMA patients with preserved native kidney and kidney transplant function were excellent.
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Thrombotic Microangiopathy as the Presenting Feature of Newly Diagnosed HIV Infection Treated with Eculizumab (Exhibit Hall, Walter E. Washington Convention Center) - Oct 14, 2019 - Abstract #KIDNEYWEEK2019KIDNEY_WEEK_4107;
Because uncontrolled HIV infection has been associated with increased complement activation, we hypothesized that HIV infection triggered dysregulation of complement activity in our patient and that treatment with eculizumab would be beneficial. Although our patient did not have a common gene mutation associated with atypical HUS, treatment with eculizumab in conjunction with ART resulted in hematologic remission as well as improvement in kidney function and discontinuation of dialysis.
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Clinical and Genetic Characteristics of Pregnancy-Associated aHUS in Japan (Exhibit Hall, Walter E. Washington Convention Center) - Oct 14, 2019 - Abstract #KIDNEYWEEK2019KIDNEY_WEEK_2943;
One case (CFH R1215G) continued eculizumab therapy every two weeks until now...Conclusion The clinical and genetic characteristics of pregnancy-associated aHUS in our cohort are; 1) postpartum TMA with HELLP syndrome is typical, 2) the recurrence rate of aHUS is low, 3) the recovery of kidney and liver functions is generally good, 4) diverse mutations in complement related factors were detected, but they might not be the sole factors responsible for the onset of the disease. Funding Government Support - Non-U.S.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, yttrium-90 (SIR-Spheres) / SIRTeX Medical
Thrombotic Microangiopathy Following Y90-Dotatate Treatment (Exhibit Hall, Walter E. Washington Convention Center) - Oct 14, 2019 - Abstract #KIDNEYWEEK2019KIDNEY_WEEK_2810;
Discussion TMA has been described in association with Y90-Dotate therapy and this case demonstrates this rare complication which carries a poor renal prognosis. Renal biopsy: glomerulus showing thrombi in capillary loops and basement membrane thickening; mesangiolysis; aneurysmal dilatation of capillary loops; background acute tubular necrosis.
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Successful Pregnancies In Kidney Transplant Recipients with Complement-Mediated Thrombotic Microangiopathy (Exhibit Hall, Walter E. Washington Convention Center) - Oct 14, 2019 - Abstract #KIDNEYWEEK2019KIDNEY_WEEK_2239;
Preventive plasma therapy was used in three pregnancies, and one patient had ongoing eculizumab (ECU) therapy during two pregnancies...Thus, becoming pregnant can be encouraged in kidney transplant recipients with native kidney cTMA. Extensive preconception counseling, however, is mandatory in such cases.
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Gender Differences in Presentation and Outcomes Among Patients with Atypical Hemolytic Uremic Syndrome (aHUS) (Exhibit Hall, Walter E. Washington Convention Center) - Oct 14, 2019 - Abstract #KIDNEYWEEK2019KIDNEY_WEEK_2223;
23 received plasma and 7 eculizumab therapy...Women presented at younger age, more often harbored disease-causing genetic variants or a CFH- or CD46-risk haplotype, and had better kidney function at last follow-up as compared to males. Table 1.*no data available in 1, 5 showed no genetic variants
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A Case of Pediatric Dense Deposit Disease (DDD) in Remission for 7 Years on Eculizumab (Exhibit Hall, Walter E. Washington Convention Center) - Oct 14, 2019 - Abstract #KIDNEYWEEK2019KIDNEY_WEEK_1774;
To date, he may have one of the longest DDD remissions on Eculizumab monotherapy. Further studies including a randomized controlled trial of Eculizumab monotherapy could define parameters for starting and discontinuing treatment.
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Recurrent C3 Glomerulonephritis (Exhibit Hall, Walter E. Washington Convention Center) - Oct 14, 2019 - Abstract #KIDNEYWEEK2019KIDNEY_WEEK_1750;
Intravenous steriods were given twice daily for 4 days, transitioned to prednisone...After 6 doses of cyclophosphamide 750mg, in 3 months renal function stabilized with creatinine 1.34mg/dl, urine protein excretion 0.8g and C3 153mg/dl...Due to repetitive steriod and immunosuppression responsiveness we conclude that recurrent cases of atypical post infectious glomerulonephritis be investigated for alternate pathway mutations. Novel anticomplement medications such as eculizumab may also be used.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, Avastin (bevacizumab) / Roche
Bevacizumab-Associated Thrombotic Microangiopathy Treated with Eculizumab (Exhibit Hall, Walter E. Washington Convention Center) - Oct 14, 2019 - Abstract #KIDNEYWEEK2019KIDNEY_WEEK_1510;
Case Description Case 1: A 68 yo woman with recurrent, metastatic ovarian high-grade serous carcinoma received pegylated liposomal doxorubicin (PLD) and bevacizumab...Case 2: A 52 yo woman with HTN and stage IIIC poorly differentiated serous carcinoma was treated for progression of disease with PLD, atezolizumab, and bevacizumab...Discussion We present two cases of severe bevacizumab-associated TMA with renal involvement who had evidence of disease improvement or stabilization with eculizumab treatment. The role of complement in VEGF inhibitor-associated TMA and treatment with complement blockade deserves further study.
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Cocaine-Associated Atypical Haemolytic Syndrome in a Genetically Susceptible Individual (Exhibit Hall, Walter E. Washington Convention Center) - Oct 14, 2019 - Abstract #KIDNEYWEEK2019KIDNEY_WEEK_1502;
It responded well to rapid treatment with eculizumab supporting the hypothesis of an underlying immunological complement-based pathogenesis. Furthermore, in the absence of gold standard diagnosis of aHUS in the acute setting, prompt treatment with eculizumab can be challenging and delayed.
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AKI Secondary to Thrombotic Microangiopathy (TMA) in a Myeloma Patient: Scleroderma Renal Crisis (SRC), A Paraneoplastic Phenomenon (Exhibit Hall, Walter E. Washington Convention Center) - Oct 14, 2019 - Abstract #KIDNEYWEEK2019KIDNEY_WEEK_1498;
Patient was treated with captopril initially for SRC and subsequently Eculizumab...TMA is known to be associated with both MM and SRC, and although the clinical presentation was suggestive of scleroderma, the negative serologies challenged the diagnosis. Further studies are required to determine whether MM can induce SRC and if chemotherapy can improve scleroderma outcomes in these patients.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, Rituxan (rituximab) / Roche, Biogen
Diagnosing Atypical HUS in the Setting of Complement Amplifying Conditions (CAC): A Case Series (Exhibit Hall, Walter E. Washington Convention Center) - Oct 14, 2019 - Abstract #KIDNEYWEEK2019KIDNEY_WEEK_1488;
Case Description Case 1: 20 Yr old African American woman with Lupus (diagnosed at age 16, chronically low complements, partially controlled with Azathioprine, Rituximab, Mycophenolate and recently started on Cyclophosphamide (CYC) for class IV lupus nephritis, received 3 doses) presented to hospital for confusion and seizures...Started on Eculizumab (ECU) for aHUS (900 mg/week for 4 weeks)...Her renal function and other lab parameters returned to normal, and remained stable for past 18 months. Discussion Clinicians should have low threshold for suspecting aHUS if CAC's do not respond to appropriate therapy.
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Clinical, Journal: The unusual couple: a clinical case of coexistence between aHUS and Fabry's disease (Pubmed Central) - Oct 13, 2019
Today, the rapid diagnosis of the disease and the timely start of treatment with eculizumab, improve outcomes of renal failure, stroke and heart attack (8-10)...The diagnostic suspicion based on anamnesis and careful research of the symptoms and then confirmed by the enzymatic dosage of alpha galactosidase or by molecular analysis, allows the early treatment of the patient with enzyme replacement therapy, guaranteeing the resolution and/or slowing down the evolution of the disease, especially in the brain, heart and kidneys. In this report, we describe the clinical case of a patient who is a carrier of both rare diseases.
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Review, Journal: Where are we with haemolytic uremic syndrome? (Pubmed Central) - Oct 12, 2019
Therefore, treatment is based on complement inhibitors like eculizumab, a drug that has revolutionised the natural history of the disease. Relapses are frequent after kidney transplant and thus confer a poor prognosis.
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Journal: Myasthenia gravis (Pubmed Central) - Oct 11, 2019
This article reviews the basic and advanced treatment options of the different disease subtypes including plasma exchange and immunoglobulins for treatment in a myasthenic crisis. Recently, clinical approval of eculizumab, a complement inhibitor, enriched the pharmacological armamentarium for AChR antibody-positive MG patients not appropriately responding to immunosuppression alone.
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Journal: Therapies Directed Against B-Cells and Downstream Effectors in Generalized Autoimmune Myasthenia Gravis: Current Status. (Pubmed Central) - Oct 8, 2019
The rarity of myasthenia gravis, heterogeneity in its clinical manifestations, and variability in immunosuppressive regimens are challenges to conducting successful trials. Nonetheless, these are promising times for myasthenia gravis, as renewed research efforts provide novel insights into its immunopathology, allowing for development of targeted therapies with increased efficacy and safety.
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Clinical, Journal: Ravulizumab: First Global Approval. (Pubmed Central) - Oct 8, 2019
Alexion is also evaluating the coadministration of subcutaneous ravulizumab with Halozyme's ENHANZE drug-delivery technology (rHuPH20), which may have the potential to further extend the dosing interval. This article summarizes the milestones in the development of ravulizumab leading to this first approval for PNH.
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Journal: Eculizumab improves fatigue in refractory generalized myasthenia gravis. (Pubmed Central) - Oct 8, 2019
P3
Compared with placebo, eculizumab was associated with improvements in perceived fatigue that strongly correlated with improvements in MG-specific outcome measures. Trial ID Registration: NCT01997229, NCT02301624.
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Journal: Diacylglycerol kinase epsilon nephropathy: late diagnosis and therapeutic implications. (Pubmed Central) - Oct 5, 2019
Eculizumab was started and after 18 months of treatment, he persisted with hypertension, decline in renal function and proteinuria...Complement blockade was stopped. This case of late diagnosis of DGKe nephropathy highlights the importance of genetic testing in patients presenting TMA during the first year of life.
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