- |||||||||| Soliris (eculizumab) / AstraZeneca, Ultomiris IV (ravulizumab IV) / AstraZeneca, Empaveli (pegcetacoplan SC) / Apellis, SOBI
Journal: Emerging drugs for the treatment of paroxysmal nocturnal hemoglobinuria. (Pubmed Central) - Apr 6, 2022
Pegcetacoplan is the first C3-inihibitor approved by FDA. Long-term safety data for novel complement inhibitors are needed.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Review, Journal: Halting targeted and collateral damage to red blood cells by the complement system. (Pubmed Central) - Apr 5, 2022
In recent years, complement inhibition has improved treatment prospects for these patients, with eculizumab now the standard of care for PNH patients...This review gives an overview of the underlying pathophysiology of complement-mediated hemolysis in PNH and AIHA, the role of therapeutic complement inhibition nowadays, and the high number of complement inhibitors currently under investigation, as for almost every complement protein, an inhibitor is being developed. The focus lies with novel therapeutics that inhibit complement activity specifically in the pathway that causes pathology or those that reduce costs or patient burden through novel administration routes.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Journal: Complement and the prothrombotic state. (Pubmed Central) - Apr 5, 2022
In 2007 and 2009, the regulatory approval of the first-in-class complement inhibitor eculizumab revolutionized the clinical management of 2 rare, life-threatening clinical conditions: paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS)...Next to PNH and aHUS, germline encoded CD59 or CD55 deficiency (the latter causing the disease Complement Hyperactivation, Angiopathic thrombosis, and Protein-Losing Enteropathy; CHAPLE), autoimmune hemolytic anemia (AIHA), (catastrophic) anti-phospholipid syndrome (APS, CAPS) and C3 glomerulopathy are considered. Parallels and distinct features among these conditions are discussed against the background of thrombosis, complement activation, and potential complement diagnostic and therapeutic avenues.
- |||||||||| Soliris (eculizumab) / AstraZeneca, Ultomiris IV (ravulizumab IV) / AstraZeneca
Journal: Application of C5 inhibitors in glomerular diseases in 2021. (Pubmed Central) - Apr 1, 2022
New data in the domain of C5-inhibition in glomerular diseases are still limited and mainly focus on 1) the efficacy of ravulizumab, a long-acting C5 inhibitor in aHUS, and 2) the use of avacopan, a C5aR antagonist, in antineutrophil cytoplasmic antibody vasculitis. Several new studies ongoing or planned for the next few years will evaluate the efficacy of C5 inhibition in secondary thrombotic microangiopathy, C3 glomerulopathy, membranous nephropathy, or immunoglobulin A nephropathy.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Clinical, Journal: A case of neuromyelitis optica associated with pulmonary Mycobacterium avium complex disease (Pubmed Central) - Mar 29, 2022
Exacerbation of the pulmonary MAC disease and the following internal counteraction with IL-6 may have resulted in a NMO relapse. The present patient was therefore administered eculizumab but not satralizumab, a humanized anti-IL-6 receptor antibody, for prevention of NMO recurrence.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Review, Journal: Current Treatment of Myasthenia Gravis. (Pubmed Central) - Mar 26, 2022
Substantial progress has been made in the treatment of MG in the last century, resulting in a change of its natural course from a disease with poor prognosis with a high mortality rate in the early 20th century to a treatable condition with a large proportion of patients attaining very good disease control. This review summarizes the current treatment options for MG, including non-immunosuppressive and immunosuppressive treatments, as well as thymectomy and targeted immunomodulatory drugs.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Journal: Eculizumab use in a tertiary care nephrology center: data from the Vienna TMA cohort. (Pubmed Central) - Mar 24, 2022
In summary, eculizumab is the treatment of choice for cTMA patients that do not respond to plasma therapy. In patients with sTMA and C3G, the response rates to therapy are much lower and therefore, the decision to start therapy needs to be considered carefully.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Review, Journal: Eculizumab use in catastrophic antiphospholipid syndrome (CAPS): Descriptive analysis from the "CAPS Registry". (Pubmed Central) - Mar 24, 2022
In patients with sTMA and C3G, the response rates to therapy are much lower and therefore, the decision to start therapy needs to be considered carefully. According to the real-world experience provided by the "CAPS Registry", eculizumab can be considered in some patients with CAPS refractory to previous therapies, especially if they present with features of complement-mediated thrombotic microangiopathy.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Clinical, Journal: Pregnancy in Women with Atypical Hemolytic Uremic Syndrome. (Pubmed Central) - Mar 23, 2022
P=N/A
Our results show that in women with aHUS, even on dialysis or with a kidney graft, pregnancy is possible with careful monitoring for aHUS flares and prematurity. Prophylactic or therapeutic eculizumab offers disease control with low-risk of fetal abnormalities.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Journal: CD55-deficiency in Jews of Bukharan descent is caused by the Cromer blood type Dr(a-) variant. (Pubmed Central) - Mar 23, 2022
Establishing the diagnosis of CD55-deficiency in a timely manner, even in patients with milder symptoms, may have a critical effect on their management and quality-of-life since treatment with the complement inhibitor eculizumab is highly effective in ameliorating disease manifestations. Awareness of founder mutations within certain populations can further guide genetic testing and prevent a diagnostic odyssey, by placing this CD55 variant high on the differential diagnosis.
- |||||||||| Soliris (eculizumab) / AstraZeneca
MICROANGIOPATHIC HEMOLYTIC ANEMIA COMPLICATED BY BACTEREMIA IN A POSTPARTUM COVID-19 PATIENT ([VIRTUAL]) - Mar 23, 2022 - Abstract #SCCM2022SCCM_1248;
Eculizumab was not initiated as blood cultures had not yet resulted...Additionally, no COVID-19 vaccines were available and no American College of Obstetrics and Gynecology existed on best practices. Regardless, timely recognition of overlapping clinical syndromes is essential in critical care medicine.
- |||||||||| Rare presentation of thrombotic microangiopathy and cytomegalovirus in a kidney transplant recipient () - Mar 19, 2022 - Abstract #NKFSCM2022NKF_SCM_958;
Tacrolimus was switched to belatacept to minimize risk for thrombotic microangiopathy (TMA)...Patient was started on IV ganciclovir and switched to PO valganciclovir after viremia was controlled...utilized eculizumab to mitigate activation of the classical complement pathway. This case illustrates how CMV infection can be a trigger for TMA in transplant recipients adding to the unusual manifestations.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Clinical, Journal, Monotherapy: Eculizumab monotherapy for NMOSD: Data from PREVENT and its open-label extension. (Pubmed Central) - Mar 17, 2022
P3
Eculizumab monotherapy provides effective long-term relapse prevention, relieving the chronic immunosuppression burden in patients with AQP4-IgG + NMOSD. ClinicalTrials.gov; PREVENT: NCT01892345; open-label extension: NCT02003144.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Clinical: Thrombotic Microangiopathy Triggered by COVID-19: Case Reports. (Pubmed Central) - Mar 15, 2022
The use of anticomplement therapies such as eculizumab should be considered in refractory cases of progressive COVID-19. Controlled clinical trials are required before a definitive statement can be made.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Journal: Size Exclusion and Ion Exchange Chromatographic Hardware Modified with a Hydrophilic Hybrid Surface. (Pubmed Central) - Mar 15, 2022
Moreover, AEX separations of intact mRNA samples (Cas9 and EPO mRNA) were improved, where it was seen that h-HST column hardware provided higher sensitivity and more repeatable peak areas from injection to injection. As such, there is significant potential in the use of h-HST chromatographic hardware to facilitate more robust and more sensitive analyses for a multitude of challenging separations and analytes.
- |||||||||| Actemra IV (tocilizumab) / Roche, JW Pharma, Soliris (eculizumab) / AstraZeneca
Review, Journal: TREATMENT OF MYASTHENIA GRAVIS PATIENTS WITH COVID-19: REVIEW OF THE LITERATURE. (Pubmed Central) - Mar 15, 2022
Concerning plasmapheresis and intravenous immunoglobulins, both can be used in COVID-19 while taking into consideration thromboembolic properties of the former and hemodynamic disturbances of the latter. As current data suggest, all known COVID-19 vaccines are safe to use in MG patients.
- |||||||||| Soliris (eculizumab) / AstraZeneca, Humira (adalimumab) / Eisai, AbbVie
Journal: Atypical HUS and Crohn's disease-interference of intestinal disease activity with complement-blocking treatment. (Pubmed Central) - Mar 11, 2022
In aHUS cases secondary to CD, close monitoring of both complement inhibition and serum ECU levels is needed as intestinal disease can interfere with complement-blocking treatment. Increased doses of ECU may be necessary to maintain therapeutic blood levels of ECU and full complement blockade, especially if the intestinal disease is not under control.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Review, Journal: Pravastatin, proton pump inhibitors, metformin, micronutrients and biologics: new horizons for the prevention or treatment of preeclampsia. (Pubmed Central) - Mar 9, 2022
Proton-pump inhibitors, metformin, and sulfasalazine are other drugs with preclinical evidence of multiple molecular actions that could resolve the pathophysiology of preeclampsia...Other treatment approaches that have transitioned to human trials (ranging from single-arm to phase III trials that have been completed or are ongoing) include folic acid, nitric oxide donors (such as L-arginine), recombinant antithrombin III, digoxin immune antigen-binding fragment, and melatonin...Recent preclinical studies have proposed novel molecular targeted strategies such neutralizing antibodies, or short interfering RNA technologies targeting soluble fms-like tyrosine kinase-1 (sFlt-1) or angiotensinogen. If new agents were discovered that are effective in preventing or treating preeclampsia the potential to improve global maternal and perinatal health is significant.
- |||||||||| Soliris (eculizumab) / AstraZeneca, Vyvgart (efgartigimod alfa-fcab) / argenx, Broteio
Numbers Needed to Treat and Costs per Improved Outcome Among Treatments for Myasthenia Gravis (In-person & Virtual) - Mar 8, 2022 - Abstract #ISPOR2022ISPOR_560;
P2, P3
NNT and cost per improved efficacy help inform comparative clinical efficacy and cost-effectiveness across MG treatments. Evidence indicates more favorable treatment benefit and economic value for EFG with fewer NNT and lower cost required to achieve improved outcomes compared to other treatments.
- |||||||||| Soliris (eculizumab) / AstraZeneca, Nulojix (belatacept) / BMS
From Typical to Atypical HUS () - Mar 6, 2022 - Abstract #NKFSCM2022NKF_SCM_683;
Because of concern for complement mediated HUS, patient was initiated on eculizumab after meningococcal vaccination and penicillin V prophylaxis...He is currently on outpatient bi-weekly eculizumab along with belatacept/MPA...Tacrolimus less likely played a major role here since patient has been on tacrolimus for many years and level was therapeutic. CONCLUSION Our case demonstrates that typical HUS can be a trigger for atypical HUS in susceptible individuals.
- |||||||||| mitomycin / Generic mfg.
Thrombotic Microangiopathy Following HIPEC Mitomycin () - Mar 6, 2022 - Abstract #NKFSCM2022NKF_SCM_631;
This case highlights the importance of pursuing a kidney biopsy in patients with unclear AKI etiology. Pathologic data might carry significant therapeutic implications
- |||||||||| Soliris (eculizumab) / AstraZeneca
Acute Kidney Injury due to Thrombotic Microangiopathy from Catastrophic Antiphospholipid Syndrome Treated with Eculizumab. () - Mar 6, 2022 - Abstract #NKFSCM2022NKF_SCM_594;
In a retrospective review of 11 CAPS cases treated with eculizumab, 5 patients responded to treatment, as demonstrated by stabilization of platelets and recovery of kidney function. CONCLUSION Our case will add to growing evidence of the use of eculizumab, a recombinant humanized monoclonal antibody that inhibits C5 activation, for treatment of TMA from APS though it needs further prospective studies on dosing, treatment duration and effectiveness.
- |||||||||| Soliris (eculizumab) / AstraZeneca
An Atypical Presentation of Atypical HUS () - Mar 6, 2022 - Abstract #NKFSCM2022NKF_SCM_563;
The most frequent mutations detected involves complement Factor H (30%), then membrane cofactor protein (10%), and lastly complement factor I/CFI (5%).2 While typical HUS usually resolves with supportive care, aHUS may require plasma exchange, if indistinguishable from TTP. For patients with aHUS, eculizumab, a mono-clonal antibody against the terminal complement component C5, should be considered.2 CONCLUSION This aHUS case is unique given patient did not present with thrombocytopenia and mutations in CFI causing aHUS are rare.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Immune Reconstitution Therapy in NMOSD (Poster Hall) - Mar 6, 2022 - Abstract #AAN2022AAN_3905;
In NMO, as a 1st step control the complement (eculizumab), with subsequent reconstitution of the immune system (AHSCBMT),and then ultimately transition to immunomodulation (satralizumab) and reserve immunosuppression (inebilizumab) as 4th line. Also utilize AHSCBMT as salvage therapy for severe breakthrough disease during or after NMO-DMTs.
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