- |||||||||| Soliris (eculizumab) / Alexion Pharma
Journal: Developments in immunosuppression. (Pubmed Central) - Jul 28, 2021
Recent knowledge creates a path towards future immunosuppression success in sensitized recipients and in those in high risk of viral infections or CNI nephrotoxicity.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, Ultomiris IV (ravulizumab IV) / Alexion Pharma
Journal: How I Treat Paroxysmal nocturnal hemoglobinuria. (Pubmed Central) - Jul 28, 2021
We review the concept of pharmacokinetic and pharmacodynamic breakthrough hemolysis and briefly discuss new complement inhibitors upstream of C5 that are in clinical development. Lastly, we discuss the rare indications for bone marrow transplantation in PNH patients.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Review, Journal: The Syndromes of Thrombotic Microangiopathy: A Critical Appraisal on Complement Dysregulation. (Pubmed Central) - Jul 27, 2021
In this review, we performed a critical appraisal on complement dysregulation and the use of therapeutic complement inhibition in TMAs associated with coexisting conditions and outline a pragmatic approach to diagnosis and treatment. For future studies, we advocate the term complement-mediated TMA as opposed to the traditional atypical HUS-type classification.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Journal: Carfilzomib-induced haemolytic and uremic syndrome: Favorable outcome with eculizumab (Pubmed Central) - Jul 24, 2021
The overall evolution was favorable and an improvement of the renal function promptly allowed the discontinuation of hemodialysis. We discuss the mechanisms that may activate the alternative complement pathway and the potential interest of a transient use of eculizumab in case of carfilzomib-induced hemolytic and uremic syndrome.
- |||||||||| Soliris (eculizumab) / AstraZeneca, Avastin (bevacizumab) / Roche
Clinical, Review, Journal: Bevacizumab-associated thrombotic microangiopathy treated with eculizumab: A case series and systematic review of the literature. (Pubmed Central) - Jul 17, 2021
We discuss the mechanisms that may activate the alternative complement pathway and the potential interest of a transient use of eculizumab in case of carfilzomib-induced hemolytic and uremic syndrome. Given the findings of this systematic review, the use of eculizumab in the treatment of bevacizumab-associated thrombotic microangiopathy warrants further study, particularly in severe cases.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Review, Journal: Peri- and Post-operative Evaluation and Management of Atypical Hemolytic Uremic Syndrome (aHUS) in Kidney Transplantation. (Pubmed Central) - Jul 11, 2021
Over the past decade, advancements in the understanding of etiopathogenesis of aHUS and approval of the anti-complement drug, eculizumab, have allowed for successful kidney transplantation in these patients...Patients in whom a genetic variant is not identified or in whom a genetic variant of uncertain significance is identified should undergo further testing to determine etiology of disease. This review aims to shed light on the diagnostic and therapeutic considerations in patients with aHUS preceding and following kidney transplantation.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Review, Journal: Complement-Based Therapy in the Management of Antibody-Mediated Rejection. (Pubmed Central) - Jul 11, 2021
Several case reports described the successful use of eculizumab in the treatment of AMR, but there are no randomized controlled studies that showed efficacy. Treatment of AMR with C1 INH, in addition to standard of care, did not change short-term outcome but long-term studies are underway.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Review, Journal: Complement-Mediated Disorders in Pregnancy. (Pubmed Central) - Jul 11, 2021
The advent of the anti-C5-antibody eculizumab to quench the complement cascade has already proven in small case series to improve maternal kidney outcomes in complement-mediated obstetric catastrophes such as P-aHUS and HELLP. In this review, we will detail the pathogenesis behind these complement-mediated pregnancy disorders, the role of complement variants in disease phenotype, and the most up-to-date experience with eculizumab in this population.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Review, Journal: Novel Complement Therapeutics in Development as Potential Treatment for Renal Disease. (Pubmed Central) - Jul 11, 2021
There are now multiple new agents in clinical trials, from oral molecules to small inhibitory RNA, that target the classical, lectin, and alternative pathways. Herein we summarize several potential renal diseases in which complement inhibitors may provide a therapeutic benefit, as well as specific complement inhibitors in development.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Journal: Scleroderma Renal Crisis: A Reversible Cause of Left Ventricular Dysfunction. (Pubmed Central) - Jul 10, 2021
The case is particularly intriguing for the favorable outcome of both symptoms and heart function following immunosuppressive therapy. We also highlight the changes observed over time with image techniques as well as in electrocardiograms.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Review, Journal: Update on C3 Glomerulopathy: A Complement-Mediated Disease. (Pubmed Central) - Jul 9, 2021
Thus, its current clinical indication in C3G is restricted to rapidly progressive forms of the disease. To summarize, in recent years, several important advances have taken place in the understanding of C3G, but still further studies are warranted to elucidate the best therapeutic strategies that could improve prognosis of this entity.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Review, Journal: Immune reconstitution therapy in NMOSD. (Pubmed Central) - Jul 8, 2021
In NMO, control the complement (eculizumab), reconstitute the immune system (AHSCT), transition to immunomodulation (satralizumab) and reserve immunosuppression (inebilizumab) as 4th line. AHSCT might also be used as rescue therapy for severe breakthrough disease after NMO-DMTs.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Clinical, Retrospective data, Journal: Shiga Toxin-Associated Hemolytic Uremic Syndrome in Adults, France, 2009-2017. (Pubmed Central) - Jul 7, 2021
According to multivariate analysis and propensity score-matching, eculizumab treatment was not associated with survival. We found that underlying conditions, especially immunodeficiency, are strongly associated with decreased survival in adults who have hemolytic uremic syndrome caused by STEC.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
[VIRTUAL] Perniosis: a rare cutaneous manifestation of paroxysmal nocturnal haemoglobinuria () - Jul 5, 2021 - Abstract #BAD2021BAD_248;
Perniosis typically requires investigation for connective tissue diseases; however, we also warn dermatologists that perniosis could be a presenting feature of underlying paroxysmal nocturnal haemoglobinuria, a rare, life-threatening condition with a high mortality rate related to thromboembolism. This case highlights an interesting and previously unreported cutaneous manifestation of PNH.
- |||||||||| Thymoglobulin (anti-thymocyte globulin (rabbit)) / Sanofi, Soliris (eculizumab) / Alexion Pharma
Journal: Kidney Allograft Rejection: Diagnosis and Treatment Practices in USA- A UNOS Survey. (Pubmed Central) - Jul 4, 2021
There are diverse management practices for diagnosing and treating rejection. An effort to harmonize these diverse practices for management of TCMR and ABMR will give an opportunity to pool data for evaluating clinical outcomes.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Clinical, Review, Journal: Streptococcus Pneumoniae-Associated Hemolytic Uremic Syndrome in the Era of Pneumococcal Vaccine. (Pubmed Central) - Jul 4, 2021
Recently, the addition of eculizumab therapy in the management of Sp-HUS for control of dysregulated complement activity has demonstrated good outcomes, although randomized clinical trials are awaited. A sustained pneumococcal vaccination program and vigilance for replacement serotypes will be the key for persistent reduction in Sp-HUS cases worldwide.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Journal: Acute renal failure and cancer treatment (Pubmed Central) - Jul 1, 2021
Gemcitabine stands as one the drug well acknowledged for eliciting thrombotic microngioapathy with potentially devastating consequences so that eculizumab has been contemplated as a possible therapy, on a case to case basis. Finally, it should be kept in mind that acute kidney injury in the setting of cancer represents a severe complication in the course of the disease."
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Journal: Hypertensive choroidopathy in atypical hemolytic-uremic syndrome. (Pubmed Central) - Jun 30, 2021
Treatment including hemodialysis, plasma exchange, systemic steroids, antihypertensive medications and eculizumab was started 1 month prior to referral...Ocular manifestations in atypical hemolytic uremic syndrome include retinal, choroidal and vitreal hemorrhages, retina and/or ischemic signs. Bilateral serous retinal detachment may also be a sign of atypical hemolytic uremic syndrome or even the first manifestation of a hypertensive event.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Review, Journal: Consensus regarding diagnosis and management of atypical hemolytic uremic syndrome. (Pubmed Central) - Jun 26, 2021
However, there remain unresolved challenges in terms of treatment duration, cost, and infectious complications. A consensus regarding diagnosis and management of TMA syndrome would enhance understanding of the disease and enable treatment decision-making.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Clinical, Journal: Molecular basis and outcomes of atypical haemolytic uraemic syndrome in Czech children. (Pubmed Central) - Jun 24, 2021
Ten patients were initially treated with plasma exchange and eight with eculizumab or with a combination of eculizumab and plasma exchange...The prevailing finding was the unusually high rate of CFHR1/CFHR3 deletions associated with anti-complement factor H antibodies (62% of patients). • The incidence of aHUS in Czech children is 0.092 (CI 0.053-0.131) cases per million inhabitants and 0.92 (CI 0.53-1.32) cases per 100,000 births for the entire reporting period.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Journal: Thrombotic Microangiopathy After Post-Transplantation Cyclophosphamide-Based GVHD Prophylaxis. (Pubmed Central) - Jun 24, 2021
The paucity of events made the determination of risk factors difficult; however, the low incidence of taTMA in this cohort may be related to the limited use of myeloablative conditioning regimens, low incidence of severe GVHD, and use of PTCy. PTCy-based GVHD prophylaxis appears to be associated with a low incidence of severe taTMA.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Journal: Monoclonal Antibodies Capable of Inhibiting Complement Downstream of C5 in Multiple Species. (Pubmed Central) - Jun 24, 2021
The C5-blocking monoclonal antibody (mAb) eculizumab, the first of the new wave of complement blocking drugs, was FDA approved for treatment of Paroxysmal Nocturnal Hemoglobinuria in 2007; its expansion into other diseases has been slow and remains restricted to rare and ultra-rare diseases such as atypical hemolytic uremic syndrome...The C5b-7 complex selective mAb 2H2, most active in rat serum, efficiently inhibited serum lytic activity in vivo for over a week from a single low dose (10 mg/kg); this mAb effectively blocked disease and protected muscle endplates from destruction in a rat myasthenia model. Targeting C7 and C7-containing terminal pathway intermediates is an innovative therapeutic approach, allowing lower drug dose and lower product cost, that will facilitate the expansion of complement therapeutics to common diseases.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Journal: Functional Hemolytic Test for Complement Alternative Pathway Convertase Activity. (Pubmed Central) - Jun 23, 2021
Such factors include C3 nephritic factors, which are autoantibodies against the C3 convertase that prolong its activity, or genetic variants resulting in a stabilized convertase complex. This chapter describes a method in which the activity and stability of the alternative pathway convertases can be measured to detect aberrant serum factors causing convertase overactivity.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, Ultomiris IV (ravulizumab IV) / Alexion Pharma
Journal: Is ravulizumab the new treatment of choice for atypical hemolytic uremic syndrome (aHUS)? (Pubmed Central) - Jun 22, 2021
Rates of complete thrombotic microangiopathy response were similar to those observed in major eculizumab trials; however, fewer patients in the ravulizumab study were able to stop dialysis, probably due to differences in the study populations. Until additional data/analyses are available, eculizumab remains the drug of choice for an acute aHUS episode, whereas ravulizumab has several advantages in maintenance treatment.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Clinical, Review, Journal: Deposition of the Membrane Attack Complex in Healthy and Diseased Human Kidneys. (Pubmed Central) - Jun 22, 2021
These deleterious effects are, consequently, targeted in the development of novel therapies that inhibit the formation of C5b-9, such as eculizumab...We summarize how these deposits are related with other histological lesions and clinical characteristics. We evaluate the prognostic relevance of these deposits in the light of possible treatment with complement inhibitors.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, Ultomiris IV (ravulizumab IV) / Alexion Pharma
Review, Journal: Concept confirmation of the Treatment Administration Satisfaction Questionnaire (TASQ) in rare paroxysmal nocturnal hemoglobinuria. (Pubmed Central) - Jun 22, 2021
It provided us useful information to find out the differences between our patients and literature, which may help us understand the disease. Most participants understood the TASQ-IV and TASQ-SC instructions (n = 9 and 10, respectively) and the TASQ-IV items were considered clear, relevant and to have suitable response options, demonstrating face and content validity of the instruments for the clinical trial setting.
|
