- |||||||||| Voydeya (danicopan) / AstraZeneca
Review, Journal: Danicopan: First Approval. (Pubmed Central) - Jun 20, 2024 Of those on prednisone therapy, the majority were able to reduce their prednisone dosage. Danicopan (Voydeya
- |||||||||| Soliris (eculizumab) / AstraZeneca
Journal: Eculizumab for Shiga-toxin-induced hemolytic uremic syndrome in adults with neurological involvement. (Pubmed Central) - Jun 19, 2024 Based on our experience and literature review, three prevailing themes were noted: 1) Early eculizumab administration optimized neurological outcomes, 2) Symptom resolution may not be immediate, neurological symptoms may initially worsen before improvement, and 3) Plasma exchange yielded no benefit. Early administration of eculizumab may reverse neurotoxicity in patients with STEC-HUS.
- |||||||||| Ultomiris (ravulizumab-cwvz) / AstraZeneca
Clinical, PK/PD data, Journal: Pharmacokinetics, pharmacodynamics, efficacy, and safety of ravulizumab in pediatric paroxysmal nocturnal hemoglobinuria. (Pubmed Central) - May 31, 2024 P3 Compared with baseline, ravulizumab improved and maintained efficacy outcomes in both groups. Ravulizumab had an acceptable safety profile with no new safety signals identified, and provided immediate, complete, and sustained terminal complement inhibition, translating to clinical benefit for pediatric patients with PNH.
- |||||||||| Soliris (eculizumab) / AstraZeneca, Rituxan (rituximab) / Roche
Review, Journal: Diagnosis and Management of Pediatric Neuropsychiatric Systemic Lupus Erythematosus: An Update. (Pubmed Central) - May 28, 2024 According to EULAR's current recommendation, strong immunosuppressants such as CYC and rituximab (RTX) should be preferred...Fingolimod, eculizumab, and JAK inhibitors are potential drugs in the pipeline. Developing targeted therapies will be possible by a better understanding of the pathological mechanisms.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Journal, Post-transplantation: "Eculizumab First" in the Management of Posttransplant Thrombotic Microangiopathy. (Pubmed Central) - May 20, 2024 This is, to our knowledge, the largest series of KT patients with PT-TMA treated with eculizumab. These data suggest that eculizumab is associated with a normalization of hemolysis indices and an important and progressive improvement of graft function.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Review, Journal: What is the role of complement in bystander hemolysis? Old concept, new insights. (Pubmed Central) - May 15, 2024 Intravascular hemolysis, results in activation of the alternative pathway, establishing a vicious cycle between complement activation and bystander hemolysis. C5 inhibitors have been used effectively in patients with hyperhemolysis syndrome and other entities characterized by bystander hemolysis.
- |||||||||| Enjaymo (sutimlimab) / Sanofi
Journal, HEOR, Cost-effectiveness, Cost effectiveness: Cost-effectiveness of sutimlimab in cold agglutinin disease. (Pubmed Central) - May 11, 2024 Exploratory threshold analyses showed that significant price reduction (>80%) or time-limited treatment (<18?months) followed by lifelong clinical remission off sutimlimab would allow sutimlimab to become cost-effective. The impact of sutimlimab on health system costs with longer term follow-up data merits future study and consideration through a distributional cost-effectiveness framework.
- |||||||||| Ultomiris (ravulizumab-cwvz) / AstraZeneca
Retrospective data, Review, Journal: Network Meta-analysis of Ravulizumab and Alternative Interventions for the Treatment of Neuromyelitis Optica Spectrum Disorder. (Pubmed Central) - May 9, 2024 This review aimed to summarize 71 registered clinical trials in the ClinicalTrials.gov database with the various treatment drugs, possible mechanisms, and novel findings related to PNH treatment. In the absence of head-to-head randomized controlled trials, NMA results suggest ravulizumab, a C5 inhibitor, is likely to be more effective in preventing NMOSD relapse in patients with AQP4-Ab+ NMOSD when compared with other treatments having different methods of action.
- |||||||||| Soliris (eculizumab) / AstraZeneca, Nulojix (belatacept) / BMS
The Outcomes of Early Thrombotic Microangiopathy in Kidney Transplantation (113-ABC, Level 1) - May 6, 2024 - Abstract #ATC2024ATC_2624; We present one of the largest data of early TMA with subsequent histological progression starting as early as 4 months, leading to reduced allograft function. Furthermore, there is a distinct decrease in both allograft and patient survival with long-term follow-up.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Does Crossing Donor-Specific Antibody at the Time of Heart Transplant Make Sense? (Poster Hall, Exhibit Hall A, Level 2) - May 6, 2024 - Abstract #ATC2024ATC_1566; Crossing DSA at the time of heart transplant can be done safely (with customized immunosuppression) but is it associated with an increased risk for first-year AMR. However, this did not impact 3-year survival or other outcomes.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Crossing Multiple Donor-Specific Antibodies at Heart Transplant: What is the Outcome? (Poster Hall, Exhibit Hall A, Level 2) - May 6, 2024 - Abstract #ATC2024ATC_1565; Crossing multiple DSA at the time of transplant does not appear to affect medium-term post-transplant outcome despite more first-year AMR. Specialized perioperative immunosuppression may be beneficial to mitigate immune risk.
- |||||||||| Actemra IV (tocilizumab) / Roche, JW Pharma
Tocilizumab for Acute Active Antibody Mediated Rejection in Kidney Transplants (Poster Hall, Exhibit Hall A, Level 2) - May 6, 2024 - Abstract #ATC2024ATC_1472; We report resolution of DSA and decline in dd-cfDNA with therapy in a significant proportion of patients. The incorporation of biomarkers like DSA, dd-cfDNA and MMDx into our study provides a framework for identifying patients that may benefit from this therapy, as well as, for designing controlled trials.
- |||||||||| Soliris (eculizumab) / AstraZeneca, Rituxan (rituximab) / Roche
Effect of Treatment of Kidney Transplant Rejection on Plasma Donor Derived-cfDNA and Molecular Activity in the Biopsy (Poster Hall, Exhibit Hall A, Level 2) - May 6, 2024 - Abstract #ATC2024ATC_532; P In contrast, there was no detectable effect of Rx for ABMR on molecular activity in the biopsy or dd-cfDNA in the blood, presumably because current Rx for ABMR are not effective. Nonetheless, in patients with ABMR, the correlation between dd-cfDNA and molecular ABMR activity in the biopsy suggests that dd-cfDNA could act as a surrogate for monitoring the success of Rx on ABMR in the kidney when new effective ABMR Rx emerge.
- |||||||||| Thymoglobulin (anti-thymocyte globulin (rabbit)) / Sanofi, Soliris (eculizumab) / AstraZeneca
Early Clinical Outcomes of 2-Dose Eculizumab for Blood Group Incompatible Living Donor Liver Transplantation (Poster Hall, Exhibit Hall A, Level 2) - May 6, 2024 - Abstract #ATC2024ATC_378; Patients were also maintained on mycophenolate and prednisone tapered to 10 mg by month 1...Indefinite aspirin and 6-month low molecular weight heparin were used for thromboprophylaxis.* There were 2 patients who followed this immunosuppression protocol... Blood group incompatibility in LDLT can be overcome using 2D-ECU, despite concerns of early rejection.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Journal: Novel factors potentially initiating acute antibody-mediated rejection in pig kidney xenografts despite an efficient immunosuppressive regimen. (Pubmed Central) - Apr 26, 2024 This observation suggests that proteinuria may be associated with the loss of any therapeutic monoclonal antibody, for example, anti-CD154 or eculizumab, in the urine, resulting in xenograft rejection...The association of a urinary infection with graft rejection has been well-documented in ABO-incompatible kidney allotransplantation based on the expression of an antigen on the invading microorganism shared with the kidney graft, generating an immune response to the graft. To our knowledge, these potential initiating factors of AMR in pig xenografts have not been highlighted previously.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Journal: Assessment of epidemiology and outcomes of adult patients with kidney-limited thrombotic microangiopathies. (Pubmed Central) - Apr 22, 2024 RL-TMA resulted from virtually all identified causes, more frequently from anti-VEGF treatment and hematological malignancies but less frequently from shigatoxin-associated hemolytic uremic syndrome (HUS), systemic sclerosis, gemcitabine and bacterial infection, and even less frequently when three or more causes/triggers were combined (RL-TMA: 5%; RH-TMA: 12%)...Among the 69 patients with proven complement-mediated aHUS, eculizumab (anti-C5 therapy) was used in 43 (62%) (RL-TMA: 35%; RH-TMA: 71%)...Adverse outcomes of RL-TMA are lower compared to RH-TMA, but remain significant. Anti-C5 therapy was rarely used in RL-TMA, even in proven complement-mediated aHUS, and its effects remain to be assessed.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Review, Journal: A Case-Based Narrative Review of Pregnancy-Associated Atypical Hemolytic Uremic Syndrome/Complement-Mediated Thrombotic Microangiopathy. (Pubmed Central) - Apr 22, 2024 The first case involves a 30-year-old woman presenting in the first trimester who was diagnosed with aHUS/CM-TMA and treated with eculizumab from 19 weeks' gestation...Her condition improved with supportive management, yet investigations assessing for aHUS/CM-TMA remained abnormal 6 months postpartum consistent with persistent complement activation but negative genetic testing. Through detailed case discussion describing tests assessing for placental health, fetal anatomy, complement activation, autoantibodies to complement regulatory proteins, and genetic testing for aHUS/CM-TMA, we describe how these results aided in the clinical diagnosis of pregnancy-associated aHUS/CM-TMA and assisted in guiding patient management including the use of anti-complement therapy.
- |||||||||| Soliris (eculizumab) / AstraZeneca
A Challenging case of SLE associated with Neuromyelitis optica: a promising therapeutic approach with eculizumab (Poster Zone) - Apr 21, 2024 - Abstract #EAACI2024EAACI_2197; In June 2022, with the onset of new neurological symptoms, the patient underwent rituximab therapy with partial benefit...Aggressive treatment is essential to minimize the risk of relapse and prevent disability. Although it is premature to draw a definitive conclusion in our specific case, eculizumab, a monoclonal antibody targeting the complement protein C5 and inhibiting the formation of the terminal complement complex, may serve as a viable long-term preventive immunotherapy option.
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