- |||||||||| Soliris (eculizumab) / AstraZeneca, Nulojix (belatacept) / BMS
THROMBOTIC MICROANGIOPATHY AND KIDNEY TRANSPLANTATION (KT). A SINGLE CENTER EXPERIENCE (MON-259; Exhibition Hall and Main Foyer) - Mar 8, 2024 - Abstract #ISNWCN2024ISN_WCN_1557;
Switch to belatacept is safe in secondary TMA. Pre-transplant screening allows to identify risk mutations; lead prophylactic therapy and quickly introduce of eculizumab in unexpected relapse.
- |||||||||| Soliris (eculizumab) / AstraZeneca
GENETIC ANALYSIS IN DIARRHEA-ASSOCIATED STEC NEGATIVE HEMOLYTIC UREMIC SYNDROME (MON-192; Exhibition Hall and Main Foyer) - Mar 8, 2024 - Abstract #ISNWCN2024ISN_WCN_1292;
Genetic analysis in aHUS patients is important in understanding the underlying pathogenesis. Eculizumab is started empirically before availability of genetic results in view of suspicion aHUS diagnosis.
- |||||||||| Soliris (eculizumab) / AstraZeneca
PHENOTYPES OF PATIENTS WITH ATYPICAL HEMOLYTIC UREMIC SYNDROME (MON-186; Exhibition Hall and Main Foyer) - Mar 8, 2024 - Abstract #ISNWCN2024ISN_WCN_1286;
In addition, a trend toward better renal outcomes with eculizumab treatment was observed in patients with the classic phenotype 1, although this difference did not reach statistical significance. Despite the absence of typical symptoms in some patients, recognizing the differences in the progression of aHUS and certain phenotypic variants allows for early detection of the disease and timely initiation of therapy.
- |||||||||| Soliris (eculizumab) / AstraZeneca
A Case of Smoldering Atypical HUS Superimposed on Familial FSGS (MON-182; Exhibition Hall and Main Foyer) - Mar 8, 2024 - Abstract #ISNWCN2024ISN_WCN_1282;
This was likely a case of two very rare entities: familial FSGS with a superimposed smoldering aHUS and highlights the importance of recognizing concurrent renal pathologies as one may be amenable to treatment. Further studies are needed in this case to clarify the significance of the c.134C>T variant.
- |||||||||| Soliris (eculizumab) / AstraZeneca
ECULIZUMAB IN TYPICAL SHIGA TOXINASSOCIATED HUS: A SINGLE CENTER EXPERIENCE (SUN-196; Exhibition Hall and Main Foyer) - Mar 8, 2024 - Abstract #ISNWCN2024ISN_WCN_1274;
Apart from a single patient with liver toxicity, the drug was safe and well tolerated. However, a one-year comparison between the group of patients treated with Eculizumab and the group of patients treated with supportive therapy alone did not show significant differences in the outcome between the 2 groups.
- |||||||||| LP-005 / Longbio Pharma
BI-FUNCTIONAL C5 ANTIBODY-FUSION PROTEIN (LP-005) WITH POTENTIAL BEST-IN-CLASS BIOACTIVITY FOR COMPLEMENT INHIBITION (MON-100; Exhibition Hall and Main Foyer) - Mar 8, 2024 - Abstract #ISNWCN2024ISN_WCN_917;
In summary, LP-005 is a novel bifunctional anti-C5 monoclonal antibody fusion protein, with highest bioactivity in CP, AP, LP. LP-005 is also engineered to change it surface charge (PI) and FcRn binding, which together has the potential to be a bestin-class drug candidate with improved pharmacokinetic properties and strongest in-vitro and in-vivo bioactivity.
- |||||||||| Keytruda (pembrolizumab) / Merck (MSD)
ATYPICAL HEMOLYTIC SYNDROME INDUCED BY PEMBROLIZUMAB. MANAGEMENT AND OUTCOMES (SUN-005; Exhibition Hall and Main Foyer) - Mar 8, 2024 - Abstract #ISNWCN2024ISN_WCN_688;
Contrary to the prior reports of Pembrolizumab related MAHA and HLH, use of corticosteroids and PLEX was not effective presumably due to the different mechanisms of immune injury. Prompt response to Eculizumab with complete renal and hematologic recovery points to the novel pathway of how Pembrolizumab can affect the immune system on the level of complement regulatory proteins.
- |||||||||| Evidence Gap Analysis of the Burden of Illness and Treatment of Myasthenia Gravis () - Mar 8, 2024 - Abstract #ISPOR2024ISPOR_2036;
Besides traditional non-specific immunosuppressive agents, recently available biologics (e.g., eculizumab, ravulizumab, efgartigimod, rozanolixizumab, zilucoplan) are increasingly used in MG to target specific components of the immune pathway. We identified several gaps in the literature, including the need for comprehensive studies to advance our understanding of the epidemiology, humanistic and economic burden, as well as the evolving treatment pathways in MG.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Efficacy of Eculizumab in Acute Refractory Pediatric Neuromyelitis Optica: A Case Report (Colorado Convention Center | Exhibit Hall B-E) - Mar 8, 2024 - Abstract #AAN2024AAN_4608;
Eculizumab is a monoclonal antibody that inhibits complement-mediated cytotoxicity and was reported to mildly improve an adult with acute AQP4-IgG seropositive NMOSD failing IV methylprednisolone (IVMP) and PLEX. This case demonstrates the potential utility of eculizumab for the acute treatment of pediatric AQP4-IgG seropositive NMOSD relapses.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Eculizumab Therapy in a Seronegative Patient with NMOSD: A Case Report (Colorado Convention Center | Exhibit Hall B-E) - Mar 8, 2024 - Abstract #AAN2024AAN_4269;
If patients with seronegative NMOSD are also thought to have oligodendrocyte and myelin damage after the disruption of astrocytes, therapies aiming to prevent astrocyte dysfunction should work on this population as evidenced by the patient whose case we report. There is a need to include these patients in clinical studies to avoid treatment delays and further accumulation of disability.
- |||||||||| Uplizna (inebilizumab-cdon) / Mitsubishi Tanabe, Amgen, Soliris (eculizumab) / AstraZeneca, Enspryng (satralizumab) / Roche
Matching-adjusted Indirect Comparison of Current Treatments for NMOSD and Evaluation of Long-term Effectiveness (Colorado Convention Center | Exhibit Hall B-E) - Mar 8, 2024 - Abstract #AAN2024AAN_4266;
Additionally, treatment with inebilizumab yields better results for expected life years and quality adjusted life years compared to either satralizumab or eculizumab. Relative efficacy and discontinuation were the key drivers of the results.
- |||||||||| Soliris (eculizumab) / AstraZeneca, Ultomiris (ravulizumab-cwvz) / AstraZeneca
Patterns of Steroid Use and Outcomes in US Patients with Generalized Myasthenia Gravis (gMG) Receiving C5 Inhibitor Therapy (C5IT) (Colorado Convention Center | Exhibit Hall B-E) - Mar 8, 2024 - Abstract #AAN2024AAN_4251;
C5ITs eculizumab and ravulizumab are approved gMG treatments that employ targeted inhibition of terminal complement activation to reduce neuromuscular junction damage. These medical claims data showed statistically significant reductions in prednisone ADD after 12-months' C5IT and gMG exacerbations and crises during 6 months of C5IT treatment compared with 6 months before C5IT initiation.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Eculizumab Versus Rituxmab for Acetylcholine Receptor-positive Generalized Myasthenia Gravis: Single-center Experience (Colorado Convention Center | Exhibit Hall B-E) - Mar 8, 2024 - Abstract #AAN2024AAN_2848;
In addition to previous findings from CHAMPION MG, this retrospective data analysis from US clinical practice provides further evidence that eculizumab or ravulizumab treatment within 2 years of gMG diagnosis may offer a greater clinical benefit compared with later initiation. Out data provides real-world evidence supporting the use of eculizumab over rituximab for the treatment of refractory, anti-AChR-ab positive MG, as eculizumab led to rapid and sustained achievement of MM.
- |||||||||| Review, Journal: Advances in the treatment of IgA nephropathy with biological agents. (Pubmed Central) - Mar 7, 2024
There are four main categories of biological agents used to treat IgA nephropathy, specifically anti-CD20 monoclonal antibodies, anti-BLyS or APRIL monoclonal antibodies, monoclonal antibodies targeting both BLyS and APRIL (telitacicept and atacicept), and monoclonal antibodies inhibiting complement system activation (narsoplimab and eculizumab). However, further research on the dosages, treatment duration, long-term efficacy, and safety of these biological agents is required.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Journal: Lupus nephritis and associated thrombotic microangiopathy (Pubmed Central) - Mar 5, 2024
The co-occurrence of lupus nephritis and thrombotic microangiopathy is described to be rare but implies the risk of fatal organ dysfunction. We report three patients in whom these two disease entities occurred in parallel, necessitating intensive immunosuppressive therapy, including complement blockade.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Clinical, Journal, HEOR: Pediatric Transplant Associated Thrombotic Microangiopathy Healthcare Utilization and Implications of Eculizumab Therapy. (Pubmed Central) - Mar 4, 2024
However, after adjusting for GI bleeding which occurred disproportionately in the eculizumab (n=6) versus supportive care (n=0) cohort, eculizumab treatment was not associated with increased total costs. More studies are needed to determine the etiology of increased HCU costs in those with HR-TA-TMA and to predict those more likely to benefit from eculizumab, reducing HCU and improving outcomes.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Review, Journal: Recurrent C3 glomerulopathy after kidney transplantation. (Pubmed Central) - Feb 27, 2024
Its treatment is mainly based on immunosuppressive therapies, specifically mycophenolate mofetil and glucocorticoids...However, challenges persist in C3G, with recurrent post-transplantation cases leading to suboptimal outcomes. This review discusses the pathophysiology and management of C3G, with a focus on its recurrence after kidney transplantation.
- |||||||||| BC5 - Pharmacology and Clinical Application of Complement Inhibitors (Ballroom B-C) - Feb 27, 2024 - Abstract #HOPA2024HOPA_69;
This review discusses the pathophysiology and management of C3G, with a focus on its recurrence after kidney transplantation. The focus of this presentation will be on the clinical application of complement inhibitors
- |||||||||| Soliris (eculizumab) / AstraZeneca, Rituxan (rituximab) / Roche
Management of refractory APS (Chamber Music Room) - Feb 25, 2024 - Abstract #LUPUS2024LUPUS_111;
In patients with associated SLE, intravenous cyclophosphamide has demonstrated be beneficial. In refractory cases, rituximab or eculizumab should be added.
- |||||||||| Journal: Impact of neuromyelitis optica spectrum disorder on employment and income in the United States. (Pubmed Central) - Feb 20, 2024
Conclusion Treatment with the anti-C5 monoclonal antibody eculizumab which targets and inhibits terminal complement system activation reversed the deleterious cascade of events in this patient with sickle cell disease. We provide a structured analysis of the impact of NMOSD on employment, work hours, and income in the United States, demonstrating its major effect on the livelihoods of patients and their caregivers.
- |||||||||| Soliris (eculizumab) / AstraZeneca
PK/PD data, Journal: Eculizumab dose tapering should take into account the nonlinearity of its pharmacokinetics. (Pubmed Central) - Feb 20, 2024
We provide a structured analysis of the impact of NMOSD on employment, work hours, and income in the United States, demonstrating its major effect on the livelihoods of patients and their caregivers. Our study investigates the nonlinear elimination of eculizumab and discusses the importance of accounting for eculizumab target-mediated elimination in therapeutic drug monitoring.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Complement Mediated Hemolytic Uremic Syndrome in an In Vitro Fertilization Pregnancy (San Diego Convention Center, Room 31A-C (Upper Level)) - Feb 20, 2024 - Abstract #ATS2024ATS_7264;
In contrast, our patient had loss of CFI function, which accounts for a minority of pregnancy-associated CM-HUS cases, and presented early in the first trimester. Similar to cases of CM-HUS in unassisted pregnancy, our patient had a favorable response to eculizumab, suggesting similar treatment approached may be utilized in IVF pregnancies.
- |||||||||| Soliris (eculizumab) / AstraZeneca, Rituxan (rituximab) / Roche
Catastrophic Antiphospholipid Syndrome After Triple Coronary Artery Bypass Grafting (San Diego Convention Center, Area E (Hall A-B2, Ground Level)) - Feb 20, 2024 - Abstract #ATS2024ATS_3481;
Traditional treatments had little effect. While prior observational studies found that APLS patients can undergo cardiothoracic surgery without significantly worse outcomes, this case shows that post-surgical complications in APLS can be severe.
- |||||||||| Soliris (eculizumab) / AstraZeneca, Ultomiris (ravulizumab-cwvz) / AstraZeneca
A Case of Atypical Hemolytic Uremic Syndrome (San Diego Convention Center, Area E (Hall A-B2, Ground Level)) - Feb 20, 2024 - Abstract #ATS2024ATS_3478;
However, he showed significant improvement with discontinuation of ticagrelor and continued treatment with Ravulizumab...Ravulizumab, a C5 inhibitor, was re-engineered from Eculizumab to have a longer half-life...It is important to recognize the implications of aHUS. Early initiation of treatment with anti-complement therapy is crucial in patients with a high suspicion for aHUS as it can induce hematologic remission and stabilize kidney function.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Hypertensive Emergency Turned Atypical HUS (San Diego Convention Center, Area E (Hall A-B2, Ground Level)) - Feb 20, 2024 - Abstract #ATS2024ATS_3477;
Eculizumab has shown to reduce recurrence of symptoms in aHUS patients, with some able to come off of dialysis. An unanswered question remains: how long should aHUS patients be treated with Eculizumab?
- |||||||||| Valcyte (valganciclovir) / Roche, Mitsubishi Tanabe, Soliris (eculizumab) / AstraZeneca
Bilateral Lung Transplant Complicated by Cytokine Storm Syndrome Due to Hemophagocytic Lymphohistocytosis (San Diego Convention Center, Area E (Hall A-B2, Ground Level)) - Feb 20, 2024 - Abstract #ATS2024ATS_3467;
Valganciclovir prophylaxis was also initiated as both the donor and transplant recipient were seropositive for cytomegalovirus and Ebstein-Barr virus...Multiple therapeutic options (plasmapheresis, steroids, eculizumab) were used early on with improvement in laboratory abnormalities, but sIL-2R levels continued to rise, correlating with high disease activity...Despite early diagnostic suspicion, therapy implementation can be challenging given the multi-organ dysfunction. Competing situations, including candidemia, prevented optimal treatment for HLH with etoposide and high-dose steroids.
- |||||||||| Soliris (eculizumab) / AstraZeneca, Kineret (anakinra) / SOBI, Rituxan (rituximab) / Roche
Anti-MDA5 Antibody-associated Juvenile Dermatomyositis and Rapidly Progressive ILD: A Pediatric Case Report (San Diego Convention Center, Area J (Hall H, Ground Level)) - Feb 20, 2024 - Abstract #ATS2024ATS_1888;
Due to elevated CH50 levels and complement staining on lung biopsy tissue, Eculizumab was added...Our case emphasizes the importance of awareness of this rare variant of JDM, and the need for a high index of suspicion in patients with a rapidly deteriorating respiratory status, even without other typical features of JDM. Despite aggressive treatment, prognosis remains challenging.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Catastrophic Antiphospholipid Syndrome as a Unique Trigger for Rapidly Progressive Interstitial Lung Disease (San Diego Convention Center, Area A (Hall A-B2, Ground Level)) - Feb 20, 2024 - Abstract #ATS2024ATS_1545;
Case reports have described varying success with steroids, plasma exchange, or immunomodulators, specifically cyclophosphamide and eculizumab (3,4,5,6,7)...Her urine culture grew Klebsiella pneumoniae and she was started on vancomycin/piperacillin/tazobactam...She continued to decline despite being on VV-ECMO for 75 days and even after undergoing bilateral lung transplant. This case displays an uncommon pulmonary insult in the setting of CAPS and highlights the need for vigilant multidisciplinary approaches to prevent the cytokine storm from resulting in multi-organ failure.
- |||||||||| Idefirix (imlifidase) / Hansa Biopharma, Sarepta Therap
Trial completion date, Trial primary completion date: ConfIdeS: Renal Function in Highly Sensitized Patients 1 Year After Desensitization With Imlifidase Prior to DD Kidney Tx (clinicaltrials.gov) - Feb 15, 2024
P3, N=64, Recruiting,
This case displays an uncommon pulmonary insult in the setting of CAPS and highlights the need for vigilant multidisciplinary approaches to prevent the cytokine storm from resulting in multi-organ failure. Trial completion date: Dec 2023 --> Jun 2025 | Trial primary completion date: Dec 2023 --> Jun 2025
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