- |||||||||| Empaveli (pegcetacoplan SC) / Apellis, SOBI
MOVING TOWARD INDIVIDUAL TREATMENT GOALS WITH PEGCETACOPLAN IN PATIENTS WITH PNH AND BONE MARROW FAILURE (CLYDE) - Feb 14, 2024 - Abstract #EBMT2024EBMT_2020;
P3
Nevertheless, patients with associated BMF syndromes achieved clinically meaningful improvements in haematologic and clinical outcomes in response to pegcetacoplan treatment in the PEGASUS and PRINCE studies. Establishing individualised treatment goals may be a more appropriate and beneficial approach for evaluating PNH-targeted treatment success in the future
- |||||||||| Empaveli (pegcetacoplan SC) / Apellis, SOBI
MOVING TOWARD INDIVIDUAL TREATMENT GOALS WITH PEGCETACOPLAN IN PATIENTS WITH PNH AND BONE MARROW FAILURE (ePoster area) - Feb 14, 2024 - Abstract #EBMT2024EBMT_2019;
P3
Nevertheless, patients with associated BMF syndromes achieved clinically meaningful improvements in haematologic and clinical outcomes in response to pegcetacoplan treatment in the PEGASUS and PRINCE studies. Establishing individualised treatment goals may be a more appropriate and beneficial approach for evaluating PNH-targeted treatment success in the future
- |||||||||| Fabhalta (iptacopan) / Novartis
COMPARATIVE EFFECTIVENESS OF IPTACOPAN VERSUS C5 INHIBITORS IN COMPLEMENT INHIBITOR-NA (ePoster area) - Feb 14, 2024 - Abstract #EBMT2024EBMT_1168;
P, P3
Limitations of APPEX include the irregular frequency of measurements in real?world clinical practice, which differs from the scheduled measurement regime in APPOINT-PNH. There was no adjustment for regional differences, with a larger proportion of Asian patients enrolled in APPOINT-PNH compared with APPEX, in which patients resided in Europe.
- |||||||||| Empaveli (pegcetacoplan SC) / Apellis, SOBI
Journal, Real-world evidence, Real-world: Real-world experience of pegcetacoplan in paroxysmal nocturnal hemoglobinuria. (Pubmed Central) - Feb 13, 2024
In this large patient cohort, treatment was well tolerated with improved hemoglobin and reticulocytes and maintained LDH control. Although BTH occurs, this is manageable by acute dose modification, with the majority of patients being maintained on pegcetacoplan.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Preclinical, Journal: Ex (Pubmed Central) - Feb 5, 2024
Active, not recruiting --> Completed Early diagnosis and/or prediction of aHUS (relapse) is pivotal in aHUS kidney transplant recipients who do not receive eculizumab prophylaxis...Our data question the routine use of the ex
- |||||||||| Uplizna (inebilizumab) / Mitsubishi Tanabe, Amgen, Soliris (eculizumab) / AstraZeneca, Enspryng (satralizumab) / Roche
Review, Journal: Advances in the long-term treatment of neuromyelitis optica spectrum disorder. (Pubmed Central) - Feb 5, 2024
We will also discuss the extension trials of each of these medications and what lead to their approval in AQP4-IgG seropositive NMOSD patients. We will then examine treatments in the pipeline for adult and pediatric NMOSD patients and conclude with discussions on treatment considerations in pregnant patients and how to approach treatment of NMOSD patients during COVID.
- |||||||||| Soliris (eculizumab) / AstraZeneca, Rituxan (rituximab) / Biogen, Zenyaku Holdings, Roche
Journal: A Case of Catastrophic Antiphospholipid Syndrome with Acute Multiorgan Thrombosis and (Pubmed Central) - Feb 1, 2024
Recently, biologics such as rituximab and eculizumab have also shown promise as potential new therapies for CAPS, as observed in our case...While the exact etiology of CAPS remained unknown, it was likely precipitated by her warfarin discontinuation and confirmed Haemophilus influenzae infection...CONCLUSIONS This case highlights the importance of early recognition and diagnosis of catastrophic antiphospholipid syndrome, a true rheumatological emergency that requires aggressive treatment to prevent irreversible complications. Our patient's presentation and response to treatment also underscores the complexity of managing CAPS and the use of newer biological therapies in refractory cases.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Observational data, Retrospective data, Journal: Rational use of eculizumab in secondary atypical hemolytic uremic syndrome. (Pubmed Central) - Jan 30, 2024
Rates of hematological remission were similar in both groups (90.9% in the eculizumab cohort and 85.7% in the control cohort). Early and short-term use of eculizumab in patients with secondary aHUS could be an effective and safe therapeutic option, assuring better renal recovery compared to patients who do not receive complement blockade.
- |||||||||| Review, Journal: Myasthenia Gravis Treatment: From Old Drugs to Innovative Therapies with a Glimpse into the Future. (Pubmed Central) - Jan 29, 2024
The aim of this review is to provide a detailed overview of the pre- and post-marketing evidence on the five pharmacological treatments most recently approved for the treatment of MG, by identifying both preclinical and clinical studies registered in clinicaltrials.gov. A description of the molecules currently under evaluation for the treatment of MG is also provided.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Review, Journal: The Role of the Complement System in the Pathogenesis of Infectious Forms of Hemolytic Uremic Syndrome. (Pubmed Central) - Jan 27, 2024
This review presents summarized data from recent studies, suggesting that complement dysregulation is a key pathogenetic factor in various types of infection-induced HUS. Separate links in the complement system are considered, the damage of which during bacterial and viral infections can lead to complement hyperactivation following by microvascular endothelial injury and development of acute renal failure.
- |||||||||| Soliris (eculizumab) / AstraZeneca
IMMUNE RELATED RESPONSE CAUSING ASD OCCLUDER DEVICE RETRIEVAL (Hall B4-5) - Jan 26, 2024 - Abstract #ACC2024ACC_4357;
Separate links in the complement system are considered, the damage of which during bacterial and viral infections can lead to complement hyperactivation following by microvascular endothelial injury and development of acute renal failure. This case illustrates the importance of considering unusual complication of cardiac devices even though the procedure itself is very safe.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Preclinical, Journal: In (Pubmed Central) - Jan 25, 2024
Our data suggest that complement activation induced by rare blocking anti-?? isoantibodies may lead to the formation of a MAC with subsequent pore formation, resulting in calcium influx and procoagulant platelet phenotype.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Journal: Thrombotic microangiopathy - the importance of a multidisciplinary approach. (Pubmed Central) - Jan 22, 2024
TMA and coexisting MG represents a heterogeneous disease spectrum, including a small subset of patients who may present with complement dysregulation. Establishment of a TMA-Team is valuable in developing the necessary institutional expertise needed to promptly recognize and appropriately manage patients with TMA.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Liver transplantation in atypical Hemolytic uremic syndrome (aHUS - Complement Factor H mutation (New Hall) - Jan 20, 2024 - Abstract #APASL2024APASL_2661;
Establishment of a TMA-Team is valuable in developing the necessary institutional expertise needed to promptly recognize and appropriately manage patients with TMA. Liver Transplant is safe and effective treatment modality for aHUS and may prevent the need of simultaneous renal transplant if done before development of renal failure.
- |||||||||| Keytruda (pembrolizumab) / Merck (MSD), Soliris (eculizumab) / AstraZeneca, Ultomiris (ravulizumab-cwvz) / AstraZeneca
Journal: Novel uses of complement inhibitors in myasthenia gravis-Two case reports. (Pubmed Central) - Jan 11, 2024
The effect of complement inhibition on cancer outcomes of ICI therapy is unknown. A possible biologic basis for complement inhibitors in reducing irAEs of ICI, especially in the presence of underlying autoimmune disease, merits evaluation.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Rapid complement mediated TMA diagnosis and early intervention in a renal intensive care unit using Nanopore technology (MTCC - Exhibit Halls DE) - Jan 10, 2024 - Abstract #ACMG2024ACMG_630;
Eculizumab infusion (anti-C5 antibody) was started at day 3, after demonstration of ACMG class 5 pathogenic variant in CFH gene, impairing complement regulation, therefore classified the patients as affected by c-TMA. Rapid nanopore sequencing with adaptive sampling can provide valuable insights for TMA managment improving patient diagnosis, with optimal and early therapeutic intervention in the first week of admission.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Eculizumab as Rescue Therapy for Severe Acute Antibody Mediated Rejection in Heart Transplant Recipients (Board #325; Poster Hall) - Jan 9, 2024 - Abstract #ISHLT2024ISHLT_1917;
After multiple rounds of treatment for ACR/AMR, the patient was started on ECU with improvement in graft function.Summary Plasmapheresis and IVIG followed by ECU demonstrated a decrease in DSAs and clinical stabilization of graft function. Further studies are warranted to define ECU's place in therapy for AMR treatment in HT.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Successful Multi-Organ Transplant in Highly Sensitized Patients with Positive Crossmatch Donor (Board #295; Poster Hall) - Jan 9, 2024 - Abstract #ISHLT2024ISHLT_1789;
Both patients continued to achieve negative DSAs on weekly analysis.Summary Multiorgan Tx can be successfully performed in HSPs with a positive crossmatch donor. Our institution's protocol using PLEX, IVIG, Eculizumab, and ATG has the potential to be safe and effective for multi-organ Txs in HSPs.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Does Crossing DSA and Its Binding Levels at the Time of Heart Transplant Impact Post-Transplant Outcomes? (Board #41; Poster Hall) - Jan 9, 2024 - Abstract #ISHLT2024ISHLT_1267;
The high binding level (> 10K MFI) groups compared to the lower DSA level groups receive more eculizumab perioperatively and had lower freedom from 1-year AMR but 3-year outcome were similar.Conclusion 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
Post-Reperfusion Complement Deposition Predicts Primary Graft Dysfunction Following Lung Transplantation (Board #138; Poster Hall) - Jan 9, 2024 - Abstract #ISHLT2024ISHLT_1108;
In multivariate logistic regression, only positive C4d staining remained highly predictive of PGD-3 (OR 7.92, 95% CI 2.97-21.1, p<0.001).Conclusion C4d staining on intra-operative lung transplant biopsy predicts patients at high risk for PGD. Using intra-operative lung biopsy C4d staining as an early clinical marker for PGD would allow earlier intervention with complement inhibitors, such as eculizumab, which could improve PGD outcomes.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Novel Treatment Protocol with Perioperative Plasmapheresis and Eculizumab for Highly Sensitized Lung Transplant Patients (Board #130; Poster Hall) - Jan 9, 2024 - Abstract #ISHLT2024ISHLT_1101;
The incidence of PGD was similar in HS patients with and without plasmapheresis and eculizumab (66.7% vs 58.6%, p=1.00 in any grade, and 33.3% vs 10.3%, p=0.13 in grade3). The postoperative infection rate was also not different between HS patients with and without plasmapheresis and eculizumab (77.8% vs 72.4%, p=1.00).Conclusion This study highlights the potential benefits of perioperative plasmapheresis and eculizumab as a novel treatment protocol for highly sensitized patients undergoing lung transplantation.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Clinical, PK/PD data, Journal: [Translated article] Pharmacokinetics of eculizumab in adult and pediatric patients with atypical hemolytic uremic syndrome and C3 glomerulopathy. (Pubmed Central) - Jan 9, 2024
The postoperative infection rate was also not different between HS patients with and without plasmapheresis and eculizumab (77.8% vs 72.4%, p=1.00).Conclusion This study highlights the potential benefits of perioperative plasmapheresis and eculizumab as a novel treatment protocol for highly sensitized patients undergoing lung transplantation. The present study reports supratherapeutic concentrations of eculizumab in patients with aHUS, and defines higher concentrations than those described in the data sheet to achieve blockade, thus encouraging the personalization of treatment with eculizumab.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Clinical, PK/PD data, Journal: Pharmacokinetics of eculizumab in adult and pediatric patients with atypical hemolytic uremic syndrome and C3 glomerulopathy. (Pubmed Central) - Jan 9, 2024
The present study reports supratherapeutic concentrations of eculizumab in patients with aHUS, and defines higher concentrations than those described in the data sheet to achieve blockade, thus encouraging the personalization of treatment with eculizumab. The present study reports supratherapeutic concentrations of eculizumab in patients with aHUS, and defines higher concentrations than those described in the data sheet to achieve blockade, thus encouraging the personalization of treatment with eculizumab.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Journal: Eculizumab for a catastrophic relapse in NMOSD: case report. (Pubmed Central) - Jan 3, 2024
She experienced a life-threatening relapse that did not respond to conventional treatment, but ultimately showed a positive response to eculizumab. Furthermore, we describe the role of sNfL.
- |||||||||| Review, Journal, IO biomarker: Guideline for the management of myasthenic syndromes. (Pubmed Central) - Dec 28, 2023
Glucocorticosteroids and the classical immunosuppressants (primarily azathioprine) are the basic immunotherapeutics to treat mild/moderate to (highly) active generalized MG/young MG and ocular MG...In (highly) active generalized MG, complement inhibitors (currently eculizumab and ravulizumab) or neonatal Fc receptor modulators (currently efgartigimod) are recommended for AChR-Ab-positive status and rituximab for muscle-specific receptor tyrosine kinase (MuSK)-Ab-positive status...Specific aspects of ocular, juvenile, and congenital myasthenia are highlighted. The guideline will be further developed based on new study results for other immunomodulators and biomarkers that aid the accurate measurement of disease activity.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Journal, IO biomarker: CNS Inflammation as the First Sign of Complement Factor I Deficiency. (Pubmed Central) - Dec 26, 2023
To halt the uncontrolled complement-mediated inflammation associated with CFI deficiency, prompt targeted blockade of the complement pathway using eculizumab may be life changing in the acute phase. Long-lasting blockade of the complement pathway is also essential to prevent relapse in this subgroup of patients.
- |||||||||| Soliris (eculizumab) / AstraZeneca, Nulojix (belatacept) / BMS
Review, Journal: Beyond Immunosuppression: The Intricate Relationship Between Tacrolimus and Microangiopathy. (Pubmed Central) - Dec 26, 2023
Tacrolimus, widely known as Prograf, has become the preferred immunosuppressant for preventing graft rejection in solid organ transplant recipients, particularly in steroid-sparing regimens. Its efficacy and reduced risk of acute and chronic rejection compared to cyclosporine have made it the preferred treatment option
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