Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.1002/pbc.31649 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Pegcetacoplan for Breakthrough Hemolysis in an Adolescent With Classical Paroxysmal Nocturnal Hemoglobinuria on Eculizumab.
Pediatr Blood Cancer
March 2025
BC Children's Hospital, Vancouver, British Columbia, Canada.
Real-world evidence of pegcetacoplan in patients with paroxysmal nocturnal haemoglobinuria: A nationwide Italian study.
Br J Haematol
February 2025
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
In this study, we collected real-world evidence on the use of pegcetacoplan among 22 Italian patients with paroxysmal nocturnal haemoglobinuria showing suboptimal response to anti-C5 treatments eculizumab and ravulizumab. Most patients exhibited a complete or good response as per the criteria of the European Bone Marrow Transplant group (Risitano et al. Front Immunol 2019) and median haemoglobin improvement from baseline was +3.
View Article and Find Full Text PDFExamining Consistency Across NICE Single Technology Appraisals: A Review of Appraisals for Paroxysmal Nocturnal Haemoglobinuria.
Pharmacoeconomics
February 2025
School of Social Policy, University of Birmingham, Birmingham, B15 2TT, UK.
In 2024, the National Institute for Health and Care Excellence (NICE) recommended two new health technologies for paroxysmal nocturnal haemoglobinuria. This review systematically compares the clinical and cost-effectiveness evidence considered within the NICE single technology appraisals of iptacopan, danicopan and pegcetacoplan, examines the consistency of the clinical evidence and economic modelling, and considers whether single technology appraisals are a suitable apparatus for consistent decision making. The studies used different follow-up lengths and used different definitions for reporting breakthrough haemolysis (BTH), but otherwise reported similar outcomes and found a significant benefit for their interventions.
View Article and Find Full Text PDF[New treatment strategies for paroxysmal nocturnal hemoglobinuria: the guideline update and novel complement-targeting drugs].
Rinsho Ketsueki
November 2024
Department of Hematology and Oncology, Graduate School of Medicine, Osaka University.
Article Synopsis
- The 2022 update of Japanese guidelines for paroxysmal nocturnal hemoglobinuria (PNH) introduced improved diagnostic criteria and a new disease severity classification, enhancing clinical management.
- It included 10 key clinical questions to guide treatment strategies and patient considerations, helping healthcare providers navigate PNH management more effectively.
- New therapies, such as pegcetacoplan and danicopan, were approved to address PNH, with a focus not just on hemolysis but also on managing residual anemia and fatigue, influenced by real-world evidence.
Dry and neovascular "wet" age-related macular degeneration: Upcoming therapies.
Indian J Ophthalmol
January 2025
Department of Ophthalmology, University of Pittsburgh School of Medicine, Pittsburg, PA, USA.
The age-related macular degeneration (AMD) field is witnessing promising advancements in therapeutic options. Breakthrough drugs such as pegcetacoplan and avacincaptad have been FDA-approved for dry AMD, marking a significant development as there were no treatment options until August 2023. While several antivascular endothelial growth factor (VEGF) inhibitors have been approved for wet AMD, challenges persist with the need for frequent dosing.
View Article and Find Full Text PDFWant AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!