AI Article Synopsis

  • Paroxysmal nocturnal hemoglobinuria (PNH) is a serious blood disorder that involves the breakdown of red blood cells, causing severe symptoms.
  • Advances in drug treatments, particularly eculizumab and ravulizumab, have improved patient outlook, and the new complement inhibitor crovalimab shows promise for better results.
  • The editorial analyzes clinical trial data and emerging therapies, highlighting crovalimab's potential for more effective and convenient treatment options for a broader patient population.

Article Abstract

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, life-threatening hematologic disease that is characterized by the destruction of red blood cells, leading to a range of severe symptoms and complications. Recent advancements in drug therapies have significantly improved the prognosis for PNH patients. This editorial comprises the impact of PNH drugs, focusing on eculizumab and ravulizumab and comparing them to the recently approved complement inhibitor, crovalimab, which targets the complement system to prevent hemolysis. The discussion includes an analysis of clinical trial data and patient outcomes. The editorial mainly addresses emerging therapies, like crovalimab, that promise to offer more comprehensive, complete blockage of the complement system and low-dose solutions, reducing the treatment hassle while simultaneously appealing to a wider range of patients.

Download full-text PDF

Source
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC11623871PMC
http://dx.doi.org/10.1097/MS9.0000000000002706DOI Listing

Publication Analysis

Top Keywords

complement system
8
advancements pnh
4
pnh treatment
4
treatment crovalimab's
4
crovalimab's clinical
4
clinical efficacy
4
efficacy paroxysmal
4
paroxysmal nocturnal
4
nocturnal hemoglobinuria
4
hemoglobinuria pnh
4

Similar Publications

Background: Deprescribing is a critical component of clinical practice, especially in geriatric medicine. Nevertheless, the attributes of patients who are prepared for, interested in, and could potentially benefit from deprescribing have not been well examined. The Patient Perceptions of Deprescribing (PPoD) evaluates patients' overall readiness for deprescribing and is complemented by an 11-item validated short form (SF-PPoD).

View Article and Find Full Text PDF

The application of extracellular vesicles (EVs) as therapeutics or nanocarriers in cell-free therapies necessitates meticulous evaluations of different features, including their identity, bioactivity, batch-to-batch reproducibility, and stability. Given the inherent heterogeneity in EV preparations, this assessment demands sensitive functional assays to provide key quality control metrics, complementing established methods to ensure that EV preparations meet the required functionality and quality standards. Here, we introduce the detectEV assay, an enzymatic-based approach for assessing EV luminal cargo bioactivity and membrane integrity.

View Article and Find Full Text PDF

Background: Microscopic colitis (MC) is a chronic inflammatory condition of the colon, primarily characterized by watery diarrhea, with normal or near-normal endoscopic findings. It encompasses two main subtypes: lymphocytic colitis and collagenous colitis.

Objective: This position paper from the Brazilian Federation of Gastroenterology aims to review current evidence on the diagnosis and management of MC in Brazil, emphasizing the need for standardization across the country's healthcare systems.

View Article and Find Full Text PDF

In March 2023, the Society of Abdominal Radiology (SAR) Disease Focused Panel (DFP) on Cholangiocarcinoma (CCA) was formed. One of its initial tasks was for creation of a lexicon specific for CCA to complement the terms related to the Liver Imaging Reporting and Data System (LI-RADS) category M. A committee was formed and vetted 15 unique terms for CCA.

View Article and Find Full Text PDF

Background And Objectives: Extent of resection (EOR) is prognostic for meningioma outcomes. DNA methylation profiling can shed light on biological drivers and therapeutic vulnerabilities. The goal of this study was to re-evaluate the impact of EOR on clinical outcomes across meningioma DNA methylation groups.

View Article and Find Full Text PDF

Want 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!