Individualized dosing of Empaveli (pegcetacoplan) may allow for a larger interval between doses in about 30% of adults with paroxysmal nocturnal hemoglobinuria (PNH), while cutting costs by about 8%, according to a study that simulated a clinical trial. The study, “Development of a target concentration intervention to…
An independent appraisal committee voted that there was adequate evidence to show that adding danicopan to a C5 inhibitor may benefit certain people with paroxysmal nocturnal hemoglobinuria (PNH), pending price listing. Committee members also decided the evidence isn’t adequate to show a net health benefit for Fabhalta (iptacopan)…
People in Japan using C5 inhibitors to treat their paroxysmal nocturnal hemoglobinuria (PNH) continue to experience symptoms like fatigue and shortness of breath, even if their hemoglobin levels surpass the median of real-world PNH populations, a survey study reports. “This study contributes to understanding the unmet needs of the…
Almost 6 out of 10 people with paroxysmal nocturnal hemoglobinuria (PNH) living in the U.S. may be being given a higher initial loading dose of Ultomiris (ravulizumab) than is recommended on the medication’s label, a database study into its clinical use reports. Researchers also observed that maintenance doses,…
Danicopan, an experimental oral medication, continued to help control anemia when taken for up to 48 weeks by adults with paroxysmal nocturnal hemoglobinuria (PNH) who experience a breakdown of red blood cells outside of blood vessels — called extravascular hemolysis — despite being on a C5 inhibitor. That’s according…
This year, PNH News brought up-to-date coverage of the latest scientific and clinical research developments in paroxysmal nocturnal hemoglobinuria (PNH). Here are the 10 most-read news stories of 2023, along with a brief description of what made them interesting to our readers. We look forward to continuing to…
Taking Novartis’ Fabhalta (iptacopan) as a monotherapy — meaning as a sole treatment — twice daily for almost one year continued to sustain hemoglobin at near-normal levels, generally without the need for blood transfusions, in adults with paroxysmal nocturnal hemoglobinuria (PNH). These new data come from an open-label…
An enzyme called lactate dehydrogenase (LDH), which is a marker of red blood cell breakdown, may be used as a proxy measure for fatigue and quality of life in people with paroxysmal nocturnal hemoglobinuria (PNH) who are being treated with Ultomiris (ravulizumab) or Soliris (eculizumab). That’s according…
People with paroxysmal nocturnal hemoglobinuria (PNH) who had a prior blood clot-related event are more likely to have such events again, regardless of whether they recently used blood thinners, a real-life registry study found. Other factors that appeared to increase their likelihood in PNH patients not on Soliris…
Unusual colon lesions caused by ischemic colitis led to the diagnosis of paroxysmal nocturnal hemoglobinuria (PNH) in an 82-year-old woman in Japan, but appropriate treatment eased her symptoms. According to the authors, her case highlights the importance of considering underlying diseases when unusual features are observed in a person…
Get regular updates delivered to your inbox.