Treatment with the approved oral med Fabhalta (iptacopan) helped maintain control of paroxysmal nocturnal hemoglobinuria (PNH) without any sudden, renewed destruction of red blood cells or new blood clots for five people in the U.S. with the rare disease — even during infections and while undergoing minimally invasive…
Discussion
A woman with paroxysmal nocturnal hemoglobinuria (PNH) developed severe breakthrough hemolysis — when red blood cell destruction resumes despite treatment to prevent the process — while preparing for a stem cell transplant. A case report said the woman, who was being treated with the complement inhibitor Empaveli (pegcetacoplan),…
Discussion
In a woman with paroxysmal nocturnal hemoglobinuria (PNH) who developed acute kidney damage and low numbers of red blood cells and platelets during a COVID-19 infection, doctors suspected another disease called complement-mediated thrombotic microangiopathy (TMA). Her condition rapidly improved after treatment with Soliris (eculizumab). “Viral insults can serve…
Discussion
Seven people with paroxysmal nocturnal hemoglobinuria (PNH) in Kenya went months to years before receiving a diagnosis, according to a case series highlighting the challenges of recognizing and treating the rare, acquired blood disorder in resource-limited settings. All seven had recurrent anemia, or a shortage of healthy red blood…
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