News

The first clinical report of a patient with paroxysmal nocturnal hemoglobinuria (PNH) switching from PiaSky (crovalimab-akkz) to Fabhalta (iptacopan) was reported recently. The patient in the report was a 72-year-old man who continued to experience PNH symptoms after an infection triggered…

Antibody-based therapy ruxoprubart (formerly NM8074) safely prevented blood cell destruction, eased anemia, and eliminated the need for blood transfusions when given as a standalone treatment in adults with paroxysmal nocturnal hemoglobinuria (PNH). That’s according to results from a Phase 2 trial (NCT05646524) announced by its developer,…

Use of the approved therapy Ultomiris (ravulizumab) keeps hemolysis, or the destruction of red blood cells, under control, reduces the need for transfusions, and is generally well tolerated for people with paroxysmal nocturnal hemoglobinuria (PNH), a real-world study in Italy has found. According to the researchers, the benefits…

A new 13-item questionnaire designed to assess fatigue and its impact on daily functioning in individuals with chronic illnesses appears to be accurate and may be appropriate for use in adults with paroxysmal nocturnal hemoglobinuria (PNH). That’s according to a new analysis evaluating the validated tool, dubbed the Functional Assessment…

Mutations in the MUC4 gene may be linked to a higher risk of dangerous blood clots in people with paroxysmal nocturnal hemoglobinuria (PNH), a study has found. Researchers believe the mutations may be associated with increased activity in part of the immune system called the complement system. In mouse…

Ultomiris (ravulizumab-cwvz) may be safe and effective as a treatment for paroxysmal nocturnal hemoglobinuria (PNH) in pregnant women, a study suggested. Of 19 pregnancies in women treated with Ultomiris, all resulted in live births, compared with four live births out of eight pregnancies previously managed with Soliris…

Early recognition of paroxysmal nocturnal hemoglobinuria (PNH) in a 39-year-old woman with unexplained anemia led to treatment starting before any blood clot, or thrombotic, complications were detected, according to a case report from Portugal. The woman sought emergency care multiple times after several weeks of worsening fatigue and recurrent…

Improved access to standard-of-care treatments could significantly improve outcomes for people with paroxysmal nocturnal hemoglobinuria (PNH) living in South India, a study found. The researchers said poor outcomes, including frequent complications, repeated hospitalizations, and increased mortality, were largely driven by greater reliance on supportive care in the region. “Supportive…

People with paroxysmal nocturnal hemoglobinuria (PNH) have abnormalities in infection-fighting immune cells called natural killer (NK) cells, a study showed, shedding light on how this blood cell disorder may dysregulate the immune system. The study, “Role of NK cells in immune escape in patients with classical…

A healthy baby was born after exposure to Ultomiris (ravulizumab-cwvz) during the first trimester of pregnancy in a 25-year-old woman with paroxysmal nocturnal hemoglobinuria (PNH), according to a new case report. The woman was on Ultomiris when she was found to be pregnant. Because little is known about…