An electronically gathered patient reported outcomes (ePRO) system customized for paroxysmal nocturnal hemoglobinuria (PNH) and aplastic anemia (AA) and may be a feasible data collection and patient guidance solution in light of high adherence, satisfaction, and performance, according to an article published in the journal Annals of Hematology. For the purpose of this single-center study,…
A new paroxysmal nocturnal hemoglobinuria (PNH) registry analysis has found that, although some intergroup differences were observed, combined use of eculizumab and immunosuppressive therapy (IST) was safe and effective. The analysis, published in Acta Haemotologica, noted that the positive results were observed regardless of the treatment sequence. “Despite what is known about treating patients with…
Proximal complement inhibitors have emerged as a potential option for addressing current limitations of terminal complement inhibitors, as they could prevent both intra- and extravascular hemolysis and improve anemia in patients with paroxysmal nocturnal hemoglobinuria (PNH). In a review article recently published in Blood Reviews, a multinational panel of experts discussed the differences between the…
A case study of a patient with Crohn’s disease complicated by paroxysmal nocturnal hemoglobinuria (PNH) mimicking recurrent intestinal ischemia has been published in Gastroenterology Nursing. A 37-year-old man presented with a 6-month history of progressive, lower right quadrant abdominal pain. The patient revealed a 7-year history of PNH, of which recurrent abdominal pain and the…
A new study has further confirmed the benefits of ravulizumab over eculizumab in patients with paroxysmal nocturnal hemoglobinuria (PNH), but also found that progressive C3d binding to PNH red cells and the subsequent onset of extravascular hemolysis could still occur. The study, published in the British Journal of Haematology, suggests that the prevention of hemolysis…
Drug-target-drug complexes (DTDCs) can form in patients with paroxysmal nocturnal hemoglobinuria (PNH) who switch from eculizumab to crovalimab. However, it is possible to reduce the proportion of large DTDCs, ensure adequate complement inhibition, and improve safety by optimizing the dosing regimen of crovalimab, according to a new study published in Clinical Pharmacology & Therapeutics. COMPOSER…
Researchers reported on a patient with untreated aplastic anemia who presented with classical paroxysmal nocturnal hemoglobinuria (PNH). The case study was published in Cureus. A 29-year-old man presented with a weeklong history of generalized weakness and intermittent nausea. He was previously diagnosed with aplastic anemia via bone marrow biopsy. The only relevant medical history was…
A new study compared ravulizumab and eculizumab in terms of quality of life (QoL), the occurrence of breakthrough hemolysis (BTH), and cost-effectiveness in patients with paroxysmal nocturnal hemoglobinuria (PNH) who live in the Netherlands. The study, published in the European Journal of Health Economics, evaluated adult patients with symptoms of high disease activity or who…
Researchers from France observed an over-representation of CFH variants in patients with paroxysmal nocturnal hemoglobinuria (PNH) and found that patients with such variants were more likely to be transfusion-dependent when receiving eculizumab (Soliris®). Next-generation sequencing unveiled rare variants in CFH, C3, and CFI, in 10.7%, 4.8%, and 3.6% of PNH patients, respectively. CFH variants, but…
BCX10013, an oral factor D inhibitor for multiple complement-mediated diseases, led to a rapid, sustained, and effective suppression of the alternative pathway of the complement system in healthy volunteers. Given the positive results of the ongoing phase 1 study, BioCryst Pharmaceuticals plans to start evaluating once-daily BCX10013 treatment in patients with paroxysmal nocturnal hemoglobinuria (PNH)…
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