Complement C5 inhibitors bring an increased risk for Neisseria infections. A novel complement C3 inhibitor, pegcetacoplan, was recently approved to treat paroxysmal nocturnal hemoglobinuria, a condition commonly treated with complement C5 inhibitors. We present a case of meningococcal sepsis in a pegcetacoplan-treated patient with aplastic anemia and paroxysmal nocturnal hemoglobinuria.
View Article and Find Full Text PDFBackground: The impact of nonneutralizing antibodies (NNAs) in moderate hemophilia is elusive.
Objectives: To explore the presence of NNAs in Nordic persons with moderate hemophilia A (MHA) and B (MHB) in relation to treatment modality, clinical outcome, history of inhibitor, and the corresponding factor VIII (FVIII)/factor IX (FIX) gene mutation.
Methods: A cross-sectional multicenter study covering persons with MHA and MHB in Sweden, Finland, and Norway.
Introduction: The impact of moderate haemophilia on health-related quality of life (HRQoL) and physical activity (PA) is not well known. In previous studies, persons with factor VIII/factor IX activity (FVIII/FIX:C) below 3 IU/dL were associated with a more severe bleeding phenotype than predicted.
Aim: To explore HRQoL and PA in patients with moderate haemophilia A (MHA) and B (MHB).
Introduction: Good health-related quality of life (HRQoL) is an important goal in the treatment of persons with haemophilia B (PwHB). Studies focusing on this population are limited, however, and data are insufficient.
Aim: To assess the HRQoL in PwHB and to compare this to data on persons with haemophilia A (PwHA), as well as to evaluate the impact of joint health on HRQoL and to identify areas of insufficient care.