Publications by authors named "R Liesner"
Introduction: The B-Natural study is a multicentre, multinational, observational study of haemophilia B (HB) designed to increase understanding of clinical manifestations, treatment and quality of life (QoL).
Aim: To characterise and compare QoL in HB across disease severity groups and individuals with inhibitors to identify gaps in treatment.
Methods: A total of 224 individuals from 107 families were enrolled from a total of 24 centres in North America (n = 16), Europe (n = 7) and Asia (n = 1).
Article Synopsis
- The International Immune Tolerance Study found that both high and low doses of immune tolerance induction (ITI) were equally effective in good risk patients, although the trial was halted due to excessive bleeding in the low dose group.
- The UKHCDO used available ITI and emicizumab (Hemlibra®) efficacy data to create new guidelines for ITI in the UK.
- The updated guidance recommends using emicizumab to lower bleeding rates, allowing for lower doses and less frequent administration of FVIII CFC in most children with hemophilia.
Background: Coagadex is a high-purity plasma-derived factor X concentrate (pdFX) developed to treat hereditary factor X deficiency (FXD).
Objective: Evaluate the efficacy and safety of pdFX administered to patients with hereditary FXD.
Methods: This was an open-label, multicenter, retrospective analysis of patients receiving pdFX for compassionate use.
PUPs B-LONG evaluated the safety and efficacy of recombinant factor IX Fc fusion protein (rFIXFc) in previously untreated patients (PUPs) with hemophilia B. In this open-label, phase 3 study, male PUPs (age <18 years) with hemophilia B (≤2 IU/dL of endogenous factor IX [FIX]) were to receive treatment with rFIXFc. Primary end point was occurrence of inhibitor development, with a secondary end point of annualized bleed rate (ABR).
View Article and Find Full Text PDFIntroduction: Inhibitors develop less frequently in haemophilia B (HB) than haemophilia A (HA). However, when present, the success of tolerization by immune tolerance induction (ITI) therapy is lower and the risk of complications higher.
Aim: To evaluate the use and outcome of ITI in patients with HB and inhibitors.