Study to Test the Safety of an Investigational Drug Given Repeatedly to Adult Men With Severe Hemophilia
Condition: Hemophilia Intervention: Drug: BAY1093884 Sponsor: Bayer Not yet recruiting
Authors: Morfini M, Farrugia A Abstract Introduction: Plenty of new FVIII/IX concentrates have been developed and entered the market of hemophilia treatment. Others are going to end the long/demanding procedures for approval. Changes of the FVIII molecule (single chain), pegylation of B-domain deleted FVIII, and fusion with Fc succeeded to improve the FVIII half-life, about 4 hours. Pegylation and fusion with albumin or Fc of rFIX caused a substantial increase of half-life, approximately 3-4 times that of FIX standard concentrates. Area covered: Extended Half-life concentrates may allow a longer time interval betwe...
Conditions: Moderate Hemophilia; Arthropathy Intervention: Other: Gait analysis Sponsor: Nantes University Hospital Not yet recruiting
Condition: Hemophilia A Interventions: Biological: Nuwiq; Biological: Octanate; Biological: Wilate; Biological: Emicizumab; Biological: Recombinant factor VIIa (rFVIIa); Biological: Activated prothrombin complex concentrate (aPCC) Sponsors: Emory University; Octapharma Not yet recruiting
ConclusionsThis study provides rich insights into the experiences of patients with EHL products and the value of reduced infusion frequency. Such data could be of value to a range of stakeholders (e.g. regulators, payers) and facilitate patient –clinician discussions to promote tailored treatment decisions.
The article Routine clinical care data for population pharmacokinetic modeling: the case for Fanhdi/Alphanate in hemophilia A patients, written by Pierre Chelle, Cindy H. T. Yeung, Santiago Bonanad, Juan Crist óbal Morales Muñoz, Margareth C. Ozelo, Juan Eduardo Megías Vericat, Alfonso Iorio, Jeffrey Spears, Roser Mir, Andrea Edginton, was originally published electronically on the publisher's internet portal (currently SpringerLink) on 21 May 2019 without open access.
Achieving the goal of zero bleeds as patients pursue active lives will require divergent dosing, according to PROPEL trial investigators who evaluated whether more exposure to FVIII improves outcomes.Medscape Medical News
AbstractHaemophilia A (HA) is caused by a lack or reduced amount of factor VIII protein (FVIII). About one-third of patients with non-severe HA carrying specific missense mutations show discrepant results between FVIII activity (FVIII:C), measured by one-stage or chromogenic two-stage assays. The aim of this study was to elucidate the mechanism underlying the assay discrepancy in vitro and in silico. Thirteen missense mutations in theFactor 8-gene associated with discrepant results in patients were transiently expressed. FVIII:C of the mutations was determined using two one-stage assays (FVIII:C1st, FVIII:CBonn) and a two-...
Condition: Hemophilia A Interventions: Biological: Damoctocog-alfa-pegol (BAY94-9027, Jivi); Biological: Rurioctocog alfa pegol (Adynovi) Sponsor: Bayer Not yet recruiting