title: Prospective evaluation of treatment regimens, efficacy and safety of a recombinant factor VIII concentrate in haemophilia A: the German EffeKt study.
abstract: BACKGROUND
  Collecting clinical data under routine conditions remains important to monitor continuously efficacy and tolerability of an established product.
  
  
  OBJECTIVE
  This prospective observational study aimed at evaluating efficacy and tolerability of full-length sucrose-formulated recombinant FVIII (rFVIII-FS; KOGENATE(®) Bayer/KOGENATE(®) FS) in routine use and analysing concomitant diseases and medication.
  
  
  METHODS
  Haemophilia A patients treated with rFVIII-FS were followed up for 24 or 36 months. Efficacy, tolerability, concomitant medication and diseases were assessed at yearly intervals.
  
  
  RESULTS
  Two hundred and twenty-one documented patients were mainly pretreated, predominantly with prophylaxis (74%). On study, 54 (31%) patients in the efficacy set (n = 174) documented regular prophylaxis, 91 patients (52.3%) failed to document their prescribed prophylactic infusions, 25 (14.4%) received on-demand treatment and four patients (2%) inhibitor adapted therapy. Patients on regular prophylaxis reported 8.4% of infusions for bleeding treatment and on-demand patients reported 55.3%. Young patients experienced mainly trauma-related bleedings and older patients reported spontaneous bleedings. Joint status was good overall. Median annual spontaneous joint bleeding rate in all groups was zero. A total of 79.0% of all bleedings were successfully treated with one or two infusions. Degenerative arthropathy and arterial hypertension were the most prominent concomitant diseases. Eighty-seven patients (40.5%) took at least one concomitant drug on study, mostly drugs for pain treatment related to haemophilic arthropathy. Two patients presented with positive inhibitor titres on study, one of them was a previously untreated patient and the other had a positive inhibitor history.
  
  
  CONCLUSIONS
  This study corroborates the tolerability and efficacy profile of rFVIII-FS.
authors.affiliation: Abteilung für innere Medizin, Vivantes Klinikum Friedrichshain, Berlin, Germany. robert.klamroth@vivantes.de.
authors.forename: R
authors.initials: R
authors.lastname: Klamroth
authors.affiliation: Medizinische Klinik III, Institut für Transfusionsmedizin, Goethe Universität, Frankfurt, Germany.
authors.forename: W
authors.initials: W
authors.lastname: Miesbach
authors.affiliation: SRH Kurpfalzkrankenhaus, Heidelberg GmbH und Hämophiliezentrum, Heidelberg, Germany.
authors.forename: P
authors.initials: P
authors.lastname: Staritz
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D002648:Child; D002675:Child, Preschool; D005169:Factor VIII / Q000627:therapeutic use*; D005500:Follow-Up Studies; D006467:Hemophilia A / Q000188:drug therapy* / Q000473:pathology; D006470:Hemorrhage; D006801:Humans; D007223:Infant; D007596:Joints / Q000503:physiopathology; D008297:Male; D008875:Middle Aged; D011446:Prospective Studies; D012720:Severity of Illness Index; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: C078147:F8 protein, human; D005169:Factor VIII
keywords: clinical study; factor VIII; haemophilia A; prophylaxis