Full-length sucrose-formulated recombinant factor VIII for treatment of previously untreated or minimally treated young children with severe haemophilia A: results of an international clinical investigation.
The safety and efficacy of a full-length sucrose-formulated recombinant factor VIII product (rFVIII-FS; Kogenate FS; Kogenate Bayer) was evaluated in previously untreated (PUPs) and minimally treated (MTP) patients with severe haemophilia A (FVIII <2%). Patients (37 PUPs; 24 MTPs) aged 0.1-25.7 months were treated with rFVIII-FS for a cumulative of 9,141 exposure days (EDs), median 114 EDs (range 4-478), on prophylactic or on-demand therapy. Eighty-nine percent of all treated bleeding episodes were successfully treated with 1 (74%) or 2 (15%) infusions. Clinical response to first infusion for each bleeding episode was rated as 'excellent' in 58%, or 'good' in 33%, of all cases. Recombinant FVIII-FS was used in 27 surgical procedures, mainly catheter implantations, which were all conducted without bleeding complications. FVIII recovery mean values (approximately 2%/kg/IU) were as expected for any licensed FVIII concentrate. FVIII neutralizing antibody formation was 15% (9/60). Aside from inhibitor formation, three adverse events were rated as 'at least possibly drug-related' for a total drug-related adverse event rate of 0.14%. No viral seroconversions were observed. Overall, excellent safety and efficacy were demonstrated with rFVIII-FS for therapy of young children with severe haemophilia A.
Kreuz
Wolfhart
W
The Center of Pediatrics III, Department of Hematology, Oncology and Hemostaseology, Johann Wolfgang Goethe-University Hospital, Frankfurt am Main, Germany. Wolfhart.Kreuz@kgu.de
Gill
Joan C
JC
Rothschild
Chantal
C
Manco-Johnson
Marilyn J
MJ
Lusher
Jeanne M
JM
Kellermann
Elke
E
Gorina
Eduard
E
Larson
Peter J
PJ
D000917:Antibody Formation; D002675:Child, Preschool; D004252:DNA Mutational Analysis; D005091:Exons / Q000235:genetics; D005169:Factor VIII / Q000008:administration & dosage* / Q000009:adverse effects / Q000235:genetics / Q000276:immunology; D006467:Hemophilia A / Q000188:drug therapy*; D006470:Hemorrhage / Q000188:drug therapy / Q000517:prevention & control; D006801:Humans; D007223:Infant; D007438:Introns / Q000235:genetics; D009154:Mutation; D016896:Treatment Outcome
C078147:F8 protein, human; D005169:Factor VIII