title: Prophylaxis and quality of life in patients with hemophilia A during routine treatment with ADVATE [antihemophilic factor (recombinant), plasma/albumin-free method] in Germany: a subgroup analysis of the ADVATE PASS post-approval, non-interventional study.
abstract: Antihemophilic factor (recombinant), plasma/albumin-free method (rAHF-PFM) was evaluated during routine practice in Germany, among consenting subjects of any age with hemophilia A (HA) and no prior exposure to rAHF-PFM. The treating physician chose the dosing and inhibitor testing frequency. Data were captured for 12 months/subject from diaries and clinic records. Of 152 subjects, 69 % had severe HA, and 89 % had >150 exposure 6 days (ED) at baseline. The majority of subjects (63 %) were treated by continuous prophylaxis (CP). Assignment to CP was more likely for subjects ≥2 years of age and for those with FVIII ≤ 2 %. Median FVIII consumption was 3,548 IU/kg/year for CP and 999 IU/kg/year for continuous on-demand (OD) therapy. Median annual bleed rate was 0.82 for CP and 4.06 for OD. Of 1,218 bleeds, 97 % were home-treated and 68 % of evaluable bleeds involved joints. Based on evaluable subjects' worst ratings, 83/91 (91 %) on CP had a rating of excellent/good for all prophylactic assessments, 55/59 (93 %) on CP and 41/42 (98 %) on OD had a rating of excellent/good for all bleeding assessments. The de novo high-titer FVIII inhibitor rate in subjects with >50 ED at baseline was 1/144 (0.69 %; 95 % CI, 0.02 % to 3.81 %). No high-titer inhibitor occurred in patients with severe HA and >50 ED at baseline. Reduced HRQOL physical scores were predicted by older age (p < 0.0001), HIV positivity (p = 0.02), and presence of ≥1 target joint (p = 0.003). ADVATE rAHF-PFM is safe and efficacious for routine CP or OD management of patients with HA.
authors: Ambulanzzentrum an der Raphaelsklinik, Institut für Thrombophilie und Hämostaseologie, Loerstraße 19, Münster, Germany. ith@haemophilie-zentrum.de
authors: Hartmut
authors: H
authors: Pollmann
authors: Robert
authors: R
authors: Klamroth
authors: Natascha
authors: N
authors: Vidovic
authors: Alexander Y
authors: AY
authors: Kriukov
authors: Joshua
authors: J
authors: Epstein
authors: Ingo
authors: I
authors: Abraham
authors: Gerald
authors: G
authors: Spotts
authors: Johannes
authors: J
authors: Oldenburg
mesh_terms: D000293:Adolescent; D000328:Adult; D018890:Chemoprevention / Q000379:methods; D002648:Child; D002675:Child, Preschool; D017277:Drug Approval; D005169:Factor VIII / Q000008:administration & dosage*; D005858:Germany; D006467:Hemophilia A / Q000188:drug therapy*; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008875:Middle Aged; D011358:Product Surveillance, Postmarketing; D011788:Quality of Life; D011994:Recombinant Proteins / Q000008:administration & dosage; D012709:Serum Albumin / Q000008:administration & dosage; D055815:Young Adult
chemical_list: D011994:Recombinant Proteins; D012709:Serum Albumin; C078147:F8 protein, human; D005169:Factor VIII