title: On-demand vs. prophylactic treatment for severe haemophilia in Norway and Sweden: differences in treatment characteristics and outcome.
abstract: Using an 11-year panel of 156 Norwegian and Swedish patients with severe haemophilia, and including retrospective case-book data from birth, we compared the differences in the haemophilia-related resource use between on-demand and prophylactic treatment. Patients treated on-demand had more surgery (arthrodeses, prostheses implantations and synovectomies) and more days lost from work. Median annual factor-concentrate consumption among adults (18+) was 211,000 IU [interquartile range (IQR) 154,000-268,000] or 3,024 IU kg-1 year(-1) for patients on prophylactic treatment and 55,000 IU (IQR 28,000-91,000) for on-demand patients (780 IU kg(-1) year(-1)). This was partly explained by the fact that the median dose per kg body weight was twice as great 28, (IQR 24-32) for prophylaxis compared with 14 (IQR 12-16) for on-demand. Prescribed dose per kg body weight was found to be an important factor explaining the variation in total annual factor-concentrate consumption per patient for both types of treatment. Other variables included in the panel-data regression analysis were the number of weeks on secondary prophylaxis for on-demand patients and age, body weight and type of haemophilia for children (0-17 years) on prophylaxis. Differences were consistently substantial and will affect both costs and benefits of the two treatment strategies.
mesh_terms: D000041:Absenteeism; D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D004334:Drug Administration Schedule; D004651:Employment / Q000706:statistics & numerical data; D005164:Factor IX / Q000008:administration & dosage* / Q000627:therapeutic use; D005169:Factor VIII / Q000008:administration & dosage* / Q000627:therapeutic use; D006395:Hemarthrosis / Q000517:prevention & control / Q000601:surgery; D006467:Hemophilia A / Q000150:complications / Q000188:drug therapy*; D002836:Hemophilia B / Q000150:complications / Q000188:drug therapy*; D006470:Hemorrhage / Q000517:prevention & control; D006760:Hospitalization / Q000706:statistics & numerical data; D006801:Humans; D007223:Infant; D008297:Male; D008875:Middle Aged; D009664:Norway; D012189:Retrospective Studies; D013548:Sweden; D016896:Treatment Outcome
chemical_list: D005169:Factor VIII; D005164:Factor IX