On‐demand vs. prophylactic treatment for severe haemophilia in Norway and Sweden: differences in treatment characteristics and outcome

Summary. 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−1year−1for 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.

On‐demand vs. prophylactic treatment for severe haemophilia in Norway and Sweden: differences in treatment characteristics and outcome Journal Articles