“We have previously shown, with a formal cost-benefit analysis using the value-of-information approach, that tailored frequency escalated prophylaxis provides substantial cost-benefit in a variety of scenarios. Our results from this study lend further support to these findings,” they wrote.
The median annualized index joint bleeding rate was 0.95 per year, but 30% of the boys in the study experienced “unacceptable breakthrough bleeding” during the study.
Overall joint health was “well preserved,” according to the researchers. The median Colorado Child Physical Examination Score at the end of the CHPS was 1 (range 0-12) for the left ankle and 1 for the right ankle, with all other joints having a median score of 0.
Activities of daily living and physical function, as well as health-related quality of life were generally good, the researchers reported.
The study was initially funded by grants from the Medical Research Council of Canada/Pharmaceutical Manufacturers Association of Canada Partnership Fund and by the Bayer/Canadian Blood Services/Héma-Québec Partnership Fund. Subsequent renewals were funded by Bayer. Dr. Feldman reported grants from Bayer during the conduct of the study and grants from Baxter/Baxalta/Shire outside the submitted work. Several coauthors also report multiple relationships with industry.
SOURCE: Feldman BM et al. Lancet Haematol. 2018 May 3. doi: 10.1016/S2352-3026(18)30048-6.