Background: When availability and/or affordability of anti-hemophilic factor concentrates are limited, optimal\nprophylaxis regimens in severe hemophilia A (HA) remain to be determined. In selected situations, low-dose\ndaily prophylaxis (LDDP) may be an effective and economical option. The goal of our study was to evaluate\nif subjects on a LDDP regimen could achieve adherence and good clinical outcome.\nMethods: Seventeen subjects (age between 15.2 and 28.4) on LDDP suffering from severe/moderate HA were\nfollowed prospectively for 2 to 3 years as part of a health-related quality of life (HRQoL) study. Bleeding and\ntreatments data were collected using electronic diaries and validated every three months. The SF-36 questionnaire\nwas administered at the beginning of the study and then every 6 months until the end of the study.\nResults: The subjects (mean age 22.0, median 21.9, standard deviation 4.06), were all from a single centre\nand on LDDP for at least 12 months as part of their routine care before entering the study. Fifteen subjects\nwere prescribed a daily dose of 500 IU factor VIII (FVIII) and 2 subjects received 1000 IU FVIII per day,\nresulting into a median dose of 7.1 IU/kg/day (ranging from 4 to 13 IU/kg/day) and of 2591 IU/kg/year.\nMedian adherence (the percentage of the prescribed daily dose received) was 84 % (mean 80 %, range 57 %\nto 94 %) throughout the study. Seventy-six bleeds in the 6 index joints and 51 other types of bleeds were\nobserved throughout the study. The median annualized bleeding rate in joints (ABRjoints) was 0.7 and the\nmedian annualized bleeding rate for all bleeds (ABRall) was 1.6. The Physical Component and Mental Component\nSummary scores of SF-36, and the Hemophilia Joint Health Score were not significantly different over the course of\nthe study (respective medians of 49.8, 52.4 and 16.0 at entry; vs. 52.5, 51.5 and 16.0 upon exit).\nConclusions: This prospective longitudinal study in youth and young adults shows that LDDP may be associated\nwith low ABRs, adequate adherence and HRQoL comparable to previously reported.
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