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Intermediate-dose versus high-dose prophylaxis for severe hemophilia: comparing outcome and costs since the 1970s.

ABSTRACT: Prophylactic treatment in severe hemophilia is very effective but is limited by cost issues. The implementation of 2 different prophylactic regimens in The Netherlands and Sweden since the 1970s may be considered a natural experiment. We compared the costs and outcomes of Dutch intermediate- and Swedish high-dose prophylactic regimens for patients with severe hemophilia (factor VIII/IX < 1 IU/dL) born between 1970 and 1994, using prospective standardized outcome assessment and retrospective collection of cost data. Seventy-eight Dutch and 50 Swedish patients, median age 24 years (range, 14-37 years), were included. Intermediate-dose prophylaxis used less factor concentrate (median: Netherlands, 2100 IU/kg per year [interquartile range (IQR), 1400-2900 IU/kg per year] vs Sweden, 4000 IU/kg per year [IQR, 3000-4900 IU/kg per year]); (P < .01). Clinical outcome was slightly inferior for the intermediate-dose regimen (P < .01) for 5-year bleeding (median, 1.3 [IQR, 0.8-2.7] vs 0 [IQR, 0.0-2.0] joint bleeds/y) and joint health (Haemophilia Joint Health Score >10 of 144 points in 46% vs 11% of participants), although social participation and quality of life were similar. Annual total costs were 66% higher for high-dose prophylaxis (mean, 180 [95% confidence interval, 163 - 196] × US$1000 for Dutch vs 298 [95% confidence interval, 271-325]) × US$1000 for Swedish patients; (P < .01). At group level, the incremental benefits of high-dose prophylaxis appear limited. At the patient level, prophylaxis should be tailored individually, and many patients may do well receiving lower doses of concentrate without compromising safety.

SUBMITTER: Fischer K

PROVIDER: S-EPMC3744988 | biostudies-literature |

REPOSITORIES: biostudies-literature

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

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Intermediate-dose versus high-dose prophylaxis for severe hemophilia: comparing outcome and costs since the 1970s.

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