Adherence to treatment regimen and bleeding rates in a prospective cohort of youth and young adults on low-dose daily prophylaxis for severe hemophilia AReportar como inadecuado

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BMC Hematology

, 16:26

Hemostasis, thrombosis and vascular biology


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.

KeywordsHemophilia A Prophylaxis Health-related quality of life Annualized bleeding rates Daily dose Adherence AbbreviationsABRAnnualized bleeding rate

CHeQoLSCanadian helixate quality of life study


HAHemophilia A

HJHSHemophilia joint health score 2.0

HRQoLHealth-related quality of life

HTCHemophilia treatment centres

IUInternational units

LDDPLow-dose daily prophylaxis

MCSMental component summary

MIDMinimally important difference

PCSPhysical component summary

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Autor: Terry Mizrahi - Jean St-Louis - Nancy L. Young - Francine Ménard - Nichan Zourikian - Evemie Dubé - Georges E. Rivard


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