Gender differences in the application of Spanish criteria for initiation of enzyme replacement therapy for fabry disease in the fabry outcome survey

Miguel Ángel Barba-Romero, Guillem Pintos-Morell

Research output: Contribution to journalArticleResearchpeer-review

Abstract

© 2016 by the authors; licensee MDPI, Basel, Switzerland. Both male/female patients with Fabry disease (FD) may receive enzyme replacement therapy (ERT). Previously published analyses of the Fabry Outcome Survey (FOS; Shire-sponsored) database suggested gender differences in timing of ERT initiation. We assessed alignment of criteria for ERT initiation in the Spanish adult population included in FOS with recommendations of a Spanish national consensus. This retrospective analysis examined baseline clinical data of 88 adults (49 females) enrolled in the FOS database up to August 2014. Thirty-five (39.8%) patients were not receiving ERT: five (12.8%) males and 30 (61.2%) females. Baseline disease severity on the FOS-derived Mainz Severity Score Index was lower in untreated males (median (interquartile range), 0.0 (0.0–1.0)) than treated males (TM; 15.0 (7.5–26.5)), and was similar in untreated and treated females. The percentage of untreated females with at least one criterion for treatment initiation was 76.7% versus 100.0% of treated females (p = 0.0340) and 97.1% (p = 0.0210) of TM. In discordance with Spanish consensus recommendations, a substantial number of females with evidence of FD who might benefit from ERT have not yet initiated treatment. These results suggest unequal gender perceptions with respect to ERT initiation in Spain.
Original languageEnglish
Article number1965
JournalInternational Journal of Molecular Sciences
Volume17
Issue number12
DOIs
Publication statusPublished - 1 Dec 2016

Keywords

  • Agalsidase alfa
  • Enzyme replacement therapy
  • Fabry Outcome Survey (FOS)
  • Fabry disease
  • Gender differences
  • Spain
  • Women

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