6d0e84c76c40fc10d5ec341add24f19b pmc_27886142.pdf 606ab606b7e91b44fd367af8d280e101ede599b9 pmc_27886142.pdf 1c53007d4a1939c4dcb643e8ae5de6626931e1a19d6ab540cf079f5f6631ff38 pmc_27886142.pdf Title: Gender Differences in the Application of Spanish Criteria for Initiation of Enzyme Replacement Therapy for Fabry Disease in the Fabry Outcome Survey Subject: 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. Keywords: agalsidase alfa; enzyme replacement therapy; Fabry disease; Fabry Outcome Survey (FOS); gender differences; Spain; women Author: Miguel-Ángel Barba-Romero and Guillem Pintos-Morell Creator: LaTeX with hyperref package Producer: pdfTeX-1.40.15 CreationDate: Thu Nov 24 07:15:54 2016 CET ModDate: Thu Nov 24 07:15:54 2016 CET Tagged: no UserProperties: no Suspects: no Form: none JavaScript: no Pages: 11 Encrypted: no Page size: 595.276 x 841.89 pts (A4) Page rot: 0 File size: 1554338 bytes Optimized: no PDF version: 1.5 name type encoding emb sub uni object ID ------------------------------------ ----------------- ---------------- --- --- --- --------- ISNODJ+URWPalladioL-Ital Type 1 Custom yes yes no 56 0 ZGXSIH+URWPalladioL-Bold Type 1 Custom yes yes no 57 0 DFTVXE+URWPalladioL-Roma Type 1 Custom yes yes no 58 0 CKBPVA+EURM10 Type 1 Builtin yes yes no 59 0 CKUPDE+TimesNewRomanPS-BoldItalicMT Type 1C WinAnsi yes yes no 63 0 IRWHZX+TimesNewRomanPSMT Type 1C WinAnsi yes yes no 64 0 EILUJF+CMSY10 Type 1 Builtin yes yes no 125 0 GKJHPD+PalatinoLinotype CID TrueType Identity-H yes yes yes 145 0 GKJIAD+PalatinoLinotype TrueType WinAnsi yes yes no 146 0 GKJIFD+PalatinoLinotype,Italic TrueType WinAnsi yes yes no 147 0 GKJIFF+PalatinoLinotype,Bold TrueType WinAnsi yes yes no 148 0 Jhove (Rel. 1.22.1, 2019-04-17) Date: 2023-02-16 02:42:03 CET RepresentationInformation: pmc_27886142.pdf ReportingModule: PDF-hul, Rel. 1.12.1 (2019-04-17) LastModified: 2020-08-10 03:42:39 CEST Size: 1554338 Format: PDF Version: 1.5 Status: Well-Formed and valid SignatureMatches: PDF-hul MIMEtype: application/pdf PDFMetadata: Objects: 0 FreeObjects: 3 IncrementalUpdates: 0 DocumentCatalog: ViewerPreferences: HideToolbar: false HideMenubar: false HideWindowUI: false FitWindow: true CenterWindow: false DisplayDocTitle: false NonFullScreenPageMode: UseNone Direction: L2R ViewArea: CropBox ViewClip: CropBox PrintArea: CropBox PageClip: CropBox PageLayout: SinglePage PageMode: UseNone Outlines: Item: Title: ぉのぴひはつふっぴどはのぜ〴〩਼㰠⽓ 䝯呯 䐠⡳散瑩潮⸲ Item: Title: げづびふぬぴびぜ〴〩਼㰠⽓ 䝯呯 䐠⡳散瑩潮⸳ Item: Title: いどびっふびびどはのぜ〴〩਼㰠⽓ 䝯呯 䐠⡳散瑩潮⸴ Item: Title: きちぴづひどちぬびぜ〴ぜ〰ちのつぜ〴ぜ〰きづぴとはつびぜ〴〩਼㰠⽓ 䝯呯 䐠⡳畢獥捴楯渮㐮ㄩ‾㸊⡜㌷㙜㌷㝜〰いづびどでのぜ〴〩਼㰠⽓ 䝯呯 䐠⡳畢獥捴楯渮㐮㈩‾㸊⡜㌷㙜㌷㝜〰こぴふつべぜ〴ぜ〰ぐはばふぬちぴどはのぜ〴〩਼㰠⽓ 䝯呯 䐠⡳畢獥捴楯渮㐮㌩‾㸊⡜㌷㙜㌷㝜〰こぴふつべぜ〴ぜ〰きづちびふひづびぜ〴〩਼㰠⽓ 䝯呯 䐠⡳畢獥捴楯渮㐮㐩‾㸊⡜㌷㙜㌷㝜〰こぴちぴどびぴどっちぬぜ〴ぜ〰ぁのちぬべびづびぜ〴〩਼㰠⽓ 䝯呯 䐠⡳散瑩潮⸵ Children: Item: Title: いづびどでのぜ〴〩਼㰠⽓ 䝯呯 䐠⡳畢獥捴楯渮㐮㈩‾㸊⡜㌷㙜㌷㝜〰こぴふつべぜ〴ぜ〰ぐはばふぬちぴどはのぜ〴〩਼㰠⽓ 䝯呯 䐠⡳畢獥捴楯渮㐮㌩‾㸊⡜㌷㙜㌷㝜〰こぴふつべぜ〴ぜ〰きづちびふひづびぜ〴〩਼㰠⽓ 䝯呯 䐠⡳畢獥捴楯渮㐮㐩‾㸊⡜㌷㙜㌷㝜〰こぴちぴどびぴどっちぬぜ〴ぜ〰ぁのちぬべびづびぜ〴〩਼㰠⽓ 䝯呯 䐠⡳散瑩潮⸵ Item: Title: こぴふつべぜ〴ぜ〰ぐはばふぬちぴどはのぜ〴〩਼㰠⽓ 䝯呯 䐠⡳畢獥捴楯渮㐮㌩‾㸊⡜㌷㙜㌷㝜〰こぴふつべぜ〴ぜ〰きづちびふひづびぜ〴〩਼㰠⽓ 䝯呯 䐠⡳畢獥捴楯渮㐮㐩‾㸊⡜㌷㙜㌷㝜〰こぴちぴどびぴどっちぬぜ〴ぜ〰ぁのちぬべびづびぜ〴〩਼㰠⽓ 䝯呯 䐠⡳散瑩潮⸵ Item: Title: こぴふつべぜ〴ぜ〰きづちびふひづびぜ〴〩਼㰠⽓ 䝯呯 䐠⡳畢獥捴楯渮㐮㐩‾㸊⡜㌷㙜㌷㝜〰こぴちぴどびぴどっちぬぜ〴ぜ〰ぁのちぬべびづびぜ〴〩਼㰠⽓ 䝯呯 䐠⡳散瑩潮⸵ Item: Title: こぴちぴどびぴどっちぬぜ〴ぜ〰ぁのちぬべびづびぜ〴〩਼㰠⽓ 䝯呯 䐠⡳散瑩潮⸵ Item: Title: ぃはのっぬふびどはのびぜ〴〩਼㰠⽓ 䝯呯 䐠嬴㈠〠删⽆楴䡝‾㸊㰼ਯ呹灥 偡来ਯ䍯湴敮瑳‵㌠〠刊⽒敳潵牣敳‵㈠〠刊⽍敤楡䉯砠嬰‰‵㤵⸲㜶‸㐱⸸㥝ਯ偡牥湴‶〠〠刊⽁湮潴猠嬠㐳‰⁒‴㔠〠删㐷‰⁒‴㠠〠删㐹‰⁒‵ㄠ〠删崊㸾਼㰊⽐牯摵捥爠⡇偌⁇桯獴獣物灴‹⸱〩ਯ䍲敡瑩潮䑡瑥
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