Impact of Nontreatment Duration and Keratopathy on Major Adverse Cardiovascular Events in Fabry Disease: A Nationwide Cohort Study

Fabry disease (FD) is a rare inherited X-linked lysosomal storage disorder that results in the progressive accumulation of glycosphingolipids in multiple organs. Early FD-specific treatments may improve clinical outcomes; however, clinical evidence about early FD treatment is limited. We aimed to de...
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Autor*in:	Aram Yang [verfasserIn] Sinae Kim [verfasserIn] Yong Jun Choi [verfasserIn]

Format:	E-Artikel
Sprache:	Englisch

Erschienen:	2024

Schlagwörter:	cardiovascular diseases comorbidity Fabry disease glycosphingolipids risk factors

Übergeordnetes Werk:	In: Journal of Clinical Medicine - MDPI AG, 2013, 13(2024), 2, p 479
Übergeordnetes Werk:	volume:13 ; year:2024 ; number:2, p 479

Links:	Link aufrufen Link aufrufen Link aufrufen Journal toc

DOI / URN:	10.3390/jcm13020479

Katalog-ID:	DOAJ096179368

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520			\|a Fabry disease (FD) is a rare inherited X-linked lysosomal storage disorder that results in the progressive accumulation of glycosphingolipids in multiple organs. Early FD-specific treatments may improve clinical outcomes; however, clinical evidence about early FD treatment is limited. We aimed to determine the cardiovascular outcomes of patients with FD who received enzyme replacement therapy. This nationwide observational study was conducted using the National Health Claims database of the Korean population with FD. The primary outcome was major adverse cardiovascular events (MACEs). MACE risk factors in FD were evaluated using time-dependent Cox regression. Between January 2007 and April 2022, 188 patients with FD were analyzed. Among them, 22 (11.7%) experienced MACE (males: 14/95 [14.7%]; females: 8/93 [8.6%]). The mean age at MACE diagnosis was 53.5 ± 11.0 years in all patients with FD, which was lower in males compared with in females (49.7 ± 9.6 vs. 60.0 ± 10.7 years, <i<p</i< = 0.030). Multivariate analysis (HR, 95% CI) revealed that age (1.042; 1.004–1.082) and duration of FD nontreatment (1.040; 1.003–1.078) were significant MACE risk factors in all patients. In males, age (1.080; 1.032–1.131), FD nontreatment duration (1.099; 1.048–1.152), and keratopathy (18.920; 4.174–85.749) were significant MACE risk factors in multivariate analysis. In females, the only significant MACE risk factor was a high Charlson comorbidity index score (1.795; 1.229–2.622). In conclusion, duration of FD nontreatment and keratopathy are significant MACE risk factors in males with FD. These findings suggest the importance of early initiation of FD-specific treatment and careful evaluation of keratopathy in males with FD.
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allfields	10.3390/jcm13020479 doi (DE-627)DOAJ096179368 (DE-599)DOAJecbbb75026704a6ab878e89eeff99fe3 DE-627 ger DE-627 rakwb eng Aram Yang verfasserin aut Impact of Nontreatment Duration and Keratopathy on Major Adverse Cardiovascular Events in Fabry Disease: A Nationwide Cohort Study 2024 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier Fabry disease (FD) is a rare inherited X-linked lysosomal storage disorder that results in the progressive accumulation of glycosphingolipids in multiple organs. Early FD-specific treatments may improve clinical outcomes; however, clinical evidence about early FD treatment is limited. We aimed to determine the cardiovascular outcomes of patients with FD who received enzyme replacement therapy. This nationwide observational study was conducted using the National Health Claims database of the Korean population with FD. The primary outcome was major adverse cardiovascular events (MACEs). MACE risk factors in FD were evaluated using time-dependent Cox regression. Between January 2007 and April 2022, 188 patients with FD were analyzed. Among them, 22 (11.7%) experienced MACE (males: 14/95 [14.7%]; females: 8/93 [8.6%]). The mean age at MACE diagnosis was 53.5 ± 11.0 years in all patients with FD, which was lower in males compared with in females (49.7 ± 9.6 vs. 60.0 ± 10.7 years, <i<p</i< = 0.030). Multivariate analysis (HR, 95% CI) revealed that age (1.042; 1.004–1.082) and duration of FD nontreatment (1.040; 1.003–1.078) were significant MACE risk factors in all patients. In males, age (1.080; 1.032–1.131), FD nontreatment duration (1.099; 1.048–1.152), and keratopathy (18.920; 4.174–85.749) were significant MACE risk factors in multivariate analysis. In females, the only significant MACE risk factor was a high Charlson comorbidity index score (1.795; 1.229–2.622). In conclusion, duration of FD nontreatment and keratopathy are significant MACE risk factors in males with FD. These findings suggest the importance of early initiation of FD-specific treatment and careful evaluation of keratopathy in males with FD. cardiovascular diseases comorbidity Fabry disease glycosphingolipids risk factors Medicine R Sinae Kim verfasserin aut Yong Jun Choi verfasserin aut In Journal of Clinical Medicine MDPI AG, 2013 13(2024), 2, p 479 (DE-627)718632478 (DE-600)2662592-1 20770383 nnns volume:13 year:2024 number:2, p 479 https://doi.org/10.3390/jcm13020479 kostenfrei https://doaj.org/article/ecbbb75026704a6ab878e89eeff99fe3 kostenfrei https://www.mdpi.com/2077-0383/13/2/479 kostenfrei https://doaj.org/toc/2077-0383 Journal toc kostenfrei GBV_USEFLAG_A SYSFLAG_A GBV_DOAJ GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2005 GBV_ILN_2009 GBV_ILN_2014 GBV_ILN_2055 GBV_ILN_2111 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 13 2024 2, p 479
spelling	10.3390/jcm13020479 doi (DE-627)DOAJ096179368 (DE-599)DOAJecbbb75026704a6ab878e89eeff99fe3 DE-627 ger DE-627 rakwb eng Aram Yang verfasserin aut Impact of Nontreatment Duration and Keratopathy on Major Adverse Cardiovascular Events in Fabry Disease: A Nationwide Cohort Study 2024 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier Fabry disease (FD) is a rare inherited X-linked lysosomal storage disorder that results in the progressive accumulation of glycosphingolipids in multiple organs. Early FD-specific treatments may improve clinical outcomes; however, clinical evidence about early FD treatment is limited. We aimed to determine the cardiovascular outcomes of patients with FD who received enzyme replacement therapy. This nationwide observational study was conducted using the National Health Claims database of the Korean population with FD. The primary outcome was major adverse cardiovascular events (MACEs). MACE risk factors in FD were evaluated using time-dependent Cox regression. Between January 2007 and April 2022, 188 patients with FD were analyzed. Among them, 22 (11.7%) experienced MACE (males: 14/95 [14.7%]; females: 8/93 [8.6%]). The mean age at MACE diagnosis was 53.5 ± 11.0 years in all patients with FD, which was lower in males compared with in females (49.7 ± 9.6 vs. 60.0 ± 10.7 years, <i<p</i< = 0.030). Multivariate analysis (HR, 95% CI) revealed that age (1.042; 1.004–1.082) and duration of FD nontreatment (1.040; 1.003–1.078) were significant MACE risk factors in all patients. In males, age (1.080; 1.032–1.131), FD nontreatment duration (1.099; 1.048–1.152), and keratopathy (18.920; 4.174–85.749) were significant MACE risk factors in multivariate analysis. In females, the only significant MACE risk factor was a high Charlson comorbidity index score (1.795; 1.229–2.622). In conclusion, duration of FD nontreatment and keratopathy are significant MACE risk factors in males with FD. These findings suggest the importance of early initiation of FD-specific treatment and careful evaluation of keratopathy in males with FD. cardiovascular diseases comorbidity Fabry disease glycosphingolipids risk factors Medicine R Sinae Kim verfasserin aut Yong Jun Choi verfasserin aut In Journal of Clinical Medicine MDPI AG, 2013 13(2024), 2, p 479 (DE-627)718632478 (DE-600)2662592-1 20770383 nnns volume:13 year:2024 number:2, p 479 https://doi.org/10.3390/jcm13020479 kostenfrei https://doaj.org/article/ecbbb75026704a6ab878e89eeff99fe3 kostenfrei https://www.mdpi.com/2077-0383/13/2/479 kostenfrei https://doaj.org/toc/2077-0383 Journal toc kostenfrei GBV_USEFLAG_A SYSFLAG_A GBV_DOAJ GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2005 GBV_ILN_2009 GBV_ILN_2014 GBV_ILN_2055 GBV_ILN_2111 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 13 2024 2, p 479
allfields_unstemmed	10.3390/jcm13020479 doi (DE-627)DOAJ096179368 (DE-599)DOAJecbbb75026704a6ab878e89eeff99fe3 DE-627 ger DE-627 rakwb eng Aram Yang verfasserin aut Impact of Nontreatment Duration and Keratopathy on Major Adverse Cardiovascular Events in Fabry Disease: A Nationwide Cohort Study 2024 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier Fabry disease (FD) is a rare inherited X-linked lysosomal storage disorder that results in the progressive accumulation of glycosphingolipids in multiple organs. Early FD-specific treatments may improve clinical outcomes; however, clinical evidence about early FD treatment is limited. We aimed to determine the cardiovascular outcomes of patients with FD who received enzyme replacement therapy. This nationwide observational study was conducted using the National Health Claims database of the Korean population with FD. The primary outcome was major adverse cardiovascular events (MACEs). MACE risk factors in FD were evaluated using time-dependent Cox regression. Between January 2007 and April 2022, 188 patients with FD were analyzed. Among them, 22 (11.7%) experienced MACE (males: 14/95 [14.7%]; females: 8/93 [8.6%]). The mean age at MACE diagnosis was 53.5 ± 11.0 years in all patients with FD, which was lower in males compared with in females (49.7 ± 9.6 vs. 60.0 ± 10.7 years, <i<p</i< = 0.030). Multivariate analysis (HR, 95% CI) revealed that age (1.042; 1.004–1.082) and duration of FD nontreatment (1.040; 1.003–1.078) were significant MACE risk factors in all patients. In males, age (1.080; 1.032–1.131), FD nontreatment duration (1.099; 1.048–1.152), and keratopathy (18.920; 4.174–85.749) were significant MACE risk factors in multivariate analysis. In females, the only significant MACE risk factor was a high Charlson comorbidity index score (1.795; 1.229–2.622). In conclusion, duration of FD nontreatment and keratopathy are significant MACE risk factors in males with FD. These findings suggest the importance of early initiation of FD-specific treatment and careful evaluation of keratopathy in males with FD. cardiovascular diseases comorbidity Fabry disease glycosphingolipids risk factors Medicine R Sinae Kim verfasserin aut Yong Jun Choi verfasserin aut In Journal of Clinical Medicine MDPI AG, 2013 13(2024), 2, p 479 (DE-627)718632478 (DE-600)2662592-1 20770383 nnns volume:13 year:2024 number:2, p 479 https://doi.org/10.3390/jcm13020479 kostenfrei https://doaj.org/article/ecbbb75026704a6ab878e89eeff99fe3 kostenfrei https://www.mdpi.com/2077-0383/13/2/479 kostenfrei https://doaj.org/toc/2077-0383 Journal toc kostenfrei GBV_USEFLAG_A SYSFLAG_A GBV_DOAJ GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2005 GBV_ILN_2009 GBV_ILN_2014 GBV_ILN_2055 GBV_ILN_2111 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 13 2024 2, p 479
allfieldsGer	10.3390/jcm13020479 doi (DE-627)DOAJ096179368 (DE-599)DOAJecbbb75026704a6ab878e89eeff99fe3 DE-627 ger DE-627 rakwb eng Aram Yang verfasserin aut Impact of Nontreatment Duration and Keratopathy on Major Adverse Cardiovascular Events in Fabry Disease: A Nationwide Cohort Study 2024 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier Fabry disease (FD) is a rare inherited X-linked lysosomal storage disorder that results in the progressive accumulation of glycosphingolipids in multiple organs. Early FD-specific treatments may improve clinical outcomes; however, clinical evidence about early FD treatment is limited. We aimed to determine the cardiovascular outcomes of patients with FD who received enzyme replacement therapy. This nationwide observational study was conducted using the National Health Claims database of the Korean population with FD. The primary outcome was major adverse cardiovascular events (MACEs). MACE risk factors in FD were evaluated using time-dependent Cox regression. Between January 2007 and April 2022, 188 patients with FD were analyzed. Among them, 22 (11.7%) experienced MACE (males: 14/95 [14.7%]; females: 8/93 [8.6%]). The mean age at MACE diagnosis was 53.5 ± 11.0 years in all patients with FD, which was lower in males compared with in females (49.7 ± 9.6 vs. 60.0 ± 10.7 years, <i<p</i< = 0.030). Multivariate analysis (HR, 95% CI) revealed that age (1.042; 1.004–1.082) and duration of FD nontreatment (1.040; 1.003–1.078) were significant MACE risk factors in all patients. In males, age (1.080; 1.032–1.131), FD nontreatment duration (1.099; 1.048–1.152), and keratopathy (18.920; 4.174–85.749) were significant MACE risk factors in multivariate analysis. In females, the only significant MACE risk factor was a high Charlson comorbidity index score (1.795; 1.229–2.622). In conclusion, duration of FD nontreatment and keratopathy are significant MACE risk factors in males with FD. These findings suggest the importance of early initiation of FD-specific treatment and careful evaluation of keratopathy in males with FD. cardiovascular diseases comorbidity Fabry disease glycosphingolipids risk factors Medicine R Sinae Kim verfasserin aut Yong Jun Choi verfasserin aut In Journal of Clinical Medicine MDPI AG, 2013 13(2024), 2, p 479 (DE-627)718632478 (DE-600)2662592-1 20770383 nnns volume:13 year:2024 number:2, p 479 https://doi.org/10.3390/jcm13020479 kostenfrei https://doaj.org/article/ecbbb75026704a6ab878e89eeff99fe3 kostenfrei https://www.mdpi.com/2077-0383/13/2/479 kostenfrei https://doaj.org/toc/2077-0383 Journal toc kostenfrei GBV_USEFLAG_A SYSFLAG_A GBV_DOAJ GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2005 GBV_ILN_2009 GBV_ILN_2014 GBV_ILN_2055 GBV_ILN_2111 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 13 2024 2, p 479
allfieldsSound	10.3390/jcm13020479 doi (DE-627)DOAJ096179368 (DE-599)DOAJecbbb75026704a6ab878e89eeff99fe3 DE-627 ger DE-627 rakwb eng Aram Yang verfasserin aut Impact of Nontreatment Duration and Keratopathy on Major Adverse Cardiovascular Events in Fabry Disease: A Nationwide Cohort Study 2024 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier Fabry disease (FD) is a rare inherited X-linked lysosomal storage disorder that results in the progressive accumulation of glycosphingolipids in multiple organs. Early FD-specific treatments may improve clinical outcomes; however, clinical evidence about early FD treatment is limited. We aimed to determine the cardiovascular outcomes of patients with FD who received enzyme replacement therapy. This nationwide observational study was conducted using the National Health Claims database of the Korean population with FD. The primary outcome was major adverse cardiovascular events (MACEs). MACE risk factors in FD were evaluated using time-dependent Cox regression. Between January 2007 and April 2022, 188 patients with FD were analyzed. Among them, 22 (11.7%) experienced MACE (males: 14/95 [14.7%]; females: 8/93 [8.6%]). The mean age at MACE diagnosis was 53.5 ± 11.0 years in all patients with FD, which was lower in males compared with in females (49.7 ± 9.6 vs. 60.0 ± 10.7 years, <i<p</i< = 0.030). Multivariate analysis (HR, 95% CI) revealed that age (1.042; 1.004–1.082) and duration of FD nontreatment (1.040; 1.003–1.078) were significant MACE risk factors in all patients. In males, age (1.080; 1.032–1.131), FD nontreatment duration (1.099; 1.048–1.152), and keratopathy (18.920; 4.174–85.749) were significant MACE risk factors in multivariate analysis. In females, the only significant MACE risk factor was a high Charlson comorbidity index score (1.795; 1.229–2.622). In conclusion, duration of FD nontreatment and keratopathy are significant MACE risk factors in males with FD. These findings suggest the importance of early initiation of FD-specific treatment and careful evaluation of keratopathy in males with FD. cardiovascular diseases comorbidity Fabry disease glycosphingolipids risk factors Medicine R Sinae Kim verfasserin aut Yong Jun Choi verfasserin aut In Journal of Clinical Medicine MDPI AG, 2013 13(2024), 2, p 479 (DE-627)718632478 (DE-600)2662592-1 20770383 nnns volume:13 year:2024 number:2, p 479 https://doi.org/10.3390/jcm13020479 kostenfrei https://doaj.org/article/ecbbb75026704a6ab878e89eeff99fe3 kostenfrei https://www.mdpi.com/2077-0383/13/2/479 kostenfrei https://doaj.org/toc/2077-0383 Journal toc kostenfrei GBV_USEFLAG_A SYSFLAG_A GBV_DOAJ GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2005 GBV_ILN_2009 GBV_ILN_2014 GBV_ILN_2055 GBV_ILN_2111 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 13 2024 2, p 479
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author	Aram Yang
spellingShingle	Aram Yang misc cardiovascular diseases misc comorbidity misc Fabry disease misc glycosphingolipids misc risk factors misc Medicine misc R Impact of Nontreatment Duration and Keratopathy on Major Adverse Cardiovascular Events in Fabry Disease: A Nationwide Cohort Study
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topic_title	Impact of Nontreatment Duration and Keratopathy on Major Adverse Cardiovascular Events in Fabry Disease: A Nationwide Cohort Study cardiovascular diseases comorbidity Fabry disease glycosphingolipids risk factors
topic	misc cardiovascular diseases misc comorbidity misc Fabry disease misc glycosphingolipids misc risk factors misc Medicine misc R
topic_unstemmed	misc cardiovascular diseases misc comorbidity misc Fabry disease misc glycosphingolipids misc risk factors misc Medicine misc R
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title	Impact of Nontreatment Duration and Keratopathy on Major Adverse Cardiovascular Events in Fabry Disease: A Nationwide Cohort Study
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title_full	Impact of Nontreatment Duration and Keratopathy on Major Adverse Cardiovascular Events in Fabry Disease: A Nationwide Cohort Study
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title_sort	impact of nontreatment duration and keratopathy on major adverse cardiovascular events in fabry disease: a nationwide cohort study
title_auth	Impact of Nontreatment Duration and Keratopathy on Major Adverse Cardiovascular Events in Fabry Disease: A Nationwide Cohort Study
abstract	Fabry disease (FD) is a rare inherited X-linked lysosomal storage disorder that results in the progressive accumulation of glycosphingolipids in multiple organs. Early FD-specific treatments may improve clinical outcomes; however, clinical evidence about early FD treatment is limited. We aimed to determine the cardiovascular outcomes of patients with FD who received enzyme replacement therapy. This nationwide observational study was conducted using the National Health Claims database of the Korean population with FD. The primary outcome was major adverse cardiovascular events (MACEs). MACE risk factors in FD were evaluated using time-dependent Cox regression. Between January 2007 and April 2022, 188 patients with FD were analyzed. Among them, 22 (11.7%) experienced MACE (males: 14/95 [14.7%]; females: 8/93 [8.6%]). The mean age at MACE diagnosis was 53.5 ± 11.0 years in all patients with FD, which was lower in males compared with in females (49.7 ± 9.6 vs. 60.0 ± 10.7 years, <i<p</i< = 0.030). Multivariate analysis (HR, 95% CI) revealed that age (1.042; 1.004–1.082) and duration of FD nontreatment (1.040; 1.003–1.078) were significant MACE risk factors in all patients. In males, age (1.080; 1.032–1.131), FD nontreatment duration (1.099; 1.048–1.152), and keratopathy (18.920; 4.174–85.749) were significant MACE risk factors in multivariate analysis. In females, the only significant MACE risk factor was a high Charlson comorbidity index score (1.795; 1.229–2.622). In conclusion, duration of FD nontreatment and keratopathy are significant MACE risk factors in males with FD. These findings suggest the importance of early initiation of FD-specific treatment and careful evaluation of keratopathy in males with FD.
abstractGer	Fabry disease (FD) is a rare inherited X-linked lysosomal storage disorder that results in the progressive accumulation of glycosphingolipids in multiple organs. Early FD-specific treatments may improve clinical outcomes; however, clinical evidence about early FD treatment is limited. We aimed to determine the cardiovascular outcomes of patients with FD who received enzyme replacement therapy. This nationwide observational study was conducted using the National Health Claims database of the Korean population with FD. The primary outcome was major adverse cardiovascular events (MACEs). MACE risk factors in FD were evaluated using time-dependent Cox regression. Between January 2007 and April 2022, 188 patients with FD were analyzed. Among them, 22 (11.7%) experienced MACE (males: 14/95 [14.7%]; females: 8/93 [8.6%]). The mean age at MACE diagnosis was 53.5 ± 11.0 years in all patients with FD, which was lower in males compared with in females (49.7 ± 9.6 vs. 60.0 ± 10.7 years, <i<p</i< = 0.030). Multivariate analysis (HR, 95% CI) revealed that age (1.042; 1.004–1.082) and duration of FD nontreatment (1.040; 1.003–1.078) were significant MACE risk factors in all patients. In males, age (1.080; 1.032–1.131), FD nontreatment duration (1.099; 1.048–1.152), and keratopathy (18.920; 4.174–85.749) were significant MACE risk factors in multivariate analysis. In females, the only significant MACE risk factor was a high Charlson comorbidity index score (1.795; 1.229–2.622). In conclusion, duration of FD nontreatment and keratopathy are significant MACE risk factors in males with FD. These findings suggest the importance of early initiation of FD-specific treatment and careful evaluation of keratopathy in males with FD.
abstract_unstemmed	Fabry disease (FD) is a rare inherited X-linked lysosomal storage disorder that results in the progressive accumulation of glycosphingolipids in multiple organs. Early FD-specific treatments may improve clinical outcomes; however, clinical evidence about early FD treatment is limited. We aimed to determine the cardiovascular outcomes of patients with FD who received enzyme replacement therapy. This nationwide observational study was conducted using the National Health Claims database of the Korean population with FD. The primary outcome was major adverse cardiovascular events (MACEs). MACE risk factors in FD were evaluated using time-dependent Cox regression. Between January 2007 and April 2022, 188 patients with FD were analyzed. Among them, 22 (11.7%) experienced MACE (males: 14/95 [14.7%]; females: 8/93 [8.6%]). The mean age at MACE diagnosis was 53.5 ± 11.0 years in all patients with FD, which was lower in males compared with in females (49.7 ± 9.6 vs. 60.0 ± 10.7 years, <i<p</i< = 0.030). Multivariate analysis (HR, 95% CI) revealed that age (1.042; 1.004–1.082) and duration of FD nontreatment (1.040; 1.003–1.078) were significant MACE risk factors in all patients. In males, age (1.080; 1.032–1.131), FD nontreatment duration (1.099; 1.048–1.152), and keratopathy (18.920; 4.174–85.749) were significant MACE risk factors in multivariate analysis. In females, the only significant MACE risk factor was a high Charlson comorbidity index score (1.795; 1.229–2.622). In conclusion, duration of FD nontreatment and keratopathy are significant MACE risk factors in males with FD. These findings suggest the importance of early initiation of FD-specific treatment and careful evaluation of keratopathy in males with FD.
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title_short	Impact of Nontreatment Duration and Keratopathy on Major Adverse Cardiovascular Events in Fabry Disease: A Nationwide Cohort Study
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up_date	2024-07-03T18:43:12.694Z
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Multivariate analysis (HR, 95% CI) revealed that age (1.042; 1.004–1.082) and duration of FD nontreatment (1.040; 1.003–1.078) were significant MACE risk factors in all patients. In males, age (1.080; 1.032–1.131), FD nontreatment duration (1.099; 1.048–1.152), and keratopathy (18.920; 4.174–85.749) were significant MACE risk factors in multivariate analysis. In females, the only significant MACE risk factor was a high Charlson comorbidity index score (1.795; 1.229–2.622). In conclusion, duration of FD nontreatment and keratopathy are significant MACE risk factors in males with FD. 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Impact of Nontreatment Duration and Keratopathy on Major Adverse Cardiovascular Events in Fabry Disease: A Nationwide Cohort Study

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