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Hughes D, Linhart A, Gurevich A, et al. Prompt Agalsidase Alfa Therapy Initiation is Associated with Improved Renal and Cardiovascular Outcomes in a Fabry Outcome Survey Analysis. Drug Des Devel Ther. 2021;15:3561-3572doi: 10.2147/DDDT.S313789.
Hughes, D., Linhart, A., Gurevich, A., Kalampoki, V., Jazukeviciene, D., Feriozzi, S. (2021). Prompt Agalsidase Alfa Therapy Initiation is Associated with Improved Renal and Cardiovascular Outcomes in a Fabry Outcome Survey Analysis. Drug design, development and therapy, 153561-3572. https://doi.org/10.2147/DDDT.S313789
Hughes, Derralynn, et al. "Prompt Agalsidase Alfa Therapy Initiation is Associated with Improved Renal and Cardiovascular Outcomes in a Fabry Outcome Survey Analysis." Drug design, development and therapy vol. 15 (2021): 3561-3572. doi: https://doi.org/10.2147/DDDT.S313789
Hughes D, Linhart A, Gurevich A, Kalampoki V, Jazukeviciene D, Feriozzi S. Prompt Agalsidase Alfa Therapy Initiation is Associated with Improved Renal and Cardiovascular Outcomes in a Fabry Outcome Survey Analysis. Drug Des Devel Ther. 2021 Aug 16;15:3561-3572. doi: 10.2147/DDDT.S313789. eCollection 2021. PMID: 34429585; PMCID: PMC8379390.
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Drug Des Devel Ther. 2021 Aug 16;15:3561-3572. doi: 10.2147/DDDT.S313789. eCollection 2021.

Prompt Agalsidase Alfa Therapy Initiation is Associated with Improved Renal and Cardiovascular Outcomes in a Fabry Outcome Survey Analysis.

Drug design, development and therapy

Derralynn Hughes, Aleš Linhart, Andrey Gurevich, Vasiliki Kalampoki, Dalia Jazukeviciene, Sandro Feriozzi,

Affiliations

  1. University College London and Royal Free London NHS Foundation Trust, London, UK.
  2. Charles University, First Faculty of Medicine, Prague, Czech Republic.
  3. Takeda Pharmaceuticals International AG, Zurich, Switzerland.
  4. Nephrology and Dialysis Unit, Belcolle Hospital, Department of Nephrology and Dialysis, Viterbo, Italy.

PMID: 34429585 PMCID: PMC8379390 DOI: 10.2147/DDDT.S313789

Abstract

BACKGROUND: The timing of enzyme replacement therapy initiation in patients with Fabry disease is hypothesized to be critical. In this study, we used Fabry Outcome Survey data to assess the impact of prompt versus delayed initiation of treatment with agalsidase alfa on cardiovascular and renal events in patients with Fabry disease.

METHODS: Available genetic data at baseline were used to define patients with mutations associated with classical versus late-onset Fabry disease. Time to cardiovascular or renal events, from treatment initiation until 120 months, was compared for patients in prompt versus delayed groups. "Prompt" was defined as treatment initiation <24 months from symptom onset (analysis A) or diagnosis (analysis B), and "delayed" was defined as ≥24 months from symptom onset (analysis A) or diagnosis (analysis B). Kaplan-Meier curves and Log rank tests compared event-free probabilities and time to first event. Multivariate Cox regression estimated hazard ratios (HRs).

RESULTS: Analysis by time from symptom onset included 1374 patients (172 prompt, 1202 delayed). In a multivariate Cox regression analysis, prompt versus delayed treatment initiation significantly reduced the probability of cardiovascular (HR=0.62;

CONCLUSION: This analysis suggests that prompt treatment initiation with agalsidase alfa provided better renal and cardiovascular outcomes than delayed treatment in patients with Fabry disease.

© 2021 Hughes et al.

Keywords: cardiomyopathies; early diagnosis; mutation; nephrology; therapeutics

Conflict of interest statement

DH reports personal fees from Takeda, Sanofi Genzyme, Amicus Therapeutics, Idorsia, Protalix Biotherapeutics, and Freeline Therapeutics, outside the submitted work. AL reports personal fees from Amicu

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