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

References

• MehtaA, ClarkeJTR, GiuglianiR, et al. Natural course of Fabry disease: changing pattern of causes of death in FOS – Fabry Outcome Survey. J Med Genet. 2009;46(8):548–552. doi:10.1136/jmg.2008.06590419473999
   PubMed Web of Science ®Google Scholar
• MehtaA, RicciR, WidmerU, et al. Fabry disease defined: baseline clinical manifestations of 366 patients in the Fabry Outcome Survey. Eur J Clin Invest. 2004;34(3):236–242. doi:10.1111/j.1365-2362.2004.01309.x15025684
   PubMed Web of Science ®Google Scholar
• MehtaA, BeckM, ElliottP, et al. Enzyme replacement therapy with agalsidase alfa in patients with Fabry’s disease: an analysis of registry data. Lancet. 2009;374(9706):1986–1996. doi:10.1016/S0140-6736(09)61493-819959221
   PubMed Web of Science ®Google Scholar
• BeckM, HughesD, KampmannC, et al. Long-term effectiveness of agalsidase alfa enzyme replacement in Fabry disease: a Fabry Outcome Survey analysis. Mol Genet Metab Rep. 2015;3:21–27. doi:10.1016/j.ymgmr.2015.02.00226937390
   PubMed Web of Science ®Google Scholar
• HughesDA, ElliottPM, ShahJ, et al. Effects of enzyme replacement therapy on the cardiomyopathy of Anderson–Fabry disease: a randomised, double-blind, placebo-controlled clinical trial of agalsidase alfa. Heart. 2008;94(2):153–158. doi:10.1136/hrt.2006.10402617483124
   PubMed Web of Science ®Google Scholar
• FellgiebelA, GartenschlägerM, WildbergerK, ScheurichA, DesnickRJ, SimsK. Enzyme replacement therapy stabilized white matter lesion progression in Fabry disease. Cerebrovasc Dis. 2014;38(6):448–456. doi:10.1159/00036929325502511
   PubMed Web of Science ®Google Scholar
• SchiffmannR, KoppJB, AustinHA, et al. Enzyme replacement therapy in Fabry disease: a randomized controlled trial. JAMA. 2001;285(21):2743–2749. doi:10.1001/jama.285.21.274311386930
   PubMed Web of Science ®Google Scholar
• WestM, NichollsK, MehtaA, et al. Agalsidase alfa and kidney dysfunction in Fabry disease. J Am Soc Nephrol. 2009;20(5):1132–1139. doi:10.1681/ASN.200808087019357250
   PubMed Web of Science ®Google Scholar
• ReisinR, PerrinA, García‐PavíaP. Time delays in the diagnosis and treatment of Fabry disease. Int J Clin Pract. 2017;71:e12914. doi:10.1111/ijcp.12914
   Web of Science ®Google Scholar
• OrtizA, AbioseA, BichetDG, et al. Time to treatment benefit for adult patients with Fabry disease receiving agalsidase β: data from the Fabry Registry. J Med Genet. 2016;53(7):495–502. doi:10.1136/jmedgenet-2015-10348626993266
   PubMed Web of Science ®Google Scholar
• GermainDP, CharrowJ, DesnickRJ, et al. Ten-year outcome of enzyme replacement therapy with agalsidase beta in patients with Fabry disease. J Med Genet. 2015;52(5):353–358. doi:10.1136/jmedgenet-2014-10279725795794
   PubMed Web of Science ®Google Scholar
• HughesDA. Fabry disease: will markers of early disease enable early treatment and better outcomes?Curr Opin Cardiol. 2016;31(4):434–439. doi:10.1097/HCO.000000000000030827205888
   PubMed Web of Science ®Google Scholar
• FeriozziS, LinhartA, RamaswamiU, et al. Effects of baseline left ventricular hypertrophy and decreased renal function on cardiovascular and renal outcomes in patients with Fabry disease treated with agalsidase alfa: a Fabry Outcome Survey Study. Clin Ther. 2020;42(12):2321–2330. doi:10.1016/j.clinthera.2020.10.00733218740
   PubMed Web of Science ®Google Scholar
• PariniR, Pintos-MorellG, HennermannJB, et al. Analysis of renal and cardiac outcomes in male participants in the Fabry Outcome Survey starting agalsidase alfa enzyme replacement therapy before and after 18 years of age. Drug Des Devel Ther. 2020;14:2149–2158. doi:10.2147/DDDT.S249433
   PubMed Web of Science ®Google Scholar
• BeckM, RicciR, WidmerU, et al. Fabry disease: overall effects of agalsidase alfa treatment. Eur J Clin Invest. 2004;34(12):838–844. doi:10.1111/j.1365-2362.2004.01424.x15606727
   PubMed Web of Science ®Google Scholar
• FeriozziS, TorrasJ, CybullaM, NichollsK, Sunder-PlassmannG, WestM. The effectiveness of long-term agalsidase alfa therapy in the treatment of Fabry nephropathy. Clin J Am Soc Nephrol. 2012;7(1):60–69. doi:10.2215/CJN.0313041122246281
   PubMed Web of Science ®Google Scholar
• HughesDA, Barba RomeroM-Á, HollakCEM, GiuglianiR, DeeganPB. Response of women with Fabry disease to enzyme replacement therapy: comparison with men, using data from FOS—the Fabry Outcome Survey. Mol Genet Metab. 2011;103(3):207–214. doi:10.1016/j.ymgme.2011.03.02221543245
   PubMed Web of Science ®Google Scholar
• DevereuxRB, AlonsoDR, LutasEM, et al. Echocardiographic assessment of left ventricular hypertrophy: comparison to necropsy findings. Am J Cardiol. 1986;57(6):450–458. doi:10.1016/0002-9149(86)90771-X2936235
   PubMed Web of Science ®Google Scholar
• KampmannC, LinhartA, BaehnerF, et al. Onset and progression of the Anderson–Fabry disease related cardiomyopathy. Int J Cardiol. 2008;130(3):367–373. doi:10.1016/j.ijcard.2008.03.00718572264
   PubMed Web of Science ®Google Scholar
• de SimoneG, DevereuxRB, DanielsSR, KorenMJ, MeyerRA, LaraghJH. Effect of growth on variability of left ventricular mass: assessment of allometric signals in adults and children and their capacity to predict cardiovascular risk. J Am Coll Cardiol. 1995;25(5):1056–1062. doi:10.1016/0735-1097(94)00540-77897116
   PubMed Web of Science ®Google Scholar
• StevensLA, SchmidCH, GreeneT, et al. Comparative performance of the CKD Epidemiology Collaboration (CKD-EPI) and the Modification of Diet in Renal Disease (MDRD) Study equations for estimating GFR levels above 60 mL/min/1.73 m2. Am J Kidney Dis. 2010;56(3):486–495. doi:10.1053/j.ajkd.2010.03.02620557989
   PubMed Web of Science ®Google Scholar
• RombachSM, SmidBE, BouwmanMG, LinthorstGE, DijkgraafMGW, HollakCEM. Long term enzyme replacement therapy for Fabry disease: effectiveness on kidney, heart and brain. Orphanet J Rare Dis. 2013;8:47. doi:10.1186/1750-1172-8-4723531228
   PubMed Web of Science ®Google Scholar
• GermainDP, WeidemannF, AbioseA, et al. Analysis of left ventricular mass in untreated men and in men treated with agalsidase-β: data from the Fabry Registry. Genet Med. 2013;15(12):958–965. doi:10.1038/gim.2013.5323703683
   PubMed Web of Science ®Google Scholar
• ArendsM, WijburgFA, WannerC, et al. Favourable effect of early versus late start of enzyme replacement therapy on plasma globotriaosylsphingosine levels in men with classical Fabry disease. Mol Genet Metab. 2017;121(2):157–161. doi:10.1016/j.ymgme.2017.05.00128495078
   PubMed Web of Science ®Google Scholar
• RamaswamiU, PariniR, Pintos-MorellG, KalkumG, KampmannC, BeckM. Fabry disease in children and response to enzyme replacement therapy: results from the Fabry Outcome Survey. Clin Genet. 2012;81(5):485–490. doi:10.1111/j.1399-0004.2011.01671.x21457233
   PubMed Web of Science ®Google Scholar
• WarnockDG, OrtizA, MauerM, et al. Renal outcomes of agalsidase beta treatment for Fabry disease: role of proteinuria and timing of treatment initiation. Nephrol Dial Transplant. 2012;27(3):1042–1049. doi:10.1093/ndt/gfr42021804088
   PubMed Web of Science ®Google Scholar
• BanikazemiM, BultasJ, WaldekS, et al. Agalsidase-beta therapy for advanced Fabry disease: a randomized trial. Ann Intern Med. 2007;146(2):77–86. doi:10.7326/0003-4819-146-2-200701160-0014817179052
   PubMed Web of Science ®Google Scholar
• WeidemannF, NiemannM, BreunigF, et al. Long-term effects of enzyme replacement therapy on Fabry cardiomyopathy: evidence for a better outcome with early treatment. Circulation. 2009;119(4):524–529. doi:10.1161/CIRCULATIONAHA.108.79452919153271
   PubMed Web of Science ®Google Scholar