Cardio-renal outcomes with long-term agalsidase alfa enzyme replacement therapy: A 10-year fabry outcome survey (FOS) analysis

FOS Study Group

doi:10.2147/DDDT.S207856

Cardio-renal outcomes with long-term agalsidase alfa enzyme replacement therapy: A 10-year fabry outcome survey (FOS) analysis

FOS Study Group

Medicine

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35 Citations (Scopus)

Abstract

Purpose: Following the publication of 5-year agalsidase alfa enzyme replacement therapy (ERT) outcomes data from the Fabry Outcome Survey (FOS), 10-year data were analyzed. Patients and methods: FOS (ClinicalTrials.gov identifier: NCT03289065) data (April 2001 to August 2018) were retrospectively analyzed. Estimated glomerular filtration rate (eGFR) and left ventricular mass indexed to height (LVMI) were analyzed after treatment start (baseline) for patients with ≥3 measurements, including baseline and year 10. Results: Median (range) age (years) of the evaluable treated renal cohort at treatment start was 48.8 (17.9–67.3) for females (n=62), 34.4 (18.0–66.8) for males (n=90). With eGFR ≥60 mL/min/1.73 m² at baseline, mean (95% CI) rate of eGFR change (eGFR/year) over 10 years was relatively stable in females (n=52; −0.55 [−1.12, +0.01]) and slightly declined in males (n=79; −1.99 [−2.45, −1.54]). With impaired kidney function (eGFR <60 mL/min/1.73 m²) at baseline, mean (95% CI) eGFR/year was stable in females (n=10; −0.14 [−1.43, +1.15]) and slightly declined in males (n=11; −2.79 [−4.01, −1.56]) over 10 years. Median (range) age (years) of the evaluable treated cardiac cohort at treatment start was 46.7 (3.7–67.3) for females (n=34), 28.2 (4.0–54.2) for males (n=35). With left ventricular hypertrophy (LVH; LVMI >48 g/m^2.7 in females, >50 g/m^2.7 in males) at baseline, mean (95% CI) LVMI/year slightly increased over 10 years in females (n=18; +1.51 [+0.91, +2.12]) and males (n=14; +0.87 (+0.19, +1.55). Without LVH at baseline, mean (95% CI) LVMI/year was stable in females (n=16; +0.52 [−0.13, +1.17]) and males (n=21; +0.57 [+0.02, +1.13]) over 10 years. Conclusion: Agalsidase alfa-treated patients with 10-year FOS data and preserved kidney function and/or normal LVMI at baseline remained largely stable; those with decreased kidney function or LVH at baseline experienced modest declines in renal function and/or increases in LVMI.

Original language	English
Pages (from-to)	3705-3715
Number of pages	11
Journal	Drug Design, Development and Therapy
Volume	13
DOIs	https://doi.org/10.2147/DDDT.S207856
Publication status	Published - 2019

Bibliographical note

Funding Information:
Under the direction of the authors, Margit Rezabek, DVM, PhD, Sally Hassan, PhD, CMPP, and Latoya M. Mitchell, PhD, CMPP, employees of Excel Medical Affairs, provided writing assistance for this publication. Editorial assistance in formatting, proofreading, copy editing, and fact checking was also provided by Excel Medical Affairs. Statistical support was provided by Patrick Engrand, PhD, of Clinical Research Services – ICC, under contract to Shire. This paper was presented at the WORLDSymposium 2016 as a poster presentation with interim findings. The poster’s abstract was published in Ramaswami U, et al Mol Genet Metab 2016;117(2):S98. https://doi.org/10.1016/j.ymgme. 2015.12.416.

Publisher Copyright:
© 2019 Ramaswami et al.

ASJC Scopus Subject Areas

Pharmacology
Pharmaceutical Science
Drug Discovery

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10.2147/DDDT.S207856

Cite this

@article{1972621f62844248a4975d9a5511f319,

title = "Cardio-renal outcomes with long-term agalsidase alfa enzyme replacement therapy: A 10-year fabry outcome survey (FOS) analysis",

abstract = "Purpose: Following the publication of 5-year agalsidase alfa enzyme replacement therapy (ERT) outcomes data from the Fabry Outcome Survey (FOS), 10-year data were analyzed. Patients and methods: FOS (ClinicalTrials.gov identifier: NCT03289065) data (April 2001 to August 2018) were retrospectively analyzed. Estimated glomerular filtration rate (eGFR) and left ventricular mass indexed to height (LVMI) were analyzed after treatment start (baseline) for patients with ≥3 measurements, including baseline and year 10. Results: Median (range) age (years) of the evaluable treated renal cohort at treatment start was 48.8 (17.9–67.3) for females (n=62), 34.4 (18.0–66.8) for males (n=90). With eGFR ≥60 mL/min/1.73 m2 at baseline, mean (95% CI) rate of eGFR change (eGFR/year) over 10 years was relatively stable in females (n=52; −0.55 [−1.12, +0.01]) and slightly declined in males (n=79; −1.99 [−2.45, −1.54]). With impaired kidney function (eGFR <60 mL/min/1.73 m2) at baseline, mean (95% CI) eGFR/year was stable in females (n=10; −0.14 [−1.43, +1.15]) and slightly declined in males (n=11; −2.79 [−4.01, −1.56]) over 10 years. Median (range) age (years) of the evaluable treated cardiac cohort at treatment start was 46.7 (3.7–67.3) for females (n=34), 28.2 (4.0–54.2) for males (n=35). With left ventricular hypertrophy (LVH; LVMI >48 g/m2.7 in females, >50 g/m2.7 in males) at baseline, mean (95% CI) LVMI/year slightly increased over 10 years in females (n=18; +1.51 [+0.91, +2.12]) and males (n=14; +0.87 (+0.19, +1.55). Without LVH at baseline, mean (95% CI) LVMI/year was stable in females (n=16; +0.52 [−0.13, +1.17]) and males (n=21; +0.57 [+0.02, +1.13]) over 10 years. Conclusion: Agalsidase alfa-treated patients with 10-year FOS data and preserved kidney function and/or normal LVMI at baseline remained largely stable; those with decreased kidney function or LVH at baseline experienced modest declines in renal function and/or increases in LVMI.",

author = "{FOS Study Group} and Uma Ramaswami and Michael Beck and Derralynn Hughes and Christoph Kampmann and Jaco Botha and Guillem Pintos-Morell and West, {Michael L.} and Niu, {Dau Ming} and Kathy Nicholls and Roberto Giugliani",

note = "Funding Information: Under the direction of the authors, Margit Rezabek, DVM, PhD, Sally Hassan, PhD, CMPP, and Latoya M. Mitchell, PhD, CMPP, employees of Excel Medical Affairs, provided writing assistance for this publication. Editorial assistance in formatting, proofreading, copy editing, and fact checking was also provided by Excel Medical Affairs. Statistical support was provided by Patrick Engrand, PhD, of Clinical Research Services – ICC, under contract to Shire. This paper was presented at the WORLDSymposium 2016 as a poster presentation with interim findings. The poster{\textquoteright}s abstract was published in Ramaswami U, et al Mol Genet Metab 2016;117(2):S98. https://doi.org/10.1016/j.ymgme. 2015.12.416. Publisher Copyright: {\textcopyright} 2019 Ramaswami et al.",

year = "2019",

doi = "10.2147/DDDT.S207856",

language = "English",

volume = "13",

pages = "3705--3715",

journal = "Drug Design, Development and Therapy",

issn = "1177-8881",

publisher = "Dove Medical Press Ltd.",

}

TY - JOUR

T1 - Cardio-renal outcomes with long-term agalsidase alfa enzyme replacement therapy

T2 - A 10-year fabry outcome survey (FOS) analysis

AU - FOS Study Group

AU - Ramaswami, Uma

AU - Beck, Michael

AU - Hughes, Derralynn

AU - Kampmann, Christoph

AU - Botha, Jaco

AU - Pintos-Morell, Guillem

AU - West, Michael L.

AU - Niu, Dau Ming

AU - Nicholls, Kathy

AU - Giugliani, Roberto

N1 - Funding Information: Under the direction of the authors, Margit Rezabek, DVM, PhD, Sally Hassan, PhD, CMPP, and Latoya M. Mitchell, PhD, CMPP, employees of Excel Medical Affairs, provided writing assistance for this publication. Editorial assistance in formatting, proofreading, copy editing, and fact checking was also provided by Excel Medical Affairs. Statistical support was provided by Patrick Engrand, PhD, of Clinical Research Services – ICC, under contract to Shire. This paper was presented at the WORLDSymposium 2016 as a poster presentation with interim findings. The poster’s abstract was published in Ramaswami U, et al Mol Genet Metab 2016;117(2):S98. https://doi.org/10.1016/j.ymgme. 2015.12.416. Publisher Copyright: © 2019 Ramaswami et al.

PY - 2019

Y1 - 2019

N2 - Purpose: Following the publication of 5-year agalsidase alfa enzyme replacement therapy (ERT) outcomes data from the Fabry Outcome Survey (FOS), 10-year data were analyzed. Patients and methods: FOS (ClinicalTrials.gov identifier: NCT03289065) data (April 2001 to August 2018) were retrospectively analyzed. Estimated glomerular filtration rate (eGFR) and left ventricular mass indexed to height (LVMI) were analyzed after treatment start (baseline) for patients with ≥3 measurements, including baseline and year 10. Results: Median (range) age (years) of the evaluable treated renal cohort at treatment start was 48.8 (17.9–67.3) for females (n=62), 34.4 (18.0–66.8) for males (n=90). With eGFR ≥60 mL/min/1.73 m2 at baseline, mean (95% CI) rate of eGFR change (eGFR/year) over 10 years was relatively stable in females (n=52; −0.55 [−1.12, +0.01]) and slightly declined in males (n=79; −1.99 [−2.45, −1.54]). With impaired kidney function (eGFR <60 mL/min/1.73 m2) at baseline, mean (95% CI) eGFR/year was stable in females (n=10; −0.14 [−1.43, +1.15]) and slightly declined in males (n=11; −2.79 [−4.01, −1.56]) over 10 years. Median (range) age (years) of the evaluable treated cardiac cohort at treatment start was 46.7 (3.7–67.3) for females (n=34), 28.2 (4.0–54.2) for males (n=35). With left ventricular hypertrophy (LVH; LVMI >48 g/m2.7 in females, >50 g/m2.7 in males) at baseline, mean (95% CI) LVMI/year slightly increased over 10 years in females (n=18; +1.51 [+0.91, +2.12]) and males (n=14; +0.87 (+0.19, +1.55). Without LVH at baseline, mean (95% CI) LVMI/year was stable in females (n=16; +0.52 [−0.13, +1.17]) and males (n=21; +0.57 [+0.02, +1.13]) over 10 years. Conclusion: Agalsidase alfa-treated patients with 10-year FOS data and preserved kidney function and/or normal LVMI at baseline remained largely stable; those with decreased kidney function or LVH at baseline experienced modest declines in renal function and/or increases in LVMI.

AB - Purpose: Following the publication of 5-year agalsidase alfa enzyme replacement therapy (ERT) outcomes data from the Fabry Outcome Survey (FOS), 10-year data were analyzed. Patients and methods: FOS (ClinicalTrials.gov identifier: NCT03289065) data (April 2001 to August 2018) were retrospectively analyzed. Estimated glomerular filtration rate (eGFR) and left ventricular mass indexed to height (LVMI) were analyzed after treatment start (baseline) for patients with ≥3 measurements, including baseline and year 10. Results: Median (range) age (years) of the evaluable treated renal cohort at treatment start was 48.8 (17.9–67.3) for females (n=62), 34.4 (18.0–66.8) for males (n=90). With eGFR ≥60 mL/min/1.73 m2 at baseline, mean (95% CI) rate of eGFR change (eGFR/year) over 10 years was relatively stable in females (n=52; −0.55 [−1.12, +0.01]) and slightly declined in males (n=79; −1.99 [−2.45, −1.54]). With impaired kidney function (eGFR <60 mL/min/1.73 m2) at baseline, mean (95% CI) eGFR/year was stable in females (n=10; −0.14 [−1.43, +1.15]) and slightly declined in males (n=11; −2.79 [−4.01, −1.56]) over 10 years. Median (range) age (years) of the evaluable treated cardiac cohort at treatment start was 46.7 (3.7–67.3) for females (n=34), 28.2 (4.0–54.2) for males (n=35). With left ventricular hypertrophy (LVH; LVMI >48 g/m2.7 in females, >50 g/m2.7 in males) at baseline, mean (95% CI) LVMI/year slightly increased over 10 years in females (n=18; +1.51 [+0.91, +2.12]) and males (n=14; +0.87 (+0.19, +1.55). Without LVH at baseline, mean (95% CI) LVMI/year was stable in females (n=16; +0.52 [−0.13, +1.17]) and males (n=21; +0.57 [+0.02, +1.13]) over 10 years. Conclusion: Agalsidase alfa-treated patients with 10-year FOS data and preserved kidney function and/or normal LVMI at baseline remained largely stable; those with decreased kidney function or LVH at baseline experienced modest declines in renal function and/or increases in LVMI.

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JO - Drug Design, Development and Therapy

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ER -

Cardio-renal outcomes with long-term agalsidase alfa enzyme replacement therapy: A 10-year fabry outcome survey (FOS) analysis

Abstract

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