Long-term follow-up of renal function in patients treated with migalastat for Fabry disease

The effect of migalastat on long-term renal outcomes in enzyme replacement therapy (ERT)-naive and ERT-experienced patients with Fabry disease is not well defined. An integrated posthoc analysis of the phase 3 clinical trials and open-label extension studies was conducted to evaluate long-term chang...

Descripción completa

Detalles Bibliográficos
Autores: Bichet, Daniel G., Torra Balcells, Roser|||0000-0001-8714-2332, Wallace, Eric, Hughes, Derralynn A|||0000-0003-4531-9173, Giugliani, Roberto, Skuban, Nina, Krusinska, Eva, Feldt-Rasmussen, Ulla, Schiffmann, Raphael, Nicholls, Kathy
Tipo de recurso: artículo
Fecha de publicación:2021
País:España
Institución:Universitat Autònoma de Barcelona
Repositorio:Dipòsit Digital de Documents de la UAB
Idioma:inglés
OAI Identifier:oai:ddd.uab.cat:248892
Acceso en línea:https://ddd.uab.cat/record/248892
https://dx.doi.org/urn:doi:10.1016/j.ymgmr.2021.100786
Access Level:acceso abierto
Palabra clave:Classic phenotype
Efficacy
Fabry disease
Chaperone
Migalastat
Renal function
α-Gal A, α-galactosidase A
Gb, globotriaosylceramide
Egfr, estimated glomerular filtration rate using the Chronic Kidney Disease Epidemiology Collaboration equation
GLP-HEK, Good Laboratory Practice-validated human embryonic kidney
LVMi, left ventricular mass index
Q1, quartile 1
Q3, quartile 3
RI, renin inhibitor
id ES_70b39baccb6ca7d6b2bc5fdda4623985
oai_identifier_str oai:ddd.uab.cat:248892
network_acronym_str ES
network_name_str España
repository_id_str
spelling Long-term follow-up of renal function in patients treated with migalastat for Fabry diseaseBichet, Daniel G.Torra Balcells, Roser|||0000-0001-8714-2332Wallace, EricHughes, Derralynn A|||0000-0003-4531-9173Giugliani, RobertoSkuban, NinaKrusinska, EvaFeldt-Rasmussen, UllaSchiffmann, RaphaelNicholls, KathyClassic phenotypeEfficacyFabry diseaseChaperoneMigalastatRenal functionα-Gal A, α-galactosidase AGb, globotriaosylceramideEgfr, estimated glomerular filtration rate using the Chronic Kidney Disease Epidemiology Collaboration equationGLP-HEK, Good Laboratory Practice-validated human embryonic kidneyLVMi, left ventricular mass indexQ1, quartile 1Q3, quartile 3RI, renin inhibitorThe effect of migalastat on long-term renal outcomes in enzyme replacement therapy (ERT)-naive and ERT-experienced patients with Fabry disease is not well defined. An integrated posthoc analysis of the phase 3 clinical trials and open-label extension studies was conducted to evaluate long-term changes in renal function in patients with Fabry disease and amenable GLA variants who were treated with migalastat for ≥2 years during these studies. The analysis included ERT-naive (n = 36 [23 females]; mean age 45 years; mean baseline estimated glomerular filtration rate (eGFR), 91.4 mL/min/mL/1.73 m 2) and ERT-experienced (n = 42 [24 females]; mean age, 50 years; mean baseline eGFR, 89.2 mL/min/1.73m 2) patients with amenable variants who received migalastat 123 mg every other day for ≥2 years. The annualized rate of change from baseline to last observation in estimated glomerular filtration rate using the Chronic Kidney Disease Epidemiology Collaboration equation (eGFR) was calculated by both simple linear regression and a random coefficient model. In ERT-naive patients, mean annualized rates of change from baseline in eGFR were - 1.6 mL/min/1.73 m 2 overall and - 1.8 mL/min/1.73 m 2 and - 1.4 mL/min/1.73 m 2 in male and female patients, respectively, as estimated by simple linear regression. In ERT-experienced patients, mean annualized rates of change from baseline in eGFR were - 1.6 mL/min/1.73 m 2 overall and - 2.6 mL/min/1.73 m 2 and - 0.8 mL/min/1.73 m 2 in male and female patients, respectively. Mean annualized rate of change in eGFR in ERT-naive patients with the classic phenotype (defined by white blood cell alpha galactosidase A [α-Gal A] activity of <3% of normal and multiorgan system involvement) was -1.7 mL/min/1.73 m 2. When calculated using the random coefficient model, which adjusted for sex, age, and baseline renal function, the annualized eGFR change was minimal (mean: -0.1 and 0.1 mL/min/1.73 m 2 in ERT-naive and ERT-experienced patients, respectively). In conclusion, patients with Fabry disease and amenable GLA variants receiving long-term migalastat treatment (≤8.6 years) maintained renal function irrespective of treatment status, sex, or phenotype.Universitat Autònoma de Barcelona 22021-01-0120212021-01-01Articlehttp://purl.org/coar/resource_type/c_6501VoRhttp://purl.org/coar/version/c_970fb48d4fbd8a85info:eu-repo/semantics/articleapplication/pdfhttps://ddd.uab.cat/record/248892https://dx.doi.org/urn:doi:10.1016/j.ymgmr.2021.100786reponame:Dipòsit Digital de Documents de la UABinstname:Universitat Autònoma de BarcelonaInglésengopen accesshttp://purl.org/coar/access_right/c_abf2Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, i la comunicació pública de l'obra, sempre que no sigui amb finalitats comercials, i sempre que es reconegui l'autoria de l'obra original. No es permet la creació d'obres derivades.https://creativecommons.org/licenses/by-nc-nd/4.0/info:eu-repo/semantics/openAccessoai:ddd.uab.cat:2488922026-06-06T12:50:31Z
dc.title.none.fl_str_mv Long-term follow-up of renal function in patients treated with migalastat for Fabry disease
title Long-term follow-up of renal function in patients treated with migalastat for Fabry disease
spellingShingle Long-term follow-up of renal function in patients treated with migalastat for Fabry disease
Bichet, Daniel G.
Classic phenotype
Efficacy
Fabry disease
Chaperone
Migalastat
Renal function
α-Gal A, α-galactosidase A
Gb, globotriaosylceramide
Egfr, estimated glomerular filtration rate using the Chronic Kidney Disease Epidemiology Collaboration equation
GLP-HEK, Good Laboratory Practice-validated human embryonic kidney
LVMi, left ventricular mass index
Q1, quartile 1
Q3, quartile 3
RI, renin inhibitor
title_short Long-term follow-up of renal function in patients treated with migalastat for Fabry disease
title_full Long-term follow-up of renal function in patients treated with migalastat for Fabry disease
title_fullStr Long-term follow-up of renal function in patients treated with migalastat for Fabry disease
title_full_unstemmed Long-term follow-up of renal function in patients treated with migalastat for Fabry disease
title_sort Long-term follow-up of renal function in patients treated with migalastat for Fabry disease
dc.creator.none.fl_str_mv Bichet, Daniel G.
Torra Balcells, Roser|||0000-0001-8714-2332
Wallace, Eric
Hughes, Derralynn A|||0000-0003-4531-9173
Giugliani, Roberto
Skuban, Nina
Krusinska, Eva
Feldt-Rasmussen, Ulla
Schiffmann, Raphael
Nicholls, Kathy
author Bichet, Daniel G.
author_facet Bichet, Daniel G.
Torra Balcells, Roser|||0000-0001-8714-2332
Wallace, Eric
Hughes, Derralynn A|||0000-0003-4531-9173
Giugliani, Roberto
Skuban, Nina
Krusinska, Eva
Feldt-Rasmussen, Ulla
Schiffmann, Raphael
Nicholls, Kathy
author_role author
author2 Torra Balcells, Roser|||0000-0001-8714-2332
Wallace, Eric
Hughes, Derralynn A|||0000-0003-4531-9173
Giugliani, Roberto
Skuban, Nina
Krusinska, Eva
Feldt-Rasmussen, Ulla
Schiffmann, Raphael
Nicholls, Kathy
author2_role author
author
author
author
author
author
author
author
author
dc.contributor.none.fl_str_mv Universitat Autònoma de Barcelona
dc.subject.none.fl_str_mv Classic phenotype
Efficacy
Fabry disease
Chaperone
Migalastat
Renal function
α-Gal A, α-galactosidase A
Gb, globotriaosylceramide
Egfr, estimated glomerular filtration rate using the Chronic Kidney Disease Epidemiology Collaboration equation
GLP-HEK, Good Laboratory Practice-validated human embryonic kidney
LVMi, left ventricular mass index
Q1, quartile 1
Q3, quartile 3
RI, renin inhibitor
topic Classic phenotype
Efficacy
Fabry disease
Chaperone
Migalastat
Renal function
α-Gal A, α-galactosidase A
Gb, globotriaosylceramide
Egfr, estimated glomerular filtration rate using the Chronic Kidney Disease Epidemiology Collaboration equation
GLP-HEK, Good Laboratory Practice-validated human embryonic kidney
LVMi, left ventricular mass index
Q1, quartile 1
Q3, quartile 3
RI, renin inhibitor
description The effect of migalastat on long-term renal outcomes in enzyme replacement therapy (ERT)-naive and ERT-experienced patients with Fabry disease is not well defined. An integrated posthoc analysis of the phase 3 clinical trials and open-label extension studies was conducted to evaluate long-term changes in renal function in patients with Fabry disease and amenable GLA variants who were treated with migalastat for ≥2 years during these studies. The analysis included ERT-naive (n = 36 [23 females]; mean age 45 years; mean baseline estimated glomerular filtration rate (eGFR), 91.4 mL/min/mL/1.73 m 2) and ERT-experienced (n = 42 [24 females]; mean age, 50 years; mean baseline eGFR, 89.2 mL/min/1.73m 2) patients with amenable variants who received migalastat 123 mg every other day for ≥2 years. The annualized rate of change from baseline to last observation in estimated glomerular filtration rate using the Chronic Kidney Disease Epidemiology Collaboration equation (eGFR) was calculated by both simple linear regression and a random coefficient model. In ERT-naive patients, mean annualized rates of change from baseline in eGFR were - 1.6 mL/min/1.73 m 2 overall and - 1.8 mL/min/1.73 m 2 and - 1.4 mL/min/1.73 m 2 in male and female patients, respectively, as estimated by simple linear regression. In ERT-experienced patients, mean annualized rates of change from baseline in eGFR were - 1.6 mL/min/1.73 m 2 overall and - 2.6 mL/min/1.73 m 2 and - 0.8 mL/min/1.73 m 2 in male and female patients, respectively. Mean annualized rate of change in eGFR in ERT-naive patients with the classic phenotype (defined by white blood cell alpha galactosidase A [α-Gal A] activity of <3% of normal and multiorgan system involvement) was -1.7 mL/min/1.73 m 2. When calculated using the random coefficient model, which adjusted for sex, age, and baseline renal function, the annualized eGFR change was minimal (mean: -0.1 and 0.1 mL/min/1.73 m 2 in ERT-naive and ERT-experienced patients, respectively). In conclusion, patients with Fabry disease and amenable GLA variants receiving long-term migalastat treatment (≤8.6 years) maintained renal function irrespective of treatment status, sex, or phenotype.
publishDate 2021
dc.date.none.fl_str_mv 2
2021-01-01
2021
2021-01-01
dc.type.none.fl_str_mv Article
http://purl.org/coar/resource_type/c_6501
VoR
http://purl.org/coar/version/c_970fb48d4fbd8a85
dc.type.openaire.fl_str_mv info:eu-repo/semantics/article
format article
dc.identifier.none.fl_str_mv https://ddd.uab.cat/record/248892
https://dx.doi.org/urn:doi:10.1016/j.ymgmr.2021.100786
url https://ddd.uab.cat/record/248892
https://dx.doi.org/urn:doi:10.1016/j.ymgmr.2021.100786
dc.language.none.fl_str_mv Inglés
eng
language_invalid_str_mv Inglés
language eng
dc.rights.none.fl_str_mv open access
http://purl.org/coar/access_right/c_abf2
https://creativecommons.org/licenses/by-nc-nd/4.0/
dc.rights.openaire.fl_str_mv info:eu-repo/semantics/openAccess
rights_invalid_str_mv open access
http://purl.org/coar/access_right/c_abf2
https://creativecommons.org/licenses/by-nc-nd/4.0/
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.source.none.fl_str_mv reponame:Dipòsit Digital de Documents de la UAB
instname:Universitat Autònoma de Barcelona
instname_str Universitat Autònoma de Barcelona
reponame_str Dipòsit Digital de Documents de la UAB
collection Dipòsit Digital de Documents de la UAB
repository.name.fl_str_mv
repository.mail.fl_str_mv
_version_ 1869410599557922816
score 15,301603

Similares en LA Referencia