• Je něco špatně v tomto záznamu ?

Clinical outcomes in patients switching from agalsidase beta to migalastat: A Fabry Registry analysis

A. Pisani, KM. Wilson, JL. Batista, I. Kantola, A. Ortiz, J. Politei, L. Al-Shaar, M. Maski, A. Crespo, E. Ponce, A. Linhart

. 2024 ; 47 (5) : 1080-1095. [pub] 20240704

Jazyk angličtina Země Spojené státy americké

Typ dokumentu časopisecké články, práce podpořená grantem

Perzistentní odkaz   https://www.medvik.cz/link/bmc25004132

Grantová podpora
Sanofi

Fabry Registry data were analyzed among 83 agalsidase beta-treated patients with Fabry disease who switched to migalastat. Outcomes (estimated glomerular filtration rate [eGFR], urine protein-creatinine ratio [UPCR], plasma globotriaosylceramide [GL-3], plasma globotriaosylsphingosine [lyso-GL-3], interventricular septal wall thickness [IVST], left posterior wall thickness [LPWT], left ventricular mass index [LVMI]) were assessed using linear mixed models to estimate annual change over time in the pre- and postswitch periods. eGFR decreased throughout both periods (preswitch: -0.85 mL/min/1.73 m2/year; postswitch: -1.96 mL/min/1.73 m2/year; both p < 0.0001), with steeper decline postswitch (ppre/post = 0.01) in both classic and late-onset patients. UPCR increased significantly postswitch (ppre/post = 0.003) among classic patients and was stable in both periods among late-onset patients. GL-3 trajectories worsened postswitch across phenotypes (ppre/post = 0.0005 classic, 0.02 late-onset). LPWT was stable preswitch (0.07 mm/year, p = 0.25) and decreased postswitch (-0.51 mm/year, p = 0.0005; ppre/post = 0.0009), primarily among late-onset patients. IVST and LVMI slopes varied significantly by phenotype. Among classic patients, IVST and LVMI were stable and decreasing, respectively preswitch and increasing postswitch (ppre/post = 0.02 IVST, 0.01 LVMI). Among late-onset patients, IVST significantly decreased postswitch (ppre/post = 0.0003); LVMI was stable over time (ppre/post = 0.89). Ultimately, eGFR and GL-3 trajectories worsened postswitch across phenotypes, while UPCR and cardiac measures worsened among classic and stabilized/improved among late-onset patients. These findings indicate variability in long-term outcomes after switching from ERT to migalastat, underscoring the importance of careful monitoring.

Citace poskytuje Crossref.org

000      
00000naa a2200000 a 4500
001      
bmc25004132
003      
CZ-PrNML
005      
20250206105944.0
007      
ta
008      
250121s2024 xxu f 000 0|eng||
009      
AR
024    7_
$a 10.1002/jimd.12773 $2 doi
035    __
$a (PubMed)38961737
040    __
$a ABA008 $b cze $d ABA008 $e AACR2
041    0_
$a eng
044    __
$a xxu
100    1_
$a Pisani, Antonio $u Department of Public Health, University of Naples Federico II, Naples, Italy
245    10
$a Clinical outcomes in patients switching from agalsidase beta to migalastat: A Fabry Registry analysis / $c A. Pisani, KM. Wilson, JL. Batista, I. Kantola, A. Ortiz, J. Politei, L. Al-Shaar, M. Maski, A. Crespo, E. Ponce, A. Linhart
520    9_
$a Fabry Registry data were analyzed among 83 agalsidase beta-treated patients with Fabry disease who switched to migalastat. Outcomes (estimated glomerular filtration rate [eGFR], urine protein-creatinine ratio [UPCR], plasma globotriaosylceramide [GL-3], plasma globotriaosylsphingosine [lyso-GL-3], interventricular septal wall thickness [IVST], left posterior wall thickness [LPWT], left ventricular mass index [LVMI]) were assessed using linear mixed models to estimate annual change over time in the pre- and postswitch periods. eGFR decreased throughout both periods (preswitch: -0.85 mL/min/1.73 m2/year; postswitch: -1.96 mL/min/1.73 m2/year; both p < 0.0001), with steeper decline postswitch (ppre/post = 0.01) in both classic and late-onset patients. UPCR increased significantly postswitch (ppre/post = 0.003) among classic patients and was stable in both periods among late-onset patients. GL-3 trajectories worsened postswitch across phenotypes (ppre/post = 0.0005 classic, 0.02 late-onset). LPWT was stable preswitch (0.07 mm/year, p = 0.25) and decreased postswitch (-0.51 mm/year, p = 0.0005; ppre/post = 0.0009), primarily among late-onset patients. IVST and LVMI slopes varied significantly by phenotype. Among classic patients, IVST and LVMI were stable and decreasing, respectively preswitch and increasing postswitch (ppre/post = 0.02 IVST, 0.01 LVMI). Among late-onset patients, IVST significantly decreased postswitch (ppre/post = 0.0003); LVMI was stable over time (ppre/post = 0.89). Ultimately, eGFR and GL-3 trajectories worsened postswitch across phenotypes, while UPCR and cardiac measures worsened among classic and stabilized/improved among late-onset patients. These findings indicate variability in long-term outcomes after switching from ERT to migalastat, underscoring the importance of careful monitoring.
650    _2
$a lidé $7 D006801
650    12
$a Fabryho nemoc $x farmakoterapie $7 D000795
650    _2
$a mužské pohlaví $7 D008297
650    _2
$a ženské pohlaví $7 D005260
650    12
$a registrace $7 D012042
650    _2
$a dospělí $7 D000328
650    12
$a 1-deoxynojirimycin $x analogy a deriváty $x terapeutické užití $x aplikace a dávkování $7 D017485
650    12
$a alfa-galaktosidasa $x terapeutické užití $7 D000519
650    _2
$a lidé středního věku $7 D008875
650    12
$a hodnoty glomerulární filtrace $7 D005919
650    12
$a izoenzymy $x terapeutické užití $7 D007527
650    _2
$a výsledek terapie $7 D016896
650    _2
$a enzymová substituční terapie $x metody $7 D056947
650    _2
$a mladý dospělý $7 D055815
650    _2
$a trihexosylceramidy $x metabolismus $7 D014281
650    _2
$a mladiství $7 D000293
650    _2
$a sfingolipidy $x krev $7 D013107
650    _2
$a glykolipidy $7 D006017
655    _2
$a časopisecké články $7 D016428
655    _2
$a práce podpořená grantem $7 D013485
700    1_
$a Wilson, Kathryn M $u Navitas Data Sciences, Pottstown, Pennsylvania, USA
700    1_
$a Batista, Julie L $u Sanofi, Cambridge, Massachusetts, USA
700    1_
$a Kantola, Ilkka $u Division of Medicine, Turku University Hospital, Turku University, Turku, Finland
700    1_
$a Ortiz, Alberto $u Jiménez Díaz Foundation University Hospital and IIS-Fundación Jiménez Díaz UAM, Madrid, Spain $u Department of Medicine, Universidad Autónoma de Madrid, Madrid, Spain
700    1_
$a Politei, Juan $u Neurology Department, Fundación SPINE, Buenos Aires, Argentina
700    1_
$a Al-Shaar, Laila $u Sanofi, Cambridge, Massachusetts, USA
700    1_
$a Maski, Manish $u Sanofi, Cambridge, Massachusetts, USA
700    1_
$a Crespo, Ana $u Sanofi, Cambridge, Massachusetts, USA
700    1_
$a Ponce, Elvira $u Sanofi, Cambridge, Massachusetts, USA
700    1_
$a Linhart, Aleš $u Department of Cardiovascular Medicine, First Faculty of Medicine, Charles University, Prague, Czech Republic $u General University Hospital, Prague, Czech Republic
773    0_
$w MED00002747 $t Journal of inherited metabolic disease $x 1573-2665 $g Roč. 47, č. 5 (2024), s. 1080-1095
856    41
$u https://pubmed.ncbi.nlm.nih.gov/38961737 $y Pubmed
910    __
$a ABA008 $b sig $c sign $y - $z 0
990    __
$a 20250121 $b ABA008
991    __
$a 20250206105939 $b ABA008
999    __
$a ok $b bmc $g 2263715 $s 1240139
BAS    __
$a 3
BAS    __
$a PreBMC-MEDLINE
BMC    __
$a 2024 $b 47 $c 5 $d 1080-1095 $e 20240704 $i 1573-2665 $m Journal of inherited metabolic disease $n J Inherit Metab Dis $x MED00002747
GRA    __
$p Sanofi
LZP    __
$a Pubmed-20250121

Najít záznam

Citační ukazatele

Možnosti archivace