-
Je něco špatně v tomto záznamu ?
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
S. Feriozzi, A. Linhart, U. Ramaswami, V. Kalampoki, A. Gurevich, D. Hughes, Fabry Outcome Survey Study Group
Jazyk angličtina Země Spojené státy americké
Typ dokumentu časopisecké články, pozorovací studie, práce podpořená grantem
NLK
ProQuest Central
od 2002-01-01 do Před 2 měsíci
Nursing & Allied Health Database (ProQuest)
od 2002-01-01 do Před 2 měsíci
Health & Medicine (ProQuest)
od 2002-01-01 do Před 2 měsíci
Health Management Database (ProQuest)
od 2002-01-01 do Před 2 měsíci
- MeSH
- alfa-galaktosidasa terapeutické užití MeSH
- dialýza ledvin MeSH
- dospělí MeSH
- enzymová substituční terapie MeSH
- Fabryho nemoc komplikace patofyziologie terapie MeSH
- hodnoty glomerulární filtrace MeSH
- izoenzymy terapeutické užití MeSH
- ledviny patofyziologie MeSH
- lidé středního věku MeSH
- lidé MeSH
- mladiství MeSH
- nemoci srdce komplikace patofyziologie terapie MeSH
- rekombinantní proteiny terapeutické užití MeSH
- renální insuficience komplikace patofyziologie terapie MeSH
- senioři MeSH
- transplantace ledvin MeSH
- výsledek terapie MeSH
- Check Tag
- dospělí MeSH
- lidé středního věku MeSH
- lidé MeSH
- mladiství MeSH
- mužské pohlaví MeSH
- senioři MeSH
- ženské pohlaví MeSH
- Publikační typ
- časopisecké články MeSH
- pozorovací studie MeSH
- práce podpořená grantem MeSH
PURPOSE: The initiation of enzyme-replacement therapy prior to the occurrence of substantial and irreversible organ damage in patients with Fabry disease is of critical importance. The Fabry Outcome Survey is an international disease registry of patients with a confirmed diagnosis of Fabry disease. In this study, data from the Fabry Outcome Survey were used for the assessment of the risks for cardiovascular and renal events in patients who received agalsidase alfa treatment. METHODS: Eligible patients were males and females aged ≥18 years with Fabry disease treated with agalsidase alfa. Cardiovascular events included myocardial infarction, left ventricular hypertrophy (LVH), heart failure, arrhythmia, conduction abnormality, and cardiac surgery. Renal events included dialysis, transplantation, and renal failure. Kaplan-Meier curves and log-rank tests were used for comparing event-free probabilities and time to first cardiovascular or renal event, from agalsidase alfa initiation to a maximum of 120 months, in patients with LVH versus normal left ventricular mass index (LVMI; ≤50 g/m2.7 in males and ≤48 g/m2.7 in females) at treatment initiation (baseline), and in patients with a low estimated glomerular filtration rate (eGFR; <90 mL/min/1.73 m2) versus normal eGFR at baseline. Multivariate Cox regression analysis was used for examining the association between key study variables and the risks for cardiovascular and renal events. FINDINGS: Among the 560 patients (269 males; 291 females) with available LVMI data, 306 (55%) had LVH and 254 (45%) had normal LVMI at baseline. The risk for a cardiovascular event was higher in the subgroup with LVH versus normal LVMI at baseline (hazard ratio [HR] = 1.57; 95% CI, 1.21-2.05; P < 0.001), but the risk for a renal event was similar between the 2 subgroups (HR = 1.90; 95% CI, 0.94-3.85; P = 0.074). Among the 1093 patients (551 males; 542 females) with available eGFR data, 433 (40%) had a low eGFR and 660 (60%) had a normal eGFR at baseline. The subgroup with a low eGFR at baseline had a significantly higher risk for a cardiovascular event (HR = 1.33; 95% CI, 1.04-1.70; P = 0.021) or a renal event (HR = 5.88; 95% CI, 2.73-12.68; P < 0.001) compared with patients with a normal eGFR at baseline. IMPLICATIONS: In the present study, the presence of LVH and/or reduced renal function at agalsidase alfa initiation was associated with a significantly higher risk for a cardiovascular or renal event, indicating that cardiovascular and renal pathologies in Fabry disease may be inter-related. Early initiation of agalsidase alfa treatment prior to the onset of severe organ damage may improve outcomes. ClinicalTrials.gov identifier: NCT03289065.
General University Hospital Prague Czech Republic
Nephrology and Dialysis Unit Belcolle Hospital Viterbo Italy
Royal Free London NHS Foundation Trust University College London London United Kingdom
Citace poskytuje Crossref.org
- 000
- 00000naa a2200000 a 4500
- 001
- bmc21019706
- 003
- CZ-PrNML
- 005
- 20210830101305.0
- 007
- ta
- 008
- 210728s2020 xxu f 000 0|eng||
- 009
- AR
- 024 7_
- $a 10.1016/j.clinthera.2020.10.007 $2 doi
- 035 __
- $a (PubMed)33218740
- 040 __
- $a ABA008 $b cze $d ABA008 $e AACR2
- 041 0_
- $a eng
- 044 __
- $a xxu
- 100 1_
- $a Feriozzi, Sandro $u Nephrology and Dialysis Unit, Belcolle Hospital, Viterbo, Italy. Electronic address: sandro.feriozzi@gmail.com
- 245 10
- $a 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 / $c S. Feriozzi, A. Linhart, U. Ramaswami, V. Kalampoki, A. Gurevich, D. Hughes, Fabry Outcome Survey Study Group
- 520 9_
- $a PURPOSE: The initiation of enzyme-replacement therapy prior to the occurrence of substantial and irreversible organ damage in patients with Fabry disease is of critical importance. The Fabry Outcome Survey is an international disease registry of patients with a confirmed diagnosis of Fabry disease. In this study, data from the Fabry Outcome Survey were used for the assessment of the risks for cardiovascular and renal events in patients who received agalsidase alfa treatment. METHODS: Eligible patients were males and females aged ≥18 years with Fabry disease treated with agalsidase alfa. Cardiovascular events included myocardial infarction, left ventricular hypertrophy (LVH), heart failure, arrhythmia, conduction abnormality, and cardiac surgery. Renal events included dialysis, transplantation, and renal failure. Kaplan-Meier curves and log-rank tests were used for comparing event-free probabilities and time to first cardiovascular or renal event, from agalsidase alfa initiation to a maximum of 120 months, in patients with LVH versus normal left ventricular mass index (LVMI; ≤50 g/m2.7 in males and ≤48 g/m2.7 in females) at treatment initiation (baseline), and in patients with a low estimated glomerular filtration rate (eGFR; <90 mL/min/1.73 m2) versus normal eGFR at baseline. Multivariate Cox regression analysis was used for examining the association between key study variables and the risks for cardiovascular and renal events. FINDINGS: Among the 560 patients (269 males; 291 females) with available LVMI data, 306 (55%) had LVH and 254 (45%) had normal LVMI at baseline. The risk for a cardiovascular event was higher in the subgroup with LVH versus normal LVMI at baseline (hazard ratio [HR] = 1.57; 95% CI, 1.21-2.05; P < 0.001), but the risk for a renal event was similar between the 2 subgroups (HR = 1.90; 95% CI, 0.94-3.85; P = 0.074). Among the 1093 patients (551 males; 542 females) with available eGFR data, 433 (40%) had a low eGFR and 660 (60%) had a normal eGFR at baseline. The subgroup with a low eGFR at baseline had a significantly higher risk for a cardiovascular event (HR = 1.33; 95% CI, 1.04-1.70; P = 0.021) or a renal event (HR = 5.88; 95% CI, 2.73-12.68; P < 0.001) compared with patients with a normal eGFR at baseline. IMPLICATIONS: In the present study, the presence of LVH and/or reduced renal function at agalsidase alfa initiation was associated with a significantly higher risk for a cardiovascular or renal event, indicating that cardiovascular and renal pathologies in Fabry disease may be inter-related. Early initiation of agalsidase alfa treatment prior to the onset of severe organ damage may improve outcomes. ClinicalTrials.gov identifier: NCT03289065.
- 650 _2
- $a mladiství $7 D000293
- 650 _2
- $a dospělí $7 D000328
- 650 _2
- $a senioři $7 D000368
- 650 _2
- $a enzymová substituční terapie $7 D056947
- 650 _2
- $a Fabryho nemoc $x komplikace $x patofyziologie $x terapie $7 D000795
- 650 _2
- $a ženské pohlaví $7 D005260
- 650 _2
- $a hodnoty glomerulární filtrace $7 D005919
- 650 _2
- $a nemoci srdce $x komplikace $x patofyziologie $x terapie $7 D006331
- 650 _2
- $a lidé $7 D006801
- 650 _2
- $a izoenzymy $x terapeutické užití $7 D007527
- 650 _2
- $a ledviny $x patofyziologie $7 D007668
- 650 _2
- $a transplantace ledvin $7 D016030
- 650 _2
- $a mužské pohlaví $7 D008297
- 650 _2
- $a lidé středního věku $7 D008875
- 650 _2
- $a rekombinantní proteiny $x terapeutické užití $7 D011994
- 650 _2
- $a dialýza ledvin $7 D006435
- 650 _2
- $a renální insuficience $x komplikace $x patofyziologie $x terapie $7 D051437
- 650 _2
- $a výsledek terapie $7 D016896
- 650 _2
- $a alfa-galaktosidasa $x terapeutické užití $7 D000519
- 655 _2
- $a časopisecké články $7 D016428
- 655 _2
- $a pozorovací studie $7 D064888
- 655 _2
- $a práce podpořená grantem $7 D013485
- 700 1_
- $a Linhart, Ales $u General University Hospital, Prague, Czech Republic
- 700 1_
- $a Ramaswami, Uma $u Royal Free London NHS Foundation Trust, University College London, London, United Kingdom
- 700 1_
- $a Kalampoki, Vasiliki $u Takeda Pharmaceutical Company, Zurich, Switzerland
- 700 1_
- $a Gurevich, Andrey $u Takeda Pharmaceutical Company, Zurich, Switzerland
- 700 1_
- $a Hughes, Derralynn $u Royal Free London NHS Foundation Trust, University College London, London, United Kingdom
- 710 2_
- $a Fabry Outcome Survey Study Group
- 773 0_
- $w MED00001165 $t Clinical therapeutics $x 1879-114X $g Roč. 42, č. 12 (2020), s. 2321-2330.e0
- 856 41
- $u https://pubmed.ncbi.nlm.nih.gov/33218740 $y Pubmed
- 910 __
- $a ABA008 $b sig $c sign $y p $z 0
- 990 __
- $a 20210728 $b ABA008
- 991 __
- $a 20210830101305 $b ABA008
- 999 __
- $a ok $b bmc $g 1690505 $s 1140152
- BAS __
- $a 3
- BAS __
- $a PreBMC
- BMC __
- $a 2020 $b 42 $c 12 $d 2321-2330.e0 $e 20201117 $i 1879-114X $m Clinical therapeutics $n Clin Ther $x MED00001165
- LZP __
- $a Pubmed-20210728