Cardio-renal outcomes with long-term agalsidase alfa enzyme replacement therapy : A 10-year fabry outcome survey (FOS) analysis
Ramaswami, Uma (Royal Free London NHS Foundation Trust. Lysosomal Disorders Unit. Institute of Immunity and Transplantation)
Beck, Michael (Centre for Paediatric and Adolescent Medicine. University Medical Centre. University of Mainz)
Hughes, D. (Royal Free London NHS Foundation Trust. Lysosomal Disorders Unit. Institute of Immunity and Transplantation)
Kampmann, C. (Centre for Paediatric and Adolescent Medicine. University Medical Centre. University of Mainz)
Botha, J. (Department of Biostatistics and Programming. Takeda)
Pintos-Morell, Guillem (Institut Germans Trias i Pujol. Hospital Universitari Germans Trias i Pujol)
West, M. L. (Department of Medicine. Dalhousie University)
Niu, D. M. (Department of Paediatrics. Taipei Veterans General Hospital)
Nicholls, K. (Royal Melbourne Hospital (Melbourne, Austràlia))
Giugliani, R. (Medical Genetics Service HCPA. Department of Genetics. UFRGS. INAGEMP)
Universitat Autònoma de Barcelona

Fecha:	2019
Resumen:	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 m 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 m) 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 in females, >50 g/m 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.
Derechos:	Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, la comunicació pública de l'obra i la creació d'obres derivades, sempre que no sigui amb finalitats comercials, i sempre que es reconegui l'autoria de l'obra original.
Lengua:	Anglès
Documento:	Article ; recerca ; Versió publicada
Materia:	Cardio-renal outcomes ; Agalsidase alfa ; Fabry disease ; Enzyme replacement therapy
Publicado en:	Drug Design, Development and Therapy, Vol. 13 (2019) , p. 3705-3715, ISSN 1177-8881

DOI: 10.2147/DDDT.S207856
PMID: 31749608

11 p, 6.0 MB

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