Kidney Function and 24-Hour Proteinuria in Patients with Fabry Disease during 36 Months of Agalsidase Alfa Enzyme Replacement Therapy: A Brazilian Experience

Thofehrn, Scheila Pretto Almeida; Netto, Cristina Brinckmann Oliveira; Cecchin, Cláudia Rafaela; Burin, Maira Graeff; Matte, Úrsula da Silveira; Brustolin, Silvia; Nunes, Ane Cláudia Fernandes; Coelho, Janice Carneiro; Tsao, Marylin; Jardim, Laura Bannach; Giugliani, Roberto; Barros, Elvino José Guardão

doi:10.3109/08860220903150296

Cited by 20 publications

(19 citation statements)

References 27 publications

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“…Thirty-four studies [25–27,32,33,35–63] were conducted largely in Europe, 16 were international studies [22–24, 64–76], 10 in the USA [28–30,77–83], nine in Asia [84–92], six in Central and South America [93–98], and two in Canada [31,34]. Cohort studies sample size ranged from six [60,93] to 2,869 [68].…”

Section: Resultsmentioning

confidence: 99%

Enzyme replacement therapy for Anderson-Fabry disease: A complementary overview of a Cochrane publication through a linear regression and a pooled analysis of proportions from cohort studies

et al. 2017

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BackgroundAnderson-Fabry disease (AFD) is an X-linked recessive inborn error of glycosphingolipid metabolism caused by a deficiency of alpha-galactosidase A. Renal failure, heart and cerebrovascular involvement reduce survival. A Cochrane review provided little evidence on the use of enzyme replacement therapy (ERT). We now complement this review through a linear regression and a pooled analysis of proportions from cohort studies.ObjectivesTo evaluate the efficacy and safety of ERT for AFD.Materials and methodsFor the systematic review, a literature search was performed, from inception to March 2016, using Medline, EMBASE and LILACS. Inclusion criteria were cohort studies, patients with AFD on ERT or natural history, and at least one patient-important outcome (all-cause mortality, renal, cardiovascular or cerebrovascular events, and adverse events) reported. The pooled proportion and the confidence interval (CI) are shown for each outcome. Simple linear regressions for composite endpoints were performed.Results77 cohort studies involving 15,305 participants proved eligible. The pooled proportions were as follows: a) for renal complications, agalsidase alfa 15.3% [95% CI 0.048, 0.303; I2 = 77.2%, p = 0.0005]; agalsidase beta 6% [95% CI 0.04, 0.07; I2 = not applicable]; and untreated patients 21.4% [95% CI 0.1522, 0.2835; I2 = 89.6%, p<0.0001]. Effect differences favored agalsidase beta compared to untreated patients; b) for cardiovascular complications, agalsidase alfa 28% [95% CI 0.07, 0.55; I2 = 96.7%, p<0.0001]; agalsidase beta 7% [95% CI 0.05, 0.08; I2 = not applicable]; and untreated patients 26.2% [95% CI 0.149, 0.394; I2 = 98.8%, p<0.0001]. Effect differences favored agalsidase beta compared to untreated patients; and c) for cerebrovascular complications, agalsidase alfa 11.1% [95% CI 0.058, 0.179; I2 = 70.5%, p = 0.0024]; agalsidase beta 3.5% [95% CI 0.024, 0.046; I2 = 0%, p = 0.4209]; and untreated patients 18.3% [95% CI 0.129, 0.245; I2 = 95% p < 0.0001]. Effect differences favored agalsidase beta over agalsidase alfa or untreated patients. A linear regression showed that Fabry patients receiving agalsidase alfa are more likely to have higher rates of composite endpoints compared to those receiving agalsidase beta.ConclusionsAgalsidase beta is associated to a significantly lower incidence of renal, cardiovascular and cerebrovascular events than no ERT, and to a significantly lower incidence of cerebrovascular events than agalsidase alfa. In view of these results, the use of agalsidase beta for preventing major organ complications related to AFD can be recommended.

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Section: Resultsmentioning

confidence: 99%

Enzyme replacement therapy for Anderson-Fabry disease: A complementary overview of a Cochrane publication through a linear regression and a pooled analysis of proportions from cohort studies

et al. 2017

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“…A Brazilian study demonstrated that patients with mild (CKD stage 1) or moderate (CKD stage 3) renal impairment maintained stable GFRs over 3 years of treatment with agalsidase alfa. Only one patient (of 11), with stage 4 CKD, progressed to ESRD despite continuing agalsidase alfa infusions, without evidence of improvement in proteinuria with ERT until kidney transplantation [97]. At the histological level, a significant correlation has been shown between the reduction in podocyte Gb3 inclusions and the cumulative dose of agalsidase alfa or beta in young, predominantly male patients (one female patient) treated for a mean of 60.3 months; irrespective of drug dosage, complete clearance of Gb3 inclusions was observed in the glomerular endothelial cells and mesangial cells in all patients after 5 years of ERT [98].…”

Section: Treatment Of Fabry Nephropathymentioning

confidence: 99%

Fabry Nephropathy: An Evidence-Based Narrative Review

Pino

Andrés

Bernabeu

et al. 2018

Kidney Blood Press Res

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“…При помощи своевременно начатой патогенетической терапии удает-ся добиться значительного уменьшения интенсивности нейропатической боли вплоть до ее полного исчезнове-ния [38], улучшения слуха [39], положительной динамики характеристик функции почек [40,41], сердечно-сосуди-стой системы [42,43], купирования гастроинтестиналь-ных симптомов болезни [44].…”

Section: лечение болезни фабриunclassified