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.jpg): Failed to open stream: No such file or directory in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 117 PLoS+One
2017 ; 12
(3
): e0173358
Nephropedia Template TP
gab.com Text
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Twit Text #
English Wikipedia
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
#MMPMID28296917
El Dib R
; Gomaa H
; Ortiz A
; Politei J
; Kapoor A
; Barreto F
PLoS One
2017[]; 12
(3
): e0173358
PMID28296917
show ga
BACKGROUND: Anderson-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. OBJECTIVES: To evaluate the efficacy and
safety of ERT for AFD. MATERIALS AND METHODS: For 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.
RESULTS: 77 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. CONCLUSIONS:
Agalsidase 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.