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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 J+Am+Soc+Nephrol
2016 ; 27
(3
): 952-62
Nephropedia Template TP
gab.com Text
Twit Text FOAVip
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English Wikipedia
Patients with Fabry Disease after Enzyme Replacement Therapy Dose Reduction and
Switch-2-Year Follow-Up
#MMPMID26185201
Lenders M
; Canaan-Kühl S
; Krämer J
; Duning T
; Reiermann S
; Sommer C
; Stypmann J
; Blaschke D
; Üçeyler N
; Hense HW
; Brand SM
; Wanner C
; Weidemann F
; Brand E
J Am Soc Nephrol
2016[Mar]; 27
(3
): 952-62
PMID26185201
show ga
Because of the shortage of agalsidase-? supply between 2009 and 2012, patients
with Fabry disease either were treated with reduced doses or were switched to
agalsidase-?. In this observational study, we assessed end organ damage and
clinical symptoms with special focus on renal outcome after 2 years of
dose-reduction and/or switch to agalsidase-?. A total of 89 adult patients with
Fabry disease who had received agalsidase-? (1.0 mg/kg body wt) for >1 year were
nonrandomly assigned to continue this treatment regimen (regular-dose group,
n=24), to receive a reduced dose of 0.3-0.5 mg/kg and a subsequent switch to 0.2
mg/kg agalsidase-? (dose-reduction-switch group, n=28), or to directly switch to
0.2 mg/kg agalsidase-? (switch group, n=37) and were followed-up for 2 years. We
assessed clinical events (death, myocardial infarction, severe arrhythmia,
stroke, progression to ESRD), changes in cardiac and renal function,
Fabry-related symptoms (pain, hypohidrosis, diarrhea), and disease severity
scores. Determination of renal function by creatinine and cystatin C-based eGFR
revealed decreasing eGFRs in the dose-reduction-switch group and the switch
group. The Mainz Severity Score Index increased significantly in these two groups
(P=0.02 and P<0.001, respectively), and higher frequencies of gastrointestinal
pain occurred during follow-up. In conclusion, after 2 years of observation, all
groups showed a stable clinical disease course with respect to serious clinical
events. However, patients under agalsidase-? dose-reduction and switch or a
direct switch to agalsidase-? showed a decline of renal function independent of
the eGFR formula used.