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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
2014 ; 25
(4
): 837-49
Nephropedia Template TP
gab.com Text
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English Wikipedia
Patients with Fabry disease after enzyme replacement therapy dose reduction
versus treatment switch
#MMPMID24556354
Weidemann F
; Krämer J
; Duning T
; Lenders M
; Canaan-Kühl S
; Krebs A
; Guerrero González H
; Sommer C
; Üçeyler N
; Niemann M
; Störk S
; Schelleckes M
; Reiermann S
; Stypmann J
; Brand SM
; Wanner C
; Brand E
J Am Soc Nephrol
2014[Apr]; 25
(4
): 837-49
PMID24556354
show ga
Because of the shortage of agalsidase-beta in 2009, many patients with Fabry
disease were treated with lower doses or were switched to agalsidase-alfa. This
observational study assessed end-organ damage and clinical symptoms during dose
reduction or switch to agalsidase-alfa. A total of 105 adult patients with Fabry
disease who had received agalsidase-beta (1.0 mg/kg body weight) for ?1 year were
nonrandomly assigned to continue this treatment regimen (regular-dose group,
n=38), receive a reduced dose of 0.3-0.5 mg/kg (dose-reduction group, n=29), or
switch to 0.2 mg/kg agalsidase-alfa (switch group) and were followed
prospectively for 1 year. We assessed clinical events (death, myocardial
infarction, severe arrhythmia, stroke, progression to ESRD); changes in cardiac,
renal, and neurologic function; and Fabry-related symptoms (neuropathic pain,
hypohidrosis, diarrhea, and disease severity scores). Organ function and
Fabry-related symptoms remained stable in the regular-dose group. In contrast,
estimated GFR decreased by about 3 ml/min per 1.73 m(2) (P=0.01) in the
dose-reduction group, and the median albumin-to-creatinine ratio increased from
114 (0-606) mg/g to 216 (0-2062) mg/g (P=0.03) in the switch group. Furthermore,
mean Mainz Severity Score Index scores and frequencies of pain attacks, chronic
pain, gastrointestinal pain, and diarrhea increased significantly in the
dose-reduction and switch groups. In conclusion, patients receiving regular
agalsidase-beta dose had a stable disease course, but dose reduction led to
worsening of renal function and symptoms. Switching to agalsidase-alfa is safe,
but microalbuminuria may progress and Fabry-related symptoms may deteriorate.
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