The natural course of Gaucher disease in The Netherlands: implications for monitoring of disease manifestations.
This retrospective study in 20 untreated type I Gaucher disease patients shows that in Dutch patients clinical manifestations of Gaucher disease type I are progressive in the majority of patients, children as well as adults. This is in contrast with studies among Jewish patients. Our results emphasize the need for a regular follow-up to enable timely initiation of enzyme therapy.
Maaswinkel-Mooij
P
P
Department of Pediatrics, Leiden University Medical Centre, The Netherlands.
Hollak
C
C
van Eysden-Plaisier
M
M
Prins
M
M
Aerts
H
H
Pöll
R
R
D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D005776:Gaucher Disease / Q000150:complications*; D006801:Humans; D008297:Male; D008875:Middle Aged; D012189:Retrospective Studies