Acceleration of retarded growth in children with Gaucher disease after treatment with alglucerase.
OBJECTIVE
The incidence and severity of growth retardation in children with type 1 Gaucher disease and the response to enzyme replacement therapy with alglucerase were studied.


METHODS
A retrospective analysis of growth in 99 children and adolescents with type 1 Gaucher disease before treatment, and in 54 of those subjects during treatment, was done. Growth was compared with gender, age, and dosage of replacement enzyme.


RESULTS
Linear growth was normal in the first 1 to 2 years of life and then decelerated. Height was at or below the 5th percentile in 50% of all subjects immediately before treatment. The mean z score was -1.49 (95% confidence interval, -1.83 to -1.16), corresponding to the 6.8th percentile for height. Seventy-two percent were below the 50th percentile and 50% were at or below the 5th percentile for mid-parental height (p <0.001). One and one-half years after treatment was started, the estimated mean z score for all subjects was -1.01, which corresponds to the 16th percentile for height. Normal growth was achieved within 4 to 30 months in eight of nine subjects who were at or below the 5th percentile. It occurred only in those receiving higher doses (60 to 120 U/kg per 4-week period) of alglucerase. There was a significant association between z scores for height before treatment and liver enlargement (r= 0.57; p < 0.01).


CONCLUSIONS
Half of the subjects who manifest type 1 Gaucher disease in childhood have growth retardation. Treatment with adequate amounts of modified enzyme replacement was effective in normalizing linear growth.
Kaplan
P
P
Department of Pediatrics, Children's Hospital of Philadelphia, PA 19104, USA.
Mazur
A
A
Manor
O
O
Charrow
J
J
Esplin
J
J
Gribble
T J
TJ
Wappner
R S
RS
Wisch
J S
JS
Weinreb
N J
NJ
D000293:Adolescent; D001827:Body Height; D002648:Child; D002675:Child, Preschool; D005260:Female; D005776:Gaucher Disease / Q000150:complications / Q000188:drug therapy*; D005962:Glucosylceramidase / Q000627:therapeutic use*; D006128:Growth; D006130:Growth Disorders / Q000150:complications / Q000188:drug therapy*; D006801:Humans; D008297:Male; D012189:Retrospective Studies
C070292:alglucerase; D005962:Glucosylceramidase