ACCELERATION OF RETARDED GROWTH IN CHILDREN WITH GAUCHER DISEASE AFTER TREATMENT WITH ALGLUCERASE

Objectives: The incidence and severity of growth retardation in childr en with type 1 Gaucher disease and the response to enzyme replacement therapy with alglucerase were studied. Study design: A retrospective a nalysis of growth in 99 children and adolescents with type 1 Gaucher d isease before treatment, and in 54 of those subjects during treatment, was done. Growth was compared with gender, age, and dosage of replace ment enzyme. Results: Linear growth was normal in the first 1 to 2 yea rs of life and then decelerated. Height was at or below the 5th percen tile in 50% of all subjects immediately before treatment. The mean z s core was -1.49 (95% confidence interval, -1.83 to -1.16), correspondin g to the 6.8th percentile for height. Seventy-two percent were below t he 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 w as started, the estimated mean z score for all subjects was -1.01, whi ch corresponds to the 16th percentile for height. Normal growth was ac hieved 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 d oses (60 to 120 U/kg per 4-week period) of alglucerase. Them was a sig nificant association between z scores for height before treatment and liver enlargement (r = 0.57; p < 0.01).Conclusions: Half of the subjec ts who manifest type 1 Gaucher disease in childhood have growth retard ation. Treatment with adequate amounts of modified enzyme replacement was effective in normalizing linear growth.