Abstract
It is held that enzyme replacement therapy (ERT) accelerates the growth rate in children with Gaucher disease, but its effect on final height has not been established with certainty. This study presents final heights of Gaucher patients followed up for 15 years.The study included 41 adults with non-neuronopathic Gaucher disease. The final height of the patients and age at puberty was compared to their mid-parental target height and to their siblings' heights.Mean final height standard deviation score (HSDS) in the patients was − 0.22, but none of the patients was abnormally short (HSDS of less than − 2.2). Mean age at menarche of the female patients (14.7 years) was significantly delayed compared to that of their mothers (P = 0.0005), and mean age at first shaving in the boys was 16 years.Our study showed that the mean final height of Gaucher patients fell below the mean of the 2000 CDC growth charts, but the patients were not of short stature (height less than the 3rd percentile). ERT treatment did not significantly impact the mean final HSDS. The onset of puberty, as indicated by the age at menarche, was delayed in girls with Gaucher disease.
Published Version
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