Results of treatment for type 3 Gaucher disease

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In this issue of The Journal, Goker-Alpan et al studied the spectrum and prevalence of cognitive defects among children with type 3 neuronopathic Gaucher disease who had received either enzyme replacement therapy or bone marrow transplantation. Neuropsychometric evaluation showed a wide spectrum of full scale IQs ranging from 39 to 124; about 60% were above average. There was a significant different in the verbal and performance IQ, and the gap was more prominent in older subjects. There was no correlation between intelligence measurements and genotype or the extent of systemic involvement. There was also no effect on IQ scores from dosage of enzyme replacement therapy, length of treatment, or age at initiation of therapy.