Growth-reductive therapy in children with marfan syndrome.

Abstract

To determine the accuracy and precision of 2 height-prediction methods in Marfan syndrome and to assess the growth-reductive effect and side effects of sex hormone treatment. In a retrospective study in 31 untreated (17 boys) and 43 treated patients (21 boys) with Marfan syndrome, we assessed bone age and predicted adult height by 2 methods. The accuracy of the methods was assessed in the untreated group. The effect of therapy was corrected for outcome in the untreated group and other confounding variables with multivariate analysis. Accuracy strongly varied with sex, chronological age, and prediction method. Overall precision was low. Treatment was started at a mean age of 12.8 +/- 1.4 years (boys) and 11.4 +/- 1.2 years (girls). With multiple regression analysis, a statistically significant effect was observed only in boys using a pharmacologic dosage (5.5 cm, 95% CI 0.96-10.1 cm; P = .02). Side effects were worsening of acne and weight and muscle gain. In adolescents with Marfan syndrome, the accuracy and precision of 2 height-prediction methods were limited. The apparent growth-reductive effect of sex hormone treatment appears similar to earlier reports on adolescents with constitutional tall stature. There were no clinically important short-term side effects.