THE EFFECT OF GH (SAIZEN) THERAPY (28 IU/M/WEEK) ON LINEAR GROWTH AND CARDIAC MORPHOLOGY IN SHORT CHILDREN WITH NOONAN SYNDROME

Abstract

Noonan Syndrome (NS) is a dysmorphic condition associated with short stature and cardiac defects (pulmonary stenosis and ventricular hypertrophy [VH]). Early studies reported linear growth increases with GH. Aneedotal reports, however, suggested an association between GH and VH progression. We therefore undertook a multi-centre open trial of GH (28IU/m2/wk Saizen daily sc) in children with NS. Entry criteria were: NS confirmed by single observer(ME) in Genetics dept(MAP), HtSDS<−2, HtVelSDS <0, normal maximal LV wall thickness(LVWT) by 2D echocardiography (single observer WJM), breast stage 1 or testicular volume <6ml and age 5-14y. 14 subjects are now receiving GH [9 male, 5 female; 9.5 ± 0.7y (range 12.6-5.1), HtSDS −2.81 ± 0.12 (−3.79 - −2.17), HtVel 4.1 ± 0.3 cm/y (2.6-6.0), maximal LVWT 7± 1mm. Linear growth and QRS voltage on ECG were monitored 3mthly, and maximal LVWT at 1y. To date (1/12/92) 7 subjects have completed 6mth and 4 subjects 1y of therapy. There has been a clinically significant increase in HtSDS and HtVel without change in maximal LVWT or ECG voltages.

In conclusion GH appears effective and not associated with significant change in cardiac morphology in Noonan Syndrome.

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