Effect of 4ˋyears of growth hormone therapy in children with Noonan syndrome in the American Norditropin Studies: Web-Enabled Research (ANSWER) Programˋ registry

作者：Peter A Lee; Judith Ross; John A Germak; Robert Gut

来源：International Journal of Pediatric Endocrinology, 2012.

DOI：10.1186/1687-9856-2012-15

• Growth hormone
• Noonan Syndrome
• Somatropin
• Short stature
• Body height
• Treatment outcome

The American Norditropin Studies: Web-Enabled Research (ANSWER) Programˋ, a US-based registry, collects long-term efficacy and safety information on patients treated with Norditropinˋ (somatropin rDNA origin, Novo Nordisk A/S) at the discretion of participating physicians. A total of 120 children (90 boys, 30 girls) with NS, naˋve to previous GH treatment, were included in this analysis.The mean (SD) baseline age of subjects (nˋ=ˋ120) was 9.2 (3.8) years. Mean (SD) HSDS increased from 每2.65 (0.73) at baseline to 每1.32 (1.11) at Y4 (nˋ=ˋ17). Subjects showed continued increase in HSDS from baseline to Y4 without significant differences between genders at Y1 or Y2. The mean (SD) GH dose was 47 (11) mcg/kg/day at baseline and 59 (16) mcg/kg/day at Y4. There was a negative correlation between baseline age and 忖HSDS at Y1 (Rˋ=ˋ每0.3156; Pˋ=ˋ0.0055) and Y2 (Rˋ=ˋ每0.3394; Pˋ=ˋ0.017). 忖HSDS at Y1 was significantly correlated with 忖HSDS at Y2 (nˋ=ˋ37; Rˋ=ˋ0.8527, Pˋ%26lt;ˋ0.0001) and Y3 (nˋ=ˋ20; Rˋ=ˋ0.5145; Pˋ=ˋ0.0203), but not Y4 (nˋ=ˋ12; Rˋ=ˋ0.4066, Pˋ=ˋ0.1896).GH treatment-naˋve patients with NS showed continued increases in HSDS during 4ˋyears of treatment with GH with no significant differences between genders up to 2ˋyears. Baseline age was negatively correlated with 忖HSDS at Y1 and Y2. Whether long-term therapy in NS results in continued increase in HSDS to adult height remains to be investigated.ClinicalTrials.gov NCT01009905