Background: GH therapy is being used worldwide to improve height outcome in children with GH deficiency (GHD), with minimal serious side effects. Early diagnosis and therapy initiation optimize growth outcomes.
Objectives: To evaluate growth and safety during the first 4 years of GH treatment in 33 GHD children.
Methods: We reviewed clinical data of 33 prepubertal children (23 boys and ten girls): 30 with isolated GH deficiency (IGHD), three with multiple pituitary hormone deficiency (MPHD). All of them were treated with a mean dose of GH=0.035 mg/kg per day and followed for at least 4 years (mean 5.98 years).
Results: The mean height SDS increased from −2.76 at baseline to −0.73 at 4 years; the change in height SDS decreased with time Table 1. Within first 4 years of therapy none of these children developed diabetes mellitus, seven patients (21.21%) presented transient increase in fasting glucose (>100 <126 mg/dl), two patients (6.06%) had transiently impaired glucose tolerance (<140 <200 mg/dl at OGTT), three patients (9.09%) developed hypothyroidism and one patient (3.03%) had transiently increased TSH levels (normal fT4 values, no clinical signs). No malignancies were observed to date.
|Parameter||Baseline||1 year||2 years||3 years||4 years|
|Chronological age (years)||7.89||8.89||9.89||10.89||11.89|
|Bone age (years)||5.76||6.62||7.44||9.46||11.07|
|IGF-1 mean values (ng/ml)||64.64||221.8||205.7||265||338|
|Height velocity (cm/year)||||10.59||7.48||6.04||6.76|
Conclusions: GH treatment significantly improves growth of GHD children, with a favorable safety profile. The maximum height velocity was observed in the first year of therapy (10.59 cm/year); the following years of treatment resulted in a lower height velocity (7.48 cm/year, 6.04 cm/year, 6.76 cm/year respectively). No severe adverse events were observed.
20 - 22 Sep 2014
European Society for Paediatric Endocrinology