Revaluation of the clinical and metabolic behavior of children with isolated growth hormone deficiency during GH treatment according to newly proposed note 39 of the Italian Medicines Agency (AIFA).

Purpose To evaluate the clinical and metabolic behavior of children with isolated growth hormone (GH)-deficiency (GHD), grouped according to the new AIFA criteria for the appropriateness of use and reimbursement of GH treatment in children. Methods The clinical and metabolic data of 310 prepubertal children (220 M, 90 F; mean age 10.8 years) grouped, according to new AIFA note 39, into group A (No 181 with a peak of GH < 8 µg/L), group B (No 103 with a peak of GH ≥ 8 and < 10 µg/L) and group C (No 26 with a peak of GH > 10 µg/L) were retrospectively analyzed. Group A and B, diagnosed as having GHD, were treated with GH for at least 24 months, while group C was analyzed only at baseline. Results At baseline, group A showed higher waist circumference than B (p=0.031) and C (p=0.041), while no difference in metabolic parameters was found between the 3 groups. After 12 and 24 months of treatment, group B showed lower height velocity (p<0.001 and p=0.049, respectively) than group A. As regards the metabolic parameters, both after 12 and 24 months of treatment, in group B we found higher fasting glucose (p<0.001 and p=0.020), insulin (p=0.002 and p=0.011), Homa-β (p=0.020 and p=0.015) and Homa-IR (both p=0.001) than group A, with concomitant lower QUICKI (both p<0.001) and HDL cholesterol (p=0.020 and p=0.011), without difference in other lipid parameters. The HbA1c levels, although always within the normal range, was found higher in group B than group A after 12 months (p=0.015). Conclusions Accordingly with the new AIFA criteria, the reduction of GH cut-off for GHD diagnosis can be supported by auxological and metabolic data. The real benefits from GH therapy in children with higher stimulated GH levels at diagnosis remains to better understand.