ESPE Abstracts

Background: Central precocious puberty (CPP), early fast puberty (EFP) and early puberty (EP) are considered as deviations from normal variability of pubertal tempo. Gonadotropin releasing hormone agonists (GnRHa) have been considered the treatment of choice for CPP for decades, and later for EFP. Several pharmaceutical formulations of GnRHa have been developed, differing in the route and frequency of administration. Treatment was administered mainly by intra-muscular injections in monthly intervals. Later, 3-month and 6-month formulations were introduced and became available in market. However, there is paucity of reports regarding their long-term efficacy and safety.

Objective: To investigate from real life data the differences in efficacy and safety of monthly and 6-monthly formulations of GnRHa among children diagnosed with CPP, EFP and EP.

Methods: This was a multi-centered historical cohort study, reviewing the medical records of all children diagnosed and treated for CPP, EFP and EP in two pediatric endocrinology units between the years 2010 and 2022. Eighty-eight patients were included, seventy-three (83%) of them females and fifteen (17%) males. Forty-eight patients were treated with 1-month formulation, and forty patients were treated with 6-month formulation. Clinical, anthropometric and laboratory hormonal parameters, as well as side effects, were compared between the groups.

Results: Median age of included patients was 8.0 years for girls (IQR 7.2, 8.6) and 9.7 years for boys (IQR 8.7, 10.3) at treatment initiation. Follow-up duration of monthly-treated group was longer (median duration 42 vs. 31 months, P = 0.015). Both formulations had similar efficiency in suppressing LH levels and delaying pubertal signs progression and bone age advancement. All patients had a minimal increase of BMI-SDS during treatment, in monthly treated group by 0.15 (IQR -0.25, 0.62) and in 6-monthly treated group by 0.18 (IQR-0.03, 0.7), P = 0.55. A similar rate of patients experienced an increase in BMI levels and were defined as obese (6.25% in monthly group vs. 7.50% in 6-month group, P >0.99). In both groups, there was almost no change in height-SDS from the beginning of treatment to its end, -0.01(IQR -0.33, 0.28) in the monthly group, and -0.06 (IQR-0.21, 0.13) in the 6-monthly group, P = 0.50. Side effects reported during follow-up were comparable, and included local pain at injection site (4.2% in monthly group vs. 2.5% in 6-monthly group, P >0.999) and behavioral changes (2.1% in monthly group vs. 5% in 6-monthly group, P = 0.589).

Conclusions: The 6-months formulation represents a convenient alternative to monthly treatment, with similar efficacy and safety.