Endocrine Abstracts

JOINT3608

Long-acting growth hormones (LAGH) constitute a therapeutic innovation in growth hormone deficiency (DGH). Its efficacy, safety, immunogenicity and bioavailability should be comparable to daily administered GH. With them it is expected to reduce the treatment burden, positively influence the quality of life of the patient and family, improve adherence and consequently therapeutic efficacy. Somatrogon, a LAGH, has been approved for use by the EMA and FDA in the child with GH deficiency.

Objectives: To evaluate the therapeutic efficacy, safety and adherence in a group of children diagnosed with GH deficiency treated with weekly LAGH (Somatrogon).

Methodology: Prepubertal patients diagnosed with GH deficiency and treated with Somatrogon. Clinical follow-up evaluating therapeutic efficacy criteria (variation of the SDS of height, relationship of height with target height, progress of bone age, puberty and adult height; safety (IGF1, IGFBP3, hormonal biochemistry, lipid and glycemic metabolism and AES) and adherence (through administration record).

Results: The follow-up during 6 years of 10 patients (2 girls and 8 boys) with GH deficiency treated with Somatrogon is presented. Five of them were initially treated with daily GH for 1 year and then continued with Somatrogon and another five from the beginning were treated with Somatrogon. 1. Therapeutic efficacy: All patients have increased their growth velocity, recovering their growth curve according to their genetic potential, with gains in height > 1SDS, with an evolution of bone age and sexual maturation according to chronological age. 2. Adult height: Four patients have reached adult height (AH), one girl with AH of 161.1 cm (SDS -0.16) and three boys with AH of 171.4 cm (SDS-0.63), 165. 7 (SDS -1.68) and 170.1 cm (SDS -0.71), respectively. 3. Safety: IGF1 levels have remained in the safe range and only in two patients, during puberty, a modification of the therapeutic dose was required. Without AES or local reactions relevant. Patients and families have expressed satisfaction with weekly administration. 4. Therapeutic adherence: It has been 100%.

Conclusions: Somatrogon (LAGH) has shown safety and efficacy in promoting linear growth in this patient´s group treated in our clinic. Similarly to daily GH in the treatment of GH deficiency; although with excellent adherence and less treatment burden. Four patients achieved adult height near their target height. LAGH are emerging as an innovative and safe treatment in the patient with growth hormone indication.