ESPE Abstracts

Introduction: Fenton (2013) defined small for gestational age children (SGA) as born with birth length and/or weight < 10th percentile for the corresponding gestational age, and clinicians use it ever since. According to the literature, 1 of 10 children born SGA does not catch-up in growth. Wit (2021) suggested to address them as short SGA children. Recombinant human growth hormone (rhGH) treatment in those children have been approved by the FDA in 2001 and two years later by EMA. Unfortunately, in some countries this treatment is still not reimbursed by the health care system, and growth outcomes data is needed.

Aim: The aim of the current study is to analyze age at start, duration, and the efficacy of rhGH treatment in short SGA children.

Patients and Methods: After receiving approval by the Ethical Committee of the Medical University of Varna, written consent from the patients was collected. All patients who were patients of the center at 31.12.2021 and were rhGH treated, were included. Data about baseline, 1st and 2nd year growth indices were retrospectively extracted from the clinical records and analysed. All parameters of interest were assessed, using SPSS by Windows, version 25.

Results: A total of 43 short SGA children were included in the current study, 26 (60.5%) of them rhGH treated with starting dose of 0.029±1.94 mg/kg/d. The average age at the commencement of rhGH in the treated group was 6.57±3.67 years. The ratio between boys and girls was 15:11. The most common clinical diagnosis was the Silver-Russell Syndrome (SRS) presenting with 15 patients (57.7%), followed by short SGA (5 patients, 19.2%), cleidocranial dysplasia (2 patients, 7.7%). Some of the other patients were with DiGeorge syndrome, Noonan syndrome, Löwe syndrome, and others. On the average, the treatment duration was 3.61±2.58 years. The average growth velocity for the first year was 9.49 ± 2.63 cm and for the 2nd year of treatment was 6.8±2.01 cm. Respectively height improved with 0.57±0.46 SDS for the first year and with 1.63±1.01 SDS for the 2nd year. The weight in our cohort increased with 0.94±1.21 SDS for the first year of the treatment and with 0.36±0.45 SDS for the 2nd year.

Conclusion: The presented patients started treatment relatively late and with lower rhGH doses than the recommended for the SGA indication. These are potential targets for the improvement of future treatment outcomes.