ESPE Abstracts

Objective: To identify factors influencing compliance with daily recombinant growth hormone (rhGH) therapy in children and adolescents with growth hormone deficiency (GHD) and born as small for gestational age (SGA) based on data from two European paediatric endocrinology centres.

Study population and methods: The study population included 130 patients with GHD or SGA from Szczecin (Poland) and Messina (Italy); 53% of patients were from the Italian centre. Data on potential factors influencing compliance were collected through various methods, including medical history, anthropometric measurements, laboratory test results, and questionnaires completed by patients and their caregivers. The collected information included age, gender, main diagnosis, duration of therapy, SDS growth rate, mid parental height (MPH), pubertal stage, parents' education, self-administration of the drug, and reported problems in therapy. At both centres, compliance with therapy was determined on the basis of the number of missed doses reported by parents; at the Polish centre, the number of empty and full rhGH vials dispensed and returned by patients was also assessed. Compliance was categorized as good (>86% of doses administered), moderate (57-86% of doses administered), or poor (<57% of doses administered) based on the number of missed doses per week.

Results: Good compliance with rhGH was documented in both the Polish (80%) and Italian (82.9%) centres, whereas poor compliace was reported in only a few patients (1.7% in Szczecin and 4.3% in Messina). In both centres, girls had better compliance than boys (85.7% in Messina and 82.4% in Szczecin). Compliance was also affected by treatment duration: in Szczecin the best compliance was observed among children treated for less than 2 years, while in Messina among those treated from 2 to 5 years. Patients who reported problems with therapy had poorer compliance, with 5% in Poland and 32.9% in Italy experiencing difficulties. However, no statistical significance was found between compliance and the presence of problems in therapy reported by patients. In the Polish center, poor compliance was associated with higher education levels of the mothers (P = 0.019). Additionally, a negative correlation was found between compliance and mid parental height (r =-0.274, P = 0.034).

Conclusion: The results of this study indicate that compliance with rhGH therapy is good in two centres in northern and southern Europe. The study highlights the impact of several factors on rhGH treatment compliance in children. These findings emphasize the need for tailored approaches to enhance compliance and optimize treatment outcomes in paediatric patient.