ESPE Abstracts (2015) 84 P-3-979

The Assessment of Quality of Life and New Technologies for Therapeutic Monitoring in a Cohort of Paediatric Patients Treated with GH

Giulia Pruccoli, Cristina Partenope, Maria Piera Ferrarello, Chiara Maria Damia, Sara Osimani, Gisella Garbetta, Giovanna Weber & Gabriella Pozzobon


San Raffaele Hospital, Milan, Italy


Background: Short stature may represent a significant psychosocial problem. The rationale for GH treatment has traditionally rested upon the clinical improvement in terms of growth and well-being. Great importance is also associated with the adherence to the therapy.

Objective and hypotheses: We have investigated the benefits obtained by GH treatment, the factors that positively influence growth, the differences between ‘objective’ and ‘subjective’ adherence and finally the health related quality of life (HrQoL) linked to the condition of short stature and to the treatment both from the children and the parents’ point of view.

Method: The target population were short stature children and adolescents with a diagnosis of GH deficiency (GHD) and/or SGA, in therapy with GH using an electronic device (easypod, Merck Serono) for the administration of the therapy. We evaluated the anthropometric parameters, automatically recorded adherence and responses to three different questionnaires on the knowledge of the treatment, the reported adherence, and the evaluation of the HrQoL.

Results: 87.5% of all patients had good adherence according to the definition of Cutfield/Hartmann and 79.5% revealed adherence rates greater than 90%. Comparing recorded against reported adherence showed that more children had higher reported adherence than recorded adherence. We also found a general good HrQoL, both from the child and from the parent’s point of view. Nevertheless, parents generally have a more pessimistic opinion of their children’s conditions. Finally 100% of patients with a better growth response during the 1st year of treatment present at the same time a better psychological adaptation, as compared to the others with an unsatisfactory response.

Conclusion: New technologies capable of accurately recording/monitoring adherence give the possibility to collect objective and consistent data. They also allow the clinician to evaluate the possibility of changes in the therapeutic regimen. Pediatricians have to consider if benefits actually ride out the discomfort, difficulties and limitations and if going on with treatment, especially in poor responders.