Abstract

BackgroundOne of the most important vulnerabilities falling the efficacy of recombinant human growth hormone (r-hGH) treatment is low adherence especially in young patients. This study was planned to describe the correlation between r-hGH treatment efficacy and adherence in real-life setting using easypod™.MethodsForty patients younger than 18 years, affected by a clinical condition in which r-hGH is available and treated with r-hGH easypod™, were enrolled in a retrospective, observational, real-world data, monocentric trial. The study design provided the retrospective collection of records collected by a questionnaire proposed to the patients and their parents and compared with registered data by the new generation electronic device r-hGH easypod™. Number of injections and doses were collected and used to assess the percentage of administered GH doses to measure treatment adherence. The r-hGH treatment efficacy was evaluated comparing standard deviation score for height (SDS) between baseline and follow-up visit, according to clinical practice.ResultsThe mean treatment adherence was 92.20% and it was inversely related to patients’ age (R = − 0.358, p = 0.023), and significantly higher in the sub-group of patients with age between 10 and 13 years. Treatment adherence showed an inverse correlation with the years of therapy (R = − 0.453, p = 0.003) and with the number of r-hGH administrations (R = − 0.392, p = 0.012). However, the height increase did not reach a significant correlation with treatment adherence (R = − 0.067, p = 0.683).ConclusionsChildren and adolescent patients with GH deficiency due to different clinical conditions show high adherence to r-hGH treatment tested by easypod™. Easypod™ could be used as an important device to control patients’ adherence in daily treatment for chronic diseases with expensive drugs.

Highlights

  • One of the most important vulnerabilities falling the efficacy of recombinant human growth hormone (r-hGH) treatment is low adherence especially in young patients

  • In Italy, r-hGH is indicated in GH deficiency (GHD), Turner syndrome (TS), short stature associated with SHOX aploinsufficiency, short children born small for gestational age (SGA), chronic renal failure (CRF) and Prader-Willi syndrome [3]

  • Forty young patients treated with r-hGH were enrolled, with a mean age of 11.2 + 2.3 years (Table 2)

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Summary

Introduction

One of the most important vulnerabilities falling the efficacy of recombinant human growth hormone (r-hGH) treatment is low adherence especially in young patients. In Italy, r-hGH is indicated in GH deficiency (GHD), Turner syndrome (TS), short stature associated with SHOX aploinsufficiency, short children born small for gestational age (SGA), chronic renal failure (CRF) and Prader-Willi syndrome [3] This treatment is primarily used in childhood and adolescence and the Chronic long-term treatments are generally characterized by low adherence. Several studies reported that up to half patients are generally not fully adherent [8] This therapy requires daily subcutaneous injections and children often have a significant load related to the daily administration of r-hGH, missing the final goal of benefits in height [4, 5]. A complete evaluation of patients’ adherence allows the clinicians to exclude poor adherence from the possible reasons for sub-optimal growth response, driving further treatment adjustment and exact interval among follow-up visits

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