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)
Summary
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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