Improved adherence and growth outcomes with jet-delivered growth hormone

Abstract

Background We previously reported improved persistence and adherence to daily recombinant growth hormone (rGH) in children using jet transjection delivery compared to using needle-based devices. This study examines the relationship between improved adherence and medium-term growth outcomes in children receiving jet-delivered rGH (JrGH) at a single centre. Methods This was a retrospective longitudinal follow-up study of children (<16 years) treated with daily JrGH (somatropin; Ferring Pharmaceuticals) in the form of Zomacton® with the Zomajet® device. Delivery schedules of home distribution services were utilised to calculate adherence, quantified as the proportion of days covered (PDC) index (PDC > 0.8 adherent, PDC ≤ 0.8 less adherent). Demography, patient history, height standard deviation scores (HTSDS) and difference from mid-parental height SDS (MPHSDS − HTSDS) were extracted from hospital records for up to 3 years of treatment. Results Of 75 patients eligible for JrGH, 52 had PDC treatment and height data for at least 1 year and 22 for 3 years. A greater proportion of patients were classified as adherent in both 1- and 3-year treated cohorts (adherent 30 [57.7%] and 14 [63.6%], less adherent 22 [42.3%] and 8 [36.4%]). After 1 year of JrGH treatment, HTSDS was not significantly different in either adherence group. After 3 years, only adherent patients demonstrated sustained year-on-year increments in HTSDS and significant improvement in target HTSDS positions (by 1.32 SDS) compared to baseline (p = 0.0008). MPHSDS – HTSDS showed a similar significant improvement at 3 years in adherent patients only (p = 0.0043). Conclusions Patients adherent to JrGH demonstrate significant growth improvement compared to baseline over 3 years.

Adherence to treatment in children with growth hormone deficiency, small for gestational age and Turner syndrome in Mexico: results of the Easypod™ connect observational study (ECOS)

“…In the present study, the observed adherence rate was above 80% up to 3 years of clinical follow-up, which contrasts with the expected adherence rate of 55-65% observed in other studies using different r-hGH delivery technologies [22,23] and confirms the findings of other reports with the same methodology [5,6,13,14,24]. Additionally, this study suggests that adherence to therapy is predictor of clinical response, a factor that has been described in other scenarios where electromechanical auto-injectors are used to deliver drugs and to assess compliance to therapy [25−30].…”

Refill Adherence Measures and Its Association with Economic, Clinical, and Humanistic Outcomes Among Pediatric Patients: A Systematic Review

“…The MPR and PDC were the most common RAMs reported (n = 19) [25][26][27][29][30][31][32][33]36,37,[39][40][41][42][43][44][45][46]55]. The RAMs used in eight other studies were equivalent to the proportion of days with medication supply, similar to MPR and PDC [21,22,34,[50][51][52][53][54].…”

“…The studies were conducted in 10 different countries, the majority (n = 33) of which were high-income countries, according to The World Bank definition [56]. These include the United States of America (n = 23) [21][22][23][24][25][26][27]29,[31][32][33][34][35]39,41,[47][48][49][50][51][52][53][54], United Kingdom (n = 3) [30,38,46], Israel (n = 2) [43,45], Taiwan (n = 1) [42], Korea (n = 1) [37], Australia (n = 1) [55], Netherlands (n = 1) [36], and Singapore (n = 1) [44]. Only two studies were conducted in upper-middle-income countries such as Brazil (n = 1) [28], and Malaysia (n = 1) [40].…”

“…Asthma (n = 9) was the most common disease state involved in the studies [24,25,28,30,31,[34][35][36]51], followed by human immunodeficiency virus (HIV) infection (n = 6) [21][22][23]47,49,50]. The sources of medication refill data were pharmacy records (n = 19) in approximately half of the studies, [21][22][23]28,33,35,37,40,44,[46][47][48][49][50][51][52][53][54][55], and administrative claims database (n = 16) in the remaining studies [24][25][26][27][29][30][31][32]34,36,38,39,[41][42][43]…”

“…Of the 27 studies with a fixed threshold to define adequate adherence, five studies derived its threshold from the study cohorts' percentiles or data distributions [25,27,33,36,41], while 10 studies utilized thresholds from prior studies [22,31,37,39,40,43,45,49,50,53]. The source of threshold used for the remaining 12 studies were not specified [21,24,34,35,38,42,44,[46][47][48]52,54]. Table S3 summarizes the reported relationship between medication adherence and ECHO.…”