Background: Adherence to GH treatment is a challenge.
Objective and hypotheses: We analysed the impact of treatment duration, treatment success, treatment indication, age, gender, pubertal stage, and height on treatment adherence (TA) to optimise treatment success.
Method: Based on the easypod autoinjector used in the Saizen-online prospective, multicenter, open-label, noninterventional study we analyzed TA in 6 months periods. TA was evaluated using proposed cut-offs (good adherence: <1 missed dose/week; medium adherence: 13 missed doses/week; poor adherence: >3 missed doses/week)1.
Results: 168 children treated with GH (71% GH deficiency, 7% Turner-Syndrome, 2% chronic renal insufficiency, 20% small-for-gestational age) were included (641 6-months observations periods). TA did not differ significantly between treatment indications (P=0.713) or gender (P=0.167). Younger age, prepubertal stage, and lower height-SDS were associated with better TA, while better treatment success and longer treatment duration were related to lower TA (table 1).
|Good adherence||Medium adherence||Poor adherence||P-value|
|Number of 6-months observation periods||373 (58.2%)||135 (21.1%)||133 (20.7%)|
|Actual height-SDS||−1.9±1.1||−1.7±1.2||−1.3±1.3||<0.0011,3; 0.0382; 0.0174|
|Treatment success (actual height-SDS height-SDS at onset of GH treatment)||+0.8 (IQR 0.21.4)||+0.7 (IQR 0.21.3)||+1.0 (IQR 0.51.5)||0.0041; 0.0023; 0.0054|
|Treatment duration (y)||1.8 (IQR 0.83.6)||3.0 (IQR 1.54.5)||2.5 (IQR 1.63.6)||<0.001 1,2,3|
|Data as n (%), mean±1 S.D., or median and interquartile range (IQR); P-values: 1) overall; 2) good versus medium; 3) good versus poor; 4) medium vs poor TA, Fishers exact, WilcoxonMannWhitney and KruskalWallis tests were used as adequate.|
Conclusion: Especially in pubertal children with good treatment success so far, TA should be critically reviewed.
Funding: This work was supported by a grant from Merck Serono GmbH, Darmstadt Germany. Study design, data collection and analysis, decision to publish, and preparation of the manuscript are solely the responsibility of the authors.
01 - 03 Oct 2015
European Society for Paediatric Endocrinology