Identifying Potentially Modifiable Factors Associated with Treatment Non-Adherence in Paediatric Growth Hormone Deficiency: A Systematic Review

Graham, Selina; Weinman, John; Auyeung, Vivian

doi:10.1159/000493211

Cited by 59 publications

(85 citation statements)

References 35 publications

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“…In a variety of prospective/retrospective, randomized/non-randomized, and controlled/ non-controlled trials, some researchers concluded that GH treatment improved ISS while others considered it ineffective. The efficacies in these studies varied dramatically [12][13][14][15][16][17][18]. Some meta-analyses showed that GHtreated ISS patients might gain more FAH than untreated children, but were still shorter than the normal populations.…”

Section: Discussionmentioning

confidence: 99%

Final adult height of children with idiopathic short stature: a multicenter study on GH therapy alone started during peri-puberty

Chen

et al. 2020

BMC Pediatr

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Background: To evaluate the efficacy of GH in improving FAH in ISS children in a multicenter study. Methods: A real-world observation was carried out. Children with ISS in seven hospitals in China were enrolled. The height gains standard deviation score and the height gain over the target height were evaluated. Results: There were 344 ISS patients (217 boys and 127 girls). The baseline average age of boys and girls was 12.7 and 11.7 years, with bone age of 11.7 and 10.1 years, respectively. The baseline height SDS of boys and girls was − 3.07 and − 2.74, and the FAH SDS was − 1.91 and − 1.38, respectively. Compared with the baseline height SDS, the FAH SDS was significantly increased in both boys and girls (both P = 0.0000). The FAH SDS was the highest (gain by 1.54 SD) in the ≥2y treatment course group. Two hundred eighteen patients (218/344, 63.4%) had a FAH SDS > − 2 SD. Among these patients, girls in the 1-2y treatment course group and ≥ 2y group had a FAH SDS higher than TH SDS. Even in the control group, a spontaneous catch-up growth of 1.16 SD was observed. A multivariate linear regression model was used to analyze the results, with FAH SDS as the dependent variable. It was found that the treatment course and baseline height SDS in the boys' model were statistically significant (P < 0.05), whereas the baseline height SDS and baseline bone age significantly affected the girls' FAH SDS (P < 0.05). Conclusions: Both girls and boys of ISS improved FAH by GH therapy even if treatments begin over 10 years old and majority of them reached TH. Some peri-puberty ISS will have a spontaneous height gain. We recommend the course of GH treatment more than 2 years for girls, and longer courses for boys.

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Section: Discussionmentioning

confidence: 99%

Final adult height of children with idiopathic short stature: a multicenter study on GH therapy alone started during peri-puberty

Chen

et al. 2020

BMC Pediatr

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“…In their systematic review Graham et al (7) categorized potentially modifiable factors associated with treatment nonadherence amongst young patients receiving GHT according to the COM-B framework. The COM-B posits that human behavior (B) results from the interaction between psychological and physical capabilities (C), opportunities provided by the social and physical environment (O) and reflective and automatic motivation (M) (23).…”

Section: Discussionmentioning

confidence: 99%

“…Two questions of the "Fear" subscale can be classified as physical opportunity factors (discomfort and pain associated with daily injection) and two as reflective motivation factors (low confidence regarding self-administration). The "Supplies" subscale can be classified as a psychological capability factor since it covers mainly failure to renew the prescription (7) and not logistical problems (such as cost, access, packaging) (24) which would fall into the physical opportunity sub-component. Thus, the BAR-GHT items could be classified within the COM-B framework, even though some subscales may not map directly onto a single sub-component.…”

Section: Discussionmentioning

confidence: 99%

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Development and Psychometric Evaluation of an Instrument Assessing Barriers to Growth Hormone Treatment (BAR-GHT)

Zwaan

Fischer-Jacobs

Wabitsch³

et al. 2020

Front. Endocrinol.

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Purpose: This paper presents development and validation of a new patient reported outcome measure (PRO), the Barriers to Growth Hormone Therapy (BAR-GHT) in a patient (child/adolescent) and a parent version. The BAR-GHT was developed to measure problems and potential barriers to GHT. Methods: The development and validation of the BAR-GHT was conducted according to the Food and Drug Administration (FDA) Guidance on the development of PROs. Concept elicitation included a literature review and open-ended interviews with young patients, parents, and clinical experts. Qualitative data were analyzed based on grounded theory principles and draft items were rated in terms of their importance and clarity. The instruments underwent psychometric validation in a German clinic-based patient population of children and adolescents who inject themselves and in a parent sample who inject their child. The statistical analysis plan included exploratory factor analysis, reliability, and validity. Results: 29 patients, 22 parents, and 4 clinical experts participated in the concept elicitation, 156 children and adolescents aged 8-18 years and 146 parents completed the validation study. Exploratory factor analysis resulted in six domains: Fear, Public Embarrassment, Annoyance, Daily Routine, Supplies, and Travel. Internal consistencies and test-retest reliabilities of the total score of both the patient version and the parent version were >0.8. Convergent and discriminant validity was demonstrated. Conclusions: The final 19-item BAR-GHT for patients aged 8-18 years and the 16-item version for parents can be considered reliable and valid PROs of barriers to GHT.

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“…Adherence is likely to be an important factor for individual responsiveness, although this has not been extensively studied. Factors that can be modified, based on pediatric studies, to improve adherence are lack of knowledge and understanding of the condition and its treatment, discomfort and pain associated with injections, and the quality of the healthcare professional-patient relationship (39).…”

Section: Adherencementioning

confidence: 99%

Personalized approach to growth hormone replacement in adults

Bunderen¹,

Glad²,

Johannsson³

et al. 2019

Archives of Endocrinology and Metabolism

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Growth hormone (GH) deficiency (GHD) in adults is well-characterized and includes abnormal body composition, reduced bone mass, an adverse cardiovascular risk profile, and impaired quality of life. In the early 1990s, it was also shown that patients with hypopituitarism without GH replacement therapy (GHRT) had excess mortality. Today, GHRT has been shown to decrease or reverse the negative effects of GHD. In addition, recent papers have shown that mortality and morbidity are approaching normal in hypopituitary patients with GHD who receive modern endocrine therapy including GHRT. Since the first dose-finding studies, it has been clear that efficacy and side effects differ substantially between patients. Many factors have been suggested as affecting responsiveness, such as sex, age, age at GHD onset, adherence, and GH receptor polymorphisms, with sex and sex steroid replacement having the greatest impact. Therefore, the individual tailoring of GH dose is of great importance to achieve sufficient efficacy without side effects. One group that stands out is women receiving oral estrogen replacement, who needs the highest dose. Serum insulin-like growth factor-1 (IGF-1) is still the most used biochemical biomarker for GH dose titration, although the best serum IGF-1 target is still debated. Patients with GHD due to acromegaly, Cushing's disease, or craniopharyngioma experience similar effects from GHRT as others.

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Identifying Potentially Modifiable Factors Associated with Treatment Non-Adherence in Paediatric Growth Hormone Deficiency: A Systematic Review

Cited by 59 publications

References 35 publications

Final adult height of children with idiopathic short stature: a multicenter study on GH therapy alone started during peri-puberty

Final adult height of children with idiopathic short stature: a multicenter study on GH therapy alone started during peri-puberty

Development and Psychometric Evaluation of an Instrument Assessing Barriers to Growth Hormone Treatment (BAR-GHT)

Personalized approach to growth hormone replacement in adults

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