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Impact of Daily Growth Hormone Adherence on Height Velocity Among Children With Growth Hormone Deficiency (GHD)
Bradley S Miller1, Jane Loftus2, Mona Kelkar3
1University of Minnesota Medical School, M Health Fairview Masonic Children's Hospital, Minneapolis, Minnesota.
Insights
Adherence to daily somatropin therapy in pediatric growth hormone deficiency showed minor, though not statistically significant, improvements in height velocity within one year. Further research is needed to confirm these findings in larger, diverse populations.
Area of Science:
- Pediatric Endocrinology
- Pharmacology
- Growth Hormone Therapy
Background:
- Pediatric growth hormone deficiency (GHD) requires consistent treatment for optimal outcomes.
- Somatropin is a common treatment, but adherence can impact efficacy.
- Real-world adherence data is crucial for understanding treatment effectiveness.
Purpose of the Study:
- To evaluate adherence to daily somatropin treatment in pediatric GHD patients.
- To assess the impact of adherence on height velocity within the first year of treatment.
- To analyze real-world adherence patterns in a US pediatric population.
Main Methods:
- Retrospective cohort study of pediatric patients (3 to <16 years) with GHD.
- Data collected from electronic health records and specialty pharmacy records (2015-2020).
- Adherence measured by Proportion of Days Covered (PDC) ≥80%; height velocity compared between adherent and nonadherent groups.
Main Results:
- 181 pediatric patients with GHD were included; 70.2% male, 73.5% white.
- Mean annualized height velocity was 10.2 cm/y in the adherent group (n=108) and 9.8 cm/y in the nonadherent group (n=66).
- The difference in height velocity between adherent and nonadherent groups was not statistically significant.
Conclusions:
- Minor improvements in average height velocity were observed with somatropin adherence, but not statistically significant.
- Potential reasons for lack of significance include small sample size, short observation period, and population heterogeneity.
- Further investigation with larger cohorts and longer follow-up is warranted to confirm findings.
Objective:
To describe adherence to daily somatropin treatment and impact on height velocity within 1 year of treatment start among patients with pediatric growth hormone deficiency in a real-world US population.
Methods:
This retrospective cohort study included pediatric patients aged ≥3 years to <16 years with pediatric growth hormone deficiency prescribed somatropin by a pediatric endocrinologist at a US-based center of excellence between January 1, 2015 and December 31, 2020. Patient data were collected using hospital electronic health records linked to a specialty pharmacy patient prescription records. Adherence, evaluated over 12 months, was measured using the proportion of days covered metric and patients were categorized as adherent if their proportion of days covered ≥80%. Height velocity was annualized to compare across adherent and nonadherent patients.
Results:
One hundred eighty-one patients were identified and included in this study, of which 70.2% were male,73.5% were white, and mean age (standard deviation [SD]) at index was 12.1 (2.8). In the height velocity analysis, 174 patients were included and the mean (SD) annualized change in height was 10.2 (5.7) cm/y in the adherent group (n = 108) and 9.8 (7.6) in the nonadherent group (n = 66). The difference in height velocity between the groups was not statistically significant.
Conclusions:
Minor improvements in average height velocity were observed in the patient group who were adherent to somatropin therapy, although not statistically significant. Lack of observed significance may be due to small sample sizes, short observation period, a likely heterogenous population in terms of growth hormone prescribing, data bias due to single-center origin, or potential patient misclassification.
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