ESPE2021 ePoster Category 2 Growth and syndromes (to include Turner syndrome) (56 abstracts)
Association of Daily Growth Hormone Injection Adherence and Height among Children with Growth Hormone Deficiency
Jane Loftus 1 , Bradley S Miller 2 , Craig Parzynski 3 , Jose Alvir 4 , Yong Chen 5 , Priti Jhingran 3 , Anu Gupta 4 , Mitch DeKoven 6 , Victoria Divino 7 , Jenny Tse 8 , Jing He 9 & Michael Wajnrajch 4
1Pfizer, Tadworth, United Kingdom; 2University of Minnesota Masonic Children’s Hospital, Minneapolis, USA; 3Genesis Research, Hoboken, USA; 4Pfizer Inc, New York, USA; 5Pfizer Inc, Collegeville, USA; 6IQVIA, Falls Church, USA; 7Cambridge, Falls Church, USA; 8IQVIA, Cambridge, USA; 9IQVIA, Plymouth Meeting, USA
Background: Use of recombinant human growth hormone (somatropin) is recommended for children with growth hormone deficiency (GHD) to normalize adult height and avoid extreme short stature. Prior research conducted among small study samples, across different study populations and health care systems, has suggested an association between adherence to somatropin and clinical outcomes, including height velocity. There is a need for further research, using real-world data (RWD) among additional populations, to establish and quantify a definitive link between adherence to somatropin and height.
Objective: To investigate the association between adherence to somatropin and change in height among children with GHD.
Methods: This retrospective cohort study utilized IQVIA PharMetrics® Plus and Ambulatory Electronic Medical Records databases. Patients were aged 3-15 years, had ≥1 diagnosis code for GHD, and newly initiated somatropin treatment between January 1, 2007 and November 30, 2019. Patients were followed for a minimum of 6 months and up to 12 months post-treatment initiation. Adherence was measured using the medication possession ratio (MPR); patients were classified as adherent (MPR ≥0.8) or non-adherent (MPR<0.8). Mean patient growth trajectories for each adherence group were modelled in the 12 months pre- and post-treatment initiation using a mixed model for repeated measures (MMRM), controlling for age, gender, and weight. Separate estimates of patient growth pre- and post- treatment initiation were included in the model.
Results: Among 201 patients newly initiated somatropin, 74.6% were male and mean age was 11.4 (SD 3.0) years. Approximately 76.6% of patients were adherent to daily somatropin therapy over the variable follow-up (mean duration of 343.3 days). Adherent and non-adherent patients were generally similar in terms of demographic and baseline clinical characteristics. In the MMRM, adjusted growth trajectories were similar between the adherent and non-adherent patients pre-treatment initiation (P = 0.15). However, growth trajectories post-initiation were significantly different (P = 0.001). On average, patients who were adherent gained an additional 1.8 cm over 1 year compared to non-adherent patients (height velocity in cm/year [95% CI]: 9.4 [8.9-9.9] vs 7.5 [6.6-8.5]), adjusted for covariates.
Conclusion: This study corroborates prior research suggesting that greater adherence to somatropin therapy is associated with improved height velocity, using RWD from a national US population. Given that suboptimal adherence to daily somatropin therapy is an issue for children with GHD, novel strategies to improve adherence may improve growth outcomes in this population.
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