ESPE Abstracts

Background: For many years, raising awareness on early referral to endocrinologist, early diagnosis, and treatment continuation for paediatric patients with growth hormone deficiency (GHD) has been continued in Japan. However, the current status of trends in age at diagnosis and of treatment continuation have not been fully clarified.

Aim: The aim of this study is to estimate the persistence to growth hormone treatment (GHT) in Japanese patients with paediatric GHD. Proportion of days covered (PDC), demographics and clinical characteristics are also explored.

Method: This was a retrospective cohort study using the JMDC claims database. Patients with paediatric GHD were identified using disease codes and those who cannot be observed 12 months prior to or 3 months after the initial diagnosis were excluded. To evaluate persistence, we selected patients with paediatric GHD receiving GHT and GH stimulation tests. Persistence was defined as the proportion of patients without interruptions (no prescription for more than 6 months) to all patients at a given point. Discontinuation of GHT was defined as a prescription-free period of at least 6 months after last prescription of GH in the observation period. PDC was defined as the ratio of the length of the period with GHT to the length of the observation period.

Result: A total of 1,594 patients were identified. Regarding persistence, the time to initial treatment interruption for 50% of the patients was less than a year (307 days). 87.0% of patients who interrupted GHT had resumed GHT. Time to discontinuation for 50% of the patients was about 6 years (1,780 days). The factors affecting longer time to treatment interruption and their hazard ratio (95%CI) were higher number of patients per facility (0.80[0.70-0.93]) and concomitant use of pituitary hormonal agents other than GH (0.62[0.39-0.99]). Mean PDC was 52.7±32.9% and proportion of patients without interruption was only 22.5%. The mean age at initial diagnosis was 7.8±3.8 years. The age at diagnosis decreased until 2016 and afterwards tended to increase.

Conclusion: Most of Japanese paediatric GHD patients experienced at least one treatment interruption and PDC was almost half. Although most of patients who experienced interruption had resumed GHT, challenges with real life treatment persistence still exist. Adequate support and education on the importance of continuation of therapy would be needed to maintain the benefits of GHT, while limitations of database analysis should be considered. Continued efforts for early referral and early diagnosis are important.