Real-World Evidence of Treatment Patterns and Costs of Turner Syndrome and Noonan Syndrome in the USA

INTRODUCTION: Short stature is a common clinical feature of Turner syndrome (TS) and Noonan syndrome (NS). Growth hormone (GH) treatment increases height in patients with TS and NS. We aimed to assess treatment patterns and costs in a real-world setting of GH-treated patients with TS and NS in the USA. METHODS: Patients with TS (aged 4 to <14 years) or NS (aged ≤18 years) were included in this retrospective analysis using the Komodo Health claims database. The study period from January 1, 2016, to September 30, 2022, included ≥6 months pre-index (baseline) and ≥1 year post-index (follow-up); the index date was the first (TS) or latest of two visits (NS) with diagnostic coding for the condition. Data were analyzed descriptively. RESULTS: Among 2,530 patients with TS (mean age SD 8.4 3.0 years) and 1,119 patients with NS (mean age 7.0 4.4 years), 36% and 16% initiated GH treatment during follow-up, respectively. Of these patients, 48%/41% were early initiators (started ≤6 months post-index), 66%/70% had high adherence (≥80% of GH treatment days covered), and 56%/52% had long persistence (treated ≥2 years). Mean GH treatment duration was 24.9 (15.9) and 17.6 (10.1) months for patients with TS and NS, respectively. Median (IQR) total all-cause costs per patient/year were USD 10,232 (USD 2,850-USD 36,590) and USD 17,937 (USD 5,616-USD 47,552), respectively. CONCLUSION: Of the few patients on GH therapy, less than half were initiated on treatment early after their TS or NS diagnosis. Greater awareness regarding early diagnosis and treatment of TS and NS is needed.