SAT-220 Real-world Evidence Of Treatment Patterns And Costs Of Turner Syndrome And Noonan Syndrome In The United States

Abstract Disclosure: A.N. Dauber: Novo Nordisk, Pfizer, Inc., BioMarin, QED. M.J. Abuzzahab: Ascendis, Lumos, Medtronic, Novo Nordisk, Rhythm, Soleno, Endo Pharmaceuticals, Neurocrine, Pfizer, Inc. J. Chen: Novo Nordisk. N. Kelepouris: Novo Nordisk. M. Fridman: Novo Nordisk. M. Shy: Novo Nordisk. S. Bunner: Novo Nordisk. A. Romano: Novo Nordisk, BioMarin. V. Bamba: Lumos Pharma, Amryt Pharma. Background: Turner and Noonan syndromes are pediatric non-growth hormone-deficiency conditions (PNGCs). Short stature is one of the most common clinical features of both genetic conditions. The syndromes also have characteristic facial features, cardiovascular anomalies, and multiple comorbidities. Growth hormone (GH) treatment significantly increases the achievement of near-normal adult height for patients with PNGCs. However, little is known about the treatment patterns for these two PNGCs in a real-world setting in the United States. We aimed to assess the age of diagnosis, GH treatment rates, time-to-treatment initiation, adherence, persistence, and costs among incident GH-treated pediatric patients diagnosed with each PNGC. Methods: This retrospective analysis used the Komodo Health claims database. The study population included patients with Turner syndrome (aged 4 to <14 years at index date) and Noonan syndrome (aged ≤18 years); a cohort of patients with each PNGC was created and analyzed separately. The index date was defined as the first visit or the latest of two visits with diagnostic coding indicating Turner syndrome or Noonan syndrome, respectively. Patients were included in the analysis if they had continuous medical and pharmacy claims for ≥6 months pre-index (baseline) after January 1, 2016, and ≥1 year post-index (follow-up) before September 30, 2022. The time-to-treatment initiation, adherence, and persistence to GH therapy were assessed and dichotomized to define early/late initiation (GH treatment start ≤6/>6 months post-index), high/low adherence (taking ≥80%/<80% of the days covered), and long/short persistence (≥2 years/<2 years of continuous GH treatment). Descriptive analyses were conducted. Healthcare costs were summarized among those with one or more claims within each setting; costs were inflation-adjusted to 2023 US dollars. Results: The mean (SD) age of the 2,530 patients with Turner syndrome at the index date was 8.4 (3.0) years. A total of 902 (36%) patients initiated GH treatment during the follow-up period. Among these patients, 48% were early initiators, 66% had high adherence, and 56% had long persistence. The mean GH treatment duration was 24.9 (15.9) months. Mean total costs (all-cause, per patient per year) were $26,512 for the overall cohort. Of the patients with Noonan syndrome, 1,119 met the study criteria; the mean age was 7.0 (4.4) years. Of the 176 (16%) patients who initiated GH treatment, 41% were early initiators, 70% had high adherence, and 52% had long persistence. The mean GH treatment duration was 17.6 (10.1) months. Mean total costs were $52,594 for the patients with Noonan syndrome. Conclusion: Many patients with Turner or Noonan syndromes were not initiated on GH therapy; over half of those on GH treatment were not initiated early in their diagnosis. There is a need for greater awareness regarding early diagnosis and treatment of PNGCs. Presentation: Saturday, July 12, 2025