Risk Factors Associated With an Aspiration Outcome in Patients With Rett Syndrome: Analysis of Real-World Data in the United States

Speaker(s)

Rashid N1, Rajagopalan K2, Gopal D2, Sikirica M3
1Acadia Pharmaceuticals, Inc, San Diego, CA, USA, 2Anlitiks Inc,, Windermere, FL, USA, 3Acadia Pharmaceuticals, San Diego, CA, USA

BACKGROUND: Rett (RTT) syndrome, is a rare progressive neurodevelopment disorder beginning in childhood, and results in multisystem clinical manifestations (e.g., respiratory, gastrointestinal (GI), and epilepsy). Real-world data is needed to better understand the risk factors associated with aspiration in patients with RTT syndrome.

OBJECTIVES: To examine risk factors associated with aspiration among incident RTT patients in the United States.

METHODS: A retrospective medical and pharmacy claims analysis from 8/1/2020 to 3/31/2023 using IQVIA APLD (Anonymized Patient Level Data) database was performed. Newly diagnosed patients with a confirmed diagnosis of RTT (ICD-10-CM: F84.2) were identified during 2/2/2021 to 3/31/2022 (index date). RTT patients were required to have no RTT diagnosis prior 2/2/2021 so that the cohort was incident, had to have 6 months prior as baseline period (pre-index), and 12 months follow up (post-index). An aspiration outcome was identified during post index using diagnosis codes. A multivariate logistic regression was conducted to identify risk factors associated with the aspiration outcome.

RESULTS: Among patients with RTT (n=7,418), there were 1,994 incident RTT patients identified and evaluated in this analysis. Mean age was 21.2 (SD:17.7); 51.1% were <18yrs of age and 78.9% were female. There were 145 (7.2%) patients with an aspiration outcome and the mean time to an aspiration outcome from RTT diagnosis was 125 days. Risk factors associated with an aspiration outcome were: Lower respiratory tract infection (LRTI) [OR: 2.49 (95% CI 1.31-4.79, p=0.0061)]; cough [OR 3.45 (95% CI 1.86-6.40, p<0.05)]; dysphagia [OR 3.44 (95% CI 2.16-5.47, p<0.05); epilepsy/convulsions [OR 1.93 (95% CI 1.31-2.85), p<0.05)].

CONCLUSIONS: History of cough, dysphagia and epilepsy/convulsions were identified as statistically signification risk factors associated with an aspiration. An aspiration event can contribute to the overall morbidity of individuals with RTT across their lifespan.

Code

CO193

Topic

Clinical Outcomes, Epidemiology & Public Health, Study Approaches

Topic Subcategory

Comparative Effectiveness or Efficacy

Disease

Rare & Orphan Diseases