Gastrointestinal manifestations in pediatric and adult patients with Rett syndrome: an analysis of US claims and physician survey data
oleh: Damian M May, Jeffrey Neul, Jesus Eric Pina-Garza, Kale Kponee-Shovein, Ambika Satija, Malena Mahendran, Nathaniel Downes, Kristy Sheng, Neema Lema, Andra Boca, Patrick Lefebvre, Victor Abler, James M Youakim, Wendy Y Cheng
| Format: | Article |
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| Diterbitkan: | Becaris Publishing Limited 2023-11-01 |
Deskripsi
Aim: Patients with Rett syndrome (RTT) experience gastrointestinal (GI) manifestations. This study aimed to describe the prevalence of GI manifestations and the associated medical costs in patients with RTT in the USA. Patients & Methods: The study combined an insurance claims database analysis with a survey of 100 physicians experienced in RTT management. Results: GI manifestations affected 43.0% of 5940 patients, with increased prevalence in pediatric patients (45.6%) relative to adult patients (40.2%). Annualized mean medical cost of managing GI manifestations was $4473. Only 5.9–8.2% of neurologists and pediatricians ranked GI symptom management among the five most important treatment goals. Conclusion: Patients with RTT experience a high burden of GI manifestations, which translate to considerable medical costs. Importantly, the prevalence of GI manifestations was likely underestimated in this study, as only those symptoms which resulted in a healthcare encounter were captured.