Gastrointestinal manifestations in pediatric and adult patients with Rett syndrome: an analysis of US claims and physician survey data
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 analysi...
| Main Authors: | , , , , , , , , , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Becaris Publishing Limited
2023-11-01
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| Series: | Journal of Comparative Effectiveness Research |
| Subjects: |
| _version_ | 1797324052742799360 |
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| author | 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 |
| author_facet | 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 |
| author_sort | Damian M May |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-03-08T05:36:46Z |
| format | Article |
| id | doaj.art-5cc5079a790f43a39c7bbd21326fc217 |
| institution | Directory Open Access Journal |
| issn | 2042-6313 |
| language | English |
| last_indexed | 2024-03-08T05:36:46Z |
| publishDate | 2023-11-01 |
| publisher | Becaris Publishing Limited |
| record_format | Article |
| series | Journal of Comparative Effectiveness Research |
| spelling | doaj.art-5cc5079a790f43a39c7bbd21326fc2172024-02-05T15:24:04ZengBecaris Publishing LimitedJournal of Comparative Effectiveness Research2042-63132023-11-0113110.57264/cer-2023-0054Gastrointestinal manifestations in pediatric and adult patients with Rett syndrome: an analysis of US claims and physician survey dataDamian M May0https://orcid.org/0000-0002-1258-6649Jeffrey Neul1Jesus Eric Pina-Garza2Kale Kponee-Shovein3Ambika Satija4Malena Mahendran5Nathaniel Downes6Kristy Sheng7Neema Lema8Andra Boca9Patrick Lefebvre10Victor Abler11James M Youakim12Wendy Y Cheng13Acadia Pharmaceuticals Inc., San Diego, CA 92130, USAVanderbilt University Medical Center, Nashville, TN 37232, USACentennial Children’s Hospital, Nashville, TN 37203, USAAnalysis Group, Inc., Boston, MA 02199, USAAnalysis Group, Inc., Boston, MA 02199, USAAnalysis Group, Inc., Montreal, H3B 0G7, CanadaAnalysis Group, Inc., Boston, MA 02199, USAAnalysis Group, Inc., Menlo Park, CA 94025, USAAnalysis Group, Inc., Menlo Park, CA 94025, USAAnalysis Group, Inc., Boston, MA 02199, USAAnalysis Group, Inc., Montreal, H3B 0G7, CanadaAcadia Pharmaceuticals Inc., San Diego, CA 92130, USAAcadia Pharmaceuticals Inc., San Diego, CA 92130, USAAnalysis Group, Inc., Boston, MA 02199, USAAim: 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.claims analysisgastrointestinal symptomsmedical costsorphan diseasephysician surveyprevalencerett syndrome |
| spellingShingle | 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 Gastrointestinal manifestations in pediatric and adult patients with Rett syndrome: an analysis of US claims and physician survey data Journal of Comparative Effectiveness Research claims analysis gastrointestinal symptoms medical costs orphan disease physician survey prevalence rett syndrome |
| title | Gastrointestinal manifestations in pediatric and adult patients with Rett syndrome: an analysis of US claims and physician survey data |
| title_full | Gastrointestinal manifestations in pediatric and adult patients with Rett syndrome: an analysis of US claims and physician survey data |
| title_fullStr | Gastrointestinal manifestations in pediatric and adult patients with Rett syndrome: an analysis of US claims and physician survey data |
| title_full_unstemmed | Gastrointestinal manifestations in pediatric and adult patients with Rett syndrome: an analysis of US claims and physician survey data |
| title_short | Gastrointestinal manifestations in pediatric and adult patients with Rett syndrome: an analysis of US claims and physician survey data |
| title_sort | gastrointestinal manifestations in pediatric and adult patients with rett syndrome an analysis of us claims and physician survey data |
| topic | claims analysis gastrointestinal symptoms medical costs orphan disease physician survey prevalence rett syndrome |
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