Managing Gastrointestinal Symptoms Resulting from Treatment with Trofinetide for Rett Syndrome: Caregiver and Nurse Perspectives.

Autor: Moore R; Caregiver Author, Redmond, OR, USA., Poulsen J; Caregiver Author, Albuquerque, NM, USA., Reardon L; Caregiver Author, Darien, CT, USA., Samples-Morris C; Caregiver Author, Jacksonville, FL, USA., Simmons H; Caregiver Author, West Chester, PA, USA., Ramsey KM; Center for Rare Childhood Disorders, Translational Genomics Research Institute, Phoenix, AZ, USA., Whatley ML; University of Alabama at Birmingham, Birmingham, AL, USA., Lane JB; University of Alabama at Birmingham, Birmingham, AL, USA. janelane@uabmc.edu.
Jazyk: angličtina
Zdroj: Advances in therapy [Adv Ther] 2024 Apr; Vol. 41 (4), pp. 1305-1317. Date of Electronic Publication: 2024 Feb 20.
DOI: 10.1007/s12325-024-02782-4
Abstrakt: Rett syndrome (RTT) is a rare genetic neurodevelopmental disorder mainly affecting female individuals. Trofinetide was recently approved as the first treatment for RTT, largely on the basis of results from the phase 3 LAVENDER trial, in which trofinetide showed improvements in core symptoms of RTT compared with placebo. However, gastrointestinal (GI) symptoms such as diarrhea and vomiting were commonly reported side effects, and taste was also a reported issue. The objective of this article is to describe the perspectives of five caregivers of girls in trofinetide clinical trials as well as those of three nurse trial coordinators, with a focus on management of GI symptoms of trofinetide treatment.Audio Abstract available for this article. Audio Abstract: Jane Lane provides an overview and discusses key findings of the article titled "Managing Gastrointestinal Symptoms Resulting from Treatment with Trofinetide for Rett Syndrome: Caregiver and Nurse Perspectives." (MP4 83274 KB).
(© 2024. The Author(s).)
Databáze: MEDLINE
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