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

Rebecca Moore; Joshua Poulsen; Lindsay Reardon; Candice Samples-Morris; Holly Simmons; Keri M Ramsey; Meagan L Whatley; Jane B Lane

doi:10.1007/s12325-024-02782-4

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

Adv Ther. 2024 Apr;41(4):1305-1317. doi: 10.1007/s12325-024-02782-4. Epub 2024 Feb 20.

Authors

Rebecca Moore¹, Joshua Poulsen², Lindsay Reardon³, Candice Samples-Morris⁴, Holly Simmons⁵, Keri M Ramsey⁶, Meagan L Whatley⁷, Jane B Lane⁸

Affiliations

¹ Caregiver Author, Redmond, OR, USA.
² Caregiver Author, Albuquerque, NM, USA.
³ Caregiver Author, Darien, CT, USA.
⁴ Caregiver Author, Jacksonville, FL, USA.
⁵ Caregiver Author, West Chester, PA, USA.
⁶ Center for Rare Childhood Disorders, Translational Genomics Research Institute, Phoenix, AZ, USA.
⁷ University of Alabama at Birmingham, Birmingham, AL, USA.
⁸ University of Alabama at Birmingham, Birmingham, AL, USA. janelane@uabmc.edu.

Abstract

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).

Keywords: Diarrhea; Rett syndrome; Taste; Trofinetide; Vomiting.

Publication types

Clinical Trial, Phase III

MeSH terms

Caregivers
Causality
Female
Gastrointestinal Diseases* / chemically induced
Gastrointestinal Diseases* / drug therapy
Glutamates / therapeutic use
Humans
Rett Syndrome* / complications
Rett Syndrome* / diagnosis
Rett Syndrome* / drug therapy

Substances

Glutamates
trofinetide