Recommendations for the management of diarrhea with trofinetide use in Rett syndrome

Background Trofinetide is a recently approved treatment for Rett syndrome (RTT), rare neurodevelopmental disorder with no previously therapy. The phase 3 LAVENDER trial showed improvements in efficacy measures compared placebo, but diarrhea rates were high trofinetide-treated participants. To manage possible diarrhea, recommendations that can be used by health care providers are needed.Research design and methods Additional analyses conducted on data to elucidate predictors of trofinetide-associated diarrhea. A panel advisors was convened refine set practical the prevention management individuals RTT treated trofinetide.Results No examined demographic or factors appeared influence Advisors recommend establishing baseline bowel activity providing caregivers information. On initiation trofinetide, constipation medications should stopped reduced, concomitant liquid sugar alcohols substituted if possible, fiber initiated. Bowel movements tracked loperamide started following onset Dietary hydration also recommended.Conclusions confers RTT-related symptoms. With these recommendations, associated trofinetide use managed, enhancing lives caregivers.