Recommendations for managing diarrhea from trofinetide use in individuals with Rett syndrome: a plain language summary

Rett syndrome is a rare genetic disorder that affects the way the brain develops. The medication trofinetide (DAYBUE™) was studied in a large clinical trial called LAVENDER, where it showed a benefit in reducing symptoms of Rett syndrome versus placebo (placebo did not contain medication but looked the same as trofinetide and was taken in the same way). The most common side effect in the trial was diarrhea (frequent and/or watery bowel movements). In order to help caregivers and healthcare providers, experts created recommendations on how to prevent and manage diarrhea if it occurs during trofinetide treatment. In the LAVENDER trial, no characteristics were found that could help to identify people who may develop diarrhea when taking trofinetide. The diarrhea management recommendations include: Keep a diary of the frequency (how often) of bowel movements and their consistency (shape, hardness/softness) before starting trofinetide On starting trofinetide, discuss stopping or reducing medicines for constipation with the healthcare provider             – Ask the healthcare provider to swap other liquid medications with sugar alcohols to a pill form if possible             – Introduce dietary fiber At the first sign of diarrhea, contact the healthcare provider and start antidiarrheal medication             – A stool (feces) diary should be kept, noting frequency and consistency along with monitoring how much liquid the person is drinking Follow a regular diet when taking trofinetide             – Those with mild dehydration can be given an oral rehydration solution, but the healthcare provider should be contacted for moderate or severe dehydration These practical recommendations may help caregivers to manage diarrhea so people can continue to take trofinetide, allowing individuals with Rett syndrome and their caregivers to experience its benefits.