Recommendations for Managing Diarrhea from Trofinetide use in Individuals with Rett Syndrome: A Plain Language Summary

Plain Language Summary

What is this summary about?

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.

What were the results?

In the LAVENDER trial, no characteristics were found that could help to identify people who may develop diarrhea when taking trofinetide.

What were the recommendations?

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

What do the results mean?

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.

Video Abstract

Tweetable abstract

Expert recommendations on how to manage diarrhea if it occurs during trofinetide (DAYBUE) treatment for Rett syndrome @RettSyndrome #trofinetide #DAYBUE #FirstTreatmentInRettSyndrome #rettsyndrome #Acadiapharma

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Keywords::

• diarrhea
• gastroenterology
• guidelines
• lay summary
• plain language summary
• rare disease
• recommendations
• Rett syndrome
• trofinetide

This article is related to:

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

Acknowledgments

The authors thank the girls and women who took part in the LAVENDER trial and their caregivers.

Financial & competing interests disclosure

V Abler is an employee of Acadia Pharmaceuticals Inc. A Beisang is a consultant to Acadia. TA Benke has received funding for consulting from Acadia Pharmaceuticals Inc., Alcyone Therapeutics, GRIN Therapeutics, GW Pharmaceuticals, the International Rett Syndrome Foundation, Marinus Pharmaceuticals, Neuren Pharmaceuticals, Neurogene, Ovid Therapeutics, Takeda Pharmaceutical Company Limited, Ultragenyx, and Zogenix; and funding for clinical trials from Acadia Pharmaceuticals Inc., GW Pharmaceuticals, Marinus Pharmaceuticals, Ovid Therapeutics, and Rett Syndrome Research Trust; all remuneration has been made to his department. B Gaucher has no disclosures to report. KJ Motil has received funding for clinical research studies from the International Rett Syndrome Foundation and funding for consulting from Acadia Pharmaceuticals Inc. D Pichard has no disclosures to report. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Jennifer L. Giel, PhD, on behalf of Evidence Scientific Solutions, Philadelphia, PA, provided medical writing and editing services in the development of this manuscript. Acadia Pharmaceuticals Inc. (San Diego, CA) provided funding for this work.

Ethical conduct of research

The LAVENDER study protocol was approved by local institutional review boards. Informed consent was obtained from parents or guardians on behalf of the trial participants.

Additional information

Funding

V Abler is an employee of Acadia Pharmaceuticals Inc. A Beisang is a consultant to Acadia. TA Benke has received funding for consulting from Acadia Pharmaceuticals Inc., Alcyone Therapeutics, GRIN Therapeutics, GW Pharmaceuticals, the International Rett Syndrome Foundation, Marinus Pharmaceuticals, Neuren Pharmaceuticals, Neurogene, Ovid Therapeutics, Takeda Pharmaceutical Company Limited, Ultragenyx, and Zogenix; and funding for clinical trials from Acadia Pharmaceuticals Inc., GW Pharmaceuticals, Marinus Pharmaceuticals, Ovid Therapeutics, and Rett Syndrome Research Trust; all remuneration has been made to his department. B Gaucher has no disclosures to report. KJ Motil has received funding for clinical research studies from the International Rett Syndrome Foundation and funding for consulting from Acadia Pharmaceuticals Inc. D Pichard has no disclosures to report. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.