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Expert Consensus on Real-World Use of Trofinetide for Rett Syndrome Using a Modified Delphi Method
Erin O'Connor Prange1, Arthur Beisang2, Davut Pehlivan3,4
1Division of Neurology Children's Hospital of Philadelphia Philadelphia Pennsylvania USA.
Annals of the Child Neurology Society
|August 7, 2026
Summary
Trofinetide is the first approved treatment for Rett syndrome (RTT). Expert consensus now guides its practical use, emphasizing early initiation and individualized care to manage symptoms and side effects effectively.
Area of Science:
- Neurology
- Rare Diseases
- Pharmacology
Background:
- Rett syndrome (RTT) is a rare neurodevelopmental disorder.
- Trofinetide represents the first FDA-approved treatment for RTT.
- Real-world experience with trofinetide has accumulated over nearly three years.
Purpose of the Study:
- Establish consensus recommendations for the practical application of trofinetide in RTT treatment.
- Provide guidance for healthcare professionals based on expert experience.
- Optimize the use of trofinetide for eligible RTT patients.
Main Methods:
- A modified Delphi process involving RTT experts was utilized.
- A steering group developed 72 consensus statements across six domains.
- Two rounds of assessment with 25 respondents achieved ≥75% agreement.
Main Results:
- Consensus supports trofinetide as standard of care for eligible patients.
- Early initiation and individualized titration are recommended.
- Trofinetide is considered efficacious, with manageable side effects.
Conclusions:
- Recommendations emphasize a flexible, patient-centered approach to trofinetide therapy.
- Optimizing efficacy and tolerability are key considerations.
- Strategies to support adherence and manage side effects are highlighted.
