Real-world benefits and tolerability of trofinetide for the treatment of Rett syndrome: The LOTUS study.
Louise Cosand, Haya Mayman, Jenny Downs and 1 others
PMID 40936177WHAT IT FOUND
Caregivers reported improvements in communication, alertness, and social connection for 71% to 90% of patients after 12 months of trofinetide.
Diarrhea was common but often manageable, with serious adverse events occurring in 4.4% of patients.
Key findings
01Caregivers reported behavioral improvements in 71% to 90% of patients during months 1 to 12, most frequently in non-verbal communication, alertness, and social interaction.
02Quality of life scores improved modestly, with the median total score rising from 55.1 at baseline to 64.9 at month 12 on a 0 to 100 scale.
03Diarrhea was reported by 79% of caregivers at some point in the first 12 weeks, though serious adverse events occurred in only 4.4% of patients.
STILL TO COME
How it was doneWhat they foundWhat it means for PTsWhat it means for OTsWhat it means for SLPs
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What it does not show
The study is observational with no control group, so it cannot determine if improvements were caused by trofinetide or other factors like natural history or concurrent care. The Behavioral Improvement Questionnaire and Gastrointestinal Health Questionnaire are new measures that have not undergone psychometric testing. Adverse events were not actively solicited, which may lead to underreporting. Data are restricted to 12 months, and the number of patients reaching later time points was limited. Reliance on caregiver reports introduces potential bias.
Declared interests
The study was funded by Acadia Pharmaceuticals, Inc., the manufacturer of trofinetide.
The easy way to misread this
Do not interpret the high rates of reported improvement as proof of efficacy. This was an uncontrolled observational study, and caregivers knew their patients were receiving the treatment. The improvements reported may reflect placebo effects, natural history, or other concurrent therapies rather than a direct drug effect.
Summarised by AI from the full paper, without a clinician reviewing it. Check it against the source before it changes what you do. Read it on PubMed →