ArticleDevelopmental medicine and child neurology2026
Real-world benefits and tolerability of trofinetide for the treatment of Rett syndrome: The LOTUS study.
Article in Developmental medicine and child neurology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers.
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The trial behind it
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Who cites it
9 citing papers in PubMed.
- Rett syndrome: MECP2 biology, multisystem pathophysiology, and the evolving therapeutic landscape.European journal of pediatrics · 2026Review
- Mapping Trofinetide Polypharmacology in Rett Syndrome: A Multi-Stage Computational Analysis.Journal of computational chemistry · 2026Article
- Real-world benefits and tolerability of trofinetide for the treatment of Rett syndrome: The LOTUS study.Developmental medicine and child neurology · 2026Article
- Expert Consensus on Real-World Use of Trofinetide for Rett Syndrome Using a Modified Delphi Method.Annals of the Child Neurology Society · 2026Article
- Real-World Treatment Landscape of Individuals with Rett Syndrome Treated and Untreated with Trofinetide in the United States.Journal of health economics and outcomes research · 2026Article
- Trofinetide Use and Treatment Patterns Among Children Aged 2-4 Years with Rett Syndrome in the United States: A Retrospective Specialty Pharmacy Linked Claims Database Analysis.International journal of general medicine · 2026Article
- Anticodon-engineered tRNAs restore full-length MeCP2 expression and function in Rett syndrome nonsense mutations.Frontiers in neurologyArticle
- Disease-modifying therapies for Rett syndrome: a review for neurologists.Frontiers in neurologyReview
- Clinical experience using trofinetide in Rett syndrome and related MECP2 diagnosis at the children's hospital of Philadelphia post approval.Frontiers in neurologyArticle
Corrections and comments
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Authors and funding
4 authors.
Funding
Abstract
aimTo describe the real-world effects of trofinetide in individuals with Rett syndrome (RTT) using the 18-month follow-up analysis of the LOTUS study.
methodCaregivers of any patients who were prescribed trofinetide under routine clinical care were eligible to participate. Assessments included the Behavioral Improvement Questionnaire (BIQ), the Quality of Life Inventory-Disability (QI-Disability) questionnaire, the Gastrointestinal Health Questionnaire, and safety monitoring. Data are reported to 12 months since the initiation of trofinetide.
resultsOverall, 227 caregivers participated in this follow-up. The median trofinetide dose at week 1 was 36.0% of the target dose and increased to over 80% by week 10 onwards; 55.2% of patients took the full trofinetide recommended daily dose. Caregivers (71%-90%) reported behavioral improvements across months 1 to 12 with the BIQ, including non-verbal communication (48%-71%), alertness (44%-70%), and social interaction and connectedness (33%-58%). The median QI-Disability total score change from baseline ranged from 4.7 to 4.6 in months 1 to 12. The incidence of diarrhea varied from weeks 1 to 12 (23%-50%) and months 4 to 12 (26%-38%); most reports of diarrhea were contained inside the patient's diaper. Safety reports were consistent with previous trofinetide clinical trials.
interpretationResults from the LOTUS study show improvements in the symptoms of RTT, with early insights into managing gastrointestinal symptoms in the context of real-world patient care.
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