Documenting Trofinetide Treatment Phenotypes in Rett Syndrome with a Dual-LLM Consensus Pipeline
Objective: Trofinetide, approved in 2023 as the first Rett syndrome treatment, lacks scalable real-world monitoring because chart review is labor-intensive. We developed a dual large language model (LLM) consensus pipeline to extract 18 treatment-response phenotypes, comprising 4 adverse effects, 13 Rett clinical features, and medication refusal, from clinical notes. Materials and Methods: We analyzed 26,763 notes from 108 patients with confirmed sustained trofinetide exposure at Texas Children's Hospital. The pipeline filters notes by keyword, classifies each through two LLMs requiring consensus, and aggregates a patient-by-timepoint matrix. Architecture and prompt were selected on a dia...
| Authors | Daniel Palacios, Guantong Qi, Mikael Guzman Karlsson et al. |
|---|---|
| Primary category | q-bio.QM |
| Published | 15 October 2026 |
| Last updated at source | 15 October 2026 |
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