Real-world use of trofinetide: a survey of practices from prescribers at Rett syndrome centers of excellence in the United States
Notice bibliographique
Résumé
Trofinetide is approved for the treatment of Rett syndrome (RTT) in the United States and Canada. The objective of this study was to describe RTT expert approach to trofinetide titration and observed impact in their practice. In May 2024, an electronic survey was sent to prescribers at RTT centers of excellence (COEs) in the United States to collect real-world experience with trofinetide dosing strategies and observed impact on tolerability. Overall, 67% (22/33) of prescribers from 89% (16/18) of COEs responded to the survey. Most respondents (95%, n = 21) indicated they titrate trofinetide and 86% (n = 19) believed titration improved tolerability. Titration strategies included initiating at a lower percentage (50%, n = 11) or initiating at lower milliliters (27%, n = 6) than label dose, or individualized starting dose based on baseline conditions (14%, n = 3). Respondents estimated a median 75% of patients achieve their label dose after titration. Respondents encourage families to remain on trofinetide for a median of ≥16 weeks to adequately evaluate efficacy; most (77%, n = 17) indicated this period should start after the patient arrives at their highest tolerable dose. Survey respondents from RTT COEs in the United States are most commonly titrating trofinetide to improve tolerability while decreasing treatment discontinuations. Trofinetide is recommended to be administered using weight-banded dosing, yet dosing flexibility has been observed in the trofinetide clinical trial program and the real worldAn electronic survey was sent in May 2024 to prescribers at Rett syndrome centers of excellence in the United States to collect their real-world experience with trofinetide dosing strategies and impact on tolerabilityMost survey respondents indicated that they titrate trofinetide (ie, initiating at a lower percentage or milliliters than label dose, or start at an individualized dose based on baseline conditions) in treatment-naïve patients and that titration improved tolerability in their practiceApproximately 75% of patients achieve their label dose after titrationRespondents encourage families to remain on trofinetide for a median of at least 16 weeks to adequately evaluate efficacy; this period should start after the patient arrives at their highest tolerable dose Trofinetide is recommended to be administered using weight-banded dosing, yet dosing flexibility has been observed in the trofinetide clinical trial program and the real world An electronic survey was sent in May 2024 to prescribers at Rett syndrome centers of excellence in the United States to collect their real-world experience with trofinetide dosing strategies and impact on tolerability Most survey respondents indicated that they titrate trofinetide (ie, initiating at a lower percentage or milliliters than label dose, or start at an individualized dose based on baseline conditions) in treatment-naïve patients and that titration improved tolerability in their practice Approximately 75% of patients achieve their label dose after titration Respondents encourage families to remain on trofinetide for a median of at least 16 weeks to adequately evaluate efficacy; this period should start after the patient arrives at their highest tolerable dose Trofinetide is approved for the treatment of Rett syndrome (RTT) in the United States and Canada. Trofinetide is recommended to be administered based on patient weight, yet flexible dosing was observed in clinical trials and the real world. Namely, clinicians are starting trofinetide at lower doses than recommended and slowly increasing the dose over time. A survey of experts at RTT centers of excellence in the United States was designed to collect and describe expert approach to, reasons for, and estimated impact of trofinetide dosing changes and increases in their practice. Most survey respondents indicated that they start trofinetide at doses lower than recommended and increase the dose with the goal of reaching the recommended dose. Experts have observed that this approach to trofinetide dosing improves trofinetide tolerability and caregiver and patient quality of life, while decreasing trofinetide discontinuation. The respondents also indicated that most patients can achieve their recommended dose and should try trofinetide for ≥16 weeks at their label dose or highest tolerable dose to adequately assess efficacy.
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Comment cette classification a été obtenuedéplier
Prédiction machine sur la base complète
Imitation des enseignantsNi prévalence calibrée, ni vérité terrain. Validation humaine à venir. Le volet Gemma est une étiquette directe du modèle pour chaque travail de la base, lue sur la notice réduite au titre. Le volet Codex est un classifieur appris des 10 348 étiquettes directes de Codex et calibré sur les taux pondérés de l'échantillon; les champs sans appui suffisant ne portent aucun appel Codex. Le mode candidate est l'union des deux volets; le consensus est leur intersection. Ces sorties portent le statut machine_predicted_unvalidated et ne sont pas des étiquettes humaines.
Scores du classifieur distillé par catégorie (deux têtes)
| Catégorie | Codex | Gemma |
|---|---|---|
| Métarecherche | 0,003 | 0,013 |
| Méta-épidémiologie (sens strict) | 0,000 | 0,000 |
| Méta-épidémiologie (sens large) | 0,000 | 0,000 |
| Bibliométrie | 0,001 | 0,001 |
| Études des sciences et des technologies | 0,001 | 0,001 |
| Communication savante | 0,001 | 0,001 |
| Science ouverte | 0,000 | 0,001 |
| Intégrité de la recherche | 0,001 | 0,001 |
| Charge utile insuffisante (le modèle a refusé de juger) | 0,001 | 0,000 |
Scores machine (provisoires)
Les deux têtes enseignantes du modèle étudiant, lues sur ce travail. Un score ordonne la base pour la relecture; il n'affirme jamais une catégorie, et le statut de validation accompagne chaque rangée tel quel.
Scores de référence d'un modèle non mature (critères de maturité non atteints, 7 itérations). Un score ordonne; il n'affirme jamais une catégorie.
score_only:v0-immature-baseline · tel quel depuis la passe de notation : score_only signifie que le nombre peut ordonner les travaux, et qu'aucune étiquette de catégorie n'en découleClassification
machine, non validéePrédiction automatique; un appel candidat d’une seule source (Gemma direct ou Codex distillé), pas un consensus.
Le détail, modèle par modèle et score par score, se trouve en fin de page sous « Comment cette classification a été obtenue ».