Real-world Use of Trofinetide: A Survey of Tolerability from US Rett syndrome Centers of Excellence
Clinical Genetics and Therapeutics
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Primary Categories:
- Clinical- Pediatric
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Secondary Categories:
- Clinical- Pediatric
Introduction:
Trofinetide was approved by the US Food and Drug Administration in March 2023 for treating Rett syndrome in patients aged ≥2 years. Diarrhea was the most common adverse event and the leading cause of treatment discontinuation in clinical trials. Here, we present real-world experience with trofinetide titration and impact on tolerability.
Methods:
Trofinetide was approved by the US Food and Drug Administration in March 2023 for treating Rett syndrome in patients aged ≥2 years. Diarrhea was the most common adverse event and the leading cause of treatment discontinuation in clinical trials. Here, we present real-world experience with trofinetide titration and impact on tolerability.
Results:
Overall, 67% (22/33) of prescribers from 89% (16/18) of COEs participated, accounting for 4.6% of trofinetide prescribers and 38.1% of trofinetide prescriptions nationwide since approval. In total, 86% (n=19) of respondents indicated that titrating trofinetide improves tolerability in treatment-naïve patients. Tolerability improvements included decreased diarrhea severity (77%, n=17), decreased trofinetide discontinuations (73%, n=16), improved caregiver quality of life (64%, n=14), and improved patient quality of life (59%, n=13). Respondents indicated that approximately 20% of patients may still discontinue trofinetide due to tolerability issues; however, discontinuation due to lack of efficacy was estimated to be much lower at approximately 5-8%.
Conclusion:
Prescribers believe trofinetide titration helps with overall tolerability, specifically to decrease diarrhea severity and treatment discontinuations while improving patient and caregiver quality of life. Tolerability issues may persist in some patients which can lead to discontinuation. Prescribers believe discontinuation due to lack of efficacy remains low.
Trofinetide was approved by the US Food and Drug Administration in March 2023 for treating Rett syndrome in patients aged ≥2 years. Diarrhea was the most common adverse event and the leading cause of treatment discontinuation in clinical trials. Here, we present real-world experience with trofinetide titration and impact on tolerability.
Methods:
Trofinetide was approved by the US Food and Drug Administration in March 2023 for treating Rett syndrome in patients aged ≥2 years. Diarrhea was the most common adverse event and the leading cause of treatment discontinuation in clinical trials. Here, we present real-world experience with trofinetide titration and impact on tolerability.
Results:
Overall, 67% (22/33) of prescribers from 89% (16/18) of COEs participated, accounting for 4.6% of trofinetide prescribers and 38.1% of trofinetide prescriptions nationwide since approval. In total, 86% (n=19) of respondents indicated that titrating trofinetide improves tolerability in treatment-naïve patients. Tolerability improvements included decreased diarrhea severity (77%, n=17), decreased trofinetide discontinuations (73%, n=16), improved caregiver quality of life (64%, n=14), and improved patient quality of life (59%, n=13). Respondents indicated that approximately 20% of patients may still discontinue trofinetide due to tolerability issues; however, discontinuation due to lack of efficacy was estimated to be much lower at approximately 5-8%.
Conclusion:
Prescribers believe trofinetide titration helps with overall tolerability, specifically to decrease diarrhea severity and treatment discontinuations while improving patient and caregiver quality of life. Tolerability issues may persist in some patients which can lead to discontinuation. Prescribers believe discontinuation due to lack of efficacy remains low.