Summary

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• This summary is based on the review of two systematic review(s)/meta-analysis(es). ^[1-2]
• In patients with Rett syndrome, trofinetide improved Rett Syndrome Behavior Questionnaire (RSBQ) scores, with a mean difference (MD) of -3.46 points (95% confidence interval (CI): -5.63 to -1.27, P = 0.0002) in one study and -3.53 points (P = 0.001) in another at a 200 mg dosage.
• Trofinetide significantly improved Clinical Global Impression Scale-Improvement (CGI-I) scores, with an MD of -0.35 (95% CI: -0.51 to -0.18, P < 0.0001) in one study and -0.34 (P < 0.0001) in another.
• No significant changes were observed in caregiver concerns (Visual Analog Scale) or Rett Motor Behavioral Assessment (MBA) outcomes in either study.
• In one study, trofinetide was associated with a significantly higher incidence of vomiting compared to placebo (odds ratio (OR): 3.17, 95% CI: 1.57 to 6.43, P = 0.001), and diarrhea was also significantly more frequent (OR: 18.51, 95% CI: 9.30 to 36.84, P ≤ 0.00001), leading to treatment discontinuation in some cases.
• In another study, diarrhea and vomiting were reported as common treatment-emergent adverse events at the 200 mg dosage, with irritability also noted as an adverse event in this dosage group.
• The studies focused on patients with Rett syndrome, primarily affecting females, and included both pediatric and adult populations. Statistically significant improvements were observed in CGI-I and RSBQ scores across these groups, with no specific differences reported based on gender or other subgroups.