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Meta-Analysis
. 2024 Oct;45(10):4767-4778.
doi: 10.1007/s10072-024-07584-8. Epub 2024 May 21.

A meta-analysis of the efficacy and safety of trofinetide in patients with rett syndrome

Mohamed Abo Zeid  1 , Amr Elrosasy  2 , Rashad G Mohamed  3 , Alina Ghazou  4 , Elarbi Goufa  5 , Nourhan Hassan  6 , Yasmine Abuzaid  7
Affiliations
Meta-Analysis

A meta-analysis of the efficacy and safety of trofinetide in patients with rett syndrome

Mohamed Abo Zeid et al. Neurol Sci. 2024 Oct.

Abstract

Background: Rett syndrome (RTT) is an uncommon inherited neurodevelopmental disorder that affects brain development, mostly in females. It results from mutation in MECP2 gene in the long arm (q) of the X chromosome.

Objective: Trofinetide is a recently developed drug that has a neuroprotective effect on neurons, and it is our aim in this meta-analysis to evaluate its efficacy and safety in treating Rett syndrome patients.

Methods: We searched 5 databases (PubMed, Scopus, Embase, Web of Science, and Cochrane Library databases) to identify randomized controlled trials (RCTs) comparing Trofinetide and placebo in patients with Rett syndrome until August 13, 2023.Our primary outcomes were the Clinical Global Impression-Improvement (CGI) and the Rett syndrome Behavior Questionnaire (RSBQ). We used Risk of Bias Assessment tool-2 (ROB2) to assess the methodological quality of the included randomized controlled trials.

Results: Three RCTs with a total of 325 patients were included with a follow-up duration ranging from one month to three months. 186 patients received the intervention drug (Trofinetide) and 138 received the placebo. Trofinetide was found to reduce CGI and RSBQ significantly more than placebo (MD = -0.35, 95% CI [-0.52 to -0.18], P 0.0001), (MD = -3.40, 95% CI [-3.69 to -3.12], P 0.00001) respectively. Most adverse events did not show any statistical difference between Trofinetide and the placebo.

Conclusion: Trofinetide offers promise as a potential effective and safe therapeutic opportunity for a population without many available treatments, with improvements seen on both CGI and RSBQ assessments and no severe adverse effects reported.

Keywords: Genetic disorder; Neurodevelopmental disorder; Quality of life; Rett syndrome; Trofientide.

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Conflict of interest statement

The authors declare that they have no competing interests.

Figures

Fig. 1
Fig. 1
PRISMA flow diagram of the included studies
Fig. 2
Fig. 2
Summary of assessment of risk of bias
Fig. 3
Fig. 3
Forest plot for Clinical Global Impression-Improvement (CGI)
Fig. 4
Fig. 4
Forest plot for Motor Behavior Assessment Scale (MBA)
Fig. 5
Fig. 5
Forest plot for Rett Syndrome Behavior Questionnaire (RSBQ)
Fig. 6
Fig. 6
Forest plot for Adverse events
Fig. 7
Fig. 7
Forest plot for Diarrhea
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Fig. 8
Forest plot for Vomiting
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Fig. 9
Forest plot for Pyrexia
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Fig. 10
Forest plot for Seizures
Fig. 11
Fig. 11
Forest plot for Serious adverse events
See this image and copyright information in PMC

References

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