Abstract Details

Title Efficacy of Pharmacological Interventions for Transthyretin Familial Amyloid Polyneuropathy (TTR-FAP): A Systematic Review and Network Meta-Analysis

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) S9 - Autonomic Disorders (12:03 PM-12:15 PM)

Poster/Presentation
Number 005

Objective To assess and compare the efficacy of disease-modifying pharmacological agents in patients with transthyretin familial amyloid polyneuropathy.

Background Transthyretin Familial Amyloid Polyneuropathy (TTR-FAP) is a life-threatening, autosomal dominant disorder causing sensorimotor and autonomic neuropathy due to amyloid fibril deposition. This network meta-analysis aims to identify the most effective pharmacological treatments for managing TTR-FAP.

Design/Methods A systematic search in PubMed/MEDLINE, Cochrane, EMBASE, Scopus, and ClinicalTrials.gov identified randomized controlled trials (RCTs) on TTR-FAP interventions up to May 2024. The primary outcomes were the Norfolk Quality of Life-Diabetic Neuropathy (QOL-DN) questionnaire and the Modified Neuropathy Impairment Score +7 (mNIS+7). Pairwise meta-analysis and network meta-analysis were performed using the frequentist approach to obtain both direct and indirect comparisons of each measure in terms of standardized mean difference (SMD).

Results The QOL-DN questionnaire was assessed in four studies with a pooled population of 644 and four pairwise comparisons, while the mNIS+7 was assessed in three studies with a pooled population of 455 and three pairwise comparisons. For the QOL-DN questionnaire, Patisiran showed the greatest effect, with an SMD of -1.04 (95% CI: -1.39 to -0.70), followed by Vutrisiran (-0.85, 95% CI: -1.15 to -0.56) and Inotersen (-0.55, 95% CI: -0.90 to -0.20), all statistically significant. Tafamidis had an SMD of -0.24 (95% CI: -0.59 to 0.12), which was not statistically significant. For mNIS+7, Patisiran again demonstrated the strongest impact, with an SMD of -1.71 (95% CI: -2.07 to -1.34). Inotersen showed an SMD of -1.02 (95% CI: -1.39 to -0.65), while Diflunisal had an SMD of -0.42 (95% CI: -0.77 to -0.07), all statistically significant.

Conclusions Patisiran and Inotersen demonstrated superior efficacy with significant results, while Vutrisiran and Diflunisal also showed notable outcomes. In contrast, Tafamidis did not yield significant results. Further research is needed to confirm these findings.

Authors/Disclosures
Vinay Suresh, MBBS PRESENTER	Dr. Suresh has nothing to disclose.
Muneeb Ahmad Muneer (Allama Iqbal Medical College)	Mr. Muneer has nothing to disclose.
Poorvikha S	Poorvikha S has nothing to disclose.
Adeeb Ali Mohammad, MBBS	Dr. Mohammad has nothing to disclose.
Archit Garg	Mr. Garg has nothing to disclose.
Arkansh Sharma	Mr. Sharma has nothing to disclose.
Vibhor Agrawal, MBBS	Dr. Agrawal has nothing to disclose.
Varsha Srinivasan	Ms. Srinivasan has nothing to disclose.
Ishita Lanjewar, MBBS	Dr. Lanjewar has nothing to disclose.
Kshitij Sonawale, MBBS	Dr. Sonawale has nothing to disclose.

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