Efficacy and safety of selpercatinib in treating RET-altered MTC: A single-arm meta-analysis

Selpercatinib is effective in the treatment of RET-altered medullary thyroid carcinoma (MTC). This study aimed to evaluate the efficacy and safety of selpercatinib in the treatment of patients with RET-altered MTC. PubMed, Embase, the Cochrane Library, and ClinicalTrials.gov were searched from their inception to April 5, 2024. Outcomes included complete response (CR), partial response (PR), stable disease (SD), objective response rate (ORR), disease control rate (DCR), and adverse events (AEs). We carried out a meta-analysis of these studies and exploratory subgroup analyses. The effect sizes for all pooled results were presented as 95% confidence intervals with upper and lower limits. The pooled CR, PR, and SD rates for all patients were 10%, 59%, and 26%, respectively. The pooled ORR in all patients was 70%, while the pooled ORR in pre-treated and non-pre-treated groups were 67% and 70%, respectively. The pooled DCR in all patients was 95%, while the pooled DCR in pre-treated and non-pre-treated groups were 96% and 95%, respectively. The most common AEs associated with selpercatinib were hypertension, alanine aminotransferase (ALT) increased and aspartate aminotransferase (AST) increased. Selpercatinib offers significant benefits to patients with RET-altered MTC with assessable CR, PR, SD, ORR, and grade 3–4 AEs; however, treatment-related AEs should be considered. •This Meta-analysis assessed the efficacy and safety of selpercatinib in RET-alter MTC.•Treatment for RET-alter MTC patients with selpercatinib resulted in a pooled CR of 10%, PR of 59%, SD of 26%, ORR of 70% and DCR of 95%.•Treatment with selpercatinib show frequent adverse events, but generally tolerable toxicity profiles.•Our data establish selpercatinib as a promising treatment option for RET-alter MTC.