Our previous study10,11 showed that the GOLP regimen achieved the best ORR (80%) in the treatment of advanced iCCA, while lenvatinib plus anti-PD1 antibody or GEMOX showed ORRs of approximately 30% in the treatment of advanced iCCA (Fig. 1A). The gene discussed is PDCD1; the disease is infantile convulsions and choreoathetosis.