Outcomes of 38 patients with PFIC3: Impact of genotype and of response to ursodeoxycholic acid therapy

•71% of patients had cirrhosis at a median age of 8.1 years of whom 66% received a liver transplant at a median age of 8.5 years.•Patients with a severe genotype (no missense variation) did not respond to UDCA treatment.•87% of patients with at least one missense variation responded partially or fully to UDCA treatment.•Biliary phospholipid level over 6.9% of total biliary lipids predicted response to UDCA therapy.•Severity of genotypes and response to UDCA treatment predicted native liver survival.