Long-term liver transplant outcomes are good in patients with PFIC1

In a study including patients with progressive familial intrahepatic cholestasis type 1 (PFIC1) who underwent a liver transplant (LT), the 10-year outcomes were positive, with excellent patient and graft survival.

Pre- and post-LT data for 7 patients, 6 of whom had confirmed or suspected identical genetics, were analyzed.

At a mean follow-up of 10.9 years, all patients were alive and graft survival was 100%.

Diarrhea persisted in 3 patients and developed in 4 patients after LT. This contributed to continuous growth failure, with mean z-scores -2.63 (weight) and -2.98 (height) at follow-up. Although allograft steatosis was common in it did not lead to significant inflammation, ballooning, or fibrosis.

Reference
Henkel SAF, Salgado CM, Reyes-Mugica M, et al. Long-term liver transplant outcomes for progressive familial intrahepatic cholestasis type 1: The Pittsburgh experience. Pediatr Transplant. 2021;25(8):e14108. doi: 10.1111/petr.14108. Epub 2021 Aug 2. PMID: 34339082.