{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,8]],"date-time":"2026-07-08T22:53:20Z","timestamp":1783551200315,"version":"3.55.0"},"reference-count":28,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2019,5,12]],"date-time":"2019-05-12T00:00:00Z","timestamp":1557619200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pediatric Transplantation"],"published-print":{"date-parts":[[2019,8]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Niemann\u2010Pick disease type C (NPC) is a rare autosomal recessive inherited disease characterized by lysosomal accumulation of free cholesterol in macrophages within multiple organs. Infantile\u2010onset NPC often presents with jaundice and hepatosplenomegaly from birth, but these symptoms usually improve during early childhood, and it rarely progresses to liver failure. We report three cases from different hospitals in Japan; the patients developed neonatal\u2010onset NPC, and liver transplantation (LT) was performed as a life\u2010saving procedure. LT was performed at 19\u00a0days, 59\u00a0days, and 4\u00a0months of age, respectively. The last patient was diagnosed with NPC before LT, while the first two patients were diagnosed with neonatal hemochromatosis at LT. In these two patients, the diagnosis of NPC was made more than a year after LT. Even though oral administration of miglustat was started soon after the diagnosis of NPC, all patients showed neurological regression and required artificial respiratory support. All patients survived more than one year after LT; however, one patient died due to tracheal hemorrhage at 4.5\u00a0years of age, and another one patient was suspected as recurrence of NPC in liver graft. In conclusion, while LT may be a temporary life\u2010saving measure in patients with neonatal\u2010onset NPC leading to liver failure, the outcome is poor especially due to neurological symptoms. A preoperative diagnosis is thus critical.<\/jats:p>","DOI":"10.1111\/petr.13462","type":"journal-article","created":{"date-parts":[[2019,5,13]],"date-time":"2019-05-13T05:59:22Z","timestamp":1557727162000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":19,"title":["Pediatric liver transplantation for neonatal\u2010onset Niemann\u2010Pick disease type C: Japanese multicenter experience"],"prefix":"10.1111","volume":"23","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-0111-028X","authenticated-orcid":false,"given":"Naoya","family":"Yamada","sequence":"first","affiliation":[{"name":"Department of Transplant Surgery Jichi Medical University Tochigi Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1973-4376","authenticated-orcid":false,"given":"Ayano","family":"Inui","sequence":"additional","affiliation":[{"name":"Department of Pediatric Hepatology and Gastroenterology 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