Outcomes of liver transplantation for Alagille syndrome after Kasai portoenterostomy: Alagille Syndrome with agenesis of extrahepatic bile ducts at porta hepatis.


Journal

Journal of pediatric surgery
ISSN: 1531-5037
Titre abrégé: J Pediatr Surg
Pays: United States
ID NLM: 0052631

Informations de publication

Date de publication:
Nov 2019
Historique:
received: 02 01 2019
revised: 23 03 2019
accepted: 19 04 2019
pubmed: 21 5 2019
medline: 20 2 2020
entrez: 21 5 2019
Statut: ppublish

Résumé

Alagille syndrome (ALGS) is an autosomal dominant disorder, characterized by a paucity of intrahepatic bile ducts, resulting in significant cholestasis, and peculiar extrahepatic features. Some ALGS patients show a considerable overlap with biliary atresia (BA), and they can undergo Kasai procedure. The purpose of this study is to show the manifestations of BA overlapped ALGS cases in our institution, and to compare the outcomes of ALGS patients following liver transplantation (LT) between those who previously underwent Kasai surgery (ALGS-Kasai group) and those who did not (ALGS-non-Kasai group). Medical records of ALGS patients who underwent LT in Kyoto University Hospital, Japan from January 1992 to March 2018 were analyzed. ALGS diagnosis was determined according to physical, radiologic, and histopathological findings. Thirty-one patients were ascertained (ALGS-Kasai: 4 males and 5 females vs. ALGS-non-Kasai: 14 males and 8 females, p = 0.43). Of 31 ALGS patients, 96.8% of children had pulmonary artery stenosis, 54.8% showed facial features, 29% revealed skeletal anomalies and 9.7% demonstrated ocular anomalies. The age at LT was significantly younger in ALGS-Kasai than ALGS-non-Kasai group (1.47 [interquartile range (IQR), 0.75-1.92] vs. 5.1 [IQR, 1.4-9.29] years; p = 0.038). Overall patient survival did not significantly differ between ALGS-Kasai (88.9%) and ALGS-non-Kasai patients (86.4%) (p = 0.84). Furthermore, the 1-year, 5-year, and 10-year patient survival rates for ALGS-Kasai group were 100%, 88.9%, and 88.9%, respectively, whereas those for ALGS-non-Kasai group were 90.9%, 90.9%, and 86.4%, respectively, with p-values of 0.36, 0.90, and 0.84, respectively. BA overlapped ALGS cases had neonatal progressive cholestasis which prompted Kasai procedure, and early liver dysfunction after Kasai led to performing LT. The ALGS-Kasai patients undergo LT at earlier ages than the ALGS-non-Kasai patients, however, overall patients' survival rates are similar between groups. Overall ALGS patients' survival rate after LT is considered high. Level III; Case-control study or Retrospective comparative study.

Sections du résumé

BACKGROUND BACKGROUND
Alagille syndrome (ALGS) is an autosomal dominant disorder, characterized by a paucity of intrahepatic bile ducts, resulting in significant cholestasis, and peculiar extrahepatic features. Some ALGS patients show a considerable overlap with biliary atresia (BA), and they can undergo Kasai procedure. The purpose of this study is to show the manifestations of BA overlapped ALGS cases in our institution, and to compare the outcomes of ALGS patients following liver transplantation (LT) between those who previously underwent Kasai surgery (ALGS-Kasai group) and those who did not (ALGS-non-Kasai group).
METHODS METHODS
Medical records of ALGS patients who underwent LT in Kyoto University Hospital, Japan from January 1992 to March 2018 were analyzed. ALGS diagnosis was determined according to physical, radiologic, and histopathological findings.
RESULTS RESULTS
Thirty-one patients were ascertained (ALGS-Kasai: 4 males and 5 females vs. ALGS-non-Kasai: 14 males and 8 females, p = 0.43). Of 31 ALGS patients, 96.8% of children had pulmonary artery stenosis, 54.8% showed facial features, 29% revealed skeletal anomalies and 9.7% demonstrated ocular anomalies. The age at LT was significantly younger in ALGS-Kasai than ALGS-non-Kasai group (1.47 [interquartile range (IQR), 0.75-1.92] vs. 5.1 [IQR, 1.4-9.29] years; p = 0.038). Overall patient survival did not significantly differ between ALGS-Kasai (88.9%) and ALGS-non-Kasai patients (86.4%) (p = 0.84). Furthermore, the 1-year, 5-year, and 10-year patient survival rates for ALGS-Kasai group were 100%, 88.9%, and 88.9%, respectively, whereas those for ALGS-non-Kasai group were 90.9%, 90.9%, and 86.4%, respectively, with p-values of 0.36, 0.90, and 0.84, respectively.
CONCLUSIONS CONCLUSIONS
BA overlapped ALGS cases had neonatal progressive cholestasis which prompted Kasai procedure, and early liver dysfunction after Kasai led to performing LT. The ALGS-Kasai patients undergo LT at earlier ages than the ALGS-non-Kasai patients, however, overall patients' survival rates are similar between groups. Overall ALGS patients' survival rate after LT is considered high.
LEVELS OF EVIDENCE METHODS
Level III; Case-control study or Retrospective comparative study.

Identifiants

pubmed: 31104835
pii: S0022-3468(19)30323-9
doi: 10.1016/j.jpedsurg.2019.04.022
pii:
doi:

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

2387-2391

Informations de copyright

Copyright © 2019 Elsevier Inc. All rights reserved.

Auteurs

Department of Surgery (HBP and Transplantation/Pediatric Surgery), Kyoto University Hospital, Kyoto 606-8507, Japan; Pediatric Surgery Division, Department of Surgery, Faculty of Medicine, Public Health and Nursing, Universitas Gadjah Mada/Dr. Sardjito Hospital, Yogyakarta 55281, Indonesia.

Masakatshu Kaneshiro (M)

Department of Surgery (HBP and Transplantation/Pediatric Surgery), Kyoto University Hospital, Kyoto 606-8507, Japan.

Tatsuya Okamoto (T)

Department of Surgery (HBP and Transplantation/Pediatric Surgery), Kyoto University Hospital, Kyoto 606-8507, Japan. Electronic address: okamotot@kuhp.kyoto-u.ac.jp.

Mari Sonoda (M)

Department of Surgery (HBP and Transplantation/Pediatric Surgery), Kyoto University Hospital, Kyoto 606-8507, Japan.

Eri Ogawa (E)

Department of Surgery (HBP and Transplantation/Pediatric Surgery), Kyoto University Hospital, Kyoto 606-8507, Japan.

Hideaki Okajima (H)

Department of Surgery (HBP and Transplantation/Pediatric Surgery), Kyoto University Hospital, Kyoto 606-8507, Japan; Department of Pediatric Surgery, Kanazawa Medical University Hospital, Kanazawa 920-0265, Japan.

Shiji Uemoto (S)

Department of Surgery (HBP and Transplantation/Pediatric Surgery), Kyoto University Hospital, Kyoto 606-8507, Japan.

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