Volume 13 Issue 1
Jan.  2022
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Tan Yule, Zhu Zhijun, Sun Liying, et al. Pediatric liver transplantation for Alagille syndrome: a single-center experience[J]. ORGAN TRANSPLANTATION, 2022, 13(1): 61-66. doi: 10.3969/j.issn.1674-7445.2022.01.010
Citation: Tan Yule, Zhu Zhijun, Sun Liying, et al. Pediatric liver transplantation for Alagille syndrome: a single-center experience[J]. ORGAN TRANSPLANTATION, 2022, 13(1): 61-66. doi: 10.3969/j.issn.1674-7445.2022.01.010
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Pediatric liver transplantation for Alagille syndrome: a single-center experience

doi: 10.3969/j.issn.1674-7445.2022.01.010

  • Department of General Surgery, Beijing Friendship Hospital, Capital Medical University, Clinical Center for Pediatric Liver Transplantation, Capital Medical University, National Clinical Research Center for Digestive Diseases, Beijing 100050, China

More Information
  • Corresponding author: Zhu Zhijun, E-mail: zhu-zhijun@outlook.com
  • Received Date: 2021-08-22
    Available Online: 2022-01-12
  • Publish Date: 2022-01-15
    Abstract
  •   Objective  To evaluate the clinical efficacy of liver transplantation in children with Alagille syndrome (ALGS).  Methods  Clinical data of 12 children with ALGS were collected and retrospectively analyzed. Clinical characteristics of children with ALGS, pathological characteristics of liver tissues, characteristics of liver transplantation, postoperative complications and follow-up of children with ALGS were analyzed.  Results  JAG1 gene mutation and typical facial features was present in all 12 children. Jaundice was the most common initial symptom, which occurred at 7 (3, 40) d after birth. Upon liver transplantation, the Z scores of height and body weight were calculated as -2.14 (-3.11, -1.83) and -2.32 (-3.12, -1.12). Five children developed severe growth retardation and 4 children with severe malnutrition. Eight of 12 children were diagnosed with cardiovascular abnormalities. Pathological examination showed that the lobular structure of the diseased livers of 4 children was basically maintained, and 8 cases of nodular liver cirrhosis in different sizes including 1 case of single early moderately-differentiated hepatocellular carcinoma. Three children were misdiagnosed with biliary atresia and underwent Kasai portoenterostomy. Eight children underwent living donor liver transplantation, three children underwent cadaveric donor liver transplantation (two cases of split liver transplantation and one case of cadaveric total liver transplantation), and one child underwent domino liver transplantation (donor liver was derived from a patient with maple syrup urine disease). during the follow-up of 30.0(24.5, 41.7) months, the survival rates of the children and liver grafts were both 100%. During postoperative follow-up, the Z scores of height and body weight were calculated as -1.24 (-2.11, 0.60) and -0.83 (-1.65, -0.43), indicating that the growth and development of the children were significantly improved after operation.  Conclusions  Liver transplantation is an efficacious treatment for children with ALGS complicated with decompensated cirrhosis, severe itching and poor quality of life. For children with ALGS complicated with cardiovascular abnormalities, explicit preoperative evaluation should be delivered, and consultation with pediatric cardiologists should be performed if necessary.

     

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    • References(31)
  • [1]
    KAMATH BM, YIN W, MILLER H, et al. Outcomes of liver transplantation for patients with Alagille syndrome: the studies of pediatric liver transplantation experience[J]. Liver Transpl, 2012, 18(8): 940-948. DOI: 10.1002/lt.23437.
    [2]
    DIAZ-FRIAS J, KONDAMUDI NP. Alagille syndrome[M]. Treasure Island (FL): StatPearls Publishing, 2021.
    [3]
    AYOUB MD, KAMATH BM. Alagille syndrome: diagnostic challenges and advances in management[J]. Diagnostics (Basel), 2020, 10(11): 907. DOI: 10.3390/diagnostics10110907.
    [4]
    KAMATH BM, BAKER A, HOUWEN R, et al. Systematic review: the epidemiology, natural history, and burden of Alagille syndrome[J]. J Pediatr Gastroenterol Nutr, 2018, 67(2): 148-156. DOI: 10.1097/MPG.0000000000001958.
    [5]
    LEONARD LD, CHAO G, BAKER A, et al. Clinical utility gene card for: Alagille syndrome (ALGS)[J]. Eur J Hum Genet, 2014, 22(3). DOI: 10.1038/ejhg.2013.140.
    [6]
    MITCHELL E, GILBERT M, LOOMES KM. Alagille syndrome[J]. Clin Liver Dis, 2018, 22(4): 625-641. DOI: 10.1016/j.cld.2018.06.001.
    [7]
    EMERICK KM, RAND EB, GOLDMUNTZ E, et al. Features of Alagille syndrome in 92 patients: frequency and relation to prognosis[J]. Hepatology, 1999, 29(3): 822-829. DOI: 10.1002/hep.510290331.
    [8]
    KAMATH BM, SCHWARZ KB, HADZIĆ N. Alagille syndrome and liver transplantation[J]. J Pediatr Gastroenterol Nutr, 2010, 50(1): 11-15. DOI: 10.1097/MPG.0b013e3181c1601f.
    [9]
    KAMATH BM, YE W, GOODRICH NP, et al. Outcomes of childhood cholestasis in Alagille syndrome: results of a multicenter observational study[J]. Hepatol Commun, 2020, 4(3): 387-398. DOI: 10.1002/hep4.1468.
    [10]
    AKDUR A, KIRNAP M, AYVAZOGLU SOY EH, et al. Unusual indications for a liver transplant: a single-center experience[J]. Exp Clin Transplant, 2017, 15(Suppl 1): 128-132. DOI: 10.6002/ect.mesot2016.P11.
    [11]
    GILBERT MA, BAUER RC, RAJAGOPALAN R, et al. Alagille syndrome mutation update: comprehensive overview of JAG1 and NOTCH2 mutation frequencies and insight into missense variant classification[J]. Hum Mutat, 2019, 40(12): 2197-2220. DOI: 10.1002/humu.23879.
    [12]
    马艳立, 宋元宗. Alagille综合征诊断治疗进展[J]. 中国当代儿科杂志, 2014, 16(11): 1188-1192. DOI: 10.7499/j.issn.1008-8830.2014.11.025.

    MA YL, SONG YZ. Advances in the diagnosis and treatment of Alagille syndrome[J]. Chin J Contemp Pediatr, 2014, 16(11): 1188-1192. DOI: 10.7499/j.issn.1008-8830.2014.11.025.
    [13]
    GUNADI, KANESHIRO M, OKAMOTO T, et al. Outcomes of liver transplantation for Alagille syndrome after Kasai portoenterostomy: Alagille syndrome with agenesis of extrahepatic bile ducts at porta hepatis[J]. J Pediatr Surg, 2019, 54(11): 2387-2391. DOI: 10.1016/j.jpedsurg.2019.04.022.
    [14]
    YANG WH, ZHANG L, XUE FS, et al. Pediatric liver transplantation for Alagille syndrome: anesthetic evaluation and perioperative management[J]. Ann Transplant, 2020, 25: e924282. DOI: 10.12659/AOT.924282.
    [15]
    SANADA Y, NAYA I, KATANO T, et al. Visceral artery anomalies in patients with Alagille syndrome[J]. Pediatr Transplant, 2019, 23(2): e13352. DOI: 10.1111/petr.13352.
    [16]
    FUJISHIRO J, SUZUKI K, WATANABE M, et al. Outcomes of Alagille syndrome following the Kasai operation: a systematic review and Meta-analysis[J]. Pediatr Surg Int, 2018, 34(10): 1073-1077. DOI: 10.1007/s00383-018-4316-3.
    [17]
    MANDATO C, ZOLLO G, VAJRO P. Cholestatic jaundice in infancy: struggling with many old and new phenotypes[J]. Ital J Pediatr, 2019, 45(1): 83. DOI: 10.1186/s13052-019-0679-x.
    [18]
    HAN S, JEON TY, HWANG SM, et al. Imaging findings of Alagille syndrome in young infants: differentiation from biliary atresia[J]. Br J Radiol, 2017, 90(1077): 20170406. DOI: 10.1259/bjr.20170406.
    [19]
    TAKEDA M, SAKAMOTO S, UCHIDA H, et al. The morphological and histopathological assessment of Alagille syndrome with extrahepatic bile duct obstruction: the importance of the differential diagnosis with subgroup "o" biliary atresia[J]. Pediatr Surg Int, 2021, 37(9): 1167-1174. DOI: 10.1007/s00383-021-04932-z.
    [20]
    DĚDIČ T, JIRSA M, KEIL R, et al. Alagille syndrome mimicking biliary atresia in early infancy[J]. PLoS One, 2015, 10(11): e0143939. DOI: 10.1371/journal.pone.0143939.
    [21]
    JANOWSKI K, OBRYCKI Ł, LITWIN M, et al. Cardiovascular risk assessment in children with chronic cholestatic liver diseases[J]. J Pediatr Gastroenterol Nutr, 2020, 71(5): 647-654. DOI: 10.1097/MPG.0000000000002874.
    [22]
    DI PINTO D, ADRAGNA M. Renal manifestations in children with Alagille syndrome[J]. Arch Argent Pediatr, 2018, 116(2): 149-153. DOI: 10.5546/aap.2018.eng.149.
    [23]
    KHANNA R, VERMA SK. Pediatric hepatocellular carcinoma[J]. World J Gastroenterol, 2018, 24(35): 3980-3999. DOI: 10.3748/wjg.v24.i35.3980.
    [24]
    胡会, 杨永臣, 张泓, 等. Alagille综合征合并肝细胞癌1例报告并文献复习[J]. 临床儿科杂志, 2017, 35(4): 253-255. DOI: 10.3969/j.issn.1000-3606.2017.04.003.

    HU H, YANG YC, ZHANG H, et al. Alagille syndrome in a child combined with hepatocellular carcinoma: a case report and literature review[J]. J Clin Pediatr, 2017, 35(4): 253-255. DOI: 10.3969/j.issn.1000-3606.2017.04.003.
    [25]
    SCHINDLER EA, GILBERT MA, PICCOLI DA, et al. Alagille syndrome and risk for hepatocellular carcinoma: need for increased surveillance in adults with mild liver phenotypes[J]. Am J Med Genet A, 2021, 185(3): 719-731. DOI: 10.1002/ajmg.a.62028.
    [26]
    VÁZQUEZ RODRÍGUEZ JA, MOLINA VILLALBA C, ESTÉVEZ ESCOBAR M. Alagille syndrome and hepatocellular carcinoma in a non-cirrhotic adult[J]. Med Clin (Barc), 2021, DOI: 10.1016/j.medcli.2021.06.010[Epubaheadofprint].
    [27]
    SCHOEN K, RIBEIRO CMF, GONÇALVES MC, et al. Hepatocellular carcinoma in an adult with Alagille syndrome: case report and literature review[J]. Radiol Case Rep, 2020, 16(1): 90-93. DOI: 10.1016/j.radcr.2020.09.027.
    [28]
    GALVEZ D, RUCH B, SHARMA A, et al. Recurrent hepatocellular carcinoma in an adult with Alagille syndrome treated with liver resection followed by liver transplantation: a rare case report[J]. Transplant Direct, 2020, 6(10): e606. DOI: 10.1097/TXD.0000000000001055.
    [29]
    AKAGI K, TANAKA T, BABA S. Successful living donor liver transplantation after stent implantation in a patient with Alagille syndrome and severe bilateral pulmonary artery stenosis[J]. Cardiol Young, 2018, 28(12): 1465-1467. DOI: 10.1017/S1047951118001476.
    [30]
    LUONG R, FEINSTEIN JA, MA M, et al. Outcomes in patients with Alagille syndrome and complex pulmonary artery disease[J]. J Pediatr, 2021, 229: 86-94. DOI: 10.1016/j.jpeds.2020.09.053.
    [31]
    MCGAHAN RK, TANG JE, IYER MH, et al. Combined liver kidney transplant in adult patient with Alagille syndrome and pulmonary hypertension[J]. Semin Cardiothorac Vasc Anesth, 2021, 25(3): 191-195. DOI: 10.1177/10892532211008742.
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