Thanh Liem Nguyen, Hoang-Phuong Nguyen, Thi Hang Bui, Thi Kieu Trang Phan, Duy Minh Ngo, Thi Thu Hien Ha, Thanh Tri Tran, Nguyen Anh Thuan Luu, Tuan Kiet Phan, Phi Duy Ho
{"title":"同种异体脐带间充质干细胞输注治疗开赛手术后胆道闭锁肝硬化的疗效。","authors":"Thanh Liem Nguyen, Hoang-Phuong Nguyen, Thi Hang Bui, Thi Kieu Trang Phan, Duy Minh Ngo, Thi Thu Hien Ha, Thanh Tri Tran, Nguyen Anh Thuan Luu, Tuan Kiet Phan, Phi Duy Ho","doi":"10.1016/j.jpedsurg.2025.162624","DOIUrl":null,"url":null,"abstract":"<p><strong>Background and aim: </strong>Although Kasai surgery has saved many patients with biliary atresia, the long-term survival rate remains low. Recently, cell therapy has been explored as a potential strategy to improve post-surgical survival. This study aims to evaluate the safety and outcomes of allogeneic umbilical cord mesenchymal stem cell (UC-MSC) infusion in management of liver cirrhosis due to Biliary Atresia after Kasai operation.</p><p><strong>Methods: </strong>A matched case-control study was conducted from April 2019 to May 2023, including 32 patients with the cirrhosis after Kasai portoenterostomy for biliary atresia. Sixteen patients in the cell therapy (CT) group, who received a single hepatic-artery infusion of UC-MSC plus standard care after a modified Kasai procedure at a median age of 9.0 ± 2.1 months (median 9.1, range 6.1-13). Sixteen matched controls underwent the same Kasai technique but received standard care only. Matching required the age at Kasai to differ by no more than 14 days (81.5 days [58-103] in the therapy group vs 76.0 days [52-112] in controls; p = 0.77) and that stool-color scores on the Stool Color Card (Taiwan Infant Stool Color Card) be identical. At enrollment the groups were comparable for age (9.0 ± 2.1 vs 9.4 ± 5.2 months; p = 0.52) and PELD score (2.31 ± 4.16 vs 2.69 ± 4.09; p = 0.80).</p><p><strong>Results: </strong>No severe adverse events related to cell therapy were observed. Over 12 months, the CT group showed significantly better biochemical profiles than controls: serum albumin 3.9 ± 0.3 vs. 3.5 ± 0.4 g/dL (p = 0.02), total bilirubin 9 ± 3 vs. 30 ± 10 μmol/dL (p = 0.04) and greater improvement in alkaline phosphatase (p = 0.03). Reductions in AST, ALT and GGT also favored the UC-MSC group but did not reach statistical significance. The CT group demonstrated a significant improvement in disease severity, with PELD scores decreased to -3.56 in the UC-MSC group and 1.64 in the control group (p < 0.01).</p><p><strong>Conclusion: </strong>Allogeneic umbilical cord mesenchymal stem cell administration was safe and may help preserve or improve liver function in patients with liver cirrhosis due to biliary atresia.</p><p><strong>Clinicaltrials: </strong></p><p><strong>Gov identifier: </strong>NCT04522869 on June 6th, 2024.</p>","PeriodicalId":16733,"journal":{"name":"Journal of pediatric surgery","volume":" ","pages":"162624"},"PeriodicalIF":2.5000,"publicationDate":"2025-09-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Outcomes of allogeneic umbilical cord mesenchymal stem cell infusion for liver cirrhosis due to biliary atresia after Kasai operation.\",\"authors\":\"Thanh Liem Nguyen, Hoang-Phuong Nguyen, Thi Hang Bui, Thi Kieu Trang Phan, Duy Minh Ngo, Thi Thu Hien Ha, Thanh Tri Tran, Nguyen Anh Thuan Luu, Tuan Kiet Phan, Phi Duy Ho\",\"doi\":\"10.1016/j.jpedsurg.2025.162624\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Background and aim: </strong>Although Kasai surgery has saved many patients with biliary atresia, the long-term survival rate remains low. Recently, cell therapy has been explored as a potential strategy to improve post-surgical survival. This study aims to evaluate the safety and outcomes of allogeneic umbilical cord mesenchymal stem cell (UC-MSC) infusion in management of liver cirrhosis due to Biliary Atresia after Kasai operation.</p><p><strong>Methods: </strong>A matched case-control study was conducted from April 2019 to May 2023, including 32 patients with the cirrhosis after Kasai portoenterostomy for biliary atresia. Sixteen patients in the cell therapy (CT) group, who received a single hepatic-artery infusion of UC-MSC plus standard care after a modified Kasai procedure at a median age of 9.0 ± 2.1 months (median 9.1, range 6.1-13). Sixteen matched controls underwent the same Kasai technique but received standard care only. Matching required the age at Kasai to differ by no more than 14 days (81.5 days [58-103] in the therapy group vs 76.0 days [52-112] in controls; p = 0.77) and that stool-color scores on the Stool Color Card (Taiwan Infant Stool Color Card) be identical. At enrollment the groups were comparable for age (9.0 ± 2.1 vs 9.4 ± 5.2 months; p = 0.52) and PELD score (2.31 ± 4.16 vs 2.69 ± 4.09; p = 0.80).</p><p><strong>Results: </strong>No severe adverse events related to cell therapy were observed. Over 12 months, the CT group showed significantly better biochemical profiles than controls: serum albumin 3.9 ± 0.3 vs. 3.5 ± 0.4 g/dL (p = 0.02), total bilirubin 9 ± 3 vs. 30 ± 10 μmol/dL (p = 0.04) and greater improvement in alkaline phosphatase (p = 0.03). Reductions in AST, ALT and GGT also favored the UC-MSC group but did not reach statistical significance. The CT group demonstrated a significant improvement in disease severity, with PELD scores decreased to -3.56 in the UC-MSC group and 1.64 in the control group (p < 0.01).</p><p><strong>Conclusion: </strong>Allogeneic umbilical cord mesenchymal stem cell administration was safe and may help preserve or improve liver function in patients with liver cirrhosis due to biliary atresia.</p><p><strong>Clinicaltrials: </strong></p><p><strong>Gov identifier: </strong>NCT04522869 on June 6th, 2024.</p>\",\"PeriodicalId\":16733,\"journal\":{\"name\":\"Journal of pediatric surgery\",\"volume\":\" \",\"pages\":\"162624\"},\"PeriodicalIF\":2.5000,\"publicationDate\":\"2025-09-02\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Journal of pediatric surgery\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1016/j.jpedsurg.2025.162624\",\"RegionNum\":2,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"PEDIATRICS\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of pediatric surgery","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1016/j.jpedsurg.2025.162624","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"PEDIATRICS","Score":null,"Total":0}
Outcomes of allogeneic umbilical cord mesenchymal stem cell infusion for liver cirrhosis due to biliary atresia after Kasai operation.
Background and aim: Although Kasai surgery has saved many patients with biliary atresia, the long-term survival rate remains low. Recently, cell therapy has been explored as a potential strategy to improve post-surgical survival. This study aims to evaluate the safety and outcomes of allogeneic umbilical cord mesenchymal stem cell (UC-MSC) infusion in management of liver cirrhosis due to Biliary Atresia after Kasai operation.
Methods: A matched case-control study was conducted from April 2019 to May 2023, including 32 patients with the cirrhosis after Kasai portoenterostomy for biliary atresia. Sixteen patients in the cell therapy (CT) group, who received a single hepatic-artery infusion of UC-MSC plus standard care after a modified Kasai procedure at a median age of 9.0 ± 2.1 months (median 9.1, range 6.1-13). Sixteen matched controls underwent the same Kasai technique but received standard care only. Matching required the age at Kasai to differ by no more than 14 days (81.5 days [58-103] in the therapy group vs 76.0 days [52-112] in controls; p = 0.77) and that stool-color scores on the Stool Color Card (Taiwan Infant Stool Color Card) be identical. At enrollment the groups were comparable for age (9.0 ± 2.1 vs 9.4 ± 5.2 months; p = 0.52) and PELD score (2.31 ± 4.16 vs 2.69 ± 4.09; p = 0.80).
Results: No severe adverse events related to cell therapy were observed. Over 12 months, the CT group showed significantly better biochemical profiles than controls: serum albumin 3.9 ± 0.3 vs. 3.5 ± 0.4 g/dL (p = 0.02), total bilirubin 9 ± 3 vs. 30 ± 10 μmol/dL (p = 0.04) and greater improvement in alkaline phosphatase (p = 0.03). Reductions in AST, ALT and GGT also favored the UC-MSC group but did not reach statistical significance. The CT group demonstrated a significant improvement in disease severity, with PELD scores decreased to -3.56 in the UC-MSC group and 1.64 in the control group (p < 0.01).
Conclusion: Allogeneic umbilical cord mesenchymal stem cell administration was safe and may help preserve or improve liver function in patients with liver cirrhosis due to biliary atresia.
期刊介绍：
The journal presents original contributions as well as a complete international abstracts section and other special departments to provide the most current source of information and references in pediatric surgery. The journal is based on the need to improve the surgical care of infants and children, not only through advances in physiology, pathology and surgical techniques, but also by attention to the unique emotional and physical needs of the young patient.
求助内容：
应助结果提醒方式：
京公网安备 11010802042870号
