title: Pilot study of umbilical cord-derived mesenchymal stem cell transfusion in patients with primary biliary cirrhosis.
abstract: OBJECTIVE
  Ursodeoxycholic acid (UDCA) treatment is an effective medical therapy for patients with primary biliary cirrhosis (PBC); however, 40% of PBC patients show an incomplete response to the UDCA therapy. This study aimed to investigate the safety and efficacy of umbilical cord-derived mesenchymal stem cell (UC-MSC) transfusion in PBC patients with an incomplete response to UDCA.
  
  
  METHODS
  We conducted a single-arm trial that included seven PBC patients with a suboptimal response to UDCA treatment. UC-MSCs were first cultured, and then 0.5 × 10(6) cells/kg body weights were infused through a peripheral vein. UC-MSCs were given three times at 4-week intervals, and patients were followed up for 48 weeks. Primary outcomes were to evaluate the safety and feasibility of UC-MSC treatment, and secondary outcomes were to evaluate liver functions and patient's quality of life.
  
  
  RESULTS
  No obvious side-effects were found in the patients treated with UC-MSCs. Symptoms such as fatigue and pruritus were obviously alleviated in most patients after UC-MSC treatment. There was a significant decrease in serum alkaline phosphatase and γ-glutamyltransferase levels at the end of the follow-up period as compared with baseline. No significant changes were observed in serum alanine aminotransferase, aspartate aminotransferase, total bilirubin, albumin, prothrombin time activity, international normalized ratio, or immunoglobulin M levels. The Mayo risk score, a prognostic index, was also stable during the treatment and follow-up period.
  
  
  CONCLUSIONS
  UC-MSC transfusion is feasible and well tolerated in patients with PBC who respond only partially to UDCA treatment, thus representing a novel therapeutic approach for patients in this subgroup. A larger, randomized controlled cohort study is warranted to confirm the clinical efficacy of UC-MSC transfusion.
authors.affiliation: Research Center for Biological Therapy, The Institute of Translational Hepatology, Beijing 302 Hospital, Beijing, China.
authors.forename: Lifeng
authors.initials: L
authors.lastname: Wang
authors.forename: Jin
authors.initials: J
authors.lastname: Li
authors.forename: Honghong
authors.initials: H
authors.lastname: Liu
authors.forename: Yuanyuan
authors.initials: Y
authors.lastname: Li
authors.forename: Junliang
authors.initials: J
authors.lastname: Fu
authors.forename: Ying
authors.initials: Y
authors.lastname: Sun
authors.forename: Ruonan
authors.initials: R
authors.lastname: Xu
authors.forename: Hu
authors.initials: H
authors.lastname: Lin
authors.forename: Siyu
authors.initials: S
authors.lastname: Wang
authors.forename: Sa
authors.initials: S
authors.lastname: Lv
authors.forename: Liming
authors.initials: L
authors.lastname: Chen
authors.forename: Zhengsheng
authors.initials: Z
authors.lastname: Zou
authors.forename: Baosen
authors.initials: B
authors.lastname: Li
authors.forename: Ming
authors.initials: M
authors.lastname: Shi
authors.forename: Zheng
authors.initials: Z
authors.lastname: Zhang
authors.forename: Fu-Sheng
authors.initials: FS
authors.lastname: Wang
mesh_terms: D000328:Adult; D002478:Cells, Cultured; D002756:Cholagogues and Choleretics / Q000627:therapeutic use; D005240:Feasibility Studies; D005260:Female; D006801:Humans; D008105:Liver Cirrhosis, Biliary / Q000628:therapy*; D008297:Male; D045164:Mesenchymal Stem Cell Transplantation / Q000379:methods*; D008875:Middle Aged; D010865:Pilot Projects; D011788:Quality of Life; D016896:Treatment Outcome; D014470:Umbilical Cord / Q000166:cytology*; D014580:Ursodeoxycholic Acid / Q000627:therapeutic use
chemical_list: D002756:Cholagogues and Choleretics; D014580:Ursodeoxycholic Acid
keywords: immune regulation; mesenchymal stem cell; primary biliary cirrhosis; ursodeoxycholic acid