The study is designed to evaluate the safety and efficacy of intravenous administration of bone marrow derived mesenchymal stem cells for patients with refractory primary biliary cirrhosis (PBC).
Primary biliary cirrhosis (PBC) is an organ-specific inflammatory disease and characterized by immune mediated destruction of intrahepatic bile ducts, then lead to liver cirrhosis and eventually failure.Currently, ursodeoxycholic acid (UDCA) is the only drug approved by the Food and Drug Administration (FDA). Novel treatment is urgently needed for patients who have an incomplete response to UDCA. Mesenchymal stem cells (MSC) represent a promising tool for cell-based therapies of autoimmune diseases. To explore the therapeutic effect of MSCs for PBC, the investigators plan to conduct an open-label, randomized clinical trial. Patients with PBC will be enrolled and randomly divided into two groups which will receive MSCs and UDCA respectively. The investigators will evaluate the efficacy and safety of MSCs for PBC by comparison of symptom improvement, survival rate and side effects in the two groups.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
20
Mesenchymal stem cells,5-50 million/kg, Intravenous infusion, One dosage,whether to give another dosage depending on patients' condition
13-15 mg/kg/day, to the end of the study
Peking Union Medical College Hospital
Beijing, China
RECRUITINGserum level of alkaline phosphatase
Serum level of alkaline phosphatase will be measured at entry, 1 months,3 months, 6 months and 24 months after therapy
Time frame: 24 months after MSCs administration
histological changes in liver biopsies
Liver biopsy of each patient will be taken before entry into therapeutic trials and at 6 months after therapy.
Time frame: 6 months after therapy
Serum levels of TNF-alpha
serum levels of TNF-alpha will be assessed before entry into therapeutic trials and at 6 months after therapy
Time frame: 6 months after therapy
changes in fatigue
changes in fatigue will be evaluated before test (baseline), 1 month,3 months and 6 months after theraphy by PBC-40 score.
Time frame: 6 months after theraphy
The occurrence of cirrhosis and its complications
Time frame: 24 months after therapy
Serum levels of Interleukin
serum levels of Interleukin will be assessed before entry into therapeutic trials and at 6 months after therapy
Time frame: 6 months after therapy
changes in pruritus severity
changes in pruritus severity will be evaluated before test (baseline), 1 month,3 months and 6 months after theraphy by VAS score.
Time frame: 6 months after therapy
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