Pilot study of umbilical cord-derived mesenchymal stem cell transfusion in patients with primary biliary cirrhosis.
J Gastroenterol Hepatol
; 28 Suppl 1: 85-92, 2013 Aug.
Article
en En
| MEDLINE
| ID: mdl-23855301
ABSTRACT
BACKGROUND AND AIM:
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.Palabras clave
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
Cordón Umbilical
/
Trasplante de Células Madre Mesenquimatosas
/
Cirrosis Hepática Biliar
Tipo de estudio:
Clinical_trials
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Observational_studies
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Prognostic_studies
/
Risk_factors_studies
Aspecto:
Patient_preference
Límite:
Adult
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Female
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Humans
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Male
/
Middle aged
Idioma:
En
Revista:
J Gastroenterol Hepatol
Asunto de la revista:
GASTROENTEROLOGIA
Año:
2013
Tipo del documento:
Article
País de afiliación:
China