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Ursodeoxycholic acid treatment in isolated chronic graft-vs.-host disease of the liver
Mutlu Arat1, Ramazan Idilman, Ender Akcaglayan Soydan
1Department of Hematology, Ankara University School of Medicine, Ankara, Turkey. arat@medicine.ankara.edu.tr
Insights
Long-term ursodeoxycholic acid (UDCA) treatment effectively improved liver tests in patients with chronic graft-vs.-host disease (cGVHD) of the liver. The therapy was safe and well-tolerated, with no major adverse events reported.
Area of Science:
- Hepatology
- Immunology
- Transplantation Medicine
Background:
- Limited data exist on long-term ursodeoxycholic acid (UDCA) treatment for chronic graft-vs.-host disease (cGVHD) of the liver.
- Isolated liver cGVHD following allogeneic hematopoietic cell transplantation requires effective and safe therapeutic options.
Purpose of the Study:
- To evaluate the efficacy of long-term UDCA treatment in patients with isolated liver cGVHD.
- To assess the tolerability and safety of UDCA in this patient population.
Main Methods:
- A prospective study included fifteen patients with de novo isolated liver cGVHD.
- Patients received UDCA at 13 mg/kg/d for one year, with clinical and laboratory assessments at regular intervals.
Main Results:
- Sixty percent of patients achieved normal liver tests, while 40% showed improvement.
- Significant reductions in serum aminotransferases, alkaline phosphatase, and gamma-glutamyl transpeptidase were observed (p < 0.01).
- Pruritus resolved in seven of nine patients, and all patients tolerated the treatment without major adverse events.
Conclusions:
- Long-term UDCA therapy is effective for individuals with cGVHD involving the liver.
- UDCA treatment is safe and well-tolerated in this patient group.
Objectives:
Data regarding the long-term treatment of ursodeoxycholic acid (UDCA) in individuals of chronic graft-vs.-host disease (cGVHD) of the liver are limited. The aims of this prospective study were to determine whether, (i) UDCA treatment is useful as a long-term treatment for individuals with limited cGVHD of the liver following allogeneic hematopoietic cell transplantation, and (ii) the tolerability of UDCA treatment in such individuals.
Methods:
Fifteen consecutive patients with de novo isolated cGVHD of the liver were included. All individuals were treated with UDCA at a dose of 13 mg/kg/d for 1 yr. Clinical evaluation and laboratory testing were assessed at 30-d intervals during UDCA therapy and every 30 d after discontinuation of UDCA for a total of 3 months.
Results:
At the end of the treatment, 60% of patients with cGVHD of the liver had normal liver tests, the remaining 40% of patients demonstrated improvement in their abnormal liver tests (partial responders), whereas none of the patients had worsening of the liver tests. When compared with baseline, there was a significant decrease in the serum aminotransferases, alkaline phosphatase and gamma-glutamyl transpeptidase levels after completion of the UDCA treatment at 12 months (p < 0.01). No significant increase in serum liver enzyme tests was observed at the third month after the completion of therapy. Pruritus in seven of nine patients resolved after UDCA treatment. All patients completed their assigned treatment with no major adverse event.
Conclusion:
Long-term UDCA therapy appears to be effective, safe and tolerable in individuals with cGVHD presenting with isolated liver involvement.
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