Primary sclerosing cholangitis (PSC) is a chronic cholestatic liver disease of unknown etiology.
Despite advances in understanding the pathophysiology underlying this disorder, no effective medical therapy has been identified for halting disease progression.
Dr Talwalkar and colleagues from Minnesota in America undertook a study in order to determine the safety and estimated efficacy of mycophenolate mofetil (MMF) for the treatment of primary sclerosing cholangitis.
The research team administered MMF 1 g daily up to a maximum of 3 g daily for 1 yr to a total of 30 patients with primary sclerosing cholangitis.
The researchers then performed liver tests for each participant at 3-month intervals with the Mayo risk score calculated at baseline and at the end of therapy.
|7 patients discontinued MMF due to adverse events possibly related to therapy |
|The American Journal of Gastroenterology|
77% of patients completed the 1 yr of therapy.
The researchers observed a significant but clinically marginal improvement in serum alkaline phosphatase level after 1 yr of therapy.
The research team found no other significant changes in liver biochemistries or Mayo risk score.
The researchers noted that 7 patients discontinued mycophenolate mofetil due to adverse events possibly related to therapy.
Adverse reactions resolved spontaneously or with dose reduction in 10 (33%) patients.
The research team noted that 1 patient developed pancreatitis, bacterial cholangitis, and sepsis during the eighth month of mycophenolate mofetil therapy.
No patient developed cytopenia on therapy.
Dr Talkwalkar concluded, "Mycophenolate mofetil does not appear to have clinically important benefits for PSC despite being tolerated by most patients."
"The results of this pilot study do not support further study of mycophenolate mofetil as a single agent in the treatment of PSC."