Combination Therapy for Patients with Primary Sclerosing Cholangitis

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The summary below is from the full report titled "Combined Therapy with Azathioprine, Prednisolone, and Ursodiol in Patients with Primary Sclerosing Cholangitis. A Case Series." It is in the 12 December 1999 issue of Annals of Internal Medicine (volume 131, pages 943-946). The authors are C. Schramm, P. Schirmacher, I. Helmreich-Becker, G. Gerken, K.H. Meyer zum Büschenfelde, and A.W. Lohse.

What is the problem and what is known about it so far?

Primary sclerosing cholangitis is a type of liver disease where the ducts that transport bile in the liver become increasingly blocked as time passes. This deadly disease is thought to be an autoimmune disease. Autoimmune means that the disease results when a person develops immune reactions against substances in his or her own body. No treatment is known to be effective at stopping the continuous worsening of this disease. Treatments that keep the immune system in check (immunosuppressants) might help if this is really an autoimmune disease.

Why did the researchers do this particular study?

The researchers report what happened when they treated patients with primary sclerosing cholangitis with the immunosuppressant drugs azathioprine and prednisolone. They gave these drugs in combination with ursodeoxycholic acid, a drug that helps to dissolve bile caught in the liver ducts. Azathioprine and prednisolone are drugs that suppress the immune system.

Who was studied?

The researchers studied 15 patients with primary sclerosing cholangitis at a teaching hospital in Germany.

How was the study done?

The researchers gave each of these patients a combination of the drugs azathioprine, prednisolone, and ursodeoxycholic acid and watched them for between 3 and 81 months (41 months on average). The patients had examinations, blood tests, samples of liver tissue taken (biopsies) to be examined under a microscope, and special tests called endoscopic retrograde cholangiography (ERCP). The ERC involves putting a tube down someone's mouth into their intestinal tract and squirting dye into the bile ducts so the degree of blockage will show up on special x-rays.

What did the researchers find?

The blood tests improved in all 15 patients. Ten of the patients had liver biopsies and 6 of these showed improvements. Ten patients had ERCP and only one of the 10 had worsening blockages of the bile duct. Two of the 15 patients had to stop the treatment because of side effects.

What were the limitations of the study?

Without a comparison group of patients who did not get this combination treatment, we cannot tell for sure that this treatment was responsible for the improvements the researchers saw in these 15 patients.

What are the implications of the study?

Combination therapy with drugs that keep the immune system in check may slow the worsening of primary sclerosing cholangitis. A more sophisticated study of this treatment, including a control group, is necessary.