Oral budesonide in the treatment of primary sclerosing cholangitis.
OBJECTIVE
This study was designed to evaluate the safety and estimate the efficacy of oral budesonide in patients with primary sclerosing cholangitis (PSC).


METHODS
Twenty-one patients with PSC were treated with 9 mg daily of oral budesonide for 1 yr.


RESULTS
Significant, but marginally important, improvement in serum alkaline phosphatase (1,235 +/- 190 vs 951 +/-206 U/L, p = 0.003) and AST levels (119 +/- 14 vs 103 +/- 19 U/L, p = 0.02) was noted at the end of the treatment period. Serum bilirubin levels increased significantly in the 18 patients who completed 1 yr of treatment (1.1 +/- 0.1 vs 1.4 +/- 0.3, p = 0.01) and no significant changes in liver tests were noted 3 months after budesonide was discontinued. The Mayo risk score did not change significantly, and although a significant improvement in the degree of portal inflammation was noted at the end of the treatment period, the degree of fibrosis and stage of disease were not significantly affected. There was a marked loss of bone mass of the femoral neck (0.851 +/- 0.02 vs 0.826 +/- 0.02 g/cm2, p = 0.002) and lumbar spine (1.042 +/- 0.02 vs 1.029 +/- 0.02 g/cm2, p = 0.09) at 1 yr of treatment with budesonide. Two patients required evaluation for liver transplantation during treatment, and two patients developed cosmetic side effects.


CONCLUSIONS
Oral budesonide appears to be of minimal, if any, benefit and it is associated with a significant worsening of osteoporosis in patients with PSC.
Angulo
P
P
Department of Laboratory Medicine and Pathology, Mayo Clinic and Foundation, Rochester, Minnesota 55905, USA.
Batts
K P
KP
Jorgensen
R A
RA
LaRusso
N A
NA
Lindor
K D
KD
D000284:Administration, Oral; D000328:Adult; D000368:Aged; D000469:Alkaline Phosphatase / Q000097:blood; D000893:Anti-Inflammatory Agents / Q000008:administration & dosage / Q000627:therapeutic use*; D001219:Aspartate Aminotransferases / Q000097:blood; D001663:Bilirubin / Q000097:blood; D015415:Biomarkers / Q000097:blood; D001706:Biopsy; D019819:Budesonide / Q000008:administration & dosage / Q000627:therapeutic use*; D015209:Cholangitis, Sclerosing / Q000097:blood / Q000188:drug therapy* / Q000473:pathology; D018450:Disease Progression; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D010865:Pilot Projects; D011379:Prognosis; D012449:Safety
D000893:Anti-Inflammatory Agents; D015415:Biomarkers; D019819:Budesonide; D001219:Aspartate Aminotransferases; D000469:Alkaline Phosphatase; D001663:Bilirubin