Management of primary sclerosing cholangitis

Abstract

Primary sclerosing cholangitis (PSC) is a rare cholestatic liver disease with major morbidity and mortality. Therapeutic management is difficult, due to lack of conclusive data and individual disease progression. High-dose UDCA was used for years as a pharmacotherapeutic agent to prevent disease progression, based on a positive trend in pilot studies, but has recently been proven to have a negative effect in advanced disease. Immunosuppressants might be useful in patients with overlap syndromes. Dominant bile duct stenoses should be treated endoscopically, and cholangiocellular carcinoma (CCC) still remains a therapeutic challenge in PSC patients. Early diagnosis of CCC must be improved and new strategies such as neoadjuvant radiochemotherapy with subsequent liver transplantation in selected patients are further options to be considered.

Keywords: Cholangiocellular carcinoma; Cholestatic liver disease; Dominant stenoses; Endoscopy; NorUDCA; Primary sclerosing cholangitis; Ursodeoxycholic acid.

Figures

Figure 1

Primary sclerosing cholangitis and a benign, high-grade stenosis in a 22-year-old man. Cholangiography shows partially bilateral intrahepatic ductal dilatations and irregularity consistent with primary sclerosing cholangitis, and a short, high-grade stenotic segment in the proximal portion of the bile duct.

Figure 2

Therapeutic algorithm in PSC and CCC[34]. Patients with resectable carcinoma should preferably undergo surgical therapy as a potentially curative approach. In advanced stages, endoscopic treatment is the first step in the management of the disease in order to reconstitute biliary drainage. Patients without metastases can undergo local treatment such as photodynamic therapy or radiochemotherapy. Cholangiocarcinoma is considered a contraindication for transplantation, but may offer a possibility for selected patients after extensive preoperative treatment within controlled studies. Systemic chemotherapy remains the only treatment option in metastatic patients.