From the Guidelines
Yes, there is hope for individuals diagnosed with Primary Sclerosing Cholangitis (PSC), as with appropriate management, many patients can live for decades after diagnosis. The diagnosis of PSC is made in the presence of typical findings of sclerosing cholangitis on high-quality cholangiography, such as magnetic resonance cholangiopancreaticography (MRCP), and after exclusion of secondary causes 1.
Key Considerations
- PSC is a chronic, progressive liver disease without a cure, but treatment focuses on managing symptoms, slowing disease progression, and addressing complications.
- Ursodeoxycholic acid (UDCA) at doses of 13-15 mg/kg/day may help some patients by improving liver biochemistry, though its effect on disease progression remains debated.
- For symptom management, cholestyramine (4-16 g/day) can help control itching, while antibiotics like ciprofloxacin or metronidazole are used for bacterial cholangitis episodes.
- Endoscopic procedures to dilate strictures and remove stones can provide significant relief for those with dominant strictures.
- Ultimately, liver transplantation remains the definitive treatment for advanced PSC, with excellent 5-year survival rates exceeding 85% 2.
Monitoring and Complications
- Regular monitoring with blood tests every 3-6 months and imaging studies annually helps track disease progression and detect complications early.
- Since PSC is often associated with inflammatory bowel disease, managing any underlying gut inflammation is important for overall health outcomes.
- Patients with PSC are at higher risk of developing cholangiocarcinoma, and regular screening is essential to detect this complication early 3.
Treatment Outcomes
- Patient and graft survival rates are comparable to those of age-matched children who undergo transplantation for other indications 3.
- Post-LT complications include intrahepatic biliary strictures, cholangitis, and disease recurrence in the graft.
- Five-year survival following LT for PSC is >80% 3.
From the Research
Diagnosis of PSC
- The diagnosis of Primary Sclerosing Cholangitis (PSC) is generally established with a bile duct imaging study, typically magnetic resonance cholangiopancreaticography (MRCP), which is more than 80% sensitive and more than 90% specific for the diagnosis of PSC 4.
- MRI of the biliary tract is the primary imaging technology for diagnosis 5.
- Cholangiography, either magnetic resonance cholangiography (MRCP) or endoscopic retrograde cholangiography, can be used to confirm the diagnosis 6.
Treatment and Management
- There is no causally directed treatment for PSC, and management is mainly focused on treatment of symptoms and addressing complications 4, 7.
- Endoscopic interventions of the bile ducts should be limited to clinically relevant strictures for balloon dilatation, biopsy, and brush cytology 5.
- Liver transplantation is currently the only life-extending therapeutic approach for eligible patients with end-stage PSC, ultimately required in approximately 40% of patients 7, 8.
- Several novel therapeutic strategies are in various stages of development, including apical sodium-dependent bile acid transporter and ileal bile acid transporter inhibitors, integrin inhibitors, peroxisome proliferator-activated receptor agonists, CCL24 blockers, recombinant FGF19, CCR2/CCR5 inhibitors, farnesoid X receptor bile acid receptor agonists, and nor-ursodeoxycholic acid 5.
Prognosis and Outcome
- The time from diagnosis to death or liver transplantation is 12 to 18 years, and the risk that a patient with PSC will die of cancer is 40% to 58% 4.
- PSC is a progressive disease, and the clinical course is marked by progressive liver disease leading to cirrhosis with its attendant complications of portal hypertension 6.
- The 10-year survival rate after liver transplantation is above 80% 4.