What is the management approach for sclerosing conditions?

From the Guidelines

The management of sclerosing conditions should prioritize controlling inflammation, preventing disease progression, and addressing specific symptoms, with a focus on improving morbidity, mortality, and quality of life. The most recent and highest quality study, 1, published in 2023, provides state-of-the-art evidence in the treatment of systemic sclerosis, highlighting the importance of early intervention and organ-specific therapies. Key aspects of management include:

• Controlling inflammation with corticosteroids, such as prednisone, and immunosuppressive agents like mycophenolate mofetil or methotrexate
• Addressing specific symptoms, such as Raynaud phenomenon and digital ulcers, with dihydropyridine calcium channel blockers, phosphodiesterase 5 inhibitors, or intravenous iloprost
• Using antifibrotic medications, such as pirfenidone or nintedanib, to slow disease progression in conditions like idiopathic pulmonary fibrosis
• Providing supportive care, including management of complications, nutritional support, and physical therapy to maintain function
• Regular monitoring with laboratory tests, imaging studies, and pulmonary function tests to assess treatment response and disease progression. According to 1, the European Alliance of Associations for Rheumatology (EULAR) recommendations for the treatment of systemic sclerosis emphasize the importance of a multidisciplinary approach and highlight the need for further research in this area. Additionally, 1 discusses the current management of systemic sclerosis, including screening for and treatment of organ-based manifestations, and consideration of overall disease modification with autologous haematopoietic stem cell transplantation. Overall, the goal of management is to improve morbidity, mortality, and quality of life for patients with sclerosing conditions, and to slow disease progression and prevent complications.

From the Research

Management Approach for Sclerosing Conditions

The management approach for sclerosing conditions, such as primary sclerosing cholangitis (PSC), involves various treatment strategies.

• Medical therapy:
  • Ursodeoxycholic acid (UDCA) has been widely used, but its effectiveness is still debated 2, 3.
  • Immunosuppressive agents like mycophenolate mofetil have shown no significant benefit 2.
  • Other medications like pentoxifylline, antibiotics, and docosahexanoic acid have been tested with limited success 4.
• Endoscopic treatment:
  • Biliary stent therapy, including dilatation and stenting, can provide relief for dominant strictures 5.
  • Self-expandable metallic stents are also being used as an innovative option 5.
• Novel pharmacological agents:
  • Modulation of bile composition, immunomodulation, targeting the gut microbiome, and targeting fibrosis are being explored as potential treatment strategies 6.
• Personalized treatment:
  • A combination of different drugs and endoscopic treatment may be the most effective approach for successful PSC therapy 6.

Treatment Outcomes

The outcomes of these treatment strategies vary, with some showing improvement in biochemical parameters or symptoms, but none have been proven to delay disease progression or improve transplant-free survival.

• UDCA:
  • No significant difference in time to treatment failure or liver transplantation 3.
  • Improvement in serum alkaline phosphatase, aspartate aminotransferase, bilirubin, and albumin levels 3.
• Biliary stent therapy:
  • Effective in 80% of patients at 1 year and 60% at 3 years 5.
  • Safe and does not increase the risk of malignant transformation or complications for liver transplant candidates 5.