[Treatments of progressive systemic sclerosis]

Area of Science:

• Rheumatology and Immunology
• Clinical Pharmacology
• Medical Research Methodology

Context:

• Progressive Systemic Sclerosis (PSS) presents diagnostic and therapeutic challenges due to low prevalence and varied clinical manifestations.
• Limited financial interest from pharmaceutical companies and lack of clear efficacy endpoints complicate clinical trial design for PSS.
• Recent advancements include earlier diagnosis via capillaroscopy and antibody assays, improved prognostic understanding through disease classification, and early detection of pulmonary hypertension.

Purpose:

• To review the current state of Progressive Systemic Sclerosis (PSS) treatment and discuss advancements in diagnosis, prognosis, and therapeutic strategies.
• To highlight effective treatments for specific visceral involvements in PSS, including renal, respiratory, and pulmonary hypertension.
• To evaluate the impact of novel therapeutic agents, such as oral prostacyclin derivatives, on PSS complications like digital necrosis.

Summary:

• Despite being incurable, PSS management has improved with earlier diagnosis (capillaroscopy, antibody assays) and better prognosis classification (limited vs. diffuse).
• Effective treatments exist for renal (ACE inhibitors), respiratory (D-penicillamine), and pulmonary hypertension (prostacyclin derivatives) in PSS.
• A trial of an oral prostacyclin derivative showed reduced digital necrosis, improved well-being, and decreased hospitalizations, with decreased von-Willebrand factor levels.

Impact:

• Improved diagnostic accuracy and prognostic stratification are enhancing the methodology for future PSS drug trials.
• Targeted therapies offer significant benefits for specific organ involvements, improving quality of life for PSS patients.
• Novel treatments like prostacyclin derivatives demonstrate potential in managing severe PSS complications and improving patient outcomes.