What is the treatment approach for scleroderma?

Treatment Approach for Scleroderma

The treatment of scleroderma requires an organ-based approach targeting specific manifestations, as there is no single therapy that modifies the overall disease course, but early intervention targeting specific organ involvement improves both quality of life and survival. 1

Classification and Initial Assessment

Scleroderma can be divided into two main subgroups:

• Localized scleroderma: Usually confined to specific regions with no internal organ involvement
• Systemic sclerosis (SSc): Further classified as:
  • Limited cutaneous SSc: Less extensive skin involvement
  • Diffuse cutaneous SSc: More extensive skin involvement with potential for severe organ complications

Initial evaluation should include:

• Clinical evaluation of cutaneous changes
• Serological tests (anti-centromere, anti-topoisomerase I, anti-RNA polymerase III antibodies)
• Pulmonary function tests
• HRCT of the chest
• Echocardiography
• Capillaroscopy 1

Organ-Specific Treatment Approaches

1. Skin Involvement

• Diffuse/extensive skin involvement:

  • First-line: Methotrexate (15 mg/week) for at least 12 months 1
  • Monitor for adverse effects of immunosuppressive treatment

• Limited lesions:

  • Topical corticosteroids
  • UVA1 phototherapy for circumscribed lesions 1

2. Vascular Complications

• Digital ulcers:

  • Bosentan for prevention of new digital ulcers 1

• Raynaud's phenomenon:

  • Fluoxetine may be considered 1
  • Calcium channel blockers (not explicitly mentioned in evidence but standard of care)

3. Pulmonary Involvement

• Interstitial Lung Disease (ILD):

  • First-line: Mycophenolate mofetil (500-1000 mg/m²) 1
  • For severe cases: Cyclophosphamide 1
  • For progressive fibrotic ILD: Nintedanib 1

• Pulmonary Hypertension:

  • Initial therapy: Combination of PDE-5 inhibitors and endothelin receptor antagonists 1
  • Additional therapy: Prostacyclin analogues if needed 1
  • Newer option: Riociguat 1

4. Gastrointestinal Involvement

• GERD: Proton pump inhibitors 1
• Motility disorders: Prokinetic agents 1
• Bacterial overgrowth: Rotating antibiotics 1
• Malnutrition: Nutritional support (critical as this is a leading cause of GI-related mortality) 1

5. Renal Involvement

• Scleroderma renal crisis: High-dose ACE inhibitors as first-line therapy 1
• Important caution: Avoid glucocorticoids or use at low doses as they may precipitate renal crisis 1

Monitoring and Follow-up

Regular monitoring is crucial for early detection of organ involvement:

• Pulmonary function tests
• HRCT of chest
• Echocardiography
• Complete joint examination including temporomandibular joint
• Ophthalmological follow-up including uveitis screening 1

Supportive Care

• Avoid skin irritants and perfumed products
• Patient education about disease management
• Psychological support (fundamental due to impact on quality of life and possible disfigurement) 1
• Address factors affecting daily function: nutrition, pain management, deconditioning, musculoskeletal issues 2

Treatment Challenges and Pitfalls

1. Timing is critical: Early intervention before irreversible damage occurs improves outcomes 2

2. No disease-modifying therapy: Currently, no treatment has been proven to modify the overall disease course 2

3. Heterogeneity: Clinical presentation varies widely, requiring careful characterization of each patient 2

4. Multisystem approach: Treatment must be holistic while focusing on dominant organ disease 2

5. Pediatric considerations: Juvenile forms require special attention due to potential growth defects in localized disease and higher mortality in systemic disease 3

6. Medication cautions: Monitor for adverse effects of immunosuppressives; be particularly cautious with glucocorticoids due to renal crisis risk 1