What is the recommended treatment plan for patients with scleroderma?

Treatment Plan 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. 1 Treatment should focus on the affected organ systems with different therapeutic strategies for each manifestation.

Skin Involvement

• First-line therapy for extensive/diffuse skin involvement: Methotrexate (15 mg/week) 1
• For limited lesions: Topical corticosteroids 1
• For circumscribed lesions: UVA1 phototherapy 1

Digital Vasculopathy

• For prevention of digital ulcers: Bosentan 1
• For Raynaud's phenomenon: Fluoxetine may be considered 1

Pulmonary Involvement

Interstitial Lung Disease (ILD)

• First-line therapy: Mycophenolate mofetil (500-1000 mg/m²) 1
• For severe cases: Cyclophosphamide 2, 1
  • More effective in patients with baseline FVC <70% of predicted and/or extensive reticular infiltrates on HRCT 2
  • Treatment effect was 9.81% improvement in FVC at 18 months in patients with ≥50% lung zone involvement on HRCT and/or mRSS ≥23/51 2
• For progressive fibrotic ILD: Nintedanib 1
• For rapidly progressive disease: Hematopoietic stem cell transplantation (HSCT) should be considered in selected patients at risk of organ failure 2
  • Should be performed only in centers with experience in this treatment 2

Pulmonary Hypertension

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

Gastrointestinal Involvement

• For GERD: Proton pump inhibitors 1
• For motility disorders: Prokinetic agents 1
• For bacterial overgrowth: Rotating antibiotics 1
• For malnutrition: Nutritional support 1

Renal Involvement

• For scleroderma renal crisis (SRC): Immediate use of high-dose ACE inhibitors is essential and significantly improves survival 2, 1
  • Studies show 1-year survival rates of 76% with ACE inhibitors vs. 15% without 2
  • 5-year survival rates of 66% with ACE inhibitors vs. 10% without 2
  • The beneficial effect remained significant after adjustment for age and blood pressure (p=0.001) 2
• Important caution: Glucocorticoids should be avoided or used 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 the chest
• Echocardiography
• Capillaroscopy
• Complete joint examination
• Ophthalmological follow-up 1

Treatment Pitfalls and Considerations

1. Glucocorticoid use: High-dose steroids can precipitate scleroderma renal crisis and should be used with extreme caution 1

2. Treatment duration: Immunosuppressive treatments like methotrexate should be continued for at least 12 months after achieving acceptable clinical improvement 1

3. Organ-specific approach: Due to the heterogeneity of scleroderma, treatment must be tailored to the specific organ systems involved rather than using a one-size-fits-all approach 1

4. Early intervention: Treatment is most effective when initiated early before irreversible organ damage occurs 1

5. HSCT considerations: While effective for rapidly progressive disease, HSCT carries significant risks and should only be performed in specialized centers after careful patient selection 2

6. Comprehensive care: Address nutrition, pain management, and psychological support as part of the treatment plan 1