Treatment of Scleroderma
The treatment of scleroderma should be organ-based, focusing on specific manifestations, with different therapeutic approaches for each affected system. 1 This approach is essential as scleroderma is a complex, heterogeneous disease affecting multiple organ systems with varying severity.
Management of Raynaud's Phenomenon and Digital Ulcers
- First-line therapy: Dihydropyridine calcium channel blockers, especially nifedipine 2, 1
- Second-line therapy: Phosphodiesterase-5 (PDE-5) inhibitors 2, 1
- Third-line therapy: Intravenous iloprost for severe cases 2, 1
- For prevention of new digital ulcers: Bosentan 2, 1
- Additional option: Fluoxetine may be considered for SSc-related Raynaud's phenomenon 2
Management of Skin Manifestations
- Limited lesions: Topical corticosteroids 1
- Circumscribed lesions: UVA1 phototherapy 1
- Extensive/diffuse skin involvement: Methotrexate (15 mg/week) is recommended as first-line therapy 2, 1
- Alternative for rapidly progressive SSc: Hematopoietic stem cell transplantation for selected patients 2, 1
Management of Interstitial Lung Disease (ILD)
- First-line therapy: Mycophenolate mofetil (500-1000 mg/m²) 1
- For severe cases: Cyclophosphamide 2, 1
- For progressive fibrotic ILD: Nintedanib 1
Management of Pulmonary Arterial Hypertension (PAH)
- Initial therapy: Combination therapy with PDE-5 inhibitors and endothelin receptor antagonists 2, 1
- Additional therapy if needed: Prostacyclin analogues 2, 1
- Newer option: Riociguat 2
Management of Gastrointestinal Involvement
- For GERD: Proton pump inhibitors 2, 1
- For motility disorders: Prokinetic agents 2, 1
- For bacterial overgrowth: Rotating antibiotics 2, 1
- For malnutrition: Nutritional support 1
Management of Scleroderma Renal Crisis
- First-line therapy: High-dose ACE inhibitors 2, 1
- Important note: 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: To assess lung function
- HRCT of the chest: To evaluate lung involvement
- Echocardiography: To detect pulmonary hypertension
- Capillaroscopy: To evaluate microvasculature
Important Clinical Considerations
Disease subtype matters: Limited scleroderma has less extensive skin involvement and typically doesn't require aggressive systemic immunosuppression for skin manifestations alone, but requires vigilant monitoring for late complications, particularly PAH 1
Timing of intervention: Early treatment before irreversible damage occurs improves both quality of life and survival 3
Avoid traditional ineffective treatments: Some previously used drugs like D-penicillamine or colchicine have been disappointing in clinical practice despite anecdotal evidence 4, 5
Comprehensive care: Address nutrition, pain, deconditioning, and psychological support due to the impact on quality of life 1, 3
Treatment duration: For medications like methotrexate, treatment should continue for at least 12 months after achieving acceptable clinical improvement 1
The management of scleroderma remains challenging, with no single treatment modifying the overall disease course. However, organ-specific therapy has shown success in improving quality of life and survival 5. Regular monitoring and early intervention for organ complications are essential components of care.