Treatment of Scleroderma
The treatment of scleroderma requires an organ-specific approach targeting the affected systems, with methotrexate combined with systemic corticosteroids being the first-line therapy for active, potentially disfiguring or disabling forms of scleroderma. 1, 2
Types of Scleroderma and Treatment Approach
- Scleroderma is classified into two major clinical subsets: systemic sclerosis (SSc) and localized scleroderma (morphea), with different treatment approaches for each 3
- Treatment must target specific organ involvement early before irreversible damage occurs to improve quality of life and survival 4
Systemic Sclerosis (SSc) Treatment
Raynaud's Phenomenon and Digital Ulcers
- Dihydropyridine-type calcium channel blockers (particularly oral nifedipine) are recommended as first-line therapy for Raynaud's phenomenon 5
- Phosphodiesterase-5 (PDE-5) inhibitors should be considered for treatment of SSc-related Raynaud's phenomenon and digital ulcers 1, 5
- Intravenous iloprost should be considered for severe Raynaud's phenomenon that fails to respond to oral therapy 5
- Fluoxetine may be used for SSc-related Raynaud's phenomenon as an alternative treatment option 1, 5
Pulmonary Arterial Hypertension (PAH)
- Treatment options for SSc-related PAH include:
- Endothelin receptor antagonists
- Prostacyclin analogues
- PDE-5 inhibitors
- Riociguat (a newer treatment option) 1
Skin and Lung Disease
- For rapidly progressive SSc with skin and/or lung involvement, hematopoietic stem cell transplantation may be considered in selected patients 1
- Immunosuppressants including methotrexate, cyclosporine, and cyclophosphamide may be used for skin and lung involvement 6
Scleroderma Renal Crisis
- Angiotensin-converting enzyme inhibitors are the cornerstone of treatment for scleroderma renal crisis 4, 6
Gastrointestinal Involvement
- Proton pump inhibitors are used for management of gastroesophageal reflux disease 7
- Prokinetic agents may be beneficial for GI motility disorders 1
Localized Scleroderma (Morphea) Treatment
Assessment and Classification
- Standardized assessment using the Localized Scleroderma Cutaneous Assessment Tool (LoSCAT) is recommended to evaluate disease activity and damage 2, 8
- Classification based on subtype, extent, and depth should guide treatment decisions 2
Treatment Algorithm
For limited, superficial lesions (circumscribed morphea):
For linear, deep, generalized, or pansclerotic morphea:
- Methotrexate (MTX) at 15 mg/m²/week (oral or subcutaneous) combined with systemic corticosteroids during the initial inflammatory phase is recommended as first-line therapy 2, 8
- MTX should be maintained for at least 12 months after achieving clinical improvement before tapering 2, 8
- For MTX-refractory or MTX-intolerant patients, mycophenolate mofetil (500-1000 mg/m²) is recommended as second-line therapy 2, 8
- Biologics, including TNF or IL-6 inhibitors, have been used in some patients with severe recalcitrant disease 2
Monitoring and Follow-up
- Regular clinical assessment using standardized tools is essential to monitor disease activity and response to treatment 2, 8
- Close monitoring for medication side effects, particularly with MTX which may cause nausea, headache, and transient hepatotoxicity 2
- MTX or alternative disease-modifying drugs should be withdrawn once the patient is in remission and off steroids for at least 1 year 1, 2
Common Pitfalls in Treatment
- Inadequate treatment duration is a common pitfall - treatment should be continued for sufficient time to prevent relapse 2
- Underestimating the importance of treating skin disease aggressively can lead to significant physical and psychological morbidity 1, 2
- For patients with facial and head involvement (such as "en coup de sabre" or Parry-Romberg syndrome), MRI of the head is highly recommended at the time of diagnosis, regardless of neurological symptoms 8