From the Guidelines
The management of scleroderma should prioritize the use of mycophenolate mofetil, nintedanib, rituximab, and tocilizumab for the treatment of skin fibrosis and interstitial lung disease (ILD), as recommended by the 2023 European Alliance of Associations for Rheumatology (EULAR) update 1. The treatment approach for scleroderma is multifaceted and involves a combination of pharmacological and non-pharmacological interventions.
- For Raynaud's phenomenon, calcium channel blockers such as nifedipine (30-60 mg daily) or amlodipine (5-10 mg daily) are recommended as first-line treatment.
- Immunosuppressants like mycophenolate mofetil (1-2 g daily) are used for skin thickening and lung involvement, with nintedanib being considered for patients with progressive ILD 1.
- Gastrointestinal symptoms are managed with proton pump inhibitors (omeprazole 20-40 mg daily) and prokinetic agents like metoclopramide (10 mg before meals).
- Pulmonary hypertension may require endothelin receptor antagonists (bosentan), phosphodiesterase-5 inhibitors (sildenafil), or prostacyclins, while renal crisis is treated with ACE inhibitors like captopril or enalapril.
- Non-pharmacological approaches, including physical therapy, occupational therapy, and protection from cold temperatures, are also essential in managing scleroderma. The 2023 EULAR update provides a comprehensive framework for the management of scleroderma, emphasizing the importance of individualized treatment based on disease subtype and organ involvement 1.
- Regular screening for organ complications, including pulmonary function tests, echocardiograms, and blood pressure monitoring, is crucial in preventing and managing complications.
- The use of autologous haematopoietic stem cell transplantation may be considered for patients with early diffuse cutaneous SSc who are at high risk of mortality, as suggested by recent evidence 1. However, the most recent and highest-quality study, the 2023 EULAR update, should be prioritized in guiding treatment decisions 1.
From the Research
Management Options for Scleroderma
The management of scleroderma involves a range of treatment options, including:
- Vasodilators, such as calcium channel blockers and angiotensin-converting enzyme inhibitors, to improve peripheral blood circulation 2
- Immunosuppressant drugs, such as methotrexate and cyclophosphamide, to prevent the synthesis and release of harmful cytokines 2
- Antifibrotic agents, such as D-penicillamine and colchicine, to inhibit or reduce fibrosis 2
- Endothelin receptor antagonists and direct renin inhibitors for the treatment of scleroderma renal crisis (SRC) 3
- Biologic agents targeting collagen, cytokines, and cell surface molecules, which may have promising therapeutic effects in systemic sclerosis (SSc) 4
Treatment of Specific Complications
For specific complications of scleroderma, the following treatment options are recommended:
- Scleroderma renal crisis (SRC): angiotensin-converting enzyme inhibitors as first-line therapy, with the addition of calcium channel blockers, angiotensin receptor blockers, or alpha-blockers as second-line therapy 5
- Pulmonary arterial hypertension (PAH): endothelin receptor agonists as first-line therapy, with the addition of phosphodiesterase-5 inhibitors and prostanoids as second-line therapy 5
- Raynaud's phenomenon: calcium channel blockers as first-line therapy, with the addition of phosphodiesterase-5 inhibitors, angiotensin receptor blockers, and prostanoids as second-line therapy 5
- Digital ulcers: calcium channel blockers as first-line therapy, with the addition of phosphodiesterase-5 inhibitors, endothelin receptor agonists, and prostanoids as second-line therapy 5
- Interstitial lung disease/pulmonary fibrosis: intravenous cyclophosphamide and mycophenolate mofetil as induction therapy, with mycophenolate mofetil as maintenance therapy 5
Novel Investigational Agents
Several novel investigational agents are being studied for the treatment of scleroderma, including:
- Rituximab, tocilizumab, and IVIG for the treatment of active SSc skin or lung disease 6
- Bortezomib, LPA-1 antagonists, anti-CCN2 therapy, anti-IL-13, and thrombin antagonists for the treatment of fibrosis 6
- Agents targeting intracellular molecular pathways and matricellular proteins, which may be a novel area of investigation 6