From the Guidelines
Scleroderma management should prioritize a multidisciplinary approach, focusing on organ-specific treatments and symptomatic relief, as outlined in the most recent EULAR recommendations 1.
Symptoms and Workup
Scleroderma is a chronic autoimmune disease characterized by excessive collagen production, leading to skin thickening and potential internal organ involvement. Symptoms include:
- Raynaud's phenomenon (fingers turning white/blue in cold)
- Skin thickening
- Joint pain
- Dysphagia
- Heartburn
- Shortness of breath
- Fatigue Workup should include a thorough history and physical examination, followed by laboratory tests including:
- Antinuclear antibodies (ANA)
- Anti-Scl-70
- Anti-centromere antibodies
- Complete blood count
- Comprehensive metabolic panel
- Urinalysis Additional testing may include:
- Pulmonary function tests
- High-resolution chest CT
- Echocardiogram
- Esophageal manometry depending on suspected organ involvement.
Management
Management is primarily symptomatic and organ-specific.
- For Raynaud's phenomenon, calcium channel blockers like nifedipine 30-60mg daily are first-line, with phosphodiesterase-5 inhibitors (sildenafil 20mg three times daily) as alternatives 1.
- Skin manifestations may be treated with methotrexate (15-25mg weekly) or mycophenolate mofetil (1-2g daily) 1.
- Gastrointestinal symptoms require proton pump inhibitors like omeprazole 20-40mg daily for reflux.
- Pulmonary arterial hypertension may require endothelin receptor antagonists (bosentan), while interstitial lung disease often requires immunosuppressants like cyclophosphamide or mycophenolate 1.
- Scleroderma renal crisis necessitates ACE inhibitors like captopril. All patients should avoid cold exposure, stop smoking, perform regular physical therapy, and receive multidisciplinary care. The disease mechanism involves autoimmunity, vascular damage, and fibroblast activation leading to excessive collagen deposition in tissues, which explains the diverse manifestations and need for targeted therapies. Key treatment considerations, as per the latest evidence 1, include the use of mycophenolate mofetil, nintedanib, rituximab, and tocilizumab for skin fibrosis and interstitial lung disease, highlighting the importance of staying updated with the latest recommendations for optimal patient care.
From the Research
Scleroderma Symptoms
- Scleroderma is a complex systemic autoimmune disorder characterized by inflammation, vasculopathy, and excessive fibrosis of the skin and multiple internal organs 2
- There are two major clinical subsets of scleroderma: systemic sclerosis (SSc) and localized scleroderma (LoS) 2
- SSc is associated with significant morbidity and mortality compared with other rheumatic diseases, with fatal outcomes often originating from organ complications such as lung fibrosis, pulmonary artery hypertension (PAH), and scleroderma renal crisis (SRC) 2
Workup
- The diagnosis of scleroderma involves a comprehensive evaluation of the patient's symptoms, medical history, and physical examination 3
- Laboratory tests and imaging studies may be ordered to confirm the diagnosis and assess the extent of organ involvement 3
- The physician needs to carefully characterize each patient to understand the specific manifestations and level of disease activity to decide appropriate treatment 3
Management
- Current treatment modalities in SSc have focused on targeting vascular damage, fibrosis, and regulation of inflammation as well as autoimmune responses 2
- Some canonical medications, including glucocorticoids, immunosuppressants, and vasodilators, have had some success in treating various manifestations in SSc patients 2
- Increasing evidence suggests that some biologic agents targeting collagen, cytokines, and cell surface molecules might have promising therapeutic effects in SSc 2
- Hematopoietic stem cell transplantation (HSCT), mostly autologous, has made great progress as a promising treatment option in severe and refractory SSc 2
- For LoS, local skin-targeted therapy is generally used, including topical application of glucocorticoids or other immunomodulatory ointments and ultraviolet (UV) irradiation 2
- Systemic immunosuppressants are also utilized in several forms of LoS 2
- Treatment of SSc complications, such as SRC, PAH, and digital ulcers, requires a multidisciplinary approach and may involve the use of endothelin receptor antagonists, phosphodiesterase-5 inhibitors, and prostacyclins 4, 5
Self-Management
- Patients with scleroderma face unique challenges, including accessing information, dealing with negative emotions, and accessing resources 6
- Strategies for overcoming these challenges include seeking connections to other people with scleroderma or scleroderma patient organizations, actively seeking out local resources, and learning to communicate and advocate more effectively 6
- Rehabilitation professionals can support people with scleroderma by providing them with information on connecting with scleroderma patient organizations or by facilitating local patient support networks 6