From the Guidelines
Systemic sclerosis, also known as scleroderma, is a chronic autoimmune disease characterized by fibrosis and vascular alterations in the skin and internal organs.
Diagnosis and Treatment
Treatment typically involves immunosuppressive therapy, such as methotrexate 10-20 mg/week or mycophenolate mofetil 500-1000 mg twice daily, to slow disease progression 1.
- Immunosuppressive agents like mycophenolate mofetil, methotrexate, cyclophosphamide, rituximab, and tocilizumab are used to treat early diffuse cutaneous SSc 1.
- Endothelin receptor antagonists like bosentan 62.5-125 mg twice daily may be used to manage pulmonary arterial hypertension, a common complication of systemic sclerosis 1.
- Disease-modifying antifibrotic agents, such as nintedanib 150 mg twice daily, may also be considered to reduce fibrosis progression 1.
Organ-Specific Therapies
- Interstitial lung disease (ILD) is usually treated with mycophenolate mofetil as the initial therapy, and then other immunosuppressives or biologic agents, and if ILD is fibrotic and progressing, anti-fibrotic therapy can be added, such as nintedanib (and possibly pirfenidone) 1.
- Raynaud phenomenon in SSc is treated with calcium channel blockers and then phosphodiesterase 5 inhibitors or intravenous iloprost 1.
- Pulmonary arterial hypertension is frequently treated with initial combination therapy (for example, with phosphodiesterase 5 inhibitors and endothelin receptor antagonists) and, if necessary, the addition of a prostacyclin analogue 1.
From the Research
Diagnosis of Systemic Sclerosis (SSc)
- Systemic sclerosis (SSc) is a complex systemic autoimmune disorder characterized by inflammation, vasculopathy, and excessive fibrosis of the skin and multiple internal organs 2
- The diagnosis of SSc is based on clinical features, molecular signatures, and identification of subpopulations with distinct clinical trajectories 3
- High-resolution computed tomography (HRCT) is the primary tool to diagnose interstitial lung disease (ILD) in patients with SSc, and full pulmonary function tests (PFTs) with diffusing capacity of the lungs for carbon monoxide (DLco) and ambulatory desaturation testing should be obtained for disease monitoring 4
Treatment of Systemic Sclerosis (SSc)
- 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
- Immunosuppressive therapy such as mycophenolate mofetil, tocilizumab, and rituximab is proposed as first-line treatment for SSc-ILD, and other immunosuppressive treatments and antifibrotic therapy are also reviewed 4
- Early combination therapy with vasodilators, immunosuppressive and antifibrotic drugs should successfully downregulate the disease progression, especially if started from the beginning 5
Treatment Options for Specific Manifestations of SSc
- Pulmonary hypertension and interstitial lung disease are the primary causes of increased mortality in SSc, and treatments targeting these manifestations are crucial 6
- Local skin-targeted therapy is generally used for localized scleroderma (LoS), 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