What is the initial management for patients with scleroderma (systemic sclerosis)?

From the Guidelines

The initial management of scleroderma (systemic sclerosis) should focus on treating specific organ manifestations and symptoms, with a thorough assessment to identify organ involvement, and treatment guided by the most recent evidence-based recommendations, such as those from the European Alliance of Associations for Rheumatology (EULAR) 1. The treatment approach is organ-based, aiming to modify the disease process and improve outcomes. For patients with Raynaud's phenomenon, first-line treatment includes dihydropyridine-type calcium antagonists, such as nifedipine, and phosphodiesterase 5 inhibitors should also be considered 1. In cases of digital ulcers, phosphodiesterase 5 inhibitors and/or intravenous iloprost are recommended, and bosentan can be used to reduce the development of new digital ulcers 1. For interstitial lung disease (ILD), mycophenolate mofetil (MMF), cyclophosphamide, or rituximab should be considered, and nintedanib can be used alone or in combination with MMF for the treatment of SSc-ILD 1. Skin manifestations can be treated with methotrexate, MMF, and/or rituximab, and tocilizumab may be considered for early, inflammatory diffuse cutaneous systemic sclerosis (dcSSc) 1. A multidisciplinary approach involving rheumatologists, pulmonologists, cardiologists, and other specialists is crucial for comprehensive care, and regular monitoring is essential to improve outcomes in patients with scleroderma. Key considerations include:

• Organ-specific treatment approaches
• Use of immunosuppressants and biologic agents for skin and lung involvement
• Management of Raynaud's phenomenon and digital ulcers
• Regular monitoring and a multidisciplinary care approach. The most recent and highest quality study, the 2023 EULAR recommendations for the treatment of systemic sclerosis, provides guidance on the management of various organ manifestations and symptoms in scleroderma 1.

From the Research

Initial Management for Patients with Scleroderma

The initial management for patients with scleroderma (systemic sclerosis) involves a holistic approach, focusing on the dominant organ disease and addressing various factors that affect daily function 2.

• The goal of therapy is to improve quality of life by minimizing specific organ involvement and subsequent life-threatening disease.
• Treatment plans must be individualized, taking into account the patient's specific manifestations and level of disease activity.
• There is no treatment that has been proven to modify the overall disease course, although therapy targeting specific organ involvement early on can improve both quality of life and survival 2.

Organ-Specific Treatment

Organ-specific treatment is crucial in managing scleroderma. For example:

• Raynaud's Phenomenon: Calcium channel blockers are used first-line, while phosphodiesterase 5 inhibitors have also been shown to be beneficial in reducing symptoms 3.
• Digital Ischemia: Administration of intravenous prostanoids is the standard of care, and bosentan has shown benefit in preventing future ulcers in patients with scleroderma 3.
• Kidney Involvement: Scleroderma "renal crisis" is a severe complication that requires prompt treatment 4.
• Lung Involvement: Pulmonary fibrosis on chest radiograph and a forced vital capacity of <55% of predicted are indicators of severe lung involvement 4.

Timing of Severe Organ Involvement

Severe organ involvement in scleroderma patients with diffuse scleroderma often occurs early in the course of the disease 4.

• Severe skin and kidney involvement typically develop within the first 3 years.
• Severe heart, lung, and GI tract involvement also often occur within the first 3 years.
• Survival for patients with severe organ involvement is markedly reduced, emphasizing the need for close monitoring and early initiation of potential disease-modifying therapies 4.

Emerging Therapies

Emerging therapies, such as stem cell therapy, are being explored for the treatment of scleroderma 5.

• High-dose immunosuppressive therapy and stem cell transplantation have shown promise in pilot studies.
• Controlled studies are currently underway to further evaluate the efficacy and safety of these therapies.