Introduction and current guidance

Introduction and current guidance

Scleroderma is an autoimmune condition that affects the skin, internal organs and blood vessels, causing scarring and thickening of the tissue in these areas. There are 2 main types of scleroderma: localised scleroderma and systemic sclerosis. Localised scleroderma is confined to the skin and underlying tissues (NHS Choices: scleroderma). Systemic sclerosis can involve internal organs, causing them to become hard and fibrous and function less efficiently (Scleroderma & Raynaud's UK: Systemic sclerosis factsheet).

There are 2 types of systemic sclerosis: limited cutaneous systemic sclerosis and diffuse cutaneous systemic sclerosis. Limited cutaneous systemic sclerosis tends to progress more slowly than diffuse cutaneous disease, although it can be associated with complications such as pulmonary hypertension. It often starts as Raynaud's phenomenon, and other symptoms include thickening of the skin over the extremities and face, red spots (dilated blood vessels) on the skin and hard lumps of calcium underneath the skin (especially the fingertips). In diffuse cutaneous systemic sclerosis, skin changes can affect the whole body. Symptoms tend to come on suddenly and get worse quickly over the first few years; then the disease settles and skin may improve. The gastrointestinal tract, heart, lungs or kidneys can be affected causing a range of symptoms such as diarrhoea or constipation, shortness of breath, high blood pressure and pulmonary hypertension (NHS Choices: scleroderma).

The aim of treatment in scleroderma is to relieve symptoms, prevent the disease getting worse, detect and treat any complications, and minimise disability through occupational therapy and physiotherapy. Because scleroderma can affect many different parts of the body, various different medicines may be needed (NHS Choices: scleroderma).

The British Society for Rheumatology (BSR) and British Health Professionals in Rheumatology (BHPR) published a guideline for treating systemic sclerosis in 2016 (NICE accredited). These advise that:

  • People with early diffuse cutaneous systemic sclerosis should be offered an immunosuppressant (methotrexate, mycophenolate [see the evidence summary on mycophenolate for scleroderma] or cyclophosphamide), although the evidence base is weak.

  • Some people might later be candidates for autologous hematopoietic stem cell transplant.

  • Skin involvement may be treated with either methotrexate or mycophenolate. Other options include cyclophosphamide, oral corticosteroid therapy (in as low a dose as possible to suppress symptoms, and with close monitoring of renal function) and possibly rituximab. Azathioprine or mycophenolate should be considered after cyclophosphamide to maintain improvement in skin sclerosis and/or lung function.

None of the immunosuppressants recommended for consideration in the BSR/BHPR guideline are licensed for use in people with scleroderma and use of any of these medicines would be off‑label.

NHS England is developing a clinical commissioning policy on Rituximab for connective tissue disease with interstitial lung disease. This evidence summary considers rituximab for treating skin involvement in systemic sclerosis: evidence for rituximab for treating lung involvement in systemic sclerosis is not discussed.