How is scleroderma treated?

Updated: Sep 09, 2019
  • Author: Sergio A Jimenez, MD, MACR, FACP, FRCP(UK Hon); Chief Editor: Herbert S Diamond, MD  more...
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Current treatment of systemic sclerosis is directed toward managing complications and providing symptomatic relief. In addition, a range of disease-modifying treatments have been investigated.

Disease-modifying treatment aims at inhibiting tissue fibrosis and vascular and immune system alterations, which are the three crucial components of disease pathogenesis. [132, 133, 134, 135, 136, 137] Therapies targeting cytokine signalling, including interleukin-6 (IL-6), and other immune-inflammatory therapies, have produced promising results. [138]

To date, the US Food and Drug Administration (FDA) has not approved any disease-modifying therapies for systemic sclerosis. In June 2015, however, the FDA granted breakthrough therapy designation to the IL-6 receptor antagonist tocilizumab, to expedite its development as a treatment for systemic sclerosis. [139]  A phase 3 trial of tocilizumab in systemic sclerosis is currently in progress. [140]

No placebo-controlled studies have demonstrated clear superiority for any drug except for a modest benefit from use of methotrexate. [141, 142] Numerous uncontrolled prospective and retrospective trials along with post-hoc analysis have suggested a beneficial effect from mycophenolate mofetil. [143, 144, 145] Retrospective uncontrolled studies also supported a beneficial role for D-penicillamine, [146] but a large high-dose versus low-dose controlled trial failed to demonstrate benefits of the higher dose versus the lower dose. [147]

Other agents are currently being studied for skin and lung involvement. For example, trials of rituximab have yielded promising results, with improvement of skin fibrosis and prevention of worsening lung fibrosis. [148, 149, 150, 151]

Phototherapy using longer-wavelength ultraviolet A (UVA) light (ie, UVA1, 340-400 nm) has proved beneficial for cutaneous lesions in scleroderma. UVA1 inhibits the inflammatory process and can soften former sclerotic skin lesions. However, data on this technique remain limited, and the most effective dose has yet to be determined. [152]

No serious systemic effects of UVA1 phototherapy have been reported. Skin pigmentation or tanning, which can persist for months, is the most common acute adverse effect; uncommon acute adverse effects include reactivation of herpes simplex, cholinergic urticaria, and transient and reversible changes in the appearance of moles. The risk of long-term adverse effects, particularly skin cancer, has not been determined. [152]

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