Emerging treatments

Mycophenolate

There is an ongoing phase 2 trial studying the effects of mycophenolate for improvement of skin and function in limited cutaneous systemic sclerosis.[48]

Treprostinil

This prostacyclin analogue has been trialled for the treatment of Raynaud's phenomenon and/or digital ulcers.[49][50] In one dual-centre, open-label, phase 1 pharmacokinetic study, oral treprostinil was effectively absorbed in 19 patients with scleroderma and was temporally associated with improved cutaneous perfusion and temperature.[51]

Targeted antibodies

There have been studies targeting tumour growth factor (TGF)-beta and other profibrotic signals.[15][52] One phase 1 open-label, multi-centre, cohort dose-escalation trial with AVID200 - a novel, potent TGF-beta receptor inhibitor - has posted initial results that the drug is well tolerated in this first study in patients with diffuse cutaneous systemic sclerosis (dcSSc).[53][54]

Rituximab

Preliminary studies of rituximab demonstrated improvements in skin and lung function.[55][56] One 2021 systematic review and meta-analysis concluded that rituximab is a promising strategy for the systemic sclerosis-associated interstitial lung disease and cutaneous fibrosis treatment.[57]

Imatinib

There have been suggestions that systemic sclerosis may improve with use of tyrosine kinase inhibitors such as imatinib.[58][59][60] One small study found that the tolerability of imatinib was poor in treating active (mostly early) dcSSc, but another phase 2a, single-arm, open-label trial found that imatinib was tolerated by most of the 24 patients with dcSSc.[61][62]

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