Innovative therapies for systemic sclerosis

  title={Innovative therapies for systemic sclerosis},
  author={Voon H. Ong and Christopher P. Denton},
  journal={Current Opinion in Rheumatology},
  • V. Ong, C. Denton
  • Published 1 May 2010
  • Biology, Medicine
  • Current Opinion in Rheumatology
Purpose of reviewThe purpose of this study is to review the evidence and recent developments leading to novel therapeutics in scleroderma. Recent findingsRecent advances have been made in understanding the key pathogenetic aspects of scleroderma, and these have led to potential targeted therapeutic agents for the management of these patients. Preliminary data from early clinical trials suggest that tyrosine kinase molecules may be potential candidates for therapy, especially in the fibrotic… 

Molecular and cellular pathways as treatment targets for biologic therapies in systemic sclerosis.

Recent advances have shed light on the complex pathogenic processes that underlie the development and progression of Systemic Sclerosis (SSc) but management of the disease remains problematic and

Cyclophosphamide as disease-modifying therapy for scleroderma: pros and cons

An evaluation of the benefits and drawbacks of cyclophosphamide in systemic sclerosis shows that identification of appropriate patients is central to management to harness its maximal potential clinical efficacy.

Targeted Therapies in Autoimmune and Inflammatory Skin Disorders

Factors that need to be considered in the future include deeper knowledge of the pathogenic mechanisms, the identification of valuable biomarkers assessing efficacy and safety and finally the more defined characterization of optimum treatment paradigms and most appropriate patient populations for the use of new therapeutic agents.

Emerging drugs for uveitis

Future treatment of uveitis will require a better understanding of the mechanisms involved in autoimmune diseases and better delivery systems in order to provide targeted treatment with minimal side effects.

Systemic Sclerosis Pathogenesis and Emerging Therapies, beyond the Fibroblast

The state of the art of the pathogenesis of this disease is reviewed and the main therapeutic targets related to each pathogenic mechanism that have been discovered so far are discussed.

Cytokines in the immunopathology of systemic sclerosis

Novel therapies that block key mediators that drive the fibrotic response are being developed and appear as potential therapeutic tools in the treatment of SSc, highlighting the importance for an effective therapy targeted towards the molecular and cellular pathways.

Whole orbital tissue culture identifies imatinib mesylate and adalimumab as potential therapeutics for Graves' ophthalmopathy

In vitro tissue-culture model may, in future, prove valuable to test novel therapeutics for their presumed effect in GO as well as in other inflammatory diseases.

Interstitial Lung Disease Definition of Pulmonary Manifestations and Complications of Scleroderma

Several studies showed that the disease modifying drug, cyclophosphamide, can reduce the progression of scleroderma ILD in patients with early symptomatic disease with stabilization and also improve the extent of skin indurations, contributing to the changing attitude towards sclerodma patients.



Disease-modifying treatment in systemic sclerosis: current status

There is still no treatment that is well tolerated and unequivocally effective currently for systemic sclerosis, but a number of novel agents including antiinterleukin-6, transforming growth factor-β-directed therapies and other novel biological agents are being developed based on new insights into the pathophysiology of disease.

Targeted therapy comes of age in scleroderma.

Targeting activated T cells: successful use of anti-CD25 monoclonal antibody basiliximab in a patient with systemic sclerosis.

The successful use of basiliximab in combination with cyclophosphamide in a patient with severe, rapidly progressive SSc is described.

B cell depletion with rituximab in patients with diffuse cutaneous systemic sclerosis.

Treatment with rituximab appeared to be safe and well tolerated among patients with diffuse cutaneous systemic sclerosis and the potential efficacy in other organs such as the lung could not be clearly evaluated in this small open-label trial.

Efficacy of rapamycin in scleroderma: a case study.

The results provide the rationale for larger clinical trials of rapamycin in scleroderma and other fibrotic disorders and allow for rapid improvement in skin stiffness and mobility.

[Successful treatment of a severe case of idiopathic pulmonary fibrosis with rapamycin].

It is presumed that partial remission is related to rapamycin which may be effective in blocking the progressive fibrosis and increased collagen synthesis thought to be pathophysiologically relevant in this disease.

Treatment with imatinib prevents fibrosis in different preclinical models of systemic sclerosis and induces regression of established fibrosis.

Results indicate that combined inhibition of the tyrosine kinase c-Abl and PDGF receptor might be effective in the later, less inflammatory stages of SSc and for the treatment of established fibrosis.

Experience with rituximab in scleroderma: results from a 1-year, proof-of-principle study

The results indicate that RTX may improve lung function in patients with SSc, and it is proposed that larger scale, multicentre studies with longer evaluation periods are needed.

Mycophenolate mofetil in diffuse cutaneous systemic sclerosis--a retrospective analysis.

MMF is very well tolerated and appears to be at least as effective as the other current therapies for dcSSc, and provides support for further evaluation of MMF in a prospective trial.

Rituximab in diffuse cutaneous systemic sclerosis: an open-label clinical and histopathological study

Rituximab appears to be well tolerated and may have potential efficacy for skin disease in dc-SSc, and there was a significant change in skin score at week 24.