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Progress Continues in Systemic Sclerosis

Daniel E. Furst, MD, Maureen D.Mayes, MD, MPH, Dinesh Khanna, MD,James R. Seibold, MD, Rajeev Saggar,MD, and Keith M. Sullivan, MD  |  Issue: March 2010  |  

Conclusions

Important advances have been gained in our understanding of the genetics, pathobiology and treatment of severe SSc. While SSc is often said to be the most fearsome of the autoimmune diseases, molecular targets for therapy have been defined and pilot trials of targeted therapies are underway. Convincing animal and human data demonstrate that the immune repertoire can be reset after hematopoietic stem cell transplantation leading to dramatic reversal of dermal fibrosis and stabilization to improvement of pulmonary disease. A pivotal National Institutes of Health–sponsored randomized trial comparing immunosuppression to immunoablative HCT is currently underway and patients are being recruited to the SCOT trial to test outcomes in these two promising treatments. Such trials will provide basic scientists with a better understanding of the regulatory pathways of SSc and rheumatologists with a context to weigh progress in the treatment of scleroderma.

Acknowledgments

This work was supported in part by NIH award AI-05419. The authors have nothing else to disclose.

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Dr. Furst is professor in the division of rheumatology at UCLA; Dr. Mayes is professor in the division of rheumatology at the University of Texas in Houston; Dr. Khanna is assistant professor of rheumatology at UCLA; Dr. Seibold is professor of rheumatology at the University of Connecticut in Farmington; Dr. Saggar a clinical instructor in the division of pulmonary and critical care at UCLA; and Dr. Sullivan is the James B. Wyngaarden Professor of Medicine in the division of cellular therapy at Duke University Medical Center in Durham, N.C.

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