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Scleroderma

Future Research Directions

Despite intensive study for several years, many questions about scleroderma remain unanswered. This is reflected by the fact that the mortality rate for scleroderma patients has not changed significantly over the past 40 years (Elhai 2012). However, as scientists continue to piece together the biologic framework of scleroderma, there is hope that therapeutics that target pathways leading to tissue fibrosis can be developed resulting in improved patient outcomes (Leask 2012).

Much of scleroderma research now focuses on developing ways to reduce the proliferation and/or activation of fibroblasts (Jungel 2011). Avenues being pursued include epigenetics and various methods of modifying growth and signaling pathways that lead to fibroblast activation (Gordon 2010). Unfortunately, some of these strategies have proved ineffective or only able to provide marginal benefits in clinical trials (Prey 2012).

One strategy that has shown some promise is suppression of the immune system with drugs that destroy B-cells or inhibit their production. Examples of drugs in this category include rituximab (Rituxan®) and mycophenolate mofetil (CellCept®). In preliminary trials, these interventions have shown some benefit (Le 2011; Mendoza 2012; Daoussis 2012). However, larger clinical trials are needed to more thoroughly assess the viability of this approach.

Other ongoing research efforts are evaluating immunosuppression followed by transplantation of autologous peripheral blood stem cells, and high-dose cyclophosphamide (NCT00114530; NCT00501995).