More effective treatment for systemic sclerosis
Systemic sclerosis is a rare autoimmune disease associated with the destruction of connective tissue, leading to vascular damage and fibrosis in various organs. Like many rare diseases, treatment of systemic sclerosis entails off-label drug usage to modify the immune response, a practice that lacks evidence from controlled trials. The EU-funded project DESSCIPHER (To decipher the optimal management of systemic sclerosis) has provided evidence for the efficacy and safety of different preventative measures and therapeutic treatments. Researchers carried out five observational trials (OTs) addressing the effect of off-label drugs during different phases of the disease, ranging from digital ulcers and arthritis to severe morbidities such as severe heart disease, interstitial lung disease and pulmonary hypertension. Data from more than 4600 patients, who received off-label drug treatment in a real-life setting were analysed and compared to the deleterious course of the disease under different therapeutic regimens. This task is being achieved also through collaboration with the pan-European EUSTAR group (EULAR Scleroderma Trials and Research group), which includes more than 190 expert centres across Europe, as well as the patient organization Federation of European Scleroderma Associations (FESCA). Of the five DESSCIPHER large-scale observational trials, four could be realised through the complete funding period, and owing to long-term nature of the treatments, the DESSCIPHER partners and contributing EUSTAR centres will continue the follow-up of the remaining four OTs beyond the project end under the combined umbrella of the EUSTAR network and World Scleroderma Foundation up to November 2017. After their completion, the project data will be merged with that of the EUSTAR cohort to become the largest available clinical database of systemic sclerosis. The DESSCIPHER study has already, and will continue to provide scientific evidence for key clinical questions and problems associated with systemic sclerosis. The work will also facilitate official use of drugs that are currently off-label. Ultimately, implementing this newly gained knowledge will lead to novel clinical guidelines for the effective diagnosis and management of systemic sclerosis patients.
Keywords
Treatment, systemic sclerosis, observational trial, autoimmune, off-label drugs
