Scleroderma or systemic sclerosis is a rare condition with many clinical manifestations including Raynaud’s phenomenon. As with many other rarely encountered diseases, drug therapy for scleroderma is often empirical with little evidence in the form of randomised controlled trials to aid drug choice.
Raynaud’s phenomenon has been recognised for well over 100 years. A considerable number of clinical trials in this area have demonstrated unequivocally the use of nifedipine as a gold standard. Large studies have also demonstrated the efficacy of iloprost. However, this drug is not as yet licensed for scleroderma in the UK or elsewhere. This presents an additional problem as information regarding the use and administration of unlicensed drugs is often sparse and post-marketing surveillance to assess safety is not routinely performed.
When looking at the other distinct conditions encountered by a patient with scleroderma it becomes evident that trials are often retrospective or limited in patient numbers. Studies investigating the use of methotrexate, antithymocyte globulin and cyclophosphamide in patients with scleroderma have been very small and in some cases not well designed. The major work on penicillamine was a retrospective trial. Again these drugs are not licensed for use in scleroderma.
Drug therapy for pulmonary hypertension secondary to scleroderma closely follows that outlined for primary pulmonary hypertension. In the US there is a patient registry for primary pulmonary hypertension that has enabled well designed, large-scale studies to demonstrate the benefits of epoprostenol in severe primary pulmonary hypertension. Hence, research in this area has progressed considerably over the last decade.
Clearly, a considerable amount of work is being carried out to elucidate new treatment regimens for scleroderma, however, evaluation of these studies is proving to be a difficult process. Designated hospital centres for scleroderma (there are currently 2 in the UK), better markers of disease activity and methods to measure improvement or deterioration in affected organs, should enable research into aetiology, disease progression and treatment to be carried out on a larger scale resulting, hopefully, in more conclusive answers.
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