The Scleroderma Society is very generously funding a study investigating the safety and effectiveness of different treatments in people with early diffuse scleroderma. Diffuse scleroderma is that type of scleroderma in which skin thickening often progresses fairly rapidly and involves not only the face, hands, forearms and feet but also the upper arms, trunk and/or thighs. There is much that can be done to help people with scleroderma. For example, there are effective treatments for the oesophageal (gullet) symptoms and treatments which help Raynaud's phenomenon. However, at present there is no treatment which is known to improve or prevent progression of skin thickening in people with diffuse scleroderma.
For this reason in 2001 the UK Systemic Sclerosis Study Group (a group of doctors interested in scleroderma) set up a study to examine the effects of different treatments for diffuse scleroderma. These treatments were selected on the basis that all might be helpful, but required further study. The design of the study is 'observational'. This means that doctors select for each person what they feel is likely to be the best approach to treatment for him/her, and then monitor things carefully according to standard guidelines. Doctors from throughout the UK have
been invited to take part. Because everyone in the study is monitored in the same way, it should be possible to make some comparisons between the different treatments. It must be emphasised that this is not what is called a 'controlled clinical trial'. Some readers will have participated in controlled clinical trials when people are randomised (as in the toss of a coin) to receive either one treatment or another, but usually neither the patient nor the doctor knows which treatment he/she is on. The observational study described here is a different type of study, but nonetheless important information will be obtained.
So far 150 people with diffuse scleroderma have been included into the study. Of these 40 have been followed for 3 years. The variables which we are measuring to check up on progress include the skin score (many readers will be familiar with this, when the doctor pinches the skin at a number of different places to assess the degree of thickening), tests of breathing and of kidney function, and questionnaires about everyday living activities.
The study is ongoing. We are currently undertaking some preliminary analysis of the results so far. The study has highlighted to doctors in the UK the importance of careful monitoring of people with early diffuse scleroderma, and should indicate which treatments should be examined further. As always, our ultimate aim is to identify safe and effective treatments. Dr Ariane HerrickApril 2007