A study of prednisolone therapy in patients with an early subtype of systemic scleroderma

Disease - Scleroderma

Lead applicant - Professor Ariane Herrick

Organisation - University of Manchester

Type of grant - Clinical Studies

Status of grant - Active

Amount of the original award - £367,593.95

Start date - 1 April 2016

Reference - 21021

Public Summary

What are the aims of this research?

Scleroderma, or systemic sclerosis, is a rare autoimmune condition that can cause skin thickening that can spread to other parts of the body and involve the internal organs. For a small number of people this can cause severe and potentially life-threatening complications such as scarring in the lungs, high blood pressure and kidney problems.

Diffuse cutaneous scleroderma is a specific type of scleroderma where patients experience skin thickening that rapidly spreads from the hands and feet to involve the arms, legs or trunk. This type of scleroderma is associated with an increased risk of severe and life-threatening complications. The aim of this research is to investigate whether treatment with the steroid prednisolone is beneficial, in terms of safety and effectiveness, in patients with early diffuse cutaneous scleroderma.

Why is this research important?

Diffuse scleroderma is a fairly rare condition with around five people per million developing the disease each year, however it has devastating impact on those affected, including an increased risk of serious complications, death and inflammation causing severe pain, itching and impaired upper and lower limb function.

Prednisolone is a steroid treatment which can help reduce inflammation and relieve severe pain, itching and disability however due to its possible side effects doctors are often reluctant to prescribe it. This clinical trial, involving treating patients with either prednisolone or placebo therapy for six months, should provide doctors with a long awaited answer about the safety and effectiveness of using this drug.

How will the findings benefit patients?

If prednisolone treatment reduces disability and pain in early diffuse scleroderma, without unacceptable side effects, then this will have a major impact on the quality of life of those affected. This short term study of the efficacy and safety of prednisolone use could lead to longer term studies, examining prevention of any potential side effects.