New drug trial for serious blister disease

June 20, 2008

It's itchy, very painful and potentially fatal but a new clinical trial by University of Nottingham scientists is hoping to give sufferers of a rare skin condition a safe and effective treatment at last.

Bullous Pemphigoid (BP) is a serious skin disease which leaves elderly people covered in large skin blisters, at risk of infection and, in around 40% of cases, death. Currently treatment is by oral steroids. Although steroids are usually very effective at getting rid of the blisters, their long term use can have serious side effects like high blood pressure, diabetes, osteoporosis and infections. But now researchers have been awarded nearly £800,000 to fund an international clinical trial of the effectiveness and safety of a simple oral antibiotic and anti-inflammatory drug, doxycycline. This antibiotic is a member of the tetracycline family, and previous reports have suggested that tetracyclines may work for pemphigoid but with far less risk of serious side effects.

The five year-long 'BLISTER' Study is being coordinated at the University of Nottingham by Professor of Dermatology, Hywel Williams, in collaboration with Professor Fenella Wojnarowska from the University of Oxford who is an expert on blistering diseases. Professor Williams and his team are seeking 250 bullous pemphigoid sufferers in the UK and Germany to take part in the randomised trial in which half will be treated with the current standard steroid drug prednisolone and half with the doxycycline. Each volunteer will be monitored for a year to see whether doxycycline works as well as prednisolone, but with fewer side effects.

Reacting to the substantial financial award of £796,770 from the National Institute for Health Research Health Technology Assessment (NIHR HTA) programme Professor Williams said,

“It is wonderful that the NHS has not turned its back on elderly people with pemphigoid just because it is a less common condition. Pemphigoid can be a serious disease, and telling a family member that we don't know how best to treat it because it is rare is of little comfort. If tetracyclines turn out to be useful for people with pemphigoid, then this could have huge implications for the way it is managed in developed and developing countries since the medicine is widely available at low cost.”

Bullous pemphigoid is an autoimmune disease which mainly affects people over sixty and is more common in the over 80s, and is on the increase in the UK. It happens when the immune system starts to malfunction and makes antibodies which attack the membrane between the top layer of skin, the epidermis, and the dermis underneath. Itchy patches appear and then tight, watery or blood-filled blisters which leave skin raw and sore when they burst, and open to infection. It's quite rare, affecting seven in a million people, yet mortality rates remain the same now as they were sixty years ago.

The BLISTER Study investigators will note each participant's condition before the course of drugs and monitor any changes or improvements after six weeks. For the important safety data, they will also record and monitor any side effects for a year after starting the trial.

Source: University of Nottingham