A trial of different corticosteroid regimens in Duchenne muscular dystrophy and a call for patient partners.

For many years a major obstacle to the standardisation of care in Duchenne Muscular Dystrophy (DMD) has been indecision about what corticosteroid regime is best to use and how to best prevent side effects. This is a difficult area to decide as the balance as to what is best for any individual patient needs to take into account not only how much benefit the drugs are giving, but also what the side effects are. No randomised controlled trial has yet been done to compare different regimes head-to-head, and unfortunately the long term results from cohorts of patients in different clinics are often uncontrolled and therefore cannot give definitive data on which regime is “best”.

It is over 20 years since steroids were shown to benefit DMD (they prolong the ability to walk as well as increase respiratory function, resulting in much less need for spinal surgery, and also stabilise cardiac function) but there remain clinics around the world that do not use steroids, and amongst those who do there is a bewildering range of different regimes in use. While the recently published care guidelines for DMD and the Cochrane review do suggest that daily steroid regimes have the greatest weight of evidence behind their use, the decision on whether to use daily prednisone or deflazacort, or if an alternative regime might in the long run be better, remains an open question.

FOR-DMD (Finding the Optimum Regimen of Corticosteroids for DMD) is a trial of different steroid regimes that has now been funded by the NIH. It compares daily prednisone and deflazacort regimes against one of the more commonly used alternative regimes (10 days on and 10 days off prednisone). The trial is led by Dr. Robert Griggs from the University of Rochester in the USA and Prof Kate Bushby from Newcastle University in the UK, linking the Muscle Study Group and TREAT-NMD network in this joint initiative.

The first patient was recruited to the study in January 2013. 300 children aged between 4 and 7 years, who have not previously been on steroids, will be recruited and followed for at least three years. The study includes a standardised, systematic process to minimise and control side effects, and the drugs will be judged not only on their ability to improve muscle function but also how acceptable the side effects are. The trial is carried out across more than 40 sites in five different countries (UK, Germany, Italy, US and Canada).

Patient advocacy groups have been an integral part of the process for the planning of the FOR-DMD steroid trial, including pushing for the study in the first place, acting as members of the planning committees, and assisting in the preparation of the grant. The first two planning meetings for FOR-DMD were kindly supported by the ENMC as part of its clinical trials programme.

Since the study has started ENMC  is supporting and advertising recruitment to ensure the success of this trial so that we can plan for the timeliest translation possible of the results into clinical practice.

We would like to invite patients and advocacy groups to become FOR-DMD patient partners. We welcome any suggestions or advice in particular on how we can work together on ways to maximise recruitment and retention through a co-ordinated publicity campaign.

If you are interested in discussing these roles further, or for any additional information please contact Shelley O’Rourke  phone: + 44-191-222-7623.