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 will test 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.
It is anticipated that the first patients will be recruited in early 2012. 300 children aged between 4 and 7 years, who have not previously been on steroids, will be recruited and followed for at least 3 years. There will be 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. It is planned that the trial will be carried out across a number of different countries – many sites have already volunteered to take part and the TREAT-NMD patient and care and trial site registries are participating in the feasibility and set up process.
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 their clinical trials unit programme.
As the grant is due to become operational very soon, we would like to broaden the participation of patients in ensuring the success of this trial so that we can plan for the timeliest translation as possible of the results into clinical practice.
We would like to invite patients and advocacy groups to become part of the FOR-DMD patient partners. We welcome any suggestions or advice but in particular, we are keen to get input on: protocol and patient information material; advice on areas of management, especially relating to weight control and behaviour management; and how we can work together on ways to maximise recruitment and retention through a co-ordinated publicity campaign. We will also be seeking to recruit a patient representative for the Data and Safety Monitoring Board. If you are interested in discussing these roles further, or for any additional information please contact Shelley O’Rourke.