I have just attended an International task force meeting on disability outcomes in MS; the main attendees were clinicians working in MS (trialists), several of the MS Societies (US, UK and Canada), ECTRIMS, industry and more importantly the FDA, EMA and Health Canada (regulatory agencies responsible for licensing drugs). Unfortunately there were no people with MS attending. It was clear that we need to establish better outcome measures to capture MS disability. Several problems were highlighted with the EDSS and MSFC (multiple sclerosis composite), the main outcome measure we use today. The main conclusion of the meeting was that we need to improve what we have, i.e. make the EDSS better and to add components to the MSFC to make it clinically meaningful. At the moment the MSFC measures cognition (PASAT), upper limb function (9-hole peg test) and lower limb function (25-ft timed walk). It is very important that the outcome measures we use and develop have clinically meaningfulness; this is particularly important for the FDA. Patient related outcome measures (PROMS) were relatively short changed in that they are probably the easiest way to get PwMS engaged with monitoring their own disease and are clinically meaningful by design as they get to the core of the issue of disability. I was particularly impressed by the concept of item banking; this is a bank of many questions or mini outcome scales that assess many different functions. PwMS create their own personalised outcome scale from items relevant to their life, for example running, playing the piano, memory, writing, working, etc. This scale is then used longitudinally to track the impact of MS over time. Another idea that was floated was the use of smart phones to collect this data via mini apps. The disappointment for me personally was how little longitudinal data we have on body fluid biomarkers and the reluctance for the group to accept serial lumbar punctures to monitor the disease. How do you feel about having annual lumbar punctures to monitor your disease?
“All in all a very good meeting; I am glad I attended.”
Very interesting, thank you. Item banking is an excellent idea, though presumably only of use longitudinally.Re the lumbar punctures – I've only had one, and had no problems with it. I know a lot of people experience a great deal of pain and discomfort though. I think you might find problems recruiting PwMS for serial LPs.
Still too much time spent on refining scales for measuring fatigue, spasticity etc etc.The real need is to understand the mechanisms behind tissue damage and develop treatments to stop / reverse them. Once this is achieved the various scales will become irrelevant.Are MS patients interested in EDSS, other scales – NO.Are patients interest in stopiing / reversing progression – YES. Moving things from benchside to bedside needs to be speedied up. The current academic approach to MS slows things down. In 2011 we should be talking of getting patients better / improving their future not about refining the measurement of the impact of this disease on diufferent functions.
Please explain how serial lumbar punctures will be useful. I'm asking because I have never had one (4 years since diagnosis, 3+ years on Betaferon, now on Tysabri)