Patient led measure of outcomes

[bobbler]

Thinking about how we can measure if treatments work. A lot of the questionnaires and scales seem to try to compare across subjects, which is really difficult for many of us, they don’t fit our experiences or severity. So people try to capture a range of experiences and we end up with huge long questionnaires. So how do we measure outcomes?

How about this
- Pick and describe in your own words 5 activities that you feel define your current limitations, include 2 things things you can only occasionally do
Examples: get to the toilet in the morning, sit up comfortably throughout the day, have a 5 minute conversation with someone , have a shower, walk to the car (I don’t know I haven’t done those last two for years but you get the idea, the usual sort of things we see on questionnaires, but defined by the patient)
- Count how many days you can do these per month, before and after interventions, without negative impact, record weekly
- Maybe add a measure of how many days are ‘good’ ‘average’ or ‘bad’ for you, record daily

This would be person specific but capture the changes which are relevant and/or important to them and how their ME/CFS affects them. It would be quicker than most things to record but I think would allow measurement of if an intervention has actually worked.

Probably needs some refinements but…thoughts?

I've just re-read this. And have picked up on the bit in bold in this:

Count how many days you can do these per month, before and after interventions, without negative impact, record weekly

And I think that is an issue. Because almost everything has a negative impact, I just have to make some wicked automatic mental calculation of pros vs cons that sort of combines in with everything else to become eg how much I can brush teeth, shower etc. almost certainly the same with time lying on back or not vs boredom.

Is the negative impact bit important?


I think I'd really struggle with assigning how many days were good, ave or bad or labelling most days that aren't terrible as something.

 

[NelliePledge]

yeah I agree there are 2 aspects 1- managing to do the activity at all, and 2-=the consequences of doing the activity.
But I think the frequency is a rough proxy for the consequences because if they were less I would go out more often.

I think without negative impact doesn’t quite work because when I go out there’s always negative impact just less or more.

 

[Kitty]

Is the negative impact bit important?

Yes it's crucial, but if we do a good enough job of coming up with revealing activity categories, and recording them objectively—i.e., things we actually did, not thought we could probably do—it should show up in long term patterns.

It won't show that we were feeling really crap when we did something, but that crapness will influence what we chose to do in the following days, the following week.

 

[hotblack]

I wonder if the people at visible would be willing to engage with this @hotblack might be worth asking

That’s an interesting idea

Is the negative impact bit important?

Good question and I understand the mental gymnastics and difficulty of capturing this. I’m not sure what the best way is, hence proposing some ideas and seeing if they stand up or can be improved.

I think I phrased it better with ‘significant negative impact’ but maybe ‘more negative impact than usual’ could work? And of course this is all subjective, so what these phrases mean is up to an individual.

My thinking was the difference between something like getting to the loo when you manage it and when it is like hell (see the examples I gave here). So in this way we could capture when an activity you regularly do or have to do becomes harder. Because that could be important and people often say capturing it is.

Maybe you’re right and it’s not important. And just are you doing the thing is what we want to capture. It will depend upon what activities people choose I guess?

Kitty puts this bit well

It won't show that we were feeling really crap when we did something, but that crapness will influence what we chose to do in the following days, the following week.

 

[Suffolkres]

'A simple tool like the disability scale we use being confirmed so if someone is more severe it’s calibratable to eg other serious illnesses..
In 2018, we put some suggestions leaflets etc for our Suffolk GP AGM and exhibition.

An A4 symptom scoring chart was part of this and has proved useful for us at home, to monitor progress, fluctuations and symptom shift over time.

Our banner was taken to RCGP AGM in Glasgow by Nigel Speight for lecture before 250 zGPs, on behalf of 25% Group Tymes Trust and Suffolk Youth & Parent Support Group.

Used by NI Groups, Joan and Tom modified, and Helen in Scotland.

We did it as a pack to raise awareness and streamline process of;

GP as first post of call, referral and diagnosis, severely and symptom Identification and monitoring over time.

It was endorsed and authorised by the CCG to assist with commissioning and direction of future service fulfillment and patient expectations plus Joint Norfolk and Suffolk Health Overview and Scrutiny recommendations.

It was the basis for our journey to where we are now, or will be on 1st August hopefully.

Bearing in mind it was pre NICE NG206.
But we never had that CBT GET problem in Suffolk who embraced CIC .....

We had Jo Edwards give material the once over too!
I will make the pack material available.

It might be worth resurrecting some of it?

 

[Kitty]

An A4 symptom scoring chart was part of this and has proved useful for us at home, to monitor progress, fluctuations and symptom shift over time.

I agree it would be useful, and if the symptoms monitored are tailored to the individual it would make record-keeping quite nimble. I wonder if it could be got down to a list of three or four? Not necessarily the most severe or troublesome symptoms, but the most revealing of how you're functioning.

It could fit in with the suggestions I've been making for monitoring of the effects of a treatment on activity. The idea is to come up with as short a list as possible of ordinary daily activities, which will show trends over time rather than day-to-day change.

It might go something like this:

• Three things you do at least once a week, even in a crash. (Regular ticks indicate no change, no ticks indicate substantial worsening)
  
  
• Two things you usually do at least once a week, but can't do in a crash. (No ticks indicate worsening)
  
  
• Two things you need to avoid doing more than once a month. (Frequent ticks indicate some improvement)
  
  
• Two things you can't even attempt but would love to be able to do. (Any tick indicates substantial improvement)

The person just checks boxes to say how many of them they did this week. The less able they are, the less box ticking they have to do. Ideally, monitoring starts six months or more before the trial.

There might be something in it. It hasn't been explored much in ME/CFS, partly because the BPS theory assumes people avoid doing things they could manage if they tried. This assumes the opposite, that people do things even though they know they'll feel awful afterwards. That they'll squeeze the pips out of even the smallest gain.

It'd probably only work for a treatment that has a significant effect or none at all. The trends might be too faint to measure marginal changes, especially as they'll be confounded by factors like weather, cold viruses, etc. It'd need to be supported by a step counter, a heart rate monitor, and a simple symptom score.

 

[hotblack]

The person just checks boxes to say how many of them they did this week. The less able they are, the less box ticking they have to do. Ideally, monitoring starts six months or more before the trial.

Great ideas! I can picture how this works, more of a weekly tally?

 

[Kitty]

I can picture how this works, more of a weekly tally?

I think so. The challenge is in picking the right activities to demonstrate your "normal", but recording whether or not you did them each week should be as easy and take as little time as possible.

 

[hotblack]

I think so. The challenge is in picking the right activities to demonstrate your "normal", but recording whether or not you did them each week should be as easy and take as little time as possible.

I guess we may not even need the number of times you did them if picking the right activities and having clearly delineated groups as you outlined in your example? It may lose some resolution/detail but could be easier to fill in and still capture any changes.

 

[Kitty]

I guess we may not even need the number of times you did them

That's what I'm trying to get away from.

When you're asking whether a treatment offers meaningful improvement, it doesn't really matter how many times a week participants can do small, ordinary things.

It's whether they've moved up a whole level, and whether they can sustain it.

Whether they eventually go up two levels as their stamina improves, and whether they can sustain that.

Or whether they go down a level, of course. That's likely to happen even in a trial with a positive result, either because people really want to be responders but eventually realise they're not, or because they do too much too soon.

 

[Trish]

When you're asking whether a treatment offers meaningful improvement, it doesn't really matter how many times a week participants can do small, ordinary things.

I'm not sure about that. It makes a big difference to me whether I can shower daily or only once a week, for example. And whether I can prepare a meal from scratch with fresh ingredients every day, or about once a month.

 

[Kitty]

I'm not sure about that. It makes a big difference to me whether I can shower daily or only once a week, for example.

Yes, of course it matters to each individual. But while it would be brilliant to find a drug that makes the difference between one and three showers a week, or being propped up in bed several hours a day instead of just one, it isn't really what we're looking for. If you're still having to spend most of the time lying down it hasn't modified the underlying process.

I'd like to anticipate trialling drugs that do act on it. If they work at all they should block, break or interrupt whatever doom loop our immune systems are stuck in. It's difficult to envisage the long term result being partial gains; presumably the faulty process is either running or not, and if it's not the handbrake should be released. (I've experienced that twice, and it's extraordinary.)

To make the case for further trials or treatment being rolled out, the research team would need to be able to show that in a convincing way. Analysing what people actually did* seems much more satisfactory than asking how they felt (it's a proxy for that anyway), and it would be backed up by data from wearables.

ETA: * I should have said asking what people did in a manageable, accessible way that they'll be willing to do for a long time!

 

[Trish]

I think this illustrates the problem in ME/CFS of the cumulative effect of activities. Being able to shower or cook every day for someone who can currently only do them once a week or a month, if it implies all other daily activities are also able to be increased by that amount too, indicates a very significant overall improvement.

 

[bobbler]

Yes, of course it matters to each individual. But while it would be brilliant to find a drug that makes the difference between one and three showers a week, or being propped up in bed several hours a day instead of just one, it isn't really what we're looking for. If you're still having to spend most of the time lying down it hasn't modified the underlying process.

I'd like to anticipate trialling drugs that do act on it. If they work at all they should block, break or interrupt whatever doom loop our immune systems are stuck in. It's difficult to envisage the long term result being partial gains; presumably the faulty process is either running or not, and if it's not the handbrake should be released. (I've experienced that twice, and it's extraordinary.)

To make the case for further trials or treatment being rolled out, the research team would need to be able to show that in a convincing way. Analysing what people actually did* seems much more satisfactory than asking how they felt (it's a proxy for that anyway), and it would be backed up by data from wearables.

ETA: * I should have said asking what people did in a manageable, accessible way that they'll be willing to do for a long time!

This is where we start to diverge a bit - I really 'got' the last comment on the idea of having a set of criteria that represented a 'stepping up'

I wonder if part of the difference might be the caveat I have in my mind that the really important bit is that anything is followed up long-term and those long-term measures become the primary outcome over eg something 6months or less (because it is the excitement of doing things too much pre- this timing with something that stimulates x ability but doesn't have behind it the 'delivery of y' that makes it not damaging that would be something which one could either term a 'false flag' or actually might turn out to be something important short-term for people in desperate situations, depending on what it actually does).

If for example we are really looking at the 12month, 18month, 24month and focusing on objective activity, but including measures such as time horizontal and the tasks are indeed things like showering then I'm more OK with this

But a real downside of the history of those we get surrounded by staff-wise with ME is coercion (even including of the flavour hidden as 'motivating' and getting us to self-gaslight). I'm not sure because of this deliberate, and sometiems inadvertent by those deluded themselves that they are helping whether 'how we feel' actually compensates for that anyway in the short-medium because I know how thinking the pain=gain stuff can affect what goes on in our heads nevermind on a form etc. So agree with envisaging coming up with something that moves away from this being a necessary balancer.

PLus of course from my experience with things that genuinely made me feel better short term there was the deterioration which I'm convinced is part of what we might need to call a 'cycle of PEM' or something better descriptive to show that PEM isn't just some short term thing, but the accumulation or thinking we are managing to cheat it by riding the edges of it can mean we then take a massive dip in function say 6months in. So it is an illness of these nasty after-effects.

And we might have genuinely 'felt good'. And even done more. BUt perhaps the fact we couldn't also requisitely up the rest to go with that (because there are only 24hrs in a day and if 1hr activity needs 23hrs rest and we start feeling good doing 2hrs, is it 36hrs res or 24hrs rest we needed but the sums didn't add up over that period) caught up with us. To me I think that would probably mostly show in our function and activity rather than 'feel' still, because having a setback after having a 'holiday' comes with all sorts of complicated 'feelings' (does this term include how our body parts 'feel' without the mind getting involved is an interesting question having had different doses of steroids poses to me, because many things can change pain and feeling of strength? which can lull us into the behaviour that only causes the deterioration much later on eg many months).

But all of this absolutely hinges on this caveat of the longer-term, and that the pattern shows consistency (hence why it being measured up to 3yrs starts to make sense because if we get a virus at 2yrs that knocks us back then there is time to say we were good at 12, 18, 30, 36months)

I also think that we need to be aware that something could be 'part of the solution' and genuinely working on ME/CFS. But only part. And whilst we assume that no damage is done that will persist for all, it might. So we need to barter with oureslves a bit I guess at what the level of improvement is that says 'wouldn't want this taken away' vs 'could be tricked' and of course that level needs to be clearly labelled so that we don't end up with people again getting away with suggesting something is a cure and we should be fixed and 'acting normally' when it merely gives us back a massive chunk of quality of life eg being able to shower once a week vs once a month, sitting up for 3hrs vs 1hr is huge.