Building an evidence base for management of severe ME (including sleep management)

Surely we should look to what personalised, palliative care approaches bring to this situation?
That in effect is what is required for the severely affected.
A management and oversight that flexibly responds to the situation as it presents itself?
Similarly a contingency for acute needs at any point tailored with a palliative, personalised approach?

This information could be gleaned (by NICE and others) from, specialist health practitioners and medics in these settings.

What evidence exists from medical notes about caution and approaches that work from ME patients when they have to be managed for another(potentially life threatening situation), like cancer, pneumonia, lymphoma surgery in acute secondary care?

 

I think there's a risk that the advice that deviates the most from what people are already doing or would instinctively do, will be seen as the intervention, the advice that is designed to be the active test. For example, if you give one group the advice: "sleep as many hours as you feel you need", and another group: "try keeping a regular sleep schedule" or "avoid sleeping during day time" then the latter has more chance to be seen as the intervention group because it gives the impression that scientists have an assumption why this will be beneficial.

Another problem I see is that if you take ME/CFS patients who've got ill and the trial only follows them for a couple of months, then I think that activity or sleep management will have very little influence on the progression of their illness. I think the usefulness of these kinds of advice will only become evident in a longer time period.

 

Yes, it can be passive. There is no need to expend energy. Just to go through range.

 

Maybe you have never seen a child with arthritis sitting in a buggy.

 

That would be good and it is what the professionals say they do. But in terms of hard evidence how do yo do controlled trials on individuals - it is impossible by definition. The problem with 'personalised care' is that it is always theory drive - you theorise what would suit this person or that.

You can glean information from professionals but how do we know it is any good? If I was asked what was advisable for they thousands of people with rheumatoid arthritis I looked after I would have to be honest and say iI did not really know because I was never in a position to do controlled studies.

 

I have not. I have seen children handicaped from polio.

 

You can get a (probably clear) pattern from observation alone. I agree that sometimes observation doesn't give enough information that is needed to find a pattern.

In the first step the aim is not to help (with the exception of severe ME where basic help is required), but to observe to possibly find a pattern. In the second step, this pattern can be tested. Everything else is guessing (as you say) and most possibly harmful for a part of participants.

I also think ME is not a lifestyle problem - I know you don't think that either. Lifestyle changes won't "be it". It's maybe the wrong focus at this point of time (no knowledge about pathomechanism, scarce information about living habits of pwME etc).

I think most people here can agree that the "lifestyle" recommendations in the guidelines or by doctors have no factual base, but are often based on ideology and prejudice, and need to go. (Better to advise nothing.) One of the major problems is, in my view, that ME is not understood and there doesn't exist a good description outside of "the community". Everything will be guessing then.
These advices may still be found in a "daily living trial", because they are common, so that information about their effectiveness may be found, too.

You can always help by listening to what a person needs; for this, no trial is needed.

 

A big problem with health professionals I've found is that because there is no evidence base they generalize:

• between the effect of activity and exercise on PwME and people with other conditions and people who are healthy
• between other patient groups and PwME with regard to toleration of dose of meds
  
• between levels of severity of ME
  
• between adults and children with ME
  
• between evidence / their professional experience for their other patients and PwME

As a result many PwME get useless or harmful advice.

So many HP's are not familiar with ME and they don't take seriously what the patient is telling them. Even in specialist clinics HP's may well be out of their depth, especially for their SA patients, but they carry on, seemingly oblivious:

• How could anyone really have so many symptoms affecting so many bodily systems?
• Exercise & activity is good for everyone, right?
  
• Sleep hygiene is good advice, right?
  
• Too much rest is bad, right?
  
• Orthostatic problems are difficult to diagnose and there is not any treatment, so there is no point in diagnosing, right?
  
• Tachycardia isn't anything to worry about, right?
  
• Your BMI is too high / low - that is the cause of your problems, right?
  
• We don't want patients to become psychologically dependent on medication to help with sleep / pain, right?

Added to that, even though HP's may not be knowledgeable themselves about they may also resistant to patients that are well informed and persist with 'generic' advice.

So what happens? Well PwME understandably do not go back to those HP's if they can avoid it and the cycle perpetuates. Not only that the specialist services that there are do not usually deliver ongoing care so there is no longitudinal monitoring of patients.

Even where outcomes are measured, and can be measured to be worsening (as has happened locally), the views of patients are not of importance. The elephant in the room is not only do we know what is actually being done and whether we know what is being done is of benefit, but also HP's entrenchment in 'management' approaches that they are currently using. Added to that CCG's cover their backsides by saying a service is NICE compliant, but that may not reflect what is actually being delivered.

It makes it very difficult to unpick what is said to be done and what is actually being delivered.

 

Presumably advice only has to adopt the status of being advice at the time it is being administered, and I suppose at the time it is being considered for regulation, guideline usage, etc. In principle you could presumably throw a number of random notions into a trial, with no one having any idea what may or may not be of any benefit (the ultimate blinding?). So, almost by definition, only once the trial results are known might something emerge that could then be deemed advice? There is obviously a catch here, because the notions I'm talking of cannot be random, though a random element might not be a bad thing. There would clearly have to be filtering to weed potential harms.

 

I agree that there is always a risk of an assumption about what researchers think is right. But a study if the sort I am thinking of would say in black and white, and maybe in bold in the information for patients that the study was being done precisely because nobody has a clue what is the right thing to do.

The sort of dissociation of tests in a trial from theory may be unfamiliar but it has been commonplace in cancer trials. Lots of cancer trials look at various combinations of more or less aggressive treatments with the researchers having no preference for which is likely to give the best survival rate - which will of course include problems with side effects.

I am very much assuming that impact of plans would need to be followed over an extended period. My first guess would be six months. Longer might be nice but might be impractical.

 

I am not sure which way you are arguing. If you are not intervening with help you cannot test for a pattern of effect. You can get a pattern of liability and symptoms but that tells you nothing about what might be worth testing to help. I agree that you might see correlations between activity and PEM but that does not necessarily indicate what policy is best longer term.

 

Johnathan,

I am confused. At this point, what are you going to measure, how and for what purpose?

 

It’s interesting to note that the current NICE guidelines for insomnia recommend sleep hygiene although they do acknowledge that there is insufficient evidence for its efficacy:

https://cks.nice.org.uk/insomnia#!scenarioRecommendation:3

Is it standard practice for NICE to recommend treatments on the basis of “expert opinion” even where there is insufficient evidence for efficacy for a particular intervention? That seems a bit of a worry to me.

 

I cannot imagine any meaningful utilitarian results without soliciting patient input, and that will be no small feat on several different levels.

 

It would be a good start.

 

The main target I see as objective evidence of reduced disability. And by disability I mean not being able to live a fulfilling life so it can cover all sorts of impaired abilities. Actometry might come in to that, as might employment and need for benefits - the things that did not change with PACE.

The purpose would be to see if any sort of plan was associated with a better or worse outcome or whether it made no difference.

 

Well we are already getting patient input. The next stage ought to be to get representative patient input from a community based cohort of patients. The UK Biobank cohort might be a start. Caroline Kingdon is constantly getting input from these people.

 

My guess is that you will find a pattern in a study that looks at everyday-life. Every being has patterns. And nearly every human being creates some day-to-day routine. My guess is, since pwME experience comparable symptoms (in different severities), that they will develop groups of similar patterns. A study on a large cohort over a longer period of time might show these different patterns.

Example: Someday during their illness, pwME will aim at preventing PEM. They will make observations about what will lead to PEM and adjust their day-to-day life.
For instance, one pattern may be that a group of pwME found that a certain (individual) amount of activity triggered PEM, so they use aids (scooter, wheelchair, carers, rest etc.) and then report stabilizing around a baseline. Some may report improvement from "very bad indeed" to baseline; some may report worsening nonetheless, some may report no change. Or another group might show an eating pattern that led to changes (better/worse) or no changes. Another group might show a sleeping pattern, like a regulated sleep plan/no sleep plan that led to improvement/worsening/no change. The changes would be measured objectively (e.g. times per day that the house was left, steps per day, ability to care for oneself, minutes per day spent reading/watching/talking etc.) and subjectively.

At this point it is just observing what kind of patterns pwME create. They do. As a byproduct one might get knowledge about whether there exist different groups like with MS (stable, relapsing/remitting, progressive, others...). One might also learn which intervention is potantially harmful. This will be relevant information when doing trials that test a plan vs. a control.

As soon as there is more knowledge about how pwME live, i.e. about their patterns, the second phase can start. A pattern (a help plan e.g.) is tested in a methodological trial.

I might think too simplified.
I think this is the aim of NIH maybe with their new study?

I think that surveys might be a source of information, too:

But they lack objective measures.

 

thanks, that helps clarify things. why are you proposing to study only severely ill patients?