What research do you want to see? (study ideas)

[Inara]

Studies replicating Dr. David Systrom's invasive CPET studies should also be done.

Is this planned by vanElzekker?
https://because.massgeneral.org/cam...gue-syndrome-exercise-challenge-study/c168141

 

[Alvin]

A discussion elsewhere and my thread have led to this question i think should be researched. Would people diagnosed with ME/CFS stop progressing if they stayed out of PEM. Of course people are diagnosed at all stages since most visit many doctors over many years before being diagnosed but for mild/moderate/severe it would be very useful to find out. Its is a piece of the puzzle we don't actually have a concrete answer to. This research would take years and have to be very well thought out before starting.

The reason i think this is important to study is that it may lead to a way to stop progression if answered correctly, if all we need to do is stay out of PEM at all costs and we will remain static then thats an important piece of symptom and disease management. Also part of the study would be patients who could stay well below PEM in case close to the threshold is enough to cause progression.
And its the exact opposite of GET/CBT, if it were proven that avoiding PEM will prevent progression let the PACErs argue with their shadows while their credibility takes a very hard hit.

 

[Sly Saint]

Would people diagnosed with ME/CFS stop progressing if they stayed out of PEM.

This research would take years and have to be very well thought out before starting.

By progressing, you mean deteriorating(?)
I don't think you could research this ethically; ie have a 'control' group who are advised not to pace themselves and stay within their energy envelope (ie like all the PACE style research effectively does by getting people to increase their activity even if they suffer for it).

But it would be good if someone were to somehow put together all the evidence (ie those using objective measures) to clearly show the energy envelope theory (see Jasons study which unfortunately only seems to use questionnaires). Nancy Klimas (see video on other thread) has done work on this and Lucinda Batemans group also.

 

[Alvin]

By progressing, you mean deteriorating(?)

Yes

I don't think you could research this ethically; ie have a 'control' group who are advised not to pace themselves and stay withing their energy envelope (ie like all the PACE style research effectively does by getting people to increase their activity even if they suffer for it).

I agree but this is an important question to answer, if we can stop deterioration by proving its caused by PEM episodes then that will improve many patient's lives until there is a treatment/cure.
It may be more ethical to do in depth surveys on patients then follow up later, how often do you get PEM, for those who have prevented it vs those who have not, and compare rate of deterioration.
Ideally we would design a study that did well below PEM, just under and PEM induced on differing timeframes but as you say that would not be ethical (though somehow its ethical to use GET according to many "experts" which is still being done...)

 

[Rick Sanchez]

Finally found this thread!

Not sure if this is possible / ethical or whatever.

But I would like to see a study with 3 different groups.

A group of people with short term ME/CFS (who have had the illness less than 3 years)
A group with long-term ME/CFS (longer than 3 years)
A control group of healthy people.

Infect the patients with the most benign sort of infection possible (something like the common cold I guess?)

Measure the patients immune system with the current technology we have and take samples from the patient

-Prior to the infection
-During the infection
-Post infection

Patients would be mild / moderate. No patients close to severe for obvious reasons.

The Mady Hornig study showed that there were significant changes in the immune system of ME/CFS patients depending on the length of the illness. Overactive immune system >> Immune exhaustion. So would be interesting to see how this played out with a benign infection, especially with comparison to healthy control.

I love the idea of researchers having started to use something like exercise to induce PEM in patients in their research. But I actually think measuring patients while they are fighting an infection would give even more interesting results.

 

[Andy]

To resurrect this thread.

I would suggest that studies that assesses the level of impairment in patients on a large scale, i.e. physical and cognitive impairment, in a way that helps support patients access state support and insurance payouts, could make a huge difference to patients lives. If there was solid evidence presented in the printed literature that, if need be, a patient could use to back up their claim for state/insurance support then far more patients would be able to live more securely and healthily.

 

[Adrian]

To resurrect this thread.

I would suggest that studies that assesses the level of impairment in patients on a large scale, i.e. physical and cognitive impairment, in a way that helps support patients access state support and insurance payouts, could make a huge difference to patients lives. If there was solid evidence presented in the printed literature that, if need be, a patient could use to back up their claim for state/insurance support then far more patients would be able to live more securely and healthily.

I think that is an interesting idea. I wonder if there could be an addition to the DecodeME project (with separate funding) that looked into these issues taking advantage of the cohort that is being developed (and even diagnostic information to get agregate levels and types of impairment).

I feel that any study should have a heavy involvement of patients to help direct into what is really impairment (why and how symptoms really causes impairments in activities).

My other thought was is this something that a patient collective could do - survey patients, collate and write up results (along with some academic support).

 

[Andy]

I think that is an interesting idea. I wonder if there could be an addition to the DecodeME project (with separate funding) that looked into these issues taking advantage of the cohort that is being developed (and even diagnostic information to get agregate levels and types of impairment).

I feel that any study should have a heavy involvement of patients to help direct into what is really impairment (why and how symptoms really causes impairments in activities).

My other thought was is this something that a patient collective could do - survey patients, collate and write up results (along with some academic support).

Yes, to all those suggestions.

To clarify the first one, that could either be a project by the DecodeME team, or a separate researcher/team. We want the cohort and/or data that is built up to be as widely used and useful as possible (subject to any applications for use of both having appropriate ethical and other necessary approval of course).

 

[Milo]

To resurrect this thread.

I would suggest that studies that assesses the level of impairment in patients on a large scale, i.e. physical and cognitive impairment, in a way that helps support patients access state support and insurance payouts, could make a huge difference to patients lives. If there was solid evidence presented in the printed literature that, if need be, a patient could use to back up their claim for state/insurance support then far more patients would be able to live more securely and healthily.

Biomarkers. We need biomarkers. And while disability support is much needed as it is difficult to prove you are disabled, we need to be careful in my view to not encourage a for-profit test that is not well accepted in the medical community. Ideally a Widely available test that does not rely on self-report, and a test that both diagnose and measure the impairment and doesn’t require anyone to mortgage their home, go hungry, or send their blood to Germany.

 

[Andy]

Biomarkers. We need biomarkers. And while disability support is much needed as it is difficult to prove you are disabled, we need to be careful in my view to not encourage a for-profit test that is not well accepted in the medical community. Ideally a Widely available test that does not rely on self-report, and a test that both diagnose and measure the impairment and doesn’t require anyone to mortgage their home, go hungry, or send their blood to Germany.

Yep, I'm not arguing against a biomarker, but we don't how long that will take, or even if we will ever get one.

So my suggestion is to cover the time in between, and actually when a biomarker emerges, it will be support for what that biomarker means. There is no point to a biomarker if there is insufficient literature to support the level of disability reported by patients. And if we have convincing literature that supports the level of disability reported by patients then it might well improve the chance of more funding for a biomarker, and/or other biomedical research.

 

[Trish]

And if we have convincing literature that supports the level of disability reported by patients then it might well improve the chance of more funding for a biomarker, and/or other biomedical research.

I wonder, on level of disability, whether it might be possible for the DecodeME project to include a simple one click question that asks participants to choose where on either the MEA or Bell scale their current function is best reflected.
MEA Disability Rating Scale
Link to scale
Bell Disability Scale
Link to Scale

 

[Kitty]

Couple of thoughts.

1) Should we use 'impairment' as well as – or instead of – 'disability'? Without getting into a long and technical discussion about usage, 'disability' is usually taken to mean socially-imposed barriers, and 'impairment' to indicate an individual's physical, sensory, and developmental differences. I argue that we suffer from both, but that may not universally be accepted.

2) Do we need to start with our own categories in order to reflect the effects of an energy limiting chronic illness? By this, I mean something like how it affects our ability to carry out:

Basic self care (eating, toileting, washing, dressing, etc)
Broader self care (accessing housing, food & clothing, banking & finance, medical care, benefits, transport, running a home, etc)
Work or post-16 study
Partner relationships
Children and family life
Friends
Social life
Interests and recreation
Physical activity

(ETA: the above are not in a suggested order of priority)

 

[Kitty]

More thoughts – or, more accurately, questions.

We've established that it's fiendishly difficult to assess our level of function due to the fact that it varies from hour to hour and day to day, and over longer cycles such as months and years. Plus, of course, our success or otherwise in managing PEM, and the effects of co-morbidities and increasing age.

In the UK, if you apply for Personal Independence Payment or PIP (one of our main disability benefits), an important principle is whether an impairment affects you for more than 50% of the time. Provided it does, it can be regarded as affecting you all of the time, and can therefore be considered as part of the application.

Is this principle useful in trying to establish an individual's broad level of function, or would something else work better?

When we're making our assessment of our own function, what period of time should we consider? An average over the previous 12 months? The last three years?

 

[Adrian]

2) Do we need to start with our own categories in order to reflect the effects of an energy limiting chronic illness?

I think one of the important things is that ideas of what imparements matter are driven from patients (and carers) rather than any academics. That way the things that really matter come across - also temporal issues around imparements need to be raised (for example, the inability to plan to see friends not knowing how well you will be on a given day; or the effects of PEM being one activity a week etc)

 

[Adrian]

In the UK, if you apply for Personal Independence Payment or PIP (one of our main disability benefits), an important principle is whether an impairment affects you for more than 50% of the time. Provided it does, it can be regarded as affecting you all of the time, and can therefore be considered as part of the application.

Is this principle useful in trying to establish an individual's broad level of function, or would something else work better?

Given things like pacing and PEM I've always thought the 50% doesn't represent a good concept. It doesn't seem to cover the notion of an imparemtent in the form of if I do X then I won't be able to do {x,y,z,....} for a time t (with bounds) but if I don't do i where i is from {x,y,z,.....} then I have the option of doing i at most days in the future.

 

[Kitty]

It doesn't seem to cover the notion of an imparemtent in the form of if I do X then I won't be able to do {x,y,z,....} for a time t (with bounds) but if I don't do i where i is from {x,y,z,.....} then I have the option of doing i at most days in the future.

I guess it depends how you look at it. This phenomenon affects me (and probably most ME patients) all of the time, so the 50% rule automatically applies anyway.

The rule may be irrelevant in any case, I was just trying to think of what general principles are required. I made a huge batch of curry yesterday, though, after washing, peeling, and chopping mounds of veg the previous day, so my brain wasn't necessarily functioning as well as it might!

I think one of the important things is that ideas of what imparements matter are driven from patients (and carers) rather than any academics.

Absolutely.

 

[DigitalDrifter]

I'd like to see research exclusively using genuine ME patients (those who have an adverse reaction to exercise and exertion). Outcome measures for treatments should include PEM thresholds, PEM duration per equivalent exertion, and actimeters where appropriate. All adverse effects and drop outs should be reported. Useless or flawed scales such as the Chalder Fatigue Scale and SF-36 should be abandoned.

All data should be freely available to all.

 

[dreampop]

Biopsy studies, I used to be interest in what a brain biopsy would show but I wonder if that's really useful if the illness is a signalling problem. I know Hartford OMF planned muscle biopsies (who knows how far along this is). I'm not sure how useful these would be anymore.

 

[Amw66]

I think that is an interesting idea. I wonder if there could be an addition to the DecodeME project (with separate funding) that looked into these issues taking advantage of the cohort that is being developed (and even diagnostic information to get agregate levels and types of impairment).

I feel that any study should have a heavy involvement of patients to help direct into what is really impairment (why and how symptoms really causes impairments in activities).

My other thought was is this something that a patient collective could do - survey patients, collate and write up results (along with some academic support).

@MEMarge re PSP research targeting

 

[Forbin]

I'd be interested in a prospective study that tries to capture data on patients as the severity of their symptoms undergoes long term shifts.

Anecdotally, it seems like some patients experience a level of improvement (though not necessarily recovery) during a window of 3 to 5 years after onset.

It would be interesting to have a lot of biological data on a group of patients at 2 years after onset and then follow up on those who improve prior to (say) the 6th year and see how their post-improvement data compares to their earlier data.

The idea would be to compare data from the same person over time as their severity changed. Obviously, you could also capture changes which might occur as severity increased. That might be harder because a lot of the decline might happen in the first year - before a patient even qualifies for a diagnosis of ME/CFS.

Anyway, it may be naïve, but I'm thinking that if you want to find clues about what's causing the problem, look at what (if anything) is changing when patients get better or worse.

The answer might be something non-intuitive, like if some readings got objectively "worse" by normal standards when some people improved - which might indicate an adaptation to the disease.