What would a good case study of recovery from ME/CFS look like?

Impressive individual cases of improvement or recovery can often be a starting point for hypothesis-generation and clinical trials in any disease.

In ME/CFS, the rituximab trial arose from observing impressive remissions in two PwME treated with rituximab for their cancer (IIRC). But lots of PwME have remissions following various interventions, but their stories don't lead to trials.

Suppose one of us had tried something out and had had a spectacular improvement. We'd surely want to share it and try to push for it to be researched. Presumably the first step would be to try to get written up as a case study. But what would make for a case study good enough to make it interesting enough to researchers to take it forward? What information would have to be in it for it to be taken seriously? How, in practice, would you have to do to get it into the medical literature?

Discuss!

 

Not a lot really. I published a case report of someone with arthritis getting better after rituximab. I had already decided to use rituximab bu the case was brought to my attention. There were no measurements before treatment - there aren't in this situation.So we just noted that the person felt better. It was published. But it needn't have been. If someone thinks they have seen a recovery following an unexpected treatment they can set up a pilot trial with actimeters or whatever. Nothing needs to be published to get that started.

 

Suppose, though, that you were the patient. You knew you were going to try an intervention and you had high enough hopes that it was going to work that you decided to start measuring some parameters, with a view to being an influential case study if it worked. What would you do? And if it did work, how would you get it published? Or at least get the right kind of information to the right researchers who might be interested in setting up a pilot trial?

 

But that's a trial, @Trish. A case report is where you get a result without having set out to do so.
If we are talking of doing a trial you need a lot more than one case.

 

That's a trial again, and it needs controls and enough patients.
For one person to do it would be hopeless. If they got better they would write to a journal but if they didn't they wouldn't - classic publication bias.

 

Admittedly I haven't read a lot of case reports, but I get the impression they focus on clinical observations that might be useful or important.

It could be a serendipitous response to treatment, a pattern of symptoms that suggest a familiar condition but turn out to be something else, or a previously unreported genetic finding.

They do all seem to be written by doctors, though that might only be because search engines tend to put the papers with most views at the top of the list.

 

We are oddly at cross-purposes!

This N=1 attempt would be a trial if it had controls and enough patients - but it's not a trial, it's a patient looking ahead before they try something and trying to get the best scientific use out of it.

How would this be different to Fluge and Mella's publishing their observation of their two ME cancer patients getting spectacularly better on rituximab?

Surely there's a way to do good hypothesis generation from observation of individual cases?

 

It would be hard for an individual patient—there are so many people telling recovery stories that the auto-response is an eye roll. It might be different if several patients experienced a similar response, passed the information on, and others benefitted too. Then it might get published.

Maybe the best case scenario is that the first patient is managed by an interested doctor who thinks the result is plausible enough to put his or her name to it, and has enough credibility to get it published and noticed.

Credibility's probably the biggest hurdle, and some of it is as much about the author and their standing as it is about the contents of the paper.

 

I am afraid that is a trial. A trial does not have to have controls or more than 1 case. My initial rituximab trial had no controls and 5 cases. Because I had objective end-points it gave a reliable result. A trial can be done by non-medical people, including patients.

The problem is if various people try something and only those with success report. For Fluge and Bella nobody was 'trying something'. The observation was purely post-hoc. It is all to do with expectations. Confirmation of expectations is a different situation from noting something unexpected. The need for guarding against subjectivity in trials is a strange and complex thing. You do not need to blind for unexpected or negative results anything like as much. People are not biased to report those.

 

I think it would require clinical verification of the ME symptoms before and after the treatment. Of course, we're still waiting for the means to measure ME symptoms reliably.

 

No. IIRC they received cyclophosphamide PLUS ritux. Fluge went with trialing ritux before cyclo.

Disabled people going back to work would be good info “to be taken seriously”. See here (trial, not a case study).

https://www.s4me.info/threads/intra...-study-2020-rekeland-mella-fluge-et-al.14925/