USA: National Institutes of Health (NIH) intramural ME/CFS study

[Simon M]

Brian Vastag asked me to post this here as he isn’t on Science 4 ME.

Hi - I'm nearing the end of week 2, visit 2 of the NIH study. Thought that I'd reiterate to the community that the study is still recruiting and still needs patients. To volunteer, contact patient coordinator Angelique Gavin (who is lovely):angelique.gavin@nih.gov and (301) 402-0880.

To reiterate the criteria: Patients must have had a sudden-onset illness and be within 5 years of the onset. The study is in all likelihood too intense for severe patients.

Lots of sub-studies have been added over the past year, since the study began, including skin and muscle biopsies and viral discovery in spinal fluid. I just donated 24 milliliters of it today, and some sterile, DNA and RNA-free vials of the "liquid gold" (in one doctor's words) were flash-frozen in liquid nitrogen and whisked away.

Visit one is a deep phenotyping visit: The volunteer is thoroughly assessed and other illnesses are ruled out. A five-doctor panel of outside ME experts then adjudicates the patient's file. If they are judged (unanimously) to have ME by CCC and Fukuda criteria, they are invited back for visit 2. Visit 2 includes a one-day exercise test (on a stationary bike), and five nights in metabolic chamber. They are assessing PEM quite thoroughly. It is fairly intense, and each patient needs to judge whether they can handle it. I am having a tough week but I'm doing a bit better than when I did a two-day exercise test three years ago for insurance purposes. So I'm grateful for that.

Volunteers will have travel paid by NIH and also receive a stipend for each visit. (About $600 for visit 1 and $1000 for visit 2). Each visit takes two weeks. Partners or family members are put up at a lodge on the NIH campus, which is very convenient to the Clinical Center, or at a nearby hotel.

Happy to answer questions on this thread. [refers to PR thread]
B
Brian

 

[MErmaid]

Here is the NIH page with more details:

https://mecfs.ctss.nih.gov/

 

[ahimsa]

For folks on twitter, here's a link to a tweet by @Tom Kindlon that you can re-tweet to spread the word:

 

[Forbin]

Patients must have had a sudden-onset illness and be within 5 years of the onset.

I don't object to this requirement, but it does reduce the pool of potential candidates quite a bit.

Not only is it estimated that 91% of patients are undiagnosed, but, in 29 percent of the cases that are diagnosed, that diagnosis took more than five years.

So something like only 6.4% of all cases may receive a diagnosis in less than 5 years, and only about 1.5% - 2% of all cases are diagnosed in less than one year - and that's without taking the sudden-onset requirement into account.

[On the other hand, 6.4% of a million cases is 64,000 cases.]

Myalgic encephalomyelitis/ chronic fatigue syndrome (ME/CFS) remains undiagnosed in up to 91% of patients. Recently, the United States-based Institute of Medicine (IOM) developed new diagnostic criteria, naming it systemic exertion intolerance disease (SEID).
https://www.tandfonline.com/doi/full/10.1080/21641846.2017.1299079

First, the often lengthy duration of time between symptom onset and diagnosis is estimated to be at least 1 year in 67 to 77 percent of patients and at least 5 years in 29 percent of patients (CFIDS Association of America, 2014; ProHealth, 2008). Quoted in the IOM report.

 

[Andy]

A Facebook post if anybody wants to share it,

 

[B_V]

I don't object to this requirement, but it does reduce the pool of potential candidates quite a bit.

Not only is it estimated that 91% of patients are undiagnosed, but, in 29 percent of the cases that are diagnosed, that diagnosis took more than five years.

So something like only 6.4% of all cases may receive a diagnosis in less than 5 years, and only about 1.5% - 2% of all cases are diagnosed in less than one year - and that's without taking the sudden-onset requirement into account.

[On the other hand, 6.4% of a million cases is 64,000 cases.]

Hi, Brian here, just signed up for the forum. Yes, that is all true. Still, NIH is having trouble recruiting. They've run 16 patients through the first visit. The NIH team has contacted about 200 patients who made it through an initial screening phone call and look like they qualify for the study, but they all disappeared and did not return phone calls. If about 10-15% of those people maintained contact and got into the study, that would get us to the 40 patients needed. I understand why people make contact and then disappear but I was surprised that 200 people had done that.

 

[Trish]

Hi @B_V, welcome to the forum. I'm pleased you've joined us here. I hope you enjoy being here as well as updating us on the NIH project.
Can you tell us how many have gone through the second stage of the trail?

 

[MErmaid]

Air travel plus adding two weeks of thorough testing is a “big ask” for many of us. I would consider it, if I could avoid the spinal puncture. Additionally, if the NIH wants more volunteers, then they need to think about dropping the age requirements. We are ill in all ages. I have sporadic energy surges through out the day. I would assume they would love to capture my data, for sure, as it would fluctuate from mild to moderate during each one of those metabolic chamber sessions.

 

[Aimossy]

Hi, Brian here, just signed up for the forum. Yes, that is all true. Still, NIH is having trouble recruiting. They've run 16 patients through the first visit. The NIH team has contacted about 200 patients who made it through an initial screening phone call and look like they qualify for the study, but they all disappeared and did not return phone calls. If about 10-15% of those people maintained contact and got into the study, that would get us to the 40 patients needed. I understand why people make contact and then disappear but I was surprised that 200 people had done that.

Did they try phoning these people just the once Brian? If so a second call round might be worth a shot.

 

[MErmaid]

Did they try phoning these people just the once Brian? If so a second call round might be worth a shot.

They are very persistent. I assume, from personal experience, that trying atleast 5 times would be more likely.

 

[Adrian]

Hi, Brian here, just signed up for the forum. Yes, that is all true. Still, NIH is having trouble recruiting. They've run 16 patients through the first visit.

I assume it is US only patients?

 

[Forbin]

The NIH team has contacted about 200 patients who made it through an initial screening phone call and look like they qualify for the study, but they all disappeared and did not return phone calls.

That is really odd. Is it possible that there is some information that is being conveyed in the initial phone call that is scaring people off? Or could they have been given a call back number that puts them on hold forever? For no one to call back sounds like a real systemic problem that may not lie with the patients. The detective in me would like to have one of these initial calls unwittingly placed to someone higher up in the study so that the senior person could see how they go (i.e. the caller would not know that they were speaking to someone up the chain at the NIH).

( BTW, Great to see you here Brian! )

 

[Allele]

If I weren't 13+ years into the illness I would apply despite the travel etc.
Do let us know if they shift that parameter at any point.

ETA: Great to see you here, Brian, thank you for joining us!

 

[Tom Kindlon]

For folks on twitter, here's a link to a tweet by @Tom Kindlon that you can re-tweet to spread the word:

Thanks.
If anyone wants to like or share it on Facebook, I posted it here:

 

[B_V]

That is really odd. Is it possible that there is some information that is being conveyed in the initial phone call that is scaring people off? Or could they have been given a call back number that puts them on hold forever? For no one to call back sounds like a real systemic problem that may not lie with the patients. The detective in me would like to have one of these initial calls unwittingly placed to someone higher up in the study so that the senior person could see how they go (i.e. the caller would not know that they were speaking to someone up the chain at the NIH).

( BTW, Great to see you here Brian! )

Ok, let me clarify. I got something wrong. The 200 number is the approximate total number of patient contacts after initial NIH phone screening. Sixteen of those are in the study. Some did not qualify due to self-diagnosis, gradual onset illness, illness longer than 5 years. The rest may qualify but disappeared after some initial contact with the patient coordinator, Angelique. She is very responsive and the phone number goes right to her desk so I don't think people are getting stuck on hold. Maybe voicemail once in a while. Anyway, I don't have an exact number of how many people have called in, made it past initial screening, been re-contacted by NIH and then disappeared. Apologies for the imprecise info.

 

[B_V]

If I weren't 13+ years into the illness I would apply despite the travel etc.
Do let us know if they shift that parameter at any point.

ETA: Great to see you here, Brian, thank you for joining us!

Thanks. With 16 patients already enrolled and examined in part 1 of the study, I doubt the inclusion criteria will change. They want to keep variables to a minimum. It does reduce the patient pool substantially but there are enough ME/CFS people to fill a 40-patient study. The NIH is looking at other ways to help recruit. For instance, several of the patients have been Mormon and the Mormon community seems active in medical research. On the other hand, the investigators are leery about drawing too many volunteers from one community for fear of introducing some unknown bias into the study.

 

[B_V]

I assume it is US only patients?

NIH takes international patients for some studies. I just saw someone from Belgium who is in the undiagnosed patient program, for instance. I will ask the patient coordinator. She did see a bump in contacts this past week, so I think the postings here, on PR, twitter, etc. did help.

 

[B_V]

NIH takes international patients for some studies. I just saw someone from Belgium who is in the undiagnosed patient program, for instance. I will ask the patient coordinator. She did see a bump in contacts this past week, so I think the postings here, on PR, twitter, etc. did help.

International patients welcome in the NIH intramural ME/CFS study. Call/email Angelique Gavin: angelique.gavin@nih.gov and (301) 402-0880

 

[duncan]

I wonder, @B_V, have you gathered your records from the NIH as they have accrued?

 

[B_V]

I wonder, @B_V, have you gathered your records from the NIH as they have accrued?

Yes, they provide online access to all of the standard test results. The research stuff is not accessible - much of it will be run after samples from all the volunteers are collected and pooled, e.g. the immune analysis of spinal fluid (they're looking at a few thousand proteins in CSF). They are running a few specialized tests for me to see if they can ID an antibody that showed up in my blood and spinal fluid. So far, I'm the only one of 16 patients to have oligoclonal bands in spinal fluid, so Ibappreciste the extra steps they are taking to figure out what my immune system is attacking. One advantage to being in the study: If weird stuff shows up, NIH will chase it down and bring in other experts to help. I had a consult with a muscle specialist and a dermatologist that weren't originally involved in the study.