TITLE: PAN / Vasculitis Chat: The VCRC Patient Registry
DATE: December 12, 2012
Ed: PSN Director
Dr. Peter Grayson: Rheumatologist
Gail: PAN Group Member
Peter: PAN Group Member
Cory: PAN Group Member
Mary H: PAN Group Member
Key Web Resources Related to this chat:
Vasculitis Clinical Research Consortium (VCRC)
The VCRC Patient Registry
The Vasculitis Foundation
The Polyarteritis Nodosa Support Network
Ed--PSN Director: Anyway, let's get it started. Since this chat forum is limiting in terms of deep detail, I’ll encourage people to visit this site to learn more about Dr. Peter Grayson:
However, Dr. Grayson, please tell us some of your background.
Dr. Grayson: I am a clinician researcher at Boston University. I focus on all kinds of vasculitis - caring for patients with the disease and doing research. I should also mention that I was trained by Dr. Peter Merkel.
Ed--PSN Director: Can you tell us about some of the research you've done, or are doing?
Dr. Grayson: Sure. I have done research in the online VCRC patient contact registry, using data from the VCRC longitudinal studies, and some of my own work in a new field of research called bioinformatics.
Ed--PSN Director: And, or, what specific areas of rheumatology hold your interest the most.
Dr. Grayson: I am interested in vasculitis and other autoimmune diseases.
Ed--PSN Director: Great. Before we go on, tell us about bioinformatics. What that means to us laypeople.
Dr. Grayson: We study how genes are expressed inside of cells. With help of computers we can take a cell from a patient with vasculitis and run 20K tests simultaneously on all the genes to see what’s going on inside the cell.
Ed--PSN Director: that's pretty amazing.
Dr. Grayson: Hopefully it will usher in a new era of discovery in medicine!
Ed--PSN Director: I'm guessing this developing technology coming into its own in last ten or so years?
Dr. Grayson: That's right and it is still emerging and changing rapidly.
Ed--PSN Director: Great. So let's get to it. And we're going to start from the very beginning.
Again, rather than into a lot of detail here about the VCRC, I would encourage people reading the transcript to go to: http://rarediseasesnetwork.epi.usf.edu/vcrc/ to learn more about it. However, summing it upthe VCRC is basically a consortium of hospitals that have gotten together to focus on research for vasculitis. Anything to add to that, Dr. Grayson?
Dr. Grayson: That about sums it up but the focus is always on patient care first and then on research.
Ed--PSN Director: And so a very critical key to the VCRC is the Patient Contact Registry. tell us a bit about what it is... physically it is a database, right? But put a definition on it for the people who are unfamiliar.
Dr. Grayson: The VCRC Patient Contact Registry is a newly developed tool to conduct vasculitis research online. Anyone who has vasculitis can join. We particularly want to recruit patients with PAN. If you register, then you will periodically get emails about all new studies that come through the registry. The emails contain details about the study and a link to the study, which you can complete online and hit a button to send it back. It’s easy and hopefully it is rewarding and fun….
Dr. Grayson: You can register at http://rarediseasesnetwork.epi.usf.edu/vcrc/registry/
Ed--PSN Director: So a patient (or caregiver) goes to this registry and puts in information about themselves------it's like an online survey in that respect?
Dr. Grayson: Yes. You sign up, provide some information about yourself and your type of vasculitis. Then you receive emails that contain research studies, which are basically online surveys.
Ed--PSN Director: Once signed up do you get regular emails or periodic updates about news and developments
Dr. Grayson: As a member of the registry you will receive newsletters detailing what’s happening in the world of vasculitis research. These come out a few times a year. The online research studies come out whenever they are available, roughly at most 2-3 per year.
Ed--PSN Director: Excellent. Now the important point, and one that unfortunately confused some people. Just signing up for the registry does not mean you are automatically enrolled in a study. The registry is simply a way to alert you to current or upcoming studies, correct?
Dr. Grayson: Correct. The registry is not a clinical trial. It’s not even a study. It’s a gateway to begin to participate in research.
Ed--PSN Director: Talk a bit about what the patient gets in terms of a benefit by signing up and more importantly what YOU as a researcher can glean from this registry/database.
Dr. Grayson: The main benefit for all patients with vasculitis, no matter what type, is that you get connected to a vasculitis community and you get the rewarding experience of participating in clinical research from the comfort of your own home. For PAN there is an added benefit. Of all the different types of vasculitis, PAN is one of the rarest. And it is very hard to recruit enough PAN patients to perform any kind of clinical research. But it is easier to recruit enough patients with a very rare disease if you do it online. So in a sense, the Contact registry is a major vehicle to conduct research in PAN. It is a way for the rarer types of vasculitis to have their voices heard. For me as researcher, I can recruit large numbers of patients with a rare disease very quickly. This opens doors to doing lots of interesting online research.
Ed--PSN Director: Now, not to put too fine a point on this. But it was a source of confusion, when you say that you're participating in research from your own home. What you mean is that simply by joining the registry you are in a sense helping research! Is that right?
Dr. Grayson: I mean that you will get links to research studies which are being conducted all online. You can complete the study usually in 20 minutes on your home computer, submit it back to us by clicking a button, and you have just participated in vasculitis research without leaving your home.
Ed--PSN Director: See, originally when this all started I had many people writing to me and saying, ‘ I joined the patient registry but I never heard if I was accepted for the PAN Study’ . They assumed joining the registry was the same as applying to be in the trial. But I think that has been explained much better, thanks!
Dr. Grayson: exactly. You always get the choice with each new online study whether or not to participate
Ed--PSN Director: so it only takes a few min to join the registry not much time or effort...
Tell us about PAN participation right now. What kind of numbers we have, and what kind of numbers we NEED to see? Also why that's so important in terms of keeping the PAN study going.
Dr. Grayson: In the registry, we have about 35 people with PAN who are actively participating in studies and perhaps a few more who are enrolled but haven't decided to participate yet. One of the things we do with the registry studies are to make comparisons between different types of vasculitis. In order for your type of vasculitis to be included in the comparisons, we generally need to recruit at least 30 people with that type of vasculitis into the study. For example, we just completed the Vasculitis Illness Perceptions Study which looked at how people with vasculitis think about the disease. We had 32 PAN patients so we were able to use that data to show that people with PAN think about vasculitis very similarly to how people with other types of vasculitis think.
Ed--PSN Director: I just wanted to clarify and re-state the VCRC Patient Registry is totally separate from the VCRC PAN Longitudinal Study. I can join the VCRC patient registry and that will put me in touch with some ongoing studies and surveys.
But, if I want to join the PAN Longitudinal Study...that's an entirely different registration process. I have to go to VCRC site and sign up for that one specifically. Is that accurate?
Dr. Grayson: That's right. The registry and the longitudinal studies are vastly different but complimentary approaches to conducting research in vasculitis. The PAN longitudinal study is a clinical study being conducted at VCRC centers, and is very different from PAN studies done in the Patient Contact Registry.
To participate in the VCRC longitudinal studies you have to physically go to one of the participating VCRC centers and be seen quarterly or at least annually.
For the studies offered by the Contact Registry you can do that from home at your computer.
Basically, everyone with PAN (or other vasculitic diseases) can participate in research via the contact registry without having to ever report to a center.
Ed--PSN Director: Thanks for clearing that up.
Let's talk briefly about security of info on the registry. Some people have told me they are reluctant about giving such detailed info. What do you say to them?
Dr. Grayson: The Contact registry is completely anonymous. And information in the studies is never shared with outside agencies.
Ed--PSN Director: It's used strictly for the VCRC purposes.
Dr. Grayson: It's governed by the VCRC, and every study is approved by an ethics committee. All results are only used for research purposes.
Ed--PSN Director: Also, can we see the results of some of the studies that have been done. Is there a place to go to read them?
Dr. Grayson: Great questions. We are hoping to provide results in the registry newsletters. I also got the great opportunity to present results from a registry study at the Vasculitis Foundation Patient Symposiums.
Ed--PSN Director: Once more - parents and caregivers. They sign the registry for someone?
Dr. Grayson: Yes. Parents and caregivers can sign up.
Ed--PSN Director: GREAT. Thanks, Dr. Grayson. You really helped clear that up and now I hope to get folks to sign up!
Dr. Grayson: That would be wonderful.
Ed--PSN Director: In the remaining minutes I'd like to just turn it over to a general Q&A ....
Peter : Are you interested in people from outside of the U.S. signing up for this patient registry?
Dr. Grayson: Yes. No country restrictions and efforts currently underway to translate the registry into many different languages. A lot of international vasculitis researchers are interested in using the registry and are more than welcome.
Peter : Do the results of the studies go out to the rheumatology doctors?
Dr. Grayson: Yes, Peter. The most recent study I completed in the registry is being submitted for publication.
Ed--PSN Director: Dr. Grayson, a more general question on vasculitis?
What research or developments in the world may be good news, or of interest for PAN and vasculitis patients.
I mean, new diagnostic tests, or less invasive treatments. What might be good news stories on the horizon?
Dr. Grayson: Ed, there are lots of new developments. In a recent national meeting, one of our colleagues from the VCRC presented findings that one type of vasculitis (giant cell arteritis) may be caused by a specific type of infection that he identified.
Ed--PSN Director: Dr. Curry, I believe?
Dr. Grayson: Yes. Dr. Curry Koening from Utah. We are always looking to finding causes of vasculitis and his finding, if proven true, cold be one of the most important findings in vasculitis in the last 50 years.
Ed--PSN Director: What are the implications of that finding in layman terms?
Dr. Grayson: If you find a bacteria that is the cause of vasculitis, you could potentially treat that bacteria and cure the disease!
Ed--PSN Director: Wow - big stuff.
Dr. Grayson: And his approach to discovering the bacteria could be applied to look in other types of vasculitis like PAN.
Peter : That really grabs one's attention.
Ed--PSN Director: Interesting, because I had never heard of research being done in that area...as a bacteria.
Dr. Grayson: Yes it's cool. But findings are preliminary so we must be cautious about it.
Ed--PSN Director: This is where patients have to realize that research isn't instant. It's a lot of study and testing.
Dr. Grayson: Yes. Definitively, Ed. Discoveries take time. Within the field of bioinformatics that we talked about earlier, there are ways to look for bacteria within human tissue that are novel and done in an automated way.
Ed--PSN Director: But reassuring to know that such studies are being done. Patients need to know that behind the scenes a considerable amount of research is being conducted. This also ties directly into the topic tonightthe VCRC patient registry.
In a very real and direct way, every vasculitis patient can help this research by joining the VCRC patient registry and participating in some of these ongoing studies. It’s not way off to say that a PAN patient, by simply sharing information about his/her disease specifics, can be playing a part in a possible breakthrough some day. Buchanan...we're getting ready to wrap up, but are you a vasculitis patient and do you have a question for Dr. Grayson?
Buchanan: Yes, thank you! My husband was diagnosed by Cleveland Clinic with P.A.N. in August after 2 renal aneurysms. We were curious if a rheumatologist Dr. is ok to be a primary Dr. to treat PAN or do I have to only to go Cleveland Clinic.
Ed--PSN Director: Great question, go ahead Dr. Grayson!
Dr. Grayson: Good questions. The answer is that it depends on the rheumatologist. I would guess that most rheumatologists wouldn't feel comfortable making the treatment decisions in PAN. It's important to talk to your rheumatologist and ask them honestly about how comfortable they are treating the disease. If you live close to an expert center, like the Cleveland Clinic, then my advice would be to go there.
Buchanan: Great information. We are in Cincinnati and fortunately C.C. is only 4 hours away. I appreciate your advice.
Ed--PSN Director: That's a very interesting answer, Dr. Grayson. I mean, we have so many people who are diagnosed and they are letting their primary doc do the treatment. We always say to immediately go to a rheumy, but you're saying that up front you should find out how familiar that rheumy is with vasculitis specifically?
Dr. Grayson: Yes. If you have the chance to go to an expert center, it is advisable. I see patients as a onetime consultation and then remain available to them or their local doctor by phone or email.
Buchanan: May I ask a question about Prednisone? I started at 60 mg. in August and am now tapering down at 2.5mg. I am curious how long I should stay on 2.5 mg...before completely stopping. I am now having some numbness in my legs and feel shaky at times. I am curious if your body reacts to the weaning process of prednisone.
Dr. Grayson: Without commenting too specifically on your case since I have never evaluated you, I would say that people can commonly experience withdrawal symptoms from prednisone, particularly when weaning off lower doses. You should discuss these symptoms with your doctor. Sometimes it indicates withdrawal and sometimes it indicates other issues.
Buchanan: Ok, thank you very much. I greatly appreciate your advice.
Ed--PSN Director: Mary, would you like to ask Dr. Grayson a question?
Mary H: If a flare is not going away and I still have 2 nodules, what would be the proper dose of pred.? I have tapered down to 4 mg should I go back in to get on a higher dose or give it time?
Dr. Grayson: Hi Mary. That is a tough question to answer without evaluating you in the clinic. In general, there is never one "proper dose of prednisone". It has to be individualized to the patient. Mary, if you are concerned that you are flaring, I would go back in and talk to your doctor.
Ed--PSN Director: Prednisone really is very, very individualized, isn't it? Everyone tolerates it so differently.
Dr. Grayson: Yes. Patients always ask me how much prednisone do I need, and the answer I was taught to say is "enough and not anymore."
Mary H: I’m okay with that. Should I expect to be fully out of the flair before I tapper all the way down? or could the Imuran still be working?
Dr. Grayson: In general, I like to see the patients fully out of the flare before tapering.
Ed--PSN Director: One last question-----------
So a lot of people join my group and they may have just been dx'ed with PAN hours earlier by their doctors and they find our group and join it. They are understandably in a total panic. Some of them read there is only a five year survival rate (untreated)...or they are just totally freaked out. What message can you give them? What should they know about PAN/vasculitis, and especially what CAN they control to help get the best care and the best chance to treat it?
Dr. Grayson: I'd say to them:
- So much progress has been made in treating these diseases in the last 20 years and there are lots of treatment options...
- Don't look on the internet for information, you will scare yourself...
- instead, find a doctor who has expertise in these diseases and talk to them or at the very least joint a support group and learn more by talking to people with the disease
- the disease is highly variable and behaves differently in different people. Each case is unique. What is on the internet is usually the worst cases and a lot of misinformation.
Peter : That is for sure!
Ed--PSN Director: I always direct patients to the Vasculitis Foundation because they are legitimate and they present information that is accurate. Well, folks, it's a wrap. Thank you to Dr. Grayson..........we REALLY appreciate your time and knowledge. I'd love to have you back again. So much to talk about and I know patients are happy to even have a few min with you.
Dr. Grayson: Thanks so much for having me. This was fun. Happy to do it anytime!