I am excited to share a recent conversation with Jessica Gavin, PRAI Board Chair & IPR Principal Investigator, to talk about the International PANS Registry (IPR) and the PANS Epidemiology Study with you! Let’s dive right in. – Gabriella True, ASPIRE President.
Please note PANDAS is a subset of PANS, we will refer to PANS throughout, so yes, we automatically include PANDAS.
Gabriella: Give us the quick elevator pitch explanation about the epidemiological studies and registry.
Jessica: We need your help to further explore the dynamics of this complex illness and its enigmatic features. Study participants must have/had a formal or suspected PANS/PANDAS diagnosis and will complete a 2-hour highly detailed survey in the International PANS Registry patient-repository. Healthy sibling controls are encouraged to participate.
Why the IPR is vital to the community and its members
Gabriella: How do a registry and the epidemiological studies help the PANS/PANDAS community?
Jessica: The purpose of this project is to build an international hub of information on Pediatric Acute-Onset Neuropsychiatric Syndrome (PANS) by which to advance knowledge of this condition through research.
I know so many people would like to have answers to so many questions like:
- Why does IVIG work for one kid but not another?
- Which kids do better with tonsillectomy?
- Are there any common environments during pregnancy?
- Does diet help alleviate PANS symptoms?
- What makes some children treatment-resistant after a period of time?
I am so grateful for what the medical community has done so far, but there are still many questions to be answered. Fortunately, many researchers would love to study PANS. Yet, they have shared that collecting data on such a large scale is time-consuming and expensive and thus poses a barrier to launching many research studies. So the IPR enables researchers to launch studies more quickly and at a lower cost.
As of today, there are no comprehensive studies that aim to understand the epidemiology of PANS. Current research focuses mainly on identifying effective interventions for individuals with PANS. IPR believes that the lack of comprehensive studies designed in collaboration with researchers, clinicians, and caregivers that PANS remains such a gray area among the medical community today. We believe the registry will encourage new scientists to study the condition, and the data will provide unique insights into the true nature of the disorder that cannot otherwise be obtained through a clinical registry alone.
Gabriella: What are the specific aims of the registry?
Jessica: The purpose of the registry is 4 fold.
- Establish a registry of 2,000 PANS cases and their unaffected siblings
- Collect epidemiological survey data,
- Provide a means to link registry data to future biobank biological specimens for enrolled cases and controls, and
- Summarize the survey
We hope that by doing so, we will be able to unlock some of the mysteries around PANS by having a broad view from pregnancy to the present time on environmental and genetic factors that may lead to a PANS diagnosis.
Gabriella: Is there an end date for the registry?
Jessica: The larger registry project will be open for quite some time. However, the large-scale Epidemiological study from Dr. Masterson, PhD, MPH from the University of Washington, concludes March 15th. To participate in her four publications, everyone who has enrolled and completed all surveys by that date.
A few basics about the registry
Here are a few rapid-fire questions and answers. Some answers will have much more detail in the next section.
Gabriella: How long does it take to fill out the registry?
Jessica: Well, it is not short, but we wanted to get as much information as possible to appeal to a broader range of researchers. So, you might not be able to do it in one sitting (and we actually recommend that you don’t to avoid survey fatigue), especially if you are filling it out for more than one person. But you can take breaks by pressing SAVE AND CONTINUE and come back and finish later.
Gabriella: I know you get asked this a lot – “My kid has PANDAS; can I still fill it out?” Jessica: Absolutely. PANS is the umbrella term, and PANDAS is a subset.
Gabriella: If my child is older than 18, do they have to fill it out themselves?
Jessica: Yes. Anybody over 18 must fill it out themselves unless a parent has power of attorney.
Gabriella: If I have two affected kids and myself, how many times do I have to fill this out?
Jessica: If you have two kids, you have to add them at the start of the survey and fill it out for each child. If you have three kids and you yourself have PANS, then you would fill out the survey for your 3 kids, and then register yourself under the same survey ID but with a separate email for yourself to link your data to theirs.
Gabriella: Can one fill it out while still waiting for a diagnosis?
Gabriella: If my kid has comorbid issues like autism, can I fill it out?
Jessica: Absolutely. No other condition precludes you from filling the registry out.
Gabriella: Are my identity and information secure?
Jessica: Absolutely. We go into further detail below. Your information is 100% de-identified. If researchers want to include you in their research, they must contact us, and we will pass on their information for you to decide if you want to contact them. Plus, at the beginning of the survey, we ask you if you are willing to participate in future research; we hope you say yes.
The Nitty Gritty about the IPR – Compliance, Privacy, Software
Gabriella: How is the registry designed?
Jessica: We are collecting quantitative and qualitative (closed-ended) survey data and health history data. To officially enroll in the registry, participants (for cases and controls) respond to a set of baseline surveys.
Gabriella: Who is leading the registry?
Jessica: The lead epidemiologist of the registry is Erin Masterson, Ph.D. University of Seattle. She was hired by PRAI/IRP as an independent consultant to lead the project. She was chosen because she was an unbiased scientist with no history of “the controversy” and genuinely cares about children. She’s been involved in dental research and the environmental impacts that may influence autism.
Gabriella: What is the International Review Board (IRB)?
Jessica: Under FDA regulations, an IRB review is a qualified group that has been designated to monitor biomedical research involving human subjects. This review serves to protect the rights and welfare of human research subjects. It ensures research complies with FDA guidelines. It certifies that all data is de-identified when shared, and specific protocols are followed.
Gabriella: What is the registry platform?
Jessica: Dacima, a Canadian software company, runs the platform the registry is built and maintained on. An epidemiologist owns the company, and it has been used for many large scale clinical trials. The data is housed in HIPPA compliant cloud storage. Interview, an independent IRB with over 20 years of experience, has certified that IPR has little to no risk to research participants!
Gabriella: Who will be included?
Jessica: This includes adopted and biological children. Adopted children will be able to share that they “Do not know information on pregnancy” if they do not have that information available to them. Children who do NOT have a control sibling are welcome to register. Controls are not specific to each child. They are only compared to the larger CASE cohort as a whole. We really need as many controls as we do cases, so we really hope everyone enrolls all their children to obtain the best data. It’s important to enroll them all upfront at the beginning of the survey because you can’t go back later and add them in without contacting the IPR help desk.
We are also collecting data on those who believe they have PANS and have been unable to receive a diagnosis and those who received a PANS diagnosis and later learned that it was a misdiagnosis. Siblings who do not have PANS also will be asked to enroll. Caregivers will be asked to respond to children and adolescents.
Gabriella: Who will be able to identify who is enrolled?
- No insurance company
- No researcher
- No doctor
- No one
If you’re still uncertain about the safety of your information, we encourage you to use pseudo names for all of your children but with a working follow up email address. That way, your children’s name isn’t stored, but you can still be contacted if a researcher is interested in including your family in the research.
Gabriella: Who can access data?
Jessica: As soon as you register, your family is assigned an ID number that is linked to your health data. Researchers or anyone interested in using the data will only see the ID number and never your name or anything to identify you. At the start of the survey, you are asked whom you are willing to share your information with. If you choose, the data can be shared with pharmaceutical companies, university researchers, public health officials, nonprofits, and even citizen scientists by requesting access through a user agreement through our data manager running IPR. So you have control over whether or not your information is shared with researchers and institutions.
Gabriella: Will the person/group doing research contact me directly?
Jessica: No. Remember, they have no access to your personal identification, so they have to come through us. If they would like to include you in their upcoming research, they will contact us, and we will contact you. We will provide you with their information if you are interested in participating. Then we are no longer the middleman; you two will proceed from there.
Gabriella: So specifically, will my family be asked to participate in studies as a result of the IPR?
Jessica: Only if you allow them to. At the start of the survey, you will be asked with whom you would like to share your de-identified information. If you choose not to share with a pharmaceutical company, for example, then your data will not be included in their data. If you choose to share your health data, then that pharmaceutical company may choose to include you in their research. We hope that you choose to share your data with everyone as we need as many minds, organizations, researchers, etc to look at this data.
Gabriella: Tell us about the controls in the registry
Jessica: The registry has pseudo-controls. We are using siblings in phase 1 (which isn’t ideal, but we wanted sibling info to see if we could see why one and not another and whether other trends emerged). In phase 2, we will add controls by recruiting in areas where families cluster who do not have any psychiatric symptoms. Adults and children are both included.
Gabriella: So Jessica, to sum up in a nutshell, let me see if I got this right.
The PANS registry is a safe and secure, HIPAA compliant platform with IRB approval built by Dacima, a leader in data-driven software, designed to collect data from at least 2,000 PANS patients and controls for researchers to launch extensive data-driven studies more quickly and at less expense to continue to answer critical questions about PANS. I can fill it out even if I am waiting on a diagnosis and no matter how old the patient is. I can also choose with whom and if I want to share my data as all data is de-identified and secure. Start filling it out today and take breaks as you need, but the sooner you get 2,000 registrants, the better for the community!
Gabriella: Any words in closing to implore people to complete the registry?
Jessica: Remember, this is for our community. This is to grow research. A PANS parent and a researcher created this registry. So we share the same concerns about data safety as you, but we did everything in our power to protect all research participants, even making it completely anonymous. The sooner we fill this registry up, the sooner we can work on getting answers. I understand it is an extremely comprehensive survey and can’t be done quickly, but we truly appreciate your time and effort in getting it done. Also, don’t forget to ask your friends to fill it out too! Thank you, Gabriella and ASPIRE, for helping us get the word out. Thank you to the families and individuals that have already filled it out. I am excited about all the great doors this will open.