Frequently Asked Questions

Why does our community need a patient registry?

The underlying biological mechanism linking infection and neuropsychiatric symptoms is hypothesized to be a pseudo-autoimmune/auto-inflammatory reaction, in which an abnormal immune response causes damage to the child’s brain tissue due to cross-reactive antibodies and/or abnormal activation of the immune system. Current research is focused on identifying effective interventions for individuals with PANS. However, to date there are no comprehensive studies that aim to understand the epidemiology of PANS.

While interviewing dozens of researchers, we were told that the barriers to study the condition were too great. Not only was there not extensive funding, but with no large scale data, it was too expensive to study.  Those same researchers said they would be enthusiastic about investigating PANS if they had data to help lessen the learning curve and microgrants to offset the cost of post-doc time in the lab.

With a lack of comprehensive studies, PANS remains a gray area among the medical community today. We believe an International PANS registry will encourage new scientists to study the condition and the patient-reported data will provide unique insights into the true nature of the disorder.

What is the specific aim of the registry project?

The aims of this project are to (1) establish a registry with a minimum of 1,000 PANS cases and 1,000 unaffected siblings (for those who do not have unaffected siblings they would simply enroll all children as CASES) (2) collect epidemiological survey data and medical/health history information (3) provide a means to link registry data to future biobanked biological specimens for enrolled cases and controls, and (4) to summarize the survey results and publish them in peer-reviewed journals. It is our 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 present time on environmental and genetic factors that may lead to a PANS diagnosis.

It is our hope that this data will allow us to identify sub-groups within the larger umbrella of PANS to try and gain insights into why current treatments are effective for some patients and fail to work for others.

WHY SHOULD I PARTICIPATE?

By adding your data to IPR you are helping to advance life-changing initiatives in research, education, and advocacy. Large scale data from thousands of patients living with PANS will allow researchers to analyze disease patterns and help answer questions most important to patients.

WHAT TYPE OF RESEARCH QUESTIONS WILL IPR HELP ANSWER?

WHY ARE WE ENROLLING SIBLINGS?

WHO CREATED IPR?
Launched as a charitable program of Pediatric Research & Advocacy Iniative (PRAI), a 501(c)(3) organization aimed at accelerating patient-centered research initiatives, IPR was developed in collaboration with the parent community,  Erin E. Masterson, PhD, MPH, and several members of the PANS scientific and medical community. 

After reviewing the submissions, they were sorted into:

• Questions that could be answered using data from the IPR.
• Questions that couldn’t be answered using Registry data, but that could shed light on other research areas.

A half a dozen practicing physicians were then asked what type of data would be useful to them.  In addition, a dozen researchers not yet studying PANS were asked what type of data might encourage them to add PANS to their investigations. A team of people within our community went through the questions that could be answered with registry data and Erin Masterson, MPH, PhD organized them into topic areas and restructured them to reduce bias. More than 2,000 community members voted on the questions that were most important to their daily lives.

You can learn more about the history of IPR by reading our story.

IS THIS REGISTRY APPROVED BY AN INSTITUTIONAL REVIEW BOARD (IRB)? 
The Registry sponsor applied for approval from the University of Washington, (UW). The IPR was granted an approved exempt determination under federal IRB rules.  This determination included a review of the Registry surveys and overall study to ensure appropriate provisions are in place to protect the privacy and safety of participating subjects and their data in the United States and Canada.  

How will you ensure the safety of participant data collected in this study?

We take your privacy very seriously.  Patient information will be entered on Dacima’s HIPAA protected web server. Exported datasets containing subject identifiers will be managed according to the Registry’s approved IRB protocol and will be saved on a secure, HIPAA-compliant, password- protected Cloud server in an electronic data file that only the project Primary Investigator (PI) and lead epidemiologist have access to. Both individuals have met the National Institute of Health’s (NIH) Protecting Research Human Participants Requirements.  

To minimize the risk of harm and protect registry participants’ rights and welfare, the IPR project team will only use coded/de-identified data for analyses. Identifying information, including contact information, will be kept securely and separately from the analytic data. Children and adolescents will not be put at risk because their caregiver will act on their behalf.  If additional collaborators are ever added to the IRB study team, an email will go out to all participants informing them of the new addition and giving them the opportunity to remove their data if they so wish. De-identified information is never attached to the health information that we share with researchers and paper copies will never be used.  

We realize security and privacy are of the utmost importance, if you have any questions we can help answer related to the protection of your health data and we have not covered those questions here, email our registry team: info@pansregistry.org

Who can register?

The target population for this registry are children, adolescents, and adults with a clinical diagnosis of PANS, as well as children with a diagnosis of PANS who later qualify for an Autoimmune Encephalitis diagnosis based on strict AE criteria. 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. Please note, as PANDAS is a subset of PANS, it is included in the overarching umbrella every time we refer to PANS in our communications.  

We are also collecting data on those who believe they have PANS and have been unable to receive a diagnosis, as well as 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 for children and adolescents. ***Important information! All children, both cases and controls must be entered at the time of eligibility, otherwise, they will not be able to participate in IPR.  Please enroll all children (both those with and those without PANS) at the start of the survey.***

Is this available in all countries?

The International PANS Registry will first launch in the United States and Canada as part of our expedited IRB approval process with additional countries planned for phase 2 roll out.

Do adults over the age of 18 qualify?

Yes.  Adults with a previous or current diagnosis of PANS qualify for IPR and may enroll themselves. We also ask that they ask their unaffected adult (or parents of their less than 18 year old) siblings to register. 

CAN INDIVIDUALS WITH CO-MORBID CONDITIONS LIKE AUTISM, MAST CELL DISEASE OR DOWN SYNDROME PARTICIPATE?

Yes.  Having more than one diagnosis does not disqualify anyone from participating in the IPR registry as we hope that researchers will find value in studying different subgroups within the PANS community.  IPR enrollment only requires that an individual has or is suspected of having PANS (or PANDAS).  We will be actively encouraging research into these areas.

What if my child is in remission, or was later diagnosed with something else, are you still interested in our participation?

Absolutely!  Having children and adults who are now actively in remission would be incredibly valuable for our research efforts.  While you are no longer in the trenches, you can help shape the future for families with PANS by participating in IPR.  Similarly, if you ever received a diagnosis of PANS and went on to be diagnosed with another condition, sharing that information can potentially help other misdiagnoses from happening.  We encourage all families with a previous or current diagnosis of PANS, regardless of current health situation to participate in IPR.

What type of information should I have on hand prior to enrollment?

Before getting started, it may help to gather any of the following documentation that you might have on hand. If you don’t have these, and can’t recall specifics, just tell us you don’t remember in your responses! We want the most accurate information possible, so we would rather know you are unsure than have you guess.  

• Medical Records
• Bloodwork and other labs
• Cunningham Panel
• Vaccine history
• Birth/Pregnancy History
• Treatments tried
• Prescription history
• Diagnosis dates
• Clinical history

WHAT AM I COMMITTING TO BY AGREEING TO PARTICIPATE IN IPR?

HOW LONG DOES THE SURVEY TO TAKE TO FILL OUT?
IPR has a number of surveys important to researchers. Each survey may be completed in 15 minutes or less. You may complete as many surveys (or as few) as you would like, and log back in at a later time to complete the rest. If you want to answer them all at once, you should expect to spend at least 30 to 45 minutes due to the vast and diverse information we are trying to obtain. Please note, we do suggest that you take your time with the surveys for most accurate information.

CAN I TAKE IPR FROM MY CELL PHONE?
While we have done our absolute best to make the survey mobile friendly, phones are notorious for having issues with complex tables.  While it can be done, we highly recommend using a desktop, ipad or laptop computer. If you have technical difficulties, do not skip the questions, but reach out to us at help@pansregistry.org so we can help you work through them.

WHAT IF I CANT FIND MY CUSTOM LINK?

Search all your email folders for “info@pansregistry.org” for your Welcome to the International PANS Registry email.  If you still can’t identify it, email us help@pansregistry.org with the email you used when registered and/or your study ID and we can retrieve it for you.

HOW WILL YOU COMMUNICATE RESEARCH FINDINGS AND OTHER INFORMATION WITH FAMILIES?

You will receive updates from us periodically, for example, when there is a new survey, important news, and/or research findings. You will also receive any opportunities to participate in clinical trials and focus groups as a result of your participation. We believe patients and their families should hear the results of research before anyone else. All IPR funded research or research receiving microgrants from the registry sponsor will be shared first with families who participated. 

HOW CAN MY HEALTHCARE PROVIDER PARTICIPATE IN IPR?

Your provider can order educational IPR brochures to display in his/her office at no cost by simply sending an email to info@pansregistry.org with the subject line BROCHURES.

CAN I REMOVE MY DATA AT A LATER DATE?

You may choose to remove your data from IPR by written request whenever you wish.  However, data already being aggragated for research purposes’ including the study participant de-identified patient information, will not be able to remove study participants once the work has begun. The data can and will be removed from the database and not used for future research purposes by written request.

WHAT IF MY CHILD’S HEALTH HISTORY CHANGES? WILL WE BE ABLE TO UPDATE THAT?

After the initial roll-out, IPR will add a “flare diary” so you can prospectively report symptoms, suspected triggers and treatment success (or not) as your child experiences them. You will also eventually be able to log back into the Registry to update us if your CONTROL child develops PANS symptoms (becomes a CASE). Stay tuned to learn more about the release of these new features! 

IF YOU DON’T NEED TO HAVE A DIAGNOSIS OF PANS FOR ELIGIBILITY, HOW WILL YOU ENSURE THE DATA ISN’T SKEWED?

Since there’s no official test for PANS, a diagnosis is subjective whether a provider has given the label or not. We know many children with an original diagnosis of OCD, Tourette’s ODD, ASD, etc. later come to find out they have PANS.  Because of the strict criteria around PANDAS and the lack of knowledge and acceptance amongst pracititoners, we’ve included patients with a diagnosis, as well as those who were unable to receive one but believe their psychiatric symptoms are immune-mediated. What is great about this survey is that if a researcher only wants to see officially diagnosed patients, they can obtain that specific information.  However, if they want to study a broader population to see what commonalities exist among that population, they can query that data as well.  This way, no child is left behind.

WHAT IF MY CHILD’S UNAFFECTED SIBLING HAS SOME SIGNS OF PANS BUT WE ARE UNSURE WHETHER THEY SHOULD BE A CASE OR CONTROL?

If one of your children has “soft signs” of PANS but you are unsure whether or not they should register as a CASE, please enroll the sibling as a CASE so that you may be able to report symptoms appropriately.

WHAT IF I HAVE A TECHNICAL PROBLEM PROHIBITING ME FROM CONTINUING?
If you have any technical difficulties, please do the following to help us fix it quickly:

1. Tell us what question you’re on and under what topic area.
2. Screen shot the message or error you see.
3. Send that screen shot by email to help@pansregistry.org and someone will get back to you quickly.

While we hope these errors are far and few between.  We have already successfully piloted to many families. So we feel confident this should be an easy process.  However, there are always unknowns.  Following the above three steps will ensure that we escalate any technical issues quickly and streamline the process for other participants.  Thank you for your patience and understanding.

HOW WILL I KNOW WHEN I’VE COMPLETED THE REGISTRY?

Upon completion of IPR you should see all your links completed on your Household Landing page letting you know you’ve completed the entire survey.  

I RECEIVED AN EMAIL SAYING I WASN’T ELIGIBLE TO ENROLL BUT I THINK I MEET THE CRITERIA. WHAT DO I DO?

WHAT IF I HAVE SUGGESTIONS OR FEEDBACK ABOUT THE REGISTRY?

We want to make the registry the best it can be, which is why we’ve involved the parent community and clinical and scientific community every step of the way. If you have any suggestions or feedback, please feel free to email us at info@pansregistry.org and let us know what we can do to make IPR even more useful.

WILL I KNOW HOW MY DATA IS BEING USED?

Your child’s anonymized information will be pooled with data from thousands of other children with PANS. The pooled information is used by researchers to identify patterns that would not otherwise be visible, and to gain insights into the causes, mechanisms, and management of the disease.

IPR will provide updates about the research studies to registered participants as soon as details are given.  What is unique about IPR is we are giving each patient control over how their data is used for research.During intake, you will be asked how you would like to share your information, so please choose what’s most comfortable for you and your family.  If you do not want anonymized data to be available for public policy, we simply will not include your child when presenting that information when we receive public health data requests. If your family is unwilling to share de-identified data with pharmaceutical companies, that is your decision as well. If you want to engage citizen scientists in the scientific process, you can opt to share your de-identified data with them.  Your data will never be sold or shared without your written consent and absolutely no one other than the trained registry staff associated with the IRB will have access to your de-identified data. We highly encourage patients to allow a diverse range of people to have access to their child’s de-identified information as the more minds on the case and the more people working on PANS, the better. Remember, this data is DE-IDENTIFIED and the IPR Data Use Committee is comprised of families with the same fears and concerns over our children’s health history and privacy as you are. We hope that participants will share their de-identified data and trust the registry committee to ensure all researchers obtaining the data will conduct research that will be of great benefit to the PANS community. We believe the power to choose who has access to your data, de-identified or otherwise, solely belongs to you.  

WHEN WILL THE PARENT COMMUNITY GET ACCESS TO REGISTRY FINDINGS?

As soon as we get reportable results, we will share it with the community. We firmly believe that patients and their families who share their data deserve to be among the first group to learn any new findings.  It is our intention to report results by email to those who have registered once we have a large enough data set to analyze.

Will this data be published?  

In order to engage more scientists so that they may want to investigate PANS, our preliminary analysis will include stratified descriptions, simple correlations and associations using multivariate regression techniques to assess relationships between PANS and potentially-related factors. The emphasis of these analyses will be on (a) characterizing symptoms and trajectories according to health, environmental and demographic information, (b) identifying potential causes and associated factors related to specific neuropsychiatric symptoms (by comparing to siblings), and (c) summarizing treatment outcomes for individuals with PANS. Results will be summarized in manuscripts that will be submitted to journals for peer-review.  In addition, Erin Masterson, PhD, University of Washington Seattle, and co-creator of IPR, will present data at multiple conferences to raise awareness and generate interest among the scientific community

IF I DONATE TO IPR WHAT IS MY MONEY GOING TO SUPPORT?

Thanks to the incredible work of the parent community, IPR is fully funded for 36 months for the administrative aspects of the registry including hosting, HIPAA-compliant CLOUD storage, the creation of one manuscript laying out how IPR was created a basic overview of data findings, marketing and analysis.  Additional funds will be needed.  The registry sponsor (PRAI) has made IPR it’s sole charitable program, therefore, 100% of funds donated to the registry go directly to support the success of the registry and entice researchers to investigate PANS with microgrants.  Our most immediate funding need is a data analyst to be the gatekeeper for the data and the conduit between researchers and patient data.  This person will be responsible for reading through requests for data and communicating it to the IPR Data Use Committee and pulling the queries for researcher.  This resource needs to be proficient in SAS statistical software and have a basic understanding of database analysis.