Learn More About Our Patient Registry
Thank you for learning more about PRAI's upcoming International PANS registry. Prior to full launch (expected May 1) we will update a more robust Q&A for specifics about the PEER platform and how to participate. For now, we hope that the information below will help you better understanding what it is we're trying to accomplish and why this project is so important for the PANS community. 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.
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.
Why does our community need a patient registry?
The underlying biological mechanism linking infection and neuropsychiatric symptoms is hypothesized to be a pseudo-autoimmune/autoinflammatory 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, which then attacks neuronal cells. 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.
PRAI believes it is because of 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 an International PANS registry will encourage new scientists to study the condition and the data will provide unique insights into the true nature of the disorder that can not otherwise be obtained through a clinical registry alone.
After a year of generating hypotheses from families who are in the trenches, we have incorporated questions into the study design that caregivers want answers to. We believe parents know their children best and the unique survey design based on their instincts will aid scientific discovery like never before.
What is the specific aim of the registry project?
The aims of this project are to (1) establish a registry of 1,000 PANS cases and their unaffected siblings (2) collect epidemiological survey data, health history tracker data (FlareDown app), and medical record information, (3) provide a means to link registry data to biobanked biological specimens for enrolled cases and controls, and (4) to summarize the survey, health history tracker and abstracted medical chart data collected for the first 1,000 registered cases and their siblings. 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.
How will the registry be designed?
We will collect quantitative and qualitative (closed-ended) survey data, health history data and medical record information for study. To officially enroll in the registry, participants (for cases and controls) will (1) respond to a set of baseline surveys. Participants will be also be asked to upload to the PEER site (2) health history data recorded via the FlareDown tracker app (cases only) and (3) medical records. Items (2) and (3) will not be required for enrollment in the Registry.
Who will be included?
The target population for this registry are children, adolescents and adults with PANS as well as children with a diagnosis of Autoimmune Encephalitis with a prior diagnosis of PANS. Siblings who do not have PANS will be asked to enroll as well. Caregivers will be asked to respond for children and adolescents. It's important to note, the data will be able to be pulled to reflect only the information scientists would like to see. So if they're only interested in PANS patients with an official diagnosis as opposed to those suspected of having PANS, they will be able to generate that information for the specific cohort they're interested in.
Does PRAI have any intention on publication?
In order to engage more scientists to investigate PANS, we highly encourage participants to choose to have their data used for official research efforts. For those participants who are willing to have their de-identified data used for publication in a peer-reviewed journal, our preliminary analysis will include 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 specifically related to the neuropsychiatric aspects of PANS (by comparing to siblings), and (c) summarizing treatment outcomes for individuals with PANS. Results will be summarized in (a) a comprehensive report and (b) in manuscripts that will be submitted to peer-reviewed journals.
Will participants be compensated or reimbursed?
Each participant that uploads their child’s full medical records will be offered compensation with a $35 gift card (per child registered) to assist with the associated copying costs and to recognize the extra time and effort it will take for parents to provide these records to the PRAI project team. Participants may choose to "no compensation" to help finance future research projects.
Who will be able to access my child's information and how?
PRAI chose the PEER platform because it was the only registry that put the power in the patients hands, and we think that's exactly where it belongs.
How will you ensure the safety of participant data collected in this study?
It is necessary to record subject identifiers (names and contact information) to make it possible to follow up with this registry of patients and sibling controls. Datasets used for analyses will be de-identified and only a non-identifying subject ID will be used. Exported datasets containing subject identifiers will be saved on a secure, password-protected server in an electronic data file that only the project PI and NIH-trained research collaborators will have access to.
Please stay tuned as we update our website with more information as this exciting project unfolds. If you have any questions we can help answer, just email our registry team: Registry@praikids.org