AXIS MG Patient Registry

Overview

The goal of the AXIS Autoimmune Neurology Registry is to learn about the experiences of adults living with autoimmune neurological disorders, starting with myasthenia gravis (MG), over time. The main questions the registry aims to answer are: * How do symptoms, daily functioning, and quality of life change over time? * What treatments do participants use, and how are these treatments related to their symptoms and health? * What challenges do participants experience with treatment, including side effects, treatment burden, missed doses, and access to care? Participants will: * Join the registry and complete an enrollment survey (online or through a mobile app) * Complete a brief online survey about their MG approximately once a month * Provide information that can help confirm their MG diagnosis * Optionally, share their medical records with the research team This is an observational study. The registry will not assign treatments or ask participants to change their medical care. Information collected through the registry may help researchers better understand MG and improve future research and care.

The AXIS Autoimmune Neurology Registry is a prospective, longitudinal, observational registry of adults in the United States with autoimmune neurological disorders. The registry is beginning with myasthenia gravis (MG), and participants with MG are currently being enrolled. The registry may expand to include other autoimmune neurological disorders in the future. The registry is decentralized. Participants complete study activities remotely through a secure website or mobile application. The registry does not assign treatments and does not ask participants to change their usual medical care. The registry collects information directly from participants to support research on the natural history and real-world management of MG. At enrollment, participants provide information about their MG diagnosis, whether they have ocular or generalized MG, age at diagnosis, demographic characteristics, education, employment, other medical conditions, prior testing, and treatments they have used. Participants are also asked about MG-related antibody testing, including acetylcholine receptor (AChR), muscle-specific kinase (MuSK), low-density lipoprotein receptor-related protein 4 (LRP4), agrin, and other antibody tests. The registry collects participant-reported information about chest imaging, thymus findings, and thymectomy when applicable. Participants identify MG treatments they have used and treatments taken during the prior month. These may include symptomatic therapies, corticosteroids, other immunosuppressive therapies, biologic or targeted therapies, intravenous or subcutaneous immunoglobulin, and plasma exchange. Additional questions may collect information about current and prior prednisone doses. At enrollment and during monthly follow-up, participants complete standardized measures of MG symptoms, functioning, and quality of life. These include: * The Myasthenia Gravis Activities of Daily Living profile (MG-ADL), which asks about talking, chewing, swallowing, breathing, brushing teeth or combing hair, rising from a chair, double vision, and eyelid droop during the previous week * The revised 15-item Myasthenia Gravis Quality of Life questionnaire (MG-QOL15r), which asks about the effect of MG on areas such as social activities, work, family responsibilities, mobility, driving, personal care, emotional well-being, and enjoyment of activities during the previous four weeks Participants are also asked to describe the severity of their MG symptoms at their worst during the prior month as a patient-reported proxy of MGFA Clinical Classification. Monthly questionnaires collect information about MG exacerbations and healthcare use, including emergency department visits, intensive care unit stays, and the use of mechanical ventilation. Questions may change or use branching logic based on a participant's earlier responses. Participants complete an enrollment questionnaire and an initial monthly questionnaire when they join. After enrollment, they are asked to complete a follow-up questionnaire approximately once per month. Participants may pause a questionnaire, save their progress, and return later. Participants provide information indicating that a healthcare provider has diagnosed them with MG. They are also asked to provide information that can help the research team confirm the diagnosis. Participants may confirm their diagnosis through one or more of the following methods: * Uploading a laboratory report showing a positive MG-related antibody test * Uploading another medical document supporting the MG diagnosis * Authorizing the registry to contact their healthcare provider * Authorizing the registry to request relevant medical records * Connecting electronic medical records through an available secure process Sharing medical records is optional. Participants may continue in the registry if their diagnosis has not yet been confirmed, although diagnosis confirmation may be required for certain participant incentives. Authorized registry staff review submitted documentation or information received from healthcare providers to determine whether the diagnosis has been confirmed. The registry uses structured electronic questionnaires and data-quality procedures to support accurate and consistent data collection. Required fields, response options, date and age validations, branching rules, and other predefined checks are built into the questionnaires. Responses may also be reviewed for completeness, ambiguity, values outside expected ranges, and inconsistencies with other information in the registry. Participants may be contacted when clarification is needed. Source data verification may be conducted by comparing participant-reported information with uploaded diagnostic documents, laboratory reports, healthcare-provider confirmation, connected electronic health information, or medical records authorized by the participant. Because medical-record sharing is optional, the amount of externally verified information may differ among participants. Study data are associated with a unique participant code. Information that directly identifies participants is stored separately, and access is limited to authorized personnel who need the information for registry operations, participant support, diagnosis verification, monitoring, or other approved purposes. Data used for research analyses are de-identified. The Principal Investigator and the Scientific Committee are responsible for monitoring the quality and integrity of the registry. The registry may also be reviewed, audited, or inspected by the institutional review board, regulatory authorities, sponsor representatives, and authorized quality or compliance personnel. The electronic system maintains records of informed consent and participant activity that can be provided for monitoring, audit, or inspection. Registry procedures are governed by the study protocol and supporting study documents. These procedures address informed consent, participant recruitment, questionnaire administration, diagnosis confirmation, medical-record authorization, participant communications and reminders, data management, confidentiality, data sharing, withdrawal, monitoring, and changes to registry procedures. Questionnaire specifications document the questions, response options, required fields, and branching logic used for data collection. There is no fixed maximum sample size. Broad recruitment is planned to support analyses of the overall MG population and clinically relevant subgroups, including groups based on MG type, symptom severity, antibody status, treatment use, age, and other participant characteristics. Statistical methods will depend on the research question and may include descriptive statistics, comparisons among participant subgroups, regression modeling, longitudinal analyses, and survival analyses. Analyses may evaluate changes in symptoms, functioning, quality of life, treatment use, and healthcare use over time. Missing data are expected in this long-term observational registry. Electronic and email reminders are used to encourage participants to complete enrollment, diagnosis-confirmation, and monthly questionnaires. Other follow-up methods may include telephone calls or mail when appropriate. Data-quality reviews will identify missing or inconsistent responses. The methods used to address missing data in an analysis will depend on the research question, the variables involved, and the amount and pattern of missingness. The registry is designed to continue over many years. Research questions, questionnaires, variables, and analyses may be updated as clinical practice changes, additional treatments become available, other autoimmune neurological disorders are added, and new research needs emerge. Any material changes to the protocol or participant-facing study materials will undergo required review and approval before implementation.