The proposed study, entitled the Collaborative Assessment of Pediatric Transverse Myelitis: Understand, Reveal, Educate, or CAPTURE study, will be the first to combine assessments from healthcare providers and patients relative to TM outcomes. The collaboration will involve multiple healthcare centers across North America, the Transverse Myelitis Association and most importantly, patients. It will assess the current state of Pediatric TM in terms of diagnosis, treatment and outcomes. Ultimately, it will lead to an improved understanding of the current status of care for individuals afflicted with TM, and reveal what are the current best practices. Patients will educate clinicians and the study will educate the broader healthcare system about what outcomes are important and achievable. It will develop a multi-metric outcome measure, based on combined patient-generated and provider-generated data that can be used in future controlled trials. Of critical significance is the specific aim to make the data available for use by patients and practitioners via a Web-based program to determine how comparable their specific case is to the studied population. Unlike other clinical research endeavors that report findings primarily in peer-reviewed publications or presentations, this study will provide its data to a controlled program that can be accessed while a patient is being evaluated for TM, to determine if the data exists to guide decision making for that individual patient.
This study will work with the Web-based program Traitwise™, to enable their platform to inform a patient or practitioner about the percent homology of their patient to [other] (sic) studied patients. When complete, this study could serve as a model for other rare disease communities, allowing them to leverage vital assets and accelerate understanding of various conditions. The proposed research will lead to meaningful improvements in patient health and quality of care in multiple ways. It will identify which treatments were the most effective and safe for patients. This data will be used to guide future acute care and controlled clinical trials. Currently, there are wide variations in clinical practice, which is evidence of the pervasive uncertainty surrounding best practices. Previous studies examining the impact of acute care treatments in TM have identified a differential response to various therapeutic options. Recognizing which patients respond to the various therapies available would be of immense value to clinicians and individual patients, by helping them navigate the acute therapy environment with more scientific guidance. The largest patient advocacy organization for TM, the Transverse Myelitis Association, has identified improved diagnostic algorithms, treatment options, and outcome measures as a critical priority for the patients they serve.
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