Pediatric Autoimmune Clinical Trial Discontinuation: an Analysis of the National Library of Medicine

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Background: With the rise in pediatric autoimmune diseases in the United States, it is both clinically relevant and timely to analyze the safety, cost-effectiveness, and efficacy of treatment options. The methodical research and study of health care services can lead to improving outcomes for patients with pediatric autoimmune diseases. Further, clinical trials are the gold standard for discovering and evaluating these often novel treatment options. Our primary objective was to assess select characteristics – phase, intervention, autoimmune disease type – of studies examining pediatric autoimmune clinical trials registered with the National Library of Medicine between 2008 to 2021. Our secondary objective assessed the rates of discontinuation of the clinical trials.

Methods: Using, we performed a cross-sectional study on all pediatric autoimmune clinical trials conducted from 2008 to 2021. Inclusion criteria were pediatric autoimmune trials with interventional treatment. We extracted the following data: the trial’s phase, intervention, autoimmune disease type, trial status (terminated, completed, active, recruiting), and intervention type. We then assessed rates of discontinuation by trial characteristics.

Results: 2009 trials returned from the search “pediatric autoimmune disease” on, 1098 of which met inclusion criteria for our research. The most common autoimmune disease was Type I Diabetes Mellitus (DM; 664 trials, 60.5%) followed by arthritis (164 trials, 15.03%). Nearly half (542, 49.4%) of the studies used pharmacologic interventions; other frequently used interventions were medical devices (200, 18.21%) and behavioral therapy (156, 14.21%). We found that 17.1% (188) of the pediatric autoimmune clinical trials were discontinued during this timeframe— the highest rates being among Pediatric Autoimmune Neuropsychiatric Disorders Associated with Streptococcal Infections (PANDAS; 2 of 7, 28.57%) and celiac disease (8 of 28; 28.57%).

Conclusion: In reviewing the substantial body of research on pediatric autoimmunity, our investigation revealed clinical trials focusing on Type I DM and drug interventions were most prevalent. This is an important finding since autoimmune diseases constitute a significant emotional, physical, and financial strain for patients and their caregivers. Therefore, it is necessary to determine which specific diagnoses constitute the majority ofsocietal burden to best direct resources. For advancements in medicine to be made, clinical trials must make it to completion and have their results published. The discontinuation of clinical trials delays advancements in the medical field and is not a productive use of research funding.
Original languageAmerican English
StatePublished - 17 Feb 2023
EventOklahoma State University Center for Health Sciences Research Week 2023 - Oklahoma State University Center for Health Sciences, 1111 W. 17th street, Tulsa, United States
Duration: 13 Feb 202317 Feb 2023


ConferenceOklahoma State University Center for Health Sciences Research Week 2023
Country/TerritoryUnited States
Internet address


  • pediatrics
  • autoimmune disease
  • clinical trials
  • discontinuation


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