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Rachel Leigh Randell

Assistant Professor of Pediatrics
Pediatrics, Rheumatology
DUMC Box 3212, Durham, NC 27710
DUMC Box 3212, Durham, NC 27710

Selected Publications

Opportunistic dried blood spot sampling validates and optimizes a pediatric population pharmacokinetic model of metronidazole.

Journal Article Antimicrob Agents Chemother · April 3, 2024 Pharmacokinetic models rarely undergo external validation in vulnerable populations such as critically ill infants, thereby limiting the accuracy, efficacy, and safety of model-informed dosing in real-world settings. Here, we describe an opportunistic appr ... Full text Link to item Cite

"Click and mortar" opportunities for digitization and consumerism in trials.

Journal Article Contemp Clin Trials · September 2023 BACKGROUND: Digitization (using novel digital tools and strategies) and consumerism (taking a consumer-oriented approach) are increasingly commonplace in clinical trials, but the implications of these changes are not well described. METHODS: We assembled a ... Full text Link to item Cite

Pharmacokinetics of hydroxychloroquine in paediatric lupus: data from a novel, direct-to-family clinical trial.

Journal Article Lupus Sci Med · November 2022 OBJECTIVE: Determine the pharmacokinetics (PK) and exposure-response of hydroxychloroquine (HCQ) and desethylhydroxychloroquine (DHCQ) in paediatric SLE (pSLE). METHODS: We conducted an exploratory phase 2, direct-to-family trial. Children enrolled in the ... Full text Link to item Cite

Pediatric rheumatologists' perspectives on diagnosis, treatment, and outcomes of Sjögren disease in children and adolescents.

Journal Article Pediatr Rheumatol Online J · September 5, 2022 BACKGROUND: Sjögren disease in children and adolescents (pedSD) presents differently than adult disease. Diagnosis and classification are controversial, optimal treatment is unknown and outcomes are poorly understood. Here, we describe the current perspect ... Full text Link to item Cite

Adverse Reactions in a Phase 1 Trial of the Anti-Malarial DM1157: An Example of Pharmacokinetic Modeling and Simulation Guiding Clinical Trial Decisions.

Journal Article Infect Dis Ther · April 2022 INTRODUCTION: There is an urgent need to develop new drugs to treat malaria due to increasing resistance to first-line therapeutics targeting the causative organism, Plasmodium falciparum (P. falciparum). One drug candidate is DM1157, a small molecule that ... Full text Link to item Cite

Assessing Patient-Reported Outcomes in Pediatric Rheumatic Diseases: Considerations and Future Directions.

Journal Article Rheum Dis Clin North Am · February 2022 For children with pediatric rheumatic diseases (PRDs), the inclusion of patient-reported outcomes (PROs) is critical to inform decision making in health care delivery and research settings. PROs are direct reports from a child on their health status, witho ... Full text Link to item Cite

Unique Aspects of Pediatric Sjögren Disease.

Journal Article Rheum Dis Clin North Am · November 2021 Sjögren disease increasingly is recognized in pediatric patients. Clinical features, primarily parotitis and sicca symptoms, and results of diagnostic tests may be different from those in adult disease. Adult criteria fail to capture most pediatric patient ... Full text Link to item Cite

Safety updates in novel therapeutics for pediatric rheumatic disease.

Journal Article Curr Opin Rheumatol · September 1, 2021 PURPOSE OF REVIEW: Biologics and novel targeted therapeutics have transformed the management of pediatric rheumatic diseases over the past two decades; however, questions about short-term and long-term safety remain. Safety data gathered from recent clinic ... Full text Link to item Cite

Childhood Sjögren syndrome: features of an international cohort and application of the 2016 ACR/EULAR classification criteria.

Journal Article Rheumatology (Oxford) · July 1, 2021 OBJECTIVE: Sjögren syndrome in children is a poorly understood autoimmune disease. We aimed to describe the clinical and diagnostic features of children diagnosed with Sjögren syndrome and explore how the 2016 ACR/EULAR classification criteria apply to thi ... Full text Link to item Cite

Delivering clinical trials at home: protocol, design and implementation of a direct-to-family paediatric lupus trial.

Journal Article Lupus Sci Med · May 2021 INTRODUCTION: Direct-to-family clinical trials efficiently provide data while reducing the participation burden for children and their families. Although these trials can offer significant advantages over traditional clinical trials, the process of designi ... Full text Link to item Cite

Painful, reappearing eruption in a medically complex 4-year-old.

Journal Article BMJ Case Rep · February 18, 2021 A 4-year-old boy with atypical, complete DiGeorge and CHARGE (coloboma, heart defects, atresia choanae, growth retardation, genital abnormalities and ear abnormalities) syndromes presented with frequent episodes of a painful, markedly erythematous eruption ... Full text Link to item Cite

Tocilizumab in Refractory Autoimmune Encephalitis: A Series of Pediatric Cases.

Journal Article Pediatr Neurol · September 2018 BACKGROUND: Autoimmune encephalitis can result in significant neurological and psychiatric morbidity and mortality in patients of all ages and often does not respond to standard therapies. Recent reports suggest efficacy of tocilizumab, a monoclonal antibo ... Full text Link to item Cite

Feasibility of salivary DNA collection in a population-based case-control study: a pilot study of pediatric Crohn's disease.

Journal Article Clin Epidemiol · 2018 BACKGROUND: Epidemiologic studies combining exposure and outcome data with the collection of biosamples are needed to study gene-environment interactions that might contribute to the etiology of complex diseases such as pediatric Crohn's disease (CD). Nati ... Full text Open Access Link to item Cite

Collecting Biospecimens From an Internet-Based Prospective Cohort Study of Inflammatory Bowel Disease (CCFA Partners): A Feasibility Study.

Journal Article JMIR Res Protoc · January 5, 2016 BACKGROUND: The Internet has successfully been used for patient-oriented survey research. Internet-based translational research may also be possible. OBJECTIVE: Our aim was to study the feasibility of collecting biospecimens from CCFA Partners, an Internet ... Full text Link to item Cite

Validation of an internet-based cohort of inflammatory bowel disease (CCFA partners).

Journal Article Inflamm Bowel Dis · March 2014 BACKGROUND: As traditional methods have become increasingly difficult, the Internet offers a mechanism for conducting survey research quickly and efficiently. However, the validity of this research depends on the ability of respondents to accurately report ... Full text Link to item Cite

Patient perception of chronic illness care in a large inflammatory bowel disease cohort.

Journal Article Inflamm Bowel Dis · June 2013 BACKGROUND: Improvements in care for inflammatory bowel diseases could use the Chronic Care Model, an evidence-based approach that has improved patient outcomes and reduced costs in other illnesses. Specific aims include (1) to explore patient perception o ... Full text Link to item Cite

4-Phenylbutyrate stimulates Hsp70 expression through the Elp2 component of elongator and STAT-3 in cystic fibrosis epithelial cells.

Journal Article J Biol Chem · December 30, 2011 Sodium 4-phenylbutyrate (4PBA) corrects trafficking of ΔF508-CFTR in Cystic Fibrosis (CF) epithelia, which is hypothesized to, at least in part, result from increased expression of Hsp70 (stress-induced 70 kDa heat shock protein). To identify other 4PBA-re ... Full text Link to item Cite