Background & Aims: Heterogeneity of treatment effects in inflammatory bowel disease (IBD) means that many individuals or patient subgroups depart from the average for whom the outcomes from traditional randomized trials may not be applicable. The N-of-1 trial is a design in which a single patient is followed over time with the treatments being randomized from period to period with the intention of finding the most effective treatment for that patient. The aim was to investigate the utility of N-of-1 trials in IBD.
Methods: To identify relevant articles for this scoping review, a MEDLINE literature search was conducted through the PubMed platform for articles published in the English language using the search terms "inflammatory bowel disease," "Crohn's disease," "ulcerative colitis," "N-of-1 trials," "single case designs," and "personalized trials."
Results: N-of-1 trials have seen a resurgence across several medical disciplines, driven by a need for more personalized medicine and patient-centered health care; their use in IBD is scarce with only 3 trials identified. Studies involving multiple N-of-1 trials can generate robust evidence for each participant and average effect estimates. The N-of-1 trial may hold potential for studying patients with IBD that are excluded from or underrepresented by randomized trials, such as those with extraintestinal manifestations, pouchitis, and proctitis. Although methodologically sound and akin to the rigor of a randomized controlled trial, the crossover periods inherent to the study design can be perceived as burdensome by patients and researchers.
Conclusions: The N-of-1 trial design provides a patient-centered means of objectively determining individual response to therapy.
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http://dx.doi.org/10.1016/j.cgh.2024.08.028 | DOI Listing |
CA Cancer J Clin
January 2025
Medical College of Wisconsin Cancer Center, Milwaukee, Wisconsin, USA.
Next-generation sequencing has revealed the disruptive reality that advanced/metastatic cancers have complex and individually distinct genomic landscapes, necessitating a rethinking of treatment strategies and clinical trial designs. Indeed, the molecular reclassification of cancer suggests that it is the molecular underpinnings of the disease, rather than the tissue of origin, that mostly drives outcomes. Consequently, oncology clinical trials have evolved from standard phase 1, 2, and 3 tissue-specific studies; to tissue-specific, biomarker-driven trials; to tissue-agnostic trials untethered from histology (all drug-centered designs); and, ultimately, to patient-centered, N-of-1 precision medicine studies in which each patient receives a personalized, biomarker-matched therapy/combination of drugs.
View Article and Find Full Text PDFJ Inherit Metab Dis
January 2025
Synaptic Metabolism and Personalized Therapies Lab, Institut de Recerca Sant Joan de Déu, Department of Neurology and MetabERN; Esplugues de Llobregat, Barcelona, Spain.
Cell trafficking alterations are a growing group of disorders and one of the largest categories of Inherited Metabolic Diseases. They have complex and variable clinical presentation. Regarding neurological manifestations they can present a wide repertoire of symptoms ranging from neurodevelopmental to neurodegnerative disorders.
View Article and Find Full Text PDFWell-designed effective interventions promoting sustainable diets are urgently needed to benefit both human and planetary health. This study evaluated the feasibility, acceptability, and potential impact of a pilot blended digital intervention aimed at promoting sustainable diets. We conducted a series of ABA n-of-1 trials with baseline, intervention, and follow-up phases over the course of a year, involving twelve participants.
View Article and Find Full Text PDFZhonghua Yi Xue Yi Chuan Xue Za Zhi
January 2025
University of Hong Kong Shenzhen Hospital, Shenzhen, Guangdong 518053, China.
Antisense oligonucleotide (ASO) was discovered several decades ago and initially used only as a research tool in the laboratory. In recent years, several ASO therapeutics have been developed for neurological disorders. Some of these therapeutics, including eteplirsen, golodirsen, viltolarsen, nusinersen and inotersen, have been approved by the Food and Drug Administration (FDA) and begun to draw the public's attention as an effective therapeutic approach.
View Article and Find Full Text PDFJ Neurogastroenterol Motil
January 2025
Department of Gastroenterology, St Mark's Hospital, London, UK.
Background/aims: Buspirone shows promise in treating disorders of gut-brain interaction (DGBIs), particularly functional dyspepsia. However, findings have been mixed.
Methods: We systematically searched for prospective studies testing buspirone for any upper gastrointestinal DGBI in 4 databases (Cochrane, PubMed, Scopus, and PsycInfo).
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