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Predictive model performance may deteriorate when applied to data sources that were not used for training, thus, external validation is a key step in successful model deployment. As access to patient-level external data sources is typically limited, we recently proposed a method that estimates external model performance using only external summary statistics. Here, we benchmark the proposed method on multiple tasks using five large heterogeneous US data sources, where each, in turn, plays the role of an internal source and the remaining-external. Results showed accurate estimations for all metrics: 95th error percentiles for the area under the receiver operating characteristics (discrimination), calibration-in-the-large (calibration), Brier and scaled Brier scores (overall accuracy) of 0.03, 0.08, 0.0002, and 0.07, respectively. These results demonstrate the feasibility of estimating the transportability of prediction models using an internal cohort and external statistics. It may become an important accelerator of model deployment.
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http://dx.doi.org/10.1038/s41746-024-01414-z | DOI Listing |
J Am Soc Nephrol
September 2025
Department of Biochemistry and Molecular Biology, Mayo Clinic, Rochester, MN, USA.
Background: Genetic modifiers are believed to play an important role in the onset and severity of polycystic kidney disease (PKD), but identifying these modifiers has been challenging due to the lack of effective methodologies.
Methods: We generated zebrafish mutants of IFT140, a skeletal ciliopathy gene and newly identified autosomal dominant PKD (ADPKD) gene, to examine skeletal development and kidney cyst formation in larval and juvenile mutants. Additionally, we utilized ift140 crispants, generated through efficient microhomology-mediated end joining (MMEJ)-based genome editing, to compare phenotypes with mutants and conduct a pilot genetic modifier screen.
JCI Insight
September 2025
Division of Nephrology, Boston University Chobanian & Avedisian School of Medicine, Boston, United States of America.
Background: Active vitamin D metabolites, including 25-hydroxyvitamin D (25D) and 1,25-dihydroxyvitamin D (1,25D), have potent immunomodulatory effects that attenuate acute kidney injury (AKI) in animal models.
Methods: We conducted a phase 2, randomized, double-blind, multiple-dose, 3-arm clinical trial comparing oral calcifediol (25D), calcitriol (1,25D), and placebo among 150 critically ill adult patients at high-risk of moderate-to-severe AKI. The primary endpoint was a hierarchical composite of death, kidney replacement therapy (KRT), and kidney injury (baseline-adjusted mean change in serum creatinine), each assessed within 7 days following enrollment using a rank-based procedure.
Cereb Cortex
August 2025
Department of Developmental Psychology, University of Amsterdam, Nieuwe Achtergracht 129b, 1018 WS Amsterdam, The Netherlands.
Social learning, a hallmark of human behavior, entails integrating other's actions or ideas with one's own. While it can accelerate the learning process by circumventing slow and costly individual trial-and-error learning, its effectiveness depends on knowing when and whose information to use. In this study, we explored how individuals use social information based on their own and others' levels of uncertainty.
View Article and Find Full Text PDFProc Natl Acad Sci U S A
September 2025
Max Planck Institute for Solar System Research, Göttingen 37077, Germany.
Turbulent convection governs heat transport in both natural and industrial settings, yet optimizing it under extreme conditions remains a significant challenge. Traditional control strategies, such as predefined temperature modulation, struggle to achieve substantial enhancement. Here, we introduce a deep reinforcement learning (DRL) framework that autonomously discovers optimal control policies to maximize heat transfer in turbulent Rayleigh-Bénard convection.
View Article and Find Full Text PDFJMIR Med Inform
September 2025
Department of Radiology, Air Force Medical Center, Air Force Medical University, Fucheng Road 30, Haidian District, Beijing, CN.
Background: Lateral malleolar avulsion fracture (LMAF) and subfibular ossicle (SFO) are distinct entities that both present as small bone fragments near the lateral malleolus on imaging, yet require different treatment strategies. Clinical and radiological differentiation is challenging, which can impede timely and precise management. On imaging, magnetic resonance imaging (MRI) is the diagnostic gold standard for differentiating LMAF from SFO, whereas radiological differentiation on computed tomography (CT) alone is challenging in routine practice.
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