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Individualized treatment rules inform tailored treatment decisions based on the patient's information, where the goal is to optimize clinical benefit for the population. When the clinical outcome of interest is survival time, most of current approaches typically aim to maximize the expected time of survival. We propose a new criterion for constructing Individualized treatment rules that optimize the clinical benefit with survival outcomes, termed as the adjusted probability of a longer survival. This objective captures the likelihood of living longer with being on treatment, compared to the alternative, which provides an alternative and often straightforward interpretation to communicate with clinicians and patients. We view it as an alternative to the survival analysis standard of the hazard ratio and the increasingly used restricted mean survival time. We develop a new method to construct the optimal Individualized treatment rule by maximizing a nonparametric estimator of the adjusted probability of a longer survival for a decision rule. Simulation studies demonstrate the reliability of the proposed method across a range of different scenarios. We further perform data analysis using data collected from a randomized Phase III clinical trial (SWOG S0819).
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http://dx.doi.org/10.1177/09622802241262525 | DOI Listing |
Turk J Pediatr
September 2025
Division of Pediatric Infectious Diseases, Faculty of Medicine, İstanbul University, İstanbul, Türkiye.
Aim: This study aimed to describe barriers and facilitators of the adherence of children with human immunodeficiency virus (HIV) to antiretroviral therapy (ART) from the perspectives of their caregivers.
Methods: In-depth interviews were held with the caregivers of 15 children. The collected data were analyzed using thematic analysis procedures.
Turk J Pediatr
September 2025
Department of Cardiorespiratory Physiotherapy and Rehabilitation, Faculty of Physical Therapy and Rehabilitation, Hacettepe University, Ankara, Türkiye.
Background: Vascular changes are observed in children with cystic fibrosis (cwCF), and gender-specific differences may impact arterial stiffness. We aimed to compare arterial stiffness and clinical parameters based on gender in cwCF and to determine the factors affecting arterial stiffness in cwCF.
Methods: Fifty-eight cwCF were included.
Turk J Pediatr
September 2025
Department of Pathology, University of Health Sciences Antalya Education and Research Hospital, Antalya, Türkiye.
Background: Delirium in patients with ulcerative colitis may be seen, especially in the elderly and in patients hospitalized for a long time. In children, Wernicke's encephalopathy may occur due to thiamine deficiency in both ulcerative colitis and Crohn's disease. We present a patient with ulcerative colitis who presented with delirium as the first symptom, did not respond to steroid treatment and improved with anti-tumor necrosis factor-alpha treatment.
View Article and Find Full Text PDFTurk J Pediatr
September 2025
Department of Pediatric Hematology and Oncology, the Affiliated Hospital of Qingdao University, Qingdao, Shandong, China.
Background: The expression and clinical correlation of BRAFV600E mutation and programmed cell death-1 ligand 1 (PD-L1) in children with Langerhans cell histiocytosis (LCH) have been reported, but the conclusions of previous studies are inconsistent. In addition, it has been reported that elevated cathepsin S (CTSS) expression is associated with various cancers. However, there is currently no research on the correlation between CTSS and LCH.
View Article and Find Full Text PDFTurk J Pediatr
September 2025
Division of Pediatric Rheumatology, Department of Pediatrics, Cerrahpaşa Faculty of Medicine, İstanbul University-Cerrahpaşa, İstanbul, Türkiye.
Background: We aimed to document childhood onset mevalonate kinase deficiency (MKD) and to explore treatment responses and diagnostic challenges in regions endemic to familial Mediterranean fever (FMF).
Methods: This retrospective study included patients under 18 years of age, diagnosed with MKD and followed for at least six months at the pediatric rheumatology department of Istanbul University - Cerrahpaşa Medical Faculty between 2016 and 2024.
Results: Of 33 patients, 51.