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To inform clinical trial design and real-world precision pediatric oncology practice, we classified diagnoses, assessed the landscape of mutations, and identified genomic variants matching trials in a large unselected institutional cohort of solid tumors patients sequenced at Dana-Farber / Boston Children's Cancer and Blood Disorders Center. Tumors were sequenced with OncoPanel, a targeted next-generation DNA sequencing panel. Diagnoses were classified according to the International Classification of Diseases for Oncology (ICD-O-3.2). Over 6.5 years, 888 pediatric cancer patients with 95 distinct diagnoses had successful tumor sequencing. Overall, 33% (n = 289/888) of patients had at least 1 variant matching a precision oncology trial protocol, and 14% (41/289) were treated with molecularly targeted therapy. This study highlights opportunities to use genomic data from hospital-based sequencing performed either for research or clinical care to inform ongoing and future precision oncology clinical trials. Furthermore, the study results emphasize the importance of data sharing to define the genomic landscape and targeted treatment opportunities for the large group of rare pediatric cancers we encounter in clinical practice.
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http://dx.doi.org/10.1038/s41467-024-49944-0 | DOI Listing |
J Invest Dermatol
September 2025
Division of Pediatrics, Department of Biomedical and Clinical Sciences, Linköping University, Linköping, Sweden. Electronic address:
Environmental factors play a role for the onset of psoriasis. We investigated if early childhood stress contributes to the development of psoriasis later in life. Questionnaires, answered by parents of children in the All Babies in Southeast Sweden (ABIS) prospective birth cohort (n = 16 145) at follow-up 1, 3, 5, and 8 years of the child were used.
View Article and Find Full Text PDFJ Med Econ
September 2025
Friedrich-Baur-Institute, Department of Neurology, Ludwig-Maximilians-University Clinic, Munich, Germany.
Objective: To describe the economic burden of Pompe Disease in patients treated with enzyme replacement therapy (ERT) in the US.
Methods: A retrospective cohort study (1 January 2012-30 September 2022) was conducted using the Merative™ MarketScan Research Databases. Patients with ≥2 outpatient or ≥1 inpatient claims of Pompe disease, ≥1 claim of ERT, and continuous enrollment in medical/prescription coverage for ≥90 days prior to diagnosis date for patients ≥2 years and ≥1-month post-index were included.
Fam Pract
August 2025
Family Medicine and Occupational Medicine, FMC Medical Center, 888/272-273, Nakhon Ratchasima 30000, Thailand.
Ann Pharmacother
July 2025
Ernest Mario School of Pharmacy, Rutgers, The State University of New Jersey, Piscataway, NJ, USA.
Objective: To compare the efficacy and safety of ceftriaxone (CRO) 1 g intravenously (IV) daily to higher doses for treating non-central nervous system (CNS) infections in adults.
Data Sources: PubMed, Embase, Scopus, and Web of Science were used. A search was run from database inception to August 8, 2023 and rerun May 12, 2025.
J Inflamm Res
August 2025
Department of Laboratory Medicine, Ruijin-Hainan Hospital, Shanghai Jiao Tong University School of Medicine (Hainan Boao Research Hospital), Ruijin, Hainan, People's Republic of China.
Purpose: Previous small-sample study suggested that fecal calprotectin (FC) combined with blood inflammatory biomarkers may aid in Crohn's disease (CD) diagnosis. This study aimed to validate the FC's diagnostic and mucosal healing assessment value of FC and a noninvasive composite index in pediatric CD.
Patients And Methods: Patients aged 2-17 years who underwent ileocolonoscopy for suspected or established CD were enrolled.