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Evaluation of Lyso-Gb1 as a biomarker for Gaucher disease treatment outcomes using data from the Gaucher Outcome Survey.
Orphanet J Rare Dis
January 2025
Department of Gastroenterology, Hepatology and Infectious Diseases, University Hospital, Heinrich- Heine University, Düsseldorf, Germany.
Background: Patients with Gaucher disease (GD) require continual monitoring; however, lack of specific disease biomarkers was a significant challenge in the past. Glucosylsphingosine (lyso-Gb1) has been shown to be a reliable, key, specific, and sensitive biomarker for diagnosis, prognosis, and treatment response in clinical studies of patients with GD. We evaluated the change in lyso-Gb1 concentration over time following enzyme replacement therapy in patients with confirmed GD using real-world data from the Gaucher Outcome Survey disease registry.
View Article and Find Full Text PDFPrognostic significance and accuracy of oncologists' estimates of survival time in recurrent ovarian cancer.
Int J Gynecol Cancer
January 2025
The NHMRC Clinical Trials Centre, The University of Sydney, Sydney, NSW, Australia; Macarthur Cancer Therapy Centre, Sydney, NSW, Australia; Western Sydney University, Department of Medicine, Sydney, NSW, Australia. Electronic address:
Objective: We evaluated the accuracy of oncologists' estimates of expected survival time in recurrent ovarian cancer.
Methods: Oncologists estimated expected survival time at baseline for each patient, who were then followed up for survival time. We hypothesized that oncologists' estimates of expected survival time would be independently significant predictors of survival, unbiased (approximately equal proportions [50%] living longer versus shorter than their expected survival time), or imprecise (<30% within 0.
Refractory and Resistant Herpes Simplex Virus Mucocutaneous Infections in Immunocompromised Patients: Literature Review and Proposed Definitions for Use in Clinical Trials.
Clin Infect Dis
January 2025
Department of Cellular Therapy and Allogeneic Stem Cell Transplantation, Karolinska University Hospital Huddinge, Karolinska Comprehensive Cancer Center, Stockholm, Sweden.
Herpes simplex virus (HSV) infection is one of the most prevalent viral infections worldwide. In general, host immunity is sufficient to clear viral shedding and recurrences, although it is insufficient to prevent subsequent virologic reactivations. In immunocompromised patients, prolonged and difficult-to-treat HSV infections may develop.
View Article and Find Full Text PDFThe severity of COVID 19 symptoms has a direct correlation with lymphopenia, affecting natural killer (NK) cells. SARS-CoV-2 specific "memory" NK cells obtained from convalescent donors can be used as cell immunotherapy. In 2022 a phase I, dose-escalation, single center clinical trial was conducted to evaluate the safety and feasibility of the infusion of CD3/CD56 NK cells against moderate/severe cases of COVID-19 (NCT04578210).
View Article and Find Full Text PDFA Rare Case of Juvenile Granulosa Cell Tumor in a Premenarchal Female: Clinical Presentation, Diagnosis, and Management.
Cureus
December 2024
2nd Pediatric Surgery Department, Athens Children's Hospital P&A Kyriakou, Athens, GRC.
Juvenile granulosa cell tumors (JGCTs), a rare type of ovarian tumor, are predominantly seen in premenarchal girls. We report a case of a 4.5-year-old girl with precocious puberty and a left ovarian JGCT, confirmed through imaging and histopathology.
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