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Gastrointestinal stromal tumors (GISTs) account for approximately 3% of gastrointestinal tumors and are the most prevalent mesenchymal tumors, primarily affecting the stomach and small intestine. In pediatrics, they are considered rare tumors and differ from GISTs that are found in adults because they often lack mutations in the KIT or platelet-derived growth factor α (PDGFRA) genes and are thereby classified as "wild-type." Currently, there are few specific guidelines for treating this type of tumor in the pediatric population. More data are needed to better understand GISTs and develop more effective targeted therapies. The present report describes the case of a patient, now an adult, who was diagnosed with a GIST during childhood and experienced several relapses. A therapeutic regimen was devised and implemented, resulting in a favorable outcome.
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http://dx.doi.org/10.1542/peds.2025-070625 | DOI Listing |
J Dermatolog Treat
December 2025
Department of Dermatology, Beijing Hospital, National Center of Gerontology, Institute of Geriatric Medicine, Chinese Academy of Medical Sciences, Beijing, China.
Background: Bullous pemphigoid (BP) is a common autoimmune subepidermal bullous disease. Dupilumab, an IL-4/IL-13 inhibitor, represents a novel therapeutic approach for BP, but real-world long-term data in super-elderly patients are limited.
Methods: This retrospective, single-center observational study included super-elderly BP patients (≥80 years) receiving dupilumab monotherapy from September 2022 to September 2024.
Ann Surg Oncol
September 2025
Department of Surgery, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
Int J Hematol
September 2025
Bone Marrow Transplantation Center, the First Affiliated Hospital, Zhejiang University School of Medicine, No. 79 Qingchun Road, Hangzhou, 310003, China.
Patients with primary plasma cell leukemia (pPCL), particularly those with extramedullary disease (EMD), face a poor prognosis even with chimeric antigen receptor (CAR)-T cell therapy. This case report describes a patient with relapsed/refractory pPCL and life-threatening malignant pleural effusion (PE) treated with intrapleural CAR-T cells targeting B-cell maturation antigens. CAR-T cell expansion within the PE was observed, along with a rapid reduction in leukemia cell count and PE volume.
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September 2025
Faculty of Health Sciences, Ben-Gurion University of the Negev, Beer-Sheva, Israel; Department of Medicine A, Assuta Ashdod University Hospital, Ashdod, Israel.
Front Immunol
September 2025
Department of Hematology, Cancer Center, the First Hospital of Jilin University, Changchun, China.
Severe aplastic anemia (SAA) is a life-threatening bone marrow failure syndrome that is caused primarily by immune-mediated destruction of hematopoietic stem cells. Traditional treatment relies on immunosuppressive therapy (IST) with antithymocyte globulin (ATG) and cyclosporine (CSA). However, the toxicity and limited availability of ATG have spurred interest in ATG-free regimens.
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