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http://dx.doi.org/10.1097/RHU.0000000000000440 | DOI Listing |
Clin Kidney J
January 2025
Department of Nephrology, The Children's Hospital, Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hangzhou, Zhejiang, China.
Background: Idiopathic nephrotic syndrome (INS) in children, commonly treated with steroids, poses challenges due to associated side effects. Rituximab, known for its efficacy in reducing relapse frequency in difficult-to-treat cases, emerges a potential first-line therapy for pediatric new-onset INS.
Method: This is a single-center, retrospective, observational study to evaluate the efficacy and safety of rituximab as a first-line therapy for pediatric INS.
Clin Cancer Res
January 2025
The University of Texas MD Anderson Cancer Center, Houston, TX, United States.
Purpose: More active high-dose chemotherapy (HDC) regimens are needed for autologous stem-cell transplantation (ASCT) for refractory lymphomas. Seeking HDC enhancement with a poly(ADP-ribose) polymerase (PARP) inhibitor, we observed marked synergy between olaparib and vorinostat/gemcitabine/busulfan/melphalan (GemBuMel) against lymphoma cell lines, mediated by inhibition of DNA damage repair. Our preclinical work led us to clinically study olaparib/vorinostat/GemBuMel with ASCT.
View Article and Find Full Text PDFJ Neurol Neurosurg Psychiatry
January 2025
Department for Neuromuscular Disease, National Hospital for Neurology and Neurosurgery, Queen Square, London, UK.
Background: We report our experience of patients with generalised myasthenia gravis (gMG) treated with efgartigimod, an neonatal Fc receptor antagonist, under the Early Access to Medicine Scheme (EAMS) in the UK.
Methods: Data from all UK patients treated with efgartigimod under the EAMS July 2022 to July 2023 were collected retrospectively. Efgartigimod was administered as per the ADAPT protocol (consisting of a treatment cycle of four infusions at weekly intervals with further cycles given according to clinical need).
J Pharm Sci
January 2025
Department of Chemical Engineering, Indian Institute of Technology Delhi. Electronic address:
Stability of complex biotherapeutics like monoclonal antibodies is paramount for their safe and efficacious use. Excipients are inactive ingredients that are added to the purified product so as to offer it a stable environment. Trehalose dihydrate is a non-reducing sugar that is commonly used as a stabilizing agent in biotherapeutic formulations under liquid and frozen states.
View Article and Find Full Text PDFVaccines (Basel)
December 2024
Hospital Universitário Cassiano Antônio Moraes da Universidade Federal do Espírito Santo (HUCAM-UFES/EBSERH), Vitória 29041-295, ES, Brazil.
Background/objectives: The effectiveness of COVID-19 vaccine in patients with immune-mediated inflammatory diseases (IMID) depends on the underlying disease, immunosuppression degree and the vaccine regimens. We evaluate the safety and immunogenicity of different COVID-19 vaccine schedules.
Methods: The SAFER study: "Safety and effectiveness of the COVID-19 Vaccine in Rheumatic Disease", is a Brazilian multicentric prospective observational phase IV study in the real-life.
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