Download full-text PDF |
Source |
---|---|
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC10344830 | PMC |
http://dx.doi.org/10.1016/j.idcr.2023.e01843 | DOI Listing |
J Pediatr Hematol Oncol
January 2025
Department of Paediatric Haematology, NHS Foundation Trust, Birmingham Children's Hospital, Birmingham, UK.
Objective: Emicizumab promotes efficacious hemostasis in persons with hemophilia A persons with hemophilia A with and without inhibitors. Primary analyses of real-world data and clinical trials have shown emicizumab efficacy and safety; however, long-term data are limited.
Methods: This retrospective study was conducted to assess real-world long-term outcomes of pediatric patients on emicizumab in our hemophilia center between the period of February 2018 and September 2023.
J Pediatr Hematol Oncol
January 2025
MVR Cancer Centre and Research Institute, Calicut, Kerala, India.
Background And Aims: Chemotherapy with alternating cycles of vincristine-doxorubicin-cyclophosphamide and ifosfamide-etoposide, along with primary tumor treatment with surgery or radiotherapy or both, constitute the usual treatment of Ewing sarcoma. The AEWS0031 study demonstrated survival benefits after interval-compressed chemotherapy without significant toxicity. The aim of this study was to assess the tolerability of dose-intensified chemotherapy in developing countries like India.
View Article and Find Full Text PDFLiver Int
February 2025
Department of Clinical Pharmacology and Toxicology, University Hospital Zürich, University of Zürich, Zürich, Switzerland.
Targeted therapies and immunotherapies have shown great promise as best-in-class treatments for several cancers with respect to efficacy and safety. While liver test abnormalities are rather common in patients treated with kinase inhibitors or immunotherapy, events of severe hepatotoxicity in these patients are rare in comparison with those associated with chemotherapeutics. The underlying mechanisms and risk factors for severe hepatotoxicity with novel oncology therapies are not well understood, complicating the drug-induced liver injury (DILI) risk assessment in the preclinical and clinical phases of drug development.
View Article and Find Full Text PDFIntroduction: Dolutegravir is now extensively used in sub-Saharan Africa as a preferred component of antiretroviral therapy (ART). There is a paucity of large studies using routinely collected data from African people living with HIV on dolutegravir-based regimens to inform HIV programmes. We reviewed data in a large programme clinic of people living with HIV on dolutegravir to determine the real-world safety and tolerability of dolutegravir and to describe drivers of treatment discontinuation.
View Article and Find Full Text PDFStat Med
February 2025
MRC Biostatistics Unit, University of Cambridge, Cambridge, UK.
There is a growing number of Phase I dose-finding studies that use a model-based approach, such as the CRM or the EWOC method to estimate the dose-toxicity relationship. It is common to assume that all patients will have similar toxicity risk given the dose regardless of patients' individual characteristics. In many trials, however, some patients' covariates (e.
View Article and Find Full Text PDFEnter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!