Targeted Therapy With Vemurafenib in Brazilian Children With Refractory Langerhans Cell Histiocytosis: Two Case Reports and Review of Literature.
Klerize Anecely de Souza SilvaIsis Maria Quezado Soares MagalhãesDaniela Elaine Roth BenincasaDaiane Keller CecconelloMariana Bohns MichalowskiPublished in: Cancer reports (Hoboken, N.J.) (2024)
These cases represent the first reported instances of off-label vemurafenib use in Brazil for the treatment of LCH and both patients have demonstrated excellent responses to the medication. However, the long-term side effects are unknown in children, and prospective studies are needed. In addition, there is a lack of epidemiological data on histiocytosis in Brazil and studies evaluating the budgetary impact of incorporating BRAF mutation research and the use of vemurafenib into the public health system. These reports could be a starting point.
Keyphrases
- end stage renal disease
- young adults
- healthcare
- ejection fraction
- newly diagnosed
- adverse drug
- chronic kidney disease
- case control
- single cell
- prognostic factors
- stem cells
- mental health
- peritoneal dialysis
- cell therapy
- electronic health record
- case report
- bone marrow
- patient reported outcomes
- emergency department
- mesenchymal stem cells
- machine learning