Langerhans cell histiocytosis (LCH) is characterized by inflammatory lesions featuring clonal infiltration. The BRAF mutation stands out as the most prevalent. Vemurafenib has emerged as a therapeutic option. The off-label use of VMF persists in Brazil. We will delineate the cases of two Brazilian children suffering from LCH with a positive response to VMF. Case Report I: The patient underwent standard therapy, but bone marrow infiltration persisted. After confirming a BRAF V600E mutation through testing, VMF was initiated, resulting in significant improvements. Case Report II: The patient initiated VMF and showed a positive response. After several months, maintenance therapy was introduced. However, skin lesions recurred upon discontinuation of VMF. She has been on VMF for a total of 31 months, maintaining remission. The cases presented represent the first reported instances of off-label VMF use in Brazil for the treatment of LCH, and both patients have demonstrated excellent responses to the medication.

A document by Daiane Cecconello. Click on the document to view its contents.

Brazil is the largest country in South America with continental dimensions and significant regional differences. Recently, the country has faced a great challenge in establishing adequate strategies to mitigate the strong impact caused by the arrival of COVID-19 in the national territory. Since the first case diagnosed on February 26th, 2020, we have gone through a 18 week period that resulted in 58.300 deaths and more than 1.370.000 confirmed cases¹. Despite having a public and universal health system, most pediatric oncology centers are located in more urbanized regions, such as state capitals or economic centers. Few reports describe the impact of SARS-CoV-2 on the population of pediatric patients accompanied by cancer or undergoing hematopoietic stem cell transplantation (HSCT)2,3,4. Less frequent still are the descriptions of the situation of these children in low or middle income countries⁵. Faced with this new and unknown scenario, the present study aims to describe the clinical presentation and the evolution of children affected by COVID-19 undergoing treatment in units of oncology, hematology or HSCT. We are analyzing the interregional differences in the presentation, management and prognosis of these children, while drawing a national panorama that can be compared with other countries through a national and multicenter registry of children evaluated for SARS-CoV-2.Through dissemination by the Brazilian Society of Pediatric Oncology (SOBOPE), Brazilian Association of Hematology and Hemotherapy (ABHH) and Brazilian Society of Bone Marrow Transplantation (SBTMO). 36 centers across the country agreed to participate in this project (Figure 1). All regions of the country are included. The variables evaluated are clinical symptoms, diagnostic method, therapeutic measures and treatment location. In addition, the repercussions of infection on baseline treatment and overall prognosis are being assessed. Despite being in an initial phase, the database already counts with 64 registered cases.This joint work of the centers for pediatric oncology and bone marrow transplantation in our country is an advance that is allowing the development of one of the largest databases on the influence of COVID19 in the treatment of children in developing countries. Considering the small number of current literature on this subject, the heterogeneity of our population and regional differences, centralization of records is essential to better understand the extent and impact of this epidemic in our country, in addition to comparing the effect of local health strategies. It is important to highlight that the Sistema Único de Saúde (SUS), one of the largest and most complex public health systems in the world, guarantees universal health care and presents itself as a significant differential in combating the pandemic in Brazil through primary care, as well as medium and high complexity assistance.The development of this study will contribute to a better understanding of the clinical presentation, evolution and impact of that of COVID-19 in pediatric cancer patients or those undergoing HSCT in middle-income countries.