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Targeted Therapy with Vemurafenib in Brazilian Children with Refractory Langerhans Cell Histiocytosis: Two Case Reports and a Comprehensive Review
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  • Klerize Anecely de Souza Silva,
  • Isis Maria Quezado Soares Magalhães,
  • Daniela Elaine Roth Benincasa,
  • Daiane Keller Cecconello,
  • Mariana Bohns Michalowski
Klerize Anecely de Souza Silva
Universidade Federal do Rio Grande do Sul

Corresponding Author:[email protected]

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Isis Maria Quezado Soares Magalhães
Hospital da Crianca de Brasilia Jose Alencar
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Daniela Elaine Roth Benincasa
Hospital Conceicao
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Daiane Keller Cecconello
Universidade Federal do Rio Grande do Sul
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Mariana Bohns Michalowski
Universidade Federal do Rio Grande do Sul
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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.
25 Jan 2024Submitted to Cancer Reports
25 Jan 2024Review(s) Completed, Editorial Evaluation Pending
25 Jan 2024Assigned to Editor
25 Jan 2024Submission Checks Completed
06 Feb 2024Reviewer(s) Assigned