OncoTargets and Therapy (Dec 2020)

Improvement in Pituitary Imaging After Targeted Therapy in Three Children with BRAF-Mutated Langerhans Cell Histiocytosis with Pituitary Involvement

  • Yang Y,
  • Wang D,
  • Li N,
  • Ma H,
  • Lian H,
  • Cui L,
  • Zhang Q,
  • Zhao X,
  • Zhang L,
  • Zhao Y,
  • Wang C,
  • Zhang L,
  • Wang T,
  • Li Z,
  • Zhang R

Journal volume & issue
Vol. Volume 13
pp. 12357 – 12363

Abstract

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Ying Yang,1 Dong Wang,1 Na Li,2 Honghao Ma,1 Hongyun Lian,1 Lei Cui,2 Qing Zhang,2 Xiaoxi Zhao,1 Liping Zhang,1 Yunze Zhao,1 Chanjuan Wang,2 Li Zhang,1 Tianyou Wang,1 Zhigang Li,2 Rui Zhang1 1Beijing Key Laboratory of Pediatric Hematology Oncology; National Key Discipline of Pediatrics; Key Laboratory of Major Diseases in Children, Ministry of Education; Hematology Oncology Center, Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health, Beijing 100045, People’s Republic of China; 2Laboratory of Hematologic Diseases, Beijing Pediatric Research Institute, Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health, Beijing 100045, People’s Republic of ChinaCorrespondence: Rui Zhang; Tianyou WangBeijing Children’s Hospital, Capital Medical University, National Center for Children’s Health, No. 56, Nanlishi Road, Xicheng District, Beijing, People’s Republic of ChinaTel +86 010-13691156162Fax +86 010-59617613Email [email protected]; [email protected]: Langerhans cell histiocytosis (LCH) is a rare myeloid neoplasia in children. BRAF mutations are associated with permanent diabetes insipidus (DI). The onset of clinical DI is always latent with imaging evidence of pituitary involvement. In this study, we reported three children with BRAF-mutated LCH with pituitary involvement who improved after targeted therapy (dabrafenib and trametinib). The results may indicate that pituitary involvement may be reversible in some patients with LCH if it is observed and treated before clinical DI. Targeted therapy may be an effective choice for LCH patients with early pituitary involvement who were irresponsive to first-line or second-line chemotherapy. However, a relapse after targeted therapy is completed in patients with persistently positive cfBRAFV600E mutations is still a challenge that needs to be resolved. The timing of introducing targeted therapy, its appropriate duration and effective combinations with chemotherapy or other targeted drugs should be explored further.Keywords: Langerhans cell histiocytosis, BRAF mutation, pituitary, children, targeted therapy

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