First-Line Treatment of Pediatric Langerhans Cell Histiocytosis

  • Carlos Rodriguez-Galindo
  • Cor van den Bos


The current first-line treatment strategies in pediatric Langerhans cell histiocytosis (LCH) are based on extent of disease at diagnosis and early response to treatment. For limited forms of disease, such as single-bone lesions and isolated skin LCH, a wait-and-watch strategy is advised after the diagnosis has been made; other forms of treatment, including local and systemic therapies, may also be considered in some circumstances. For more extensive forms of LCH, systemic therapy is indicated. The treatment backbones most often used are those of vinblastine and corticosteroids, or cytarabine and corticosteroids. In multifocal bone LCH, the optimal treatment duration has not been established yet. For multisystem LCH, it has become clear that early intensification in unresponsive disease is very important. Prolongation of maintenance treatment to a year has resulted in a decrease in the reactivation rates. The effect of further prolongation is being studied in the current LCH-IV protocol of the Histiocyte Society. It is to be expected that in the (near) future, the use of BRAF and MEK inhibitors may be introduced in first-line therapy, especially for very high-risk patients. For these and other patients, recently identified risk factors need to be validated in ongoing and future trials, and new risk factors need to be identified. This should subsequently allow for the development of more refined risk-adapted treatment strategies.


Langerhans cell histiocytosis Pediatric Children Treatment First-line treatment Vinblastine Cytarabine Corticosteroids 







Associazione Italiana Ematologia Oncologia Pediatrica




Gene encoding for B-Raf protein (serine/threonine protein kinase B-Raf)


Mutation of BRAF gene with substitution of glutamate for valine at amino acid position 600


Central nervous system


Circulating tumor DNA


CXC chemokine receptor type 4


Deutsche Arbeitsgemeinschaft für Leukämieforschung und –Behandlung im Kindesalter e.V. – Histiozytose X


Diabetes insipidus


Deoxyribonucleic acid


Event-free survival


Japan Langerhans Cell Histiocytosis Study Group


Langerhans Cell Histiocytosis


Gene encoding for mitogen-activated protein kinase kinase


Magnetic resonance imaging


Multisystem Langerhans Cell Histiocytosis




Non-active disease


Neurodegenerative central nervous system LCH




Pediatric Oncology Group


Psoralen plus ultraviolet A


Risk organ








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Copyright information

© Springer International Publishing AG 2018

Authors and Affiliations

  1. 1.Departments of Global Pediatric Medicine and OncologySt. Jude Children’s Research HospitalMemphisUSA
  2. 2.Emma Children’s Hospital/Academic Medical Center, Department of Pediatric OncologyAmsterdamThe Netherlands

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