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First-Line Treatment of Pediatric Langerhans Cell Histiocytosis

  • Carlos Rodriguez-Galindo
  • Cor van den Bos
Chapter

Abstract

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.

Keywords

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

Abbreviations

2-CdA

Cladribine

6-MP

6-Mercaptopurine

AIEOP

Associazione Italiana Ematologia Oncologia Pediatrica

ARA-C

Cytarabine

BRAF

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

BRAF-V600E

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

CNS

Central nervous system

ctDNA

Circulating tumor DNA

CXCR-4

CXC chemokine receptor type 4

DAL-HX

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

DI

Diabetes insipidus

DNA

Deoxyribonucleic acid

EFS

Event-free survival

JLSG

Japan Langerhans Cell Histiocytosis Study Group

LCH

Langerhans Cell Histiocytosis

MEK

Gene encoding for mitogen-activated protein kinase kinase

MRI

Magnetic resonance imaging

MS-LCH

Multisystem Langerhans Cell Histiocytosis

MTX

Methotrexate

NAD

Non-active disease

ND-CNS LCH

Neurodegenerative central nervous system LCH

PDN

Prednisone

POG

Pediatric Oncology Group

PUVA

Psoralen plus ultraviolet A

RO

Risk organ

VBL

Vinblastine

VCR

Vincristine

VP-16

Etoposide

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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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