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자료유형
학술저널
저자정보
Jin Kyung Suh (Division of Pediatric Hematology/Oncology, Department of Pediatrics, Asan Medical Center Children’s) Sunghan Kang (Division of Pediatric Hematology/Oncology, Department of Pediatrics, Asan Medical Center Children’s) 김혜리 (울산대학교) 임호준 (울산대학교) 고경남 (울산대학교)
저널정보
대한혈액학회 Blood Research Blood Research Vol.56
발행연도
2021.4
수록면
65 - 69 (5page)
DOI
10.5045/br.2021.2021013

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Langerhans cell histiocytosis (LCH) is the most common histiocytic disorder caused by the clonal expansion of myeloid precursors that differentiate into CD1a+/CD207+ cells in the lesion. Advances in genomic sequencing techniques have improved our understanding of the pathophysiology of LCH. Activation of the mitogen-activated protein kinase (MAPK) pathway is a key molecular mechanism involved in the development of LCH. Recurrent BRAF mutations and MAP2K1 mutations are the major molecular alterations involved in the activation of the MAPK pathway. Recent studies have supported the “misguided myeloid differentiation model” of LCH, where the extent of disease is defined by the differentiation stage of the cell in which the activating somatic MAPK mutation occurs, suggesting LCH. Several studies have advocated the efficacy of targeted therapy using BRAF inhibitors with a high response rate, especially in patients with high-risk or refractory LCH. However, the optimal treatment scheme for children remains unclear. This review outlines recent advances in LCH, focusing on understanding the molecular pathophysiology, emerging targeted therapy options, and their clinical implications.

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