Langerhans cell histiocytosis
Top Cited Papers
- 16 April 2020
- journal article
- review article
- Published by American Society of Hematology in Blood
- Vol. 135 (16), 1319-1331
- https://doi.org/10.1182/blood.2019000934
Abstract
Langerhans cell histiocytosis (LCH) is caused by clonal expansion of myeloid precursors that differentiate into CD1a+/CD207+ cells in lesions that leads to a spectrum of organ involvement and dysfunction. The pathogenic cells are defined by constitutive activation of the MAPK signaling pathway. Treatment of LCH is risk-adapted: patients with single lesions may respond well to local treatment, whereas patients with multisystem disease require systemic therapy. Although survival rates for patients without organ dysfunction is excellent, mortality rates for patients with organ dysfunction may reach 20%. Despite progress made in the treatment of LCH, disease reactivation rates remain above 30%, and standard second-line treatment is yet to be established. Treatment failure is associated with increased risks for death and long-term morbidity, including LCH-associated neurodegeneration. Early case series report promising clinical responses in patients with relapsed and refractory LCH treated with BRAF or MEK inhibitors, although potential for this strategy to achieve cure remains uncertain.Keywords
This publication has 122 references indexed in Scilit:
- Management of adult patients with Langerhans cell histiocytosis: recommendations from an expert panel on behalf of Euro-Histio-NetOrphanet Journal of Rare Diseases, 2013
- Optimal Therapy for Adults with Langerhans Cell Histiocytosis Bone LesionsPLOS ONE, 2012
- Adult Langerhans cells derive predominantly from embryonic fetal liver monocytes with a minor contribution of yolk sac–derived macrophagesThe Journal of Experimental Medicine, 2012
- Recurrent BRAF mutations in Langerhans cell histiocytosisBlood, 2010
- Adult Langerhans cell histiocytosis and sclerosing cholangitis: a case report and review of the literatureHepatology International, 2010
- Cell-Specific Gene Expression in Langerhans Cell Histiocytosis Lesions Reveals a Distinct Profile Compared with Epidermal Langerhans CellsThe Journal of Immunology, 2010
- Targeting Oncogenic BRAF in Human CancerCurrent Topics in Microbiology and Immunology, 2010
- Neurodegenerative central nervous system Langerhans cell histiocytosis and coincident hydrocephalus treated with vincristine/cytosine arabinosidePediatric Blood & Cancer, 2009
- Expansion of Regulatory T Cells in Patients with Langerhans Cell HistiocytosisPLoS Medicine, 2007
- Epidemiologic study of Langerhans cell histiocytosis in childrenThe Journal of Pediatrics, 1997