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References from Real-world experience with targeted therapy in patients with histiocytic neoplasms in the Netherlands and in Belgium. Local targets link to admitted publications; unresolved targets remain external evidence.
Revised classification of histiocytoses and neoplasms of the macrophage-dendritic cell lineages
10.1182/blood-2016-01-690636 · 2016 · External reference
The 5th edition of the World Health Organization classification of haematolymphoid tumours: myeloid and histiocytic/dendritic neoplasms
10.1038/s41375-022-01613-1 · 2022 · External reference
The international consensus classification of mature lymphoid neoplasms: a report from the clinical advisory committee
10.1182/blood.2022015851 · 2022 · External reference
Demographics and additional haematologic cancers of patients with histiocytic/dendritic cell neoplasms
10.1111/his.15127 · 2024 · External reference
Incidence, prevalence and survival in patients with Langerhans cell histiocytosis: a national registry study from England, 2013-2019
10.1111/bjh.18459 · 2022 · External reference
Recurrent BRAF mutations in Langerhans cell histiocytosis
10.1182/blood-2010-04-279083 · 2010 · External reference
High prevalence of BRAF V600E mutations in Erdheim-Chester disease but not in other non-Langerhans cell histiocytoses
10.1182/blood-2012-05-430140 · 2012 · External reference
Frequent detection of BRAF(V600E) mutations in histiocytic and dendritic cell neoplasms
10.1111/his.12416 · 2014 · External reference
Somatic activating ARAF mutations in Langerhans cell histiocytosis
10.1182/blood-2013-06-511139 · 2014 · External reference
Mutually exclusive recurrent somatic mutations in MAP2K1 and BRAF support a central role for ERK activation in LCH pathogenesis
10.1182/blood-2014-05-577825 · 2014 · External reference
High prevalence of somatic MAP2K1 mutations in BRAF V600E negative Langerhans cell histiocytosis
10.1182/blood-2014-05-577361 · 2014 · External reference
MAP2K1 and MAP3K1 mutations in Langerhans cell histiocytosis
10.1002/gcc.22247 · 2015 · External reference
Alternative genetic mechanisms of BRAF activation in Langerhans cell histiocytosis
10.1182/blood-2016-08-733790 · 2016 · External reference
New somatic BRAF splicing mutation in Langerhans cell histiocytosis
10.1186/s12943-017-0690-z · 2017 · External reference
Diverse and targetable kinase alterations drive histiocytic neoplasms
10.1158/2159-8290.cd-15-0913 · 2016 · External reference
Activating mutations in CSF1R and additional receptor tyrosine kinases in histiocytic neoplasms
10.1038/s41591-019-0653-6 · 2019 · External reference
Clinicogenomic associations in childhood Langerhans cell histiocytosis: an international cohort study
10.1182/bloodadvances.2022007947 · 2023 · External reference
Efficacy of MEK inhibition in patients with histiocytic neoplasms
10.1038/s41586-019-1012-y · 2019 · External reference
Management of adult patients with Langerhans cell histiocytosis: recommendations from an expert panel on behalf of Euro-Histio-Net
10.1186/1750-1172-8-72 · 2013 · External reference
International expert consensus recommendations for the diagnosis and treatment of Langerhans cell histiocytosis in adults
10.1182/blood.2021014343 · 2022 · External reference
Erdheim-Chester disease: consensus recommendations for evaluation, diagnosis, and treatment in the molecular era
10.1182/blood.2019003507 · 2020 · External reference
Consensus recommendations for the diagnosis and clinical management of Rosai-Dorfman-Destombes disease
10.1182/blood-2018-03-839753 · 2018 · External reference
Therapy prolongation improves outcome in multisystem Langerhans cell histiocytosis
10.1182/blood-2012-09-455774 · 2013 · External reference
The clinical spectrum of Erdheim-Chester disease: an observational cohort study
10.1182/bloodadvances.2016001784 · 2017 · External reference
Phenotypes and survival in Erdheim-Chester disease: results from a 165-patient cohort
10.1002/ajh.25055 · 2018 · External reference
Neurologic and oncologic features of Erdheim–Chester disease: a 30-patient series
10.1093/neuonc/noaa008 · 2020 · External reference
Clinicopathological features, treatment approaches, and outcomes in Rosai-Dorfman disease
10.3324/haematol.2019.219626 · 2020 · External reference
Neurodegenerative central nervous system Langerhans cell histiocytosis and coincident hydrocephalus treated with vincristine/cytosine arabinoside
10.1002/pbc.22326 · 2010 · External reference
Evaluation and treatment of Langerhans cell histiocytosis patients with central nervous system abnormalities: current views and new vistas
10.1002/pbc.26784 · 2018 · External reference
Histiocytic sarcoma: a population-based analysis of incidence, demographic disparities, and long-term outcomes
10.1182/blood-2017-10-812495 · 2018 · External reference
Malignant histiocytosis comprises a phenotypic spectrum that parallels the lineage differentiation of monocytes, macrophages, dendritic cells, and Langerhans cells
10.1016/j.modpat.2023.100268 · 2023 · External reference
Histiocytosis and the nervous system: from diagnosis to targeted therapies
10.1093/neuonc/noab107 · 2021 · External reference
Neurodegenerative central nervous system disease as late sequelae of Langerhans cell histiocytosis. report from the Japan LCH Study Group
10.3324/haematol.11827 · 2008 · External reference
Incidence and risk factors for clinical neurodegenerative Langerhans cell histiocytosis: a longitudinal cohort study
10.1111/bjh.15577 · 2018 · External reference
The cognitive spectrum in neurodegenerative Langerhans cell histiocytosis
2014 · External reference
Long-term MR imaging course of neurodegenerative Langerhans cell histiocytosis
10.3174/ajnr.a0509 · 2007 · External reference
Neuropathology of CNS disease in Langerhans cell histiocytosis
10.1093/brain/awh403 · 2005 · External reference
Central nervous system disease in Langerhans cell histiocytosis
10.1016/j.jpeds.2010.03.001 · 2010 · External reference
CNS Langerhans cell histiocytosis: common hematopoietic origin for LCH-associated neurodegeneration and mass lesions
10.1002/cncr.31348 · 2018 · External reference
Circulating senescent myeloid cells infiltrate the brain and cause neurodegeneration in histiocytic disorders
10.1016/j.immuni.2023.11.011 · 2023 · External reference
Targeted cancer therapy
10.1038/nature03095 · 2004 · External reference
Dramatic efficacy of vemurafenib in both multisystemic and refractory Erdheim-Chester disease and Langerhans cell histiocytosis harboring the BRAF V600E mutation
10.1182/blood-2012-07-446286 · 2013 · External reference
Vemurafenib use in an infant for high-risk Langerhans cell histiocytosis
10.1001/jamaoncol.2015.0736 · 2015 · External reference
Vemurafenib in multiple nonmelanoma cancers with BRAF V600 mutations
10.1056/nejmoa1502309 · 2015 · External reference
Reproducible and sustained efficacy of targeted therapy with vemurafenib in patients with BRAF V600E -mutated Erdheim-Chester disease
10.1200/jco.2014.57.1950 · 2015 · External reference
Targeted inhibition of the MAPK pathway: emerging salvage option for progressive life-threatening multisystem LCH
10.1182/bloodadvances.2016003533 · 2017 · External reference
Vemurafenib for BRAF V600–mutant Erdheim-Chester disease and Langerhans cell histiocytosis
10.1001/jamaoncol.2017.5029 · 2018 · External reference
Mechanisms of resistance to RAF inhibitors in melanoma
10.1038/jid.2011.147 · 2011 · External reference
Single-agent dabrafenib for BRAFV600E-mutated histiocytosis
10.3324/haematol.2017.185298 · 2018 · External reference
A phase I and pharmacokinetic study of oral dabrafenib in children and adolescent patients with recurrent or refractory BRAF V600 mutation-positive solid tumors
10.1158/1078-0432.ccr-17-3572 · 2019 · External reference
Dabrafenib, alone or in combination with trametinib, in BRAF V600–mutated pediatric Langerhans cell histiocytosis
10.1182/bloodadvances.2022008414 · 2023 · External reference
Rosai–Dorfman disease with activating KRAS mutation — response to cobimetinib
10.1056/nejmc1713676 · 2017 · External reference
Trametinib in histiocytic sarcoma with an activating MAP2K1 (MEK1) mutation
10.1056/nejmc1511490 · 2018 · External reference
Efficacy of the MEK inhibitor cobimetinib for wild-type BRAF Erdheim-Chester disease
10.1111/bjh.14284 · 2018 · External reference
Outcomes after treatment with cobimetinib in patients with Rosai-Dorfman disease based on KRAS and MEK alteration status
10.1001/jamaoncol.2022.4432 · 2022 · External reference
Successful treatment of non-Langerhans cell histiocytosis with the MEK inhibitor trametinib: a multicenter analysis
10.1182/bloodadvances.2022009013 · 2023 · External reference
FDA approval summary: vemurafenib for the treatment of patients with Erdheim-Chester disease with the BRAFV600 mutation
10.1634/theoncologist.2018-0295 · 2018 · External reference
ALK-positive histiocytosis: a new clinicopathologic spectrum highlighting neurologic involvement and responses to ALK inhibition
10.1182/blood.2021013338 · 2022 · External reference
Allele-specific mechanisms of activation of MEK1 mutants determine their properties
10.1158/2159-8290.cd-17-1452 · 2018 · External reference
Vulvar Langerhans cell histiocytosis: clinicopathologic characteristics, mutational profile, and treatment of 4 patients in a single-center cohort
10.1016/j.jdcr.2023.03.024 · 2023 · External reference
Childhood-onset Erdheim-Chester disease in the molecular era: clinical phenotypes and long-term outcomes of 21 patients
10.1182/blood.2023020569 · 2023 · External reference
International cooperative ataxia rating scale for pharmacological assessment of the cerebellar syndrome. The Ataxia Neuropharmacology Committee of the World Federation of Neurology
10.1016/s0022-510x(96)00231-6 · 1997 · External reference
Targeted therapies in 54 patients with Erdheim-Chester disease, including follow-up after interruption (the LOVE study)
10.1182/blood-2017-03-771873 · 2017 · External reference
Vemurafenib for refractory multisystem Langerhans cell histiocytosis in children: an international observational study
10.1200/jco.19.00456 · 2019 · External reference
Clinical responses and persistent BRAF V600E+ blood cells in children with LCH treated with MAPK pathway inhibition
10.1182/blood-2018-10-878363 · 2019 · External reference
Effectiveness and safety of dabrafenib in the treatment of 20 Chinese children with BRAFV600E-mutated Langerhans cell histiocytosis
10.4143/crt.2020.769 · 2021 · External reference
Dabrafenib and trametinib in Langerhans cell histiocytosis and other histiocytic disorders
2024 · External reference
Pediatric histiocytic sarcoma clonally related to precursor B-cell acute lymphoblastic leukemia with homozygous deletion of CDKN2A encoding p16INK4A
10.1002/pbc.22810 · 2011 · External reference
PAX5 P80R-mutated B-cell acute lymphoblastic leukemia with transformation to histiocytic sarcoma: clonal evolution assessment using NGS-based immunoglobulin clonality and mutation analysis
10.1007/s00428-022-03428-y · 2023 · External reference
Transdifferentiation of B-lymphoblastic leukemia to histiocytic sarcoma after immunotherapy
10.1002/ajh.26983 · 2023 · External reference
Real-world toxicity experience with BRAF/MEK inhibitors in patients with Erdheim-Chester disease
10.1634/theoncologist.2019-0606 · 2020 · External reference
Low-dose vemurafenib monotherapy in BRAF V600E -mutated Erdheim-Chester disease
10.1080/10428194.2020.1783447 · 2020 · External reference
Outcomes after interruption of targeted therapy in patients with histiocytic neoplasms
10.1111/bjh.18964 · 2023 · External reference
Vemurafenib acts as a molecular on-off switch governing systemic inflammation in Langerhans cell histiocytosis
10.1182/bloodadvances.2021005442 · 2022 · External reference
Vemurafenib combined with cladribine and cytarabine results in durable remission of pediatric BRAF V600E–positive LCH
10.1182/bloodadvances.2022009067 · 2023 · External reference
Dual BRAF/MEK blockade restores CNS responses in BRAF-mutant Erdheim–Chester disease patients following BRAF inhibitor monotherapy
10.1093/noajnl/vdaa024 · 2020 · External reference
A brain-penetrant RAF dimer antagonist for the noncanonical BRAF oncoprotein of pediatric low-grade astrocytomas
2017 · External reference
The type II RAF inhibitor tovorafenib in relapsed/refractory pediatric low-grade glioma: the phase 2 FIREFLY-1 trial
10.1038/s41591-023-02668-y · 2024 · External reference
Children with refractory histiocytosis treated by MAPKi have a high risk of neurodegeneration: an international study
2023 · External reference
Vemurafenib: the first drug approved for BRAF-mutant cancer
10.1038/nrd3847 · 2012 · External reference
Inhibition of mutated, activated BRAF in metastatic melanoma
10.1056/nejmoa1002011 · 2010 · External reference
Cellular distribution of mutations and association with disease risk in Langerhans cell histiocytosis without BRAF V600E
10.1182/bloodadvances.2022007519 · 2022 · External reference
Lineage switching of the cellular distribution of BRAFV600E in multisystem Langerhans cell histiocytosis
10.1182/bloodadvances.2021006732 · 2023 · External reference
BRAF-V600E expression in precursor versus differentiated dendritic cells defines clinically distinct LCH risk groups
10.1084/jem.20130977 · 2014 · External reference
Circulating cell-free BRAF V600E as a biomarker in children with Langerhans cell histiocytosis
10.1111/bjh.14695 · 2017 · External reference
Hematopoietic origin of Langerhans cell histiocytosis and Erdheim-Chester disease in adults
10.1182/blood-2016-12-757823 · 2017 · External reference
Longitudinal assessment of peripheral blood BRAFV600E levels in patients with Langerhans cell histiocytosis
10.1038/s41390-018-0238-y · 2019 · External reference
Bone marrow–derived myeloid progenitors as driver mutation carriers in high- and low-risk Langerhans cell histiocytosis
10.1182/blood.2020005209 · 2020 · External reference
Circulating cell-free BRAF V600E during chemotherapy is associated with prognosis of children with Langerhans cell histiocytosis
10.3324/haematol.2019.229187 · 2020 · External reference
A circulating subset of BRAF V600E -positive cells in infants with high-risk Langerhans cell histiocytosis treated with BRAF inhibitors
10.1111/bjh.17721 · 2021 · External reference
BRAF V600E mutation in cell-free DNA, rather than in lesion tissues, at diagnosis is an independent prognostic factor in children with Langerhans cell histiocytosis
10.1158/1535-7163.mct-20-1075 · 2021 · External reference
Screening for neurodegeneration in Langerhans cell histiocytosis with neurofilament light in plasma
10.1111/bjh.18247 · 2022 · External reference
Response to mitogen-activated protein kinase inhibition of neurodegeneration in Langerhans cell histiocytosis monitored by cerebrospinal fluid neurofilament light as a biomarker: a pilot study
10.1111/bjh.17781 · 2022 · External reference