Disease · MONDO_0018153

Erdheim-Chester disease

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. Erdheim-Chester disease. Diseases, record MONDO_0018153. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0018153

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Disease

Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.

Name: Erdheim-Chester disease; Open Targets
Open Targets ID: MONDO_0018153; Open Targets
Other names: lipogranulomatosis, polyostotic sclerosing histiocytosis; Open Targets
Disease Ontology: DOID:4329; Disease Ontology
MeSH: D031249; MeSH
NCI Thesaurus: C53972; NCI Thesaurus
Orphanet: ORPHA:35687; Orphanet
Highest stage of its drugs: Phase 2, for 9 of its 9 drugs, from ClinicalTrials.gov; Open Targets

Drugs9

The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.

Drug	ChEMBL ID	Type	Open Targets' stage	Stage reported by	Reports	Of them trials	Trials shown here	Source
CETUXIMAB	CHEMBL1201577	Antibody	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
COBIMETINIB	CHEMBL2146883	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
DABRAFENIB	CHEMBL2028663	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
DABRAFENIB MESYLATE	CHEMBL2105729	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
LENALIDOMIDE	CHEMBL848	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
TOCILIZUMAB	CHEMBL1237022	Antibody	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
TRAMETINIB	CHEMBL2103875	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
TRAMETINIB DIMETHYL SULFOXIDE	CHEMBL2105741	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
VEMURAFENIB	CHEMBL1229517	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets

Targets9

The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.

Target	Protein	Its drugs that act on it	Mechanisms	Highest stage of those drugs	Gene in Genes	Source
BRAF	Serine/threonine-protein kinase B-raf	3	Serine/threonine-protein kinase B-raf inhibitor	Phase 2	https://conductscience.org/library/genes/673	Drugs and targets
MAP2K1	Dual specificity mitogen-activated protein kinase kinase 1	3	Dual specificity mitogen-activated protein kinase kinase 1 inhibitor; Dual specificity mitogen-activated protein kinase kinase; MEK1/2 inhibitor	Phase 2	https://conductscience.org/library/genes/5604	Drugs and targets
MAP2K2	Dual specificity mitogen-activated protein kinase kinase 2	3	Dual specificity mitogen-activated protein kinase kinase 2 inhibitor; Dual specificity mitogen-activated protein kinase kinase; MEK1/2 inhibitor	Phase 2	https://conductscience.org/library/genes/5605	Drugs and targets
CRBN	CRL4(CRBN) E3 ubiquitin ligase	1	CRL4(CRBN) E3 ubiquitin ligase inhibitor; CRL4(CRBN) E3 ubiquitin ligase modulator	Phase 2	https://conductscience.org/library/genes/51185	Drugs and targets
CUL4A	CRL4(CRBN) E3 ubiquitin ligase	1	CRL4(CRBN) E3 ubiquitin ligase inhibitor; CRL4(CRBN) E3 ubiquitin ligase modulator	Phase 2	https://conductscience.org/library/genes/8451	Drugs and targets
DDB1	CRL4(CRBN) E3 ubiquitin ligase	1	CRL4(CRBN) E3 ubiquitin ligase inhibitor; CRL4(CRBN) E3 ubiquitin ligase modulator	Phase 2	https://conductscience.org/library/genes/1642	Drugs and targets
EGFR	Epidermal growth factor receptor	1	Epidermal growth factor receptor erbB1 inhibitor	Phase 2	https://conductscience.org/library/genes/1956	Drugs and targets
IL6R	Interleukin-6 receptor subunit alpha	1	Interleukin-6 receptor alpha subunit inhibitor	Phase 2	https://conductscience.org/library/genes/3570	Drugs and targets
RBX1	CRL4(CRBN) E3 ubiquitin ligase	1	CRL4(CRBN) E3 ubiquitin ligase inhibitor; CRL4(CRBN) E3 ubiquitin ligase modulator	Phase 2	https://conductscience.org/library/genes/9978	Drugs and targets

Trials6

Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT01524978	A Study of Vemurafenib in Participants With BRAF V600 Mutation-Positive Cancers	Phase 2	208	2017-11-20	Multiple Myeloma, Neoplasms	CETUXIMAB, VEMURAFENIB	Its record names the disease	ClinicalTrials.gov
NCT02523040	Lenalidomide for Adult Histiocyte Disorders	Phase 2	12	2025-04-25	Langerhans Cell Histiocytosis (LCH), Histiocytoses Erdheim-chester Disease, Histiocytic Sarcoma (HS)	LENALIDOMIDE	Its record names the disease	ClinicalTrials.gov
NCT02281760	Dabrafenib and Trametinib in People With BRAF V600E Mutation Positive Lesions in Erdheim Chester Disease	Phase 2	9	2021-06-07	BRAF V600E Mutation	DABRAFENIB, TRAMETINIB	Its record names the disease	ClinicalTrials.gov
NCT04079179	Cobimetinib in Refractory Langerhans Cell Histiocytosis (LCH), and Other Histiocytic Disorders	Phase 2	90		Langerhan's Cell Histiocytosis, Juvenile Xanthogranuloma, Erdheim-Chester Disease, Rosai Dorfman Disease, Neuro-Degenerative Disease, Histiocytic Sarcoma, Histiocytic Disorders, Malignant	COBIMETINIB	Its record names the disease	ClinicalTrials.gov
NCT01727206	Pilot Study of Tocilizumab in Patients With Erdheim-Chester Disease	Phase 2	3		Erdheim-Chester Disease	TOCILIZUMAB	Its record names the disease	ClinicalTrials.gov
NCT03794297	Dabrafenib and Trametinib in Treating Patients With Erdheim Chester Disease With BRAF V600 Mutations	Phase 2	0		Erdheim-Chester Disease	DABRAFENIB MESYLATE, TRAMETINIB DIMETHYL SULFOXIDE	Its record names the disease	ClinicalTrials.gov

Comparisons in its trials1

Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.

Comparison	Group 1, with the outcome	Group 2, with the outcome	Participants	Trial
Cohort 3b: Colorectal Cancer - Vemurafenib + Cetuximab vs Pooled Arm - Vemurafenib: serious adverse events	11 of 27	91 of 181	208	NCT01524978

Source Trial comparisons

Papers from its trials4

The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.

PMID	Listed as	Trials listing it	Source
37934000	DERIVED	1	PubMed
36877575	DERIVED	1	PubMed
30351999	DERIVED	1	PubMed
26287849	DERIVED	1	PubMed

Data dates

The date of each source this release read.

Open Targets: 2026-09-18; Open Targets
Drug links and their checks, from Drugs and targets: 2026-09-18; Drugs and targets
ClinicalTrials.gov, from Trials: 2026-11-12; Trials
Comparisons, from Trial comparisons: 2026-03-23; Trial comparisons
PubMed, for the checks: 2026-11-06; PubMed
MeSH, for the checks: 2026-11-10; MeSH

Sources 6

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ChEMBL, through Open TargetsCC BY-SA 3.0
ClinicalTrials.govNo license stated
NLM's MeSHNLM's terms
Open TargetsCC0 1.0
PubMedNLM's terms

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
ClinicalTrials.govnone stated
NLM's MeSHNLM's
Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
PubMedNLM's terms

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this disease from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/diseases/v1/MONDO_0018153

GET https://conductscience.org/api/diseases/v1/MONDO_0018153/drugs

API reference For AI assistants