Disease · MONDO_0015540

hemophagocytic syndrome

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. hemophagocytic syndrome. Diseases, record MONDO_0015540. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0015540

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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: hemophagocytic syndrome; Open Targets
Open Targets ID: MONDO_0015540; Open Targets
Other names: HLH, Hemophagocytic Lymphohistiocytosis; Open Targets
Disease Ontology: DOID:0050120; Disease Ontology
NCI Thesaurus: C34792; NCI Thesaurus
NCI Thesaurus: C35439; NCI Thesaurus
Orphanet: ORPHA:158032; Orphanet
Highest stage of its drugs: Phase 3, for 22 of its 42 drugs, from ClinicalTrials.gov; Open Targets

Drugs42

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
ETOPOSIDE	CHEMBL44657	Small molecule	Phase 3	ClinicalTrials.gov	23	23	23	Drugs and targets
DEXAMETHASONE	CHEMBL384467	Small molecule	Phase 3	ClinicalTrials.gov	12	12	12	Drugs and targets
METHYLPREDNISOLONE	CHEMBL650	Small molecule	Phase 3	ClinicalTrials.gov	11	11	11	Drugs and targets
RUXOLITINIB	CHEMBL1789941	Small molecule	Phase 3	ClinicalTrials.gov	8	8	8	Drugs and targets
CYCLOPHOSPHAMIDE	CHEMBL1200796	Small molecule	Phase 3	ClinicalTrials.gov	6	6	6	Drugs and targets
DOXORUBICIN	CHEMBL53463	Small molecule	Phase 3	ClinicalTrials.gov	6	6	6	Drugs and targets
CYCLOSPORINE	CHEMBL160	Protein	Phase 3	ClinicalTrials.gov	5	5	5	Drugs and targets
EMAPALUMAB	CHEMBL3989977	Antibody	Phase 3	ClinicalTrials.gov	5	5	5	Drugs and targets
PEGASPARGASE	CHEMBL2108546	Enzyme	Phase 3	ClinicalTrials.gov	5	5	4	Drugs and targets
ANTILYMPHOCYTE IMMUNOGLOBULIN (HORSE)	CHEMBL2108981		Phase 3	ClinicalTrials.gov	4	4	0	Drugs and targets

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See all 42 drugs

Targets55

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
TOP2A	DNA topoisomerase 2-alpha	5	DNA topoisomerase II alpha inhibitor; DNA topoisomerase II inhibitor	Phase 3	https://conductscience.org/library/genes/7153	Drugs and targets
NR3C1	Glucocorticoid receptor	4	Glucocorticoid receptor agonist	Phase 3	https://conductscience.org/library/genes/2908	Drugs and targets
JAK1	Tyrosine-protein kinase JAK1	2	Tyrosine-protein kinase JAK1 inhibitor	Phase 3	https://conductscience.org/library/genes/3716	Drugs and targets
PDCD1	Programmed cell death protein 1	2	Programmed cell death protein 1 antagonist	Phase 2	https://conductscience.org/library/genes/5133	Drugs and targets
POLA1	DNA polymerase (alpha/delta/epsilon)	2	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2	https://conductscience.org/library/genes/5422	Drugs and targets
POLA2	DNA polymerase (alpha/delta/epsilon)	2	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2	https://conductscience.org/library/genes/23649	Drugs and targets
POLD1	DNA polymerase (alpha/delta/epsilon)	2	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2	https://conductscience.org/library/genes/5424	Drugs and targets
POLD2	DNA polymerase (alpha/delta/epsilon)	2	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2	https://conductscience.org/library/genes/5425	Drugs and targets
POLD3	DNA polymerase (alpha/delta/epsilon)	2	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2	https://conductscience.org/library/genes/10714	Drugs and targets
POLD4	DNA polymerase (alpha/delta/epsilon)	2	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2	https://conductscience.org/library/genes/57804	Drugs and targets

1 to 10 of 55

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Trials48

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. 1 more is not shown, since the checks do not confirm it.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT02069899	A Study for Long-term Follow-up of Hemophagocytic Lymphohistiocytosis (HLH) Participants Who Received Treatment With Emapalumab (NI-0501), an Anti-interferon Gamma Monoclonal Antibody	Phase 2/3	58	2022-06-28	Hemophagocytic Lymphohistiocytosis	EMAPALUMAB	Its record names the disease	ClinicalTrials.gov
NCT01998633	Reduced Intensity Conditioning for Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (BMT CTN 1204)	Phase 2	47	2018-06-01	Hemophagocytic Lymphohistiocytosis, Chronic Active Epstein-Barr Virus Infection, Chronic Granulomatous Disease, HIGM-1, Leukocyte Adhesion Deficiency, IPEX	ALEMTUZUMAB, FLUDARABINE, MELPHALAN	Its record names the disease	ClinicalTrials.gov
NCT00950846	Umbilical Cord Blood Transplant for Congenital Pediatric Disorders	Not applicable	40	2023-10-30	Congenital Pediatric Disorders	BUSULFAN, CYCLOPHOSPHAMIDE, FLUDARABINE	Its record names the disease	ClinicalTrials.gov
NCT03312751	Study to Assess the Efficacy and Safety of Emapalumab in Primary Haemophagocytic Lymphohistiocytosis	Phase 3	35	2024-03-12	Primary Hemophagocytic Lymphohistiocytosis	EMAPALUMAB	Its record names the disease	ClinicalTrials.gov
NCT01104025	Hybrid Immunotherapy for Hemophagocytic LymphoHistiocytosis	Phase 2	31	2020-10-19	Hemophagocytic Lymphohistiocytosis	DEXAMETHASONE, ETOPOSIDE	Its record names the disease	ClinicalTrials.gov
NCT02385110	Alemtuzumab or Tocilizumab in Combination With Etoposide and Dexamethasone for the Treatment of Adult Patients With Hemophagocytic Lymphohistiocytosis	Phase 2	18	2024-01-17	Leukemia	ALEMTUZUMAB, DEXAMETHASONE, ETOPOSIDE, METHOTREXATE, TOCILIZUMAB	Its record names the disease	ClinicalTrials.gov
NCT03985423	A Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Emapalumab in Adult Patients With HLH	Phase 2/3	7	2023-10-06	Hemophagocytic Lymphohistiocytoses	EMAPALUMAB	Its record names the disease	ClinicalTrials.gov
NCT02400463	A Pilot Study of Ruxolitinib in Secondary Hemophagocytic Syndrome	Phase 2	6	2020-11-18	Hemophagocytic Syndrome (HPS)	RUXOLITINIB	Its record names the disease	ClinicalTrials.gov
NCT00426101	Treatment Protocol for Hemophagocytic Lymphohistiocytosis 2004	Phase 3	368		Hemophagocytic Lymphohistiocytosis	CYCLOSPORINE, DEXAMETHASONE, ETOPOSIDE	Its record names the disease	ClinicalTrials.gov
NCT00334672	Combination Chemotherapy Followed By Donor Stem Cell Transplant in Treating Patients With Hemophagocytic Lymphohistiocytosis	Phase 3	288		Nonneoplastic Condition	BUSULFAN, CYCLOPHOSPHAMIDE, CYCLOSPORINE, DEXAMETHASONE, ETOPOSIDE, HYDROCORTISONE, METHOTREXATE, MYCOPHENOLATE MOFETIL	Its record names the disease	ClinicalTrials.gov

1 to 10 of 48

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Comparisons in its trials3

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	Source
Group 1: Alemtuzumab + Etoposide + Dexamethasone vs Group 2: Etoposide + Dexamethasone + Tocilizumab: deaths from any cause	1 of 2	7 of 16	18	NCT02385110	Trial comparisons
Group 1: Alemtuzumab + Etoposide + Dexamethasone vs Group 2: Etoposide + Dexamethasone + Tocilizumab: serious adverse events	1 of 2	9 of 16	18	NCT02385110	Trial comparisons
Group 1: Alemtuzumab + Etoposide + Dexamethasone vs Group 2: Etoposide + Dexamethasone + Tocilizumab: Number of Participants With a Response to Alemtuzumab or Tocilizumab in Combination With Etoposide and Dexamethasone for the Treatment of Adult Patients With Hemophagocytic Lymphohistiocytosis	1 of 1	8 of 8	9	NCT02385110	Trial comparisons

Papers from its trials16

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
32374962	RESULT	1	PubMed
28935695	RESULT	1	PubMed
26289641	RESULT	1	PubMed
42628036	DERIVED	1	PubMed
42241708	DERIVED	1	PubMed
39040100	DERIVED	1	PubMed
38662147	DERIVED	1	PubMed
37042921	DERIVED	1	PubMed
37001971	DERIVED	1	PubMed
36453638	DERIVED	1	PubMed

1 to 10 of 16

See all 16 papers from its trials

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_0015540

GET https://conductscience.org/api/diseases/v1/MONDO_0015540/drugs?page=2

API reference For AI assistants