Histiocytosis
diseaseOn this page
Also known as histiocytic and dendritic cell neoplasmshistiocytic infiltratehistiocytic syndrome
Summary
Histiocytosis (MONDO:0002637) is a disease (an umbrella term covering 5 Mondo subtypes) and 17 clinical trials. Top therapeutic interventions include vemurafenib, fapi ga-68, and plixorafenib. A subtype of lymphatic system disorder — broader associated-gene and molecular evidence is on the parent page (see Disease family below).
At a glance
- Umbrella term: 5 Mondo subtypes
- Clinical trials: 17
Clinical features
No curated clinical features (Orphanet) for this disease.
Identifiers
Disease identifiers
| Field | Value |
|---|---|
| Canonical name | histiocytosis |
| Mondo ID | MONDO:0002637 |
| MeSH | D015614 |
| DOID | DOID:3405 |
| NCIT | C3106 |
| UMLS | C0019618 |
| MedGen | 6845 |
| Is cancer (heuristic) | no |
Also known as: histiocytic and dendritic cell neoplasms · histiocytic infiltrate · histiocytic syndrome
Disease family
This is a subtype of lymphatic system disorder. Genetic, therapeutic, and trial evidence is largely curated at the broader-term level — see the parent page for the associated-gene cohort and molecular evidence.
Classification path: disease › human disease › disease by body system or component › immune system disorder › lymphoid system disorder › lymphatic system disorder › histiocytosis
Related subtypes (13): lymphatic system cancer, bubonic plague, splenic disorder, lymphocele, lymph node disorder, lymphangitis, lymphogranuloma venereum, lymphangiectasis, hemophagocytic syndrome, plastic bronchitis, lymphedema, lymphatic vessel neoplasm, pseudolymphoma
Subtypes (5): reticulohistiocytic granuloma, malignant histiocytosis, non-Langerhans cell histiocytosis, Langerhans cell histiocytosis, ALK+ histiocytosis
Genetics & variants
GWAS landscape
No GWAS associations recorded — common-variant (GWAS) studies don’t cover this disease (typical for Mendelian / rare diseases). See the curated gene cohort and Mendelian overlap below.
Variant details and genetic-evidence tiers
No tiered GWAS variants or ClinVar records for this disease.
Genes & proteins
No associated-gene cohort resolved for this disease. Atlas builds the molecular and therapeutic sections — associated genes, protein families, druggability, pathways, interactions, and drug associations — by aggregating over a disease’s associated genes (resolved via GWAS / GenCC / ClinVar / CIViC), and none resolved here. This is expected for antibody-mediated, autoimmune, or otherwise non-gene-defined conditions; the curated evidence for this disease is its clinical features, GWAS susceptibility, and clinical trials (above).
Function
No pathway enrichment — requires an associated-gene cohort.
Therapeutics
Drugs indicated for this disease
No approved or late-stage (phase ≥3) drug is indicated for this disease; the following are in earlier-phase trials only.
Earlier-phase candidates (phase 2, investigational — efficacy not yet established): Alemtuzumab, Cladribine, Etanercept, Fludarabine Phosphate, Melphalan, Ruxolitinib, Trametinib, Vemurafenib.
Clinical trials & evidence
Clinical trials
Clinical trials: 17.
Phase distribution (across all retrieved trials)
| Phase | Trials |
|---|---|
| Not specified | 8 |
| PHASE2 | 5 |
| PHASE1/PHASE2 | 2 |
| PHASE3 | 1 |
| PHASE1 | 1 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT04943211 | PHASE3 | RECRUITING | Determination of Molecular Status, the Efficacy and Safety of Fluorodeoxyglucose in PET-CT Imaging |
| NCT01966367 | PHASE1/PHASE2 | ACTIVE_NOT_RECRUITING | CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation |
| NCT04943198 | PHASE2 | RECRUITING | Optimization of the Time and Dosage of Vemurafenib in BRAF Positive Juvenile Patients With Refractory Histiocytosis |
| NCT04943224 | PHASE2 | RECRUITING | Optimization of the Time and Dosage of Trametinib in BRAF Negative Juvenile Patients |
| NCT05786924 | PHASE1/PHASE2 | RECRUITING | Phase 1/2 Trial of S241656 in Selected RAS/MAPK Mutation- Positive Malignancies |
| NCT00145626 | PHASE2 | COMPLETED | HLA-Nonidentical Stem Cell and Natural Killer Cell Transplantation for Children Less the Two Years of Age With Hematologic Malignancies |
| NCT01049854 | PHASE2 | COMPLETED | CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant |
| NCT01050439 | PHASE2 | TERMINATED | Unrelated Donor Transplant for Malignant and Non-Malignant Disorders |
| NCT02012231 | PHASE1 | TERMINATED | Phase I/IIa Study to Evaluate the Safety, PK, PD, and Preliminary Efficacy of PLX8394 in Patients With Advanced Cancers. |
| NCT03127709 | Not specified | ACTIVE_NOT_RECRUITING | A Study of Memory, Thinking, and Brain Imaging in Adults With Histiocytosis |
| NCT04665674 | Not specified | RECRUITING | Adult Pulmonary Langerhans Cell Histiocytosis: a National Registry-based Prospective Cohort Study |
| NCT05915208 | Not specified | RECRUITING | Histiocytic Disorder Follow-up Study |
| NCT06573671 | Not specified | ENROLLING_BY_INVITATION | Histiocytosis in Injecting Drug Users |
| NCT01225718 | Not specified | COMPLETED | Ceftriaxone in Non-neutropenic Fever |
| NCT02668120 | Not specified | COMPLETED | Search Elevated Serum Alkaline Phosphatase as a Marker of Prenatal Intervillositis Chronic Histiocytic |
| NCT04437381 | Not specified | UNKNOWN | Molecular Targets for the Treatment of Histiocytosis |
| NCT05803629 | Not specified | UNKNOWN | Characterizing Histiocytosis With 68Ga-FAPI PET/CT |
Drugs tested across these trials (top 30)
| Molecule | Max phase | Trials referencing |
|---|---|---|
| VEMURAFENIB | 4 | 1 |
| FAPI GA-68 | 2 | 1 |
| PLIXORAFENIB | 2 | 1 |
| CHEMBL3415553 | 0 | 1 |
| CHEMBL4209555 | 0 | 1 |
| CHEMBL5433950 | 0 | 1 |
| PLX-4720 | 0 | 1 |
Related Atlas pages
- Drugs: Vemurafenib