Hairy cell leukemia

Summary

Hairy cell leukemia (MONDO:0018935) is a cancer with 2 cohort genes (2 CIViC-evidence somatic drivers) and 44 clinical trials. Molecularly, BRAF V600E confers sensitivity to Vemurafenib in Hairy Cell Leukemia (CIViC Level B); 1 further subtype–drug associations are mapped below. Top therapeutic interventions include rituximab, cladribine, and vemurafenib.

At a glance

• Classification: Cancer
• Prevalence: 1-9 / 1 000 000 (Europe) [Orphanet-validated]
• Cohort genes: 2
• Clinical trials: 44
• Precision-medicine evidence (CIViC): 2 subtype–drug associations

Clinical features

Epidemiology

Prevalence records

9 prevalence record(s), Orphanet:

Type	Class	Value	Geography	Validation
Annual incidence	1-9 / 1 000 000	0.29	Europe	Validated
Lifetime Prevalence	1-9 / 100 000	3.12	Europe	Validated
Annual incidence	1-9 / 1 000 000	0.33	United States	Validated
Annual incidence	1-9 / 1 000 000	0.29	United Kingdom	Validated
Annual incidence	1-9 / 1 000 000	0.47	Iceland	Validated
Annual incidence	<1 / 1 000 000	0.035	Hong Kong	Validated
Annual incidence	1-9 / 1 000 000	0.37	Denmark	Validated
Point prevalence	1-9 / 100 000		Europe	Not yet validated
Annual incidence	1-9 / 1 000 000	0.24	Israel	Not yet validated

Identifiers

Disease identifiers

Field	Value
Canonical name	hairy cell leukemia
Mondo ID	MONDO:0018935
EFO	EFO:1000956
MeSH	D007943
Orphanet	58017
DOID	DOID:285
ICD-10-CM	C91.4
ICD-11	82152208
NCIT	C7402
SNOMED CT	118613001
UMLS	C0023443
MedGen	9727
GARD	0006560
MedDRA	10019053, 10019055
NORD	1213
Is cancer (heuristic)	yes

Also known as: classic hairy cell leukaemia · hairy cell leukemia · HCL · HCL-C · leukemic reticuloendotheliosis

Data availability: 22 cell lines.

Disease family

An umbrella term covering 4 Mondo subtypes.

Classification path: human disease › disease by etiologic mechanism › cancer or benign tumor › neoplastic disease or syndrome › neoplasm › hematopoietic and lymphoid system neoplasm › hematopoietic and lymphoid cell neoplasm › leukemia › chronic leukemia › B-cell chronic lymphocytic leukemia › hairy cell leukemia

Related subtypes (1): chronic lymphocytic leukemia/small lymphocytic lymphoma

Subtypes (4): intrapelvic lymph node leukemic reticuloendotheliosis, splenic manifestation of hairy cell leukemia, refractory hairy cell leukemia, hairy cell leukemia variant

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

Mendelian disease overlap and somatic drivers

GenCC: 0 · Orphanet: 22 · OMIM-shared: 0 · Dual-evidence (GWAS+Mendelian): 0

Somatic driver evidence (intOGen + CIViC, cohort fanout)

Gene	intOGen role	Cancer types	CIViC
BRAF	Act	BLCA,BRCA,CHOL,CLLSLL,COAD,COADREAD,CSCC,DLBCLNOS,GBM,GIST,HGGNOS,LGGNOS,LUAD,MEL,MLYM,NSCLC,OVT,PAST,PCM,PRAD,PRCC,PROSTATE,READ,SACA,SKCM,STAD,UCEC,WDTC	CIViC #5
KRAS	Act	ALL,AML,ANSC,BLADDER,BLCA,BRCA,CEAD,CESC,CHOL,CLLSLL,COAD,COADREAD,DLBCLNOS,EGC,ESCA,ESCC,HCC,LUAD,LUSC,MEL,MGCT,MT,NSCLC,OVT,PAAD,PANCREAS,PAST,PCM,PRAD,PRCC,READ,STAD,STOMACH,UCEC,UCS,WDTC	CIViC #30

Orphanet rare-disease linkage (cohort genes)

Gene	Orphanet ID	Rare disease
BRAF	Orphanet:1340	Cardiofaciocutaneous syndrome
BRAF	Orphanet:146	Differentiated thyroid carcinoma
BRAF	Orphanet:251615	Pilomyxoid astrocytoma
BRAF	Orphanet:389	Langerhans cell histiocytosis
BRAF	Orphanet:500	Noonan syndrome with multiple lentigines
BRAF	Orphanet:54595	Craniopharyngioma
BRAF	Orphanet:58017	Classic hairy cell leukemia
BRAF	Orphanet:626	Large/giant congenital melanocytic nevus
BRAF	Orphanet:648	Noonan syndrome
BRAF	Orphanet:840	Syringocystadenoma papilliferum
BRAF	Orphanet:96253	Cushing disease
KRAS	Orphanet:1333	Familial pancreatic carcinoma
KRAS	Orphanet:1340	Cardiofaciocutaneous syndrome
KRAS	Orphanet:144	Lynch syndrome
KRAS	Orphanet:146	Differentiated thyroid carcinoma
KRAS	Orphanet:2396	Encephalocraniocutaneous lipomatosis
KRAS	Orphanet:251615	Pilomyxoid astrocytoma
KRAS	Orphanet:2612	Linear nevus sebaceus syndrome
KRAS	Orphanet:268114	RAS-associated autoimmune leukoproliferative disease
KRAS	Orphanet:3339	Oculoectodermal syndrome
KRAS	Orphanet:648	Noonan syndrome
KRAS	Orphanet:86834	Juvenile myelomonocytic leukemia

Cohort genes → proteins

2 cohort genes, 2 distinct canonical proteins.

Evidence partition

Subset	Genes
civic_only	2

Cohort genes (full)

Symbol	HGNC	Ensembl	UniProt	Name	Evidence
BRAF	HGNC:1097	ENSG00000157764	P15056	Serine/threonine-protein kinase B-raf	civic_evidence
KRAS	HGNC:6407	ENSG00000133703	P01116	GTPase KRas	civic_evidence

Cohort function summary

Lead sentence per gene, UniProt-curated.

Symbol	Protein name	Function (lead sentence)
BRAF	Serine/threonine-protein kinase B-raf	Protein kinase involved in the transduction of mitogenic signals from the cell membrane to the nucleus.
KRAS	GTPase KRas	Ras proteins bind GDP/GTP and possess intrinsic GTPase activity.

Protein-family classification

Druggable: 2 · Difficult: 0 · Unknown: 0 · Druggable fraction: 1.0

Family distribution

Cohort families vs a genome-wide background (hypergeometric, BH-FDR; fold = observed/expected). Counts kept; sorted by enrichment, so the catch-all Other/Unknown bucket no longer leads.

Family	Genes	Fold	FDR
Kinase	1	13.9×	0.142
Enzyme (other)	1	6.0×	0.160

Per-gene assignment

Symbol	Family	Druggable?	EC	InterPro (top 3)
BRAF	Kinase	yes	2.7.10.2	Prot_kinase_dom, Ser-Thr/Tyr_kinase_cat_dom, PKC_DAG/PE
KRAS	Enzyme (other)	yes	3.6.5.2	Small_GTPase, Small_GTP-bd, Small_GTPase_Ras-type

Expression context

Cohort genes with no expression data: 0.

2 cohort genes are a single-cell marker in ≥1 SCXA experiment.

Breadth distribution (Bgee present_calls)

Bucket	Genes
narrow (1-5 tissues)	0
moderate (6-20)	0
broad (>20)	2
unknown	0

Top tissues across cohort

Tissue	Cohort genes
buccal mucosa cell	1
calcaneal tendon	1
colonic epithelium	1
nipple	1
pylorus	1
trigeminal ganglion	1

Per-gene tissue summary (top 30)

Symbol	Bgee breadth	FANTOM5 breadth	SCXA	Top tissues
BRAF	265	ubiquitous	marker	buccal mucosa cell, colonic epithelium, calcaneal tendon
KRAS	298	ubiquitous	marker	trigeminal ganglion, pylorus, nipple

Protein interactions among cohort

Intra-cohort edges: 1.

Hub genes (top 10 by interactor count)

Symbol	Interactor count
KRAS	14,509
BRAF	7,394

Intra-cohort edges

A	B	Sources
BRAF	KRAS	biogrid_interaction, intact, string_interaction

Structural data

PDB: 2 · AlphaFold-only: 0 · No structure: 0

Cohort genes with PDB structures (top 30)

Symbol	UniProt	PDB entries
KRAS	P01116	511
BRAF	P15056	131

Function

Pathway analysis

Distinct Reactome pathways touched by cohort: 100. Enrichment computed across 2 evidence-associated genes (2 with Reactome annotation).

Pathways by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 2 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

Pathway	Cohort genes	Fold	FDR	Sample cohort genes
RAF activation	2	335.9×	2e-04	BRAF, KRAS
Signaling by high-kinase activity BRAF mutants	2	317.2×	2e-04	BRAF, KRAS
MAP2K and MAPK activation	2	285.5×	2e-04	BRAF, KRAS
Signaling by RAF1 mutants	2	278.5×	2e-04	BRAF, KRAS
Negative regulation of MAPK pathway	2	265.6×	2e-04	BRAF, KRAS
Signaling by moderate kinase activity BRAF mutants	2	253.8×	2e-04	BRAF, KRAS
Paradoxical activation of RAF signaling by kinase inactive BRAF	2	253.8×	2e-04	BRAF, KRAS
Signaling downstream of RAS mutants	2	253.8×	2e-04	BRAF, KRAS
Signaling by BRAF and RAF1 fusions	2	170.4×	4e-04	BRAF, KRAS
RAF/MAP kinase cascade	2	61.1×	0.003	BRAF, KRAS
Signaling by RAS GAP mutants	1	1903.3×	0.004	KRAS
Signaling by RAS GTPase mutants	1	1903.3×	0.004	KRAS
Signaling by MRAS-complex mutants	1	1427.5×	0.005	BRAF
Activation of RAS in B cells	1	1142.0×	0.006	KRAS
Signalling to p38 via RIT and RIN	1	1142.0×	0.006	BRAF
Negative feedback regulation of MAPK pathway	1	951.7×	0.006	BRAF
ARMS-mediated activation	1	815.7×	0.006	BRAF
RAS signaling downstream of NF1 loss-of-function variants	1	815.7×	0.006	KRAS
Estrogen-stimulated signaling through PRKCZ	1	815.7×	0.006	KRAS
SOS-mediated signalling	1	713.8×	0.006	KRAS
Prolonged ERK activation events	1	713.8×	0.006	BRAF
SHOC2 M1731 mutant abolishes MRAS complex function	1	713.8×	0.006	BRAF
Gain-of-function MRAS complexes activate RAF signaling	1	713.8×	0.006	BRAF
Activated NTRK3 signals through RAS	1	634.4×	0.006	KRAS
EGFR Transactivation by Gastrin	1	571.0×	0.006	KRAS
SHC-related events triggered by IGF1R	1	571.0×	0.006	KRAS
Signaling by FGFR3	1	571.0×	0.006	BRAF
RUNX3 regulates p14-ARF	1	571.0×	0.006	KRAS
Activated NTRK2 signals through RAS	1	571.0×	0.006	KRAS
Signaling by FGFR4	1	519.1×	0.006	BRAF

GO biological processes by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 2 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

GO term	Cohort genes	Fold	FDR	Sample cohort genes
visual learning	2	306.4×	7e-04	BRAF, KRAS
MAPK cascade	2	153.2×	0.001	BRAF, KRAS
negative regulation of neuron apoptotic process	2	110.9×	0.002	BRAF, KRAS
response to mineralocorticoid	1	8426.0×	0.002	KRAS
forebrain astrocyte development	1	2808.7×	0.004	KRAS
CD4-positive or CD8-positive, alpha-beta T cell lineage commitment	1	2808.7×	0.004	BRAF
response to isolation stress	1	2106.5×	0.004	KRAS
positive regulation of axon regeneration	1	1685.2×	0.004	BRAF
negative regulation of synaptic vesicle exocytosis	1	1685.2×	0.004	BRAF
response to gravity	1	1404.3×	0.004	KRAS
CD4-positive, alpha-beta T cell differentiation	1	1404.3×	0.004	BRAF
positive regulation of gene expression	2	38.7×	0.004	BRAF, KRAS
myeloid progenitor cell differentiation	1	1203.7×	0.004	BRAF
positive regulation of D-glucose transmembrane transport	1	1053.2×	0.004	BRAF
head morphogenesis	1	1053.2×	0.004	BRAF
establishment of protein localization to membrane	1	936.2×	0.004	BRAF
negative regulation of fibroblast migration	1	766.0×	0.005	BRAF
type I pneumocyte differentiation	1	766.0×	0.005	KRAS
myoblast proliferation	1	702.2×	0.005	KRAS
endothelial cell apoptotic process	1	648.1×	0.005	BRAF
positive regulation of cellular senescence	1	648.1×	0.005	KRAS
negative regulation of epithelial cell differentiation	1	601.9×	0.005	KRAS
regulation of T cell differentiation	1	601.9×	0.005	BRAF
regulation of synaptic transmission, GABAergic	1	526.6×	0.005	KRAS
regulation of long-term neuronal synaptic plasticity	1	495.6×	0.005	KRAS
striated muscle cell differentiation	1	495.6×	0.005	KRAS
glial cell proliferation	1	443.5×	0.005	KRAS
epithelial tube branching involved in lung morphogenesis	1	421.3×	0.005	KRAS
face development	1	401.2×	0.005	BRAF
synaptic vesicle exocytosis	1	383.0×	0.005	BRAF

Therapeutics

Drugs indicated for this disease

4 approved. Disease-direct ChEMBL indications, not inferred from the associated-gene cohort below.

Drug	Development status
Cladribine	Approved (phase 4)
INTERFERON ALFA-2B	Approved (phase 4)
Moxetumomab Pasudotox	Approved (phase 4)
Pentostatin	Approved (phase 4)

Earlier-phase candidates (phase 2, investigational — efficacy not yet established): Acetaminophen, Bendamustine, Binimetinib, Diphenhydramine, Encorafenib, Epinephrine, INTERFERON ALFA-2A, Obinutuzumab, Rituximab, Vemurafenib.

Drug target analysis

Approved (phase 4): 2 · Phase ≥3: 2 · Phased (≥1): 2 · Undrugged: 0

Druggability breadth: 2 of 2 evidence-associated genes (100%) have a ChEMBL target (buckets above are over the deeply-mined display cohort).

Genes with an approved drug

The molecule shown is one approved compound that hits the gene — not necessarily a drug of choice or one indicated for this disease.

Symbol	Example approved molecule
BRAF	VEMURAFENIB
KRAS	VEMURAFENIB

Top cohort targets by molecule count

Symbol	Molecules	Max phase
BRAF	48	4
KRAS	11	4

Drugs targeting cohort genes (top 30)

Molecule	Max phase	Targets in cohort
VEMURAFENIB	4	BRAF, KRAS
PONATINIB	4	BRAF
FEDRATINIB	4	BRAF
SORAFENIB	4	BRAF
DASATINIB ANHYDROUS	4	BRAF
RUXOLITINIB	4	BRAF
INFIGRATINIB PHOSPHATE	4	BRAF
INFIGRATINIB	4	BRAF
REGORAFENIB	4	BRAF
DABRAFENIB	4	BRAF, KRAS
COBIMETINIB	4	BRAF
NILOTINIB	4	BRAF
ABEMACICLIB	4	BRAF
ENCORAFENIB	4	BRAF
TOVORAFENIB	4	BRAF
PAZOPANIB	4	BRAF
DASATINIB	4	BRAF
ERLOTINIB	4	BRAF
GEFITINIB	4	BRAF
IMATINIB	4	BRAF
LONAFARNIB	4	KRAS
SOTORASIB	4	KRAS
ADAGRASIB	4	KRAS
MASITINIB	3	BRAF
AVUTOMETINIB	3	BRAF
NAPORAFENIB	3	BRAF
QUERCETIN	3	BRAF
MOTESANIB	3	BRAF
OPNURASIB	3	KRAS
DORAMAPIMOD	2	BRAF

Bioactivity and enzyme data

Enzyme cohort genes (≥1 EC): 2.

Cohort genes with ChEMBL bioactivity (full, sorted by assay count)

Symbol	Assays	Type breakdown
BRAF	1,442	Binding:1400, Functional:37, ADMET:5
KRAS	861	Binding:829, Functional:32

Cohort enzymes (BRENDA EC)

Symbol	EC numbers	Names
BRAF	2.7.10.2, 2.7.11.1	non-specific protein-tyrosine kinase, non-specific serine/threonine protein kinase
KRAS	3.6.5.2	small monomeric GTPase

Cohort genes with high screening signal

≥100 ChEMBL assays — a studied-ness signal; see Therapeutics for approved-drug status.

Symbol	ChEMBL assays
BRAF	1,442
KRAS	861

Pharmacogenomics

Cohort genes with a PharmGKB record: 2; with CPIC/DPWG dosing guidelines: 0.

No cohort gene has a CPIC/DPWG genotype-guided dosing guideline (PharmGKB).

Drug repurposing candidates

27 approved/phased drugs hit cohort targets but don’t yet appear in disease-level clinical trials. Target-inhibition rationale is strongest for cancer driver genes; a bioactivity hit is a screening signal, not a treatment claim.

Compound	Max phase	Cohort target (bioactivity)
PONATINIB	4	BRAF
FEDRATINIB	4	BRAF
SORAFENIB	4	BRAF
DASATINIB ANHYDROUS	4	BRAF
RUXOLITINIB	4	BRAF
INFIGRATINIB PHOSPHATE	4	BRAF
INFIGRATINIB	4	BRAF
REGORAFENIB	4	BRAF
DABRAFENIB	4	BRAF, KRAS
COBIMETINIB	4	BRAF
NILOTINIB	4	BRAF
ABEMACICLIB	4	BRAF
PAZOPANIB	4	BRAF
DASATINIB	4	BRAF
ERLOTINIB	4	BRAF
GEFITINIB	4	BRAF
IMATINIB	4	BRAF
LONAFARNIB	4	KRAS
SOTORASIB	4	KRAS
ADAGRASIB	4	KRAS
MASITINIB	3	BRAF
AVUTOMETINIB	3	BRAF
NAPORAFENIB	3	BRAF
QUERCETIN	3	BRAF
MOTESANIB	3	BRAF
OPNURASIB	3	KRAS
DORAMAPIMOD	2	BRAF

Druggability pyramid

Cohort genes binned by druggability tier (high → low):

Tier	Definition	Genes	Symbols
A	Approved (phase 4 drug)	2	BRAF, KRAS
B	Phased (≥1) drug, not yet approved	0
C	Druggable family + PDB, no drug	0
D	Druggable family + AlphaFold only, no drug	0
E	Difficult family or no structure, no drug	0

Undrugged target profiles

0 cohort genes are undrugged. Ranked by ‘starting-point quality’ (assay depth + drugged-partner adjacency).

Clinical trials & evidence

Clinical trials

Clinical trials: 44.

Phase distribution (across all retrieved trials)

Phase	Trials
PHASE2	16
Not specified	13
PHASE1	11
PHASE1/PHASE2	3
PHASE2/PHASE3	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT02131753	PHASE2/PHASE3	RECRUITING	Therapy Optimisation for the Treatment of Hairy Cell Leukemia
NCT00412594	PHASE2	RECRUITING	Cladribine and Rituximab in Treating Patients With Hairy Cell Leukemia
NCT00923013	PHASE2	ACTIVE_NOT_RECRUITING	Cladribine With Simultaneous or Delayed Rituximab to Treat Hairy Cell Leukemia
NCT01059786	PHASE2	ACTIVE_NOT_RECRUITING	Randomized Phase II Trial of Rituximab With Either Pentostatin or Bendamustine for Multiply Relapsed or Refractory Hairy Cell Leukemia
NCT01841723	PHASE2	ACTIVE_NOT_RECRUITING	Ibrutinib in Treating Patients With Relapsed Hairy Cell Leukemia
NCT03410875	PHASE2	ACTIVE_NOT_RECRUITING	A Phase II Study of the BRAF Inhibitor, Vemurafenib, Plus Obinutuzumab in Patients With Previously Untreated Classical Hairy Cell Leukemia
NCT04322383	PHASE2	RECRUITING	Binimetinib for People With Relapsed/Refractory BRAF Wild Type Hairy Cell Leukemia and Variant
NCT04324112	PHASE2	RECRUITING	Encorafenib Plus Binimetinib for People With BRAF V600 Mutated Relapsed/Refractory HCL
NCT06311227	PHASE2	RECRUITING	Venetoclax for the Treatment of Patients With Relapsed Hairy Cell Leukemia
NCT06561360	PHASE2	RECRUITING	A Study of Vemurafenib and Obinutuzumab Compared to Cladribine and Rituximab in People With Hairy Cell Leukemia (HCL)
NCT06965114	PHASE1/PHASE2	RECRUITING	Testing the Combination of Anti-cancer Drugs, Tovorafenib Plus Rituximab, in Patients With Hairy Cell Leukemia
NCT00001567	PHASE2	COMPLETED	A Phase II Efficacy Study of Roferon-A in Hairy Cell Leukemia
NCT00321555	PHASE2	COMPLETED	LMB-2 to Treat Hairy Cell Leukemia
NCT00924040	PHASE2	TERMINATED	Retreatment Protocol for BL22 Immunotherapy in Relapsed or Refractory Hairy Cell Leukemia
NCT01711632	PHASE2	COMPLETED	BRAF Inhibitor, Vemurafenib, in Patients With Relapsed or Refractory Hairy Cell Leukemia
NCT02157181	PHASE2	COMPLETED	Treatment of Hairy Cell Leukaemia Variant and Relapsing Hairy Cell Leukaemia With Cladribine Plus Rituximab
NCT02362035	PHASE1/PHASE2	COMPLETED	ACP-196 (Acalabrutinib) in Combination With Pembrolizumab, for Treatment of Hematologic Malignancies
NCT03010358	PHASE1/PHASE2	COMPLETED	Entospletinib and Obinutuzumab in Treating Patients With Relapsed Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma, or Non-Hodgkin Lymphoma
NCT03739606	PHASE2	WITHDRAWN	Flotetuzumab in Treating Patients With Recurrent or Refractory CD123 Positive Blood Cancer
NCT05388123	PHASE2	COMPLETED	Low Dose Vemurafenib and Rituximab in Hairy Cell Leukemia
NCT02153580	PHASE1	ACTIVE_NOT_RECRUITING	Cellular Immunotherapy Following Chemotherapy in Treating Patients With Recurrent Non-Hodgkin Lymphomas, Chronic Lymphocytic Leukemia, or B-Cell Prolymphocytic Leukemia
NCT03805932	PHASE1	ACTIVE_NOT_RECRUITING	Moxetumomab Pasudotox-tdfk (Lumoxiti(TM)) and Either Rituximab (Rituxan(R)) or Ruxience for Relapsed Hairy Cell Leukemia
NCT04775745	PHASE1	RECRUITING	Study of Oral Administration of LP-168 in Patients With Relapsed or Refractory B-cell Malignancies.
NCT04815356	PHASE1	RECRUITING	Phase I Study of Anti-CD22 Chimeric Receptor T Cells in Patients With Relapsed/Refractory Hairy Cell Leukemia and Variant
NCT06340737	PHASE1	RECRUITING	AutologousCD22 Chimeric Antigen Receptor (CAR)T Cells in w/Recurrent/Refractory B Cell Lymphomas
NCT00462189	PHASE1	UNKNOWN	Safety Study of CAT-8015 Immunooxin in Patients With HCL With Advance Disease
NCT00586924	PHASE1	COMPLETED	A Phase I, Multicenter Dose Escalation Study in Patients With Hairy Cell Leukemia
NCT00608361	PHASE1	COMPLETED	Dasatinib in Treating Patients With Solid Tumors or Lymphomas That Are Metastatic or Cannot Be Removed By Surgery
NCT02012231	PHASE1	TERMINATED	Phase I/IIa Study to Evaluate the Safety, PK, PD, and Preliminary Efficacy of PLX8394 in Patients With Advanced Cancers.
NCT04578600	PHASE1	COMPLETED	CC-486, Lenalidomide, and Obinutuzumab for the Treatment of Recurrent or Refractory CD20 Positive B-cell Lymphoma
NCT04681105	PHASE1	COMPLETED	Flotetuzumab for the Treatment of Relapsed or Refractory Advanced CD123-Positive Hematological Malignancies
NCT01087333	Not specified	RECRUITING	Collection of Human Samples to Study Hairy Cell and Other Leukemias, and to Develop Recombinant Immunotoxins for Cancer Treatment
NCT02863692	Not specified	RECRUITING	Registry of the German CLL Study Group
NCT05859932	Not specified	NOT_YET_RECRUITING	Assessing Medical Trial Experiences of Hairy Cell Leukemia Patients
NCT06764524	Not specified	RECRUITING	Hairy Cell Leukemia: Harnessing the Full Power of Extracellular Vesicles to Improve Patient Care Management
NCT06774677	Not specified	RECRUITING	Decoding the Extracellular Vesicles-driven Communication in the Microenvironment of Hairy Cell Leukemia to Improve Patient Care Management
NCT06781515	Not specified	RECRUITING	Assessment of Disease Burden in Hairy Cell Leukemia
NCT00898079	Not specified	COMPLETED	Collecting and Storing Malignant, Borderline Malignant Neoplasms, and Related Samples From Young Patients With Cancer
NCT01720758	Not specified	COMPLETED	Assessment of the V600E Mutation in the B-RAF Gene in Chronic Lymphoproliferative Disease
NCT02883946	Not specified	COMPLETED	Rituximab in Hairy Cell Leukemia: a Multicenter Retrospective Study

Drugs tested across these trials (top 30)

Molecule	Max phase	Trials referencing
RITUXIMAB	4	6
CLADRIBINE	4	4
VEMURAFENIB	4	3
BENDAMUSTINE	4	2
BINIMETINIB	4	2
MOXETUMOMAB PASUDOTOX	4	2
ACALABRUTINIB	4	1
DIPHENHYDRAMINE	4	1
ENCORAFENIB	4	1
FAMOTIDINE	4	1
IBRUTINIB	4	1
IBUPROFEN	4	1
INTERFERON ALFA-2A	4	1
OBINUTUZUMAB	4	1
PENTOSTATIN	4	1
RANITIDINE	4	1
TOVORAFENIB	4	1
ENTOSPLETINIB	3	1
BL22	2	1
FLOTETUZUMAB	2	1
PLIXORAFENIB	2	1
RACEPINEPHRINE	2	1
CHEMBL4776881	0	4
CHEMBL3415553	0	3
CHEMBL4209555	0	3
PLX-4720	0	3
CHEMBL3647964	0	1
CHEMBL4466205	0	1
CHEMBL4747506	0	1
CHEMBL5187554	0	1

Precision-medicine subtype map (CIViC)

Drug × molecular subtype: 2 predictive associations from 8 curated evidence items; also 1 diagnostic.

Molecular subtype	Therapy	Effect	Level	CIViC
BRAF V600E	Vemurafenib	Sensitivity/Response	CIViC B	EID11315 +6
KRAS G12D	Vemurafenib	Resistance	CIViC C	EID1580