Hematopoietic and lymphoid cell neoplasm

Summary

Hematopoietic and lymphoid cell neoplasm (MONDO:0044881) is a cancer (an umbrella term covering 8 Mondo subtypes) with 4 cohort genes (4 CIViC-evidence somatic drivers) and 786 clinical trials. Molecularly, ETV6::NTRK3 Fusion confers sensitivity to Larotrectinib in Hematologic Cancer (CIViC Level C); 4 further subtype–drug associations are mapped below. Top therapeutic interventions include trametinib, 2-mercaptoethanesulfonic acid, and afatinib.

At a glance

• Classification: Cancer
• Umbrella term: 8 Mondo subtypes
• Cohort genes: 4
• Clinical trials: 786
• Precision-medicine evidence (CIViC): 5 subtype–drug associations

Clinical features

No curated clinical features (Orphanet) for this disease.

Identifiers

Disease identifiers

Field	Value
Canonical name	hematopoietic and lymphoid cell neoplasm
Mondo ID	MONDO:0044881
NCIT	C27134
UMLS	C0376544
MedGen	91264
GARD	0025916
Is cancer (heuristic)	yes

Also known as: haematological neoplasm · haematological tumour · haematopoietic and lymphoid neoplasms · haematopoietic cancer · haematopoietic cell tumour · haematopoietic malignancy, NOS · haematopoietic neoplasm · haematopoietic neoplasms including lymphomas · haematopoietic tumour · hematologic cancer · hematologic malignancy · hematologic neoplasm · hematological neoplasm · hematological tumor · hematopoietic and lymphoid cell neoplasm · hematopoietic and lymphoid neoplasms · hematopoietic cancer · hematopoietic cell tumor · hematopoietic malignancy, NOS · hematopoietic neoplasm (+7 more)

Disease family

An umbrella term covering 8 Mondo subtypes.

Classification path: disease › 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

Related subtypes (8): cavernous hemangioma, thymus neoplasm, bone marrow neoplasm, STAT3-related early-onset multisystem autoimmune disease, myeloid hemopathy, lymphoid hemopathy, lymph node neoplasm, spleen neoplasm

Subtypes (8): central nervous system hematopoietic neoplasm, refractory hematologic cancer, leukemia, lymphoid neoplasm, myeloid neoplasm, histiocytic and dendritic cell neoplasm, myeloid/lymphoid neoplasms associated with eosinophilia and abnormality of PDGFRA, PDGFRB, FGFR1 or JAK2, myelodysplastic syndrome with excess blasts

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: 24 · OMIM-shared: 0 · Dual-evidence (GWAS+Mendelian): 0

Somatic driver evidence (intOGen + CIViC, cohort fanout)

Gene	intOGen role	Cancer types	CIViC
DDX41			CIViC #11932
EGFR	Act	BRCA,COADREAD,GB,GBM,HGGNOS,LGGNOS,LUAD,LUSC,NSCLC,PAST,PCM,READ,SIC	CIViC #19
FGFR3	Act	BLADDER,BLCA,HNSC,LUSC,PCM,PLMESO,UTUC	CIViC #23
ATM	LoF	BLCA,BRCA,CCRCC,CHOL,CLLSLL,COAD,COADREAD,ESCA,HCC,LUAD,LUSC,MEL,NSCLC,PAAD,PANCREAS,PANET,PCM,PLMESO,PRAD,PROSTATE,STAD,UCEC,UTUC,WDTC	CIViC #69

Orphanet rare-disease linkage (cohort genes)

Gene	Orphanet ID	Rare disease
DDX41	Orphanet:488647	DDX41-related hematologic malignancy predisposition syndrome
EGFR	Orphanet:251576	Gliosarcoma
EGFR	Orphanet:251579	Giant cell glioblastoma
FGFR3	Orphanet:15	Achondroplasia
FGFR3	Orphanet:1860	Thanatophoric dysplasia type 1
FGFR3	Orphanet:2363	Lacrimoauriculodentodigital syndrome
FGFR3	Orphanet:251576	Gliosarcoma
FGFR3	Orphanet:251579	Giant cell glioblastoma
FGFR3	Orphanet:35099	Non-syndromic bicoronal craniosynostosis
FGFR3	Orphanet:429	Hypochondroplasia
FGFR3	Orphanet:53271	Muenke syndrome
FGFR3	Orphanet:794	Saethre-Chotzen syndrome
FGFR3	Orphanet:85164	Camptodactyly-tall stature-scoliosis-hearing loss syndrome
FGFR3	Orphanet:85165	Severe achondroplasia-developmental delay-acanthosis nigricans syndrome
FGFR3	Orphanet:93262	Crouzon syndrome-acanthosis nigricans syndrome
FGFR3	Orphanet:93274	Thanatophoric dysplasia type 2
ATM	Orphanet:100	Ataxia-telangiectasia
ATM	Orphanet:1331	Familial prostate cancer
ATM	Orphanet:145	Hereditary breast and/or ovarian cancer syndrome
ATM	Orphanet:227535	Hereditary breast cancer
ATM	Orphanet:370109	Ataxia-telangiectasia variant
ATM	Orphanet:440437	Familial colorectal cancer Type X
ATM	Orphanet:52416	Mantle cell lymphoma
ATM	Orphanet:67038	B-cell chronic lymphocytic leukemia

Cohort genes → proteins

4 cohort genes, 4 distinct canonical proteins.

Evidence partition

Subset	Genes
civic_only	4

Cohort genes (full)

Symbol	HGNC	Ensembl	UniProt	Name	Evidence
DDX41	HGNC:18674	ENSG00000183258	Q9UJV9	Probable ATP-dependent RNA helicase DDX41	civic_evidence
EGFR	HGNC:3236	ENSG00000146648	P00533	Epidermal growth factor receptor	civic_evidence
FGFR3	HGNC:3690	ENSG00000068078	P22607	Fibroblast growth factor receptor 3	civic_evidence
ATM	HGNC:795	ENSG00000149311	Q13315	Serine-protein kinase ATM	civic_evidence

Cohort function summary

Lead sentence per gene, UniProt-curated.

Symbol	Protein name	Function (lead sentence)
DDX41	Probable ATP-dependent RNA helicase DDX41	Multifunctional protein that participates in many aspects of cellular RNA metabolism.
EGFR	Epidermal growth factor receptor	Receptor tyrosine kinase binding ligands of the EGF family and activating several signaling cascades to convert extracellular cues into appropriate cellular responses.
FGFR3	Fibroblast growth factor receptor 3	Tyrosine-protein kinase that acts as a cell-surface receptor for fibroblast growth factors and plays an essential role in the regulation of cell proliferation, differentiation and apoptosis.
ATM	Serine-protein kinase ATM	Serine/threonine protein kinase which activates checkpoint signaling upon double strand breaks (DSBs), apoptosis and genotoxic stresses such as ionizing ultraviolet A light (UVA), thereby acting as a DNA damage sensor.

Protein-family classification

Druggable: 3 · Difficult: 0 · Unknown: 1 · Druggable fraction: 0.75

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	3	20.8×	4e-04
Other/Unknown	1	0.5×	0.962

Per-gene assignment

Symbol	Family	Druggable?	EC	InterPro (top 3)
DDX41	Other/Unknown	no		Helicase_C-like, DEAD/DEAH_box_helicase_dom, Helicase_ATP-bd
EGFR	Kinase	yes	2.7.10.1	Rcpt_L-dom, Prot_kinase_dom, Ser-Thr/Tyr_kinase_cat_dom
FGFR3	Kinase	yes	2.7.10.1	Prot_kinase_dom, Ser-Thr/Tyr_kinase_cat_dom, Ig_sub2
ATM	Kinase	yes	2.7.11.1	PI3/4_kinase_cat_dom, PIK-rel_kinase_FAT, FATC_dom

Expression context

Cohort genes with no expression data: 0.

4 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)	4
unknown	0

Top tissues across cohort

Tissue	Cohort genes
granulocyte	1
lower esophagus mucosa	1
right frontal lobe	1
gingiva	1
gingival epithelium	1
nipple	1
skin of hip	1
upper arm skin	1
upper leg skin	1
calcaneal tendon	1
colonic epithelium	1
corpus callosum	1

Per-gene tissue summary (top 30)

Symbol	Bgee breadth	FANTOM5 breadth	SCXA	Top tissues
DDX41	274	ubiquitous	marker	granulocyte, right frontal lobe, lower esophagus mucosa
EGFR	285	ubiquitous	marker	nipple, gingiva, gingival epithelium
FGFR3	262	broad	marker	upper leg skin, skin of hip, upper arm skin
ATM	286	ubiquitous	marker	calcaneal tendon, colonic epithelium, corpus callosum

Protein interactions among cohort

Intra-cohort edges: 0.

Hub genes (top 10 by interactor count)

Symbol	Interactor count
EGFR	18,421
ATM	7,383
FGFR3	4,510
DDX41	2,388

Structural data

PDB: 4 · AlphaFold-only: 0 · No structure: 0

Cohort genes with PDB structures (top 30)

Symbol	UniProt	PDB entries
EGFR	P00533	388
FGFR3	P22607	15
ATM	Q13315	14
DDX41	Q9UJV9	5

Function

Pathway analysis

Distinct Reactome pathways touched by cohort: 114. Enrichment computed across 4 evidence-associated genes (4 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 4 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

Pathway	Cohort genes	Fold	FDR	Sample cohort genes
t(4;14) translocations of FGFR3	1	2855.0×	0.014	FGFR3
Signaling by FGFR3 fusions in cancer	1	2855.0×	0.014	FGFR3
Constitutive Signaling by Aberrant PI3K in Cancer	2	63.4×	0.014	EGFR, FGFR3
PLCG1 events in ERBB2 signaling	1	713.8×	0.017	EGFR
Sensing of DNA Double Strand Breaks	1	475.8×	0.017	ATM
FGFR3b ligand binding and activation	1	407.9×	0.017	FGFR3
PTK6 promotes HIF1A stabilization	1	407.9×	0.017	EGFR
Inhibition of Signaling by Overexpressed EGFR	1	317.2×	0.017	EGFR
EGFR interacts with phospholipase C-gamma	1	285.5×	0.017	EGFR
EGFR Transactivation by Gastrin	1	285.5×	0.017	EGFR
STING mediated induction of host immune responses	1	259.6×	0.017	DDX41
Signaling by activated point mutants of FGFR3	1	237.9×	0.017	FGFR3
TP53 Regulates Transcription of Caspase Activators and Caspases	1	237.9×	0.017	ATM
Pexophagy	1	237.9×	0.017	ATM
Defective homologous recombination repair (HRR) due to PALB2 loss of function	1	237.9×	0.017	ATM
FGFR3c ligand binding and activation	1	219.6×	0.017	FGFR3
Phospholipase C-mediated cascade; FGFR3	1	219.6×	0.017	FGFR3
IRF3-mediated induction of type I IFN	1	203.9×	0.017	DDX41
ERBB2 Activates PTK6 Signaling	1	203.9×	0.017	EGFR
Diseases of DNA Double-Strand Break Repair	1	203.9×	0.017	ATM
Defective homologous recombination repair (HRR) due to BRCA2 loss of function	1	203.9×	0.017	ATM
GRB2 events in EGFR signaling	1	190.3×	0.017	EGFR
Regulation of innate immune responses to cytosolic DNA	1	190.3×	0.017	DDX41
Stabilization of p53	1	190.3×	0.017	ATM
TFAP2 (AP-2) family regulates transcription of growth factors and their receptors	1	190.3×	0.017	EGFR
SHC1 events in EGFR signaling	1	178.4×	0.017	EGFR
Constitutive Signaling by EGFRvIII	1	178.4×	0.017	EGFR
ERBB2 Regulates Cell Motility	1	178.4×	0.017	EGFR
p53-Dependent G1 DNA Damage Response	1	178.4×	0.017	ATM
p53-Dependent G1/S DNA damage checkpoint	1	178.4×	0.017	ATM

GO biological processes by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 4 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

GO term	Cohort genes	Fold	FDR	Sample cohort genes
negative regulation of developmental growth	1	4213.0×	0.008	FGFR3
negative regulation of cardiocyte differentiation	1	4213.0×	0.008	EGFR
establishment of RNA localization to telomere	1	2106.5×	0.008	ATM
establishment of protein-containing complex localization to telomere	1	2106.5×	0.008	ATM
fibroblast growth factor receptor apoptotic signaling pathway	1	2106.5×	0.008	FGFR3
positive regulation of telomerase catalytic core complex assembly	1	2106.5×	0.008	ATM
pre-B cell allelic exclusion	1	1404.3×	0.008	ATM
bone maturation	1	1404.3×	0.008	FGFR3
cellular response to nitrosative stress	1	1404.3×	0.008	ATM
positive regulation of protein kinase C signaling	1	1404.3×	0.008	EGFR
morphogenesis of an epithelial fold	1	1053.2×	0.008	EGFR
response to UV-A	1	1053.2×	0.008	EGFR
positive regulation of phospholipase activity	1	842.6×	0.008	FGFR3
peptidyl-serine autophosphorylation	1	842.6×	0.008	ATM
regulation of peptidyl-tyrosine phosphorylation	1	842.6×	0.008	EGFR
cGAS/STING signaling pathway	1	842.6×	0.008	DDX41
negative regulation of telomere capping	1	842.6×	0.008	ATM
positive regulation of ERK1 and ERK2 cascade	2	42.6×	0.008	EGFR, FGFR3
positive regulation of MAPK cascade	2	40.3×	0.008	EGFR, FGFR3
positive regulation of phosphatidylinositol 3-kinase/protein kinase B signal transduction	2	39.2×	0.008	EGFR, FGFR3
positive regulation of transcription by RNA polymerase II	3	11.2×	0.008	DDX41, EGFR, ATM
regulation of telomere maintenance via telomerase	1	702.2×	0.009	ATM
positive regulation of telomere maintenance via telomere lengthening	1	702.2×	0.009	ATM
salivary gland morphogenesis	1	601.9×	0.009	EGFR
lipoprotein catabolic process	1	601.9×	0.009	ATM
positive regulation of cell migration	2	30.9×	0.009	EGFR, ATM
V(D)J recombination	1	526.6×	0.010	ATM
protein insertion into membrane	1	526.6×	0.010	EGFR
meiotic telomere clustering	1	468.1×	0.010	ATM
ubiquitin-dependent endocytosis	1	468.1×	0.010	EGFR

Therapeutics

Drug target analysis

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

Druggability breadth: 4 of 4 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
EGFR	LEVODOPA
FGFR3	PONATINIB
ATM	AMIODARONE HYDROCHLORIDE

Top cohort targets by molecule count

Symbol	Molecules	Max phase
EGFR	175	4
FGFR3	64	4
ATM	35	4
DDX41	1	2

Drugs targeting cohort genes (top 30)

Molecule	Max phase	Targets in cohort
LEVODOPA	4	EGFR
CLOTRIMAZOLE	4	EGFR
ERLOTINIB HYDROCHLORIDE	4	EGFR
CISPLATIN	4	EGFR
PONATINIB	4	EGFR, FGFR3
AFATINIB	4	EGFR
CHROMIC CHLORIDE	4	EGFR
BACITRACIN	4	EGFR
ZINC CHLORIDE	4	EGFR
LAPATINIB DITOSYLATE	4	EGFR
VEMURAFENIB	4	EGFR
FEDRATINIB	4	EGFR, FGFR3
AXITINIB	4	EGFR, FGFR3
SORAFENIB	4	EGFR, FGFR3
DASATINIB ANHYDROUS	4	EGFR
NICLOSAMIDE	4	EGFR
SELUMETINIB	4	EGFR
TERFENADINE	4	EGFR
ALECTINIB	4	EGFR
NERATINIB	4	EGFR
IBRUTINIB	4	EGFR
AFATINIB DIMALEATE	4	EGFR
CABOZANTINIB	4	EGFR
DACOMITINIB	4	EGFR
DACOMITINIB ANHYDROUS	4	EGFR
CERITINIB	4	EGFR, FGFR3
VANDETANIB	4	EGFR, FGFR3
TRIBROMSALAN	4	EGFR
BOSUTINIB	4	EGFR
BITHIONOL	4	EGFR

Bioactivity and enzyme data

Enzyme cohort genes (≥1 EC): 3.

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

Symbol	Assays	Type breakdown
EGFR	6,531	Binding:6211, Functional:173, ADMET:138, Toxicity:9
FGFR3	975	Binding:948, Functional:18, ADMET:9
ATM	240	Binding:233, Functional:5, ADMET:2
DDX41	7	Binding:7

Cohort enzymes (BRENDA EC)

Symbol	EC numbers	Names
EGFR	2.7.10.1	receptor protein-tyrosine kinase
FGFR3	2.7.10.1	receptor protein-tyrosine kinase
ATM	2.7.11.1	non-specific serine/threonine protein kinase

Cohort genes with high screening signal

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

Symbol	ChEMBL assays
EGFR	6,531
FGFR3	975
ATM	240

Pharmacogenomics

Cohort genes with a PharmGKB record: 4; 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)
LEVODOPA	4	EGFR
CLOTRIMAZOLE	4	EGFR
ERLOTINIB HYDROCHLORIDE	4	EGFR
CISPLATIN	4	EGFR
CHROMIC CHLORIDE	4	EGFR
BACITRACIN	4	EGFR
ZINC CHLORIDE	4	EGFR
LAPATINIB DITOSYLATE	4	EGFR
VEMURAFENIB	4	EGFR
AXITINIB	4	EGFR, FGFR3
SORAFENIB	4	EGFR, FGFR3
DASATINIB ANHYDROUS	4	EGFR
NICLOSAMIDE	4	EGFR
SELUMETINIB	4	EGFR
TERFENADINE	4	EGFR
ALECTINIB	4	EGFR
NERATINIB	4	EGFR
IBRUTINIB	4	EGFR
AFATINIB DIMALEATE	4	EGFR
CABOZANTINIB	4	EGFR
DACOMITINIB	4	EGFR
DACOMITINIB ANHYDROUS	4	EGFR
CERITINIB	4	EGFR, FGFR3
VANDETANIB	4	EGFR, FGFR3
TRIBROMSALAN	4	EGFR
BOSUTINIB	4	EGFR
BITHIONOL	4	EGFR

Druggability pyramid

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

Tier	Definition	Genes	Symbols
A	Approved (phase 4 drug)	3	EGFR, FGFR3, ATM
B	Phased (≥1) drug, not yet approved	1	DDX41
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: 786.

Phase distribution (across all retrieved trials)

Phase	Trials
Not specified	360
PHASE2	152
PHASE1	136
PHASE1/PHASE2	60
PHASE3	39
EARLY_PHASE1	20
PHASE4	12
PHASE2/PHASE3	7

Top trials by phase / activity

NCT	Phase	Status	Title
NCT03023631	PHASE4	ACTIVE_NOT_RECRUITING	Gardasil 9 Vaccine in Preventing HPV Infection in Patients With Hematologic Malignancies Undergoing Donor Stem Cell Transplant
NCT03844360	PHASE4	RECRUITING	Dose Individualization of Antineoplastic Drugs and Anti-Infective Drug in Children With Hematoplastic Disease
NCT04296305	PHASE4	ACTIVE_NOT_RECRUITING	Effect of Opioid Infusion Rate on Abuse Liability Potential of Intravenous Hydromorphone for Cancer Pain
NCT06582186	PHASE4	NOT_YET_RECRUITING	Individualized First Maintenance Doses of Voriconazole Through a Multiparametric Algorithm
NCT07590648	PHASE4	NOT_YET_RECRUITING	Early Discontinuation of Antibiotics in Paediatric High-risk Febrile Neutropenia
NCT00067730	PHASE4	COMPLETED	A Safety Evaluation of Drotrecogin Alfa (Activated) in Patients With Blood Cancer, Severe Infection Related to Bone Marrow Transplantation
NCT02895529	PHASE4	TERMINATED	A Study Comparing the Efficacy of Intravenous Followed by Oral Itraconazole With Intravenous Caspofungin For Empiric Antifungal Therapy in Neutropenic Participants With Hematological Malignancy
NCT03908138	PHASE4	UNKNOWN	RDD Versus VDD in Newly Diagnosed Patients With Multiple Myeloma
NCT04673175	PHASE4	TERMINATED	Ceftolozane-Tazobactam for Directed Treatment of Pseudomonas Aeruginosa Bacteremia and Pneumonia in Patients With Hematological Malignancies and Hematopoietic Stem Cell Transplantation
NCT04745910	PHASE4	COMPLETED	Pegloticase for the Reduction of Uric Acid in Patients With Tumor Lysis Syndrome
NCT04952766	PHASE4	COMPLETED	Study Evaluating SARS-CoV-2 (COVID-19) Humoral Response After BNT162b2 Vaccine in Immunocompromised Adults Compared to Healthy Adults
NCT05061654	PHASE4	WITHDRAWN	CEFtolozane-Tazobactam for the Empiric Anti-bacterial Treatment of Neutropenic Fever in Hematology Patients
NCT03486873	PHASE3	RECRUITING	Long-term Safety and Efficacy Extension Study for Participants With Advanced Tumors Who Are Currently on Treatment or in Follow-up in a Pembrolizumab (MK-3475) Study (MK-3475-587/KEYNOTE-587)
NCT04089358	PHASE3	ACTIVE_NOT_RECRUITING	Mobile Health and Social Media Physical Activity Intervention Among Adolescent and Young Adult Childhood Cancer Survivors, the StepByStep Study
NCT04188418	PHASE3	ACTIVE_NOT_RECRUITING	Fentanyl Buccal Tablet or Morphine for Exertional Dyspnea in Cancer Patients
NCT04448184	PHASE3	RECRUITING	Platelet Transfusions in Hematopoietic Stem Cell Transplantation (The PATH III Trial)
NCT04829539	PHASE3	ACTIVE_NOT_RECRUITING	Comparing Brief Behavioral Therapy (BBT-CI) and Healthy Eating Education Learning (HEAL) for Cancer-Related Sleep Problems While Receiving Cancer Treatment
NCT04939090	PHASE3	ACTIVE_NOT_RECRUITING	Olanzapine Versus Megestrol Acetate for the Treatment of Loss of Appetite Among Advanced Cancer Patients
NCT05711667	PHASE3	RECRUITING	A Study of the Drug Letermovir as Prevention of Cytomegalovirus Infection After Stem Cell Transplant in Pediatric Patients
NCT06278896	PHASE3	NOT_YET_RECRUITING	Early Neutropenic Fever De-escalation of Antibiotics Study
NCT06378138	PHASE2/PHASE3	RECRUITING	ICP-248 in Combination With Orelabrutinib in Treatment-naïve Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (APEX-03)
NCT06401356	PHASE3	RECRUITING	An Extension Study for Patients Previously Enrolled in Studies With Pelabresib
NCT06449586	PHASE3	ACTIVE_NOT_RECRUITING	CMV-specific T Cell Immunity Test Indicated Prophylaxis of Letermovir After All-HSCT
NCT07186192	PHASE3	RECRUITING	Digital Health Intervention for Self-Management and Telemonitoring in Chimeric Antigen Receptor (CAR-T) Therapy
NCT07485855	PHASE3	RECRUITING	Influenza Vaccination Strategy for Patients With Hematologic Malignancy
NCT07511127	PHASE3	RECRUITING	Comparing the Efficacy of Different Durations of Maribavir Treatment Regimens in Allo-HSCT
NCT00044759	PHASE3	COMPLETED	Study Comparing the Safety and Efficacy of Piperacillin/Tazobactam to Cefepime in Patients With Hematologic Malignancy or Lymphoma
NCT00075478	PHASE3	COMPLETED	Total-Body Irradiation With or Without Fludarabine Phosphate Followed By Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer
NCT00138112	PHASE3	COMPLETED	Trial of Prophylactic Versus Empirical Vancomycin for the Prevention of Streptococcal Sepsis After Hematopoietic Cell Transplantation
NCT00165282	PHASE2/PHASE3	COMPLETED	Mindfulness Meditation in Bone Marrow Transplantation
NCT00469729	PHASE2/PHASE3	COMPLETED	Efficacy and Safety Study of StemEx®, to Treat Subjects With High Risk Hematologic Malignancies, Following Myeloablative Therapy
NCT00750737	PHASE3	COMPLETED	Oral Posaconazole Three Times Per Day vs Weekly High Dose Amphotericin B Lipid Complex (ABLC)
NCT01217723	PHASE3	UNKNOWN	Thymoglobulin in Unrelated Hematopoietic Progenitor Cell Transplantation
NCT01231412	PHASE3	COMPLETED	Graft-Versus-Host Disease Prophylaxis in Treating Patients With Hematologic Malignancies Undergoing Unrelated Donor Peripheral Blood Stem Cell Transplant
NCT01237639	PHASE3	COMPLETED	Study of Red Blood Cell Transfusion Triggers in Patients Undergoing Hematopoietic Stem Cell Transplantation
NCT01345019	PHASE3	COMPLETED	Denosumab Compared to Zoledronic Acid in the Treatment of Bone Disease in Patients With Multiple Myeloma
NCT01437787	PHASE3	COMPLETED	Phase III Study of SAR302503 in Intermediate-2 and High Risk Patients With Myelofibrosis
NCT01503515	PHASE3	COMPLETED	Caspofungin Acetate, Fluconazole, or Voriconazole in Preventing Fungal Infections in Patients Following Donor Stem Cell Transplant
NCT01602211	PHASE3	COMPLETED	INSPIRE: Internet and Social-media Program With Information and Resources for Long-Term Cancer Survivors Who Underwent Stem Cell Transplant
NCT02623309	PHASE3	COMPLETED	Studyof Allogeneic Hematopoietic Stem Cell Transplantation From One Haplotype Mismatch Related Donor or From an Unrelated Donor in Elderly Patients

Drugs tested across these trials (top 30)

Molecule	Max phase	Trials referencing
TRAMETINIB	4	7
2-MERCAPTOETHANESULFONIC ACID	4	4
AFATINIB	4	4
CRIZOTINIB	4	4
FEDRATINIB	4	4
COPANLISIB	4	3
DABRAFENIB MESYLATE	4	3
LETERMOVIR	4	3
PALIFERMIN	4	3
PENTOSTATIN	4	3
PIPERACILLIN	4	3
PLERIXAFOR	4	3
ROMIDEPSIN	4	3
BINIMETINIB	4	2
CAPIVASERTIB	4	2
CARVEDILOL	4	2
CEFTOLOZANE	4	2
DUVELISIB	4	2
ENASIDENIB	4	2
FLUDARABINE PHOSPHATE	4	2
INOTUZUMAB OZOGAMICIN	4	2
MARAVIROC	4	2
MUROMONAB-CD3	4	2
OMACETAXINE MEPESUCCINATE	4	2
PONATINIB	4	2
POSACONAZOLE	4	2
RELATLIMAB	4	2
ROMIPLOSTIM	4	2
THIOTEPA	4	2
TRASTUZUMAB EMTANSINE	4	2

Precision-medicine subtype map (CIViC)

Drug × molecular subtype: 5 predictive associations from 5 curated evidence items; also 4 predisposing, 3 diagnostic, 1 oncogenic.

Molecular subtype	Therapy	Effect	Level	CIViC
ETV6::NTRK3 Fusion	Larotrectinib	Sensitivity/Response	CIViC C	EID6395
ATM Mutation	Olaparib	Sensitivity/Response	CIViC D	EID455
EGFR D770_N771insNPG	Erlotinib	Sensitivity/Response	CIViC D	EID4491
EGFR D770_N771insNPG	Cetuximab	Sensitivity/Response	CIViC D	EID4492
EGFR E746_A750del	Erlotinib	Sensitivity/Response	CIViC D	EID4208