Childhood leukemia

Summary

Childhood leukemia (MONDO:0004355) is a cancer with 3 cohort genes (3 CIViC-evidence somatic drivers) and 1,723 clinical trials. Molecularly, TP53 Wildtype confers sensitivity to RG7112 in Leukemia (CIViC Level B); 8 further subtype–drug associations are mapped below. Top therapeutic interventions include cyclophosphamide anhydrous, mercaptopurine anhydrous, and cytarabine.

At a glance

• Classification: Cancer
• Cohort genes: 3
• Clinical trials: 1,723
• Precision-medicine evidence (CIViC): 9 subtype–drug associations

Clinical features

No curated clinical features (Orphanet) for this disease.

Identifiers

Disease identifiers

Field	Value
Canonical name	childhood leukemia
Mondo ID	MONDO:0004355
DOID	DOID:7757
NCIT	C4989
UMLS	C1332977
MedGen	234132
GARD	0023954
Is cancer (heuristic)	yes

Also known as: childhood leukaemia (disease) · childhood leukemia · childhood leukemia (disease) · leukaemia · leukaemia (disease) of childhood · leukemia · leukemia (disease) of childhood · paediatric leukaemia (disease) · pediatric leukemia (disease)

Data availability: 27 cell lines.

Disease family

An umbrella term covering 3 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 › leukemia › childhood leukemia

Related subtypes (11): chronic leukemia, chronic erythremia, testicular leukemia, central nervous system leukemia, aleukemic leukemia, splenic manifestation of leukemia, monocytic leukemia, myeloid leukemia, lymphoid leukemia, acute leukemia, mast cell leukemia

Subtypes (3): childhood acute lymphoblastic leukemia, neonatal leukemia, childhood acute myeloid leukemia

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

Somatic driver evidence (intOGen + CIViC, cohort fanout)

Gene	intOGen role	Cancer types	CIViC
SF3B1	Act	AML,BLCA,BRCA,CHOL,CLLSLL,HCC,LUNG,MBL,MEL,PAAD,PCM,PRAD,SKCM,UM	CIViC #44
TP53	LoF	ACC,ALL,AML,ANGS,ANSC,BCC,BL,BLADDER,BLCA,BRCA,CCRCC,CEAD,CESC,CHOL,CHRCC,CLLSLL,COAD,COADREAD,CSCC,DLBCLNOS,EGC,ES,ESCA,ESCC,GB,GBC,GBM,GIST,HCC,HGGNOS,HNSC,LGGNOS,LIPO,LMS,LNM,LUAD,LUSC,MBL,MEL,MLYM,MT,NBL,NETNOS,NHL,NPC,NSCLC,OS,OVT,PAAD,PANCREAS,PAST,PCM,PLMESO,PRAD,PRCC,PROSTATE,RCC,READ,SACA,SARCNOS,SCLC,SIC,SKCM,SKIN,SOFT_TISSUE,STAD,STOMACH,THYM,UCEC,UCS,UTUC,VULVA,WDTC,WT	CIViC #45
FLT3	Act	ALL,AML	CIViC #24

Orphanet rare-disease linkage (cohort genes)

Gene	Orphanet ID	Rare disease
SF3B1	Orphanet:39044	Uveal melanoma
SF3B1	Orphanet:75564	Acquired idiopathic sideroblastic anemia
TP53	Orphanet:1333	Familial pancreatic carcinoma
TP53	Orphanet:145	Hereditary breast and/or ovarian cancer syndrome
TP53	Orphanet:1501	Adrenocortical carcinoma
TP53	Orphanet:210159	Adult hepatocellular carcinoma
TP53	Orphanet:251576	Gliosarcoma
TP53	Orphanet:251579	Giant cell glioblastoma
TP53	Orphanet:251899	Choroid plexus carcinoma
TP53	Orphanet:2807	Papilloma of choroid plexus
TP53	Orphanet:293199	Pleomorphic rhabdomyosarcoma
TP53	Orphanet:3318	Essential thrombocythemia
TP53	Orphanet:524	Li-Fraumeni syndrome
TP53	Orphanet:52688	Myelodysplastic syndrome
TP53	Orphanet:585909	B-lymphoblastic leukemia/lymphoma with t(9;22)(q34.1;q11.2)
TP53	Orphanet:667662	Breast implant-associated anaplastic large cell lymphoma
TP53	Orphanet:668	Osteosarcoma
TP53	Orphanet:67038	B-cell chronic lymphocytic leukemia
TP53	Orphanet:70573	Small cell lung cancer
TP53	Orphanet:96253	Cushing disease
TP53	Orphanet:99756	Alveolar rhabdomyosarcoma
TP53	Orphanet:99757	Embryonal rhabdomyosarcoma
FLT3	Orphanet:102724	Acute myeloid leukemia with t(8;21)(q22;q22) translocation
FLT3	Orphanet:585909	B-lymphoblastic leukemia/lymphoma with t(9;22)(q34.1;q11.2)
FLT3	Orphanet:589534	Mixed phenotype acute leukemia with t(9;22)(q34.1;q11.2)
FLT3	Orphanet:589595	Mixed phenotype acute leukemia with t(v;11q23.3)
FLT3	Orphanet:98829	Acute myeloid leukemia with abnormal bone marrow eosinophils inv(16)(p13q22) or t(16;16)(p13;q22)
FLT3	Orphanet:98832	Acute myeloid leukemia with minimal differentiation
FLT3	Orphanet:98833	Acute myeloblastic leukemia without maturation
FLT3	Orphanet:98834	Acute myeloblastic leukemia with maturation
FLT3	Orphanet:99861	Precursor T-cell acute lymphoblastic leukemia

Cohort genes → proteins

3 cohort genes, 3 distinct canonical proteins.

Evidence partition

Subset	Genes
civic_only	3

Cohort genes (full)

Symbol	HGNC	Ensembl	UniProt	Name	Evidence
SF3B1	HGNC:10768	ENSG00000115524	O75533	Splicing factor 3B subunit 1	civic_evidence
TP53	HGNC:11998	ENSG00000141510	P04637	Cellular tumor antigen p53	civic_evidence
FLT3	HGNC:3765	ENSG00000122025	P36888	Receptor-type tyrosine-protein kinase FLT3	civic_evidence

Cohort function summary

Lead sentence per gene, UniProt-curated.

Symbol	Protein name	Function (lead sentence)
SF3B1	Splicing factor 3B subunit 1	Component of the 17S U2 SnRNP complex of the spliceosome, a large ribonucleoprotein complex that removes introns from transcribed pre-mRNAs.
TP53	Cellular tumor antigen p53	Multifunctional transcription factor that induces cell cycle arrest, DNA repair or apoptosis upon binding to its target DNA sequence.
FLT3	Receptor-type tyrosine-protein kinase FLT3	Tyrosine-protein kinase that acts as a cell-surface receptor for the cytokine FLT3LG and regulates differentiation, proliferation and survival of hematopoietic progenitor cells and of dendritic cells.

Protein-family classification

Druggable: 1 · Difficult: 1 · Unknown: 1 · Druggable fraction: 0.33

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	9.2×	0.313
Transcription factor	1	2.8×	0.482
Other/Unknown	1	0.6×	0.914

Per-gene assignment

Symbol	Family	Druggable?	EC	InterPro (top 3)
SF3B1	Other/Unknown	no		ARM-like, SF3b_su1, ARM-type_fold
TP53	Transcription factor	no		p53_tumour_suppressor, p53-like_TF_DNA-bd_sf, p53_tetrameristn
FLT3	Kinase	yes	2.7.10.1	Prot_kinase_dom, Ser-Thr/Tyr_kinase_cat_dom, Tyr_kinase_rcpt_3_CS

Expression context

Cohort genes with no expression data: 0.

3 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)	3
unknown	0

Top tissues across cohort

Tissue	Cohort genes
ventricular zone	2
epithelium of nasopharynx	1
tibia	1
ganglionic eminence	1
tendon of biceps brachii	1
cerebellar cortex	1
cerebellar hemisphere	1
male germ line stem cell (sensu Vertebrata) in testis	1

Per-gene tissue summary (top 30)

Symbol	Bgee breadth	FANTOM5 breadth	SCXA	Top tissues
SF3B1	295	ubiquitous	marker	tibia, ventricular zone, epithelium of nasopharynx
TP53	223	ubiquitous	marker	ventricular zone, ganglionic eminence, tendon of biceps brachii
FLT3	166	broad	marker	male germ line stem cell (sensu Vertebrata) in testis, cerebellar hemisphere, cerebellar cortex

Protein interactions among cohort

Intra-cohort edges: 0.

Hub genes (top 10 by interactor count)

Symbol	Interactor count
TP53	22,736
SF3B1	4,582
FLT3	3,570

Structural data

PDB: 3 · AlphaFold-only: 0 · No structure: 0

Cohort genes with PDB structures (top 30)

Symbol	UniProt	PDB entries
TP53	P04637	313
SF3B1	O75533	74
FLT3	P36888	11

Function

Pathway analysis

Distinct Reactome pathways touched by cohort: 85. Enrichment computed across 3 evidence-associated genes (3 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 3 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

Pathway	Cohort genes	Fold	FDR	Sample cohort genes
FLT3 mutants bind TKIs	1	3806.7×	0.001	FLT3
KW2449-resistant FLT3 mutants	1	3806.7×	0.001	FLT3
semaxanib-resistant FLT3 mutants	1	3806.7×	0.001	FLT3
crenolanib-resistant FLT3 mutants	1	3806.7×	0.001	FLT3
gilteritinib-resistant FLT3 mutants	1	3806.7×	0.001	FLT3
lestaurtinib-resistant FLT3 mutants	1	3806.7×	0.001	FLT3
midostaurin-resistant FLT3 mutants	1	3806.7×	0.001	FLT3
pexidartinib-resistant FLT3 mutants	1	3806.7×	0.001	FLT3
ponatinib-resistant FLT3 mutants	1	3806.7×	0.001	FLT3
quizartinib-resistant FLT3 mutants	1	3806.7×	0.001	FLT3
sorafenib-resistant FLT3 mutants	1	3806.7×	0.001	FLT3
sunitinib-resistant FLT3 mutants	1	3806.7×	0.001	FLT3
tandutinib-resistant FLT3 mutants	1	3806.7×	0.001	FLT3
linifanib-resistant FLT3 mutants	1	3806.7×	0.001	FLT3
tamatinib-resistant FLT3 mutants	1	3806.7×	0.001	FLT3
Loss of function of TP53 in cancer due to loss of tetramerization ability	1	3806.7×	0.001	TP53
Regulation of TP53 Expression	1	1903.3×	0.003	TP53
Transcriptional activation of cell cycle inhibitor p21	1	951.7×	0.005	TP53
Activation of NOXA and translocation to mitochondria	1	634.4×	0.007	TP53
RUNX3 regulates CDKN1A transcription	1	543.8×	0.007	TP53
STAT5 Activation	1	543.8×	0.007	FLT3
FLT3 signaling through SRC family kinases	1	543.8×	0.007	FLT3
FLT3 signaling by CBL mutants	1	543.8×	0.007	FLT3
PI5P Regulates TP53 Acetylation	1	423.0×	0.008	TP53
Activation of PUMA and translocation to mitochondria	1	380.7×	0.009	TP53
STAT5 activation downstream of FLT3 ITD mutants	1	380.7×	0.009	FLT3
TP53 Regulates Transcription of Caspase Activators and Caspases	1	317.2×	0.010	TP53
TP53 Regulates Transcription of Death Receptors and Ligands	1	317.2×	0.010	TP53
Urea cycle	1	292.8×	0.010	TP53
Regulation of TP53 Activity through Association with Co-factors	1	271.9×	0.010	TP53

GO biological processes by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 3 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 helicase activity	1	5617.3×	0.005	TP53
cellular response to actinomycin D	1	5617.3×	0.005	TP53
regulation of intrinsic apoptotic signaling pathway by p53 class mediator	1	5617.3×	0.005	TP53
negative regulation of G1 to G0 transition	1	5617.3×	0.005	TP53
positive regulation of mitochondrial membrane permeability	1	2808.7×	0.005	TP53
oligodendrocyte apoptotic process	1	2808.7×	0.005	TP53
negative regulation of glucose catabolic process to lactate via pyruvate	1	2808.7×	0.005	TP53
negative regulation of pentose-phosphate shunt	1	2808.7×	0.005	TP53
leukocyte homeostasis	1	1872.4×	0.005	FLT3
obsolete homolactic fermentation	1	1872.4×	0.005	TP53
signal transduction by p53 class mediator	1	1872.4×	0.005	TP53
negative regulation of miRNA processing	1	1872.4×	0.005	TP53
intrinsic apoptotic signaling pathway in response to hypoxia	1	1872.4×	0.005	TP53
regulation of fibroblast apoptotic process	1	1872.4×	0.005	TP53
T cell proliferation involved in immune response	1	1404.3×	0.005	TP53
pro-B cell differentiation	1	1404.3×	0.005	FLT3
positive regulation of programmed necrotic cell death	1	1404.3×	0.005	TP53
oxidative stress-induced premature senescence	1	1404.3×	0.005	TP53
B cell lineage commitment	1	1123.5×	0.005	TP53
T cell lineage commitment	1	1123.5×	0.005	TP53
mRNA transcription	1	1123.5×	0.005	TP53
positive regulation of RNA polymerase II transcription preinitiation complex assembly	1	1123.5×	0.005	TP53
positive regulation of thymocyte apoptotic process	1	1123.5×	0.005	TP53
cellular response to UV-C	1	1123.5×	0.005	TP53
regulation of apoptotic process	2	55.6×	0.005	TP53, FLT3
regulation of mitochondrial membrane permeability involved in apoptotic process	1	936.2×	0.006	TP53
myeloid progenitor cell differentiation	1	802.5×	0.006	FLT3
viral process	1	802.5×	0.006	TP53
mitochondrial DNA repair	1	802.5×	0.006	TP53
lymphocyte proliferation	1	802.5×	0.006	FLT3

Therapeutics

Drug target analysis

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

Druggability breadth: 3 of 3 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
TP53	NITROFURANTOIN
FLT3	PONATINIB

Top cohort targets by molecule count

Symbol	Molecules	Max phase
TP53	196	4
FLT3	143	4
SF3B1	1	2

Drugs targeting cohort genes (top 30)

Molecule	Max phase	Targets in cohort
NITROFURANTOIN	4	TP53
DIOSMIN	4	TP53
VERTEPORFIN	4	TP53
CANDESARTAN CILEXETIL	4	TP53
DIENESTROL	4	TP53
CLOTRIMAZOLE	4	TP53
COLCHICINE	4	TP53
NABUMETONE	4	TP53
SALMETEROL XINAFOATE	4	TP53
AMIODARONE HYDROCHLORIDE	4	TP53
FURAZOLIDONE	4	TP53
AMOXAPINE	4	TP53
RALOXIFENE HYDROCHLORIDE	4	TP53
NICARDIPINE HYDROCHLORIDE	4	TP53
SULCONAZOLE NITRATE	4	TP53
PYRITHIONE ZINC	4	TP53
LACTIC ACID	4	TP53
OXYMETHOLONE	4	TP53
CHLOROXINE	4	TP53
PROPIOLACTONE	4	TP53
CLOMIPRAMINE HYDROCHLORIDE	4	TP53
PHENYL AMINOSALICYLATE	4	TP53
THIORIDAZINE HYDROCHLORIDE	4	TP53
AMITRIPTYLINE HYDROCHLORIDE	4	TP53
ETHOPROPAZINE HYDROCHLORIDE	4	TP53
MECHLORETHAMINE HYDROCHLORIDE	4	TP53
ECONAZOLE NITRATE	4	TP53
TRIFLUPROMAZINE HYDROCHLORIDE	4	TP53
PROCHLORPERAZINE EDISYLATE	4	TP53
DEQUALINIUM CHLORIDE	4	TP53

Bioactivity and enzyme data

Enzyme cohort genes (≥1 EC): 1.

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

Symbol	Assays	Type breakdown
FLT3	3,132	Binding:3096, Functional:24, ADMET:8, Toxicity:4
TP53	869	Binding:775, ADMET:83, Functional:10, Toxicity:1
SF3B1	22	Binding:22

Cohort enzymes (BRENDA EC)

Symbol	EC numbers	Names
FLT3	2.7.10.1	receptor protein-tyrosine kinase

Cohort genes with high screening signal

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

Symbol	ChEMBL assays
TP53	869
FLT3	3,132

Pharmacogenomics

Cohort genes with a PharmGKB record: 3; with CPIC/DPWG dosing guidelines: 0.

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

Drug repurposing candidates

30 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)
NITROFURANTOIN	4	TP53
DIOSMIN	4	TP53
VERTEPORFIN	4	TP53
CANDESARTAN CILEXETIL	4	TP53
DIENESTROL	4	TP53
CLOTRIMAZOLE	4	TP53
COLCHICINE	4	TP53
NABUMETONE	4	TP53
SALMETEROL XINAFOATE	4	TP53
AMIODARONE HYDROCHLORIDE	4	TP53
FURAZOLIDONE	4	TP53
AMOXAPINE	4	TP53
RALOXIFENE HYDROCHLORIDE	4	TP53
NICARDIPINE HYDROCHLORIDE	4	TP53
SULCONAZOLE NITRATE	4	TP53
PYRITHIONE ZINC	4	TP53
LACTIC ACID	4	TP53
OXYMETHOLONE	4	TP53
CHLOROXINE	4	TP53
PROPIOLACTONE	4	TP53
CLOMIPRAMINE HYDROCHLORIDE	4	TP53
PHENYL AMINOSALICYLATE	4	TP53
THIORIDAZINE HYDROCHLORIDE	4	TP53
AMITRIPTYLINE HYDROCHLORIDE	4	TP53
ETHOPROPAZINE HYDROCHLORIDE	4	TP53
MECHLORETHAMINE HYDROCHLORIDE	4	TP53
ECONAZOLE NITRATE	4	TP53
TRIFLUPROMAZINE HYDROCHLORIDE	4	TP53
PROCHLORPERAZINE EDISYLATE	4	TP53
DEQUALINIUM CHLORIDE	4	TP53

Druggability pyramid

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

Tier	Definition	Genes	Symbols
A	Approved (phase 4 drug)	2	TP53, FLT3
B	Phased (≥1) drug, not yet approved	1	SF3B1
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: 1,723.

Phase distribution (across all retrieved trials)

Phase	Trials
PHASE2	553
PHASE1/PHASE2	185
PHASE1	179
PHASE3	144
PHASE2/PHASE3	20
PHASE4	15
EARLY_PHASE1	4

Top trials by phase / activity

NCT	Phase	Status	Title
NCT06918054	PHASE4	RECRUITING	Hepatoprotective for Children and Adolescent With Acute Lymphoblastic Leukemia
NCT00003398	PHASE4	COMPLETED	Bone Marrow Transplantation in Treating Patients With Hematologic Cancer
NCT00362544	PHASE4	COMPLETED	PROPHYSOME: Pilot Study on Safety of a Weekly Administration of 10mg/kg of AmBisome® in Antifungal Prophylaxis Treatment of Allogeneic Stem-cell Transplantation and Acute Leukaemia
NCT00365508	PHASE4	COMPLETED	Counseling and Nicotine Replacement Therapy in Helping Adult Smokers Quit Smoking
NCT00415103	PHASE4	COMPLETED	AMENO-2: Aprepitant Plus Palonosetron Versus Granisetron in the Prevention of Nausea and the Emesis Induced by Chemotherapy in Patients Treated With Haematopoietic Progenitors
NCT00666978	PHASE4	COMPLETED	Health Education Counseling With or Without Bupropion in Helping African Americans Stop Smoking
NCT00808171	PHASE4	COMPLETED	Evaluation of the Analgesy With Emla and/or Nitrous Oxide in Pediatric Patients for Lumbar Puncture
NCT01191541	PHASE4	COMPLETED	Cytarabine (Ara-C) in Children With Acute Promyelocytic Leukemia (APL)
NCT01222819	PHASE4	TERMINATED	SubCutaneous (SC) Versus Intravenous (IV) Granulocyte Colony Stimulating Factors (G-CSF) for the Treatment of Neutropenia in Hospitalized Haemato-oncological Patients
NCT02095951	PHASE4	COMPLETED	Preemptive Ethanol Lock Therapy in Pediatric Bloodstream Infection
NCT02347878	PHASE4	UNKNOWN	Self-control Trial to Evaluate the Role of Aprepitant in the Prophylaxis of Post-lumbar-punture-headache (PLPH)
NCT02784561	PHASE4	UNKNOWN	Study of Busulfan and FLAG Conditioning Regimen for Allogeneic Peripheral Blood Stem Cell Transplantation
NCT03677596	PHASE4	COMPLETED	A Study Of Two Inotuzumab Ozogamicin Doses in Relapsed/ Refractory Acute Lymphoblastic Leukemia Transplant Eligible Patients
NCT04187755	PHASE4	COMPLETED	Cefepime vs Ceftazidime as Empirical Therapy for Neutropenic Fever
NCT04636918	PHASE4	UNKNOWN	Ikervis for DED Due to GVHD Post Allo-HSCT
NCT03057054	PHASE3	ACTIVE_NOT_RECRUITING	Lactobacillus Plantarum in Preventing Acute Graft Versus Host Disease in Children Undergoing Donor Stem Cell Transplant
NCT04547049	PHASE3	ACTIVE_NOT_RECRUITING	A Study Comparing Haploidentical Hematopoietic Stem Cell Transplantations (HSCTs) From Young Non-first-degree and Older First-degree Donors in Hematological Malignancies
NCT06578247	PHASE3	RECRUITING	Quizartinib or Placebo Plus Chemotherapy in Newly Diagnosed Patients With FLT3-ITD Negative AML
NCT07082452	PHASE3	NOT_YET_RECRUITING	A Multicenter Trial Evaluating Efficacy and Safety of A Reduced Venetoclax Exposure To Seven Days Versus Standard Continuous Venetoclax Exposure Combined With Azacitidine in Treatment Naïve Subjects With Acute Myeloid Leukemia Who Are Ineligible for Intensive Induction
NCT07202052	PHASE2/PHASE3	RECRUITING	Role of Antibiotic Therapy or Immunoglobulin On iNfections in hAematoLogy Platform Trial (RATIONAL-PT)
NCT07202065	PHASE2/PHASE3	RECRUITING	Role of Antibiotic Therapy or Immunoglobulin On iNfections in hAematoLogy Dosing Immunoglobulin (Dose Ig)
NCT07202078	PHASE2/PHASE3	RECRUITING	Role of Antibiotic Therapy or Immunoglobulin On iNfections in hAematoLogy: Starting Immunoglobulin (Start Ig)
NCT07202091	PHASE2/PHASE3	RECRUITING	Role of Antibiotic Therapy or Immunoglobulin On iNfections in hAematoLogy Immunoglobulin Stopping or Extension (Stop Ig)
NCT07296445	PHASE3	NOT_YET_RECRUITING	A Trial to Investigate Whether Oral Arsenic Trioxide Is Similar to Intravenous Arsenic Trioxide in Pharmacokinetics, Safety, and Efficacy (LATITUDE/SDKARS-301)
NCT00000591	PHASE3	COMPLETED	T-Cell Depletion in Unrelated Donor Marrow Transplantation
NCT00002456	PHASE3	COMPLETED	Graft-Versus-Host Disease Prevention in Treating Patients Who Are Undergoing Bone Marrow Transplantation
NCT00002499	PHASE2/PHASE3	UNKNOWN	Combination Chemotherapy in Treating Children With Relapsed Acute Lymphocytic Leukemia
NCT00002514	PHASE3	COMPLETED	Stem Cell Transplantation Compared With Standard Chemotherapy in Treating Patients With Acute Lymphoblastic Leukemia in First Remission
NCT00002517	PHASE3	COMPLETED	Combination Chemotherapy in Treating Children With Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome
NCT00002534	PHASE3	COMPLETED	Bone Marrow Transplantation in Treating Patients With Acute Leukemia in First or Second Remission
NCT00002549	PHASE3	UNKNOWN	Combination Chemotherapy Followed by Bone Marrow or Peripheral Stem Cell Transplantation in Treating Patients With Acute Myelogenous Leukemia
NCT00002658	PHASE3	UNKNOWN	Combination Chemotherapy, Biological Therapy, and Bone Marrow Transplantation in Treating Patients With Acute Myeloid Leukemia
NCT00002700	PHASE3	COMPLETED	Chemotherapy With or Without Bone Marrow Transplantation in Treating Patients With Acute Lymphoblastic Leukemia
NCT00002701	PHASE3	UNKNOWN	Combination Chemotherapy With or Without Bone Marrow Transplantation in Treating Patients With Acute Promyelocytic Leukemia
NCT00002719	PHASE3	COMPLETED	Combination Chemotherapy With or Without G-CSF in Treating Older Patients With Acute Myeloid Leukemia
NCT00002742	PHASE3	COMPLETED	Antifungal Therapy for Fever and Neutropenia in Patients Receiving Treatment for Hematologic Cancer
NCT00002744	PHASE3	COMPLETED	Combination Chemotherapy in Treating Children With Newly Diagnosed Acute Lymphoblastic Leukemia
NCT00002757	PHASE3	COMPLETED	TITLE:Less Intensive Therapy for Children With Non-Hodgkin’s Lymphoma
NCT00002766	PHASE3	COMPLETED	Comparison of Two Combination Chemotherapy Regimens in Treating Adults With Previously Untreated Leukemia or Lymphoma
NCT00002771	PHASE3	UNKNOWN	Chemotherapy, Interferon, and Bone Marrow Transplantation in Treating Patients With Chronic Myelogenous Leukemia

Drugs tested across these trials (top 30)

Molecule	Max phase	Trials referencing
CYCLOPHOSPHAMIDE ANHYDROUS	4	269
MERCAPTOPURINE ANHYDROUS	4	110
CYTARABINE	4	44
IDARUBICIN	4	40
MITOXANTRONE HYDROCHLORIDE	4	38
IMATINIB	4	37
ASPARAGINASE	4	35
BUSULFAN	4	35
DAUNORUBICIN HYDROCHLORIDE	4	33
2-MERCAPTOETHANESULFONIC ACID	4	30
THIOGUANINE	4	29
ETOPOSIDE	4	23
LEUCOVORIN	4	23
SARGRAMOSTIM	4	20
THIOTEPA	4	19
VINCRISTINE SULFATE	4	18
FILGRASTIM	4	16
FLUDARABINE PHOSPHATE	4	16
TRETINOIN	4	11
AMSACRINE	4	6
AMPHOTERICIN B	4	2
APREPITANT	4	2
BUPROPION HYDROCHLORIDE	4	1
CEFEPIME	4	1
CEFTAZIDIME	4	1
CYCLOSPORINE	4	1
GRANISETRON	4	1
LIDOCAINE HYDROCHLORIDE	4	1
NICOTINE	4	1
PALONOSETRON	4	1

Precision-medicine subtype map (CIViC)

Drug × molecular subtype: 9 predictive associations from 9 curated evidence items; also 1 oncogenic.

Molecular subtype	Therapy	Effect	Level	CIViC
TP53 Wildtype	RG7112	Sensitivity/Response	CIViC B	EID2965
TP53 Deleterious Mutation	RG7112	Resistance	CIViC B	EID2966
FLT3 D835Y	Lestaurtinib	Sensitivity/Response	CIViC D	EID11095
FLT3 D835Y	Sorafenib	Sensitivity/Response	CIViC D	EID11096
FLT3 ITD	Sunitinib	Sensitivity/Response	CIViC D	EID7374
FLT3 ITD	Sunitinib + Sorafenib	Sensitivity/Response	CIViC D	EID7382
SF3B1 K700E	Etoposide + Olaparib	Sensitivity/Response	CIViC D	EID10139
TOP2A TOP2A/90	Etoposide	Resistance	CIViC D	EID9634
TOP2A TOP2A/90	Etoposide + Pixantrone + DVP Regimen + 4’-(9-acridinylamino)methanesulfon-m-anisidide + MVT Regimen	Resistance	CIViC D	EID9635