Acute biphenotypic leukemia

Summary

Acute biphenotypic leukemia (MONDO:0020322) is a cancer and 37 clinical trials. Molecularly, KMT2A::v Fusion confers sensitivity to EPZ004777 in Acute Biphenotypic Leukemia (CIViC Level D). Top therapeutic interventions include mycophenolate mofetil, cladribine, and clofarabine. A subtype of acute lymphoblastic leukemia — broader associated-gene and molecular evidence is on the parent page (see Disease family below).

At a glance

• Classification: Cancer
• Clinical trials: 37
• Precision-medicine evidence (CIViC): 1 subtype–drug association

Clinical features

No curated clinical features (Orphanet) for this disease.

Identifiers

Disease identifiers

Field	Value
Canonical name	acute biphenotypic leukemia
Mondo ID	MONDO:0020322
EFO	EFO:1000828
MeSH	D015456
Orphanet	98837
DOID	DOID:9953
NCIT	C4673
SNOMED CT	278453007
UMLS	C0023464
MedGen	7318
GARD	0025156
MedDRA	10067399
Is cancer (heuristic)	yes

Also known as: B- and T-cell mixed leukaemia · B- and T-cell mixed leukemia

Data availability: 5 cell lines.

Disease family

This is a subtype of acute lymphoblastic leukemia. 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: 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 › lymphoid neoplasm › precursor lymphoblastic lymphoma/leukemia › acute lymphoblastic leukemia › acute biphenotypic leukemia

Related subtypes (11): childhood acute lymphoblastic leukemia, prolymphocytic leukemia, adult acute lymphoblastic leukemia, null-cell leukemia, B-cell acute lymphoblastic leukemia, B-cell chronic lymphocytic leukemia, T-cell acute lymphoblastic leukemia, lymphoblastic leukemia, acute, with lymphomatous features, plasma cell leukemia, precursor B-cell acute lymphoblastic leukemia, precursor T-cell acute lymphoblastic leukemia

Subtypes (4): mixed phenotype acute leukemia with BCR-ABL1, mixed phenotype acute leukemia with MLL rearranged, mixed phenotype acute leukemia, B/myeloid, mixed phenotype acute leukemia,T/myeloid

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

0 approved, 7 in late-stage (phase 3) trials. Disease-direct ChEMBL indications, not inferred from the associated-gene cohort below.

Drug	Development status
Cytarabine	Phase 3 (in late-stage trials)
Dexamethasone	Phase 3 (in late-stage trials)
Doxorubicin	Phase 3 (in late-stage trials)
Filgrastim	Phase 3 (in late-stage trials)
Hydrocortisone	Phase 3 (in late-stage trials)
Methotrexate	Phase 3 (in late-stage trials)
Pegaspargase	Phase 3 (in late-stage trials)

Earlier-phase candidates (phase 2, investigational — efficacy not yet established): Asparaginase Erwinia Chrysanthemi, Bortezomib, Cladribine, Clofarabine, Idarubicin, Mitoxantrone, Rituximab, Sorafenib, Venetoclax, Vincristine, Vorinostat.

Clinical trials & evidence

Clinical trials

Clinical trials: 37.

Phase distribution (across all retrieved trials)

Phase	Trials
PHASE2	22
PHASE1	7
PHASE1/PHASE2	6
PHASE3	1
Not specified	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT01951885	PHASE3	COMPLETED	Tac, Mini-MTX, MMF Versus Tac, MTX for GVHD Prevention
NCT02115295	PHASE2	RECRUITING	Cladribine, Idarubicin, Cytarabine, and Venetoclax in Treating Patients With Acute Myeloid Leukemia, High-Risk Myelodysplastic Syndrome, or Blastic Phase Chronic Myeloid Leukemia
NCT02727803	PHASE2	RECRUITING	Personalized NK Cell Therapy in CBT
NCT03399773	PHASE2	ACTIVE_NOT_RECRUITING	Infusion of Expanded Cord Blood Cells in Addition to Single Cord Blood Transplant in Treating Patients With Acute Leukemia, Chronic Myeloid Leukemia, or Myelodysplastic Syndromes
NCT03683433	PHASE2	RECRUITING	Enasidenib and Azacitidine in Treating Patients With Recurrent or Refractory Acute Myeloid Leukemia and IDH2 Gene Mutation
NCT03779854	PHASE2	RECRUITING	Naive T Cell Depletion for Preventing Chronic Graft-versus-Host Disease in Children and Young Adults With Blood Cancers Undergoing Donor Stem Cell Transplant
NCT04047641	PHASE1/PHASE2	RECRUITING	Cladribine, Idarubicin, Cytarabine, and Quizartinib in Treating Patients With Newly Diagnosed, Relapsed, or Refractory Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome
NCT04128501	PHASE2	ACTIVE_NOT_RECRUITING	Venetoclax and Azacitidine for the Treatment of Acute Myeloid Leukemia in the Post-Transplant Setting
NCT04797767	PHASE1/PHASE2	RECRUITING	Venetoclax and CLAG-M for the Treatment of Acute Myeloid Leukemia and High-Grade Myeloid Neoplasms
NCT04975919	PHASE2	ACTIVE_NOT_RECRUITING	Venetoclax in Combination With Decitabine and Cedazuridine for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia
NCT05589896	PHASE1/PHASE2	RECRUITING	A First-in-Human Study of HLA-Partially to Fully Matched Allogenic Cryopreserved Deceased Donor Bone Marrow Transplantation for Patients With Hematologic Malignancies
NCT00719888	PHASE2	COMPLETED	Umbilical Cord Blood Transplant, Cyclophosphamide, Fludarabine, and Total-Body Irradiation in Treating Patients With Hematologic Disease
NCT00796068	PHASE2	COMPLETED	Treosulfan, Fludarabine Phosphate, and Total-Body Irradiation in Treating Patients With Hematological Cancer Who Are Undergoing Umbilical Cord Blood Transplant
NCT01028716	PHASE2	TERMINATED	Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies
NCT01690520	PHASE2	COMPLETED	Donor Umbilical Cord Blood Transplant With or Without Ex-vivo Expanded Cord Blood Progenitor Cells in Treating Patients With Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Chronic Myelogenous Leukemia, or Myelodysplastic Syndromes
NCT01858740	PHASE2	COMPLETED	Selective Depletion of CD45RA+ T Cells From Allogeneic Peripheral Blood Stem Cell Grafts in Preventing GVHD in Children
NCT02044796	PHASE1/PHASE2	COMPLETED	Filgrastim, Cladribine, Cytarabine, and Mitoxantrone Hydrochloride in Treating Patients With Newly Diagnosed or Relapsed/Refractory Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndromes
NCT02135874	PHASE2	COMPLETED	Clofarabine, Idarubicin, Cytarabine, Vincristine Sulfate, and Dexamethasone in Treating Patients With Newly Diagnosed or Relapsed Mixed Phenotype Acute Leukemia
NCT02220985	PHASE2	COMPLETED	Selective Depletion of CD45RA+ T Cells From Allogeneic Peripheral Blood Stem Cell Grafts From HLA-Matched Related and Unrelated Donors in Preventing GVHD
NCT02397720	PHASE2	COMPLETED	Nivolumab and Azacitidine With or Without Ipilimumab in Treating Patients With Refractory/Relapsed or Newly Diagnosed Acute Myeloid Leukemia
NCT02399917	PHASE2	TERMINATED	Lirilumab and Azacitidine in Treating Patients With Refractory or Relapsed Acute Myeloid Leukemia
NCT02728050	PHASE1/PHASE2	COMPLETED	Filgrastim, Cladribine, Cytarabine, and Mitoxantrone With Sorafenib in Treating Patients With Newly-Diagnosed, Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome
NCT02793544	PHASE2	COMPLETED	HLA-Mismatched Unrelated Donor Bone Marrow Transplantation With Post-Transplantation Cyclophosphamide
NCT03096782	PHASE2	COMPLETED	Umbilical Cord Blood Transplant With Added Sugar and Chemotherapy and Radiation Therapy in Treating Patients With Leukemia or Lymphoma
NCT03195010	PHASE2	TERMINATED	Management of Platelet Transfusion Therapy in Patients With Blood Cancer or Treatment-Induced Thrombocytopenia
NCT03267186	PHASE2	COMPLETED	Ibrutinib in Preventing Acute Leukemia in Patients After Reduced-Intensity Conditioning and Stem Cell Transplant
NCT03404193	PHASE2	TERMINATED	Venetoclax and Decitabine in Treating Participants With Relapsed/Refractory Acute Myeloid Leukemia or Relapsed High-Risk Myelodysplastic Syndrome
NCT03739606	PHASE2	WITHDRAWN	Flotetuzumab in Treating Patients With Recurrent or Refractory CD123 Positive Blood Cancer
NCT04874194	PHASE1/PHASE2	TERMINATED	Omacetaxine and Venetoclax for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome Harboring Mutant RUNX1
NCT02159495	PHASE1	ACTIVE_NOT_RECRUITING	Genetically Modified T-cell Immunotherapy in Treating Patients With Relapsed/Refractory Acute Myeloid Leukemia and Persistent/Recurrent Blastic Plasmacytoid Dendritic Cell Neoplasm
NCT03326921	PHASE1	RECRUITING	HA-1 T TCR T Cell Immunotherapy for the Treatment of Patients With Relapsed or Refractory Acute Leukemia After Donor Stem Cell Transplant
NCT01619761	PHASE1	UNKNOWN	NK Cells in Cord Blood Transplantation
NCT02529813	PHASE1	COMPLETED	CD19-Specific T-cells in Treating Patients With Advanced Lymphoid Malignancies
NCT03807063	PHASE1	WITHDRAWN	Rivogenlecleucel Donor Lymphocyte Immunotherapy in Treating Patients With Recurrent Blood Cancers After Stem Cell Transplant
NCT04526795	PHASE1	TERMINATED	Fludarabine, Cytarabine, and Pegcrisantaspase for the Treament of Relapsed or Refractory Leukemia
NCT05170828	PHASE1	WITHDRAWN	Cryopreserved MMUD BM With PTCy for Hematologic Malignancies
NCT01804101	Not specified	COMPLETED	Liposomal Cytarabine-Daunorubicin CPX-351 in Treating Patients With Untreated Myelodysplastic Syndrome or Acute Myeloid Leukemia

Drugs tested across these trials (top 30)

Molecule	Max phase	Trials referencing
MYCOPHENOLATE MOFETIL	4	10
CLADRIBINE	4	5
CLOFARABINE	4	3
MITOXANTRONE	4	3
2-MERCAPTOETHANESULFONIC ACID	4	2
DECITABINE	4	2
ENASIDENIB	4	2
VENETOCLAX	4	2
CEDAZURIDINE	4	1
GILTERITINIB	4	1
MIDOSTAURIN	4	1
OMACETAXINE MEPESUCCINATE	4	1
QUIZARTINIB	4	1
SORAFENIB	4	1
THIOTEPA	4	1
TISAGENLECLEUCEL	4	1
TREOSULFAN	4	1
DILANUBICEL	2	1
LIRILUMAB	2	1
RIMIDUCID	2	1
RIVOGENLECLEUCEL	2	1
CHEMBL4776881	0	5
CHEMBL3970001	0	2
CHEMBL4790597	0	1
CHEMBL5075192	0	1
CHEMBL5173987	0	1
CHEMBL5199540	0	1

Precision-medicine subtype map (CIViC)

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

Molecular subtype	Therapy	Effect	Level	CIViC
KMT2A::v Fusion	EPZ004777	Sensitivity/Response	CIViC D	EID7582