Acute myeloblastic leukemia without maturation

Summary

Acute myeloblastic leukemia without maturation (MONDO:0005224) is a cancer and 1 clinical trial. A subtype of acute myeloid leukemia by FAB classification — broader associated-gene and molecular evidence is on the parent page (see Disease family below).

At a glance

• Classification: Cancer
• Prevalence: <1 / 1 000 000 (Europe) [Orphanet-validated]
• Clinical trials: 1

Clinical features

Epidemiology

Prevalence records

1 prevalence record(s), Orphanet:

Type	Class	Value	Geography	Validation
Annual incidence	<1 / 1 000 000	0.01	Europe	Validated

Identifiers

Disease identifiers

Field	Value
Canonical name	acute myeloblastic leukemia without maturation
Mondo ID	MONDO:0005224
EFO	EFO:0003027
Orphanet	98833
DOID	DOID:0081086
NCIT	C3249
SNOMED CT	359640008
UMLS	C0026998
MedGen	10232
GARD	0000526
Is cancer (heuristic)	yes

Also known as: acute granulocytic leukaemia without maturation · acute granulocytic leukemia without maturation · acute M1 myeloid leukaemia · acute M1 myeloid leukemia · acute myeloblastic leukaemia M1 · acute myeloblastic leukaemia type 1 · acute myeloblastic leukemia M1 · acute myeloblastic leukemia type 1 · acute myelocytic leukaemia without maturation · acute myelocytic leukemia without maturation · acute myelogenous leukaemia without maturation · acute myelogenous leukemia without maturation · acute myeloid leukaemia without maturation · acute myeloid leukaemia without maturation (FAB M1) · acute myeloid leukemia without maturation · acute myeloid leukemia without maturation (FAB M1) · AML M1 · AML without maturation · AWM · FAB M1 (+20 more)

Data availability: 3 cell lines.

Disease family

This is a subtype of acute myeloid leukemia by FAB classification. 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 by etiologic mechanism › cancer or benign tumor › neoplastic disease or syndrome › neoplasm › hematopoietic and lymphoid system neoplasm › hematopoietic and lymphoid cell neoplasm › leukemia › myeloid leukemia › acute myeloid leukemia › acute myeloid leukemia by FAB classification › acute myeloblastic leukemia without maturation

Related subtypes (8): acute myeloid leukemia with minimal differentiation, myeloid sarcoma, acute erythroid leukemia, acute myelomonocytic leukemia M4, acute megakaryoblastic leukemia, acute panmyelosis with myelofibrosis, acute basophilic leukemia, acute myeloblastic leukemia with maturation

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

1 approved, 13 in late-stage (phase 3) trials. Disease-direct ChEMBL indications, not inferred from the associated-gene cohort below.

Drug	Development status
Cytarabine	Approved (phase 4)
Aldesleukin	Phase 3 (in late-stage trials)
Asparaginase	Phase 3 (in late-stage trials)
Busulfan	Phase 3 (in late-stage trials)
Dexamethasone	Phase 3 (in late-stage trials)
Etoposide	Phase 3 (in late-stage trials)
Filgrastim	Phase 3 (in late-stage trials)
Fludarabine Phosphate	Phase 3 (in late-stage trials)
Hydrocortisone	Phase 3 (in late-stage trials)
Idarubicin	Phase 3 (in late-stage trials)
Methotrexate	Phase 3 (in late-stage trials)
Thioguanine	Phase 3 (in late-stage trials)
Tipifarnib	Phase 3 (in late-stage trials)
Valspodar	Phase 3 (in late-stage trials)

Clinical trials & evidence

Clinical trials

Clinical trials: 1.

Phase distribution (across all retrieved trials)

Phase	Trials
PHASE1/PHASE2	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT02944162	PHASE1/PHASE2	UNKNOWN	CAR-pNK Cell Immunotherapy for Relapsed/Refractory CD33+ AML