Acquired aplastic anemia

disease

On this page

Also known as rare acquired aplastic anaemia

Summary

Acquired aplastic anemia (MONDO:0015610) is a disease and 9 clinical trials. Top therapeutic interventions include eltrombopag. A subtype of aplastic anemia — broader associated-gene and molecular evidence is on the parent page (see Disease family below).

At a glance

Clinical trials: 9

Clinical features

No curated clinical features (Orphanet) for this disease.

Identifiers

Disease identifiers

Field	Value
Canonical name	acquired aplastic anemia
Mondo ID	MONDO:0015610
Orphanet	164823
SNOMED CT	55907008
UMLS	C0271907
MedGen	543648
GARD	0020058
Is cancer (heuristic)	no

Also known as: acquired aplastic anemia · rare acquired aplastic anaemia

Disease family

This is a subtype of aplastic anemia. 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 › human disease › disease by body system or component › hematologic disorder › anemia › aplastic anemia › acquired aplastic anemia

Subtypes (3): primary myelofibrosis, primary acquired red cell aplasia, paroxysmal nocturnal hemoglobinuria

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

No druggable-target or therapeutic data for this disease’s cohort.

Clinical trials & evidence

Clinical trials

Clinical trials: 9.

Phase distribution (across all retrieved trials)

Phase	Trials
Not specified	4
PHASE2	3
PHASE3	1
PHASE1/PHASE2	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT03413306	PHASE3	UNKNOWN	Eltrombopag+hATG+CsA vs. hATG+CsA in Children With Severe AA
NCT06839456	PHASE1/PHASE2	RECRUITING	Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT
NCT01343953	PHASE2	COMPLETED	Cord Blood Transplantation in Severe Aplastic Anemia
NCT01530555	PHASE2	COMPLETED	Prospective Phase II Study of Rabbit Antithymocyte Globulin (ATG, Thymoglobuline®, Genzyme) With Ciclosporin for Patients With Acquired Aplastic Anaemia
NCT01759732	PHASE2	UNKNOWN	Haploidentical Stem Cell Transplantation for Children With Acquired Severe Aplastic Anemia
NCT03047746	Not specified	ACTIVE_NOT_RECRUITING	Unrelated And Partially Matched Related Donor PSCT w/ T Cell Receptor (TCR) αβ Depletion for Patients With BMF
NCT07259863	Not specified	NOT_YET_RECRUITING	Clinico-Epidemiological Profile Of Acquired Aplastic Anemia Among Children At Assiut Governorate .
NCT01719341	Not specified	UNKNOWN	Study of Non-Myeloablative Haplo-identical Haematopoietic Stem Cell Transplantation in Patients With Haematological Malignancies or Acquired Aplastic Anaemia
NCT03904134	Not specified	COMPLETED	Clinical Transplant-Related Long-term Outcomes of Alternative Donor Allogeneic Transplantation (BMT CTN 1702)

Drugs tested across these trials (top 30)

Molecule	Max phase	Trials referencing
ELTROMBOPAG	4	1

Drugs: Eltrombopag