GATA1-related cytopenia
MONDO:0100089
A heterogeneous inherited disorder marked by varying combinations of thrombocytopenia, dyserythropoietic anemia, macrothrombocytopenia, β‑thalassemia‑like red‑cell abnormalities, and/or neutropenia, reflecting impaired transcriptional regulation of erythroid and megakaryocytic development. Affected individuals may present with mucocutaneous bleeding, anemia, abnormal platelet morphology, or multilineage cytopenias, with severity ranging from isolated thrombocytopenia to syndromic tri‑lineage involvement.
Research intelligence
Directly mapped studies only · unique clinical trialsCurrent registry observations. Phase counts may overlap. Study start dates are not registry registration dates. Investigational interventions are not approved-treatment recommendations.
Medications under investigation
Verified disease and drug mappings that co-occur within the same study; not evidence of effectiveness.
MONDO disease family
Recruiting studies by city
Trial status
Trial phase
Study start year
Investigated drug interventions
Drugs being studied
Disease identity and terminology: MONDO. Trial relationships: ClinicalTrials.gov.