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Aplastic anemia without a known cause.
Features include always present findings: Low red blood cell count (anemia); and very common findings: Bone marrow hypocellularity. 11 total HPO annotations.
Organ System | Phenotype Count | Example Features |
|---|---|---|
Blood and immune system | 7 | Reticulocytopenia, Gingival bleeding, Low platelet count (thrombocytopenia) |
Biomarker and diagnostic research for idiopathic aplastic anemia has been reported in the published literature.
Phenotype severity distribution: 1 always present feature, 1 very common feature, 2 common features.
Estimated prevalence: 1-9 in 1,000,000 (Rare).
109 clinical trials registered, 49 recruiting. Interventions under study include drug therapy, other interventions, procedural interventions, and biologic therapy. Pipeline includes 5 PHASE4, 8 PHASE3, 41 PHASE2. Research is sponsored by a mix of industry and academic institutions.
NCT ID | Title | Phase | Sponsor | Status |
|---|---|---|---|---|
[NCT01351545](https://clinicaltrials.gov/study/NCT01351545) |
Data assembled from 5 of 12 sources · Last updated Sep 17, 2026, 11:53 PM UTC
European rare disease database
Genetic and Rare Diseases Info Center
Eyes
1 |
Retinal hemorrhage |
Bones and joints | 1 | Bone marrow hypocellularity |
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs) |
— |
Center for International Blood and Marrow Transplant Research |
RECRUITING |
[NCT05126849](https://clinicaltrials.gov/study/NCT05126849) | Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation With Post-transplant Cyclophosphamide in Patients With Acquired Refractory Aplastic Anemia or in Relapse After Immunosuppression | PHASE2 | Assistance Publique - Hôpitaux de Paris | RECRUITING |
[NCT06622694](https://clinicaltrials.gov/study/NCT06622694) | CD7 CAR T Cells (RD13-02) in the Treatment of Relapsed/Refractory Severe Aplastic Anemia | PHASE1 | Institute of Hematology & Blood Diseases Hospital, China | RECRUITING |
[NCT06633328](https://clinicaltrials.gov/study/NCT06633328) | CD7 CAR-T Bridging to alloHSCT for Severe Aplastic Anemia | EARLY_PHASE1 | Zhejiang University | RECRUITING |
[NCT04558736](https://clinicaltrials.gov/study/NCT04558736) | Haploidentical HCT for Severe Aplastic Anemia | PHASE2 | St. Jude Children's Research Hospital | RECRUITING |
14 publications have been identified in PubMed for idiopathic aplastic anemia. Research spans Case Report / Case Series (36%), Basic Science / Preclinical (21%), and Review / Meta-Analysis (14%).
Research Type | Count | % of Total |
|---|---|---|
Patient case studies | 5 | 36% |
Laboratory research | 3 | 21% |
Research summaries | 2 | 14% |
Disease patterns and progression | 2 | 14% |
Testing and diagnosis research | 1 | 7% |
Clinical study results | 1 | 7% |
Papadimitriou K (2026). [PMID: 38715430](https://pubmed.ncbi.nlm.nih.gov/38715430/). *Interv Neuroradiol*. [Review / Meta-Analysis]
Bukhari SI (2026). [PMID: 41445363](https://pubmed.ncbi.nlm.nih.gov/41445363/). *Expert review of hematology*. [Diagnostic / Biomarker]
Abbas D (2026). [PMID: 41478762](https://pubmed.ncbi.nlm.nih.gov/41478762/). *La Revue de medecine interne*. [Case Report / Case Series]
Pinc Z (2025). [PMID: 41188636](https://pubmed.ncbi.nlm.nih.gov/41188636/). *Journal of clinical immunology*. [Basic Science / Preclinical]
Lee AC (2025). [PMID: 40829064](https://pubmed.ncbi.nlm.nih.gov/40829064/). *Journal of pediatric hematology/oncology*. [Case Report / Case Series]
Maeda T (2025). [PMID: 40259860](https://pubmed.ncbi.nlm.nih.gov/40259860/). *British journal of haematology*. [Case Report / Case Series]
Kwon YD (2025). [PMID: 40358701](https://pubmed.ncbi.nlm.nih.gov/40358701/). *Annals of hematology*. [Epidemiology / Natural History]
Palla S (2025). [PMID: 40209608](https://pubmed.ncbi.nlm.nih.gov/40209608/). *Blood cells, molecules & diseases*. [Basic Science / Preclinical]
Maeda T (2024). [PMID: 38296352](https://pubmed.ncbi.nlm.nih.gov/38296352/). *British journal of haematology*. [Case Report / Case Series]
Foucault A (2024). [PMID: 39726306](https://pubmed.ncbi.nlm.nih.gov/39726306/). *Discovery medicine*. [Review / Meta-Analysis]
AI-curated news mentioning idiopathic aplastic anemia
Updated Jul 31, 2026
A recent population-based study investigates the incidence, outcomes, and health-related quality of life in childhood aplastic anemia. The findings contribute valuable insights into the disease's impact on affected children.
A nationwide post-marketing surveillance study in Japan evaluates the long-term safety and effectiveness of romiplostim in patients with refractory aplastic anemia. The findings contribute to understanding treatment outcomes in this rare disease.
A new nomogram has been developed and validated for early prognostic prediction in children with aplastic anemia undergoing cyclosporine monotherapy. This tool aims to enhance treatment outcomes by providing timely prognostic insights.