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Disease

Anemia, Aplastic

Late-stage therapeutic developmentEmerging research
MeSHAnemia, Aplastic
1
Publications
10
Clinical trials
11
Associated genes
4
Related proteins
1985
Latest publication
Current focus
Perforin-1 biologyTherapeutic developmentThrombopoietin biology
Latest activity
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Recent clinical, regulatory, research and industry developments relating to this disease.

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

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Clinical trials

5 sponsors · 0 new · 0 completed in the last 12 months (net +0)

The current development programme across all trial phases.

Clinical programme
10
All trials
1
Active
2
Late-stage
6
Completed
Recently completed

Efficacy and Safety of Thrombopoetin-Receptor Agonist Eltrombopag in in Combination With Ciclosporin A in Moderate Aplastic Anemia (EMAA): Prospective Randomized Multicenter Study

Phase 2/3 · Completed · B. Höchsmann

Horse ATG/CsA in Aplastic Anemia Patients Unresponsive to or With a Suboptimal Response to Rabbit ATG/CsA Treatment

Phase 2 · Completed · National Heart, Lung, and Blood Institute (NHLBI)

Fludarabine-based Conditioning for Allogeneic Marrow Transplantation From HLA-compatible Unrelated Donors in Severe Aplastic Anemia (BMT CTN #0301)

Phase 1/2 · Completed · Medical College of Wisconsin

A Pilot Study of a Thrombopoietin-Receptor Agonist (TPO-R Agonist), Eltrombopag, in Aplastic Anemia Patients With Immunosuppressive-Therapy Refractory Thrombocytopenia

Phase 2 · Completed · National Heart, Lung, and Blood Institute (NHLBI)

A Pilot Study of Recombinant Humanized Anti- Cluster of Differentiation Antigen 20 (Anti-CD20) Antibody (Rituximab) in Patients With Moderate Aplastic Anemia, Pure Red Cell Aplasia, or Diamond Blackfan Anemia

Phase 2 · Completed · National Heart, Lung, and Blood Institute (NHLBI)

Multi-Center Trial of Anti-Thymocyte Globulin in Treatment of Aplastic Anemia and Other Hematologic Disorders

Phase 3 · Completed · National Heart, Lung, and Blood Institute (NHLBI)

Research activity

1 papers

Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.

Leading journals1
  • Annals of internal medicine1
Leading researchers7
  • Bennett JM1
  • Catovsky D1
  • Daniel MT1
  • Flandrin G1
  • Galton DA1
  • Gralnick HR1
  • Sultan C1

Associated genes

11 matches

Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.

NBN0.72TERT0.71PRF10.65SBDS0.57CSF3R0.56PALD10.56MPL0.54ACTRT30.52BCAP290.28ADH50.19ANAPC20.01

Disease biology

4 matches

Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.

Telomerase reverse transcriptasevia TERT0.71Perforin-1via PRF10.65Granulocyte colony-stimulating factor receptorvia CSF3R0.56Thrombopoietin receptorvia MPL0.54

Reference

Authoritative identity, definition & identifiers.

Defined in MeSH

A form of anemia in which the bone marrow fails to produce adequate numbers of peripheral blood elements.

Identifiers
MeSHAnemia, Aplastic
References & data sources
  • Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
  • Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
  • Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
  • Related entities are derived from literature co-mention (studied together) — associative, not causal.