Trial reportAnnals of medicine2023
Avatrombopag, a promising novel thrombopoietin receptor agonist for refractory/relapsed/intolerant non-severe aplastic anemia: a phase 2 single-arm clinical trial.
Trial report in Annals of medicine, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 3 registered trials, which are not on this map. Cited by 10 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Efficacy and Safety of Avatrombopag in Non-severe Aplastic Anemia - a Multicenter Prospective Single Arm Study
Ciclosporin + Antithymocyte Immunoglobulin + Avatrombpag Versus Ciclosporin + Avatrombpag for the Treatment of Severe Aplastic Anemia in the Elderly
An Exploratory Study on the Efficacy and Safety of Lusutrombopag in the Treatment of Recurrent/Refractory/Intolerable NSAA
Who cites it
10 citing papers in PubMed, 13 citations in OpenAlex.
- [Efficacy and safety of romiplostim N01 for refractory aplastic anemia: a retrospective single-center study].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2026Article
- Efficacy and safety of different TPO-RAs combined with cyclosporine in the treatment of NSAA.Therapeutic advances in hematology · 2026Article
- Efficacy and safety of avatrombopag in aplastic anemia: a comprehensive review of clinical evidence.Therapeutic advances in hematology · 2026Review
- Effective treatment of refractory aplastic anemia with romiplostim after failure of multiple thrombopoietin receptor agonists: a single-center retrospective study.Annals of medicine · 2025Article
- FZD6 mediates the contributing role of miR-199a-5p in the adipogenesis of bone marrow mesenchymal stem cells derived from aplastic anemia.Scientific reports · 2025Article
- Avatrombopag for the Treatment of Immune Thrombocytopenia.European journal of haematology · 2025Review
- Exploring the Potential of Pyridine Carboxylic Acid Isomers to Discover New Enzyme Inhibitors.Drug design, development and therapy · 2025Review
- Cyclosporine plus avatrombopag versus cyclosporine monotherapy for first-line treatment of elderly patients with transfusion-dependent non-severe aplastic anemia: a single center retrospective study.Annals of medicine · 2024Article
- Modified Delphi panel consensus recommendations for management of severe aplastic anemia.Blood advances · 2024Article
- Avatrombopag for adults with early versus chronic immune thrombocytopenia.American journal of hematology · 2024Observational
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
introductionThe therapeutic options for thrombocytopenia in non-severe aplastic anaemia (NSAA) are limited. Avatrombopag (AVA) is prescribed for thrombocytopenic diseases but not for NSAA.
methodsHerein, we conducted a phase 2, non-randomized, single-arm trial to explore the efficacy and safety of AVA in refractory/relapsed/intolerant NSAA. AVA dose was initiated at 20 mg/d and titrated to a maximum of 60 mg/d. The primary endpoint was the haematological response at 3 months.
resultsTwenty-five patients were analyzed. The overall response rate (ORR) at 3 months was 56% (14/25), with 12% (3/25) achieving a complete response (CR). At a median follow-up of 7 (3-10) months, the OR and CR rates were 52% and 20%, respectively. Responders had a shorter duration of diagnosis of AVA administration than non-responders (10 (6-80)
conclusionAVA is effective and well-tolerated in NSAA patients who are refractory, relapsed, or intolerant to CsA/tacrolimus ± eltrombopag. Earlier treatment and relapsed/intolerant AA may show a better short-term response rate. More studies are needed to define the optimal dose and the long-term efficacy (NCT04728789).
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.