Trial reportThe New England journal of medicine2012
Eltrombopag and improved hematopoiesis in refractory aplastic anemia.
Trial report in The New England journal of medicine, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. It is linked to 5 registered trials, which are not on this map. Cited by 264 papers, 5 of them syntheses that pooled it.
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.
A Pilot Study of a Thrombopoietin-Receptor Agonist (TPO-R Agonist), Eltrombopag, in Aplastic Anemia Patients With Immunosuppressive-Therapy Refractory Thrombocytopenia
Randomized Clinical Trial of the Use of & Cyclosporine in Children With Sever Idiopathic Aplastic Anemia
Open Label Single Arm Prospective Pilot Trial of Eltrombopag in Patients 1 Year to 18 Years of Age Undergoing Intensive Chemotherapy for Malignant Solid Tumors (CCPO011)
Avatrombopag in TPO-RA Refractory Aplastic Anemia Patients Safety and Efficacy Study --Single-arm, Multicenter, Open, Phase Π Clinical Study
Efficacy and Safety Study of Umbilical Cord Blood Transplantation (UCBT) With Total Marrow Irradiation (TMI)-Based Conditioning Regimen for Adults With Refractory/Relapsed Aplastic Anemia (AA).
Who cites it
264 citing papers in PubMed, 5 syntheses or guidelines pooled it, 531 citations in OpenAlex.
- American Society of Hematology 2026 guidelines for the diagnosis and management of severe and very severe acquired aplastic anemia.Blood advances · 2026Guideline
- Indications for haematopoietic cell transplantation and CAR-T for haematological diseases, solid tumours and immune disorders: 2025 EBMT practice recommendations.Bone marrow transplantation · 2025Guideline
- Efficacy of Eltrombopag with Immunosuppressive Therapy Versus Immunosuppressive Therapy Alone on Severe Aplastic Anaemia: A Systematic Review and Meta-analysis.Clinical drug investigation · 2023Pooled it
- Haploidentical hematopoietic stem cell transplantation in aplastic anemia: a systematic review and meta-analysis of clinical outcome on behalf of the severe aplastic anemia working party of the European group for blood and marrow transplantation (SAAWP of EBMT).Bone marrow transplantation · 2020Pooled it
- Efficacy and Safety of Eltrombopag for Aplastic Anemia: A Systematic Review and Meta-analysis.Clinical drug investigation · 2019Pooled it
- Eltrombopag Added to Standard Immunosuppressive Treatment as Front-Line Therapy for Severe Aplastic Anemia: Long-Term Outcomes of the Phase-3 Randomized Superiority EBMT-SAAWP RACE Study.American journal of hematology · 2026Trial
- Trial
- Hetrombopag Added to Cyclosporine as the First-Line Treatment for Patients With Non-Severe Aplastic Anemia: A Phase 2 Multicenter Trial.American journal of hematology · 2026Trial
- Phase II study of the triple combination of rabbit ATG, ciclosporin and eltrombopag in patients with transfusion-dependent aplastic anaemia: West Japan Hematology Study Group (W-JHS) AA02 trial.British journal of haematology · 2026Trial
- Eltrombopag in combination with immunosuppressive therapy in pediatric severe aplastic anemia: phase 2 ESCALATE trial.Blood advances · 2025Trial
- Predictive factors of romiplostim response in patients with refractory aplastic anemia: data from two clinical trials.Annals of hematology · 2025Trial
- Avatrombopag, a promising novel thrombopoietin receptor agonist for refractory/relapsed/intolerant non-severe aplastic anemia: a phase 2 single-arm clinical trial.Annals of medicine · 2023Trial
- Efficacy and safety of eltrombopag in Chinese patients with refractory or relapsed severe aplastic anemia.Scientific reports · 2023Trial
- Alternative treatment modality for severe aplastic anemia in a resource-limited setting: a single-institution prospective cohort study from Upper Egypt.Annals of hematology · 2023Trial
- Eltrombopag for Low-Risk Myelodysplastic Syndromes With Thrombocytopenia: Interim Results of a Phase II, Randomized, Placebo-Controlled Clinical Trial (EQOL-MDS).Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2023Trial
- Eltrombopag with or without Tacrolimus for relapsed/refractory acquired aplastic anaemia: a prospective randomized trial.Blood cancer journal · 2023Trial
- Efficacy of combined immunosuppression with or without eltrombopag in children with newly diagnosed aplastic anemia.Blood advances · 2023Trial
- Iron chelation of hetrombopag in aplastic anemia: a post hoc analysis of a phase II study.Annals of hematology · 2022Trial
- Eltrombopag in Combination with Rabbit Anti-thymocyte Globulin/Cyclosporine A in Immunosuppressive Therapy-naïve Patients with Aplastic Anemia in Japan.Internal medicine (Tokyo, Japan) · 2021Trial
- Efficacy and safety of romiplostim in refractory aplastic anaemia: a Phase II/III, multicentre, open-label study.British journal of haematology · 2021Trial
204 more citing papers are in PubMed but not listed here.
Corrections and comments
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Authors and funding
14 authors at 1 institution in 1 country.
Funding
Abstract
backgroundSevere aplastic anemia, which is characterized by immune-mediated bone marrow hypoplasia and pancytopenia, can be treated effectively with immunosuppressive therapy or allogeneic transplantation. One third of patients have disease that is refractory to immunosuppression, with persistent, severe cytopenia and a profound deficit in hematopoietic stem cells and progenitor cells. Thrombopoietin may increase the number of hematopoietic stem cells and progenitor cells.
methodsWe conducted a phase 2 study involving patients with aplastic anemia that was refractory to immunosuppression to determine whether the oral thrombopoietin mimetic eltrombopag (Promacta) can improve blood counts. Twenty-five patients received eltrombopag at a dose of 50 mg, which could be increased, as needed, to a maximum dose of 150 mg daily, for a total of 12 weeks. Primary end points were clinically significant changes in blood counts or transfusion independence. Patients with a response continued to receive eltrombopag.
resultsEleven of 25 patients (44%) had a hematologic response in at least one lineage at 12 weeks, with minimal toxic effects. Nine patients no longer needed platelet transfusions (median increase in platelet count, 44,000 per cubic millimeter). Six patients had improved hemoglobin levels (median increase, 4.4 g per deciliter); 3 of them were previously dependent on red-cell transfusions and no longer needed transfusions. Nine patients had increased neutrophil counts (median increase, 1350 per cubic millimeter). Serial bone marrow biopsies showed normalization of trilineage hematopoiesis in patients who had a response, without increased fibrosis. Monitoring of immune function revealed no consistent changes.
conclusionsTreatment with eltrombopag was associated with multilineage clinical responses in some patients with refractory severe aplastic anemia. (Funded by the National Heart, Lung, and Blood Institute; ClinicalTrials.gov number, NCT00922883.).
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What OpenQuestion holds
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.