Trial reportCancer2018
Addition of eltrombopag to immunosuppressive therapy in patients with newly diagnosed aplastic anemia.
Trial report in Cancer, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 papers, 4 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
24 citing papers in PubMed, 4 syntheses or guidelines pooled it, 36 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
- Initiation timing and treatment duration of eltrombopag added to immunosuppressive therapy in aplastic anemia: a systematic review and meta-analysis.Frontiers in oncology · 2026Pooled it
- Efficacy and safety of immunosuppressive therapy combined with eltrombopag for severe aplastic anemia: a systematic review and meta-analysis.Systematic reviews · 2024Pooled it
- Comparisons Between Frontline Therapy and a Combination of Eltrombopag Plus Immunosuppression Therapy and Human Leukocyte Antigen-Haploidentical Hematopoietic Stem Cell Transplantation in Patients With Severe Aplastic Anemia: A Systematic Review.Frontiers in oncology · 2021Pooled it
- 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
- 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 with horse anti-thymocyte globulin and cyclosporine in acquired aplastic anemia.International journal of hematology · 2026Article
- Review
- A Multicenter Retrospective Study Comparing Immunosuppressive Therapy Combined with Eltrombopag to Immunosuppressive Therapy Alone as Frontline Treatment for Pediatric Severe Aplastic AnemiaTurkish journal of haematology : official journal of Turkish Society of Haematology · 2025Article
- Long-Term Follow-Up of Eltrombopag Treatment for Patients with Cyclosporin A Refractory/Relapsed Transfusion-Dependent Non-Severe Aplastic Anemia: A Report from a Single Center in China.Acta haematologica · 2025Article
- Modified Delphi panel consensus recommendations for management of severe aplastic anemia.Blood advances · 2024Article
- Review
- Progress in medical therapy in aplastic anemia: why it took so long?International journal of hematology · 2024Review
- Multicenter evaluation of the addition of eltrombopag to immunosuppressive therapy for adults with severe aplastic anemia.International journal of hematology · 2023Article
- Prolonged use of eltrombopag in patients with severe aplastic anemia in the real world.Clinical and experimental medicine · 2023Article
- A multicentre trial of intensive immunosuppressive therapy combined with umbilical cord blood for the treatment of severe aplastic anaemia.Annals of hematology · 2022Article
- A New Immunosuppressive Therapy for Very Severe Aplastic Anemia in Children with Autoantibodies.Current medical science · 2022Article
- Adding eltrombopag to immunosuppression: the importance of predicting outcome.Haematologica · 2022Article
- Predicting Response of Severe Aplastic Anemia to Rabbit-Antithymocyte Immunoglobulin Based Immunosuppressive Therapy Combined With Eltrombopag.Frontiers in immunology · 2022Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
16 authors at 1 institution in 1 country.
Funding
Abstract
backgroundThe immune-mediated destruction of hematopoietic stem cells is implicated in the pathophysiology of aplastic anemia (AA). Immunosuppressive therapy (IST) using antithymocyte globulin and cyclosporine is successful in this setting. Eltrombopag is active in patients with refractory AA, presumably by increasing the bone marrow progenitors.
methodsThis phase 2 trial initially was designed to evaluate standard IST in newly diagnosed patients with severe AA and later was amended to add eltrombopag to simultaneously address immune destruction and stem cell depletion. The primary outcome was the overall response rate (ORR) at 3 months and 6 months.
resultsA total of 38 patients were enrolled: 17 (45%) received IST alone and 21 (55%) received additional eltrombopag. The ORR was 74%. Patients receiving IST plus eltrombopag had a similar ORR (76% vs 71%; P = .72), complete remission rate (38% vs 29%; P = .73), and median time to response (84 days vs 57 days; P = .30) compared with those receiving IST alone. The 2-year overall survival rate in the IST group was 91% compared with 82% for those patients treated with IST plus eltrombopag (P = .82). No cumulative toxicities were noted after the addition of eltrombopag.
conclusionsThe addition of eltrombopag to standard IST was well tolerated and resulted in similar responses.
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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.