Trial reportBritish journal of haematology2021
Eltrombopag added to immunosuppression for children with treatment-naïve severe aplastic anaemia.
Trial report in British journal of haematology, 2021. 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 trial NCT01623167 (Eltrombopag Added to Standard Immunosuppression in Treatment-Naive Severe Aplastic Anemia), which is not on this map. Cited by 38 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.
Eltrombopag Added to Standard Immunosuppression in Treatment-Naive Severe Aplastic Anemia
Who cites it
38 citing papers in PubMed, 4 syntheses or guidelines pooled it, 58 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
- 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
- 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
- Eltrombopag in combination with immunosuppressive therapy in pediatric severe aplastic anemia: phase 2 ESCALATE trial.Blood advances · 2025Trial
- 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
- Safety, pharmacokinetics, and preliminary efficacy of hetrombopag in pediatric patients with aplastic anemia: A pilot study.Annals of hematology · 2026Article
- Evolving Patterns of TPO-RA Use in Children: A Decade of Single-Centre Experience and Narrative Review.International journal of molecular sciences · 2026Review
- Review
- Romiplostim for aplastic anemia: beyond an eltrombopag alternative.Haematologica · 2026Article
- Translational potential of γδ T cells in hematologic diseases: from immunobiology to therapeutic innovation.Frontiers in immunology · 2026Review
- Up-front alternative donor HCT in severe aplastic anemia: gaps and opportunities to translate evidence into practice.Blood advances · 2025Review
- 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
- ESCALATing care? Eltrombopag in pediatric aplastic anemia.Blood advances · 2025Article
- Towards graft-Haematologica · 2025Review
- Macrophage polarization, inflammatory monocytes, and impaired MDSCs are associated with murine and human immune aplastic anemia.Journal of leukocyte biology · 2025Article
- Adjunctive effects of eltrombopag on immunosuppressive therapy for childhood aplastic anemia.International journal of hematology · 2025Article
Corrections and comments
- Commented on by
- Erratum issuedCorrigendum.2022
Authors and funding
11 authors at 2 institutions in 2 countries.
Funding
Abstract
Acquired severe aplastic anaemia (SAA) has an immune pathogenesis, and immunosuppressive therapy (IST) with anti-thymocyte globulin and cyclosporine is effective therapy. Eltrombopag (EPAG) added to standard IST was associated with higher overall and complete response rates in patients with treatment-naïve SAA compared to a historical IST cohort. We performed a paediatric subgroup analysis of this trial including all patients aged <18 years who received EPAG plus standard IST (n = 40 patients) compared to a historical cohort (n = 87) who received IST alone. Response, relapse, clonal evolution, event-free survival (EFS), and overall survival were assessed. There was no significant difference in either the overall response rate (ORR) or complete response rate at 6 months (ORR 70% in EPAG group, 72% in historical group, P = 0·78). Adults (≥18 years) had a significantly improved ORR of 82% with EPAG compared to 58% historically (P < 0·001). Younger children had lower response rates than did adolescents. The trend towards relapse was higher and EFS significantly lower in children who received EPAG compared to IST alone. Addition of EPAG added to standard IST did not improve outcomes in children with treatment-naïve SAA. EPAG in the paediatric population should not automatically be considered standard of care. Registration: clinicaltrials.gov (NCT01623167).
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Identifiers
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.