Trial reportBlood advances2023
Efficacy of combined immunosuppression with or without eltrombopag in children with newly diagnosed aplastic anemia.
Trial report in Blood advances, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03413306 (A Phase II Multicenter Randomized Study of Eltrombopag Combined With Cyclosporine and hATG Versus hATG and CsA as First Line Treatment in Pediatric Patients With Severe Acquired Aplastic Anemia), which is not on this map. Cited by 21 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.
A Phase II Multicenter Randomized Study of Eltrombopag Combined With Cyclosporine and hATG Versus hATG and CsA as First Line Treatment in Pediatric Patients With Severe Acquired Aplastic Anemia
Who cites it
21 citing papers in PubMed, 4 syntheses or guidelines pooled it, 35 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 in combination with immunosuppressive therapy in pediatric severe aplastic anemia: phase 2 ESCALATE trial.Blood advances · 2025Trial
- 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
- Review
- Safety and efficacy of romiplostim in children with acquired aplastic anemia who are naïve to immunosuppressive therapy.Haematologica · 2026Article
- Romiplostim for aplastic anemia: beyond an eltrombopag alternative.Haematologica · 2026Article
- 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
- Adjunctive effects of eltrombopag on immunosuppressive therapy for childhood aplastic anemia.International journal of hematology · 2025Article
- Efficacy and influencing factors of immunosuppressive therapy combined with or without eltrombopag in children with severe aplastic anemia.Frontiers in medicine · 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
- Article
- Progress in medical therapy in aplastic anemia: why it took so long?International journal of hematology · 2024Review
- The treatment and outcome prediction analysis of pediatric acquired severe aplastic anemia.American journal of stem cells · 2024Review
- The state of the art in the treatment of severe aplastic anemia: immunotherapy and hematopoietic cell transplantation in children and adults.Frontiers in immunology · 2024Review
- The efficacy and the safety of eltrombopag in pediatric patients with severe aplastic anemia: a systematic review.Frontiers in pediatrics · 2023Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
36 authors at 8 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
We compared the efficacy and safety of eltrombopag (ELTR) combined with immunosuppressive therapy (IST) and IST alone in treatment-naïve children with severe (SAA) and very severe (vSAA) aplastic anemia. Ninety-eight pediatric patients were randomized to receive horse antithymocyte globulin (hATG) and cyclosporin A (CsA) with (n = 49) or without (n = 49) ELTR. The primary endpoint was the overall response rate (ORR) at 4 months. After 4 months, nonresponders were crossed over to the alternative group. In all patients, the ORR in ELTR + IST and IST groups was similar (65% vs 53%; P = .218); however, the complete response (CR) rate was significantly higher in the ELTR + IST group (31% vs 12%; P = .027). In severity subgroups, the ORR was 89% vs 57% (P = .028) in favor of IST + ELTR in SAA, but it did not differ in patients with vSAA (52% vs 50%; P = .902). At 6 months after the crossover, 61% of initial ELTR(-) patients achieved a response compared with 17% of initial ELTR(+) patients (P = .016). No significant difference in ELTR + IST and IST groups was observed in the 3-year overall survival (OS) (89% vs 91%; P = .673) or the 3-year event-free survival (EFS) (53% vs 41%; P = .326). There was no unexpected toxicity related to ELTR. Adding ELTR to standard IST was well tolerated and increased the CR rate. The greatest benefit from ELTR combined with IST was observed in patients with SAA but not in those with vSAA. The second course of IST resulted in a high ORR in initial ELTR(-) patients who added ELTR and had limited efficacy among patients who received ELTR upfront. This trial was registered at Clinicaltrials.gov as #NCT03413306.
Indexed as
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.