Trial reportBlood advances2025
Eltrombopag in combination with immunosuppressive therapy in pediatric severe aplastic anemia: phase 2 ESCALATE trial.
Trial report in Blood advances, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03025698 (A Phase II, Open-label, Non-controlled, Intra-patient Dose-escalation Study to Characterize the Pharmacokinetics After Oral Administration of Eltrombopag in Pediatric Patients With Refractory, Relapsed or Treatment Naive Severe Aplastic Anemia or Recurrent Aplastic Anemia), which is not on this map. Cited by 8 papers.
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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 Phase II, Open-label, Non-controlled, Intra-patient Dose-escalation Study to Characterize the Pharmacokinetics After Oral Administration of Eltrombopag in Pediatric Patients With Refractory, Relapsed or Treatment Naive Severe Aplastic Anemia or Recurrent Aplastic Anemia
Who cites it
8 citing papers in PubMed.
- PTCy-based allo-BMT platform as a curative, accessible alternative for pediatric and young adult severe aplastic anemia.Blood advances · 2026Article
- Evolving Patterns of TPO-RA Use in Children: A Decade of Single-Centre Experience and Narrative Review.International journal of molecular sciences · 2026Review
- Eltrombopag Combined With Cyclosporine A in the Treatment of Pediatric Patients With SAA: Establishing the Range for Eltrombopag Concentrations.Clinical and translational science · 2026Article
- Real-world use of thrombopoietic agents in treatment-naïve children with severe aplastic anemia: a multicenter retrospective study.Scientific reports · 2026Article
- Blood Transfusion Practices in Rural Hospitals: Indications, Challenges, and Practical Solutions.Cureus · 2026Review
- Machine learning mortality prediction model for cyclosporine therapy in pediatric aplastic anemia.Annals of hematology · 2026Article
- Real-world retrospective analysis of clinical outcomes in pediatric acquired aplastic anemia: a single-center 10-year cohort study.Frontiers in pediatrics · 2026Article
- ESCALATing care? Eltrombopag in pediatric aplastic anemia.Blood advances · 2025Article
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Authors and funding
26 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
abstractSevere aplastic anemia (SAA) is a rare, life-threatening disease with acquired pancytopenia and hypocellular bone marrow. ESCALATE evaluated eltrombopag in combination with immunosuppressive therapy (IST) in pediatric patients (aged 1 to <18 years) with relapsed/refractory (R/R) or treatment-naïve SAA. The eltrombopag starting dose was 25 mg/d for patients aged 1 to <6 years and 50 mg/d for patients aged 6 to <18 years; dose modifications (maximum dose, 150 mg/d) were allowed to achieve a target platelet count of 50 × 109/L to 200 × 109/L. Eltrombopag was administered with cyclosporine A, with or without horse antithymocyte globulin, for 26 weeks and could be extended if clinically beneficial. Fifty-one patients were treated (R/R SAA, n = 14; treatment-naïve SAA, n = 37). Data were analyzed overall and as 2 cohorts: R/R and treatment-naïve cohorts. The overall response rate (ORR; per North American Pediatric Aplastic Anemia Consortium criteria) at 26 weeks was 54.9% in both cohorts combined and 71.4% and 48.6% in the R/R and treatment-naïve cohorts, respectively; most responders had sustained responses after discontinuing eltrombopag. Among baseline transfusion-dependent patients, 66.7% and 76.7% achieved red blood cell and platelet transfusion independence, respectively, with rates of 70% and 80% for the R/R cohort and 65.6% and 75.8% for the treatment-naïve cohort, respectively. The most common treatment-related adverse events were abnormalities in liver function tests, including increased bilirubin (43.1%), alanine aminotransferase (37.3%), and aspartate aminotransferase (33.3%). Eltrombopag with IST showed a trend toward a favorable ORR in the R/R cohort, with no new safety signals. This trial was registered at www.clinicaltrials.gov as #NCT03025698.
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