Eltrombopag for Newly Diagnosed Pediatric Immune Thrombocytopenia Requiring Treatment
Bibliographic record
Abstract
Importance: Eltrombopag, a thrombopoietin receptor agonist, is approved by the US Food and Drug Administration for children with chronic immune thrombocytopenia. Efficacy of eltrombopag during the newly diagnosed phase of pediatric immune thrombocytopenia is unknown. Objective: To determine if the proportion of patients with a platelet response is significantly greater in patients with newly diagnosed immune thrombocytopenia treated with eltrombopag than in those treated with standard therapy (first-line treatments). Design, Setting, and Participants: This phase 3, randomized clinical trial enrolled patients (aged 1-<18 years) with newly diagnosed primary immune thrombocytopenia (platelet count <30 × 109/L who required pharmacological treatment but did not have severe bleeding or need a rapid increase in platelet count) from May 7, 2019, to January 25, 2024, at 23 centers participating in the Pediatric ITP Consortium of North America. Final follow-up occurred on February 26, 2025. Interventions: Eltrombopag was administered orally based on a standard dosing schedule (n = 78) vs standard therapy (investigator choice of glucocorticoids, intravenous immunoglobulin, or anti-D immunoglobulin) (n = 40). Main Outcomes and Measures: The primary outcome was a sustained platelet response defined as 3 or more of 4 platelet counts greater than 50 × 109/L during weeks 6 to 12 without rescue treatment. The secondary outcomes included bleeding scores, change in health-related quality of life, and serious adverse events. Results: Of 118 pediatric patients (median age, 8 years [IQR, 4-12 years]; 49% were male), 63% experienced an initial treatment failure after observation or medical therapy. Enrollment ended after a planned interim analysis met a prespecified threshold for efficacy. Of 71 patients in the eltrombopag group, 46 (65% [95% CI, 54%-76%]) had a sustained platelet response compared with 13 of 37 patients (35% [95% CI, 20%-51%]) in the standard therapy group (between-group difference, 30% [95% CI, 11%-49%]; P = .002), which crossed the monitoring boundary for efficacy. Overall, there was no between-group difference in the number and type of adverse events. Conclusions and Relevance: In pediatric patients with newly diagnosed immune thrombocytopenia requiring pharmacological treatment, eltrombopag resulted in a higher rate of sustained platelet response compared with standard therapy. Eltrombopag may be an effective option for pediatric patients with newly diagnosed immune thrombocytopenia with nonsevere bleeding who warrant medical intervention. Trial Registration: ClinicalTrials.gov Identifier: NCT03939637.
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.001 | 0.001 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.001 |
| Bibliometrics | 0.000 | 0.000 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.000 | 0.000 |
| Open science | 0.000 | 0.000 |
| Research integrity | 0.000 | 0.001 |
| Insufficient payload (model declined to judge) | 0.002 | 0.000 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".