Trial reportPediatric blood & cancer2016
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Determine the Effect of Romiplostim on Health-Related Quality of Life in Children with Primary Immune Thrombocytopenia and Associated Burden in Their Parents.
Trial report in Pediatric blood & cancer, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03324087 (A Multi-Center Prospective Study of Evaluating Health Related Quality of Life in Adult With Immune Thrombocytopenia in China Using Real-world Data), which is not on this map. Cited by 20 papers, 1 of them a synthesis 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 Multi-Center Prospective Study of Evaluating Health Related Quality of Life in Adult With Immune Thrombocytopenia in China Using Real-world Data
Who cites it
20 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Comparative efficacy of 19 drug therapies for patients with idiopathic thrombocytopenic purpura: a multiple-treatments network meta-analysis.Annals of hematology · 2022Pooled it
- Health-related quality of life in Chinese children with immune thrombocytopaenia: a multicentre study.BMJ paediatrics open · 2026Article
- Platelet-related parameters at initial diagnosis for predicting the risk of chronicity in children with newly diagnosed immune thrombocytopenia.Frontiers in pediatrics · 2026Article
- Rilzabrutinib for the Treatment of Immune Thrombocytopenia.European journal of haematology · 2025Review
- Advances in management of pediatric chronic immune thrombocytopenia: a narrative review.Journal of Yeungnam medical science · 2023Article
- RilzabrutinibTherapeutic advances in hematology · 2023Article
- Romiplostim in children with newly diagnosed or persistent primary immune thrombocytopenia.Annals of hematology · 2021Review
- Delayed treatment-free response after romiplostim discontinuation in pediatric chronic immune thrombocytopenia.Yeungnam University journal of medicine · 2021Article
- Outcomes of Eltrombopag Treatment and Development of Iron Deficiency in Children with Immune Thrombocytopenia in TurkeyTurkish journal of haematology : official journal of Turkish Society of Haematology · 2020Article
- The Need for Comprehensive Care for Persons with Chronic Immune Thrombocytopenic Purpura.Journal of blood medicine · 2020Review
- Romiplostim for the Treatment of Immune Thrombocytopenia: Spotlight on Patient Acceptability and Ease of Use.Patient preference and adherence · 2020Review
- Updated international consensus report on the investigation and management of primary immune thrombocytopenia.Blood advances · 2019Article
- The child with immune thrombocytopenia: to treat or not to treat, is that still the question?Haematologica · 2019Article
- Second-line treatments in children with immune thrombocytopenia: Effect on platelet count and patient-centered outcomes.American journal of hematology · 2019Observational
- Romiplostim for the management of pediatric immune thrombocytopenia: drug development and current practice.Blood advances · 2019Review
- A Narrative Approach to Describe QoL in Children With Chronic ITP.Frontiers in pediatrics · 2019Article
- Efficacy and safety of thrombopoietin receptor agonists in children with chronic immune thrombocytopenia: a meta-analysis.Oncotarget · 2018Article
- Immune thrombocytopenia: improving quality of life and patient outcomes.Patient related outcome measures · 2018Review
- Spotlight on romiplostim in the treatment of children with chronic immune thrombocytopenia: design, development, and potential place in therapy.Drug design, development and therapy · 2017Review
- Second-line therapies in immune thrombocytopenia.Hematology. American Society of Hematology. Education Program · 2016Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundChronic immune thrombocytopenia (ITP) in children can negatively impact their health-related quality of life (HRQoL) and impose a burden on their parents. This study sought to examine the effect of romiplostim on HRQoL and parental burden in children with primary ITP. PROCEDURE: This was a phase 3, randomized, double-blind, placebo-controlled study. Children aged <18 years with ITP ≥6 months were randomly assigned to receive romiplostim or placebo for 24 weeks. The Kids' ITP Tool (KIT) was used to measure HRQoL and was administered to patients and/or their parents at baseline and weeks 8, 16, and 25. Mean KIT scores at each assessment and mean changes in KIT scores from baseline were calculated overall by treatment group and platelet response status. Psychometric properties of the KIT were evaluated and the minimally important difference (MID) was estimated for different KIT versions.
resultsSixty-two patients (42 romiplostim and 20 placebo) were enrolled. Changes in KIT scores by treatment group showed numerically greater and more often statistically significant improvements from baseline to each assessment for children receiving romiplostim versus placebo. Mixed-effects analysis demonstrated statistically significantly greater reduction in parental burden from baseline in the romiplostim group versus placebo. Ranges for the MID were estimated as 9-13 points for the Child Self-Report version and 11-13 points for the Parent Impact version.
conclusionsThe treatment with romiplostim may be associated with improved HRQoL in children with primary ITP and reduced burden to their parents.
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