Trial reportBlood2015
Effects of eltrombopag on platelet count and platelet activation in Wiskott-Aldrich syndrome/X-linked thrombocytopenia.
Trial report in Blood, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 44 papers, 2 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.
Effects Of Eltrombopag On Thrombocytopenia, Platelet Function and Bleeding In Patients With Wiskott-Aldrich Syndrome/X-Linked Thrombocytopenia.
Evaluation of Perioperative Eltrombopag for the Management of Elective Surgery and Invasive Acts in Patients With Inherited Thrombocytopenia
Who cites it
44 citing papers in PubMed, 2 syntheses or guidelines pooled it, 94 citations in OpenAlex.
- Alternative agents to prophylactic platelet transfusion for preventing bleeding in people with thrombocytopenia due to chronic bone marrow failure: a meta-analysis and systematic review.The Cochrane database of systematic reviews · 2016Pooled it
- Alternatives, and adjuncts, to prophylactic platelet transfusion for people with haematological malignancies undergoing intensive chemotherapy or stem cell transplantation.The Cochrane database of systematic reviews · 2016Pooled it
- Eltrombopag for the treatment of inherited thrombocytopenias: a phase II clinical trial.Haematologica · 2020Trial
- Bleeding tendency and platelet function during treatment with romiplostim in children with severe immune thrombocytopenic purpura.International journal of hematology · 2017Trial
- Thrombopoietin receptor agonists in inherited thrombocytopenias: an evolving mosaic of evidence.Research and practice in thrombosis and haemostasis · 2026Article
- Eltrombopag use in surgical procedures in inherited thrombocytopenia: expanding the role of thrombopoietin-receptor agonists.Research and practice in thrombosis and haemostasis · 2026Article
- Evolving Patterns of TPO-RA Use in Children: A Decade of Single-Centre Experience and Narrative Review.International journal of molecular sciences · 2026Review
- Identification of a novel nonsense mutation (c.1369 C > T) in the WAS gene in a neonate: a case report and literature review.BMC pediatrics · 2026Review
- Venous Thromboembolism in Inherited Platelet Disorders: A Clinical Challenge.Diagnostics (Basel, Switzerland) · 2025Review
- X-Linked Thrombocytopenia in a 6-Year-Old Boy.Turkish archives of pediatrics · 2025Article
- How I treat Wiskott-Aldrich syndrome.Blood · 2025Review
- Long-term outcome in Wiskott-Aldrich syndrome and X-linked thrombocytopenia patients: an observational -prospective multi-center study of the Italian Primary Immune Deficiency Network (IPINET).EClinicalMedicine · 2025Article
- Expert consensus on the off-label use in China of drugs for rare hematologic diseases (2024 edition).Frontiers in pharmacology · 2024Review
- Changing profile of platelet activity and turnover indices during treatment response of immune thrombocytopenia.Clinical and experimental medicine · 2022Article
- Treatment of inherited thrombocytopenias.Haematologica · 2022Review
- A thrombopoietin receptor agonist to rescue an unusual platelet transfusion-induced reaction in a p.V1316M-associated von Willebrand disease type 2B patient.Therapeutic advances in hematology · 2022Article
- Miniaturized 3D bone marrow tissue model to assess response to Thrombopoietin-receptor agonists in patients.eLife · 2021Article
- Role of Thrombopoietin Receptor Agonists in Inherited Thrombocytopenia.International journal of molecular sciences · 2021Review
- Learning the Ropes of Platelet Count Regulation: Inherited Thrombocytopenias.Journal of clinical medicine · 2021Review
- Hematopoietic Stem Cell Therapy for Wiskott-Aldrich Syndrome: Improved Outcome and Quality of Life.Journal of blood medicine · 2021Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
12 authors at 8 institutions in 2 countries.
Funding
Abstract
unlabelledBecause Wiskott-Aldrich syndrome (WAS) and X-linked thrombocytopenia (XLT) patients have microthrombocytopenia, hemorrhage is a major problem. We asked whether eltrombopag, a thrombopoietic agent, would increase platelet counts, improve platelet activation, and/or reduce bleeding in WAS/XLT patients. In 9 WAS/XLT patients and 8 age-matched healthy controls, platelet activation was assessed by whole blood flow cytometry. Agonist-induced platelet surface activated glycoprotein (GP) IIb-IIIa and P-selectin in WAS/XLT patients were proportional to platelet size and therefore decreased compared with controls. In contrast, annexin V binding showed no differences between WAS/XLT and controls. Eltrombopag treatment resulted in an increased platelet count in 5 out of 8 patients. Among responders to eltrombopag, immature platelet fraction in 3 WAS/XLT patients was significantly less increased compared with 7 pediatric chronic immune thrombocytopenia (ITP) patients. Platelet activation did not improve in 3 WAS/XLT patients whose platelet count improved on eltrombopag. IN
conclusion(1) the reduced platelet activation observed in WAS/XLT is primarily due to the microthrombocytopenia; and (2) although the eltrombopag-induced increase in platelet production in WAS/XLT is less than in ITP, eltrombopag has beneficial effects on platelet count but not platelet activation in the majority of WAS/XLT patients. This trial was registered at www.clinicaltrials.gov as #NCT00909363.
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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.