Evidence map›Paper›PMID 30864154›Full record

ReviewBritish journal of haematology2019

How I manage patients with Wiskott Aldrich syndrome.

Elizabeth Rivers, Austen Worth, Adrian J Thrasher, Siobhan O Burns

Registry-linked trialOpen access · bronzeAbstract readReview
In one paragraph

Review in British journal of haematology, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT06591260 (Role of Endomyocardial Biopsy and Aetiology-based Treatment in Pediatric Patients with Inflammatory Heart Disease in Arrhythmic and Non-arrhythmic Clinical Presentations), which is not on this map. Cited by 30 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
30citing papers in PubMed, 1 pooled it
4.1field-weighted citation impact, top 5% of its field
1 · What the graph read from 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.

2 · The registry

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.

NCT06591260 recruitingnot on this map

Role of Endomyocardial Biopsy and Aetiology-based Treatment in Pediatric Patients with Inflammatory Heart Disease in Arrhythmic and Non-arrhythmic Clinical Presentations: an Integrated Approach for the Optimal Diagnostic and Therapeutic Management

Typeobservational_patient_registrySponsorScientific Institute San RaffaeleRan2013 to 2030Enrolled20ConditionsMyocarditis, Ventricular Arrhythmia, Inflammatory Cardiomyopathy, Genetic PredispositionArmssupport treatment, cardiac medical treatment, aetiology-specific treatment, device implant, arrhythmia ablation
3 · Its place in the literature

Who cites it

30 citing papers in PubMed, 1 synthesis or guideline pooled it, 58 citations in OpenAlex.

  1. Pooled it
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  11. [Research progress on the cGAS-STING signaling pathway in immune-mediated inflammatory diseases in children].Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics · 2025
    Review
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

4 authors at 3 institutions in 1 country.

Elizabeth RiversUniversity College London Great Ormond Street Institute of Child Health, London, UK.
Austen WorthGreat Ormond Street Hospital for Children NHS Foundation Trust, London, UK.
Adrian J ThrasherUniversity College London Great Ormond Street Institute of Child Health, London, UK.
Siobhan O BurnsDepartment of Immunology, Royal Free London NHS Foundation Trust, London, UK.ORCID 0000-0002-3356-9506
Great Ormond Street Hospital · GBGreat Ormond Street Hospital for Children NHS Foundation Trust · GBRoyal Free London NHS Foundation Trust · GB

Funding

The Wellcome Trust 090233/Z/09/ZWellcome Trust 090233Wellcome Trust 201250Wellcome Trust 201250/Z/16/Z
6 · The paper itself

Abstract

Wiskott Aldrich syndrome (WAS) is a primary immunodeficiency disease resulting in recurrent infections, eczema and microthrombocytopaenia. In its classical form, significant combined immune deficiency, autoimmune complications and risk of haematological malignancy necessitate early correction with stem cell transplantation or gene therapy. A milder form, X-linked thrombocytopaenia (XLT), shares similar bleeding risk from thrombocytopaenia but is not associated with other significant clinical features and is generally managed conservatively. Here, we detail our approach to the diagnosis and treatment of classical WAS and XLT.

Indexed as

Autoimmune DiseasesConservative TreatmentEczemaFemaleGenetic TechniquesGenetic TherapyHematopoietic Stem Cell TransplantationHeterozygoteHumansInfection ControlMaleMutationRisk FactorsThrombocytopeniaWiskott-Aldrich Syndromehaematopoietic stem cell transplantimmunodeficiencyWiskott Aldrich syndromeX-linked thrombocytopenia

Identifiers

PMID30864154
PMCPMC7612067
OpenAlexW2921913141

What OpenQuestion holds

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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.