Evidence map›Paper›PMID 40612895›Full record

ArticleTH open : companion journal to thrombosis and haemostasis2025

Prospective, Observational Study of the Clinical Outcomes of FVIII Treatment in Adults and Adolescents with Severe Haemophilia A.

Pratima Chowdary, Liane Khoo, Michael Wang, Hervé Chambost, Anthony K C Chan, Annemieke Willemze, Johannes Oldenburg

Erratum issuedAbstract read
In one paragraph

Article in TH open : companion journal to thrombosis and haemostasis, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

7 authors.

Pratima ChowdaryKatharine Dormandy Haemophilia and Thrombosis Centre, Royal Free Hospital, London, United Kingdom.
Liane KhooHaemophilia Treatment Centre, Royal Prince Alfred Hospital, Sydney, Australia.
Michael WangHemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Colorado, United States.
Hervé ChambostAP-HM, Department of Pediatric Hematology Oncology, Children Hospital La Timone & Aix Marseille University, INSERM, INRA, C2VN, Marseille, France.
Anthony K C ChanDepartment of Pediatrics, McMaster Children's Hospital, McMaster University, Ontario, Canada.
Annemieke WillemzeSanofi, Amsterdam, The Netherlands.
Johannes OldenburgInstitute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn, Germany.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Objective: To assess real-world treatment patterns and outcomes in previously treated patients ≥12 years old with severe haemophilia A treated with marketed factor VIII (FVIII) replacement products. Methods: Data were collected prospectively between 25 January 2019 and 30 November 2020 across 45 sites in 17 countries. Primary endpoint was annualized bleed rate (ABR). Secondary endpoints included factor consumption, bleed treatment, joint health, and safety. Exploratory endpoints included pain and quality of life outcomes. Results: A total of 157 patients received ≥1 FVIII injection (prophylaxis Conclusions: Prophylaxis using standard or extended half-life FVIII replacement therapies achieves adequate haemostatic control in only about half of patients, with some experiencing very poor outcomes. Real-world data highlight the urgent need to optimize prophylaxis to enhance haemostatic control, ideally achieving a zero ABR and its associated benefits.

Indexed as

clinical studyfactor VIIIhaemophilia aobservational studyprospective studies

Identifiers

PMID40612895
PMCPMC12223957

What OpenQuestion holds

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Registered trials

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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.