Evidence map›Paper›PMID 37529276›Full record

ArticleTherapeutic advances in hematology2023

Real-world data in patients with congenital hemophilia and inhibitors: final data from the FEIBA Global Outcome (FEIBA GO) study.

Carmen Escuriola Ettingshausen, Cedric Hermans, Pål A Holme, Ana R Cid, Kate Khair, Johannes Oldenburg, Claude Négrier, Jaco Botha, Aurelia Lelli, Jerzy Windyga

Abstract read
In one paragraph

Article in Therapeutic advances in hematology, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing 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

3 citing papers in PubMed.

  1. Observational
  2. Article
  3. Review
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

10 authors.

Carmen Escuriola EttingshausenHämophilie-Zentrum Rhein Main - HZRM GmbH, Mörfelden-Walldorf, Germany.
Cedric HermansHemostasis and Thrombosis Unit, Division of Haematology, Cliniques Universitaires Saint-Luc, UCLouvain, Brussels, Belgium.ORCID https://orcid.org/0000-0001-5429-8437
Pål A HolmeDepartment of Haematology, Oslo University Hospital and Institute for Clinical Medicine, University of Oslo, Oslo, Norway.
Ana R CidUnidad de Hemostasia y Trombosis, Hospital Universitario y Politécnico La Fe, València, Spain.
Kate KhairCentre for Outcomes and Experience Research in Children's Health, Illness and Disability (ORCHID), Great Ormond Street Hospital for Children, London, UK.
Johannes OldenburgInstitute of Experimental Hematology and Transfusion Medicine, Bonn University Clinic, Bonn, Germany.
Claude NégrierUniversity Claude Bernard Lyon 1, Lyon, France.
Jaco BothaTakeda Pharmaceuticals International AG, Zürich, Switzerland.
Aurelia LelliTakeda Pharmaceuticals International AG, Zürich, Switzerland.
Jerzy WindygaDepartment of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Background: The bypassing agent, activated prothrombin complex concentrate [aPCC, FEIBA (factor VIII inhibitor bypass activity); Baxalta US Inc, a Takeda company, Lexington, MA, USA], is indicated for the treatment of bleeding episodes, perioperative management, and routine prophylaxis in patients with hemophilia A or B with inhibitors. In certain countries, aPCC is also indicated for the treatment of bleeding episodes and perioperative management in patients with acquired hemophilia A. Objectives: To describe long-term, real-world effectiveness, safety, and quality-of-life outcomes for patients with congenital hemophilia A or B and high-responding inhibitors receiving aPCC treatment in routine clinical practice. Design: FEIBA Global Outcome (FEIBA GO; EUPAS6691) was a prospective, observational study. Methods: Investigators determined the treatment regimen and clinical monitoring frequency. The planned patient observation period was 4 years. Data are from the safety analysis set (patients who received ⩾1 aPCC infusion). Results: Overall, 50 patients received either aPCC prophylaxis ( Conclusion: This study demonstrated the long-term, real-world effectiveness and consistent safety profile of aPCC as on-demand therapy and prophylactic treatment in patients with hemophilia and high-responding inhibitors. Trial registry: FEIBA Global Outcome Study; EUPAS6691 https://www.encepp.eu/encepp/viewResource.htm?id=32774.

Indexed as

factor VIII inhibitor bypassing activityhemophilia Ahemophilia Binhibitorsobservational study

Identifiers

PMID37529276
PMCPMC10387704

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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.