Evidence map›Paper›PMID 38572481›Full record

ArticleResearch and practice in thrombosis and haemostasis2024

Noninterventional study assessing joint health in persons with hemophilia A after switching to turoctocog alfa pegol: design of pathfinderReal.

Cihan Ay, Olga Benitez-Hidalgo, Gillian Gidley, Maria Elisa Mancuso, Davide Matino, Azusa Nagao, Laszlo Nemes, John Waller, Johannes Oldenburg

Registry-linked trialOpen access · goldAbstract read
In one paragraph

Article in Research and practice in thrombosis and haemostasis, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT05621746 (Non-Interventional Study of the Change in Joint Health in Adult Patients With Haemophilia A After Switching to Prophylaxis With Turoctocog Alfa Pegol), which is not on this map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
0.9field-weighted citation impact, top 26% 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.

NCT05621746 recruitingnot on this map

Non-Interventional Study of the Change in Joint Health in Adult Patients With Haemophilia A After Switching to Prophylaxis With Turoctocog Alfa Pegol (N8-GP)

TypeobservationalSponsorNovo Nordisk A/SRan2022 to 2026Enrolled100ConditionsHaemophilia AArmsEsperoct
3 · Its place in the literature

Who cites it

1 citing paper in PubMed, 2 citations in OpenAlex.

  1. The haemophilia joint health score for the assessment of joint health in patients with haemophilia.Haemophilia : the official journal of the World Federation of Hemophilia · 2024
    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

9 authors at 9 institutions in 9 countries.

Cihan AyClinical Division of Haematology and Haemostaseology, Department of Medicine I, Medical University of Vienna, Vienna, Austria.
Olga Benitez-HidalgoHemophilia Unit, Hematology Department, Hospital Vall d'Hebron, Barcelona, Spain.
Gillian GidleyHaemophilia Comprehensive Care Centre, Department of Haematology, Leeds Teaching Hospitals Trust, Leeds, United Kingdom.
Maria Elisa MancusoCentre for Thrombosis and Hemorrhagic Diseases, IRCCS Humanitas Research Hospital, Rozzano, Milan, Italy.
Davide MatinoDepartment of Medicine, Thrombosis and Atherosclerosis Research Institute, McMaster University, Hamilton, Ontario, Canada.
Azusa NagaoDepartment of Blood Coagulation, Ogikubo Hospital, Tokyo, Japan.
Laszlo NemesNational Hemophilia Center and Hemostasis Department, Central Hospital of Northern Pest - Military Hospital, Budapest, Hungary.
John WallerNovo Nordisk, Zurich, Switzerland.
Johannes OldenburgDepartment of Molecular Haemostasis, University of Bonn, Bonn, Germany.
Humanitas University · ITLeeds Teaching Hospitals NHS Trust · GBMedical University of Vienna · ATMilitary Hospital · PKNovo Nordisk (Switzerland) · CHOgikubo Hospital · JPThrombosis and Atherosclerosis Research Institute · CAUniversity of Bonn · DEVall d'Hebron Hospital Universitari · ES

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Background: Joint damage affects the quality of life of persons with hemophilia A. The long-term safety and efficacy of turoctocog alfa pegol (N8-GP) prophylaxis in persons with hemophilia A has been investigated in pivotal phase 3 trials in children, adolescents, and adults (pathfinder program). However, there is a lack of data on joint health in adult persons with hemophilia A treated with N8-GP. Objectives: To describe the design of the ongoing pathfinderReal study investigating the joint health status in adult persons with hemophilia A after switching to N8-GP. Methods: pathfinderReal is a multicountry, noninterventional, single-arm study (NCT05621746) of joint health in adult (≥18 years) male persons with hemophilia A who have switched to N8-GP. Patients enrolled in other interventional studies and those who have previously terminated N8-GP treatment will be excluded. Approximately 124 adults with hemophilia A will be enrolled and followed up for a maximum of 24 months. Data from routine clinical assessments of patients' joint health will be collected. The primary endpoint is change in Hemophilia Joint Health Score (defined as a change in total score of ≤2) from initiation of N8-GP treatment until the end of the study. Secondary endpoints include number of bleeding episodes, number and resolution of target joints, patient-reported outcomes of problem joint score, pain score, and change in physical function levels. An exploratory endpoint is included to measure the number of patients achieving improved Hemophilia Joint Health Score from the initiation of N8-GP until the end of the study. Conclusion: The pathfinderReal study will provide insights regarding the impact of N8-GP on joint health in persons with hemophilia A in a real-world setting.

Indexed as

factor VIII, hemarthrosis, hemophilia A, joint, N8-GP

Identifiers

PMID38572481
PMCPMC10987329
OpenAlexW4392352631

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

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.