Evidence map›Paper›PMID 42786406›Full record

ArticleThe patient2026

Experiences with Efanesoctocog Alfa: Exit Interviews with Caregivers of Previously Treated XTEND-Kids Phase III Trial Patients with Haemophilia A.

Lynn Malec, Manuel Carcao, Mary Mathias, Nana Kragh, Jennifer Dumont, Linda Bystrická, Sriya Gunawardena, Amanda Wilson, Katherine Kosa, Dana DiBenedetti

Registry-linked trialAbstract read
PubMed Publisher
In one paragraph

Article in The patient, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04759131 (A Phase 3 Open-label, Multicenter Study of the Safety, Efficacy, and Pharmacokinetics of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein), 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
–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.

NCT04759131 phase3completednot on this map

A Phase 3 Open-label, Multicenter Study of the Safety, Efficacy, and Pharmacokinetics of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein (rFVIIIFc-VWF-XTEN; BIVV001) in Previously Treated Pediatric Patients <12 Years of Age With Severe Hemophilia A

TypeinterventionalSponsorBioverativ, a Sanofi companyRan2021 to 2023Enrolled74ConditionsHemophilia AArmsefanesoctocog alfa (BIVV001)
3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Article
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.

Lynn MalecVersiti Blood Research Institute, Milwaukee, WI, USA.
Manuel CarcaoDivision of Haematology/Oncology, Hospital for Sick Children, University of Toronto, Toronto, ON, Canada.
Mary MathiasHaemophilia Centre, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.
Nana KraghGlobal Health Economics and Outcomes Research, Sobi, Stockholm, Sweden.
Jennifer DumontSanofi, Cambridge, MA, USA.
Linda BystrickáSobi, Basel, Switzerland.
Sriya GunawardenaSanofi, Morristown, NJ, USA.
Amanda WilsonSanofi, Cambridge, MA, USA.
Katherine KosaPatient-Centered Outcomes Assessment, RTI Health Solutions, Durham, NC, USA.
Dana DiBenedettiPatient-Centered Outcomes Assessment, RTI Health Solutions, Durham, NC, USA. ddibenedetti@rti.org.ORCID http://orcid.org/0000-0003-2294-9519

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundUntil recently, most treatments for haemophilia A consisted of regular prophylaxis with factor VIII (FVIII) replacement therapy, which, to be effective, needed to be given multiple times per week. Efanesoctocog alfa is a once-weekly FVIII replacement therapy that has demonstrated highly effective bleed prevention in patients aged < 12 years with haemophilia A during the 52-week phase III XTEND-Kids trial (ClinicalTrials.gov identifier: NCT04759131).

objectivesWe conducted exit interviews with caregivers of XTEND-Kids trial patients to understand their experiences with efanesoctocog alfa.

methodsCaregivers were asked questions, via telephone exit interviews, on their pre- and post-trial experiences to determine the impact of efanesoctocog alfa on their and their child's lives. Interviews were audio-recorded, transcribed, and analysed using an applied thematic analysis to identify key themes and trends. Quantitative data were obtained to inform the qualitative analysis.

resultsInterviews were conducted with 19 caregivers of 20 children with haemophilia A. Prior to the trial, caregivers described substantial impacts on children's mood and emotional wellbeing (reported for 19/20 [95%] of the children by their caregivers), physical activity (14/20; 70%), and caregiver burden. After the switch to efanesoctocog alfa, most caregivers reported improvements across multiple domains, including reduced bleeding and bruising, improved mood and emotional wellbeing (18/19; 94.7%), and increased physical activity (10/14; 71.4%). All reported at least one improvement in their child; many described reduced treatment burden due to less frequent injections.

conclusionsCaregiver-reported outcomes suggest that efanesoctocog alfa may meaningfully reduce disease and treatment burden, with improvements observed across children's daily functioning and emotional wellbeing, and caregiver experience.

Identifiers

PMID42786406

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.