Evidence map›Paper›PMID 41583536›Full record

ArticleTherapeutic advances in hematology2026

Outcomes in participants switching from FVIII replacement therapy to efanesoctocog alfa prophylaxis in XTEND-1: a post hoc analysis.

Sophie Susen, Roshni Kulkarni, Keiji Nogami, Flora Peyvandi, Barbara Konkle, Elena Santagostino, Umer Khan, Annemieke Willemze, Linda Bystrická, Jennifer Dumont and 1 more

Registry-linked trialAbstract read
In one paragraph

Article in Therapeutic advances in hematology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04161495 (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.

NCT04161495 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 Patients ≥12 Years of Age With Severe Hemophilia A

TypeinterventionalSponsorBioverativ, a Sanofi companyRan2019 to 2022Enrolled159ConditionsFactor VIII DeficiencyArmsefanesoctocog alfa (BIVV001)
3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. 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

11 authors.

Sophie SusenHemostasis and Transfusion Department, Heart and Lung Institute, CHU de Lille, Bd du Professeur Jules Leclercq, 59037 Lille Cedex, France.ORCID https://orcid.org/0000-0001-5953-163X
Roshni KulkarniDepartment of Pediatrics and Human Development, Michigan State University, East Lansing, MI, USA.
Keiji NogamiNara Medical University, Nara, Japan.
Flora PeyvandiAngelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
Barbara KonkleWashington Center for Bleeding Disorders and the University of Washington, Seattle, WA, USA.ORCID https://orcid.org/0000-0002-3959-8797
Elena SantagostinoSobi, Basel, Switzerland.
Umer KhanSanofi, Cambridge, MA, USA.
Annemieke WillemzeSanofi, Amsterdam, The Netherlands.
Linda BystrickáSobi, Basel, Switzerland.
Jennifer DumontSanofi, Cambridge, MA, USA.
Pratima ChowdaryKatharine Dormandy Haemophilia and Thrombosis Centre, Royal Free Hospital, London, UK.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Background: Efanesoctocog alfa is a first-in-class high-sustained factor VIII (FVIII) replacement therapy. In the phase III XTEND-1 (NCT04161495) study, once-weekly efanesoctocog alfa prophylaxis (50 IU/kg) was well-tolerated and achieved high-sustained factor levels in the normal to near-normal range (>40%) for most of the week. Objective: To report outcomes in previously treated participants with severe haemophilia A aged ⩾12 years from an observational study who switched to efanesoctocog alfa prophylaxis during XTEND-1. Design: Paired assessment of participants from an observational study who enrolled in the phase III XTEND-1 study. Methods: Seventy-eight participants switched from marketed standard half-life (SHL) or extended half-life (EHL) FVIII prophylaxis to once-weekly efanesoctocog alfa prophylaxis (50 IU/kg). Endpoints included annualized bleed rates (ABRs), treatment of bleeding episodes, injection frequency and FVIII consumption. Results: Pre-study, 44 (56%) and 34 (44%) participants received SHL FVIII or EHL FVIII prophylaxis, respectively. In the overall population, a significant reduction in ABR from 2.96 to 0.69 ( Conclusion: Collectively, the results of this post hoc analysis demonstrate the benefits of once-weekly efanesoctocog alfa prophylaxis over SHL or EHL FVIII prophylaxis on bleed rates, injection frequency and consumption. Trial registration: Observational study: 242HA201/OBS16221; XTEND-1: NCT04161495 (https://clinicaltrials.gov/study/NCT04161495).

Indexed as

clinical studyfactor VIIIhaemophilia aprospective studiestreatment switching

Identifiers

PMID41583536
PMCPMC12824134

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
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.