Evidence map›Paper›PMID 40099428›Full record

Trial reportHaemophilia : the official journal of the World Federation of Hemophilia2025

Perioperative Management With Efanesoctocog Alfa in Patients With Haemophilia A in the Phase 3 XTEND-1 and XTEND-Kids Studies.

Robert Klamroth, Annette von Drygalski, Cedric Hermans, Young-Shil Park, Anthony K C Chan, Alphan Kupesiz, María Teresa Alvarez-Román, Lynn Malec, Elena Santagostino, Graham Neill and 5 more

2 registry-linked trialsAbstract readClinical Trial, Phase III
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Haemophilia : the official journal of the World Federation of Hemophilia, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing 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)
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

4 citing papers in PubMed.

  1. Article
  2. Article
  3. Review
  4. 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

15 authors.

Robert KlamrothVivantes Klinikum, Friedrichshain, Berlin, and Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn, Germany.ORCID https://orcid.org/0000-0003-4194-8183
Annette von DrygalskiDepartment of Medicine, Division of Hematology/Oncology, University of California San Diego, San Diego, California, USA.
Cedric HermansHaemostasis and Thrombosis Unit, Division of Haematology, Cliniques Universitaires Saint-Luc, Université Catholique de Louvain, Brussels, Belgium.ORCID https://orcid.org/0000-0001-5429-8437
Young-Shil ParkDepartment of Pediatrics, Division of Hematology/Oncology, Kyung Hee University Hospital at Gangdong, Seoul, South Korea.ORCID https://orcid.org/0000-0002-6643-4245
Anthony K C ChanMcMaster Children's Hospital, McMaster University, Hamilton, Canada.ORCID https://orcid.org/0000-0003-1551-3995
Alphan KupesizAkdeniz University, Antalya, Turkey.ORCID https://orcid.org/0000-0001-8827-5567
María Teresa Alvarez-RománHematology Department, Hospital La Paz, Madrid, Spain.ORCID https://orcid.org/0000-0003-3296-4288
Lynn MalecVersiti Blood Research Institute, Milwaukee, Wisconsin, USA.ORCID https://orcid.org/0000-0001-9173-7179
Elena SantagostinoSobi, Basel, Switzerland.
Graham NeillSanofi, Reading, UK.
Linda BystrickáSobi, Basel, Switzerland.ORCID https://orcid.org/0000-0002-9787-7866
Jennifer DumontSanofi, Cambridge, Massachusetts, USA.
Lydia Abad-FranchSobi, Basel, Switzerland.
Lila-Sabrina FetitaSanofi, Paris, France.
Liane KhooInstitute of Haematology, Royal Prince Alfred Hospital, Sydney, NSW, Australia.

Funding

Sanofi (Cambridge, MA)Sobi (Stockholm, Sweden)
6 · The paper itself

Abstract

introductionThe Phase 3 studies, XTEND-1 (NCT04161495) and XTEND-Kids (NCT04759131), showed once-weekly efanesoctocog alfa provided high-sustained factor VIII (FVIII) activity levels that translated into highly effective bleed prevention in patients with severe haemophilia A.

aimThis analysis evaluated the efficacy and safety of efanesoctocog alfa for perioperative management during XTEND-1 and XTEND-Kids.

methodsPatients undergoing major or minor surgery were to receive a single preoperative 50 IU/kg dose, with additional 30 or 50 IU/kg doses every 2-3 days as needed following major surgery. Outcomes assessed included FVIII activity levels, number and dose of efanesoctocog alfa injections, surgeon's/investigator's assessment of haemostatic response, total factor consumption, estimated blood loss, number and type of blood transfusions, and safety.

resultsIn XTEND-1, 11 adults/adolescents underwent 12 evaluable major surgeries (6 orthopaedic). Eleven surgeries had one preoperative dose (median [range]: 49.9 [13-52] IU/kg); one had no preoperative dose. Median (range) total consumption from Day -1 to 14 was 163.3 (45-361) IU/kg. In XTEND-Kids, two children underwent major surgery with a single preoperative loading dose (60.4 and 61.9 IU/kg). Across trials, 15 adults/adolescents underwent 18 minor surgeries and 8 children underwent 9 minor surgeries, with a single preoperative dose or no preoperative dose (5 surgeries in adults/adolescents). Haemostatic response was rated excellent for all surgeries. No surgeries required blood transfusion. No safety concerns or inhibitor development was reported.

conclusionEfanesoctocog alfa provided highly effective perioperative protection in patients with severe haemophilia A.

trial registrationXTEND-1: NCT04161495 https://clinicaltrials.gov/study/NCT04161495; XTEND-Kids: NCT04759131 https://clinicaltrials.gov/study/NCT04759131.

Indexed as

Factor VIIIHemophilia APerioperative CareAdolescentAdultBlood Loss, SurgicalChildFemaleHumansMaleMiddle AgedTreatment OutcomeYoung AdultFactor VIIIadultsBIVV001childrenhaematologyhaemophiliahaemostasissurgical procedures, operative

Identifiers

PMID40099428
PMCPMC12175115

What OpenQuestion holds

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LicenceCC BY
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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.