Evidence map›Paper›PMID 39667975›Full record

Trial reportEuropean journal of haematology2025

BAY 81-8973 Demonstrates Long-Term Safety and Efficacy in Children With Severe Haemophilia A: Results From the LEOPOLD Kids Extension Study.

Rolf Ljung, Anthony K C Chan, Sanjay P Ahuja, Maria Elisa Mancuso, Jose Francisco Cabre Marquez, Florian Volk, Victor Blanchette, Bryce A Kerlin, Sonata Saulyte Trakymiene, Heidi Glosli and 1 more

Registry-linked trialAbstract readClinical Trial, Phase IIIMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Trial report in European journal of haematology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT01311648 (A Multicenter Phase III Uncontrolled Open-label Trial to Evaluate Safety and Efficacy of BAY81-8973 in Children With Severe Hemophilia A Under Prophylaxis Therapy), which is not on this map. Not yet cited in PubMed.

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0cells of the map it votes in
0citing papers in PubMed
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1 · What the graph read from it

What it found

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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.

NCT01311648 phase3completednot on this map

A Multicenter Phase III Uncontrolled Open-label Trial to Evaluate Safety and Efficacy of BAY81-8973 in Children With Severe Hemophilia A Under Prophylaxis Therapy

TypeinterventionalSponsorBayerRan2011 to 2020Enrolled94ConditionsHaemophilia AArmsRecombinant Factor VIII (Kovaltry, BAY81-8973)
3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

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.

Rolf LjungDepartment of Clinical Sciences Lund-Pediatrics, Lund University, Lund, Sweden.ORCID https://orcid.org/0000-0003-3999-8747
Anthony K C ChanMcMaster Children's Hospital, McMaster University, Hamilton, Ontario, Canada.
Sanjay P AhujaDepartment of Pediatric Hematology & Oncology, University Hospitals Rainbow Babies and Children's Hospital, Cleveland, Ohio, USA.
Maria Elisa MancusoCenter for Thrombosis and Hemorrhagic Diseases, IRCCS Humanitas Research Hospital, Milan, Italy.
Jose Francisco Cabre MarquezGlobal Medical Affairs, Bayer, Mexico City, Mexico.
Florian VolkClinStat GmbH, Bayer AG, Huerth, Germany.
Victor BlanchetteDivision of Hematology/Oncology, the Hospital for Sick Children, Toronto, Ontario, Canada; Department of Pediatrics, Faculty of Medicine, University of Toronto, Toronto, Ontario, Canada.
Bryce A KerlinDivision of Hematology/Oncology/Bone Marrow Transplant, Nationwide Children's Hospital, Columbus, Ohio, USA.
Sonata Saulyte TrakymieneClinic of Children's Diseases, Faculty of Medicine, Vilnius University, Vilnius University Hospital Santaros Klinikos, Vilnius, Lithuania.
Heidi GlosliCenter for Rare Disorders and Department of Pediatric Research, Oslo University Hospital, Oslo, Norway.
Gili KenetSheba Medical Center, The Israeli National Hemophilia Center, The Amalia Biron Thrombosis Research Institute and The Sackler Medical School, Tel Aviv University, Tel-Hashomer, Israel.

Funding

Bayer
6 · The paper itself

Abstract

objectivesTo report the long-term safety and efficacy of BAY 81-8973 in the LEOPOLD Kids extension phase.

methodsPatients received BAY 81-8973 (25-50 IU/kg) at least twice weekly. The primary endpoint was safety, assessed in all patients who entered the extension phase (n = 82). Efficacy endpoints were assessed in patients without high-titre inhibitors/immune tolerance induction (n = 67).

resultsChildren (n = 82) received BAY 81-8973 for a median of 3.1 years per patient and a median of 405 exposure days per patient. Long-term BAY 81-8973 treatment was well tolerated, with no cases of de novo inhibitor development in the extension phase. Annualised bleeding rates (ABRs) within 48 h of prophylaxis were low for all bleeds (median [IQR], 0.7 [0-1.9]; mean, 1.4 [SD, 2.1]) and for joint bleeds (median [IQR], 0 [0-0.7]; mean, 0.5 [SD, 1.1]) (n = 67). Twenty-one of 67 patients (31.3%) had zero bleeds within 48 h of prophylaxis; the treatment response was 'good'/'excellent' in 87.9% of bleeds, and most bleeds resolved with ≤ 2 BAY 81-8973 infusions (83.5%).

conclusionLong-term BAY 81-8973 treatment is well tolerated and maintains low ABRs for all bleeds and joint bleeds in children with severe haemophilia A.

trial registrationClinicalTrials.gov identifier: NCT01311648.

Indexed as

Factor VIIIHemophilia AAdolescentChildChild, PreschoolFemaleHemorrhageHumansInfantMaleSeverity of Illness IndexTreatment OutcomeFactor VIIIclinical trialfactor VIIIhaemophilia Along‐termprophylaxis

Identifiers

PMID39667975
PMCPMC11798761

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