Evidence map›Paper›PMID 40323006›Full record

Observational studyHaemophilia : the official journal of the World Federation of Hemophilia2025

Real-World Unmet Needs of Patients With Haemophilia A and Haemophilia B With or Without Inhibitors: End-of-Study Results From the explorer6 Non-Interventional Study.

Allison P Wheeler, Aby Abraham, Chris Barnes, Renée Brown Frandsen, Roseline d'Oiron, Hermann Eichler, Kingsley Hampton, Francisco José López-Jaime, Chuhl Joo Lyu, Camila Martins Mazini Tavares and 5 more

Registry-linked trialAbstract readMulticenter StudyObservational Study
PubMed Full text at PMC Publisher
In one paragraph

Observational study 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 trial NCT03741881 (A Prospective, Multi-national, Non-interventional Study in Haemophilia A and B Patients With or Without Inhibitors Treated According to Routine Clinical Treatment Practice), which is not on this map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing 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.

NCT03741881 completednot on this map

A Prospective, Multi-national, Non-interventional Study in Haemophilia A and B Patients With or Without Inhibitors Treated According to Routine Clinical Treatment Practice (Explorer™6)

TypeobservationalSponsorNovo Nordisk A/SRan2018 to 2021Enrolled231ConditionsHaemophilia A, Haemophilia A With Inhibitors, Haemophilia B, Haemophilia B With InhibitorsArmsNo treatment given
3 · Its place in the literature

Who cites it

2 citing papers in PubMed.

  1. Article
  2. Observational
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.

Allison P WheelerWashington Centre for Bleeding Disorders, Seattle, Washington, USA.ORCID https://orcid.org/0000-0003-3967-4873
Aby AbrahamDepartment of Haematology, Christian Medical College, Vellore, India.
Chris BarnesThe Royal Children's Hospital, Melbourne, VIC, Australia.
Renée Brown FrandsenNovo Nordisk A/S, Søborg, Denmark.
Roseline d'OironCentre de Référence de l'Hémophilie et de Maladies Hémorragiques Rares, Hôpital Bicêtre AP-HP and INSERM Hémostase inflammation thrombose HITH U1176, Université Paris-Saclay, Le Kremlin-Bicêtre, France.ORCID https://orcid.org/0000-0002-4843-7805
Hermann EichlerInstitute of Clinical Haemostaseology and Transfusion Medicine, Saarland University, Homburg, Germany.ORCID https://orcid.org/0000-0002-1372-0619
Kingsley HamptonDepartment of Cardiovascular Science, University of Sheffield, Sheffield, UK.ORCID https://orcid.org/0000-0002-5091-7197
Francisco José López-JaimeHemostasis and Thrombosis Unit, Málaga Regional University Hospital, IBIMA, Málaga, Spain.
Chuhl Joo LyuDepartment of Paediatric Haematology-Oncology, Yonsei Cancer Center, Severance Hospital, Yonsei University College of Medicine, Seoul, South Korea.
Camila Martins Mazini TavaresNovo Nordisk A/S, Søborg, Denmark.
Keiji NogamiDepartment of Paediatrics, Nara Medical University, Kashihara, Nara, Japan.ORCID https://orcid.org/0000-0002-2415-2194
Christopher SuttonDepartment of Paediatrics and Child Health, Faculty of Health Sciences, University of Limpopo and Pietersburg and Mankweng Hospitals, Limpopo, South Africa.
Jerzy WindygaDepartment of Haemostasis Disorders and Internal Medicine, Laboratory of Haemostasis and Metabolic Diseases, Institute of Haematology and Transfusion Medicine, Warsaw, Poland.
Bulent ZulfikarDepartment of Paediatric Haematology/Oncology, Istanbul University Oncology Institute, Istanbul, Turkey.ORCID https://orcid.org/0000-0002-7586-6939
Giancarlo CastamanCentre for Bleeding Disorders, Department of Oncology, Careggi University Hospital, Florence, Italy.ORCID https://orcid.org/0000-0003-4973-1317

Funding

Novo Nordisk A/S
6 · The paper itself

Abstract

introductionHaemophilia is associated with high disease and treatment burdens. Prospective evaluation of data from patients with haemophilia helps understand and define unmet needs, optimise treatment and improve healthcare outcomes.

aimTo present end-of-study data from explorer6 (NCT03741881), a prospective, non-interventional study across multiple countries in patients with haemophilia (haemophilia A or B without [HA or HB] or with inhibitors [HAwI or HBwI]).

methodsPatients ≥12 years old with severe HA, severe/moderate HB or HAwI/HBwI of any severity were treated according to the local standard of care (SoC). The number of bleeding episodes from enrolment up to a maximum of 115 weeks, physical activity based on data collected by a wrist-worn physical activity tracker, target joints and Haemophilia Joint Health Score (HJHS) measurements were assessed.

resultsA total of 231 patients across 33 countries were enrolled. The mean annualised bleeding rate (ABR) (standard deviation) for treated bleeding episodes was investigated for patients receiving prophylaxis (HA: 4.7 [5.9]; HB: 2.2 [3.0]; HAwI: 10.3 [8.5]; HBwI: 12.4 [14.1]) and those receiving on-demand (OnD) treatment (HA: 21.5 [17.7]; HB: 10.5 [8.6]; HAwI: 15.2 [14.8]; HBwI: 9.3 [13.3]). Physical activity levels were lowest among patients with HBwI. Target joints were similar among haemophilia types and treatments. Overall, mean and median HJHS total scores were lower for patients receiving prophylaxis compared with OnD treatment.

conclusionThe explorer6 study assessed a large haemophilia patient population in a real-world setting across 33 countries. The results indicate that an unmet need remains among patients receiving OnD treatment and those with inhibitors.

trial registrationClinicalTrials.gov identifier: NCT03741881.

Indexed as

Hemophilia AHemophilia BAdolescentAdultFemaleHemorrhageHumansMaleMiddle AgedProspective StudiesYoung Adultanti‐tissue factor pathway inhibitor (TFPI)factor VIIIhaemophiliaprophylaxistreatment burden

Identifiers

PMID40323006
PMCPMC12462599

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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.