A multicenter prospective observational cohort study to improve diagnostic yield and clinical management of persons with a Bleeding Disorder of Unknown Cause (BDUC-iN study protocol)
Research and Practice in Thrombosis and Haemostasis, vol. 10, pp. 106882
Abstract
Background: In half of individuals referred for evaluation of bleeding symptoms, no hemostatic defect is identified with current standard hemostasis tests. These individuals are classified as having a bleeding disorder of unknown cause (BDUC). The pathophysiological mechanisms underlying the bleeding tendency in BDUC are unknown. The absence of a clear, molecular diagnosis leads to significant challenges for healthcare professionals in terms of diagnostics and clinical management, as well as insecurity in patients and caregivers. Objectives: The BDUC in the Netherlands (BDUC-iN) study aims to improve diagnostic accuracy and optimize treatment strategies among individuals with BDUC by evaluating healthcare delivery, patient outcomes, health-related quality of life and by identifying underlying pathophysiological mechanisms of the bleeding tendency. Methods: This is a multicenter prospective observational cohort study aiming to include 500 persons with BDUC. Diagnostic, therapeutic and fundamental research questions are organized in dedicated work packages. Conclusions: Clinical data is collected longitudinally over a 10-year follow-up period. Quality of life and quality of care are assessed using validated questionnaires. Advanced hemostasis tests and proteogenomics are performed to characterize bleeding phenotypes and potentially identify hemostatic defects.Conclusions: The BDUC-iN study applies a structured approach to address current challenges in BDUC. It aims to improve clinical management and patient outcomes by attaining diagnostic accuracy and developing pathophysiologically-based treatment strategies. In turn, this will support the development of (evidence-based) clinical guidelines and shape future research initiatives in BDUC.
Authors 20
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Erasmus MC · Erasmus MC - Sophia Children’s Hospital
Affiliation as printed
Department of Pediatric Hematology and Oncology, Erasmus MC Sophia Children's Hospital, University Medical Center Rotterdam, Rotterdam, Netherlands
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Maastricht University Medical Centre · Maastricht University
Affiliation as printed
CARIM-Cardiovascular Research Institute Maastricht, Maastricht University, Maastricht, Netherlands
Department of Hematology, Maastricht University Medical Center and Hemophilia Treatment Center Nijmegen-Eindhoven-Maastricht, Maastricht, Netherlands
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Maastricht University Medical Centre · Maastricht University
Affiliation as printed
CARIM-Cardiovascular Research Institute Maastricht, Maastricht University, Maastricht, Netherlands
Department of Hematology, Maastricht University Medical Center and Hemophilia Treatment Center Nijmegen-Eindhoven-Maastricht, Maastricht, Netherlands
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Maastricht University Medical Centre · Maastricht University
Affiliation as printed
CARIM-Cardiovascular Research Institute Maastricht, Maastricht University, Maastricht, Netherlands
Department of Hematology, Maastricht University Medical Center and Hemophilia Treatment Center Nijmegen-Eindhoven-Maastricht, Maastricht, Netherlands
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Maastricht University Medical Centre · Maastricht University
Affiliation as printed
Department of Hematology, Maastricht University Medical Center and Hemophilia Treatment Center Nijmegen-Eindhoven-Maastricht, Maastricht, Netherlands
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Maastricht University Medical Centre · Maastricht University
Affiliation as printed
CARIM-Cardiovascular Research Institute Maastricht, Maastricht University, Maastricht, Netherlands
Department of Hematology, Maastricht University Medical Center and Hemophilia Treatment Center Nijmegen-Eindhoven-Maastricht, Maastricht, Netherlands
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Affiliation as printed
Department of Molecular Hematology, Sanquin Research, Amsterdam, Netherlands
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Maastricht University Medical Centre · Maastricht University
Affiliation as printed
CARIM-Cardiovascular Research Institute Maastricht, Maastricht University, Maastricht, Netherlands
Central Diagnostic Laboratory, Maastricht University Medical Center, Maastricht, Netherlands
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Affiliation as printed
Department of Molecular Hematology, Sanquin Research, Amsterdam, Netherlands
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Utrecht University · University Medical Center Utrecht
Affiliation as printed
Center for Benign Haematology, Thrombosis and Haemostasis, Van Creveldkliniek, University Medical Center Utrecht, Utrecht, Netherlands
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Radboud University Nijmegen · Radboud University Medical Center
Affiliation as printed
Department Hematology, Radboud University Medical Center, Nijmegen, Netherlands
Hemophilia Treatment Centre Nijmegen-Eindhoven-Maastricht, Nijmegen, Netherlands
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Sanquin · Emma Kinderziekenhuis · Amsterdam University Medical Centers · University of Amsterdam
Affiliation as printed
Department Pediatric Hematology, Amsterdam UMC Emma Children's Hospital, University of Amsterdam, Amsterdam, Netherlands
Department of Molecular Cellular Hemostasis, Sanquin Research, Amsterdam, Netherlands
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University Medical Center Groningen
Affiliation as printed
Department Hematology, University Medical Center Groningen, Groningen, Netherlands
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Leiden University Medical Center
Affiliation as printed
Department of Medicine-Thrombosis and Hemostasis, Leiden University Medical Center, Leiden, Netherlands
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Affiliation as printed
Department Hematology, Maxima Medical Center, Veldhoven, Netherlands
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Affiliation as printed
Department Hematology, Erasmus MC, University Medical Center Rotterdam, Rotterdam, Netherlands
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Affiliation as printed
Department Hematology, Erasmus MC, University Medical Center Rotterdam, Rotterdam, Netherlands
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Affiliation as printed
Department Hematology, Erasmus MC, University Medical Center Rotterdam, Rotterdam, Netherlands
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Erasmus MC · Erasmus MC - Sophia Children’s Hospital
Affiliation as printed
Department of Pediatric Hematology and Oncology, Erasmus MC Sophia Children's Hospital, University Medical Center Rotterdam, Rotterdam, Netherlands
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Maastricht University Medical Centre · Maastricht University
Affiliation as printed
CARIM-Cardiovascular Research Institute Maastricht, Maastricht University, Maastricht, Netherlands
Department of Hematology, Maastricht University Medical Center and Hemophilia Treatment Center Nijmegen-Eindhoven-Maastricht, Maastricht, Netherlands
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