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1. A Blended Physiotherapy Intervention for Persons With Hemophilic Arthropathy: Development Study

Author

Timmer, Merel A, Kloek, Corelien J J, de Kleijn, Piet, Kuijlaars, Isolde A R, Schutgens, Roger E G, Veenhof, Cindy, and Pisters, Martijn F

Subjects
Computer applications to medicine. Medical informatics, R858-859.7, Public aspects of medicine, RA1-1270
Abstract

BackgroundJoint bleeds are the hallmark of hemophilia, leading to a painful arthritic condition called as hemophilic arthropathy (HA). Exercise programs are frequently used to improve the physical functioning in persons with HA. As hemophilia is a rare disease, there are not many physiotherapists who are experienced in the field of hemophilia, and regular physiotherapy sessions with an experienced physiotherapist in the field of hemophilia are not feasible for persons with HA. Blended care is an innovative intervention that can support persons with HA at home to perform the advised physical activities and exercises and provide self-management information. ObjectiveThe aim of this study was to develop a blended physiotherapy intervention for persons with HA. MethodsThe blended physiotherapy intervention, namely, e-Exercise HA was developed by cocreation with physiotherapists, persons with HA, software developers, and researchers. The content of e-Exercise HA was compiled using the first 3 steps of the Center for eHealth Research roadmap model (ie, contextual inquiry, value specification, and design), including people with experience in the development of previous blended physiotherapy interventions, a literature search, and focus groups. ResultsA 12-week blended intervention was developed, integrating face-to-face physiotherapy sessions with a web-based app. The intervention consists of information modules for persons with HA and information modules for physiotherapists, a graded activity program using a self-chosen activity, and personalized video-supported exercises. The information modules consist of text blocks, videos, and reflective questions. The patients can receive pop-ups as reminders and give feedback on the performance of the prescribed activities. ConclusionsIn this study, we developed a blended physiotherapy intervention for persons with HA, which consists of information modules, a graded activity program, and personalized video-supported exercises.

Published
2020
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2. Congenital fibrinogen disorders: a retrospective clinical and genetic analysis of the Prospective Rare Bleeding Disorders Database

Author

Mohsenian, Samin, Palla, Roberta, Menegatti, Marzia, Cairo, Andrea, Lecchi, Anna, Casini, Alessandro, Neerman-Arbez, Marguerite, Asselta, Rosanna, Scardo, Sara, Siboni, Simona Maria, Blatny, Jan, Zapletal, Ondrej, Schved, Jean-Francois, Giansily-Blaizot, Muriel, Halimeh, Susan, Daoud, Mohamad Ayman, Platokouki, Helen, Pergantou, Helen, Schutgens, Roger E. G., Van Haaften-Spoor, Monique, Brons, Paul, Laros-van Gorkom, Britta, Van Pinxten, Elise, Borhany, Munira, Fatima, Naveena, Mikovic, Danijela, Saracevic, Marko, Özdemir, Gül Nihal, Ay, Yılmaz, Makris, Michael, Lockley, Caryl, Mumford, Andrew, Harvey, Andrew, Austin, Steve, Shapiro, Amy, Williamson, Adrianna, McGuinn, Catherine, Goldberg, Ilene, De Moerloose, Philippe, and Peyvandi, Flora

Published
2024
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4. Efficacy of a 1:1 ratio VWF/FVIII concentrate in patients with von Willebrand disease.

Author

Kuppens, Geoffrey Z. L., Fischer, Kathelijn, van Galen, Karin P. M., van Beers, Eduard J., Van der Valk, Paul R., Kremer Hovinga, Idske C. L., van Vulpen, Lize F. D., and Schutgens, Roger E. G.

Subjects
VON Willebrand disease, BLOOD coagulation disorders, VON Willebrand factor, BLOOD coagulation factor VIII, BLOOD coagulation factors
Abstract

Introduction: Patients with von Willebrand disease (VWD) require administration of von Willebrand factor (VWF) concentrates peri‐operatively. Concerns about FVIII accumulation after repetitive injections of a 1:1 ratio VWF/FVIII clotting factor concentrate (CFC) led this study to explore the recovery and FVIII accumulation over time. Methods: This monocentre study examined patients with VWD receiving perioperative 1:1 ratio CFC infusions. CFC dosing was based on body weight and endogenous VWF/FVIII activity. FVIII and VWF activity was monitored at T0 (baseline), T1 (15 min postinfusion), and trough levels at T2‐T6 (24‐120 h). Results: We included 125 patients, undergoing 125 procedures (63 major surgeries, 62 minor), with a median of two CFC infusions (IQR 1–3). With a mean administered dose of 35.7 IU/kg CFC, recovery rates of FVIII and VWF were 2.6 IU/dL per IU/kg and 2.4 IU/dL per IU/kg, respectively. Mean FVIII levels at T0 were 62 (SD 51.9), T1: 164 (SD 80.4), T2: 155 (SD 62.8), T3: 162 (SD 59.8), T4: 124 (SD 78.4), and T5: 120 (SD 65.3) IU/dL. Mean VWF activity levels at T0 were 29 (SD 25.0), T1: 133 (SD 43.7), T2: 92 (SD 37.2), and T3: 86 (SD 37.5) IU/dL. Subgroup analysis in 47 patients with more than three infusions, showed no accumulation of mean FVIII levels. Conclusion: This perioperative study demonstrated excellent FVIII and VWF recovery of a 1:1 ratio VWF product in patients with VWD. Stable FVIII and VWF activity levels were observed after repeated infusions, without accumulation. Most major surgeries required only three CFC infusions. [ABSTRACT FROM AUTHOR]

Published
2024
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6. Apixaban versus no anticoagulation after anticoagulation-associated intracerebral haemorrhage in patients with atrial fibrillation in the Netherlands (APACHE-AF): a randomised, open-label, phase 2 trial

Author

Schreuder, Floris H B M, van Nieuwenhuizen, Koen M, Hofmeijer, Jeannette, Vermeer, Sarah E, Kerkhoff, Henk, Zock, Elles, Luijckx, Gert-Jan, Messchendorp, Gert P, van Tuijl, Julia, Bienfait, H Paul, Booij, Suzanne J, van den Wijngaard, Ido R, Remmers, Michel J M, Schreuder, Antonia H C M L, Dippel, Diederik W, Staals, Julie, Brouwers, Paul J A M, Wermer, Marieke J H, Coutinho, Jonathan M, Kwa, Vincent I H, van Gelder, Isabelle C, Schutgens, Roger E G, Zweedijk, Berber, Algra, Ale, van Dalen, Jan Willem, Jaap Kappelle, L, Rinkel, Gabriel J E, van der Worp, H Bart, and Klijn, Catharina J M

Published
2021
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8. Perioperative pharmacokinetic-guided factor VIII concentrate dosing in haemophilia (OPTI-CLOT trial): an open-label, multicentre, randomised, controlled trial

Author

Cnossen, Marjon H., Leebeek, Frank W.G., Mathôt, Ron A.A., Fijnvandraat, Karin, Coppens, Michiel, Meijer, Karina, Kruip, Marieke J.H.A., Polinder, Suzanne, Lock, Janske, Hazendonk, Hendrika C.A.M., Van Moort, Iris, Heijdra, Jessica M., Goedhart, Marie C.H.J., Al Arashi, Wala, Preijers, Tim, De Jager, Nico C.B., Bukkems, Laura H., Cloesmeijer, Michael E., Janssen, Alexander, Tamminga, Rienk Y.J., Brons, Paul, Schols, Saskia E.M., Eikenboom, Jeroen C.J., Van der Meer, Felix J.M., Schutgens, Roger E.G., Fischer, Kathelijne, Van Galen, Karin P.M., Beckers, Erik E.A.M., Heubel-Moenen, Floor C.J.I., Nieuwenhuizen, Laurens, Ypma, Paula, Driessens, Mariëtte H.E., Van Vliet, Ineke, Collins, Peter W., Liesner, Ri, Chowdary, Pratima, Millar, Carolyn M., Hart, Dan, Keeling, David, van Moort, Iris, Bukkems, Laura H, Hazendonk, Hendrika C A M, van der Bom, Johanna G, Laros-van Gorkom, Britta A P, Beckers, Erik A M, van der Meer, Felix J M, Schutgens, Roger E G, Leebeek, Frank W G, Mathôt, Ron A A, and Cnossen, Marjon H

Published
2021
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10. Limited value of testing for factor XIII and α2‐antiplasmin deficiency in patients with a bleeding disorder of unknown cause.

Author

Ariëns, Sander, Huisman, Albert, Hovinga, Idske C. L. Kremer, Urbanus, Rolf T., van Galen, Karin P. M., van Vulpen, Lize F. D., Fischer, Kathelijn, and Schutgens, Roger E. G.

Subjects
BLOOD testing, VON Willebrand disease, BLOOD coagulation factors, HEMORRHAGE, ABSOLUTE value
Abstract

Introduction: In patients with an increased bleeding tendency, extensive diagnostic blood testing is often performed. When results of tier 1 assays of primary haemostasis are normal, protocols recommend additional testing to rule out rare disorders including coagulation factor XIII (FXIII) and α2‐antiplasmin (α2AP) deficiency. Aim: To evaluate the added diagnostic value of FXIII and α2AP levels in patients with a bleeding disorder of unknown cause (BDUC). Methods: A retrospective monocentre cohort study between August 2011 and August 2023 was conducted. In all patients with bleeding tendencies and normal diagnostic tests for von Willebrand disease and platelet function, FXIII and α2AP were measured. Results: We included 158 consecutive patients; mean ISTH‐BAT scores were 8.2 (SD ± 3.7) in children, 6.2 (SD ± 2.1) in men and 10.6 (SD ± 3.3) in women. Median age was 37 (range 5–79) years, 88.6% of patients were female. Patients displayed median FXIII activity of 111% (IQR = 97–131) and median α2AP activity of 112% (IQR = 103–119). Three (1.9%) patients had FXIII levels < 50%, respectively 43%, 45% and 46%. Corresponding ISTH‐BAT scores were 7, 12 and 14. No α2AP levels < 60% was observed. No significant association was found between FXIII levels and ISTH‐BAT scores. Conclusion: In our cohort of BDUC patients, no clinical relevant FXIII deficiencies were detected; absolute values were well above the 30% cutoff considered adequate for normal haemostasis. No α2AP deficiencies were detected. These data suggest that in BDUC patients, measuring FXIII or AP activity is of limited value. [ABSTRACT FROM AUTHOR]

Published
2024
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11. Metabolic blood profile and response to treatment with the pyruvate kinase activator mitapivat in patients with sickle cell disease.

Author

van Dijk, Myrthe J., Ruiter, Titine J. J., van der Veen, Sigrid, Rab, Minke A. E., van Oirschot, Brigitte A., Bos, Jennifer, Derichs, Cleo, Rijneveld, Anita W., Cnossen, Marjon H., Nur, Erfan, Biemond, Bart J., Bartels, Marije, Schutgens, Roger E. G., van Solinge, Wouter W., Jans, Judith J. M., van Beers, Eduard J., and van Wijk, Richard

Published
2024
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12. Subclinical synovial proliferation in patients with severe haemophilia A: The value of ultrasound screening and biochemical markers

Author

van Bergen, Eline D. P., primary, van Leeuwen, Flora H. P., additional, Foppen, Wouter, additional, Timmer, Merel A., additional, Schutgens, Roger E. G., additional, Mastbergen, Simon C., additional, Lafeber, Floris P. J. G., additional, de Jong, Pim A., additional, Fischer, Kathelijn, additional, and van Vulpen, Lize F. D., additional

Published
2023
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14. Towards Personalized Treatment in Haemophilia: The Role of Genetic Factors in Iron and Heme Control to Identify Patients at Risk for Haemophilic Arthropathy.

Author

van Vulpen, Lize F. D., Mastbergen, Simon C., Foppen, Wouter, Fischer, Kathelijn, Lafeber, Floris P. J. G., and Schutgens, Roger E. G.

Subjects
BLOOD coagulation factor VIII, HEMOPHILIA treatment, JOINT diseases, HEME, HEMOPHILIACS, IRON
Abstract

The treatment landscape for haemophilia is changing rapidly, creating opportunities for personalized treatment. As major morbidity is still caused by haemophilic arthropathy, understanding the factors affecting joint damage and joint damage progression might lead to more individualized treatment regimens. We investigated the association of HFE mutations or HMOX1 polymorphisms affecting iron/heme handling with radiographic joint damage in 252 haemophilia patients (severe and moderate). Although iron levels and transferrin saturation were significantly increased in the 95 patients with an HFE mutation, neither carrying this mutation nor the HMOX1 polymorphism was associated with radiographic joint damage, and the same was true after adjustment for well-known factors associated with arthropathy. In conclusion, this study does not support the hypothesis that HFE mutations or HMOX1 polymorphisms can be used to predict the development of haemophilic arthropathy. [ABSTRACT FROM AUTHOR]

Published
2024
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15. Clotting factor activity levels and bleeding risk in people with haemophilia playing sports

Author

Versloot, Olav, primary, Kemler, Ellen, additional, Blokzijl, Johan, additional, Timmer, Merel, additional, Schuuring, Marleen, additional, van Galen, Karin P. M., additional, Kremer Hovinga, Idske Cornelia L., additional, van der Valk, Paul R., additional, van Vulpen, Lize F. D., additional, Schutgens, Roger E. G., additional, van Koppenhagen, Casper F., additional, van der Net, Janjaap, additional, and Fischer, Kathelijn, additional

Published
2023
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16. Structural changes after ankle joint distraction in haemophilic arthropathy: an explorative study investigating biochemical markers and 3D joint space width

Author

van Bergen, Eline D. P., primary, Mastbergen, Simon C., additional, Lafeber, Floris P. J. G., additional, Bay‐Jensen, Anne‐Christine, additional, Madsen, Sofie F., additional, Port, Helena, additional, Foppen, Wouter, additional, Schutgens, Roger E. G., additional, Jansen, Mylène P., additional, and van Vulpen, Lize F. D., additional

Published
2023
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18. The efficacy of the entire-vial dosing of emicizumab: Real-world evidence on plasma concentrations, bleeds, and drug waste

Author

Afd Pharmacoepi & Clinical Pharmacology, PECP – Centre for Clinical Therapeutics, Pharmacoepidemiology and Clinical Pharmacology, Donners, Anouk A M T, van der Zwet, Konrad, Rademaker, Carin M A, Egberts, Toine C G, Schutgens, Roger E G, Fischer, Kathelijn, Afd Pharmacoepi & Clinical Pharmacology, PECP – Centre for Clinical Therapeutics, Pharmacoepidemiology and Clinical Pharmacology, Donners, Anouk A M T, van der Zwet, Konrad, Rademaker, Carin M A, Egberts, Toine C G, Schutgens, Roger E G, and Fischer, Kathelijn

Published
2023

19. One-year safety and efficacy of mitapivat in sickle cell disease: follow-up results of a phase 2, open-label study

Author

CDL Rode cel, Unit Opleiding Aios, Circulatory Health, CDL Research analisten, Poli Van Creveldkliniek Medisch, Haematologie patientenzorg, Child Health, Genetica Sectie Metabole Diagnostiek, Brain, Centraal Diagnostisch Laboratorium, Infection & Immunity, CDL Cluster Speciële Diagnostiek, van Dijk, Myrthe J, Rab, Minke A E, van Oirschot, Brigitte A, Bos, Jennifer, Derichs, Cleo, Rijneveld, Anita W, Cnossen, Marjon H, Nur, Erfan, Biemond, Bart J, Bartels, Marije, Jans, Judith J M, van Solinge, W W, Schutgens, Roger E G, van Wijk, Richard, van Beers, Eduard J, CDL Rode cel, Unit Opleiding Aios, Circulatory Health, CDL Research analisten, Poli Van Creveldkliniek Medisch, Haematologie patientenzorg, Child Health, Genetica Sectie Metabole Diagnostiek, Brain, Centraal Diagnostisch Laboratorium, Infection & Immunity, CDL Cluster Speciële Diagnostiek, van Dijk, Myrthe J, Rab, Minke A E, van Oirschot, Brigitte A, Bos, Jennifer, Derichs, Cleo, Rijneveld, Anita W, Cnossen, Marjon H, Nur, Erfan, Biemond, Bart J, Bartels, Marije, Jans, Judith J M, van Solinge, W W, Schutgens, Roger E G, van Wijk, Richard, and van Beers, Eduard J

Published
2023

20. Toward responsible clinical n-of-1 strategies for rare diseases

Author

Medical Humanities Onderzoek Team 1, Circulatory Health, JC onderzoeksprogramma Methodologie, Poli Van Creveldkliniek Medisch, Child Health, Infection & Immunity, Neurologen, Brain, Genetica Klinische Genetica, Defelippe, Victoria M, J M W van Thiel, Ghislaine, Otte, Willem M, Schutgens, Roger E G, Stunnenberg, Bas, Cross, Helen J, O'Callaghan, Finbar, De Giorgis, Valentina, Jansen, Floor E, Perucca, Emilio, Brilstra, Eva H, Braun, Kees P J, Medical Humanities Onderzoek Team 1, Circulatory Health, JC onderzoeksprogramma Methodologie, Poli Van Creveldkliniek Medisch, Child Health, Infection & Immunity, Neurologen, Brain, Genetica Klinische Genetica, Defelippe, Victoria M, J M W van Thiel, Ghislaine, Otte, Willem M, Schutgens, Roger E G, Stunnenberg, Bas, Cross, Helen J, O'Callaghan, Finbar, De Giorgis, Valentina, Jansen, Floor E, Perucca, Emilio, Brilstra, Eva H, and Braun, Kees P J

Published
2023

21. No immunological changes after factor VIII product switch: An in depth analysis in haemophilia A patients

Author

CTI Boes, Poli Van Creveldkliniek Medisch, Child Health, Circulatory Health, Zorg en O&O, Infection & Immunity, Schep, Sarah J, Fischer, Kathelijn, Boes, Marianne, Schutgens, Roger E G, CTI Boes, Poli Van Creveldkliniek Medisch, Child Health, Circulatory Health, Zorg en O&O, Infection & Immunity, Schep, Sarah J, Fischer, Kathelijn, Boes, Marianne, and Schutgens, Roger E G

Published
2023

22. Structural changes after ankle joint distraction in haemophilic arthropathy: an explorative study investigating biochemical markers and 3D joint space width

Author

Poli Van Creveldkliniek Medisch, MS Reumatologie/Immunologie/Infectie, Infection & Immunity, Regenerative Medicine and Stem Cells, Lab Reumatologie/Klinische Immunologie, MS Radiologie, Child Health, Circulatory Health, Onderzoek Reumatologie, van Bergen, Eline D P, Mastbergen, Simon C, Lafeber, Floris P J G, Bay-Jensen, Anne-Christine, Madsen, Sofie F, Port, Helena, Foppen, Wouter, Schutgens, Roger E G, Jansen, Mylène P, van Vulpen, Lize F D, Poli Van Creveldkliniek Medisch, MS Reumatologie/Immunologie/Infectie, Infection & Immunity, Regenerative Medicine and Stem Cells, Lab Reumatologie/Klinische Immunologie, MS Radiologie, Child Health, Circulatory Health, Onderzoek Reumatologie, van Bergen, Eline D P, Mastbergen, Simon C, Lafeber, Floris P J G, Bay-Jensen, Anne-Christine, Madsen, Sofie F, Port, Helena, Foppen, Wouter, Schutgens, Roger E G, Jansen, Mylène P, and van Vulpen, Lize F D

Published
2023

23. Functional decline in persons with haemophilia and factors associated with deterioration

Author

Poli Van Creveldkliniek Medisch, Fysiotherapiewetenschap, Brain, Child Health, Circulatory Health, Infection & Immunity, Blokzijl, Johan, Pisters, Martijn F, Veenhof, Cindy, Schutgens, Roger E G, Timmer, Merel A, Poli Van Creveldkliniek Medisch, Fysiotherapiewetenschap, Brain, Child Health, Circulatory Health, Infection & Immunity, Blokzijl, Johan, Pisters, Martijn F, Veenhof, Cindy, Schutgens, Roger E G, and Timmer, Merel A

Published
2023

24. Blood coagulation and beyond: Position paper from the Fourth Maastricht Consensus Conference on Thrombosis

Author

CDL Cluster Onderzoek en Onderwijs, Circulatory Health, Poli Van Creveldkliniek Medisch, Child Health, Infection & Immunity, Akbalut, Cengiz, Arisz, Ryanne, Baaten, Constance, Baildildinova, Gaukhar, Barakzie, Aarazo, Bauersachs, Rupert, Ten Berg, Jurriën M, van den Broek, Wout, de Boer, Hetty C, Broker, Vanessa, Buka, Richard, Ten Cate, Hugo, Cate, Arina Ten, De Luca, Ciro, De Simone, Ilaria, Dignat-George, Françoise, Freson, Kathleen, Gazzaniga, Giulia, van Gorp, Eric, Habibi, Anxhela, Henskens, Yvonne M C, Iding, Aaron F J, Khan, Abdullah, Koenderink, Gijsje, Konkoth, Akhil, Lacroix, Romaric, Lahiri, Trisha, Lam, Wilbur, Lamerton, Rachel, Lorusso, Roberto, Luo, Qi, Maas, Coen, McCarty, Owen J T, van der Meijden, Paola, Meijers, Joost, Mohapatra, Adarsh, Nevo, Neta, Pallares Robles, Alejandro, Poncelet, Philippe, Reinhardt, Christoph, Ruf, Wolfram, Saraswat, Ronald, Schonichen, Claudia, Schutgens, Roger E G, Simioni, Paolo, Spada, Stefano, Spronk, Henri M H, Tazhibayeva, Karlygash, Thachil, Jecko, Vacik-Diaz, Rocio, Veninga, Alicia, Verhamme, Peter, Visser, Chantal, Watson, Steve P, Wenzel, Philip, Willems, Ruth, Willers, Anne, Zhang, Pengyu, Zifkos, Konstantinos, van Zonneveld, Anton-Jan, CDL Cluster Onderzoek en Onderwijs, Circulatory Health, Poli Van Creveldkliniek Medisch, Child Health, Infection & Immunity, Akbalut, Cengiz, Arisz, Ryanne, Baaten, Constance, Baildildinova, Gaukhar, Barakzie, Aarazo, Bauersachs, Rupert, Ten Berg, Jurriën M, van den Broek, Wout, de Boer, Hetty C, Broker, Vanessa, Buka, Richard, Ten Cate, Hugo, Cate, Arina Ten, De Luca, Ciro, De Simone, Ilaria, Dignat-George, Françoise, Freson, Kathleen, Gazzaniga, Giulia, van Gorp, Eric, Habibi, Anxhela, Henskens, Yvonne M C, Iding, Aaron F J, Khan, Abdullah, Koenderink, Gijsje, Konkoth, Akhil, Lacroix, Romaric, Lahiri, Trisha, Lam, Wilbur, Lamerton, Rachel, Lorusso, Roberto, Luo, Qi, Maas, Coen, McCarty, Owen J T, van der Meijden, Paola, Meijers, Joost, Mohapatra, Adarsh, Nevo, Neta, Pallares Robles, Alejandro, Poncelet, Philippe, Reinhardt, Christoph, Ruf, Wolfram, Saraswat, Ronald, Schonichen, Claudia, Schutgens, Roger E G, Simioni, Paolo, Spada, Stefano, Spronk, Henri M H, Tazhibayeva, Karlygash, Thachil, Jecko, Vacik-Diaz, Rocio, Veninga, Alicia, Verhamme, Peter, Visser, Chantal, Watson, Steve P, Wenzel, Philip, Willems, Ruth, Willers, Anne, Zhang, Pengyu, Zifkos, Konstantinos, and van Zonneveld, Anton-Jan

Published
2023

25. Magnetic resonance imaging evidence for subclinical joint bleeding in a Dutch population of people with severe hemophilia on prophylaxis

Author

Cancer, Researchgr. Systems Radiology, Poli Van Creveldkliniek Medisch, Child Health, Circulatory Health, Infection & Immunity, Regenerative Medicine and Stem Cells, MS Radiologie, van Leeuwen, Flora H P, van Bergen, Eline D P, Timmer, Merel A, van Vulpen, Lize F D, Schutgens, Roger E G, de Jong, Pim A, Fischer, Kathelijn, Foppen, Wouter, Cancer, Researchgr. Systems Radiology, Poli Van Creveldkliniek Medisch, Child Health, Circulatory Health, Infection & Immunity, Regenerative Medicine and Stem Cells, MS Radiologie, van Leeuwen, Flora H P, van Bergen, Eline D P, Timmer, Merel A, van Vulpen, Lize F D, Schutgens, Roger E G, de Jong, Pim A, Fischer, Kathelijn, and Foppen, Wouter

Published
2023

26. MarioHeart: Novel In-Vitro Flow Model for Testing Heart Valve Prostheses and Anticoagulant Therapies

Author

Onderzoek CTC, CTC, Circulatory Health, Regenerative Medicine and Stem Cells, Poli Van Creveldkliniek Medisch, Child Health, Infection & Immunity, Devos, Maxime, Liesdek, Omayra C D, Suyker, Willem J L, van Tuijl, Sjoerd, Schutgens, Roger E G, van de Vosse, Frans N, de Heer, Linda M, Rutten, Marcel C M, Onderzoek CTC, CTC, Circulatory Health, Regenerative Medicine and Stem Cells, Poli Van Creveldkliniek Medisch, Child Health, Infection & Immunity, Devos, Maxime, Liesdek, Omayra C D, Suyker, Willem J L, van Tuijl, Sjoerd, Schutgens, Roger E G, van de Vosse, Frans N, de Heer, Linda M, and Rutten, Marcel C M

Published
2023

27. Proof of concept and feasibility of a blended physiotherapy intervention for persons with haemophilic arthropathy

Author

Child Health, Poli Van Creveldkliniek Medisch, Circulatory Health, Fysiotherapiewetenschap, Infection & Immunity, Brain, Timmer, Merel A, Kuijlaars, Isolde A R, Kloek, Corelien, de Kleijn, Piet, Schutgens, Roger E G, Veenhof, Cindy, Pisters, Martijn F, Child Health, Poli Van Creveldkliniek Medisch, Circulatory Health, Fysiotherapiewetenschap, Infection & Immunity, Brain, Timmer, Merel A, Kuijlaars, Isolde A R, Kloek, Corelien, de Kleijn, Piet, Schutgens, Roger E G, Veenhof, Cindy, and Pisters, Martijn F

Published
2023

33. Lupus anticoagulant associates with thrombosis in patients with COVID-19 admitted to intensive care units

Author

Noordermeer, Tessa, Schutgens, Roger E. G., Visser, Chantal, Rademaker, Emma, de Maat, Moniek P. M., Jansen, A. J. Gerard, Limper, Maarten, Cremer, Olaf L., Kruip, Marieke J. H. A., Endeman, Henrik, Maas, Coen, de Laat, Bas, Urbanus, Rolf T., van de Beek, D., Brouwer, M. C., de Bruin, S., Coppens, M., van Es, N., van Haaps, T. F., Juffermans, N. P., Muller, M. C. A., Vlaar, A. P. J., Hertogh, C. M. P. M., Heunks, L. M. A., Hugtenburg, J. G., van Kooten, J., Nossent, E. J., Smulders, Y., Tuinman, P. R., Noordegraaf, A. Vonk, Grootenboers, M. J. J. H., van Guldener, C., Kant, M., Lansbergen, A., Faber, J., Hajer, G., Stemerdink, A., van den Akker, J., Bierings, R., Endeman, H., Goeijenbier, M., Hunfeld, N. G. M., van Gorp, E. C. M., Gommers, D. A. M. P. J., Koopmans, M. P. G., Kruip, M. J. H. A., Kuiken, T., Langerak, T., Leebeek, Lauw, M. N., de Maat, M. P. M., Noack, D., Paats, M. S., Raadsen, M. P., Rockx, B., Rokx, C., Schurink, C. A. M., Tong-Minh, K., van den Toorn, L., den Uil, C. A., Visser, C., Boutkourt, F., Roest, T., Douma, R. A., de Haan, L. R., ten Wolde, M., Bemelmans, R. H. H., Festen, B., Stads, S., de Jager, C. P. C., Simons, K. S., Antoni, M. L., Bos, M. H., Burggraaf, J. L. I., Cannegieter, S. C., Eikenboom, H. C. J., den Exter, P. L., Geelhoed, J. J. M., Huisman, M. V., de Jonge, E., Kaptein, F. H. J., Klok, F. A., Kroft, L. J. M., Lijfering, W. M., Nab, L., Ninaber, M. K., Putter, H., Ramai, S. R. S., da Rocha Rondon, A. M., Roukens, A. H. E., Stals, M. A. M., Versteeg, H. H., Vliegen, H. W., van Vlijmen, B. J. M., van de Berg, T., Bruggemann, R., van Bussel, B. C. T., ten Cate, H., ten Cate-Hoek, A., Hackeng, T. M., Henskens, ir. Y., Hulshof, A., Mulder, M., Olie, R. H., Schurgers, L., Spaetgens, B., Spronk, H., Spruit, M. A., Winckers, K., Nieuwenhuizen, L., Franken, B., Schrover, I. M., de Waal, E. G. M., Beishuizen, A., Cornet, A., Krabbe, J., Kramers, K., Leentjens, J., de Mast, Q., Middeldorp, S., Brouwer, R. E., Ellerbroek, J. L. J., Tijmensen, J., Hovens, M. M. C., Oostdijk, E. A. N., Westerhof, B. D., Faber, L. M., van den Biggelaar, M., Meijers, J. C. M., Voorberg, J., Kevenaar, M. E., Soei, Y. L., Wils, E. J., Croles, F. N., de Laat, B., Kamphuisen, P. W., Vink, R., Lisman, T., Meijer, K., van Tichelaar, Y. I. G., Cremer, O. L., Geersing, G., Kaasjager, H. A. H., Kusadasi, N., Huisman, A., Maas, C., Nijkeuter, M., Schutgens, R. E. G., Creveldkliniek, Van, Urbanus, R. T., Westerink, J., Faber, H. J., Koster, S. C. E., van Montfort, P., van Twist, D. J. L., RS: Carim - B01 Blood proteins & engineering, Biochemie, Hematology, Intensive Care, Neurology, ANS - Neuroinfection & -inflammation, Intensive Care Medicine, ACS - Pulmonary hypertension & thrombosis, AII - Inflammatory diseases, Vascular Medicine, ACS - Amsterdam Cardiovascular Sciences, Graduate School, ACS - Microcirculation, Medical Microbiology and Infection Prevention, ARD - Amsterdam Reproduction and Development, Experimental Vascular Medicine, Landsteiner Laboratory, ACS - Atherosclerosis & ischemic syndromes, Elderly care medicine, APH - Aging & Later Life, Clinical pharmacology and pharmacy, APH - Health Behaviors & Chronic Diseases, Pulmonary medicine, Internal medicine, ACS - Diabetes & metabolism, Intensive care medicine, General practice, and Amsterdam Gastroenterology Endocrinology Metabolism

Subjects
lnfectious Diseases and Global Health Radboud Institute for Health Sciences [Radboudumc 4], lupus anticoagulant, risk factor, critically ill, Vascular damage Radboud Institute for Health Sciences [Radboudumc 16], lnfectious Diseases and Global Health Radboud Institute for Molecular Life Sciences [Radboudumc 4], COVID-19, Hematology, thrombosis, Healthcare improvement science Radboud Institute for Health Sciences [Radboudumc 18]
Abstract

Contains fulltext : 286889.pdf (Publisher’s version ) (Open Access) BACKGROUND: Thrombosis is a frequent and severe complication in patients with coronavirus disease 2019 (COVID-19) admitted to the intensive care unit (ICU). Lupus anticoagulant (LA) is a strong acquired risk factor for thrombosis in various diseases and is frequently observed in patients with COVID-19. Whether LA is associated with thrombosis in patients with severe COVID-19 is currently unclear. OBJECTIVE: To investigate if LA is associated with thrombosis in critically ill patients with COVID-19. PATIENTS/METHODS: The presence of LA and other antiphospholipid antibodies was assessed in patients with COVID-19 admitted to the ICU. LA was determined with dilute Russell's viper venom time (dRVVT) and LA-sensitive activated partial thromboplastin time (aPTT) reagents. RESULTS: Of 169 patients with COVID-19, 116 (69%) tested positive for at least one antiphospholipid antibody upon admission to the ICU. Forty (24%) patients tested positive for LA; of whom 29 (17%) tested positive with a dRVVT, 19 (11%) tested positive with an LA-sensitive aPTT, and 8 (5%) tested positive on both tests. Fifty-eight (34%) patients developed thrombosis after ICU admission. The odds ratio (OR) for thrombosis in patients with LA based on a dRVVT was 2.5 (95% confidence interval [CI], 1.1-5.7), which increased to 4.5 (95% CI, 1.4-14.3) in patients at or below the median age in this study (64 years). LA positivity based on a dRVVT or LA-sensitive aPTT was only associated with thrombosis in patients aged less than 65 years (OR, 3.8; 95% CI, 1.3-11.4) and disappeared after adjustment for C-reactive protein. CONCLUSION: Lupus anticoagulant on admission is strongly associated with thrombosis in critically ill patients with COVID-19, especially in patients aged less than 65 years.

Published
2022

34. Ankle joint distraction is a promising alternative treatment for patients with severe haemophilic ankle arthropathy

Author

van Bergen, Eline D. P., primary, Mastbergen, Simon C., additional, Vogely, H. Charles, additional, Balani, Tanya N., additional, de Kleijn, Piet, additional, Foppen, Wouter, additional, van Roermund, Peter M., additional, Lafeber, Floris P. J. G., additional, Schutgens, Roger E. G., additional, and van Vulpen, Lize F. D., additional

Published
2022
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35. Enhanced hepatic clearance of hyposialylated platelets explains thrombocytopenia in GNE-related macrothrombocytopenia

Author

Noordermeer, Tessa, primary, van Asten, Ivar, additional, Schutgens, Roger E. G., additional, Lakerveld, Anke J., additional, Koekman, Cornelis A., additional, Hage, Kay Y., additional, Sebastian, Silvie A. E., additional, Huisman, Albert, additional, van den Heuvel, Dave J., additional, Gerritsen, Hans C., additional, Korporaal, Suzanne J. A., additional, Bierings, Marc, additional, van der Smagt, Jasper J., additional, van Gijn, Mariëlle E., additional, and Urbanus, Rolf T., additional

Published
2022
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37. Design of a Prospective Study on Pharmacokinetic-Guided Dosing of Prophylactic Factor Replacement in Hemophilia A and B (OPTI-CLOT TARGET Study)

Author

Goedhart, Tine M H J, Bukkems, Laura H, Coppens, Michiel, Fijnvandraat, Karin J, Schols, Saskia E M, Schutgens, Roger E G, Eikenboom, Jeroen, Heubel-Moenen, Floor C J I, Ypma, Paula F, Nieuwenhuizen, L, Meijer, K, Leebeek, Frank W G, Mathôt, Ron A A, Cnossen, Marjon H, Goedhart, Tine M H J, Bukkems, Laura H, Coppens, Michiel, Fijnvandraat, Karin J, Schols, Saskia E M, Schutgens, Roger E G, Eikenboom, Jeroen, Heubel-Moenen, Floor C J I, Ypma, Paula F, Nieuwenhuizen, L, Meijer, K, Leebeek, Frank W G, Mathôt, Ron A A, and Cnossen, Marjon H

Abstract

In resource-rich countries, almost all severe hemophilia patients receive prophylactic replacement therapy with factor concentrates to prevent spontaneous bleeding in joints and muscles to decrease the development of arthropathy and risk of long-term disability. Pharmacokinetic (PK)-guided dosing can be applied to individualize factor replacement therapy, as interindividual differences in PK parameters influence factor VIII (FVIII) and FIX activity levels. PK-guided dosing may therefore lead to more optimal safeguarding of FVIII/FIX levels during prophylaxis and on demand treatment. The OPTI-CLOT TARGET study is a multicenter, nonrandomized, prospective cohort study that aims to investigate the reliability and feasibility of PK-guided prophylactic dosing of factor concentrates in hemophilia-A and -B patients in daily clinical practice. At least 50 patients of all ages on prophylactic treatment using standard half-life (SHL) and extended half-life (EHL) factor concentrates will be included during 9 months and will receive PK-guided treatment. As primary endpoint, a minimum of four FVIII/FIX levels will be compared with FVIII/FIX levels as predicted by Bayesian forecasting. Secondary endpoints are the association of FVIII and FIX levels with bleeding episodes and physical activity, expectations and experiences, economic analyses, and optimization of population PK models. This study will lead to more insight in the reliability and feasibility of PK-guided dosing in hemophilia patients. Moreover, it will contribute to personalization of treatment by greater knowledge of dosing regimens needed to prevent and treat bleeding in the individual patient and provide evidence to more clearly associate factor activity levels with bleeding risk.

Published
2022

38. Lupus anticoagulant associates with thrombosis in patients with COVID-19 admitted to intensive care units:A retrospective cohort study

Author

Noordermeer, Tessa, Schutgens, Roger E. G., Visser, C., de Maat, M. P.M., Jansen, Gerard, Limper, Maarten, Kruip, M. J.H.A., Endeman, H., Maas, C., de Laat, Bas, Urbanus, R. T., Bierings, Ruben, Noordermeer, Tessa, Schutgens, Roger E. G., Visser, C., de Maat, M. P.M., Jansen, Gerard, Limper, Maarten, Kruip, M. J.H.A., Endeman, H., Maas, C., de Laat, Bas, Urbanus, R. T., and Bierings, Ruben

Abstract

Background: Thrombosis is a frequent and severe complication in patients with coronavirus disease 2019 (COVID-19) admitted to the intensive care unit (ICU). Lupus anticoagulant (LA) is a strong acquired risk factor for thrombosis in various diseases and is frequently observed in patients with COVID-19. Whether LA is associated with thrombosis in patients with severe COVID-19 is currently unclear. Objective: To investigate if LA is associated with thrombosis in critically ill patients with COVID-19. Patients/Methods: The presence of LA and other antiphospholipid antibodies was assessed in patients with COVID-19 admitted to the ICU. LA was determined with dilute Russell's viper venom time (dRVVT) and LA-sensitive activated partial thromboplastin time (aPTT) reagents. Results: Of 169 patients with COVID-19, 116 (69%) tested positive for at least one antiphospholipid antibody upon admission to the ICU. Forty (24%) patients tested positive for LA; of whom 29 (17%) tested positive with a dRVVT, 19 (11%) tested positive with an LA-sensitive aPTT, and 8 (5%) tested positive on both tests. Fifty-eight (34%) patients developed thrombosis after ICU admission. The odds ratio (OR) for thrombosis in patients with LA based on a dRVVT was 2.5 (95% confidence interval [CI], 1.1–5.7), which increased to 4.5 (95% CI, 1.4–14.3) in patients at or below the median age in this study (64 years). LA positivity based on a dRVVT or LA-sensitive aPTT was only associated with thrombosis in patients aged less than 65 years (OR, 3.8; 95% CI, 1.3–11.4) and disappeared after adjustment for C-reactive protein. Conclusion: Lupus anticoagulant on admission is strongly associated with thrombosis in critically ill patients with COVID-19, especially in patients aged less than 65 years.

Published
2022

39. Design of a Prospective Study on Pharmacokinetic-Guided Dosing of Prophylactic Factor Replacement in Hemophilia A and B (OPTI-CLOT TARGET Study)

Author

Poli Van Creveldkliniek Medisch, Child Health, Circulatory Health, Infection & Immunity, Goedhart, Tine M H J, Bukkems, Laura H, Coppens, Michiel, Fijnvandraat, Karin J, Schols, Saskia E M, Schutgens, Roger E G, Eikenboom, Jeroen, Heubel-Moenen, Floor C J I, Ypma, Paula F, Nieuwenhuizen, L, Meijer, K, Leebeek, Frank W G, Mathôt, Ron A A, Cnossen, Marjon H, Poli Van Creveldkliniek Medisch, Child Health, Circulatory Health, Infection & Immunity, Goedhart, Tine M H J, Bukkems, Laura H, Coppens, Michiel, Fijnvandraat, Karin J, Schols, Saskia E M, Schutgens, Roger E G, Eikenboom, Jeroen, Heubel-Moenen, Floor C J I, Ypma, Paula F, Nieuwenhuizen, L, Meijer, K, Leebeek, Frank W G, Mathôt, Ron A A, and Cnossen, Marjon H

Published
2022

40. Gene Therapy for Hemophilia A: How Long Will It Last?

Author

Poli Van Creveldkliniek Medisch, Child Health, Circulatory Health, Infection & Immunity, Schutgens, Roger E G, Poli Van Creveldkliniek Medisch, Child Health, Circulatory Health, Infection & Immunity, and Schutgens, Roger E G

Published
2022

41. Menstrual problems in chronic immune thrombocytopenia: A monthly challenge - a cohort study and review

Author

Poli Van Creveldkliniek Medisch, Child Health, MS VPG/Gynaecologie, MS Verloskunde, Circulatory Health, Infection & Immunity, van Dijk, Wobke E M, Punt, Marieke C, van Galen, Karin P M, van Leeuwen, Jeanette, Lely, A Titia, Schutgens, Roger E G, Poli Van Creveldkliniek Medisch, Child Health, MS VPG/Gynaecologie, MS Verloskunde, Circulatory Health, Infection & Immunity, van Dijk, Wobke E M, Punt, Marieke C, van Galen, Karin P M, van Leeuwen, Jeanette, Lely, A Titia, and Schutgens, Roger E G

Published
2022

42. Safety and efficacy of mitapivat, an oral pyruvate kinase activator, in sickle cell disease: a phase 2, open-label study

Author

CDL Rode cel, Unit Opleiding Aios, Circulatory Health, Poli Van Creveldkliniek Medisch, Haematologie patientenzorg, Child Health, Genetica Sectie Metabole Diagnostiek, Centraal Diagnostisch Laboratorium, Infection & Immunity, CDL Cluster Speciële Diagnostiek, van Dijk, Myrthe J, Rab, Minke A E, van Oirschot, Brigitte A, Bos, Jennifer, Derichs, Cleo, Rijneveld, Anita W, Cnossen, Marjon H, Nur, Erfan, Biemond, Bart J, Bartels, Marije, Jans, Judith J M, van Solinge, Wouter W, Schutgens, Roger E G, van Wijk, Richard, van Beers, Eduard J, CDL Rode cel, Unit Opleiding Aios, Circulatory Health, Poli Van Creveldkliniek Medisch, Haematologie patientenzorg, Child Health, Genetica Sectie Metabole Diagnostiek, Centraal Diagnostisch Laboratorium, Infection & Immunity, CDL Cluster Speciële Diagnostiek, van Dijk, Myrthe J, Rab, Minke A E, van Oirschot, Brigitte A, Bos, Jennifer, Derichs, Cleo, Rijneveld, Anita W, Cnossen, Marjon H, Nur, Erfan, Biemond, Bart J, Bartels, Marije, Jans, Judith J M, van Solinge, Wouter W, Schutgens, Roger E G, van Wijk, Richard, and van Beers, Eduard J

Published
2022

43. Predicting Individual Changes in Terminal Half-Life After Switching to Extended Half-Life Concentrates in Patients With Severe Hemophilia

Author

Poli Van Creveldkliniek Medisch, Child Health, Circulatory Health, Infection & Immunity, Versloot, Olav, Iserman, Emma, Chelle, Pierre, Germini, Federico, Edginton, Andrea N, Schutgens, Roger E G, Iorio, Alfonso, Fischer, Kathelijn, Poli Van Creveldkliniek Medisch, Child Health, Circulatory Health, Infection & Immunity, Versloot, Olav, Iserman, Emma, Chelle, Pierre, Germini, Federico, Edginton, Andrea N, Schutgens, Roger E G, Iorio, Alfonso, and Fischer, Kathelijn

Published
2022

44. Reduced cardiovascular morbidity in patients with hemophilia: results of a 5-year multinational prospective study

Author

Poli Van Creveldkliniek Medisch, Child Health, Circulatory Health, Infection & Immunity, Van Der Valk, Paul, Makris, Michael, Fischer, Kathelijn, Tait, Robert C, Chowdary, Pratima, Collins, Peter W, Meijer, Karina, van Vulpen, Lize F D, Mauser-Bunschoten, Eveline, Schutgens, Roger E G, Poli Van Creveldkliniek Medisch, Child Health, Circulatory Health, Infection & Immunity, Van Der Valk, Paul, Makris, Michael, Fischer, Kathelijn, Tait, Robert C, Chowdary, Pratima, Collins, Peter W, Meijer, Karina, van Vulpen, Lize F D, Mauser-Bunschoten, Eveline, and Schutgens, Roger E G

Published
2022

45. Relapse of immune thrombocytopenia after COVID-19 vaccination

Author

Poli Van Creveldkliniek Medisch, Child Health, Infection & Immunity, Circulatory Health, van Dijk, Wobke Else Maria, Schutgens, Roger E G, Poli Van Creveldkliniek Medisch, Child Health, Infection & Immunity, Circulatory Health, van Dijk, Wobke Else Maria, and Schutgens, Roger E G

Published
2022

46. Prevalence, burden and treatment effects of vaginal bleeding in women with (suspected) congenital platelet disorders throughout life: a cross-sectional study

Author

Poli Van Creveldkliniek Medisch, Geboortecentrum voorzitterschap, Child Health, Poli VCK, Circulatory Health, Infection & Immunity, Punt, Marieke C, Ruigrok, Nienke D, Bloemenkamp, Kitty W M, Uitslager, Nanda, Urbanus, Rolf T, Groot, Evelyn, Kremer Hovinga, Idske C L, Schutgens, Roger E G, van Galen, Karin P M, T rombocytopathy in the Netherlands (TiN) Study Group, Poli Van Creveldkliniek Medisch, Geboortecentrum voorzitterschap, Child Health, Poli VCK, Circulatory Health, Infection & Immunity, Punt, Marieke C, Ruigrok, Nienke D, Bloemenkamp, Kitty W M, Uitslager, Nanda, Urbanus, Rolf T, Groot, Evelyn, Kremer Hovinga, Idske C L, Schutgens, Roger E G, van Galen, Karin P M, and T rombocytopathy in the Netherlands (TiN) Study Group

Published
2022

47. Emicizumab Dosing in Children and Adults with Hemophilia A: Simulating a User-Friendly and Cost-Efficient Regimen

Author

Poli Van Creveldkliniek Medisch, Child Health, Circulatory Health, Apotheek Opleiding, Apotheek Veiligheid en Kwaliteit, Infection & Immunity, Bukkems, Laura, Fischer, Kathelijn, Kremer Hovinga, Idske, Donners, Anouk Anna Marie Therese, Fijnvandraat, Karin, Schutgens, Roger E G, Cnossen, M, Mathôt, Ron A, Poli Van Creveldkliniek Medisch, Child Health, Circulatory Health, Apotheek Opleiding, Apotheek Veiligheid en Kwaliteit, Infection & Immunity, Bukkems, Laura, Fischer, Kathelijn, Kremer Hovinga, Idske, Donners, Anouk Anna Marie Therese, Fijnvandraat, Karin, Schutgens, Roger E G, Cnossen, M, and Mathôt, Ron A

Published
2022

49. Safety and efficacy of mitapivat, an oral pyruvate kinase activator, in sickle cell disease: A phase 2, open‐label study

Author

van Dijk, Myrthe J., primary, Rab, Minke A. E., additional, van Oirschot, Brigitte A., additional, Bos, Jennifer, additional, Derichs, Cleo, additional, Rijneveld, Anita W., additional, Cnossen, Marjon H., additional, Nur, Erfan, additional, Biemond, Bart J., additional, Bartels, Marije, additional, Jans, Judith J. M., additional, van Solinge, Wouter W., additional, Schutgens, Roger E. G., additional, van Wijk, Richard, additional, and van Beers, Eduard J., additional

Published
2022
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50. SYMPHONYconsortium: Orchestrating personalized treatment for patients with bleeding disorders

Author

Cnossen, Marjon H., Moort, Iris, Reitsma, Simone H., Maat, Moniek P. M., Schutgens, Roger E. G., Urbanus, Rolf T., Lingsma, Hester F., Mathot, Ron A. A., Gouw, Samantha C., Meijer, Karina, Bredenoord, Annelien L., Graaf, Rieke, Fijnvandraat, Karin, Meijer, Alexander B., Akker, Emile, Bierings, Ruben, Eikenboom, Jeroen C. J., Biggelaar, Maartje, Haas, Masja, Voorberg, Jan, Leebeek, Frank W. G., Cnossen, Marjon H., Reitsma, Simone H., Haas, Masja, Biggelaar, Maartje, Leebeek, Frank W. G., Voorberg, Jan, Maat, Moniek P. M., Schutgens, Roger E. G., Urbanus, Rolf T., Lingsma, Hester F., Mathot, Ron A. A., Gouw, Samantha C., Meijer, Karina, Bredenoord, Annelien L., Graaf, Rieke, Fijnvandraat, Karin, Meijer, Alexander B., Akker, Emile, Bierings, Ruben, Eikenboom, Jeroen C. J., Moort, Iris, Arisz, Ryanne A., Zivkovic, Minka, Hoorn, E. Shannon, Bukkems, Laura H., Goedhart, Tine M. C. H. J., Romano, Lorenzo G. R., Al Arashi, Wala, Cloesmeijer, Michael E., Janssen, Alexander, Brands, Martijn R., Baas, Lieke, Castillo Alferez, Jessica, Zhang, Huan, Laan, Sebastiaan N. J., Boender, Johan, Bom, Johanna G., Bos, Mettine H. A., Burdorf, Lex, Coppens, Michiel, Driessens, Mariette, Fischer, Kathelijne F., Haverman, Lotte, Hazelzet, Jan A., Huisman, Elise J., Jansen, Natalie, Jong, Sean, Kruip, Marieke, Leeuwen, Nikki, Meer, Felix, Meijer, Stephan, Amstel, Hans Kristian Ploos, Polinder, Suzanne, Schols, Saskia E. M., Wijfjes, Guus, Kluft, Kees, Heerde, Waander L., Goedhart, Geertje, Uyl, Carin, Timp, Jasmijn, Stekelenburg, Anke, Moenen, Floor, Ypma, Paula, Nieuwenhuizen, Laurens, and Plat, Arnoud

Abstract

Treatment choices for individual patients with an inborn bleeding disorder are increasingly challenging due to increasing options and rising costs for society. We have initiated an integrated interdisciplinary national research program. The SYMPHONY consortium strives to orchestrate personalized treatment in patients with an inborn bleeding disorder, by unraveling the mechanisms behind interindividual variations of bleeding phenotype. The SYMPHONY consortium will investigate patients with an inborn bleeding disorder, both diagnosed and not yet diagnosed. Research questions are categorized under the themes: (1) diagnosis, (2) treatment, and (3) fundamental research, and consist of work packages addressing specific domains. Importantly, collaborations between patients and talented researchers from different areas of expertise promise to augment the impact of the SYMPHONY consortium, leading to unique interactions and intellectual property. SYMPHONY will perform research on all aspects of care, treatment individualization in patients with inborn bleeding disorders, as well as diagnostic innovations and results of molecular genetics and cellular model technology with regard to the hemostatic process. We believe that these research investments will lead to health‐care innovations with long‐term clinical and societal impact. This consortium has been made possible by a governmental, competitive grant from the Netherlands Organization for Scientific Research (NWO) within the framework of the NWA‐ORC Call grant agreement NWA.1160.18.038.

Published
2022
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