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1. Inhibitor development according to concentrate after 50 exposure days in severe hemophilia: data from the European HAemophilia Safety Surveillance (EUHASS)

Author

Ay, Cihan, Male, Christoph, Hermans, Cedric, Verhamme, Peter, Lissitchkov, Toshko, Antoniades, Marios, Penka, Miroslav, Blatny, Jan, Komrska, Vladimir, Poulsen, Lone Hvitfeldt, Kampmann, Peter, Lehtinen, Anna-Elina, Susen, Sophie, Dargaud, Yesim, Biron, Christine, D'Oiron, Roseline, Harroche, Annie, Klamroth, Robert, Oldenburg, Johannes, Buehrlen, Martina, Miesbach, Wolfgang, Langer, Florian, Spannag, Patrick, Oliveri, Martin, Platokouki, Helen, Nomikou, Efrosyni, Katsarou, Olga, Garypidou, Vasileia, Economou, Marina, Nemes, Laszlo, Nolan, Beatrice, O'Connell, Niamh, Paolo, Radossi, Castaman, Giancarlo, Peyvandi, Flora, Rocino, Angiola, Zanon, Ezio, Tagliaferri, Annarita, Agnelli, Giancarlo, De Crisotofaro, Raimondo, Schinco, Piercarla, Tosetto, Alberto, Lejniece, Sandra, Gailiute, Neringa, Gatt, Alexander, Mäkelburg, Anja, Laros-van Gorkom, Britta, Brons, Paul, Leebeek, Frank W.G., Schutgens, Roger, Windyga, Jerzy, Catarino, Cristina, Aires, Anabela, Fraga, Cristina, Morais, Sara, Araújo, Fernando, Serban, Margit, Davydkin, Igor, Batorova, Angelika, Anzej Doma, Sasa, Martinez, Laura Segura, Palomo Bravo, Angeles, Ortega, Immaculada Soto, Bonanad, Santiago, Baghaei, Fariba, Astermark, Jan, Holmström, Margareta, Fontana, Pierre, Schmugge, Markus, Zulfikar, Bulent, Kavakli, Kaan, Khan, Mohammed, Benson, Gary, Lester, Will, Andrew, Page, Catherine, Bagot, Pinto, Fernando, Salta, Styliani, Farrelly, Cathy, Matthias, Mary, Laffan, Mike, Thynn Thynn, Yee, Mcdonald, Vickie, Austin, Steve, Bella, Madan, Hay, Charles, Grainger, John, Talks, Kate, Shapiro, Susie, Maclean, Rhona, Payne, Jeanette, Fischer, Kathelijn, Lassila, Riitta, Gouw, Samantha C., Hollingsworth, Rob, Lambert, Thierry, Kaczmarek, Radek, Carbonero, Diana, and Makris, Mike

Published
2024
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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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3. The bleeding phenotype in people with nonsevere hemophilia

Author

Kloosterman, Fabienne R., Zwagemaker, Anne-Fleur, Bagot, Catherine N., Beckers, Erik A.M., Castaman, Giancarlo, Cnossen, Marjon H., Collins, Peter W., Hay, Charles, Hof, Michel, Laros-van Gorkom, Britta, Leebeek, Frank W.G., Male, Christoph, Meijer, Karina, Pabinger, Ingrid, Shapiro, Susan, Coppens, Michiel, Fijnvandraat, Karin, and Gouw, Samantha C.

Published
2022
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4. Relationships of sensory processing sensitivity with creativity and empathy in an adult sample.

Author

Laros-van Gorkom, Britta A. P., Damatac, Christienne G., Stevelmans, Inez, and Greven, Corina U.

Subjects
OPENNESS to experience, SENSORIMOTOR integration, INDIVIDUAL differences, EMPATHY, AFFECT (Psychology), CREATIVE ability
Abstract

Introduction: Sensory Processing Sensitivity (SPS) describes individual differences in sensitivity to environments, but there is little research on potential positive correlates of SPS. Hereby we investigate whether SPS and its Aesthetic Sensitivity (AES) component are associated with different facets of creativity and empathy. Methods: Questionnaires on SPS, creativity and empathy were administered to 296 participants and data were analyzed using hierarchical multiple regression. Results: Higher SPS total and AES scores were associated with more creative ideas (SPS: β = 0.294, pfdr < 0.001; AES: β = 0.484, pfdr < 0.001). Only AES was associated with more creative activities (AES: β = 0.292, pfdr < 0.001). Furthermore, higher SPS total and AES scores were associated with more overall empathy (SPS: β = 0.428, pfdr < 0.001; AES: β = 0.373, pfdr < 0.001), affective empathy (SPS: β = 0.507, pfdr < 0.001; AES: β = 0.331, pfdr < 0.001), cognitive empathy (SPS: β = 0.2692, pfdr < 0.001; AES: β = 0.347, pfdr < 0.001), and less emotional disconnection (SPS: β = 0.234, pfdr β 0.001; AES: β = 0.210, pfdr β 0.001). Most associations remained significant after controlling for openness to experience, and the other SPS components of ease of excitation and low sensory threshold and gender, age, and education. Discussion: We conclude that SPS and AES are associated with creativity and empathy. Strengthening these positive aspects might help highly sensitive people flourish. [ABSTRACT FROM AUTHOR]

Published
2025
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7. Treatment‐related risk factors for inhibitor development in non‐severe hemophilia A after 50 cumulative exposure days: A case‐control study

Author

Abdi, Amal, Eckhardt, Corien L., van Velzen, Alice S., Vuong, Caroline, Coppens, Michiel, Castaman, Giancarlo, Hart, Dan P., Hermans, Cedric, Laros‐van Gorkom, Britta, Leebeek, Frank W.G., Mancuso, Maria Elisa, Mazzucconi, Maria G., McRae, Simon, Oldenburg, Johannes, Male, Christoph, van der Bom, Johanna G., Fijnvandraat, Karin, and Gouw, Samantha C.

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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11. ADAMTS‐13 and bleeding phenotype in von Willebrand disease

Author

Boender, Johan, Nederlof, Angelique, Meijer, Karina, Mauser‐Bunschoten, Evelien P., Cnossen, Marjon H., Fijnvandraat, Karin, van der Bom, Johanna G., de Meris, Joke, Laros‐van Gorkom, Britta A.P., van Galen, Karin P.M., Eikenboom, Jeroen, de Maat, Moniek P.M, Leebeek, Frank W.G., Coppens, M., Nieuwenhuizen, L., Tamminga, R.Y.J., Ypma, P.F., Smiers, F.J.W., Beckers, E., Brons, P., and Atiq, F.

Published
2020
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12. One piece of the puzzle: Population pharmacokinetics of FVIII during perioperative Haemate P®/Humate P® treatment in von Willebrand disease patients

Author

de Jager, Nico C.B., Bukkems, Laura H., Heijdra, Jessica M., Hazendonk, Carolien H.C.A.M., Fijnvandraat, Karin, Meijer, Karina, Eikenboom, Jeroen, Laros - van Gorkom, Britta A.P., Leebeek, Frank W.G., Cnossen, Marjon H., Mathôt, Ron A.A., Collins, P.W., Kruip, M.J.H.A., Polinder, S., Lock, J., van Moort, I., Goedhart, M.C.H.J., Coppens, M., Peters, M., Preijers, T., Brons, P., van der Meer, F.J.M., Schutgens, R.E.G., Fischer, K., Driessens, M.H.E., Zwaan, C.M., van Vliet, I., Liesner, R., Chowdary, P., and Keeling, D.

Published
2020
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13. Peri-operative desmopressin combined with pharmacokinetic-guided factor VIII concentrate in non-severe haemophilia A patients

Author

Romano, Lorenzo G. R., Schutte, Lisette M., van Hest, Reinier M., Meijer, Karina, Laros-van Gorkom, Britta A. P., Nieuwenhuizen, Laurens, Eikenboom, Jeroen, Heubel-Moenen, Floor C. J. I., Uitslager, Nanda, Coppens, Michiel, Fijnvandraat, Karin, Driessens, Mariette H. E., Polinder, Suzanne, Cnossen, Marjon H., Leebeek, Frank W. G., Mathot, Ron A. A., Kruip, Marieke J. H. A., Romano, Lorenzo G. R., Schutte, Lisette M., van Hest, Reinier M., Meijer, Karina, Laros-van Gorkom, Britta A. P., Nieuwenhuizen, Laurens, Eikenboom, Jeroen, Heubel-Moenen, Floor C. J. I., Uitslager, Nanda, Coppens, Michiel, Fijnvandraat, Karin, Driessens, Mariette H. E., Polinder, Suzanne, Cnossen, Marjon H., Leebeek, Frank W. G., Mathot, Ron A. A., and Kruip, Marieke J. H. A.

Published
2024

14. Peri-operative desmopressin combined with pharmacokinetic-guided factor VIII concentrate in non-severe haemophilia A patients

Author

Poli VCK, Romano, Lorenzo G.R., Schütte, Lisette M., van Hest, Reinier M., Meijer, Karina, Laros-van Gorkom, Britta A.P., Nieuwenhuizen, Laurens, Eikenboom, Jeroen, Heubel-Moenen, Floor C.J.I., Uitslager, Nanda, Coppens, Michiel, Fijnvandraat, Karin, Driessens, Mariëtte H.E., Polinder, Suzanne, Cnossen, Marjon H., Leebeek, Frank W.G., Mathôt, Ron A.A., Kruip, Marieke J.H.A., Poli VCK, Romano, Lorenzo G.R., Schütte, Lisette M., van Hest, Reinier M., Meijer, Karina, Laros-van Gorkom, Britta A.P., Nieuwenhuizen, Laurens, Eikenboom, Jeroen, Heubel-Moenen, Floor C.J.I., Uitslager, Nanda, Coppens, Michiel, Fijnvandraat, Karin, Driessens, Mariëtte H.E., Polinder, Suzanne, Cnossen, Marjon H., Leebeek, Frank W.G., Mathôt, Ron A.A., and Kruip, Marieke J.H.A.

Published
2024

15. Transition readiness among adolescents and young adults with haemophilia in the Netherlands: Nationwide questionnaire study

Author

Brands, Martijn R., primary, Janssen, Ebony A. M., additional, Cnossen, Marjon H., additional, Smit, Cees, additional, van Vulpen, Lize F. D., additional, van der Valk, Paul R., additional, Eikenboom, Jeroen, additional, Heubel‐Moenen, Floor C. J. I., additional, Hooimeijer, Louise, additional, Ypma, Paula, additional, Nieuwenhuizen, Laurens, additional, Coppens, Michiel, additional, Schols, Saskia E. M., additional, Laros‐van Gorkom, Britta A. P., additional, Leebeek, Frank W. G., additional, Driessens, Mariëtte H. E., additional, Rosendaal, Frits R., additional, van der Bom, Johanna G., additional, Fijnvandraat, Karin, additional, and Gouw, Samantha C., additional

Published
2023
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16. Transition readiness among adolescents and young adults with haemophilia in the Netherlands:Nationwide questionnaire study

Author

Brands, Martijn R., Janssen, Ebony A.M., Cnossen, Marjon H., Smit, Cees, van Vulpen, Lize F.D., van der Valk, Paul R., Eikenboom, Jeroen, Heubel-Moenen, Floor C.J.I., Hooimeijer, Louise, Ypma, Paula, Nieuwenhuizen, Laurens, Coppens, Michiel, Schols, Saskia E.M., Laros-van Gorkom, Britta A.P., Leebeek, Frank W.G., Driessens, Mariëtte H.E., Rosendaal, Frits R., van der Bom, Johanna G., Fijnvandraat, Karin, Gouw, Samantha C., Brands, Martijn R., Janssen, Ebony A.M., Cnossen, Marjon H., Smit, Cees, van Vulpen, Lize F.D., van der Valk, Paul R., Eikenboom, Jeroen, Heubel-Moenen, Floor C.J.I., Hooimeijer, Louise, Ypma, Paula, Nieuwenhuizen, Laurens, Coppens, Michiel, Schols, Saskia E.M., Laros-van Gorkom, Britta A.P., Leebeek, Frank W.G., Driessens, Mariëtte H.E., Rosendaal, Frits R., van der Bom, Johanna G., Fijnvandraat, Karin, and Gouw, Samantha C.

Abstract

Introduction: Care for adolescents with haemophilia is transferred from paediatric to adult care around the age of 18 years. Transition programs help to prepare adolescents for this transfer and prevent declining treatment adherence. Evaluating transition readiness may identify areas for improvement. Objective: Assess transition readiness among Dutch adolescents and young adults with haemophilia, determine factors associated with transition readiness, and identify areas of improvement in transition programs. Methods: All Dutch adolescents and young adults aged 12–25 years with haemophilia were invited to participate in a nationwide questionnaire study. Transition readiness was assessed using multiple-choice questions and was defined as being ready or almost ready for transition. Potential factors associated with transition readiness were investigated, including: socio-demographic and disease-related factors, treatment adherence, health-related quality of life, and self-efficacy. Results: Data of 45 adolescents and 84 young adults with haemophilia (47% with severe haemophilia) were analyzed. Transition readiness increased with age, from 39% in 12–14 year-olds to 63% in 15–17 year-olds. Nearly all post-transition young adults (92%, 77/84) reported they were ready for transition. Transition readiness was associated with treatment adherence, as median VERITAS-Pro treatment adherence scores were worse in patients who were not ready (17, IQR 9–29), compared to those ready for transition (11, IQR 9–16). Potential improvements were identified: getting better acquainted with the adult treatment team prior to transition and information on managing healthcare costs. Conclusions: Nearly all post-transition young adults reported they were ready for transition. Improvements were identified regarding team acquaintance and preparation for managing healthcare costs.

Published
2023

17. Transition readiness among adolescents and young adults with haemophilia in the Netherlands: Nationwide questionnaire study

Author

Poli Van Creveldkliniek Medisch, Brands, Martijn R, Janssen, Ebony A M, Cnossen, Marjon H, Smit, Cees, van Vulpen, Lize F D, van der Valk, Paul R, Eikenboom, Jeroen, Heubel-Moenen, Floor C J I, Hooimeijer, Louise, Ypma, Paula, Nieuwenhuizen, Laurens, Coppens, Michiel, Schols, Saskia E M, Laros-van Gorkom, Britta A P, Leebeek, Frank W G, Driessens, Mariëtte H E, Rosendaal, Frits R, van der Bom, Johanna G, Fijnvandraat, Karin, Gouw, Samantha C, Poli Van Creveldkliniek Medisch, Brands, Martijn R, Janssen, Ebony A M, Cnossen, Marjon H, Smit, Cees, van Vulpen, Lize F D, van der Valk, Paul R, Eikenboom, Jeroen, Heubel-Moenen, Floor C J I, Hooimeijer, Louise, Ypma, Paula, Nieuwenhuizen, Laurens, Coppens, Michiel, Schols, Saskia E M, Laros-van Gorkom, Britta A P, Leebeek, Frank W G, Driessens, Mariëtte H E, Rosendaal, Frits R, van der Bom, Johanna G, Fijnvandraat, Karin, and Gouw, Samantha C

Published
2023

19. Little discrepancy between one-stage and chromogenic FVIII/IX assays in a large international cohort of persons with non-severe hemophilia A and B

Author

Zwagemaker, Anne-Fleur, primary, Kloosterman, Fabienne R., additional, Gouw, Samantha C., additional, Boyce, Sara, additional, Brons, Paul, additional, Cnossen, Marjon H., additional, Collins, Peter W., additional, Eikenboom, Jeroen, additional, Hay, Charles, additional, Hengeveld, Rutger C.C., additional, Jackson, Shannon, additional, Klopper-Tol, Caroline A.M., additional, Kruip, Marieke J.H.A., additional, Laros-van Gorkom, Britta, additional, Male, Christoph, additional, Nieuwenhuizen, Laurens, additional, Shapiro, Susan, additional, Fijnvandraat, Karin, additional, and Coppens, M., additional

Published
2022
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20. Factor VIII gene (F8) mutation and risk of inhibitor development in nonsevere hemophilia A

Author

Eckhardt, Corien L., van Velzen, Alice S., Peters, Marjolein, Astermark, Jan, Brons, Paul P., Castaman, Giancarlo, Cnossen, Marjon H., Dors, Natasja, Escuriola-Ettingshausen, Carmen, Hamulyak, Karly, Hart, Daniel P., Hay, Charles R.M., Haya, Saturnino, van Heerde, Waander L., Hermans, Cedric, Holmström, Margareta, Jimenez-Yuste, Victor, Keenan, Russell D., Klamroth, Robert, Laros-van Gorkom, Britta A.P., Leebeek, Frank W.G., Liesner, Ri, Mäkipernaa, Anne, Male, Christoph, Mauser-Bunschoten, Evelien, Mazzucconi, Maria G., McRae, Simon, Meijer, Karina, Mitchell, Michael, Morfini, Massimo, Nijziel, Marten, Oldenburg, Johannes, Peerlinck, Kathelijne, Petrini, Pia, Platokouki, Helena, Reitter-Pfoertner, Sylvia E., Santagostino, Elena, Schinco, Piercarla, Smiers, Frans J., Siegmund, Berthold, Tagliaferri, Annarita, Yee, Thynn T., Kamphuisen, Pieter Willem, van der Bom, Johanna G., and Fijnvandraat, Karin

Published
2013
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24. Importance of Genotyping in von Willebrand Disease to Elucidate Pathogenic Mechanisms and Variability in Phenotype

Author

Atiq, Ferdows, Boender, Johan, Van Heerde, Waander L., Tellez Garcia, Juan M., Schoormans, Selene C., Krouwel, Sandy, Cnossen, Marjon H., Laros-Van Gorkom, Britta A.P., De Meris, Joke, Fijnvandraat, Karin, Van Der Bom, Johanna G., Meijer, Karina, Van Galen, Karin P.M., Eikenboom, Jeroen, Leebeek, Frank W.G., Atiq, Ferdows, Boender, Johan, Van Heerde, Waander L., Tellez Garcia, Juan M., Schoormans, Selene C., Krouwel, Sandy, Cnossen, Marjon H., Laros-Van Gorkom, Britta A.P., De Meris, Joke, Fijnvandraat, Karin, Van Der Bom, Johanna G., Meijer, Karina, Van Galen, Karin P.M., Eikenboom, Jeroen, and Leebeek, Frank W.G.

Abstract

Genotyping is not routinely performed at diagnosis of von Willebrand disease (VWD). Therefore, the association between genetic variants and pathogenic mechanism or the clinical and laboratory phenotype is unknown in most patients, especially in type 1 VWD. To investigate whether genotyping adds to a better understanding of the pathogenic mechanisms and variability in phenotype, we analyzed the VWF gene in 390 well-defined VWD patients, included in the WiN study. A VWF gene variant was found in 155 patients (61.5%) with type 1, 122 patients (98.4%) with type 2, and 14 patients (100%) with type 3 VWD. Forty-eight variants were novel. For each VWF gene variant, the pathogenic mechanisms associated with reduced VWF levels was investigated using the FVIII:C/VWF:Ag and VWFpp/VWF:Ag ratios. In type 1 VWD, reduced synthesis or secretion of VWF was most frequently found in patients with nonsense variants, frameshift variants, and deletions, whereas rapid clearance of VWF was mainly found in patients with missense variants. Furthermore, type 1 VWD patients with and without a VWF gene variant were clearly distinct in their clinical features such as age of diagnosis, laboratory phenotype, and bleeding phenotype. In type 2 VWD, 81% of variants were associated with an increased clearance of VWF. To conclude, we identified the pathogenic mechanisms associated with various VWF gene variants in type 1, 2, and 3 VWD patients. Additionally, major differences in the phenotype of type 1 VWD patients with and without a variant were observed, which may be of importance for clinical management.

Published
2022

25. Combining factor VIII levels and thrombin/plasmin generation:A population pharmacokinetic-pharmacodynamic model for patients with haemophilia A

Author

Bukkems, Laura H., Valke, Lars L.F.G., Barteling, Wideke, Laros-van Gorkom, Britta A.P., Blijlevens, Nicole M.A., Cnossen, Marjon H., van Heerde, Waander L., Schols, Saskia E.M., Mathôt, Ron A.A., Bukkems, Laura H., Valke, Lars L.F.G., Barteling, Wideke, Laros-van Gorkom, Britta A.P., Blijlevens, Nicole M.A., Cnossen, Marjon H., van Heerde, Waander L., Schols, Saskia E.M., and Mathôt, Ron A.A.

Abstract

Aims: Prophylactic treatment of haemophilia A patients with factor VIII (FVIII) concentrate focuses on maintaining a minimal trough FVIII activity level to prevent bleeding. However, due to differences in bleeding tendency, the pharmacokinetic (PK)-guided dosing approach may be suboptimal. An alternative approach could be the addition of haemostatic pharmacodynamic (PD) parameters, reflecting a patient's unique haemostatic balance. Our aim was to develop a population PK/PD model, based on FVIII activity levels and Nijmegen Haemostasis Assay (NHA) patterns, a global haemostatic assay that measures thrombin/plasmin generation simultaneously. Methods: PK/PD measurements were collected from 30 patients treated with standard half-life FVIII concentrate. The relationship between FVIII activity levels and the thrombin/plasmin generation parameters (thrombin potential, thrombin peak height and plasmin peak height), were described by sigmoidal Emax functions. Results: The obtained EC50 value was smallest for the normalized thrombin potential (11.6 IU/dL), followed by normalized thrombin peak height (56.6 IU/dL) and normalized plasmin peak height (593 IU/dL), demonstrating that normalized thrombin potential showed 50% of the maximal effect at lower FVIII activity levels. Substantial inter-individual variability in the PD parameters, such as EC50 of thrombin potential (86.9%) was observed, indicating that, despite similar FVIII activity levels, haemostatic capacity varies significantly between patients. Conclusion: These data suggest that dosing based on patients' individual PK/PD parameters may be beneficial over dosing solely on individual PK parameters. This model could be used as proof-of-principle to examine the application of PK/PD-guided dosing. However, the relation between the PD parameters and bleeding has to be better defined.

Published
2022

26. Von Willebrand disease type 2M:Correlation between genotype and phenotype

Author

Maas, Dominique P.M.S.M., Atiq, Ferdows, Blijlevens, Nicole M.A., Brons, Paul P T, Krouwel, Sandy, Laros-van Gorkom, Britta A.P., Leebeek, Frank W G, Nieuwenhuizen, Laurens, Schoormans, Selene C.M., Simons, Annet, Meijer, Daniëlle, van Heerde, Waander L., Schols, Saskia E.M., Maas, Dominique P.M.S.M., Atiq, Ferdows, Blijlevens, Nicole M.A., Brons, Paul P T, Krouwel, Sandy, Laros-van Gorkom, Britta A.P., Leebeek, Frank W G, Nieuwenhuizen, Laurens, Schoormans, Selene C.M., Simons, Annet, Meijer, Daniëlle, van Heerde, Waander L., and Schols, Saskia E.M.

Abstract

Background: An appropriate clinical diagnosis of von Willebrand disease (VWD) can be challenging because of a variable bleeding pattern and laboratory phenotype. Genotyping is a powerful diagnostic tool and may have an essential role in the diagnostic field of VWD. Objectives: To unravel the clinical and laboratory heterogeneity of genetically confirmed VWD type 2M patients and to investigate their relationship. Methods: Patients with a confirmed VWD type 2M genetic variant in the A1 or A3 domain of von Willebrand factor (VWF) and normal or only slightly aberrant VWF multimers were selected from all subjects genotyped at the Radboud university medical center because of a high suspicion of VWD. Bleeding scores and laboratory results were analyzed. Results: Fifty patients had a clinically relevant genetic variant in the A1 domain. Median bleeding score was 5. Compared with the nationwide Willebrand in the Netherlands study type 2 cohort, bleeding after surgery or delivery was reported more frequently and mucocutaneous bleedings less frequently. Median VWF activity/VWF antigen (VWF:Act/VWF:Ag) ratio was 0.32, whereas VWF collagen binding activity/VWF antigen (VWF:CB/VWF:Ag) ratio was 0.80. Variants in the A3 domain were only found in two patients with low to normal VWF:Act/VWF:Ag ratios (0.45, 1.03) and low VWF:CB/VWF:Ag ratios (0.45, 0.63). Conclusion: Genetically confirmed VWD type 2M patients have a relatively mild clinical phenotype, except for bleeding after surgery and delivery. Laboratory phenotype is variable and depends on the underlying genetic variant. Addition of genotyping to the current phenotypic characterization may improve diagnosis and classification of VWD.

Published
2022

27. Importance of Genotyping in von Willebrand Disease to Elucidate Pathogenic Mechanisms and Variability in Phenotype

Author

Poli Van Creveldkliniek Medisch, Child Health, Atiq, Ferdows, Boender, Johan, Van Heerde, Waander L., Tellez Garcia, Juan M., Schoormans, Selene C., Krouwel, Sandy, Cnossen, Marjon H., Laros-Van Gorkom, Britta A.P., De Meris, Joke, Fijnvandraat, Karin, Van Der Bom, Johanna G., Meijer, Karina, Van Galen, Karin P.M., Eikenboom, Jeroen, Leebeek, Frank W.G., Poli Van Creveldkliniek Medisch, Child Health, Atiq, Ferdows, Boender, Johan, Van Heerde, Waander L., Tellez Garcia, Juan M., Schoormans, Selene C., Krouwel, Sandy, Cnossen, Marjon H., Laros-Van Gorkom, Britta A.P., De Meris, Joke, Fijnvandraat, Karin, Van Der Bom, Johanna G., Meijer, Karina, Van Galen, Karin P.M., Eikenboom, Jeroen, and Leebeek, Frank W.G.

Published
2022

28. Combining factor VIII levels and thrombin/plasmin generation: A population pharmacokinetic‐pharmacodynamic model for patients with haemophilia A

Author

Bukkems, Laura H., primary, Valke, Lars L. F. G., additional, Barteling, Wideke, additional, Laros‐van Gorkom, Britta A. P., additional, Blijlevens, Nicole M. A., additional, Cnossen, Marjon H., additional, van Heerde, Waander L., additional, Schols, Saskia E. M., additional, and Mathôt, Ron A. A., additional

Published
2022
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29. BMI is an important determinant of VWF and FVIII levels and bleeding phenotype in patients with von Willebrand disease

Author

Atiq, Ferdows, Fijnvandraat, Karin, van Galen, Karin P. M., Laros-van Gorkom, Britta A. P., Meijer, Karina, de Meris, Joke, Coppens, Michiel, Mauser-Bunschoten, Eveline P., Cnossen, Marjon H., van der Bom, Johanna G., Eikenboom, Jeroen, Leebeek, Frank W. G., Coppens, M., Kors, A., Zweegman, S., de Meris, J., Goverde, G. J., Jonkers, M. H., Dors, N., Nijziel, M. R., Nieuwenhuizen, L., Meijer, K., Tamminga, R. Y. J., van der LindenHagaZiekenhuis, P. W., Ypma, P. F., Eikenboom, H. C. J., van der Bom, J. G., Smiers, F. J. W., Granzen, B., Hamulyák, K., Brons, P., Laros‐van Gorkom, B. A. P., Leebeek, F. W. G., Cnossen, M. H., Atiq, F., Mauser-Bunschoten, E. P., van Galen, K. P. M., Hematology, Pediatrics, Paediatric Haematology, ACS - Pulmonary hypertension & thrombosis, Vascular Medicine, ARD - Amsterdam Reproduction and Development, VU University medical center, Kindergeneeskunde, MUMC+: MA Medische Staf Kindergeneeskunde (9), RS: FHML non-thematic output, and Real World Studies in PharmacoEpidemiology, -Genetics, -Economics and -Therapy (PEGET)

Subjects
Male, medicine.medical_specialty, Letter, Vascular damage Radboud Institute for Health Sciences [Radboudumc 16], E–Only Articles, Hemorrhage, von Willebrand Disease, Type 2, von Willebrand Disease, Type 1, Body Mass Index, Text mining, Internal medicine, von Willebrand Factor, Correspondence, Von Willebrand disease, Medicine, Humans, In patient, Hematology, Factor VIII, business.industry, Middle Aged, Blood coagulation factors, medicine.disease, Phenotype, Immunology, Female, business, Body mass index
Abstract

Contains fulltext : 208376.pdf (Publisher’s version ) (Open Access)

Published
2019
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31. Perioperative pharmacokinetic-guided factor VIII concentrate dosing in haemophilia (OPTI-CLOT trial): an open-label, multicentre, randomised, controlled trial

Author

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

Published
2021
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32. Von Willebrand Factor Multimer Densitometric Analysis:Validation of the Clinical Accuracy and Clinical Implications in von Willebrand Disease

Author

Boender, Johan, Atiq, Ferdows, Cnossen, Marjon H., Van Der Bom, Johanna G., Fijnvandraat, Karin, De Meris, Joke, De Maat, Moniek P.M., Van Galen, Karin P.M., Laros-Van Gorkom, Britta A.P., Meijer, Karina, Eikenboom, Jeroen, Leebeek, Frank W.G., Boender, Johan, Atiq, Ferdows, Cnossen, Marjon H., Van Der Bom, Johanna G., Fijnvandraat, Karin, De Meris, Joke, De Maat, Moniek P.M., Van Galen, Karin P.M., Laros-Van Gorkom, Britta A.P., Meijer, Karina, Eikenboom, Jeroen, and Leebeek, Frank W.G.

Published
2021

33. Treatment-related risk factors for inhibitor development in non-severe hemophilia A after 50 cumulative exposure days: a case-control study.

Author

UCL - SSS/IREC/CARD - Pôle de recherche cardiovasculaire, UCL - (SLuc) Service d'hématologie, Abdi, Amal, Eckhardt, Corien L, van Velzen, Alice S, Vuong, Caroline, Coppens, Michiel, Castaman, Giancarlo, Hart, Dan P, Hermans, Cédric, Laros-van Gorkom, Britta, Leebeek, Frank W G, Mancuso, Maria Elisa, Mazzucconi, Maria G, McRae, Simon, Oldenburg, Johannes, Male, Christoph, van der Bom, Johanna G, Fijnvandraat, Karin, Gouw, Samantha C, INSIGHT Study Group, UCL - SSS/IREC/CARD - Pôle de recherche cardiovasculaire, UCL - (SLuc) Service d'hématologie, Abdi, Amal, Eckhardt, Corien L, van Velzen, Alice S, Vuong, Caroline, Coppens, Michiel, Castaman, Giancarlo, Hart, Dan P, Hermans, Cédric, Laros-van Gorkom, Britta, Leebeek, Frank W G, Mancuso, Maria Elisa, Mazzucconi, Maria G, McRae, Simon, Oldenburg, Johannes, Male, Christoph, van der Bom, Johanna G, Fijnvandraat, Karin, Gouw, Samantha C, and INSIGHT Study Group

Abstract

BACKGROUND: Non-severe hemophilia A patients have a life-long inhibitor risk. Yet, no studies analyzed risk factors for inhibitor development after 50 factor VIII (FVIII) exposure days (EDs). OBJECTIVES: This case-control study investigated treatment-related risk factors for inhibitor development in non-severe hemophilia A and assessed whether these risk factors were different for early versus late inhibitor development. PATIENTS/METHODS: Non-severe hemophilia A patients (FVIII:C 2-40%) were selected from the INSIGHT study. Inhibitor-positive patients were defined as early (<50 EDs) or late (> 50EDs) cases and matched to 1-4 inhibitor-negative controls by year of birth, cumulative number of EDs and center/country. We investigated treatment intensity during the last 10 EDs prior to inhibitor development. Intensive treatment was defined as: surgery, peak treatment (10 consecutive EDs) and high mean FVIII dose (>45 IU/kg/ED). Odds ratios (OR) were calculated by logistic regression. RESULTS: Of 2,709 patients, we analyzed 63 early and 26 late cases and 195 and 71 respectively matched controls. Peak treatment was associated with early and late inhibitor risk (crude OR 1.8, 95%CI 1.0-3.4; 4.0, 95%CI 1.1-14.3). This association was slightly less pronounced after adjustment for mean FVIII dose. High mean FVIII dose was also associated with early and late inhibitor risk (crude OR 2.8, 95%CI 1.5-5.1; 4.5, 95%CI 1.2-16.6). Surgery increased inhibitor risk for early cases. This was less pronounced for late cases. CONCLUSIONS: Our findings suggest that intensive FVIII treatment remains a risk factor for inhibitor development in non-severe hemophilia A after more than 50 EDs. Therefore, persistent caution is required throughout the life-time treatment course.

Published
2021

34. Dosing of factor VIII concentrate by ideal body weight is more accurate in overweight and obese haemophilia A patients

Author

van Moort, Iris, Preijers, Tim, Hazendonk, Hendrika C.A.M., Schutgens, Roger E.G., Laros-van Gorkom, Britta A.P., Nieuwenhuizen, Laurens, van der Meer, Felix J.M., Fijnvandraat, Karin, Leebeek, Frank W.G., Meijer, Karina, Mathôt, Ron A.A., Cnossen, Marjon H., van Moort, Iris, Preijers, Tim, Hazendonk, Hendrika C.A.M., Schutgens, Roger E.G., Laros-van Gorkom, Britta A.P., Nieuwenhuizen, Laurens, van der Meer, Felix J.M., Fijnvandraat, Karin, Leebeek, Frank W.G., Meijer, Karina, Mathôt, Ron A.A., and Cnossen, Marjon H.

Abstract

Aims: Under- and, especially, overdosing of replacement therapy in haemophilia A patients may be prevented by application of other morphometric variables than body weight (BW) to dose factor VIII (FVIII) concentrates. Therefore, we aimed to investigate which morphometric variables best describe interindividual variability (IIV) of FVIII concentrate pharmacokinetic (PK) parameters. Methods: PK profiling was performed by measuring 3 FVIII levels after a standardized dose of 50 IU kg−1 FVIII concentrate. A population PK model was constructed, in which IIV for clearance (CL) and central volume of distribution (V1) was quantified. Relationships between CL, V1 and 5 morphometric variables (BW, ideal BW [IBW], lean BW, adjusted BW, and body mass index [BMI]) were evaluated in normal weight (BMI < 25 kg m−2), overweight (BMI 25–30 kg m−2) and obese haemophilia A patients (BMI > 30 kg m−2). Results: In total, 57 haemophilia A patients (FVIII≤0.05 IU mL−1) were included with median BW of 83 kg (range: 53–133) and median age of 48 years (range: 18–77). IBW best explained observed variability between patients, as IIV for CL and V1 was reduced from 45.1 to 37.6 and 26.% to 14.1%, respectively. CL, V1 and half-life were similar for all BMI categories. The national recommended dosing schedule did not result in adequate trough levels, both in case of dosing based on BW and IBW. However, dosing based on IBW prevented unnecessary high FVIII peaks. Conclusion: IBW is the most suitable morphometric variable to explain interindividual FVIII PK variability and is more appropriate to dose overweight and obese patients.

Published
2021

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

Author

Poli Van Creveldkliniek Medisch, Circulatory Health, Infection & Immunity, Child Health, van Moort, Iris, Preijers, Tim, Bukkems, Laura H., Hazendonk, Hendrika C.A.M., van der Bom, Johanna G., Laros-van Gorkom, Britta A.P., Beckers, Erik E.A.M., Nieuwenhuizen, Laurens, van der Meer, Felix J.M., Ypma, Paula, Coppens, Michiel, Fijnvandraat, Karin, Schutgens, Roger E.G., Meijer, Karina, Leebeek, Frank W.G., Mathôt, Ron A.A., Cnossen, Marjon H., “OPTI-CLOT” study group, Poli Van Creveldkliniek Medisch, Circulatory Health, Infection & Immunity, Child Health, van Moort, Iris, Preijers, Tim, Bukkems, Laura H., Hazendonk, Hendrika C.A.M., van der Bom, Johanna G., Laros-van Gorkom, Britta A.P., Beckers, Erik E.A.M., Nieuwenhuizen, Laurens, van der Meer, Felix J.M., Ypma, Paula, Coppens, Michiel, Fijnvandraat, Karin, Schutgens, Roger E.G., Meijer, Karina, Leebeek, Frank W.G., Mathôt, Ron A.A., Cnossen, Marjon H., and “OPTI-CLOT” study group

Published
2021

36. Von Willebrand Factor Multimer Densitometric Analysis: Validation of the Clinical Accuracy and Clinical Implications in von Willebrand Disease

Author

Poli Van Creveldkliniek Medisch, Child Health, Boender, Johan, Atiq, Ferdows, Cnossen, Marjon H., Van Der Bom, Johanna G., Fijnvandraat, Karin, De Meris, Joke, De Maat, Moniek P.M., Van Galen, Karin P.M., Laros-Van Gorkom, Britta A.P., Meijer, Karina, Eikenboom, Jeroen, Leebeek, Frank W.G., Poli Van Creveldkliniek Medisch, Child Health, Boender, Johan, Atiq, Ferdows, Cnossen, Marjon H., Van Der Bom, Johanna G., Fijnvandraat, Karin, De Meris, Joke, De Maat, Moniek P.M., Van Galen, Karin P.M., Laros-Van Gorkom, Britta A.P., Meijer, Karina, Eikenboom, Jeroen, and Leebeek, Frank W.G.

Published
2021

37. Dosing of factor VIII concentrate by ideal body weight is more accurate in overweight and obese haemophilia A patients

Author

Poli Van Creveldkliniek Medisch, Infection & Immunity, Circulatory Health, van Moort, Iris, Preijers, Tim, Hazendonk, Hendrika C.A.M., Schutgens, Roger E.G., Laros-van Gorkom, Britta A.P., Nieuwenhuizen, Laurens, van der Meer, Felix J.M., Fijnvandraat, Karin, Leebeek, Frank W.G., Meijer, Karina, Mathôt, Ron A.A., Cnossen, Marjon H., the OPTI-CLOT study group, Poli Van Creveldkliniek Medisch, Infection & Immunity, Circulatory Health, van Moort, Iris, Preijers, Tim, Hazendonk, Hendrika C.A.M., Schutgens, Roger E.G., Laros-van Gorkom, Britta A.P., Nieuwenhuizen, Laurens, van der Meer, Felix J.M., Fijnvandraat, Karin, Leebeek, Frank W.G., Meijer, Karina, Mathôt, Ron A.A., Cnossen, Marjon H., and the OPTI-CLOT study group

Published
2021

39. A Dominant-Negative GFI1B Mutation in the Gray Platelet Syndrome

Author

Monteferrario, Davide, Bolar, Nikhita A., Marneth, Anna E., Hebeda, Konnie M., Bergevoet, Saskia M., Veenstra, Hans, Laros-van Gorkom, Britta A.P., MacKenzie, Marius A., Khandanpour, Cyrus, Botezatu, Lacramiora, Fransen, Erik, Van Camp, Guy, Duijnhouwer, Anthonie L., Salemink, Simone, Willemsen, Brigith, Huls, Gerwin, Preijers, Frank, Van Heerde, Waander, Jansen, Joop H., Kempers, Marlies J.E., Loeys, Bart L., Van Laer, Lut, and Van der Reijden, Bert A.

Published
2014
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40. Von Willebrand Factor Multimer Densitometric Analysis: Validation of the Clinical Accuracy and Clinical Implications in Von Willebrand Disease

Author

Boender, Johan, primary, Atiq, Ferdows, additional, Cnossen, Marjon H., additional, van der Bom, Johanna G., additional, Fijnvandraat, Karin, additional, de Meris, Joke, additional, de Maat, Moniek P. M., additional, van Galen, Karin P. M., additional, Laros-van Gorkom, Britta A. P., additional, Meijer, Karina, additional, Eikenboom, Jeroen, additional, and Leebeek, Frank W. G., additional

Published
2021
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41. Semiautomatic VWF Multimer Densitometric Analysis: Validation of the Clinical Accuracy and Clinical Implications in Von Willebrand Disease

Author

Atiq, Ferdows, primary, Boender, Johan, additional, Cnossen, Marjon H., additional, van der Bom, Johanna G, additional, Fijnvandraat, Karin, additional, de Meris, Joke, additional, de Maat, Moniek P.M., additional, Van Galen, Karin P.M., additional, Laros-Van Gorkom, Britta A.P., additional, Meijer, Karina, additional, Eikenboom, Jeroen C.J., additional, and Leebeek, Frank W.G., additional

Published
2020
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42. Platelet CD34 expression in a patient with a partial deletion of transcription factor subunit CBFB

Author

Bergen, Maaike G.J.M., primary, Saes, Joline L., additional, Simons, Annet, additional, Hebeda, Konnie M., additional, Henskens, Yvonne M.C., additional, Barteling, Wideke, additional, Huys, Erik, additional, Laros‐van Gorkom, Britta A.P., additional, Schols, Saskia E.M., additional, Preijers, Frank W., additional, Jongmans, Marjolijn C.J., additional, Jansen, Joop H., additional, and Reijden, Bert A., additional

Published
2020
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43. Platelet CD34 expression in a patient with a partial deletion of transcription factor subunit CBFB

Author

Genetica Klinische Genetica, Child Health, Cancer, van Bergen, Maaike G J M, Saes, Joline L, Simons, Annet, Hebeda, Konnie M, Henskens, Yvonne M C, Barteling, Wideke, Huys, Erik, Laros-van Gorkom, Britta A P, Schols, Saskia E M, Preijers, Frank W, Jongmans, Marjolijn C J, Jansen, Joop H, van der Reijden, Bert A, Genetica Klinische Genetica, Child Health, Cancer, van Bergen, Maaike G J M, Saes, Joline L, Simons, Annet, Hebeda, Konnie M, Henskens, Yvonne M C, Barteling, Wideke, Huys, Erik, Laros-van Gorkom, Britta A P, Schols, Saskia E M, Preijers, Frank W, Jongmans, Marjolijn C J, Jansen, Joop H, and van der Reijden, Bert A

Published
2020

44. von Willebrand Factor and Factor VIII Clearance in Perioperative Hemophilia A Patients

Author

Poli Van Creveldkliniek Medisch, Circulatory Health, Infection & Immunity, van Moort, Iris, Bukkems, Laura H, Heijdra, Jessica M, Schutgens, Roger E G, Laros-van Gorkom, Britta A P, Nieuwenhuizen, Laurens, van der Meer, Felix J M, Fijnvandraat, Karin, Ypma, Paula, de Maat, Moniek P M, Leebeek, Frank W G, Meijer, Karina, Eikenboom, Jeroen, Mathôt, Ron A A, Cnossen, Marjon H, “OPTI-CLOT” study group, Poli Van Creveldkliniek Medisch, Circulatory Health, Infection & Immunity, van Moort, Iris, Bukkems, Laura H, Heijdra, Jessica M, Schutgens, Roger E G, Laros-van Gorkom, Britta A P, Nieuwenhuizen, Laurens, van der Meer, Felix J M, Fijnvandraat, Karin, Ypma, Paula, de Maat, Moniek P M, Leebeek, Frank W G, Meijer, Karina, Eikenboom, Jeroen, Mathôt, Ron A A, Cnossen, Marjon H, and “OPTI-CLOT” study group

Published
2020

45. Validation of a perioperative population factor VIII pharmacokinetic model with a large cohort of pediatric hemophilia a patients.

Author

Preijers, Tim, Liesner, Ri, Hazendonk, Hendrika C. A. M., Chowdary, Pratima, Driessens, Mariëtte H. E., Hart, Dan P., Laros‐van Gorkom, Britta A. P., van der Meer, Felix J. M., Meijer, Karina, Fijnvandraat, Karin, Leebeek, Frank W. G., Mathôt, Ron A. A., and Cnossen, Marjon H.

Subjects
HEMOPHILIACS, PHARMACOKINETICS, BLOOD coagulation factor VIII, BAYESIAN analysis, BLOOD coagulation factors, BLOOD coagulation factor VIII antibodies, BODY weight
Abstract

Aims: Population pharmacokinetic (PK) models are increasingly applied to perform individualized dosing of factor VIII (FVIII) concentrates in haemophilia A patients. To guarantee accurate performance of a population PK model in dose individualization, validation studies are of importance. However, external validation of population PK models requires independent data sets and is, therefore, seldomly performed. Therefore, this study aimed to validate a previously published population PK model for FVIII concentrates administrated perioperatively. Methods: A previously published population PK model for FVIII concentrate during surgery was validated using independent data from 87 children with severe haemophilia A with a median (range) age of 2.6 years (0.03–15.2) and body weight of 14 kg (4–57). First, the predictive performance of the previous model was evaluated with MAP Bayesian analysis using NONMEM v7.4. Subsequently, the model parameters were (re)estimated using a combined dataset consisting of the previous modelling data and the data available for the external validation. Results: The previous model underpredicted the measured FVIII levels with a median of 0.17 IU mL−1. Combining the new, independent and original data, a dataset comprising 206 patients with a mean age of 7.8 years (0.03–77.6) and body weight of 30 kg (4–111) was obtained. Population PK modelling provided estimates for CL, V1, V2, and Q: 171 mL h−1 68 kg−1, 2930 mL 68 kg−1, 1810 mL 68 kg−1, and 172 mL h−1 68 kg−1, respectively. This model adequately described all collected FVIII levels, with a slight median overprediction of 0.02 IU mL−1. Conclusions: This study emphasizes the importance of external validation of population PK models using real‐life data. [ABSTRACT FROM AUTHOR]

Published
2021
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47. Desmopressin treatment combined with clotting factor VIII concentrates in patients with non-severe haemophilia A: protocol for a multicentre single-armed trial, the DAVID study

Author

Schütte, Lisette M, primary, Cnossen, Marjon H, additional, van Hest, Reinier M, additional, Driessens, Mariette H E, additional, Fijnvandraat, Karin, additional, Polinder, Suzanne, additional, Beckers, Erik A M, additional, Coppens, Michiel, additional, Eikenboom, Jeroen, additional, Laros-van Gorkom, Britta A P, additional, Meijer, Karina, additional, Nieuwenhuizen, Laurens, additional, Mauser-Bunschoten, Evelien P, additional, Leebeek, Frank W G, additional, Mathôt, Ron A A, additional, and Kruip, Marieke J H A, additional

Published
2019
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48. Sports participation and physical activity in patients with von Willebrand disease

Author

Atiq, Ferdows, Mauser-Bunschoten, Eveline P, Eikenboom, Jeroen, van Galen, Karin P M, Meijer, Karina, de Meris, Joke, Cnossen, Marjon H, Beckers, Erick A M, Laros-van Gorkom, Britta A P, Nieuwenhuizen, Laurens, van der Bom, Johanna G, Fijnvandraat, Karin, Leebeek, Frank W G, WiN study group, Atiq, Ferdows, Mauser-Bunschoten, Eveline P, Eikenboom, Jeroen, van Galen, Karin P M, Meijer, Karina, de Meris, Joke, Cnossen, Marjon H, Beckers, Erick A M, Laros-van Gorkom, Britta A P, Nieuwenhuizen, Laurens, van der Bom, Johanna G, Fijnvandraat, Karin, Leebeek, Frank W G, and WiN study group

Published
2019

49. The prevalence and burden of hand and wrist bleeds in von Willebrand disease

Author

van Deukeren, Désirée, Mauser-Bunschoten, Eveline P, Schutgens, Roger E G, Eikenboom, Jeroen, Meijer, Karina, Fijnvandraat, Karin, Laros-van Gorkom, Britta A P, Cnossen, Marjon, de Meris, Joke, van der Bom, Johanna G, Leebeek, Frank W G, van Galen, Karin P M, van Deukeren, Désirée, Mauser-Bunschoten, Eveline P, Schutgens, Roger E G, Eikenboom, Jeroen, Meijer, Karina, Fijnvandraat, Karin, Laros-van Gorkom, Britta A P, Cnossen, Marjon, de Meris, Joke, van der Bom, Johanna G, Leebeek, Frank W G, and van Galen, Karin P M

Published
2019

50. The prevalence and burden of hand and wrist bleeds in von Willebrand disease

Author

Poli Van Creveldkliniek Medisch, Circulatory Health, Other research (not in main researchprogram), van Deukeren, Désirée, Mauser-Bunschoten, Eveline P, Schutgens, Roger E G, Eikenboom, Jeroen, Meijer, Karina, Fijnvandraat, Karin, Laros-van Gorkom, Britta A P, Cnossen, Marjon, de Meris, Joke, van der Bom, Johanna G, Leebeek, Frank W G, van Galen, Karin P M, Poli Van Creveldkliniek Medisch, Circulatory Health, Other research (not in main researchprogram), van Deukeren, Désirée, Mauser-Bunschoten, Eveline P, Schutgens, Roger E G, Eikenboom, Jeroen, Meijer, Karina, Fijnvandraat, Karin, Laros-van Gorkom, Britta A P, Cnossen, Marjon, de Meris, Joke, van der Bom, Johanna G, Leebeek, Frank W G, and van Galen, Karin P M

Published
2019

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