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5. Enhanced VWF clearance in low VWF pathogenesis: limitations of the VWFpp/VWF:Ag ratio and clinical significance

Author

Montgomery, R., Flood, V., Haberichter, S., Abshire, T., Weiler, H., Lillicrap, D., James, P., O’Donnell, J., Ng, C., Di Paola, J., Sadler, B., Bennett, C., Sidonio, R., Manco-Johnson, M., Journeycake, J., Zia, A., Lusher, J., Rajpurkar, M., Shapiro, A., Lentz, S., Gill, J., Leissinger, C., Ragni, M., Tarantino, M., Roberts, J., Doherty, Dearbhla, Lavin, Michelle, Byrne, Mary, Nolan, Margaret, O’Sullivan, Jamie M., Ryan, Kevin, O’Connell, Niamh M., Haberichter, Sandra L., Christopherson, Pamela A., Di Paola, Jorge, James, Paula D., and O’Donnell, James S.

Published
2023
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6. Challenges in the management of women with type 2B von Willebrand disease during pregnancy and the postpartum period: evidence from literature and data from an international registry and physicians’ survey—communication from the Scientific and Standardization Committees of the International Society on Thrombosis and Haemostasis

Author

Miljic, Predrag, Noureldin, Abdelrahman, Lavin, Michelle, Kazi, Sajida, Sanchez-Luceros, Analia, James, Paula D., and Othman, Maha

Published
2023
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9. Surgical management of patients with von Willebrand disease: summary of 2 systematic reviews of the literature

Author

Brignardello-Petersen, Romina, El Alayli, Abdallah, Husainat, Nedaa, Kalot, Mohamad, Shahid, Shaneela, Aljabirii, Yazan, Britt, Alec, Alturkmani, Hani, El-Khechen, Hussein, Motaghi, Shahrzad, Roller, John, Dimassi, Ahmad, Abughanimeh, Omar, Madoukh, Bader, Arapshian, Alice, Grow, Jean M., Kouides, Peter, Laffan, Michael, Leebeek, Frank W.G., O'Brien, Sarah H., Tosetto, Alberto, James, Paula D., Connell, Nathan T., Flood, Veronica, and Mustafa, Reem A.

Published
2022
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10. Gynecologic and obstetric management of women with von Willebrand disease: summary of 3 systematic reviews of the literature

Author

Brignardello-Petersen, Romina, El Alayli, Abdallah, Husainat, Nedaa, Kalot, Mohamad A., Shahid, Shaneela, Aljabirii, Yazan, Britt, Alec, Alturkmani, Hani, El-Khechen, Hussein, Motaghi, Shahrzad, Roller, John, Abdul-Kadir, Rezan, Couper, Susie, Kouides, Peter, Lavin, Michelle, Ozelo, Margareth C., Weyand, Angela, James, Paula D., Connell, Nathan T., Flood, Veronica H., and Mustafa, Reem A.

Published
2022
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11. von Willebrand factor levels in the diagnosis of von Willebrand disease: a systematic review and meta-analysis

Author

Kalot, Mohamad A., Husainat, Nedaa, El Alayli, Abdallah, Abughanimeh, Omar, Diab, Osama, Tayiem, Sammy, Madoukh, Bader, Dimassi, Ahmad B., Qureini, Aref, Ameer, Barbara, Eikenboom, Jeroen C.J., Giraud, Nicolas, McLintock, Claire, McRae, Simon, Montgomery, Robert R., O'Donnell, James S., Scappe, Nikole, Sidonio, Robert F., Jr, Brignardello-Petersen, Romina, Flood, Veronica H., Connell, Nathan T., James, Paula D., and Mustafa, Reem A.

Published
2022
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12. Bleeding assessment tools in the diagnosis of VWD in adults and children: a systematic review and meta-analysis of test accuracy

Author

Kalot, Mohamad A., Husainat, Nedaa, Tayiem, Sammy, El Alayli, Abdallah, Dimassi, Ahmad B., Diab, Osama, Abughanimeh, Omar, Madoukh, Bader, Qureini, Aref, Ameer, Barbara, Di Paola, Jorge, Eikenboom, Jeroen C.J., Jacobs-Pratt, Vicky, McLintock, Claire, Montgomery, Robert, O'Donnell, James S., Sidonio, Robert, Jr., Brignardello-Petersen, Romina, Flood, Veronica, Connell, Nathan T., James, Paula D., and Mustafa, Reem A.

Published
2021
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15. ASH ISTH NHF WFH 2021 guidelines on the diagnosis of von Willebrand disease

Author

James, Paula D., Connell, Nathan T., Ameer, Barbara, Di Paola, Jorge, Eikenboom, Jeroen, Giraud, Nicolas, Haberichter, Sandra, Jacobs-Pratt, Vicki, Konkle, Barbara, McLintock, Claire, McRae, Simon, R. Montgomery, Robert, O'Donnell, James S., Scappe, Nikole, Sidonio, Robert, Jr, Flood, Veronica H., Husainat, Nedaa, Kalot, Mohamad A., and Mustafa, Reem A.

Published
2021
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16. ASH ISTH NHF WFH 2021 guidelines on the management of von Willebrand disease

Author

Connell, Nathan T., Flood, Veronica H., Brignardello-Petersen, Romina, Abdul-Kadir, Rezan, Arapshian, Alice, Couper, Susie, Grow, Jean M., Kouides, Peter, Laffan, Michael, Lavin, Michelle, Leebeek, Frank W.G., O'Brien, Sarah H., Ozelo, Margareth C., Tosetto, Alberto, Weyand, Angela C., James, Paula D., Kalot, Mohamad A., Husainat, Nedaa, and Mustafa, Reem A.

Published
2021
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17. Type 1 VWD classification revisited - novel insights from combined analysis of the LoVIC and WiN studies

Author

Poli Van Creveldkliniek Medisch, Child Health, Atiq, Ferdows, Blok, Robin, van Kwawegen, Calvin, Doherty, Dearbhla, Lavin, Michelle, van der Bom, Johanna G, O'Connell, Niamh M, de Meris, Joke, Ryan, Kevin, Schols, Saskia E M, Byrne, Mary B, Heubel-Moenen, Floor C J I, van Galen, Karin P M, Preston, Roger J S, Cnossen, Marjon H, Fijnvandraat, Karin, Baker, Ross Ian, Meijer, Karina, James, Paula D, Di Paola, Jorge, Eikenboom, Jeroen C J, Leebeek, Frank W G, O'Donnell, James S, Poli Van Creveldkliniek Medisch, Child Health, Atiq, Ferdows, Blok, Robin, van Kwawegen, Calvin, Doherty, Dearbhla, Lavin, Michelle, van der Bom, Johanna G, O'Connell, Niamh M, de Meris, Joke, Ryan, Kevin, Schols, Saskia E M, Byrne, Mary B, Heubel-Moenen, Floor C J I, van Galen, Karin P M, Preston, Roger J S, Cnossen, Marjon H, Fijnvandraat, Karin, Baker, Ross Ian, Meijer, Karina, James, Paula D, Di Paola, Jorge, Eikenboom, Jeroen C J, Leebeek, Frank W G, and O'Donnell, James S

Published
2024

18. Illustrated State‐of‐the‐Art Capsules of the ISTH 2020 Congress

Author

Ariens, Robert, Becattini, Cecilia, Bender, Markus, Bergmeier, Wolfgang, Castoldi, Elisabetta, Devreese, Katrien, Ellis, Martin, Gailani, David, Ignjatovic, Vera, James, Paula D., Kerrigan, Steven, Lambert, Michele, Lee, Lai Heng, Levi, Marcel, Maugeri, Norma, Meijers, Joost, Melero‐Martin, Juan, Michelson, Alan D., Mingozzi, Federico, Neeves, Keith, Ni, Heyu, Olsson, Anna‐Karin, Prohászka, Zoltán, Ranson, Marie, Riva, Nicoletta, Senis, Yotis, van Ommen, Cornelia H., Vaughan, Douglas E., and Weisel, John

Published
2020
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20. Type 1 VWD classification revisited - novel insights from combined analysis of the LoVIC and WiN studies

Author

Atiq, Ferdows, primary, Blok, Robin, additional, van Kwawegen, Calvin, additional, Doherty, Dearbhla, additional, Lavin, Michelle, additional, van der Bom, Johanna G, additional, O'Connell, Niamh M, additional, de Meris, Joke, additional, Ryan, Kevin, additional, Schols, Saskia E.M., additional, Byrne, Mary B, additional, Heubel-Moenen, Floor C.J.I., additional, van Galen, Karin P.M., additional, Preston, Roger J.S., additional, Cnossen, Marjon H., additional, Fijnvandraat, Karin, additional, Baker, Ross Ian, additional, Meijer, Karina, additional, James, Paula D., additional, Di Paola, Jorge, additional, Eikenboom, Jeroen C.J., additional, Leebeek, Frank W.G., additional, and O'Donnell, James S., additional

Published
2023
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25. Special considerations in GI bleeding in VWD patients

Author

Chornenki, Nicholas L.J., Ocran, Edwin, and James, Paula D.

Subjects
von Willebrand Diseases, von Willebrand Factor, Humans, Hematology, Gastrointestinal Hemorrhage, Von Willebrand Disease, Angiodysplasia
Abstract

Gastrointestinal (GI) bleeding is an important cause of morbidity and mortality in von Willebrand disease (VWD). It has been noted that GI bleeding caused by angiodysplasia is overrepresented in VWD patients compared to other causes. The bleeding from angiodysplasia is notoriously difficult to treat; recurrences and rebleeds are common. A growing body of basic science evidence demonstrates that von Willebrand factor negatively regulates angiogenesis through multiple pathways. VWD is clinically highly associated with angiodysplasia. The predisposition to angiodysplasia likely accounts for many of the clinical difficulties related to managing GI bleeding in VWD patients. Diagnosis and treatment are challenging with the current tools available, and much further research is needed to further optimize care for these patients with regard to acute treatment, prophylaxis, and adjunctive therapies. In this review we provide an overview of the available literature on GI bleeding in VWD and explore the molecular underpinnings of angiodysplasia-related GI bleeding. Considerations for diagnostic effectiveness are discussed, as well as the natural history and recurrence of these lesions and which therapeutic options are available for acute and prophylactic management.

Published
2022

30. The endothelial cell receptor stabilin-2 regulates VWF-FVIII complex half-life and immunogenicity

Author

Swystun, Laura L., Lai, Jesse D., Notley, Colleen, Georgescu, Ilinca, Paine, A. Simonne, Mewburn, Jeff, Nesbitt, Kate, Schledzewski, Kai, Geraud, Cyrill, Kzhyshkowska, Julia, Goerdt, Sergij, Hopman, Wilma, Montgomery, Robert R., James, Paula D., and Lillicrap, David

Subjects
Cell receptors -- Health aspects, Endothelium -- Genetic aspects -- Health aspects, Von Willebrand factor -- Health aspects, Immune response -- Genetic aspects, Health care industry
Abstract

Quantitative abnormalities of the von Willebrand factor-factor VIII (VWF-FVIII) complex associate with inherited bleeding or thrombotic disorders. Receptor-mediated interactions between plasma VWF-FVIII and phagocytic or immune cells can influence their hemostatic and immunogenic activities. Genetic association studies have demonstrated that variants in the STAB2 gene, which encodes the scavenger receptor stabilin-2, associate with plasma levels of VWF-FVIII. However, the mechanistic basis and pathophysiological consequences of this association are unknown. We have demonstrated that stabilin-2-expressing cells bind and internalize human VWF and FVIII in a VWF- dependent manner, and stabilin-2- deficient mice displayed prolonged human VWF-FVIII half-life compared with controls. The stabilin-2 variant p.E2377K significantly decreased stabilin-2 expression and impaired VWF endocytosis in a heterologous expression system, and common STAB2 variants associated with plasma VWF levels in type 1 von Willebrand disease patients. STAB2-deficient mice displayed a decreased immunogenic response to human VWF-FVIII complex, while coinfusion of human VWF-FVIII with the stabilin-2 ligand hyaluronic acid attenuated the immune response to exogenous FVIII. Collectively, these data suggest that stabilin-2 functions as both a clearance and an immunoregulatory receptor for VWF-FVIII, making stabilin-2 a novel molecular target for modification of the half-life of VWF-FVIII and the immune response to VWF-FVIII concentrates., Introduction The coagulation factors von Willebrand factor (VWF) and factor VIII (FVIII) circulate in the plasma as a noncovalent complex, with VWF serving as a carrier to protect FVIII from [...]

Published
2018
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31. Von Willebrand disease: Gaining a global perspective

Author

O'Sullivan, Jamie M., primary, Tootoonchian, Ellia, additional, Ziemele, Baiba, additional, Makris, Michael, additional, Federici, Augusto B., additional, Khayat Djambas, Claudia, additional, El Ekiaby, Magdy, additional, Rotellini, Dawn, additional, Sidonio, Robert F., additional, Iorio, Alfonso, additional, Coffin, Donna, additional, Pierce, Glenn F., additional, Stonebraker, Jeffrey, additional, James, Paula D., additional, and Lavin, Michelle, additional

Published
2023
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33. Clinical and laboratory variability in a cohort of patients diagnosed with type 1 VWD in the United States

Author

Flood, Veronica H., Christopherson, Pamela A., Gill, Joan Cox, Friedman, Kenneth D., Haberichter, Sandra L., Bellissimo, Daniel B., Udani, Rupa A., Dasgupta, Mahua, Hoffmann, Raymond G., Ragni, Margaret V., Shapiro, Amy D., Lusher, Jeanne M., Lentz, Steven R., Abshire, Thomas C., Leissinger, Cindy, Hoots, W. Keith, Manco-Johnson, Marilyn J., Gruppo, Ralph A., Boggio, Lisa N., Montgomery, Kate T., Goodeve, Anne C., James, Paula D., Lillicrap, David, Peake, Ian R., and Montgomery, Robert R.

Published
2016
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37. Enhanced VWF clearance in low VWF pathogenesis: limitations of the VWFpp/VWF:Ag ratio and clinical significance

Author

Doherty, Dearbhla, primary, Lavin, Michelle, additional, Byrne, Mary, additional, Nolan, Margaret, additional, O’Sullivan, Jamie M., additional, Ryan, Kevin, additional, O’Connell, Niamh M., additional, Haberichter, Sandra L., additional, Christopherson, Pamela A., additional, Di Paola, Jorge, additional, James, Paula D., additional, and O’Donnell, James S., additional

Published
2023
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40. The Empower Study - Design of a Randomized Clinical Trial to Assess the Efficacy and Safety of a Plasma-Derived Von Willebrand Factor / Factor VIII Concentrate for Heavy Menstrual Bleeding in Women with Von Willebrand Disease

Author

Sholzberg, Michelle, primary, Tang, Grace H, additional, James, Paula D., additional, Meffe, Filomena, additional, Rimmer, Emily, additional, Jackson, Shannon, additional, Sun, Haowei Linda, additional, Robinson, Sue, additional, Floros, Georgina, additional, Aratia, Shamshah, additional, Jüni, Peter, additional, and da Costa, Bruno R., additional

Published
2022
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41. Take Control. Period. -Interactive Patient Education Website Enhances Adherence to Tranexamic Acid and Iron Supplementation in Women with Heavy Menstrual Bleeding

Author

Norman, Savannah, primary, Ling, Ying, additional, Filomena, Meffe, additional, Aratia, Shamshah, additional, Robertson, Deborah, additional, Jiwajee, Aziz, additional, Tang, Grace, additional, James, Paula D., additional, Mosko, Jeff, additional, and Sholzberg, Michelle, additional

Published
2022
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46. Surgical management of patients with von Willebrand disease:summary of 2 systematic reviews of the literature

Author

Brignardello-Petersen, Romina, Alayli, Abdallah El, Husainat, Nedaa, Kalot, Mohamad, Shahid, Shaneela, Aljabirii, Yazan, Britt, Alec, Alturkmani, Hani, El-Khechen, Hussein, Motaghi, Shahrzad, Roller, John, Dimassi, Ahmad, Abughanimeh, Omar, Madoukh, Bader, Arapshian, Alice, Grow, Jean M., Kouides, Peter, Laffan, Michael, Leebeek, Frank W.G., O’Brien, Sarah H., Tosetto, Alberto, James, Paula D., Connell, Nathan T., Flood, Veronica, Mustafa, Reem A., Brignardello-Petersen, Romina, Alayli, Abdallah El, Husainat, Nedaa, Kalot, Mohamad, Shahid, Shaneela, Aljabirii, Yazan, Britt, Alec, Alturkmani, Hani, El-Khechen, Hussein, Motaghi, Shahrzad, Roller, John, Dimassi, Ahmad, Abughanimeh, Omar, Madoukh, Bader, Arapshian, Alice, Grow, Jean M., Kouides, Peter, Laffan, Michael, Leebeek, Frank W.G., O’Brien, Sarah H., Tosetto, Alberto, James, Paula D., Connell, Nathan T., Flood, Veronica, and Mustafa, Reem A.

Abstract

von Willebrand disease (VWD) is the most common inherited bleeding disorder. The management of patients with VWD who are undergoing surgeries is crucial to prevent bleeding complications. We systematically summarized the evidence on the management of patients with VWD who are undergoing major and minor surgeries to support the development of practice guidelines. We searched Medline and EMBASE from inception through October 2019 for randomized clinical trials (RCTs), comparative observational studies, and case series that compared maintaining factor VIII (FVIII) levels or von Willebrand factor (VWF) levels at .0.50 IU/mL for at least 3 days in patients undergoing major surgery, and those with options for perioperative management of patients undergoing minor surgery. Two authors screened and abstracted data and assessed the risk of bias. We conducted meta-analyses when possible. We evaluated the certainty of the evidence using the Grading of Recommendations, Assessment, Development and Evaluations (GRADE) approach. We included 7 case series for major surgeries and 2 RCTs and 12 case series for minor surgeries. Very-low-certainty evidence showed that maintaining FVIII levels or VWF levels of .0.50 IU/mL for at least 3 consecutive days showed excellent hemostatic efficacy (as labeled by the researchers) after 74% to 100% of major surgeries. Low- to very-low-certainty evidence showed that prescribing tranexamic acid and increasing VWF levels to 0.50 IU/mL resulted in fewer bleeding complications after minor procedures compared with increasing VWF levels to 0.50 IU/mL alone. Given the low-quality evidence for guiding management decisions, a shared-decision model leading to individualized therapy plans will be important in patients with VWD who are undergoing surgical and invasive procedures.

Published
2022

49. Randomized trials of therapeutic heparin for COVID‐19: A meta‐analysis

Author

Sholzberg, Michelle, da Costa, Bruno R., Tang, Grace H., Rahhal, Hassan, AlHamzah, Musaad, Baumann Kreuziger, Lisa, Ní Áinle, Fionnuala, Almarshoodi, Mozah Obaid, James, Paula D., Lillicrap, David, Carrier, Marc, Beckett, Andrew, Fralick, Michael, Middeldorp, Saskia, Lee, Agnes Y.Y., Thorpe, Kevin E., Negri, Elnara Márcia, Cushman, Mary, and Jüni, Peter

Published
2021
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50. von Willebrand factor levels in the diagnosis of von Willebrand disease: a systematic review and meta-analysis

Author

Kalot, Mohamad A., primary, Husainat, Nedaa, additional, El Alayli, Abdallah, additional, Abughanimeh, Omar, additional, Diab, Osama, additional, Tayiem, Sammy, additional, Madoukh, Bader, additional, Dimassi, Ahmad B., additional, Qureini, Aref, additional, Ameer, Barbara, additional, Eikenboom, Jeroen C.J., additional, Giraud, Nicolas, additional, McLintock, Claire, additional, McRae, Simon, additional, Montgomery, Robert R., additional, O’Donnell, James S., additional, Scappe, Nikole, additional, Sidonio, Robert F., additional, Brignardello-Petersen, Romina, additional, Flood, Veronica H., additional, Connell, Nathan T., additional, James, Paula D., additional, and Mustafa, Reem A., additional

Published
2021
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