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Article ; Online: Diagnosis and treatment of von Willebrand disease in 2024 and beyond.

James, Paula / Leebeek, Frank / Casari, Caterina / Lillicrap, David

Haemophilia : the official journal of the World Federation of Hemophilia

2024 Volume 30 Suppl 3, Page(s) 103–111

Abstract: Manuscript background and aim: The diagnosis and clinical care of patients with von Willebrand disease (VWD) has continued to evolve since the characterization of the von Willebrand factor (VWF) gene in 1985. This condition is almost certainly the most ... ...

Abstract	Manuscript background and aim: The diagnosis and clinical care of patients with von Willebrand disease (VWD) has continued to evolve since the characterization of the von Willebrand factor (VWF) gene in 1985. This condition is almost certainly the most common inherited bleeding disorder, and the major symptomatic burden of the disease is experienced by females during their reproductive years. Diagnosis relies on the identification of a personal and family history of excessive mucocutaneous bleeding, and laboratory features consistent with quantitative and/or qualitative abnormalities of VWF. This review focuses on three aspects of VWD management, with current updates and a look into the future. Manuscript themes: First, we will address the role of genetics in the diagnosis and possible therapies for VWD. With current technologies, VWD genetic diagnosis is usually confined to the confirmation of type 2 subtypes of the disease and type 3 VWD analysis for family planning. While type 3 VWD is a potential candidate for the application of gene therapy, no treatments are currently close to entering the clinic. Second, the peri-procedural management of patients with VWD remains an important element of care. The choice of product, its dose and schedule all require careful consideration depending upon the type and disruptive nature of the planned procedure. Lastly, in addition to gene therapy, several other novel therapeutic interventions are also being developed for bleeding and prophylaxis in VWD. These include a VWF aptamer interfering with VWF clearance and bioengineered forms of VWF.
MeSH term(s)	Female ; Humans ; von Willebrand Diseases/diagnosis ; von Willebrand Diseases/genetics ; von Willebrand Diseases/therapy ; von Willebrand Factor/genetics ; von Willebrand Factor/therapeutic use ; von Willebrand Disease, Type 3 ; Hemorrhage/diagnosis
Chemical Substances	von Willebrand Factor
Language	English
Publishing date	2024-03-13
Publishing country	England
Document type	Review ; Journal Article
ZDB-ID	1229713-6
ISSN	1365-2516 ; 1351-8216 ; 1355-0691
ISSN (online)	1365-2516
ISSN	1351-8216 ; 1355-0691
DOI	10.1111/hae.14970
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Zs.A 4249: Show issues			Location: Je nach Verfügbarkeit (siehe Angabe bei Bestand) bis Jg. 1994: Bestellungen von Artikeln über das Online-Bestellformular Jg. 1995 - 2021: Lesesall (2.OG) ab Jg. 2022: Lesesaal (EG)
Zs.MO 450: Show issues

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Article ; Online: New and emerging therapies for women, girls, and people with the potential to menstruate with VWD.

Casari, Caterina / Leung, Jennifer / James, Paula D

Blood advances

2023 Volume 7, Issue 24, Page(s) 7501–7505

Abstract: Innovation in therapies for patients with von Willebrand disease (VWD) has lagged far behind that for hemophilia, creating inequity in the bleeding disorder community. Although currently existing treatments of antifibrinolytics, desmopressin, and plasma- ... ...

Abstract	Innovation in therapies for patients with von Willebrand disease (VWD) has lagged far behind that for hemophilia, creating inequity in the bleeding disorder community. Although currently existing treatments of antifibrinolytics, desmopressin, and plasma-derived von Willebrand factor replacement are considered effective, multiple studies report poor quality of life in patients with VWD, especially those with heavy menstrual bleeding (HMB). This disconnect underscores the need for novel therapies that are safe and effective and that consider a patient's specific contraceptive and reproductive needs. Recombinant von Willebrand factor is the most recent new therapy for VWD; the data specific to women are reviewed. We also present emerging data on emicizumab for the treatment of VWD, BT200 (rondoraptivon pegol), generalized hemostatic therapies (VGA039 and HMB-011), as well as treatments based on nanotechnology (platelet-inspired nanoparticles and KB-V13A12). We are optimistic as we move toward pivotal clinical trials for these elegant and innovative treatments.
MeSH term(s)	Humans ; Female ; von Willebrand Diseases/drug therapy ; von Willebrand Factor/therapeutic use ; Quality of Life ; Menorrhagia ; Antifibrinolytic Agents/therapeutic use
Chemical Substances	von Willebrand Factor ; Antifibrinolytic Agents
Language	English
Publishing date	2023-11-14
Publishing country	United States
Document type	Journal Article
ZDB-ID	2915908-8
ISSN	2473-9537 ; 2473-9529
ISSN (online)	2473-9537
ISSN	2473-9529
DOI	10.1182/bloodadvances.2023010716
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Article ; Online: von Willebrand factor: from figurant to main character in the scene of inflammation.

Lenting, Peter J / Texier, Alexis / Casari, Caterina

Journal of thrombosis and haemostasis : JTH

2023 Volume 21, Issue 4, Page(s) 710–713

MeSH term(s)	Humans ; von Willebrand Factor ; Inflammation ; von Willebrand Diseases ; Factor VIII
Chemical Substances	von Willebrand Factor ; Factor VIII (9001-27-8)
Language	English
Publishing date	2023-01-21
Publishing country	England
Document type	Journal Article ; Research Support, Non-U.S. Gov't
ZDB-ID	2112661-6
ISSN	1538-7836 ; 1538-7933
ISSN (online)	1538-7836
ISSN	1538-7933
DOI	10.1016/j.jtha.2023.01.014
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Article ; Online: Towards novel treatment options in von Willebrand disease.

Lenting, Peter J / Kizlik-Manson, Claire / Casari, Caterina

Haemophilia : the official journal of the World Federation of Hemophilia

2021 Volume 28 Suppl 4, Page(s) 5–10

Abstract: Deficiency or dysfunction of von Willebrand factor (VWF) is associated with a bleeding disorder known as von Willebrand disease (VWD). The clinical manifestations of VWD are heterogeneous, and are in part dictated by the structural or functional defects ... ...

Abstract	Deficiency or dysfunction of von Willebrand factor (VWF) is associated with a bleeding disorder known as von Willebrand disease (VWD). The clinical manifestations of VWD are heterogeneous, and are in part dictated by the structural or functional defects of VWF. The tools to control bleeding in VWD are dominated by VWF concentrates, desmopressin and antifibrinolytic therapy. In view of these treatments being considered as effective, it is surprising that quality-of-life studies consistently demonstrate a significant mental and physical burden in VWD patients, particularly in women. Apparently, the current weaponry to support the management of VWD is insufficient to fully address the needs of the patients. It is important therefore to continue to search for innovative treatment options which could better serve the VWD patients. In this short review, two of such options are discussed in more detail: emicizumab to correct for the deficiency of factor VIII (FVIII), and the pegylated aptamer BT200 to increase endogenous levels of the VWF/FVIII complex.
MeSH term(s)	Antifibrinolytic Agents/therapeutic use ; Deamino Arginine Vasopressin/therapeutic use ; Factor VIII/therapeutic use ; Female ; Hemorrhage/drug therapy ; Hemostatics/therapeutic use ; Humans ; von Willebrand Diseases/drug therapy ; von Willebrand Factor/therapeutic use
Chemical Substances	Antifibrinolytic Agents ; Hemostatics ; von Willebrand Factor ; Factor VIII (9001-27-8) ; Deamino Arginine Vasopressin (ENR1LLB0FP)
Language	English
Publishing date	2021-02-09
Publishing country	England
Document type	Journal Article
ZDB-ID	1229713-6
ISSN	1365-2516 ; 1351-8216 ; 1355-0691
ISSN (online)	1365-2516
ISSN	1351-8216 ; 1355-0691
DOI	10.1111/hae.14518
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Zs.A 4249: Show issues			Location: Je nach Verfügbarkeit (siehe Angabe bei Bestand) bis Jg. 1994: Bestellungen von Artikeln über das Online-Bestellformular Jg. 1995 - 2021: Lesesall (2.OG) ab Jg. 2022: Lesesaal (EG)
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Article ; Online: Fitusiran reduces bleeding in Factor X-deficient mice.

Verhenne, Sebastien / McCluskey, Genevieve / Maynadié, Hortense / Adam, Frédéric / Casari, Caterina / Panicot-Dubois, Laurence / Crescence, Lydie / Dubois, Christophe / Denis, Cecile V / Lenting, Peter J / Christophe, Olivier D

Blood

2024

Abstract: Factor X (FX)-deficiency is a rare bleeding disorder manifesting a bleeding tendency caused by low FX activity levels. We aimed to explore the use of fitusiran (an investigational siRNA that silences antithrombin expression) to increase thrombin ... ...

Abstract	Factor X (FX)-deficiency is a rare bleeding disorder manifesting a bleeding tendency caused by low FX activity levels. We aimed to explore the use of fitusiran (an investigational siRNA that silences antithrombin expression) to increase thrombin generation and the in vivo hemostatic potential under conditions of FX-deficiency. We therefore developed a novel model of inducible FX-deficiency, generating mice expressing <1% FX activity and antigen (f10low-mice). Compared to control f10WT-mice, f10low-mice had 6- and 4-fold prolonged clotting times in Prothrombin Time- and activated Partial Prothrombin Time-assays, respectively (p<0.001). Thrombin generation was severely reduced, irrespective whether tissue factor or factor XIa was used as initiator. In vivo analysis revealed near-absent thrombus formation in a laser-induced vessel injury-model. Furthermore, in two distinct bleeding models, f10low-mice displayed an increased bleeding tendency compared to f10WT-mice. In the tail-clip assay blood loss was increased from 12±16 microliter to 590±335 microliter (p<0.0001). In the saphenous vein puncture (SVP)-model, the number of clots generated was reduced from 19±5 clots/30 min for f10WT-mice to 2±2 clots/30 min (p<0.0001) for f10low-mice. In both models, bleeding was corrected upon infusion of purified FX. Treatment of f10low-mice with fitusiran (2x10 mg/kg at one-week interval) resulted in 17±6% residual antithrombin activity and increased thrombin generation (4-fold and 2-3-fold increase in endogenous thrombin potential and thrombin peak, respectively). In the SVP-model, the number of clots was increased to 8±6 clots/30 min (p=0.0029). Altogether, we demonstrate that reduction of antithrombin levels is associated with improved hemostatic activity under conditions of FX-deficiency.
Language	English
Publishing date	2024-04-15
Publishing country	United States
Document type	Journal Article
ZDB-ID	80069-7
ISSN	1528-0020 ; 0006-4971
ISSN (online)	1528-0020
ISSN	0006-4971
DOI	10.1182/blood.2023023404
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Article ; Online: Exciting times for the ISTH early career professionals.

Kulkarni, Ketan / Chiasakul, Thita / Riva, Nicoletta / Eslick, Renée / Casari, Caterina

Journal of thrombosis and haemostasis : JTH

2020 Volume 18, Issue 10, Page(s) 2437–2438

Language	English
Publishing date	2020-10-29
Publishing country	England
Document type	Editorial
ZDB-ID	2112661-6
ISSN	1538-7836 ; 1538-7933
ISSN (online)	1538-7836
ISSN	1538-7933
DOI	10.1111/jth.15070
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Article ; Online: Acquired platelet disorders.

Casari, Caterina / Bergmeier, Wolfgang

Thrombosis research

2016 Volume 141 Suppl 2, Page(s) S73–5

Abstract: In contrast to congenital platelet disorders, which are rare, acquired platelet dysfunctions are more common in clinical practice. Their main causes are medications and systemic/hematologic diseases. Typical clinical manifestations are mucosal bleeding, ... ...

Abstract	In contrast to congenital platelet disorders, which are rare, acquired platelet dysfunctions are more common in clinical practice. Their main causes are medications and systemic/hematologic diseases. Typical clinical manifestations are mucosal bleeding, epistaxis, or superficial epidermal bleeding normally of modest entity. In most cases, the molecular mechanisms underlying impaired platelet function are not fully established, making it difficult to optimize patient care. We here provide a short overview of the various forms of acquired platelet disorders, with a particular focus on recent mechanistic studies on platelet dysfunction in von Willebrand disease.
MeSH term(s)	Animals ; Blood Platelet Disorders/blood ; Blood Platelet Disorders/complications ; Blood Platelet Disorders/etiology ; Blood Platelet Disorders/metabolism ; Blood Platelets/metabolism ; Blood Platelets/pathology ; Hemostasis ; Humans ; Liver Diseases/blood ; Liver Diseases/complications ; Liver Diseases/metabolism ; Myeloproliferative Disorders/blood ; Myeloproliferative Disorders/complications ; Myeloproliferative Disorders/metabolism ; Uremia/blood ; Uremia/complications ; Uremia/metabolism ; von Willebrand Diseases/blood ; von Willebrand Diseases/complications ; von Willebrand Diseases/metabolism
Language	English
Publishing date	2016-05
Publishing country	United States
Document type	Journal Article ; Review
ZDB-ID	121852-9
ISSN	1879-2472 ; 0049-3848
ISSN (online)	1879-2472
ISSN	0049-3848
DOI	10.1016/S0049-3848(16)30371-1
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Article ; Online: Impact of the COVID-19 pandemic on education and clinical training.

Al Moosawi, Muntadhar / Iding, Aaron / Armstrong, Paul / Chiasakul, Thita / Campbell, Robert / Casari, Caterina

Journal of thrombosis and haemostasis : JTH

2021 Volume 19, Issue 9, Page(s) 2099–2100

MeSH term(s)	COVID-19 ; Disease Outbreaks ; Humans ; Influenza A Virus, H1N1 Subtype ; Pandemics ; SARS-CoV-2
Language	English
Publishing date	2021-03-05
Publishing country	England
Document type	Editorial
ZDB-ID	2112661-6
ISSN	1538-7836 ; 1538-7933
ISSN (online)	1538-7836
ISSN	1538-7933
DOI	10.1111/jth.15470
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Article: A thrombopoietin receptor agonist to rescue an unusual platelet transfusion-induced reaction in a p.V1316M-associated von Willebrand disease type 2B patient.

Casari, Caterina / Favier, Remi / Legendre, Paulette / Kauskot, Alexandre / Adam, Frederic / Picard, Veronique / Lenting, Peter T / Denis, Cecile V / Proulle, Valerie

Therapeutic advances in hematology

2022 Volume 13, Page(s) 20406207221076812

Abstract: This report describes the first case of splenic injury in a patient with p.V1316M-associated von Willebrand disease type 2B (VWD2B) with chronic thrombocytopenia, successfully treated with nonoperative management including von Willebrand factor (VWF) ... ...

Abstract	This report describes the first case of splenic injury in a patient with p.V1316M-associated von Willebrand disease type 2B (VWD2B) with chronic thrombocytopenia, successfully treated with nonoperative management including von Willebrand factor (VWF) replacement therapy, and platelet transfusions relayed by a thrombopoietin receptor agonist (TPO-RA, Eltrombopag). Eltrombopag was initially introduced to rescue an unusual post-platelet-transfusion reaction exacerbating the thrombocytopenia. In-depth analysis of the dramatic platelet count drop and VWF measurements timeline ruled out an allo-immune reaction and supported an alternative hypothesis of a sudden platelet clearance as a consequence of stress-induced release of abnormal VWF. One year later, a second life-threatening bleeding episode required urgent surgery successfully managed with VWF replacement therapy and platelet transfusions. Eltrombopag was further introduced in the post-surgery period to allow bleeding-free and platelet-transfusion-free successful recovery. Treatment decisions are particularly challenging in patients with VWD2B, and this case highlights how such decisions can benefit from understanding the molecular origin of platelet count fluctuations observed in these patients. Here, we successfully used a new therapeutic approach combining VWF-replacement therapy and initial platelet-transfusion relayed by TPO-RA to optimize patient management. Plain language summary: A combination of von Willebrand factor replacement and thrombopoietin receptor agonist in thrombocytopenic patients with von Willebrand disease type 2B: a new therapy approach to optimize patient management?Therapeutic management of patients with von Willebrand disease type 2B are particularly challenging in case of severe thrombocytopenia.Treatment includes von Willebrands factor replacement therapy and iterative platelet transfusions.We describe the first case of splenic injury in a patient with p.V1316M-associated von Willebrand disease type 2B successfully treated with nonoperative management including von Willebrand factor replacement therapy and platelet transfusions relayed by a thrombopoietin receptor agonist.We showed that the unusual post-platelet-transfusion reaction associated with a dramatic platelet count drop was a consequence of stress-induced release of abnormal von Willebrand factor.The combination of von Willebrand factor replacement therapy and thrombopoietin receptor agonist may offer a new therapeutic approach to optimize patient management.
Language	English
Publishing date	2022-02-16
Publishing country	England
Document type	Journal Article
ZDB-ID	2585183-4
ISSN	2040-6215 ; 2040-6207
ISSN (online)	2040-6215
ISSN	2040-6207
DOI	10.1177/20406207221076812
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Article ; Online: Differences in venous clot structures between hemophilic mice treated with emicizumab versus factor VIII or factor VIIIFc.

Sefiane, Thibaud / Maynadié, Hortense / Ettingshausen, Carmen Escurola / Muczynski, Vincent / Heiligenstein, Xavier / Dumont, Julien / Christophe, Olivier D / Denis, Cécile V / Casari, Caterina / Lenting, Peter J

Haematologica

2023

Abstract: Recombinant factor VIII (rFVIII), rFVIIIFc and emicizumab are established treatment options in the management of hemophilia A. Each has its unique mode of action, which can influence thrombin generation kinetics and therefore also the kinetics of ... ...

Abstract	Recombinant factor VIII (rFVIII), rFVIIIFc and emicizumab are established treatment options in the management of hemophilia A. Each has its unique mode of action, which can influence thrombin generation kinetics and therefore also the kinetics of thrombin substrates. Such differences may potentially result in clots with different structural and physical properties. A starting observation of incomplete wound closure in a patient on emicizumab-prophylaxis led us employ a relevant mouse model in which we noticed that emicizumab-induced clots appeared less stable compared to FVIII-induced clots. We thus analyzed fibrin formation in vitro and in vivo. In vitro fibrin formation was faster and more abundant in the presence of emicizumab compared to rFVIII/rFVIIIFc. Furthermore, the time-interval between the initiation of fibrin formation and factor XIII activation was twice as long for emicizumab compared to rFVIII/rFVIIIFc. Scanning-electron microscopy and immunofluorescent spinning-disk confocal-microscopy of in vivo generated clots confirmed increased fibrin formation in the presence of emicizumab. Unexpectedly, we also detected a different morphology between rFVIII/rFVIIIFc- and emicizumab-induced clots. Contrary to the regular fibrin-mesh obtained with rFVIII/rFVIIIFc, fibrin-fibers appeared to be fused into large patches upon emicizumabtreatment. Moreover, fewer red blood cells were detected in regions where these fibrin patches were present. The presence of highly-dense fibrin-structures associated with a diffuse fiber-structure in emicizumab-induced clots was also observed when using superresolution imaging. We hypothesize that the modified kinetics of thrombin, fibrin and factor XIIIa generation contribute to differences in structural and physical properties between clots formed in the presence of FVIII or emicizumab.
Language	English
Publishing date	2023-12-07
Publishing country	Italy
Document type	Journal Article
ZDB-ID	2333-4
ISSN	1592-8721 ; 0017-6567 ; 0390-6078
ISSN (online)	1592-8721
ISSN	0017-6567 ; 0390-6078
DOI	10.3324/haematol.2023.284142
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