DDAVP treatment in a child with von Willebrand disease type 2M

Mauz‐Körholz, Christine; Budde, Ulrich; Körholz, Dieter; Göbel, U.

doi:10.1007/pl00014349

Cited by 7 publications

(5 citation statements)

References 12 publications

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“…To summarize from earlier, as an optimized assay system and compared with vWF:RCof, the vWF:CBA (1) can better distinguish HMW multimer-deficient vWD subtypes (i.e., types 2A and 2B) from type 1 vWD, (2) is technically similar to that of a vWF:Ag ELISA, so it can be performed in parallel with little additional effort, (3) can better detect the absence of vWF in type 3 vWD and the presence of very low levels of vWF in severe type 1 vWD. In addition, the vWF:CBA is a better marker of DDAVP responsiveness than vWF:RCof is, 44,54,[73][74][75][76] and its usefulness in animal vWD diagnosis and treatment is also emerging. 76,77 The use of vWF:CBA in various disease states in addition to congenital vWD is now recognized.…”

Section: Benefits and Limitations Of The Vwf:cbamentioning

confidence: 99%

“…Even in type 2M vWD, most reported studies to date essentially concur that vWF:CBA is also reduced in parallel to vWF:RCof in most cases. 16,37,74,91,92 Given the great benefits of the vWF:CBA and the wealth of current knowledge already noted, it has to be asked: is it time to stop performing vWF:RCof and vWF:Multimers? The simple answer is: not just yet.…”

Section: Vwf:cba: a Replacement For Vwf:rcof And Vwf:multimersmentioning

confidence: 99%

See 1 more Smart Citation

von Willebrand Factor Collagen-Binding (Activity) Assay in the Diagnosis of von Willebrand Disease: A 15-Year Journey

Favaloro¹

2002

Semin Thromb Hemost

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The correct diagnosis and classification of von Willebrand disease (vWD) is crucial because the presenting biological activity of von Willebrand factor (vWF) determines both the hemorrhagic risk and the subsequent clinical management. A variety of laboratory assays may be employed, not necessarily restricted to assessments of vWF. This article discusses the relative strengths and limitations of various functional or discriminatory vWF assays with a special focus on the vWF:collagen-binding activity (vWF:CBA) assay. This is a functional vWF assay that relies on the property of vWF adhesion to collagen. The vWF:CBA was first described approximately 15 years ago. The journey from that time point has been an interesting one, and the vWF:CBA is now gaining more widespread acceptance. There are now many published studies confirming the superiority of the vWF:CBA over the vWF ristocetin cofactor (vWF:RCof) activity as a functional screening diagnostic test process for vWD. However, both tests may be required in order to appropriately diagnose all forms of vWD. The relationship of these assays with multimer analysis is also discussed. In summary, an optimized vWF:CBA detects primarily high-molecular-weight (HMW) vWF forms and probably only about 30% of the total plasma vWF pool detected by vWF antigen (vWF:Ag). Because these HMW vWF forms are missing in types 2A and 2B vWD, the vWF:CBA is extremely useful in the detection of these qualitative vWD subtypes. In addition, however, concordance of vWF:CBA with vWF:Ag in unison with low vWF levels may alternatively suggest a type 1 vWD, and an absence of both vWF:Ag and vWF:CBA will suggest type 3 vWD. The vWF:CBA is also being investigated in various disease states, as is its usefulness as an effective marker of functional HMW vWF in both desmopressin (DDAVP) and factor-concentrate therapy in vWD.

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Section: Benefits and Limitations Of The Vwf:cbamentioning

confidence: 99%

Section: Vwf:cba: a Replacement For Vwf:rcof And Vwf:multimersmentioning

confidence: 99%

von Willebrand Factor Collagen-Binding (Activity) Assay in the Diagnosis of von Willebrand Disease: A 15-Year Journey

Favaloro¹

2002

Semin Thromb Hemost

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“…In most patients with the classic IIA phenotype, DDAVP increases the FVIII activity, without beneficial effects on the primary hemostatic system. On the other hand, patients with other subtypes of type 2A and patients with type 2M may respond very well (123,163). A DDAVP challenge test should therefore be performed in these patients before allocating them to treatment with blood products.…”

Section: Therapy Of Congenital Von Willebrand Diseasementioning

confidence: 99%

VON WILLEBRAND FACTORANDVON WILLEBRAND DISEASE

Budde¹,

Schneppenheim²

2001

Rev Clin Exp Hematol

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von Willebrand disease (vWD) is caused by quantitative and/or qualitative defects of the von Willebrand factor (vWF), a multimeric high molecular weight glycoprotein. Typically, it affects the primary hemostatic system, which results in a mucocutaneous bleeding tendency simulating a platelet function defect. The vWF promotes its function in two ways: (i) by initiating platelet adhesion to the injured vessel wall under conditions of high shear forces, and (ii) by its carrier function for factor VIII in plasma. Accumulating knowledge of the different clinical phenotypes and the pathophysiological basis of the disease translated into a classification that differentiated between quantitative and qualitative defects by means of quantitative and functional parameters, and by analyzing the electrophoretic pattern of vWF multimers. The advent of molecular techniques provided the opportunity for conducting genotype-phenotype studies which have recently helped, not only to elucidate or confirm important functions of vWF and its steps in post-translational processing, but also many disease causing defects. Acquired von Willebrand syndrome (avWS) has gained more attention during the recent years. An international registry was published and recommendation by the Scientific and Standardization Committee of the International Society on Thrombosis and Haemostasis in 2000. It concluded that avWS, although not a frequent disease, is nevertheless probably underdiagnosed. This should be addressed in future prospective studies. The aim of treatment is the correction of the impaired hemostatic system of the patient, ideally including the defects of both primary and secondary hemostasis. Desmopressin is the treatment of choice in about 70% of patients, mostly with type 1, while the others merit treatment with concentrates containing vWF.

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“…Mauz-Körholz et al 63 used a VWF:CB, along with VWF:RCo, VWF:Ag, and FVIII:C to explore DDAVP treatment in a child with 2M VWD, with good increments in all 2 hours post-DDAVP, and concluding that in some type 2M VWD cases, DDAVP administration might be an effective treatment in cases of elective surgery, dispensing with VWF replacement by pooled blood products. Kertzscher et al 64 explored the potential utility of the VWF:CB to characterize platelet VWF content in 24 patients with various forms of VWD.…”

Section: A History Of the Von Willebrand Factor Collagen-binding Assaymentioning

confidence: 99%

The Role of the von Willebrand Factor Collagen-Binding Assay (VWF:CB) in the Diagnosis and Treatment of von Willebrand Disease (VWD) and Way Beyond: A Comprehensive 36-Year History

Favaloro

2023

Semin Thromb Hemost

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The von Willebrand factor (VWF) collagen binding (VWF:CB) assay was first reported for use in von Willebrand diagnostics in 1986, by Brown and Bosak. Since then, the VWF:CB has continued to be used to help diagnose von Willebrand disease (VWD) (correctly) and also to help assign the correct subtype, as well as to assist in the monitoring of VWD therapy, especially desmopressin (DDAVP). However, it is important to recognize that the specific value of any VWF:CB is predicated on the use of an optimized VWF:CB, and that not all VWF:CB assays are so optimized. There are some good commercial assays available, but there are also some “not-so-good” commercial assays available, and these may continue to give the VWF:CB “a bad reputation.” In addition to VWD diagnosis and management, the VWF:CB found purpose in a variety of other applications, from assessing ADAMTS13 activity, to investigation into acquired von Willebrand syndrome (especially as associated with use of mechanical circulatory support or cardiac assist devices), to assessment of VWF activity in disease states in where an excess of high-molecular-weight VWF may accumulate, and lead to increased (micro)thrombosis risk (e.g., coronavirus disease 2019, thrombotic thrombocytopenic purpura). The VWF:CB turns 37 in 2023. This review is a celebration of the utility of the VWF:CB over this nearly 40-year history.

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DDAVP treatment in a child with von Willebrand disease type 2M

Abstract: In conclusion, type 2M vWD might be effectively treated with DDAVP administration in cases of elective surgery, dispensing with vWF replacement by pooled blood products.

Cited by 7 publications

References 12 publications

von Willebrand Factor Collagen-Binding (Activity) Assay in the Diagnosis of von Willebrand Disease: A 15-Year Journey

von Willebrand Factor Collagen-Binding (Activity) Assay in the Diagnosis of von Willebrand Disease: A 15-Year Journey

VON WILLEBRAND FACTORANDVON WILLEBRAND DISEASE

The Role of the von Willebrand Factor Collagen-Binding Assay (VWF:CB) in the Diagnosis and Treatment of von Willebrand Disease (VWD) and Way Beyond: A Comprehensive 36-Year History

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