A better approach to monitoring of therapy in von Willebrand disease?

Favaloro, Emmanuel J.

doi:10.1160/th08-07-0449

Cited by 11 publications

(13 citation statements)

References 16 publications

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“…For DDAVP, PFA-100 CT corrections depend on the type of VWD and tend to reflect correction of functional VWF, notably that detected by the VWF:CB. 18,76,83,84 Thus, for VWD patients with an abnormal baseline CT, DDAVP is generally able to normalize the CT for most type 1 VWD individuals, but only for some type 2 VWD individuals. The response of the PFA-100 to factor concentrate therapy, as provided to DDAVP nonresponders, is less striking.…”

Section: Role Of the Pfa-100 In The Identification Of Vwdmentioning

confidence: 95%

“…The role of the PFA-100 in the monitoring of therapy for VWD has also been evaluated by several studies, 49,50,[74][75][76][77][78][79][80][81][82][83][84] and has previously been summarized by this author. [15][16][17][18] Where evaluated, PFA-100 CT responses also tended to provide more utility than did the SBT.…”

Section: Role Of the Pfa-100 In The Identification Of Vwdmentioning

confidence: 99%

“…Normalization of abnormal PFA-100 CTs tends to mimic normalization of plasma VWF, in particular functional VWF assessed in vitro using the VWF:CB assay, and most likely because this assay best reflects the functional adhesive activity of HMW VWF. 18,76,83,84 Nevertheless, lack of correction of PFA-100 CT does not necessarily reflect treatment failure, and appropriate prospective studies are still required to definitively identify the role of the PFA-100 in this setting.…”

Section: Role Of the Pfa-100 In The Identification Of Vwdmentioning

confidence: 99%

See 2 more Smart Citations

Clinical Utility of the PFA-100

Favaloro

2008

Semin Thromb Hemost

288

285

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Section: Role Of the Pfa-100 In The Identification Of Vwdmentioning

confidence: 95%

Section: Role Of the Pfa-100 In The Identification Of Vwdmentioning

confidence: 99%

Section: Role Of the Pfa-100 In The Identification Of Vwdmentioning

confidence: 99%

See 1 more Smart Citation

Clinical Utility of the PFA-100

Favaloro

2008

Semin Thromb Hemost

288

285

View full text Add to dashboard Cite

“…Nevertheless, future work will no doubt be forthcoming, and I await with great interest clarification of these issues [45,46].…”

Section: Laboratory Monitoring Of Concentrate Efficacy In Von Willebrmentioning

confidence: 99%

Von Willebrand factor/factor VIII concentrates in the treatment of von Willebrand disease

Batlle

López-Fernández

Fraga

et al. 2009

Blood Coagulation &Amp; Fibrinolysis

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Therapy for von Willebrand disease (VWD) aims to restore the hemostatic function conferred by von Willebrand factor (VWF), which facilitates platelet adhesion and aggregation, and serves to increase potentially low coagulation factor VIII (FVIII) in plasma. In patients unresponsive to desmopressin (DDAVP), the preferred treatment is with plasma-derived VWF-containing FVIII concentrates. Only a few of the available VWF/FVIII concentrates have been licensed for use in VWD based on prospective studies. The efficacy of VWF/FVIII concentrates depends on the content and quality of VWF and FVIII. Several studies have demonstrated the variability of the VWF contents, as well as the differences in the VWF multimer patterns (including the high molecular weight VWF multimers that are most effective in restoring hemostasis), among these concentrates. Treating physicians should be aware of these disparities and the potential clinical implications for patients with different VWD subtypes. Dosing has traditionally been calculated based on the FVIII content of the products, although dosing based on VWF functional activity [e.g., VWF ristocetin cofactor activity (VWF:RCo)] addresses the primary protein deficiency in VWD patients. Several clinical studies have demonstrated the efficacy of concentrates dosed according to VWF:RCo. Dosing is generally consistent across VWD subtypes, although patients with severe phenotypes or undergoing major procedures may require more infusions or longer treatment duration. Other considerations for the use of VWF-containing concentrates include laboratory monitoring of efficacy and safety issues such as thrombosis risk and thromboprophylaxis.

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“…[33][34][35] It is very well known in routine daily practice that distinction between type 1 or 2 in cases of VWD 2A(IIE) and 2M is frequently not possible due to the limited test set used by laboratories and to the lower limit of sensitivity for these assays including VWF multimeric analysis, VWF:Ag and VWF:RCo 36,37 as recommended by the ISTH-SSC. 38 The present data clearly demonstrates that VWD type 2A (IIE) as a distinct VWD phenotype truly exists if sensitive functional tests such as VWF:RCo, VWF:CB and PFA100 39,40 plus DDAVP response curves and VWFpp/VWF:Ag ratios are used. Our findings also confirm recent data in defining the VWD 2A type (IIE) in other missense mutations in the D3 domain of the VWF gene as a defect of VWF clearance.…”

Section: Discussionmentioning

confidence: 55%

Autosomal dominant C1149R von Willebrand disease: phenotypic findings and their implications

Pérez‐Rodríguez

García-Rivero²,

Lourés³

et al. 2009

Haematologica

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BackgroundMutation C1149R in the von Willebrand factor (VWF) gene has been thought to cause autosomal dominant severe type 1 von Willebrand disease (VWD). Design and MethodsEight patients from three unrelated families with this mutation were included in the present study who had distinct VWF abnormalities, not described in earlier studies. ResultsThe patients showed notably low levels of VWF antigen (VWF:Ag), VWF ristocetin cofactor activity (VWF:RCo), VWF collagen binding (VWF:CB), and a reduced ristocetininduced platelet aggregation (RIPA). VWF:RCo/VWF:Ag and VWF:CB/VWF:Ag ratios were lower than 0.7. At basal conditions, all the VWF multimers were decreased in plasma, with a clearly lower relative proportion of the high molecular weight VWF multimers (HMWM). In high-resolution agarose gels, a large decrease in the relative proportions of the satellite bands was seen. The patients had a brief good response to desmopressin (DDAVP) administration, but the released VWF half-life was shorter than normal, indicating an accelerated clearance of their VWF. Platelet VWF was abnormal. ConclusionsWe conclude from the results obtained in these patients for plasma phenotypic data that this mutation should be classified as a VWD type 2A (IIE). DDAVP therapy may be somewhat helpful for this mutation, at least for mild to moderate bleeding. These data provide evidence that for VWD classification factors other than basal VWF, such as DDAVP response and platelet VWF, should be considered.

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A better approach to monitoring of therapy in von Willebrand disease?

Cited by 11 publications

References 16 publications

Clinical Utility of the PFA-100

Clinical Utility of the PFA-100

Von Willebrand factor/factor VIII concentrates in the treatment of von Willebrand disease

Autosomal dominant C1149R von Willebrand disease: phenotypic findings and their implications

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