Page 14 - Haematologica Vol. 107 - September 2022
P. 14

EDITORIAL
C.V. Denis and P.J. Lenting
 plication of this molecule: designed as an antithrombotic agent, which could be used for VWF-dependent throm- botic complications (arterial thrombosis, thrombotic thrombocytopenic purpura); it may now also find a use in the treatment of bleeding disorders such as mild/moderate hemophilia A and certain types of VWD (Figure 1). It is unusual to find both features in a single molecule!
In conclusion, Kovacevic et al. present an elegant ap-
proach to improve endogenous VWF and FVIII levels through a single subcutaneous administration of BT200. We look forward to seeing additional clinical data.
Disclosures
No conflicts of interest to disclose.
Contributions
CVD and PJL wrote the manuscript.
References
1. Pipe SW, Montgomery RR, Pratt KP, Lenting PJ, Lillicrap D. Life in the shadow of a dominant partner: the FVIII-VWF association and its clinical implications for hemophilia A. Blood. 2016;128(16):2007-2016.
2. Federici AB. The use of desmopressin in von Willebrand disease: the experience of the first 30 years (1977-2007). Haemophilia. 2008;14(Suppl 1):5-14.
3. Franchini M, Zaffanello M, Lippi G. The use of desmopressin in mild hemophilia A. Blood Coagul Fibrinolysis. 2010;21(7):615-619.
4. Mannucci PM, Bettega D, Cattaneo M. Patterns of development of tachyphylaxis in patients with haemophilia and von Willebrand disease after repeated doses of desmopressin (DDAVP). Br J Haematol. 1992;82(1):87-93.
5. Castaman G, Tosetto A, Rodeghiero F. Reduced von Willebrand factor survival in von Willebrand disease: pathophysiologic and clinical relevance. J Thromb Haemost. 2009;7 (Suppl 1):71-74.
6. Miesbach W, Krekeler S, Duck O, et al. Clinical assessment of efficacy and safety of DDAVP. Hamostaseologie. 2010;30(Suppl 1):S172-175.
7. Stoof SC, Cnossen MH, de Maat MP, Leebeek FW, Kruip MJ. Side effects of desmopressin in patients with bleeding disorders. Haemophilia. 2016;22(1):39-45.
8. Denis CV, Kwack K, Saffaripour S, et al. Interleukin 11 significantly increases plasma von Willebrand factor and factor VIII in wild type and von Willebrand disease mouse models. Blood. 2001;97(2):465-472.
9. Olsen EH, McCain AS, Merricks EP, et al. Comparative response of plasma VWF in dogs to up-regulation of VWF mRNA by interleukin-11 versus Weibel-Palade body release by desmopressin (DDAVP). Blood. 2003;102(2):436-441.
10. Ragni MV, Jankowitz RC, Jaworski K, Merricks EP, Kloos MT, Nichols TC. Phase II prospective open-label trial of recombinant interleukin-11 in women with mild von Willebrand disease and refractory menorrhagia. Thromb Haemost. 2011;106(4):641-645.
11. Ragni MV, Novelli EM, Murshed A, Merricks EP, Kloos MT, Nichols TC. Phase II prospective open-label trial of recombinant interleukin-11 in desmopressin-unresponsive von Willebrand disease and mild or moderate haemophilia A. Thromb Haemost. 2013;109(2):248-254.
12. Kovacevic KD, Grafeneder J, Schörgenhofer C, et al. The von Willebrand factor A-1 domain binding aptamer BT200 elevates plasma levels of von Willebrand factor and factor VIII: a first-in- human trial. Haematologica. Haematologica.107(9):2121-2132.
13. Zhu S, Gilbert JC, Hatala P, et al. The development and characterization of a long acting anti-thrombotic von Willebrand factor (VWF) aptamer. J Thromb Haemost. 2020;18(5):1113-1123.
14. Gilbert JC, DeFeo-Fraulini T, Hutabarat RM, et al. First-in-human evaluation of anti von Willebrand factor therapeutic aptamer ARC1779 in healthy volunteers. Circulation. 2007;116(23):2678-2686.
15. Kovacevic KD, Greisenegger S, Langer A, et al. The aptamer BT200 blocks von Willebrand factor and platelet function in blood of stroke patients. Sci Rep. 2021;11(1):3092.
16. Jilma-Stohlawetz P, Knobl P, Gilbert JC, Jilma B. The anti-von Willebrand factor aptamer ARC1779 increases von Willebrand factor levels and platelet counts in patients with type 2B von Willebrand disease. Thromb Haemost. 2012;108(2):284-290.
17. Zhu S, Gilbert JC, Liang Z, et al. Potent and rapid reversal of the von Willebrand factor inhibitor aptamer BT200. J Thromb Haemost. 2020;18(7):1695-1704.
 Haematologica | 107 September 2022
2013






































































   12   13   14   15   16