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Description of the First Cases with ADAMTS13 Mutations in Hungary
CONCLUSIONS:Clinical and molecular characterization of the first 5 Hungarian cases with ADAMTS13 mutations was presented. Compound heterozygous, damaging ADAMTS13 mutations with deficient plasma ADAMTS13 activity were associated with serious clinical consequences. In case of thrombotic microangiopathy in young patients with stroke, or HELLP syndrome, complete workup for ADAMTS13 activity and inhibitors is necessary, and genetic analysis should be considered. Three new ADAMTS13 mutations (c.1016_1017delCA, c.3199T>A , c.2839C>T) were identified and shown to be associated with decreased plasma ADAMTS13 activity.Disclos...
Source: Blood - November 21, 2018 Category: Hematology Authors: Reti, M., Sinkovits, G., Cseprekal, O., Csuka, D., Szilagyi, A., Farkas, Z., Klucsik, Z., Szederjesi, A., Wagner, L., Reusz, G., Kremer Hovinga, J. A., Rigo, J., Masszi, T., Prohaszka, Z. Tags: 311. Disorders of Platelet Number or Function Source Type: research

Prospective Study of Apixaban for Primary Prevention of Venous Thromboembolism in Patients with Multiple Myeloma Receiving Immunomodulatory Therapy
ConclusionsIn this pilot study of 50 patients, low-dose apixaban was safe and well tolerated as thromboprophylaxis for patients with MM receiving IMiDs. No patients experienced VTE, major hemorrhage, stroke, or MI. Further randomized studies are needed to validate apixaban as a standard primary prevention anti-thrombotic strategy for patients with MM receiving IMiDs.DisclosuresMoslehi: Bristol-Myers Squibb: Consultancy, Research Funding. Jagasia: Incyte Corporation: Membership on an entity's Board of Directors or advisory committees.
Source: Blood - November 21, 2018 Category: Hematology Authors: Cornell, R. F., Goldhaber, S. Z., Engelhardt, B. G., Moslehi, J., Jagasia, M., Patton, D., Harrell, S. L., Hall, R. L., Wyatt, H., Piazza, G. Tags: 332. Antithrombotic Therapy: Poster I Source Type: research

Outcome of Patients with Relapsed/Refractory (R/R) Chronic Lymphocytic Leukemia (CLL) Treated with Ibrutinib within a Named Patient Program (NPP) in Italy. a Real-Life Retrospective Study
Conclusions. The results of this real-life study show that in unselected patients with R/R CLL the clinical activity of ibrutinib was comparable to that reported in CTs. However, a third of patients discontinued ibrutinib within 24 months from the start of treatment. An earlier introduction of ibrutinib in the treatment approach of R/R patients, a careful surveillance and management of toxicities will optimise the clinical benefits of ibrutinib in CLL patients treated in the clinical practice.DisclosuresMauro: Abbvie: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Shire: Membership on...
Source: Blood - November 21, 2018 Category: Hematology Authors: Mauro, F. R., Soddu, S., Frustaci, A. M., Orsucci, L., Motta, M., Scarfo, L., Zinzani, P. L., Falzetti, F., Farina, L., Marasca, R., Cortelezzi, A., Carlo-Stella, C., Molica, S., Coscia, M., Zaja, F., Laurenti, L., de Fabritiis, P., Gaidano, G., Gobbi, M. Tags: 642. CLL: Therapy, excluding Transplantation: Poster II Source Type: research

Platelet-derived VWF is not essential for normal thrombosis and hemostasis but fosters ischemic stroke injury in mice
Von Willebrand factor (VWF) is a key hemostatic protein synthesized in both endothelial cells and megakaryocytes. Megakaryocyte-derived VWF is stored in α-granules of platelets and is enriched in hyperactive "ultra-large" VWF multimers. To elucidate the specific contribution of platelet VWF in hemostasis and thrombosis, we performed crossed bone marrow transplantations between C57BL/6J and Vwf–/– mice to generate chimeric mice. Chimeric mice specifically lacking platelet VWF showed normal tail bleeding and carotid artery thrombosis, similar to wild-type mice. Chimeric mice with VWF present only in platele...
Source: Blood - October 1, 2015 Category: Hematology Authors: Verhenne, S., Denorme, F., Libbrecht, S., Vandenbulcke, A., Pareyn, I., Deckmyn, H., Lambrecht, A., Nieswandt, B., Kleinschnitz, C., Vanhoorelbeke, K., De Meyer, S. F. Tags: Platelets and Thrombopoiesis, Thrombosis and Hemostasis Source Type: research