0000000000699455
AUTHOR
C. Caracciolo
Clinical efficacy of highly purified, doubly virus-inactivated factor VIII/von Willebrand factor concentrate (Fanhdi® ) in the treatment of von Willebrand disease: a retrospective clinical study
Summary. The goal of therapy in patients with von Willebrand disease (vWD) is to correct the dual defect of primary haemostasis and intrinsic coagulation reflected by low levels of von Willebrand factor (vWF) and factor VIII coagulant activity (FVIII:C). Factor VIII/von Willebrand factor (FVIII/vWF) concentrates are currently the treatment of choice in vWD patients unresponsive to desmopressin (DDAVP). However, only few studies on their clinical use are available so far. The main objective of this study was to retrospectively evaluate the clinical efficacy of a highly purified, doubly virus-inactivated FVIII/vWF concentrate with a high content of FVIII/vWF (Fanhdi®). Twenty-two patients wit…
CALR MUTATIONS IN SICILIAN ESSENTIAL THROMBOCYTHEMIA AND MYELOFIBROSIS PATIENTS.
Background. Essential thrombocythemia (ET) and primary myelofibrosis (MF) are myeloproliferative neoplasms characterized by the overproduction of mature cells such as platelets (ET) or early bone marrow fibrosis due to scarring induced by highly proliferating myeloid progenitors and pathological stimulation of local fibroblasts (MF). Somatic mutations in CALR gene have recently identified in the majority of JAK2-V617F and MPL negative ET and MF patients. In this study we evaluated the frequency and type of CALR mutations and their clinical and hematological features. Methods. A total of 54 patients, 29 ET and 25 MF patient, was included in this study. All patients were JAK2 V617F and MPL ne…