Oral Communication

Inherited factor VII (FVII) deficiency is the most common of the rare autosomal recessive bleeding disorders (RBD), with an estimated prevalence of 1 per 300,000 in European countries.1,2 It is likely that the prevalence is higher in those countries where consanguineous marriages are frequent.We here report STER study results on 112 evaluable enrolled cases

Bone-marrow derived haematopoietic and endothelial progenitors and reticulocytes after aerobic and anaerobic exercise in athletes

Bleeding symptoms at disease presentation and prediction of ensuing bleeding in inherited FVII deficiency.

SummaryIndividuals with inherited factor VII (FVII) deficiency display bleeding phenotypes ranging from mild to severe, with 30% of patients having always been asymptomatic (non-bleeding). In 626 FVII-deficient individuals, by analysing data from the International Factor VII (IF7) Registry and the Seven Treatment Evaluation Registry (STER), we determined whether bleeding type at disease presentation and FVII coagulant activity (FVIIc) predict ensuing bleeds. At disease presentation/diagnosis, 272 (43.5%) individuals were non-bleeding, 277 (44.2%) had minor bleeds, and 77 (12.3%) had major bleeds. During a median nine-year index period (IP) observation, 87.9% of non-bleeding individuals at p…

Inhibitors to factor VII in congenital factor VII deficiency.

Recombinant, activated factor VII for surgery in factor VII deficiency: a prospective evaluation - the surgical STER

Oral Idarubicin in Maintenance Therapy of Acute Myeloid Leukemia

More than half of all acute myeloid leukaemia (AML) patients are over 60 years. The disease free survival (DFS) and overall survival (OS) rate of these patients is poor. These unsatisfactory results are associated with adverse cytogenetic characteristics, prior myelods-plasia, adverse phenotypic features, MDR and BCL2 overexpression. Furthermore a large fraction of patients achieving CR early relapses. This is due to two factors: acquired tumor cell drug resistance and tumor re-growth. Maintenance therapy could provide a means to keep leukemic growth under control. We enrolled 31 elderly previous responder patients to standard induction therapy to receive maintenance oral IDA 3mg/m2 daily d…

Pharmacokinetic properties of recombinant FVIIa in inherited FVII deficiency account for a large volume of distribution at steady state and a prolonged pharmacodynamic effect.

Pharmacokinetic properties of recombinant FVIIa in inherited FVII deficiency account for a large volume of distribution at steady state and a prolonged pharmacodynamic effect -

Thrombosis in inherited factor VII deficiency

Thrombosis in congenital factor (F) VII deficiency was investigated through extensive phenotypic and molecular-genetic studies. Patients with a history of thrombosis among 514 entries in the FVII Deficiency Study Group database were evaluated. Thrombotic events were arterial in one case, disseminated intravascular coagulation in another and venous in seven. Gene mutations were characterized in eight patients: three were homozygous, three compound heterozygous and two heterozygous. FXa and IIa generation assays were consistent with the genetic lesions. One patient was heterozygous for the FV Leiden and one for the FIIG20210A mutation. In seven patients, surgical interventions and/or replacem…

Invasive procedures and minor surgery in factor VII deficiency

Factor VII deficiency: evidence that in minor surgery one-day replacement therapy is sufficient

Successful treatment of resistant thrombotic thrombocytopenic purpura/hemolytic uremic syndrome with autologous peripheral blood stem and progenitor (CD34+) cell transplantation

The first-line treatment of thrombotic thrombocytopenic purpura-hemolytic uremic syndrome (TTP-HUS syndrome) induces a response and survival rate of approximately 85%, even if a considerable number of patients relapse; nevertheless, a number of these patients are resistant to conventional management. Immunoablation followed by stem cell transplantation has been shown to be capable of inducing remissions in a large spectrum of experimental autoimmune disorders. We report here the case of a 20-year-old male patient with the TTP-HUS syndrome who was resistant to conventional treatment and was transplanted with autologous immunoselected CD34+ PBPC after conditioning with cyclosphosphamide, anti…

Autologous peripheral blood stem and progenitor (CD34+) cell transplantation for systemic lupus erythematosus complicated by Evans syndrome.

Immunoablation followed by allogeneic stem cell (SC) transplantation has been shown to be capable of curing a large spectrum of experimental autoimmune disorders, hereditary and/or induced. Superimposable results, albeit with some exceptions, have been obtained in human patients affected by coincidental autoimmune and blood diseases. However, both because of encouraging experimental results and of the procedure's greater safety, autologous SC are being increasingly utilized worldwide. Case reports are being collected in the registry of the European Group for Blood and Marrow Transplantation (EBMT)/European League against Rheumatism (EULAR) Autoimmune Disease Stem Cell Project. Among the se…

Pharmacokinetic of Factor VII