Haematologica
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Multicenter Study
Clinical use of Haemate P in inherited von Willebrand's disease: a cohort study on 100 Italian patients.
Plasma-derived concentrates containing von Willebrand factor and factor VIII (VWF/FVIII concentrates) are the mainstay of treatment of patients with inherited von Willebrand's disease (VWD) who are unresponsive or have a contraindication to desmopressin (DDAVP) therapy. Only a few clinical studies are available on the use of these VWF/FVIII concentrates in large numbers of cases and within the same country. The aim of our study was to collect retrospective data on the efficacy and safety of Haemate P (CSL Behring, Marburg, Germany) in a large cohort of well-characterized VWD patients after the introduction of the guidelines for VWD management in Italy. ⋯ Based on this retrospective study conducted in a large cohort of Italian patients (n=100) and covering a long period of observation (36 months), Haemate P was shown to be effective and safe for the clinical management of patients with VWD, whether given on demand or as prophylaxis.
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A probabilistic model was used to compare cryoprecipitate to viral inactivated, commercial fibrinogen concentrate to evaluate with regard to the recipient's risk of exposure to an emergent AIDS-like epidemic. In patients who occasionally need a therapeutic dose of fibrinogen, commercial fibrinogen would be marginally safer than cryoprecipitate if the new pathogen were sensitive to inactivation. But there is a potential high risk of exposure if the emerging agent withstands inactivation. In most of the analyzed scenarios, cryoprecipitate is safer than commercial fibrinogen as long as the odds that the new agent is sensitive to inactivation are lower than 1.000 to 1.
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Pulmonary hypertension (PH) is increasingly observed in sickle cell disease (SCD) and beta-thalassemia (beta-thal), but there is no information on its prevalence in patients with HbS/beta-thal. The amino-terminal fragment of B-type natriuretic peptide (NT-proBNP) is considered as an independent prognostic factor in PH. The aim of this study was to evaluate the incidence of PH and its correlation with clinical and laboratory findings, including NT-proBNP, in patients with HbS/beta-thal. ⋯ The incidence of PH in patients with HbS/beta-thal is similar to that observed in patients with SCD. Serum NT-proBNP is a strong indicator of PH in HbS/beta-thal. The correlation between PH and reticulocyte counts and ferritin suggests that the degree of hemolysis and iron overload is implicated in the pathogenesis of PH in HbS/beta-thal.
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Alemtuzumab is a humanized monoclonal antibody directed against lymphocytes through the CD-52 receptor, an antigen being found on > 95% of peripheral blood lymphocytes and monocytes, and to a smaller extent on granulocytes. It is an effective immunotherapeutic agent in patients with malignancies such as non-Hodgkin lymphoma, B cell chronic lymphocytic leukemia and T cell pro- lymphocytic leukemia. ⋯ Digestive complications have more rarely been described, i.e.: gastroenteritis and peritonitis. We recently observed a case of particular interest as the patient with T cell prolymphocytic leukaemia treated with alemtuzumab, exhibited symptomatic reactivation of CMV infection and developed subsequently typhlitis.
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High-dose chemotherapy with allogeneic stem cell transplantation (SCT) has proven to be a successful treatment for low-grade lymphoma (LGL), but is associated with considerable transplant-related mortality (TRM). In an effort to reduce toxic mortality while maintaining the graft-versus-leukemia effect, allogeneic SCT has been combined with a reduced-intensity conditioning (RIC) regimen. The aim of this study was to determine the outcome of patients with LGL treated with RIC allogeneic SCT. ⋯ Although associated with significant TRM, RIC allogeneic SCT in advanced chemosensitive disease leads to long-term survival.