1,721,228 research outputs found
ACTOR VII CONGENITAL DEFICIENCY: CLINICAL PICTURE AND CLASSIFICATION OF THE VARIANTS
No full textIn congenital factor VII deficiency the clinical picture is related to the levels of factor VII coagulant activity; when factor VII:C levels are very low the bleeding episodes can occur frequently. The most frequent bleedings are menorrhagia and metrorrhagia in females and hemarthrosis in both sexes. There are 3 immunological variants of factor VII deficiency: VII-, VIIR and VII+. Conversely, the genetic variants are 2: one characterized by no discrepancy between VII:C and VII:Ag (found in the heterozygotes of VII- and VIIR variants) and the other, in which a discrepancy between VII:C and VII:Ag is found (heterozygotes from VII+ kindreds). In factor VII deficiency, most commonly the human and ox tissue factors show the highest sensitivity to the coagulation defect, whereas the one extracted from rabbits is definitely less sensitive; the definition of functional variants is based upon a different reactivity to homologous and/or heterologous tissue thromboplastins. In no case was a PIVKA-VII-like protein found and none of the factor VII defective molecules reacted to the generation of important kallikrein activity
PISA post-marketing italian surveillance study on advate treatment of haemophilia A patients in order to evaluate its efficacy, safety and immunogenicity
No full textTreatment of congenital factor VII deficiency with a new concentrate
No full textA new factor VII concentrate, made from ACD plasma by a process involving successive absorptions of cryoprecipitate supernatant on DEAE Sephadex and of the resulting supernatant on A1(OH) 3, was administered to 10 patients with severe factor VII deficiency. 5 patients received only one dose for treatment of a single bleeding episode, the remaining 5 were given multiple infusions (47) for spontaneous hemorrhages or for the prevention of surgical bleeding. In vivo factor VII recovery ranged from 43 to 126% (average 88%) of the assayed in vitro activity of the concentrate. A dose of 0.5 u/kg was found to produce a 1% rise of the plasma factor VII levels. The mean half-life on injected factor VII as assessed in 7 kinetic studies was 205 min (range 168-234). Spontaneous bleeding was easily controlled by the concentrate and major surgical procedures (two tonsillectomies) could be performed without complications. 1 patient developed HBsAg positive hepatitis, but otherwise no serious side effects were observed. Factor VII concentrate reduces the risk of precipitating circulatory overload associated with the use of plasma and avoids the unnecessary rise of factor II, IX and X, which follows prothrombin complex concentrates
Tailored versus standard dose prophylaxis in children with hemophilia A.
No full textProphylaxis is universally recognized as the treatment of choice in people with hemophilia, and tailored prophylaxis is the consistent modification of the standard weight-based dosing regimen. A large number of factors guide the choice of a specific tailored regimen, and different regimens are under evaluation. Tailored low-dose frequent regimens are likely to be cost-effective, but they are less accepted by patients. Escalating dose regimens seem to be quite effective in preventing bleedings and, consequently, arthropathy, although data on long-term outcomes are still not available. Pharmacokinetic-driven approaches have been also proposed. Sensitive and validated tools able to reliably measure the different outcomes are necessary in this setting. With regard to the evaluation of arthropathy, magnetic resonance imaging and ultrasound are promising imaging techniques in detecting early joint damage. Factor VIII trough levels can be considered a measure of the efficacy of FVIII infused, although other factors influence the bleeding pattern. Global assays of coagulation could provide more complete information on the hemostatic potential of a sample and predict bleeding phenotype. These techniques are also promising for the individualization of prophylaxis regimens, potentially resulting in less frequent dosing, more comfortable and less expensive approaches
Deflazacort in thrombocytopenia: a comparison with prednisone.
No full textDeflazacort is a new glucocorticoid which in previous studies was found to be about 0.8 times as potent as prednisone. Twelve outpatients affected by chronic idiopathic thrombocytopenic purpura, in whom the maintenance dose of corticosteroid had already been established, were given 6 mg deflazacort for each 5 mg prednisone equivalent. Effectiveness of deflazacort therapy was estimated on the basis of the results of platelet count, bleeding time, tourniquet test, and physical signs related to platelet function. Tolerability was evaluated on the basis of laboratory data, side effects, and body weight. Deflazacort was administered for 54--263 days (mean 114.5) and, at the same mean daily dose of prednisone, succeeded in keeping platelet number unchanged. As far as bleeding time, tourniquet test, and physical signs are concerned, deflazacort therapy gave a further improvement compared to prednisone. Both treatments were very well tolerated. The high number of white blood cells and neutrophils during prednisone therapy was restored to the normal range by deflazacort
A RETROSPECTIVE STUDY ON ORAL ANTICOAGULANT PROPHYLAXIS IN 103 ITALIAN PATIENTS WITH HEREDITARY THROMBOPHILIA AND THROMBOSIS
No full textThe clinical records of 103 Italian patients with inherited thrombophilia and thrombosis were reviewed to estimate the incidence of thrombotic recurrences and major bleeding complications according to the different duration of oral anticoagulant prophylaxis (OAP). The incidence of the first thrombotic recurrence was 2.9, 7.4 and 10.8 x 100 patients/year, respectively, in subjects receiving lifelong OAP, stopping OAP after a mean of 9 months (range 1-30 months) or not receiving OAP. The probability to remain free from thrombotic recurrences in patients undergoing lifelong OAP, as estimated by the Kaplan-Meier method, was significantly higher in comparison with untreated patients (p less than 0.001), but did not reach the statistical significance in comparison with patients who stopped prophylaxis. The incidence of further thrombotic recurrences was 1.2, 21.1 and 22.3 x 100 patients/year, respectively, in the three groups defined above. The difference between patients who prolonged indefinitely OAP vs those who stopped or did not receive OAP was statistically significant (p = 0.003). Two intracranial bleedings, one of which fatal, were observed in patients undergoing lifelong OAP, whereas no major bleeding complications occurred in the other two groups. Our study supports the recommendations to continue indefinitely OAP in patients with inherited thrombophilia and recurrent thrombosis, but suggests caution in starting lifelong prophylaxis soon after the first thrombotic event in all patients
RECOMBINANT ALPHA 2b INTERFERON IN THE TREATMENT OF REFRACTORY AUTOIMMUNE THROMBOCYTOPENIC PURPURA
No full text- …
