1,721,005 research outputs found
Feasibility of prophylaxis and immune tolerance induction regimens in haemophilic children using fully implantable central venous catheters
No full textVenous access represents the major barrier to the feasibility of prophylaxis and immune tolerance induction (ITI) in haemophilic children. Ports improve treatment feasibility, but their duration is limited by infectious complications. This study aimed at evaluating whether or not ports allow haemophilic children to maintain the treatment regimen in the long term. Children were prospectively followed-up and underwent port removal either for complications or transition to peripheral veins. Of 27 ports (17 used for prophylaxis and 10 for ITI), 25 were removed after a median of 3.3 years. Inhibitor children showed a younger age at port insertion (P = 0.02), an earlier occurrence of infections (P = 0.006) at a higher rate (P = 0.00001) and an earlier removal for infection (P = 0.05) than non-inhibitor patients. Daily port use was associated with earlier infections at a higher rate compared to less frequent use (P = 0.02). Port removal after a median of 0.8 years prevented ITI completion in 50% of children, while it hampered the maintenance of prophylaxis in 27% of patients. This study showed that ports improved the feasibility of prophylaxis in the majority of non-inhibitor children, while they were not suitable for inhibitor children who require a prolonged ITI regimen with daily infusions
Immune tolerance induction with recombinant factor VIII in hemophilia A patients with high responding inhibitors
No full textImmune tolerance induction (ITI) eradicates inhibitors in patients with hemophilia A. This study was designed to investigate the success rate of ITI in high-responding inhibitor patients with severe hemophilia A using recombinant factor VIII (rFVIII). Twenty-six patients received different ITI regimens until a normal recovery (&rt; 66%) and half-life (&rt; 6 h) of infused FVIII was achieved. In order to maximize the chance of success, the initiation of ITI was deferred in the majority of patients until the inhibitor declined to < 10 BU. Twenty-two patients (85%) had baseline inhibitor levels < 10 BU (median 2.3 BU) when ITI began. Within a median of 6 months, immune tolerance was achieved in 19 of 26 patients (73%) including 12/17 (70%) with intron 22 inversion, 5/7 (71%) with other null mutations and two with small deletion/insertions in the F8 gene. In conclusion, recombinant FVIII induces a high rate of immune tolerance even in carriers of null F8 mutations
Solvent/detergent plasma for prevention of bleeding in recessively inherited coagulation disorders : dosing, pharmacokinetics and clinical efficacy
No full textBackground and Objectives. This open-label multicenter trial of solvent/detergent (SD) plasma involving 17 patients with recessively inherited coagulation disorders (one afibrinogenemia, four FV, six combined FV and FVIII, one FX and five FXI deficiencies) evaluated the pharmacokinetics of the deficient factors and hemostatic efficacy. Design and Methods. In vivo recovery (IVR) of the deficient coagulation factor was determined in a non-bleeding state in all patients and the mean values for FV, FVIII, FX, FXI and fibrinogen were 1.3, 1.2, 1.5, 1.3 and 1.5 dL/kg, respectively. The mean plasma half-life of FV, FVIII and FX was 18, 43 and 33 hours, respectively. All patients underwent replacement therapy for elective procedures at risk of bleeding (surgery in 14 cases and vaginal delivery in two patients), except one treated for a central nervous system surgical emergency. Results. Treatment courses with SD plasma were judged fully effective in 13/16 cases (81%). In the remaining three cases, mild bleeding occurred after major surgery in a FV deficient patient with a factor level of 43% and in a FXI deficient patient when factor levels were between 20% and 41%; and after minor surgery in a patient with FV and FVIII deficiency when factor levels were 41% and 18%, respectively. Bleeding was controlled by continuing or increasing treatment with SD plasma. Interpretation and Conclusions. These results suggest that, even though the current absolute risk of blood-borne infections associated with fresh-frozen plasma is relatively small, SD plasma should be preferred in patients with recessively inherited coagulation disorders who need replacement therapy when virus-inactivated single-factor concentrates are not available
A prospective randomized trial of high and standard dosages of recombinant factor VIIa for treatment of hemarthroses in hemophiliacs with inhibitors
No full textAim: A multicenter randomized open-label crossover prospective trial was designed to compare the efficacy, safety, and cost of std. and high dosages of recombinant factor VIIa (rFVIIa) for home treatment of hemarthroses in hemophiliacs with inhibitors. Methods: Patients were instructed to treat, within 6 h from the onset of bleeding, four consecutive hemarthroses of ankles, knees, or elbows, either with the rFVIIa std. dose of 90 mg kg-1 (repeated as necessary every 3 h) or with a single high dose of 270 mg kg-1. Patients who did not achieve a clin. success within 9 h continued rFVIIa treatment with repeated std. doses. Response to treatment was assessed for up to 48 h by patients/caregivers, who reported on a Visual Analog Scale (VAS) graded from 0 to 100 the improvement in symptoms and also rated the responses as effective, partially effective or ineffective. Success was defined a treatment course rated as effective and with a VAS score &rt;=70 and failure a treatment course rated as ineffective and VAS score ?30, whereas treatment courses that did not fulfill these criteria were considered partial responses. Results: Twenty hemophiliacs with inhibitors were originally enrolled (median age: 27 years), 18 of them treated 32 hemarthroses assigned to the std.-dosage and 36 to the high-dosage regimen, during the study period of 18 mo. Forty-eight hemarthroses (71%) occurred in target joints. Success rates for std.- and high-dosage regimens were similar: 31% and 25% at 9 h, 53% and 50% at 24 h, 66% and 64% at 48 h, the end point for outcome assessment. The median no. of rFVIIa infusions needed to achieve a successful course was significantly greater for the std.-dosage (n= 3) than for the high-dosage regimen (n= 1), and the median amt. of rFVIIa ultimately used per successful course was identical (270 mg kg-1). Conclusion: Our results indicate that a high-dosage regimen with rFVIIa for home treatment of hemarthroses is effective, safe, does not imply an increased consumption of rFVIIa and requires the infusion of a smaller no. of rFVIIa doses. Its convenience is particularly relevant in cases with difficult venous access and in hemorrhages into target joints
Cognitive and psychological profiles in treatment compliance: a study in an elderly population with hemophilia
Full text linkElderly patients with hemophilia have to face new challenges linked to concomitant pathologies and concurrent use of different treatments. In order to promote optimal care in the elderly hemophilia population, this study is aimed to analyze treatment compliance in relation to the presence of comorbidities and the role of potential determinants that can affect compliance (positively or negatively), including health-related quality of life, cognitive decline, and sociodemographic parameters (eg, living situation, partnership, presence of caregivers). This will be an observational study of elderly patients with hemophilia (aged >60 years). Patients will be interviewed during their routine medical visits. The data interview will pertaining to several dimension of treatment management. This study will detect more vulnerable patients with special care needs and will highlight psychological factors that should be considered for future psychosocial interventions
Going Beyond Counting First Authors in Author Co-citation Analysis
Full text linkThe present study examines one of the fundamental aspects of author co-citation analysis (ACA) - the way co-citation
counts are defined. Co-citation counting provides the data on which all subsequent statistical analyses and mappings
are based, and we compare ACA results based on two different types of co-citation counting - the traditional type that
only counts the first one among a cited work's authors on the one hand and a non-traditional type that takes into
account the first 5 authors of a cited work on the other hand. Results indicate that the picture produced through this non-traditional author co-citation counting contains more coherent author groups and is therefore considerably clearer. However, this picture represents fewer specialties in the research field being studied than that produced through the traditional first-author co-citation counting when the same number of top-ranked authors is selected and analyzed. Reasons for these effects are discussed
Variations on the Author
Full text link“Variations on the Author” discusses two of Eduardo Coutinho’s recent films (Um Dia na Vida, from 2010, and Últimas Conversas, posthumously released in 2015) and their contribution to the general question of documentary authorship. The director’s filmography is characterized by a consistent yet self-effacing form of authorial self-inscription: Coutinho often features as an interviewer that rather than express opinions propels discourses; an interviewer that is good at listening. This mode of self-inscription characterizes him as an author who is not expressive but who is nonetheless markedly present on the screen. In Um Dia na Vida, however, Coutinho is completely absent form the image, while Últimas Conversas, on the contrary, includes a confessional prologue that moves the director from the margins to the center of his films. This article examines the ways in which these works stand out in the filmography of a director who offers new insights into the notion of cinematic authorship
Appropriate Similarity Measures for Author Cocitation Analysis
Full text linkWe provide a number of new insights into the methodological discussion about author cocitation analysis. We first argue that the use of the Pearson correlation for measuring the similarity between authors’ cocitation profiles is not very satisfactory. We then discuss what kind of similarity measures may be used as an alternative to the Pearson correlation. We consider three similarity measures in particular. One is the well-known cosine. The other two similarity measures have not been used before in the bibliometric literature. Finally, we show by means of an example that our findings have a high practical relevance.information science;Pearson correlation;cosine;similarity measure;author cocitation analysis
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