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Extended Half-life rFVIII for the Treatment of Hemophilia A: Drugs Consumption and Patients’ Perspective
Full text linkProphylaxis is recognized as the most effective treatment regimen for patients with severe hemophilia A. Prophylaxis with standard half-life (SHL) FVIII products requires frequent intravenous administrations, at least two-three times per week. Frequency of injections is reported as one of the major obstacles to adherence to treatment and caused impairment in quality of life (QoL) and possible clinical implications. The extended half-life (EHL) rFVIII products recently reimbursed by Italian National Health Service (NHS), give the possibility of prophylactic dosage regimens characterized by reduced administration frequency with the potential to increase adherence to therapy and to improve QoL and clinical outcomes. Based on the approved dosing regimens the minimum and maximum number of administrations per year and the annual consumption for the marketed EHL rFVIII products were estimated. Compared to Adynovi® and Elocta®, Jivi® is the drug associated with the lowest number of administrations per year while versus Esperoct® is associated to a slightly higher maximum number of administrations per year. Furthermore, Jivi® has an annual mean consumption per kg lower than Adynovi®, Elocta® and Esperoct® (-24%, -27% and -22%, respectively). The contemporary reduction of number of injections per year and the lower annual mean consumption (IU) represent important benefits for the patient. From the economic point of view, at the prices published in Italian Official Journal, Jivi® weighted average annual expenditure per patient is 26% lower than Adynovi® and 29% lower than Elocta® and a saving is possible even considering a 10% increase in Jivi®’s price per IU versus comparators. Among the EHL rFVIII concentrates Jivi® appears to be more suitable to cover patients’ needs due to the possibility to adopt different dosage regimens (up to every 7 days) and is associated with the lowest average annual consumption per patient allowing a more predictable budget forecast and overall reducing the NHS expenditure
Cerebral Embolism Beyond Atrial Fibrillation: Interatrial Block
Full text linkThis paper is devoted to conditions that imply a risk for cerebral embolism beyond atrial fibrillation (AF). We focus on advanced interatrial block (IAB) and its relationship with stroke in elderly patients with no documented arrhythmias. Advanced IAB is manifested in the surface electrocardiogram (ECG) as a P-wave duration >120 ms plus biphasic morphology (positive and negative deflection) in leads II, III, and aVF. Several data suggest that AF is not necessarily the leading cause of stroke, but rather a risk factor. In fact, a high stroke risk has been described even in the absence of AF in patients with high CHA2DS2VASc-score (Congestive Heart failure, hypertension, Age ≥75 years [doubled], Diabetes, Stroke [doubled], Vascular disease, Age 65-74 years, Sex category [female sex]). Moreover, excessive atrial ectopy and short atrial runs also increase stroke risk. Some of the previously mentioned stroke risk factors in patients without documented arrhythmias might be combined to determine thrombotic risk. That risk is particularly high in elderly patients with advanced IAB, structural heart disease, CHA2DS2-VASc score ≥3, and frequent ambient atrial arrhythmias. Systematic screening for advanced IAB in elderly patients can be performed with a simple surface ECG. Advanced IAB is a risk marker of stroke
Real-life Diagnostic and Therapeutic Approach to CLL: A New Proposal from an Expert Panel in Tuscany Region
Full text linkBACKGROUND: In the last years genomic and somatic alterations have shown to play a pivotal role in the pathogenesis of chronic lymphocytic leukemia (CLL) and new prognostic factors have been identified accordingly.AIM: To describe a real-life diagnostic and therapeutic approach to CLL that takes into account the role of genomic and somatic prognostic factors in the risk stratification of developing progressive disease, and treatment decision.METHODS: This new proposal has been developed and validated by ten key opinion leaders from Tuscany Region during two Expert Meetings. The approach suggested comes from their experience in daily clinical practice and is supported by guidelines recommendations, clinical trials results, and drugs prescribing conditions in Italy.RESULTS: Beside TP53 deletion or mutated status, the Expert Panel highlighted the importance of the IGHV mutation status characterization, since the diagnosis, in order to identify patients who will have a more aggressive progression. Furthermore, just before starting treatment, to obtain useful prognostic information and indication in the selection of the therapy, they recommend cytogenetic analysis for the detection of del(11q), trisomy 12, del(13q), del(17p), conventional karyotyping of stimulated CLL cells, TP53 sequencing, and molecular genetic analysis to detect IGHV mutation status.CONCLUSIONS: The Expert Panel recognized the limitations associated with traditional staging systems in identifying patients who will have a more aggressive disease course and predicting response to treatment and suggested a real-life diagnostic and therapeutic approach to CLL to update the current patient management in light of recent advances that have improved understanding of CLL
Critical Review of the Pivotal Studies of Four rFVIII Products for the Treatment of Hemophilia A Patients: The Role of Octocog Alfa
Full text linkINTRODUCTION: Hemophilia A is a rare congenital bleeding disorder caused by a deficiency of clotting factor VIII (FVIII). The severe form of the disease is characterized by spontaneous bleeds, especially into the joints. Prophylaxis, based on regularly intravenous administration of the missing factor to avoid hemorrhages, represents the gold standard of treatment. In recent years, new products that significantly improve the treatment management options for patients with hemophilia have become available in the market.OBJECTIVE: To critically evaluate the pivotal studies of recombinant FVIII (rFVIII) products, approved in Europe within the first half of 2018 having obtained the indication for a prophylaxis dosing regimen based also on a twice weekly infusion frequency or even less, highlighting their limitations or strengths.METHODS: A systematic literature search was conducted, and several databases (PubMed and Embase) were consulted.RESULTS: Nine clinical trials involving patients with severe hemophilia A without inhibitor were included in this analysis. Four rFVIII products (Elocta®, Biogen, Cambridge, MA, USA; Kovaltry®, Bayer HealthCare Pharmaceuticals, Germany; Afstyla®, CSL Behring GmbH, Germany; Adynovi®, Baxalta Innovation GmbH, Austria) with different pharmacokinetic profiles were evaluated. The trials included in this analysis had different designs and heterogeneous methods were utilized to assess the study outcomes. The baseline characteristics of the patients enrolled in the studies were also often different and sometimes not adequately described. LEOPOLD II, a trial to compare prophylaxis to on-demand therapy with an unmodified rFVIII product (Kovaltry®, octocog alfa), was the only completely randomized trial that enrolled a more critical patient population in terms of compromised joint condition than the other studies. Based on these side-by-side comparison, Octocog alfa reported similar efficacy, in terms of annualized bleeding rate, to the other rFVIII products, including extended half-life.CONCLUSIONS: Even without structural modifications, octocog alfa may be considered a useful treatment option for two times a week prophylaxis in a selected population of haemophilia patients
Erratum: Effectiveness of Long-Acting Injectable Antipsychotics in Schizophrenia: A Literature Review and Bayesian Meta-Analysis Informing Economic Considerations
Full text linkThere was an error in the relapse rate reduction reported in Figure 2 in this article by Zaniolo et al. [Farmeconomia. Health economics and therapeutic pathways 2019; 20: 13-24; https://doi.org/10.7175/fe.v20i1.1393]. The online version has been corrected on 13 February 2019
An Integrated Management Model of Patients With Atrial Fibrillation: The Experience of the Local Health Unit Tuscany North-West
Full text linkAtrial fibrillation (AF) is the most prevalent form of alteration in cardiac rhythm and is associated with a high economic burden resulting from both clinical consequences and impact on patients’ quality of life. Goals of treatment include symptom control and, in the high-risk patients, the prevention of thromboembolic complications. The advent of novel oral anticoagulant agents (NOACs) has improved the management of patients with non-valvular AF (NVAF) by overcoming limitations associated with traditional oral anticoagulation drugs. NOACs are associated with a lower risk of stroke, systemic embolism, and mortality compared to vitamin K antagonists (VKAs) and with a lower risk of fatal, major, and intracranial bleeding. This supplement aims at sharing the virtuous management model of AF patients in the Local Health Unit Tuscany North-West and promoting the importance of a multidisciplinary management, which involves cardiologists and general practitioners (GPs), not only in terms of clinical outcomes, but also of therapeutic appropriateness and economic sustainability
Atypical Presentation of Periodic Paralysis: A Case Report
Full text linkHypokalemic periodic paralysis (HPP) is a kind of periodic paralysis, which is a heterogeneous group of muscle diseases. It is characterized by episodes of flaccid and sudden muscle weakness.Here, we present a case of HPP. The patient was referred to our department because of severe dizziness and fall. After a comprehensive evaluation, a markedly low potassium was detected. The patient’s symptoms resolved after replacement of potassium and he was discharged without deficits. Even though the literature reports an association with exercise, carbohydrate load, and stress, further workup in our patient revealed no association with these precipitants. A proper differential diagnosis should rule out other causes of weakness and paralysis, thus allowing a timely treatment
Statins and Immune-Mediated Necrotizing Myopathy
Full text linkStatins are a well-recognized cause of a variety of skeletal myopathic effects, which generally resolve when discontinuing the treatment. Among autoimmune manifestations associated with statins, there is immune-mediated necrotizing myopathy (IMNM).The present article summarizes the main features of statin-related IMNM, describing diagnosis, classification, epidemiology, treatment, and the main autoantibodies detected.Statins are a well-recognized cause of a variety of skeletal myopathic effects, which generally resolve when discontinuing the treatment. Among autoimmune manifestations associated with statins, there is immune-mediated necrotizing myopathy (IMNM).The present article summarizes the main features of statin-related IMNM, describing diagnosis, classification, epidemiology, treatment, and the main autoantibodies detected.Although it is impossible to define the precise number, it evident that more than 550 statin-related IMNM cases have been described in the literature. Among IMNM, two forms must be distinguished: with anti-3-hydroxy-3-methylglutaryl-coenzyme A reductase (HMGCR) and with anti-signal recognition particle (SRP) antibodies. The differential diagnosis should be made between the IMNM and self-limited statin-related myopathy, drug-induced rhabdomyolysis, and nonautoimmune myopathies.Patients who have failed to normalize high creatine phosphokinase (CPK) after statin withdrawal should be tested for anti-HMGCR antibodies and, if these are positive, undergo muscle biopsy to confirm the diagnosis of IMNM. Pharmacological therapy of IMNM, not yet based on evidence, involves the use of high-dose corticosteroids, immunosuppressant drugs used alone or in combination, intravenous immunoglobulins (IVIg) or plasmapheresis
