IntroductIonChronic hepatitis C represents a major public health problem worldwide. The estimated average global prevalence is close to 3% (ranging from 0.1% to 5% in different countries). Therefore, there are about 175 million carriers of hepatitis C virus (HCV) worldwide (24) . Approximately 30% of infected patients develop cirrhosis in 20-30 years and 1%-4% are considered at high risk of developing hepatocellular carcinoma (23) . The chronicity of hepatitis C is characterized by persistent HCV for 6 months. Prospective studies show that 60% to 85% of these infections progress to chronicity (15,24) . AbstrAct -Context -Pharmacovigilance studies aim to detect, assess, understand and prevent risks of adverse effects of medications or any other possible drug related problem. Alpha interferon is being produced by Bio-Manguinhos/Fiocruz, Rio de Janeiro, RJ, Brazil and used in the treatment of chronic hepatitis C at Brazilian National Health System. Objective -To study the safety profile and effectiveness of alpha interferon in a sample of Brazilian patients with chronic hepatitis C genotypes 2 and 3, in Porto Alegre, RS, Brazil. Method -We followed a cohort of chronic hepatitis C genotypes 2 and 3 patients treated with alpha interferon plus ribavirin in a specialized outpatient clinic in southern Brazil. Adverse events were collected and classified according to severity in monthly structured interviews. To measure effectiveness, hepatitis C viral load was evaluated before, at the end and 24 weeks after the treatment. Results -We followed 141 patients during the study period, of which 52.5% were female with mean age of 52 years. The most frequent adverse events were fatigue (84%), headache (79%) and myalgia (75%). There were 13 treatment interruptions due to adverse events, 9 of those considered serious adverse events. Virological response at end of treatment was 54.6% and after 24 weeks 39.7%, considering all patients who started treatment. Conclusion -The product produced by Bio-Manguinhos has similar efficacy and adverse event and sustained virological response profiles comparable to those found in the literature. This is the first study of pharmacovigilance performed with the Brazilian product. These data will be useful for planning and management of this disease in Brazil. HEADINGs -Hepatitis C, chronic. Interferon alpha. Ribavirin. Adverse drug reaction reporting systems.The authors declare no conflicts of interest. Study carried out at
Pompe disease (PD) is a glycogen storage disorder caused by deficient activity of acid alpha-glucosidase (GAA). We sought to review the latest available evidence on the safety and efficacy of recombinant human GAA enzyme replacement therapy (ERT) for late-onset PD (LOPD). Methods: We systematically searched the MEDLINE (via PubMed), Embase, and Cochrane databases for prospective clinical studies evaluating ERT for LOPD on pre-specified outcomes. A meta-analysis was also performed. Results: Of 1601 articles identified, 22 were included. Studies were heterogeneous and with very low certainty of evidence for most outcomes. The following outcomes showed improvements associated with GAA ERT, over a mean follow-up of 32.5 months: distance walked in the 6-min walking test (6MWT) (mean change 35.7 m (95% confidence interval [CI] 7.78, 63.75)), physical domain of the SF-36 quality of life (QOL) questionnaire (mean change 1.96 (95% CI 0.33, 3.59)), and time on ventilation (TOV) (mean change −2.64 h (95% CI −5.28, 0.00)). There were no differences between the pre- and post-ERT period for functional vital capacity (FVC), Walton and Gardner-Medwin Scale score, upper-limb strength, or total SF-36 QOL score. Adverse events (AEs) after ERT were mild in most cases. Conclusion: Considering the limitations imposed by the rarity of PD, our data suggest that GAA ERT improves 6MWT, physical QOL, and TOV in LOPD patients. ERT was safe in the studied population. PROSPERO register: 135102.
BackgroundMore than 50% of patients infected with chronic hepatitis C virus (HCV) do not respond to treatment with conventional interferon (IFN) combined with ribavirin (RBV). The aim of our study was to evaluate the effectiveness of retreatment with peginterferon alfa-2a or 2b (PEG-IFN 2a or 2b) concomitantly with RBV in patients with HCV genotype 2 and 3, which were non-responders or relapsers to initial treatment with IFN / RBV and to identify possible predictors of sustained virological response (SVR).MethodsFrom September 2003 to March 2009 a cohort of 216 patients who had previously failed therapy with a regimen of standard interferon and ribavirin, were followed in a specialized service implemented in the Brazilian Unified Health System, Rio Grande do Sul. All patients were retreated with PEG-IFN 2a or 2b per week, associated with RBV, through oral route, with doses determined according to weight (1,000 mg if weight ≤ 75 Kg and 1,250 mg if ≥ 75 Kg) per day for 48 weeks. The HCV-RNA was tested by Polymerase Chain Reaction (PCR). Virological Response (VR) within 48 weeks and SVR in the 72 weeks was considered for evaluation of treatment efficacy. Analyses were performed in patients who received at least one dose of PEG-IFN.ResultsThe SVR rate for non-responders to previous treatment was 34.4% and for relapsers was 50% (p = 0.031). As predictive factors that contribute to improve SVR, were identified the age (p = 0.005), to be relapsers to previous treatment (p = 0.023) and present liver biopsy examination Metavir F0-F2 (p = 0.004). In assessing the safety profile, 51 patients (23.6%) discontinued treatment prematurely.ConclusionsThis alternative retreatment for patients who have failed prior therapies for anti-HCV, has demonstrated promising SVR rate, provided that it includes a careful selection of patients with predictors of response and adverse events monitored.
BackgroundBehçet's disease (BD) is a multisystem disease in which central nervous system involvement – neuro-Behçet's disease (NBD) – may strike young patients with devastating consequences. In this regard, early diagnosis and treatment is essential to prevent injury.ObjectivesThis study aimed to analyze NBD clinical features compared to non-neurological BD in order to distinguish disease patterns.MethodsA retrospective study was performed in 101 BD outpatients from a single tertiary center followed between 2011 and 2016. BD diagnosis was based on the 2014 International Criteria for Behçet's Disease. Demographic, clinical and imaging features of 28 NBD patients were compared to 73 BD patients without neurological involvement.ResultsEarlier disease onset was found in NBD compared to BD (26.0±10.2 vs. 30.2±8.8 years, p=0.04). There were no differences between genders incidences, with a female predominance in both groups (64.3 vs. 72.6%, p>0.05). Over half of patients (53.6%) presented NBD as the first symptom and the mean time between diagnosis and NBD onset was 3.8±5.9 years. Uveitis was less frequent in NBD patients (25% vs. 47.9%, p=0.04), together with cutaneous disease (50% vs. 76.7%, p=0.01) and articular involvement (17.9% vs. 46.5%, p=0.01). Oral ulcers, genital ulcers, intestinal and vascular involvement frequencies were similar in both groups. Regarding NBD presentation, brainstem involvement was the most prevalent (67.9%), followed by central venous thrombosis (32.1%), aseptic meningitis (17.9%), stroke (3.6%) and peripheral neuropathy (3.6%). Most patients (82.1%) had a single neurological attack whereas relapsing disease was found in 18.5%.ConclusionsOur study found an earlier disease onset in NBD patients and a lower frequency of ocular, cutaneous and articular involvement. Moreover, several patients may unfold the disease as NBD, with lack of other manifestations. In addition, brainstem lesions occurred in most patients. Recognizing these disease patterns might support to expedite NBD diagnosis.Disclosure of InterestNone declared
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