Coelho T.,Hospital de Santo Antonio |
Maia L.F.,Hertie Institute for Clinical Brain Research |
Da Silva A.M.,Hospital de Santo Antonio |
Cruz M.W.,Hospital Universitario Clementino Fraga Filho |
And 13 more authors.
Neurology | Year: 2012
Objectives: To evaluate the efficacy and safety of 18 months of tafamidis treatment in patients with early-stage V30M transthyretin familial amyloid polyneuropathy (TTR-FAP). Methods: In this randomized, double-blind trial, patients received tafamidis 20 mg QD or placebo. Coprimary endpoints were the Neuropathy Impairment Score-Lower Limbs (NIS-LL) responder analysis (<2-point worsening) and treatment-group difference in the mean change from baseline in Norfolk Quality of Life-Diabetic Neuropathy total score (TQOL) in the intent-to-treat (ITT) popuation (n = 125). These endpoints were also evaluated in the efficacy-evaluable (EE; n = 87) population. Secondary endpoints, including changes in neurologic function, nutritional status, and TTR stabilization, were analyzed in the ITT population. Results: There was a higher-than-anticipated liver transplantation dropout rate. No differences were observed between the tafamidis and placebo groups for the coprimary endpoints, NIS-LL responder analysis (45.3% vs 29.5% responders; p = 0.068) and change in TQOL (2.0 vs 7.2; p = 0.116) in the ITT population. In the EE population, significantly more tafamidis patients than placebo patients were NIS-LL responders (60.0% vs 38.1%; p = 0.041), and tafamidis patients had better-preserved TQOL (0.1 vs 8.9; p = 0.045). Significant differences in most secondary endpoints favored tafamidis. TTR was stabilized in 98% of tafamidis and 0% of placebo patients (p < 0.0001). Adverse events were similar between groups. Conclusions: Although the coprimary endpoints were not met in the ITT population, tafamidis was associated with no trend toward more NIS-LL responders and a significant reduction in worsening of most neurologic variables, supporting the hypothesis that preventing TTR dissociation can delay peripheral neurologic impairment. Classification of evidence: This study provides Class II evidence that 20 mg tafamidis QD was associated with no difference in clinical progression in patients with TTR-FAP, as measured by the NIS-LL and the Norfolk QOL-DN score. Secondary outcomes demonstrated a significant delay in peripheral neurologic impairment with tafamidis, which was well tolerated over 18 months. Copyright © 2012 by AAN Enterprises, Inc.
Garnica M.,Federal University of Rio de Janeiro |
Nucci M.,Federal University of Rio de Janeiro |
Nucci M.,Hospital Universitario Clementino Fraga Filho
Current Fungal Infection Reports | Year: 2013
Fusarium spp. are molds widely distributed in nature as soil saprophytes. Human infections by Fusarium spp. occur both in immunocompetent and immunocompromised hosts. The most common forms in immunocompetent individuals are onychomycosis and keratitis. By contrast, disseminated fusariosis affects the immunocompromised host, especially hematopoietic cell transplant recipients and patients with acute leukemia. Severe neutropenia and T-cell immunodeficiency are the most important predisposing factors. Infection in compromised hosts is frequently fatal, and successful outcome is largely determined by the degree and persistence of immunosuppression and the extent of infection. The frequency of invasive fusariosis is increasing in some regions, especially in South America. © 2013 Springer Science+Business Media New York.
Guerra R.L.,Federal University of Rio de Janeiro |
Dorman S.E.,Center for Tuberculosis Research |
Luiz R.R.,Hospital Universitario Clementino Fraga Filho |
Conde M.B.,Federal University of Rio de Janeiro
International Journal of Tuberculosis and Lung Disease | Year: 2013
SETTING: Primary health care unit in Rio de Janeiro City, Brazil. OBJECTIVE: To estimate and compare the cost-effectiveness of strategies used for passive case finding of pulmonary tuberculosis (PTB) cases using tests available at the primary care level. DESIGN: Data on PTB suspects were reviewed, and a decision model was developed using sputum smear microscopy and chest radiography (CXR) according to three different strategies for PTB detection. A cost-effectiveness analysis was performed to estimate the cost per correct PTB diagnosis. Mycobacterial culture was used to calculate the effectiveness of the strategies. Unit costs of health resource utilisation were obtained from the payer's perspective (the Brazilian Public Health System). RESULTS: For the evaluation of 254 PTB suspects, the total costs of strategies ranged from US$5369 to US$5944; the probability of a correct PTB diagnosis ranged from 0.66 to 0.86; the number of visits required to complete the diagnostic process ranged from two to three, and cost per PTB case identified ranged from US$47.93 to US$53.07. The cost-effectiveness of the three strategies studied varied between US$56.69 and US$72.55 per correct PTB case detected. CONCLUSION: A strategy in which sputum smears and CXR were requested for all PTB suspects at the initial evaluation was cost-effective, had a high probability of correct PTB diagnosis and could be accomplished in two visits. © 2013 The Union.
Nucci M.,Hospital Universitario Clementino Fraga Filho
Current Fungal Infection Reports | Year: 2011
Persistent candidemia refers to the continued isolation of the same Candida species in the blood of a candidemic patient. Its incidence and clinical consequences are not well understood because of the lack of a homogeneous definition for persistent candidemia and the absence of prospective studies in which blood cultures were performed at prespecified times. Data from randomized clinical trials indicate that between 8% and 15% of candidemic patients have persistently positive blood cultures at the end of treatment, and the few studies that have attempted to evaluate the impact of persistent candidemia on outcomes have lacked appropriate analysis to conclude whether persistent candidemia is associated with worse outcome. On the other hand, assuming that it represents therapeutic failure, major causes for persistent candidemia include host factors (probably the most important), drug resistance, low serum levels of drugs, endovascular infection, deep-tissue abscesses, and infection associated with prosthetic material. © 2010 Springer Science+Business Media, LLC.
Fernandes N.C.,Federal University of Rio de Janeiro |
De Andrade L.R.,Hospital Universitario Clementino Fraga Filho
Anais Brasileiros de Dermatologia | Year: 2010
Reticulate acropigmentation of Dohi is a rare dyschromic disorder of autosomal dominant inheritance. Most cases have been originally described in Japan. The case of a girl with lesions of typical distribution and morphology is reported. Skin biopsy was not considered essential for diagnosis. After literature review, it was concluded that this is the third case of the disorder reported in Brazil. ©2010 by Anais Brasileiros de Dermatologia.