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Negra M.D.,Institute Infectologia Emilio Ribas | De Carvalho A.P.,Hospital Infantil Joana de Gusmao | De Aquino M.Z.,Instituto da Crianca Do Hospital das Clinicas da FMUSP | Da Silva M.T.N.,University of Campinas | And 8 more authors.
Pediatric Infectious Disease Journal | Year: 2012

Background: There are few data on the safety and antiviral activity of tenofovir disoproxil fumarate (TDF) in HIV-1 infected adolescents. Methods: A randomized, double-blinded, placebo-controlled study was conducted. Ninety adolescents (12 to <18 years) who were viremic while receiving antiretroviral treatment were randomized to receive TDF 300 mg (mean, 216.8 mg/m) or placebo in combination with an optimized background regimen (OBR) for 48 weeks. The primary efficacy endpoint was time-weighted average change in plasma HIV-1 RNA from baseline at week 24 Results: Eighty-seven subjects (45 TDF, 42 placebo) received the study drug. Through week 24, the median time-weighted average change in plasma HIV-1 RNA was not different between the TDF and placebo groups (-1.6 versus-1.6 log10copies/mL, P = 0.55). The percentages of subjects who achieved HIV-1 RNA <400 copies/mL were similar at week 24 (40.9 versus 41.5%). One fourth of subjects in the TDF and placebo groups (24.4 versus 28.6%) had at least 3 active agents in the OBR. Many subjects in both groups had baseline genotypic resistance to TDF (48.9 versus 33.3%). TDF was generally safe and well tolerated. There were no statistically significant differences in changes of renal function and bone mineral density between the 2 groups. Conclusion: This study of TDF in combination with an OBR in antiretroviral- experienced adolescents did not meet its primary or secondary efficacy endpoints. The effectiveness of the OBR and baseline genotypic resistance to TDF in both groups may have confounded the efficacy findings. No clinically relevant TDF-related renal or bone toxicities were observed in this adolescent population. Copyright © 2012 by Lippincott Williams & Wilkins. Source


Giugliani R.,Medical Genetics Service | Giugliani R.,Federal University of Rio Grande do Sul | Giugliani R.,National Institute of Population Medical Genetics | Herber S.,Research and Postgraduation Group | And 3 more authors.
Pediatric Endocrinology Reviews | Year: 2014

Mucopolysaccharidosis VI (MPS VI) is a lysosomal storage disorder caused by deficient activity of Arylsulphatase B (ARSB). The disease is progressive and multisystemic, usually leading to death in the first decades of life. In addition to supportive management, specific treatments for MPS VI are the hematopoietic stem cell transplantation (HSCT) and enzyme replacement therapy (ERT). Both are effective for some aspects of the disease, but fail in correcting important clinical features, such as bone deformities and heart valve thickening. Based on that, new treatments are currently being tested to be used alone or in combination with the current therapies. Here we summarize some of these new approaches and the preliminary results obtained, reporting their limitations and indicating possible future trends in MPS VI treatment. We discuss intrathecal ERT, gene therapy and therapies based on anti-inflammatory molecules, among other approaches. Finally, we highlight the importance of early treatment and diagnosis for a better outcome in these patients. Source


Neto A.K.,Servico de Ortopedia e Traumatologia | Oliveira M.A.,Medico Ortopedista e Traumatologista | Stipp W.N.,Hospital Infantil Joana de Gusmao
Revista Brasileira de Ortopedia | Year: 2011

Objective: Presenting a retrospective review of the results of treatment of children with septic arthritis of the hip and the investigation of the relationship between prognostic factors and the clinical results obtained. Methods: The patients evaluated (46) comprising 47 hips, who attended Orthopedics HIJG's ambulatory clinic, answered a questionnaire, underwent physical examinations and radiographs of the pelvis, as well as the review of data from medical records. The patients were classified in terms of clinical and radiographic point of view according to both the classifications of Hunka and Choi, and were divided into groups for statistical comparison. Group 1 consisted of patients classified as Type IA and group 2 consisted of patients classified as Type IB to Type IVB. Results: The disease affected 30 boys, affecting the right hip in 26 children and both sides in one child. The average age at the presentation was 50 months, being higher in group 1 (p = 0.023). The synovial fluid culture was positive in 23 patients and Staphylococcus aureus grew in 18 of them. Symptoms lasted on average for 3.5 days before surgery, the wait being shorter in group 1 (p = 0.03). The treatment outcome was satisfactory in most cases. Conclusion: Young patients, who did not limp during the initial medical exam, with synovial fluid culture positive for Staphylococcus aureus and delay in undergoing surgical treatment, were associated with poor outcomes. Source


Della Negra M.,Institute Infectologia Emilio Ribas | Carvalho A.P.,Hospital Infantil Joana de Gusmao | De Aquino M.Z.,Instituto da Crianca Do Hospital das Clinicas da FMUSP | Pinto J.A.,Federal University of Minas Gerais | And 5 more authors.
Pediatric Infectious Disease Journal | Year: 2015

Background: Reports of long-term tenofovir disoproxil fumarate (TDF) treatment in HIV-infected adolescents are limited. We present final results from the open-label (OL) TDF extension following the randomized, placebo (PBO)-controlled, double-blind phase of GS-US-104-0321 (Study 321). Methods: HIV-infected 12- to 17-year-olds treated with TDF 300 mg or PBO with an optimized background regimen (OBR) for 24-48 weeks subsequently received OL TDF plus OBR in a single arm study extension. HIV-1 RNA and safety, including bone mineral density (BMD), was assessed in all TDF recipients. Results: Eighty-one subjects received TDF (median duration 96 weeks). No subject died or discontinued OL TDF for safety/tolerability. At week 144, proportions with HIV-1 RNA <50 copies/mL were 30.4% (7 of 23 subjects with baseline HIV-1 RNA >1000 c/mL initially randomized to TDF), 41.7% (5 of 12 subjects with HIV-1 RNA <1000 c/mL who switched PBO to TDF) and 0% (0 of 2 subjects failed randomized PBO plus OBR with HIV-1 RNA >1000 c/mL and switched PBO to TDF). Viral resistance to TDF occurred in 1 subject. At week 144, median decrease in estimated glomerular filtration rate was 38.1 mL/min/1.73 m2 (n = 25). Increases in median spine (+12.70%, n = 26) and total body less head BMD (+4.32%, n = 26) and height-age adjusted Z-scores (n = 21; +0.457 for spine, +0.152 for total body less head) were observed at week 144. Five of 81 subjects (6%) had persistent >4% BMD decreases from baseline. Conclusions: Some subjects had virologic responses to TDF plus OBR, and TDF resistance was rare. TDF was well tolerated and can be considered for treatment of HIV-infected adolescents. © 2015 Wolters Kluwer Health, Inc. Source


De Lima L.R.A.,Federal University of Santa Catarina | De Lima L.R.A.,Health Science University | Da Silva R.C.R.,Federal University of Santa Catarina | De C.B. Giuliano I.,Federal University of Santa Catarina | And 4 more authors.
Jornal de Pediatria | Year: 2013

Objective: To describe bone mineral density (BMD) and bone mineral content (BMC) in children and adolescents infected with the human immunodeficiency virus (HIV), and to compare them with data from the National Health and Nutrition Examination Survey IV (NHANES IV). Method: The study included 48 children and adolescents (7 to 17 years old) infected with HIV through vertical transmission. BMC and BMD were measured by dual energy absorptiometry X-ray, by calculating z-scores based on data from NHANES IV. The information on clinical and laboratory parameters of infection by HIV was obtained from medical records. Physical activity, calcium intake, and skeletal maturation were also assessed. Descriptive and inferential statistical procedures were used, with levels of significance set at 5%. Results: Seropositive patients presented lower values compared to data from NHANES IV in all z-scores of bone mass (mean = -0.52 to -1.22, SD = 0.91 and 0.84, respectively). Based on the subtotal z-BMD, there was a prevalence of 16.7% of children and adolescents with low bone mass for age. Individuals using protease inhibitors presented a lower total z-BMD when compared to the group that did not use (-1.31 vs. -0.79, p = 0.02). There were no bone mass differences in relation to physical activity and calcium intake. Conclusions: In the present sample children and adolescents living with HIV have low bone mass for age, and the use of protease inhibitors appears to be related to such decreases. Copyright © 2013 Sociedade Brasileira de Pediatria. Published by Elsevier Editora Ltda. All rights reserved. Source

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