A systematic review of new advances in the management of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): focus on galsulfase

Regina P El Dib1, Gregory M Pastores21Department of Surgery, McMaster University, McMaster Institute of Urology, Hamilton, Ontario, Canada; 2Department of Neurology and Pediatrics, New York University School of Medicine, New York, NY, USAIntroduction: Mucopolysaccharidosis type VI (MPS VI, Maroteaux-Lamy syndrome) is an autosomal recessive lysosomal storage disorder, characterized primarily by skeletal dysplasia and joint contracture. It is caused by a deficiency of N-acetylgalactosamine-4-sulfatase (arylsulfatase B), for which a recombinant formulation (galsulfase) is available as replacement therapy. Objective: To evaluate the effectiveness and safety of galsulfase compared to placebo or no interventions, for treating MPS VI. We also considered studies evaluating different doses of galsulfase.Methods: A systematic review of the literature was conducted. A computerized electronic search in MEDLINE, EMBASE, CENTRAL, SciELO, and LILACS was carried on to identify any randomized trials that met our inclusion criteria.Results: Two studies were included in the review. Because the number of studies was small, our analysis probably did not find any statistically significant difference. Long-term follow-up will be required to ascertain full clinical benefit, on both event-free survival and quality of life measures.Conclusions: There is some evidence to support the use of galsulfase in the treatment of MPS VI; however due to the very low quantity of included studies we could not analyze it in an appropriate way. This review highlights the need for continued research into the use of enzyme replacement therapy for MPS VI.Keywords: mucopolysaccharidosis VI, Maroteaux-Lamy syndrome, galsulfase, naglazyme, systematic revie

Efetividade da higienização de brinquedos infantis na redução microbiana: revisão sistemática da literatura

Objective: It is to assess the effectiveness of the sanitation of infant toys, when compared to no intervention in the reduction of the microbial load of the toys, in different environments where the care of children exists. Method: It is a systematic review of the literature from 2003 to 2013, held through the bases of data SciELO, LILACS, IBECS, MEDLINE, EMBASE, Scopus, PUBMED and through dissertation and theses banks of USP, UNESP and UNICAMP digital libraries. The obtained data were tabulated and submitted to the analysis of subgroups, which was done descriptively. Results: In total, 7762 researches were raises, which after evaluation of the reviewers, resulted in 12 studies relevant to the theme. Predominant were the comparative researches of the microbial loads before and after the intervention of the sanitation of the toys, and the methods of sanitation were much varied. Conclusion: Several sanitation methods were consideres effective, however the heterogeneity of methods didn´t make possible the identification of the best evidence, showing the need of larger investigation for the elaboration of sanitation strategies of infant toys.Objetivo: Avaliar a efetividade de estratégias de higienização de brinquedos infantis, quando comparadas a nenhuma intervenção na redução da carga microbiana dos brinquedos, em diferentes ambientes onde há o cuidado de crianças. Método: Revisão sistemática da literatura de 2003 a 2013, realizada nas bases de dados SciELO, Lilacs, IBECS, MEDLINE, Embase, Scopus, PubMed e bancos de dissertações e teses das bibliotecas digitais da USP, Unesp e Unicamp. Os dados foram tabulados e submetidos a análise de subgrupos de forma descritiva. Resultados: Foram levantados 7762 trabalhos, que após avaliação dos revisores, resultaram em 12 pesquisas relevantes ao tema. Predominantemente estudos comparativos de cargas microbianas, com grande variabilidade metodológica, trazendo propostas de higienização diversas. Conclusão: Várias estratégias de higienização foram consideradas eficazes, porém a heterogeneidade de métodos não possibilitou a identificação da melhor evidência, mostrando a necessidade de maior investigação do tema para a elaboração de estratégias de higienização de brinquedos infantis

Evaluación del uso de collar cervical de inmovilización después de la descompresión y fusion cervical por acceso anterior: Revisión sistemática

This study aims to evaluate safety and effectiveness of the use of orthesis in postoperative degenerative disorders of the cervical spine. Although widely used, there are not defined criteria for the use of cervical collars, and their importance on cervical arthrodesis. A systematic literature review was undertaken and a multicentre controlled clinical trial comprising 32 specialized services, with a total of 257 patients who met the inclusion criteria of the review. Clinical and radiographic outcomes were compared in post surgical patients that used or not rigid cervical collar during 6 to 12 weeks after surgery. The evidence available on literature, although weak, suggests that cervical collar decreases the clinical scores in first weeks after surgery. Furthermore, it does not alter the rate of fusion when used concomitantly with plaque and screws through anterior access in cervical degenerative diseases on a single anatomical level.Este estudio tiene como objetivo evaluar la eficacia y seguridad de la utilización de ortesis después de la cirugía de trastornos degenerativos de la columna cervical. Aunque ampliamente utilizado, no existen criterios para la aplicación de collares cervicales y su importancia en la artrodesis cervical. Se realizó una revisión sistemática de la literatura y un estudio multicéntrico controlado, compuesto de 32 departamentos, con un total de 257 pacientes que cumplieron los criterios de inclusión de la revisión. Se compararon los resultados clínicos y radiográficos de pacientes, en un solo nivel anatómico, que usaron o no el collar cervical duro durante 6 a 12 semanas después de la cirugía. La evidencia disponible en la literatura, aunque débil, sugiere que el uso del collar cervical reduce las puntuaciones clínicas en las primeras semanas después de la cirugía. Además, no altera la tasa de fusión cuando se utiliza de forma concomitante con la placa y tornillos colocados por acceso anterior en la enfermedad degenerativa cervical en un solo nivel anatómico.Este estudo tem como objetivo avaliar a efetividade e a segurança do uso de órteses no pós-operatório de afecções degenerativas da coluna cervical. Apesar de amplamente utilizados, não existem critérios definidos para a aplicação de colares cervicais e sua relevância na artrodese cervical. Foi realizada uma revisão sistemática da literatura, além de um estudo multicêntrico controlado, composto por 32 serviços, com um total de 257 pacientes que satisfizeram os critérios de inclusão da revisão. Foram comparados desfechos clínicos e radiográficos de pacientes operados em um único nível anatômico que fizeram uso ou não de colar cervical rígido por 6 a 12 semanas após a cirurgia. As evidências disponíveis na literatura, apesar de fracas, sugerem que o uso do colar cervical diminui os escores clínicos nas primeiras semanas após o procedimento cirúrgico. Além disto, não altera a taxa de fusão quando utilizado concomitantemente à placa e aos parafusos por acesso anterior, em doenças degenerativas cervicais em um único nível anatômico.Santa Casa da Misericórdia de SantosUniversidade Estadual PaulistaHospital do Servidor Público Estadual Francisco Morato de OliveiraUniversidade Federal de São Paulo (UNIFESP)UNIFESP, ISS, SantosSciEL

Analisis isi buku “Islam Kita, Islam Nusantara” karya Mohamad Guntur Romli

Munculnya wacana tentang Islam Nusantara pada tahun 2015 sempat memancing munculnya berbagai reaksi dari kalangan muslim di Negeri ini. Pro dan Kontra terus saja bergulir, bahkan hingga saat ini. Meski begitu bagi para pendukung gagasan Islam Nusantra, wacana tersebut justru harus dihadirkan sebagai solusi atas permasalahan umat. Atas dasar hal itu, Guntur Romli kemudian menyajikan sebuah buku berjudul “Islam Kita, Islam Nusantara” sebagai sebuah jawaban atas tudingan miring kepada para pengusung gagasan Islam Nusantara yang notabene sama sekali tidak merubah ajaran Islam, sebaliknya justru menghadirkan Islam rahamatan lil alamin dalam bingkai kearifan hazanah bangsa. Berangkat dari fenomena tersebut, maka peneliti berusaha mengkaji dan mengetahui isi dari buku “Islam Kita, Islam Nusantara”. Metode yang digunakan adalah analisis isi sebagai pisau analisa kulaitatif, diharapkan peneliti mampu menemukan dan merefleksikan esensi dan subtasnsi dari Buku “Islam Kita, Islam Nusantara” itu sendiri. Dari penelitian ini dapat diketahui bahwa bahwa Islam Nusantara yang terdapat dalam buku karya Guntur Romli yang berjudul “Islam Kita, Islam Nusantara” adalah sebagai berikut:Pertama,Islam Nusantara Bukanlah aliran baru atau firqoh melainkan sebuah cara berislam yang sesuai dengan manhaj ahlussunnah wal jama’ah serta dilaksanakakn dalam konteks budaya lokal nusantara ( selama budaya itu tidak bertentangan dengan hukum syari’at)Kedua,Perbedaan antatra kaum tradisional dan modernis yang pernah terjadi pada paruh awal abad ke-20 tentu sudah tidak lagi relevan dengan kondisi saat ini, dimana NU sebagai kelompok yang di identikan dengan tradisionalis dan Muhammadiyah sebagai modernis justru sudah mampu berelaborasi dalam menampakkan wajah Islam yang Rahmatan Lil ‘Alamin.Ketiga,Islam Nusantara terbukti mampu menunjukan sinergi dengan nilai-nilai esensial Islam, Pancasila dan HAM dengan berbagai dinamika sosial yang terjadi di Negara Kesatuan Republik Indonesia. Hal ini bisa terwujud karena Islam Nusantara memiliki karakter Toleran, tasamuh, tawasuth (moderat), tawazun (berimbang)

Efficacy and safety of blood transfusion in obstetric patients: systematic review of the literature

Objectives: To evaluate the efficacy of blood transfusion compared to no intervention in obstetric patients. Material and methods: A systematic review was performed with Cochrane Database of Clinical Trials, PubMed, EMBASE and LILACS databases searched as of September, 2016. Two authors independently selected relevant clinical trials, assessed their methodological quality and extracted data, using the GRADE approach. Results: Five studies within a total of 6,297 met the inclusion criteria, with women generally aged 20–40 years. Three included studies allocated women to receive blood transfusion or no intervention. Two other studies allocated women with either restricted or full blood supplies. The major issue regarding risk of bias was the extent of concealment of randomization and blinding. There was no statistically significant difference between blood transfusion versus no transfusion or restricted blood supply on mortality (relative risk 0.82 [95% confidential interval 0.32 to 2.09], p = 0.68; two studies; I2 = not applicable). Conclusions: Very low-quality evidence suggests no significant difference between blood transfusion and no intervention in obstetric patients, underlining the need for more robust clinical trials evaluating this area

Electronic nicotine delivery systems and/or electronic non-nicotine delivery systems for tobacco smoking cessation or reduction: a systematic review and meta-analysis

Objective A systematic review and meta-analysis to investigate the impact of electronic nicotine delivery systems (ENDS) and/or electronic non-nicotine delivery systems (ENNDS) versus no smoking cessation aid, or alternative smoking cessation aids, in cigarette smokers on long-term tobacco use. Data sources Searches of MEDLINE, EMBASE, PsycInfo, CINAHL, CENTRAL and Web of Science up to December 2015. Study selection Randomised controlled trials (RCTs) and prospective cohort studies. Data extraction Three pairs of reviewers independently screened potentially eligible articles, extracted data from included studies on populations, interventions and outcomes and assessed their risk of bias. We used the Grading of Recommendations Assessment, Development and Evaluation approach to rate overall certainty of the evidence by outcome. Data synthesis Three randomised trials including 1007 participants and nine cohorts including 13?115 participants proved eligible. Results provided by only two RCTs suggest a possible increase in tobacco smoking cessation with ENDS in comparison with ENNDS (RR 2.03, 95% CI 0.94 to 4.38; p=0.07; I2=0%, risk difference (RD) 64/1000 over 6 to 12?months, low-certainty evidence). Results from cohort studies suggested a possible reduction in quit rates with use of ENDS compared with no use of ENDS (OR 0.74, 95% CI 0.55 to 1.00; p=0.051; I2=56%, very low certainty). Conclusions There is very limited evidence regarding the impact of ENDS or ENNDS on tobacco smoking cessation, reduction or adverse effects: data from RCTs are of low certainty and observational studies of very low certainty. The limitations of the cohort studies led us to a rating of very low-certainty evidence from which no credible inferences can be drawn. Lack of usefulness with regard to address the question of e-cigarettes\u27 efficacy on smoking reduction and cessation was largely due to poor reporting. This review underlines the need to conduct well-designed trials measuring biochemically validated outcomes and adverse effects

Differences between the real and the desired worlds in the results of clinical trials

OBJECTIVE:We refer to the effectiveness (known as pragmatic or real world) and efficacy (known as explanatory or desired or ideal world) of interventions. However, these terms seem to be randomly chosen by investigators who design clinical trials and do not always reflect the true purpose of the study. A pragmatic-explanatory continuum indicator summary tool was thus developed with the aim of identifying the characteristics of clinical trials that distinguish between effectiveness and efficacy issues. We verified whether clinical trials used the criteria proposed by the indicator summary tool, and we categorized these clinical trials according to a new classification.METHOD:A systematic survey of randomized clinical trials was performed. We added a score ranging from 0 (more efficacious) to 10 (more effective) to each domain of the indicator summary tool and proposed the following classifications: high efficacy

Pelvic floor and abdominal muscle cocontraction in women with and without pelvic floor dysfunction: a systematic review and meta-analysis

There is an ongoing discussion regarding abdominal muscle (AbM) and pelvic floor muscle (PFM) synergism. Therefore, this study aimed to investigate the cocontraction between AbMs and PFMs in women with or without pelvic floor dysfunction (PFD). The following databases were searched up to December 21, 2018: MEDLINE, EMBASE, LILACS, PEDro and CENTRAL. We included any study that assessed the cocontraction between PFMs and AbMs in women with and without PFD. Two reviewers independently screened eligible articles and extracted data. The outcomes were extracted and analyzed as continuous variables with random effect models. Twenty studies were included. A meta-analysis did not show differences in women with and without PFD. However, a sensitivity analysis suggested cocontraction of the transversus abdominis (TrA) during PFM contraction in healthy women (standardized mean difference (SMD) 1.02 [95% confidence interval (CI) 1.90 to 0.14], P=0.02; I2 = not applicable; very low quality of evidence). Women with PFD during contraction of PFMs showed cocontraction of the obliquus internus (OI) (SMD 1.10 [95% CI 0.27 to 1.94], P=0.01; I2 = not applicable; very low quality of evidence), and obliquus externus (OE) (SMD 2.08 [95% CI 1.10 to 3.06], Po0.0001; I 2 = not applicable; very low quality of evidence). Increased cocontraction of the TrA may be associated with maximal contraction of PFMs in women without PFD. On the other hand, there is likely an increased cocontraction with the OI and OE in women with PFD