Impact of achondroplasia on Latin American patients: a systematic review and meta-analysis of observational studies.

BACKGROUND: Achondroplasia (ACH), the most common form of disproportionate short stature, is caused by a pathogenic variant in the fibroblast growth factor receptor 3 gene. Recent advances in drug therapy for ACH have highlighted the importance of elucidating the natural history and socioeconomic burden of this condition. Recognition that there are many potential issues for the patient with ACH is the first step in planning cost-effective interventions in Latin America (LATAM), a vast geographic territory comprising countries with multicultural characteristics and wide socioeconomic differences. We conducted a systematic literature review to characterize the impact of ACH on affected individuals and on healthcare resources in LATAM countries. METHODS: Searches of the global medical literature as well as regional and local medical literature up to August 2020. Observational studies on patients with ACH from any LATAM country. Pairs of reviewers independently screened eligible articles, extracted data from included studies, and assessed their risk of bias. RESULTS: Fifty-three unique studies (28 case series and cross-sectional studies and 25 case reports) including data on 1604 patients were eligible. Of these studies, 11 had data available for meta-analysis. Both premature mortality and all-cause mortality in the pooled studies was 15% [95% Confidence Interval (CI) 1.0E-3 to 0.47; I2 = 82.9%, p = 0.0029; three studies, n = 99 patients]. Frequency of cardio-respiratory-metabolic disorders was 17% [95% CI 0.04-0.37; I2 = 90.3%, p < 0.0001; four studies, n = 230 patients]; nervous system disorders was 18% [95% CI 0.07-0.33; I2 = 84.6%, p < 0.0001; six studies, n = 262 patients]; ear, nose, throat and speech disorders was 32% [95% CI 0.18-0.48; I2 = 73.4%, p = 0.0046; five studies, n = 183 patients]; and spinal issues including stenosis, compression and associated pain was 24% [95% CI 0.07-0.47; I2 = 91.3%, p < 0.0001; five studies, n = 235 patients]. CONCLUSIONS: There is currently evidence of high clinical burden in ACH patients in LATAM countries. Establishing the impact of ACH provides the necessary foundation for planning tailored and effective public health interventions.

Probing for congenital nasolacrimal duct obstruction: a systematic review and meta-analysis of randomized clinical trials.

PURPOSE: Lacrimal probing is the treatment of choice for congenital nasolacrimal duct obstruction that does not have a spontaneous resolution; however, there is no consensus about the best time for probing and if it is superior to other therapies. The present study aimed to evaluate the effectiveness of lacrimal probing compared with other treatments/no intervention to treat congenital nasolacrimal duct obstruction. METHODS: A systematic review of literature in PubMed, EMBASE, CENTRAL, clinicaltrials.gov, and LILACS databases up to December 2019 was performed. Randomized clinical trials that enrolled children diagnosed with congenital nasolacrimal duct obstruction and undergoing lacrimal probing were considered. Data extraction and a risk of bias assessment were conducted independently and in duplicate. The overall quality of evidence for each outcome was conducted using the Grading of Recommendations, Assessment, Development, and Evaluation classification system. RESULTS: Four randomized clinical trials involving 423 participants were eligible. No statistically significant differences were observed in resolution rates between early probing and observation/late probing (two studies; risk ratio 1.00 [95% confidence interval 0.76-1.33]; p=0.99; low certainty evidence). One study reported better resolution rates with bicanalicular silicone stent intubation compared with late probing in the complex congenital nasolacrimal duct obstruction cases subgroup (risk ratio 0.56 [95% confidence interval 0.34-0.92]; p=0.02; moderate certainty evidence). CONCLUSIONS: Low certainty evidence suggests that early probing has the same success rate as late probing. Evidence of moderate certainty suggests that late probing has a lower success rate than bicanalicular silastic intubation in patients with complex congenital nasolacrimal duct obstructione.

Probing for congenital nasolacrimal duct obstruction: a systematic review and meta-analysis of randomized clinical trials / Sondagem no tratamento da obstrução lacrimonasal congênita: revisão sistemática de ensaios clínicos randomizados e metanálise

ABSTRACT Purpose: Lacrimal probing is the treatment of choice for congenital nasolacrimal duct obstruction that does not have a spontaneous resolution; however, there is no consensus about the best time for probing and if it is superior to other therapies. The present study aimed to evaluate the effectiveness of lacrimal probing compared with other treatments/no intervention to treat congenital nasolacrimal duct obstruction. Methods: A systematic review of literature in PubMed, EMBASE, CENTRAL, clinicaltrials.gov, and LILACS databases up to December 2019 was performed. Randomized clinical trials that enrolled children diagnosed with congenital nasolacrimal duct obstruction and undergoing lacrimal probing were considered. Data extraction and a risk of bias assessment were conducted independently and in duplicate. The overall quality of evidence for each outcome was conducted using the Grading of Recommendations, Assessment, Development, and Evaluation classification system. Results: Four randomized clinical trials involving 423 participants were eligible. No statistically significant differences were observed in resolution rates between early probing and observation/late probing (two studies; risk ratio 1.00 [95% confidence interval 0.76-1.33]; p=0.99; low certainty evidence). One study reported better resolution rates with bicanalicular silicone stent intubation compared with late probing in the complex congenital nasolacrimal duct obstruction cases subgroup (risk ratio 0.56 [95% confidence interval 0.34-0.92]; p=0.02; moderate certainty evidence). Conclusions: Low certainty evidence suggests that early probing has the same success rate as late probing. Evidence of moderate certainty suggests that late probing has a lower success rate than bicanalicular silastic intubation in patients with complex congenital nasolacrimal duct obstructione.

RESUMO Objetivo: A sondagem lacrimal tem sido o tratamento de escolha para a obstrução lacrimonasal congênita que não apresenta resolução espontânea. Contudo, não há consenso sobre qual é a melhor época para a realização da sondagem e se ela é melhor do que outras terapias. O objetivo foi avaliar a efetividade da sondagem lacrimal no tratamento da obstrução lacrimonasal congênita. Método: Uma revisão sistemática da literatura foi realizada usando as plataformas eletrônicas PubMed, EMBASE, CENTRAL, clinicaltrials.gov e LILACS até o período de dezembro de 2019. Foram considerados ensaios clínicos randomizados envolvendo crianças com obstrução lacrimonasal congênita submetidas a sondagem lacrimal. A extração dos dados e avaliação do risco de viés foram feitas por dois autores independentemente. A análise da qualidade da evidência para cada desfecho foi realizada por meio do sistema GRADE (Grading of Recommendations Assessment, Development and Evaluation). Resultados: Quatro ensaios clínicos randomizados foram incluídos, envolvendo 423 participantes. A metanálise mostrou que não houve diferença estatística na resolução da obstrução lacrimonasal congênita entre o grupo submetido à sondagem lacrimal precoce e o submetido à observação/sondagem tardia (2 estudos; risco médio 1.00 [intervalo de confiança de 95% 0.76, 1.33] p=0,99, I2=79%, baixa certeza de evidência). Um estudo evidenciou melhores resultados da intubação bicanalicular com silicone em comparação a sondagem tardia no subgrupo das obstruções lacrimonasais congênitas complexas, (1 estudo; risco médio 0.56 [intervalo de confiança de 95% 0.34, 0.92] p=0,02, moderada certeza de evidência). Conclusões: Há evidências de baixa qualidade de que a sondagem precoce tem a mesma taxa de sucesso que a sondagem tardia. Evidências de moderada certeza sugerem que a sondagem tardia tem menor chance de sucesso do que a intubação bicanalicular com silicone em casos de obstruções lacrimonasais congênitas complexas.

The ROPScore as a Screening Algorithm for Predicting Retinopathy of Prematurity in a Brazilian Population.

OBJECTIVES: To evaluate the accuracy of the ROPScore algorithm as a predictor of retinopathy of prematurity (ROP). METHODS: A prospective cohort of 220 preterm infants with a birth weight ≤1500 g and/or gestational age ≤32 weeks was included. The ROPScore was determined in the sixth week of life in 181 infants who then survived until a corrected gestational age of 45 weeks. The sensitivity, specificity, and positive (PPV) and negative predictive values (NPV) of the algorithm were analyzed. RESULTS: ROP was found in 17.6% of the preterm infants. The sensitivity of this test for any stage of ROP was 87.5%, while that for severe ROP was 95.4% (21/22 cases). The PPV and NPV were 59.6% and 97%, respectively, for any stage of ROP and 44.7% and 99.25%, respectively, for severe ROP. The ROPScore could therefore hypothetically reduce the number of ophthalmologic examinations required to detect ROP by 71.8%. CONCLUSION: The ROPScore is a useful screening tool for ROP and may optimize examinations and especially the identification of severe ROP.

Strategies to optimize MEDLINE and EMBASE search strategies for anesthesiology systematic reviews. An experimental study

ABSTRACT BACKGROUND: A high-quality electronic search is essential for ensuring accuracy and comprehensiveness among the records retrieved when conducting systematic reviews. Therefore, we aimed to identify the most efficient method for searching in both MEDLINE (through PubMed) and EMBASE, covering search terms with variant spellings, direct and indirect orders, and associations with MeSH and EMTREE terms (or lack thereof). DESIGN AND SETTING: Experimental study. UNESP, Brazil. METHODS: We selected and analyzed 37 search strategies that had specifically been developed for the field of anesthesiology. These search strategies were adapted in order to cover all potentially relevant search terms, with regard to variant spellings and direct and indirect orders, in the most efficient manner. RESULTS: When the strategies included variant spellings and direct and indirect orders, these adapted versions of the search strategies selected retrieved the same number of search results in MEDLINE (mean of 61.3%) and a higher number in EMBASE (mean of 63.9%) in the sample analyzed. The numbers of results retrieved through the searches analyzed here were not identical with and without associated use of MeSH and EMTREE terms. However, association of these terms from both controlled vocabularies retrieved a larger number of records than did the use of either one of them. CONCLUSIONS: In view of these results, we recommend that the search terms used should include both preferred and non-preferred terms (i.e. variant spellings and direct/indirect order of the same term) and associated MeSH and EMTREE terms, in order to develop highly-sensitive search strategies for systematic reviews.

The Ropscore as a screening algorithm for predicting retinopathy of prematurity in a brazilian population

OBJECTIVES: To evaluate the accuracy of the ROPScore algorithm as a predictor of retinopathy of prematurity (ROP). METHODS: A prospective cohort of 220 preterm infants with a birth weight ≤1500 g and/or gestational age ≤32 weeks was included. The ROPScore was determined in the sixth week of life in 181 infants who then survived until a corrected gestational age of 45 weeks. The sensitivity, specificity, and positive (PPV) and negative predictive values (NPV) of the algorithm were analyzed. RESULTS: ROP was found in 17.6% of the preterm infants. The sensitivity of this test for any stage of ROP was 87.5%, while that for severe ROP was 95.4% (21/22 cases). The PPV and NPV were 59.6% and 97%, respectively, for any stage of ROP and 44.7% and 99.25%, respectively, for severe ROP. The ROPScore could therefore hypothetically reduce the number of ophthalmologic examinations required to detect ROP by 71.8%. CONCLUSION: The ROPScore is a useful screening tool for ROP and may optimize examinations and especially the identification of severe ROP.

Anesthesia-Related and Perioperative Cardiac Arrest in Low- and High-Income Countries: A Systematic Review With Meta-Regression and Proportional Meta-Analysis.

The anesthesia-related cardiac arrest (CA) rate is a quality indicator to improve patient safety in the perioperative period. A systematic review with meta-analysis of the worldwide literature related to anesthesia-related CA rate has not yet been performed.This study aimed to analyze global data on anesthesia-related and perioperative CA rates according to country's Human Development Index (HDI) and by time. In addition, we compared the anesthesia-related and perioperative CA rates in low- and high-income countries in 2 time periods.A systematic review was performed using electronic databases to identify studies in which patients underwent anesthesia with anesthesia-related and/or perioperative CA rates. Meta-regression and proportional meta-analysis were performed with 95% confidence intervals (CIs) to evaluate global data on anesthesia-related and perioperative CA rates according to country's HDI and by time, and to compare the anesthesia-related and perioperative CA rates by country's HDI status (low HDI vs high HDI) and by time period (pre-1990s vs 1990s-2010s), respectively.Fifty-three studies from 21 countries assessing 11.9 million anesthetic administrations were included. Meta-regression showed that anesthesia-related (slope: -3.5729; 95% CI: -6.6306 to -0.5152; P = 0.024) and perioperative (slope: -2.4071; 95% CI: -4.0482 to -0.7659; P = 0.005) CA rates decreased with increasing HDI, but not with time. Meta-analysis showed per 10,000 anesthetics that anesthesia-related and perioperative CA rates declined in high HDI (2.3 [95% CI: 1.2-3.7] before the 1990s to 0.7 [95% CI: 0.5-1.0] in the 1990s-2010s, P < 0.001; and 8.1 [95% CI: 5.1-11.9] before the 1990s to 6.2 [95% CI: 5.1-7.4] in the 1990s-2010s, P < 0.001, respectively). In low-HDI countries, anesthesia-related CA rates did not alter significantly (9.2 [95% CI: 2.0-21.7] before the 1990s to 4.5 [95% CI: 2.4-7.2] in the 1990s-2010s, P = 0.14), whereas perioperative CA rates increased significantly (16.4 [95% CI: 1.5-47.1] before the 1990s to 19.9 [95% CI: 10.9-31.7] in the 1990s-2010s, P = 0.03).Both anesthesia-related and perioperative CA rates decrease with increasing HDI but not with time. There is a clear and consistent reduction in anesthesia-related and perioperative CA rates in high-HDI countries, but an increase in perioperative CA rates without significant alteration in the anesthesia-related CA rates in low-HDI countries comparing the 2 time periods.

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 (<25), moderate efficacy (25-50), moderate effectiveness (51-75), and high effectiveness (<75).RESULTS:A total of 844 randomized trials were analyzed. No analyzed trials used the criteria proposed by the indicator summary tool. Approximately 44% of the trials were classified as having moderate effectiveness, and 43.82% were classified as having moderate efficacy.CONCLUSIONS:Most clinical trials used the term “efficacy” to illustrate the application of results in clinical practice, but the majority of those were classified as having moderate effectiveness according to our proposed score. The classification based on the 0-100 score is still highly subjective and can be easily misunderstood in all domains based on each investigator’s own experiences and knowledge.

Review of the efficacy and safety of cryoablation for the treatment of small renal masses.

PURPOSE: Small renal masses are increasingly being discovered incidentally on imaging for another reason. The standard of care of these masses involves excision by open or laparoscopic techniques. Recently, ablative techniques, such as radiofrequency ablation (RFA) and cryoablation, have taken a more prominent role in the treatment algorithm of these masses. We evaluate the effectiveness and safety of cryoablation to treat renal tumours. METHODS: A review of the literature was conducted. There was no language restriction. Studies were obtained from the following sources: the Cochrane Library, PUBMED, EMBASE and LILACS. RESULTS: There was no clinical trial identified in the literature. Thus, we described the results from 23 case series and retrospective studies with a reasonable sample size (number of reported patients in each study ≥30), with a total of 2104 analyzed tumours from 2038 patients. There was wide variability in the outcomes reported, but success rates were generally good. Follow-up was generally short, but some series reported outcomes at 5 years. The most common complications reported were hemorrhage (some of the patients requiring transfusion), perinephric hematoma and urine leaks. CONCLUSION: Cryoablation presents a feasible treatment for patients with small renal masses. Only short-term data are available and, as such, meaningful conclusions regarding long-term cancer control cannot be made. More rigorous studies are needed.

Avaliação do uso de imobilização externa após descompressão e fusão cervical por via anterior: Revisão sistemática / Evaluation of the use of cervical restraint collar after anterior cervical fusion and decompression: Systematic review / Evaluación del uso de collar cervical de inmovilización después de la descompresión y fusion cervical por acceso anterior: Revisión sistemática

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.

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.

Review of the efficacy and safety of radiofrequency ablation for the treatment of small renal masses.

BACKGROUND: Small renal masses are increasingly being discovered incidentally on imaging performed for another reason. The standard of care for these masses involves excision by open or laparoscopic techniques. Recently, ablative techniques such as radiofrequency ablation (RFA) and cryoablation have taken a more prominent role in the treatment algorithm for these masses. We sought to evaluate the efficacy and safety of radiofrequency ablation in the treatment of renal tumours. METHODS: We conducted a review of the literature. There was no language restriction. We obtained studies from the following sources: the Cochrane Library, PubMed, EMBASE, LILACS and Current Controlled Trials. RESULTS: We identified no clinical trials in the literature. Thus we described the results from case series and retrospective studies with a reasonable sample size (number of reported patients in each study > 65). Most patients undergoing RFA had T1a disease with a mean tumour size of about 3 cm. Radiofrequency ablation was usually performed percutaneously with image guidance. Reported follow-up was short and ranged from 1 to 30 months. Most series used radiographic response as a surrogate for cancer control. The rates of local recurrence of the tumour were as high as 13.0% (average 8.5%) and were slightly higher than those associated with cryoablation and partial nephrectomy. Complications included hemorrhage, ureteral strictures and loss of a renal unit. CONCLUSION: Our review demonstrates that RFA is a suitable and promising therapy in patients with small renal tumours who are considered to be poor candidates for more involved surgery. However, clinical trials with long-term data are needed to establish the oncological efficacy.