Enzyme replacement therapy for the treatment of Hunter disease: A systematic review with narrative synthesis and meta-analysis.

BACKGROUND: In the last 10 years enzyme replacement therapy (ERT) has become an alternative for the treatment of patients with Hunter disease (HD). Nevertheless, the information regarding efficacy and safety is scarce and mainly based on the pivotal trials. This scarcity is especially evident for adults and severe forms of HD. METHODS: A systematic review of publications in the electronic databases PUBMED, EMBASE and Cochrane Central was undertaken. Clinical trials and observational studies were included. The data about efficacy and security were retrieved and analysed with Review Manager version 5.3. RESULTS: 677 records were found, 559 remaining after the removal of duplicates. By title and abstract review, 427 were excluded. Full reading of the rest was made (122 publications) and 42 were finally included. It was not possible to perform meta-analysis of all the endpoints due to high heterogeneity in the reporting and measuring of variables in each publication. Eight clinical trials were included, 6 with high risk of bias. The quality of the other studies was low in 12%, average in 68% and good in 21%. Main findings were: a reduction in the elimination of glycosaminoglycans (GAG) in urine in all the studies (26/26), decrease in liver and spleen size (18/18), increase of 52.59 m (95% CI, 36, 42-68.76, p < .001) in the 6-min walk test (TM6M), increase in forced vital capacity (FVC) of 9.59% (95% CI 4.77-14.51, p < .001), reduction of the left ventricular mass index of 3.57% (95% CI 1.2-5.93) and reduction in mortality (OR) of 0.44 (0.27-0.71). DISCUSSION: The data suggests a clear and consistent effect of ERT in HD reducing the accumulation of GAGs in the body, demonstrated by the reduction of its urinary excretion, as well as by the reduction of its deposits (spleen, liver and heart). Likewise, there is an improvement in physical and respiratory function. In addition, a reduction in mortality has been observed. Lack of studies, small size of the samples, and methodological deficiencies are the main limitations to establish definite conclusions. CONCLUSIONS: The data suggests that ERT is effective and safe in the treatment of HD. There is a need to evaluate patient-centred outcomes and the impact on quality of life.

Factors associated with malaria and arboviral disease in patients with imported febrile syndrome: a retrospective cohort study. / Factores asociados con malaria y arboviriasis en pacientes con síndrome febril importado: estudio de cohortes retrospectivo.

OBJECTIVES: To identify predictors of malaria and arboviral disease in patients with febrile syndrome who seek care after traveling from tropical or subtropical locations. MATERIAL AND METHODS: Observational retrospective cohort study. We collected demographic, epidemiologic, and clinical data; laboratory findings; and the clinical and final microbiologic diagnoses. Multivariate analysis was used to calculate indices of diagnostic accuracy (sensitivity, specificity, and predictive values) and coefficients of probability of combinations of variables. RESULTS: Data for 291 patients with febrile syndrome were included; 108 had malaria (37.1%), 28 had an arboviral disease (9.6%), and 155 had other causes of fever (53.3%). Multivariate analysis showed patients most likely to have malaria were those from sub-Saharan Africa, adjusted odds ratio (aOR) of 45.85 (95% CI, 9.45-222.49); immigrants who returned to visit friends and relatives (VFR), aOR of 3.55 (95% CI, 1.21-10.46); or had platelet concentrations 150 000/mm3, aORa of 16.47 (95% CI, 5.46-49.70) or headache, aOR of 10.62 (95% CI, 3.20-35.28). The combination of these 4 variables gave a positive probability coefficient (PPC) of 23.72 (95% CI, 5.76-97.62). Patients with febrile syndrome most likely to have an arboviral disease were those from Central or South America, OR 5.07 (95% CI, 1.73-14.92), and those who had exanthems, OR 5.10 (95% CI, 1.72-17.02) or joint pain, OR 14.50 (95% CI, 3.05-68.80). The combination of these 3 variables gave a PPC of 20.66 (95% CI, 7.74-55.21). CONCLUSION: Patients with febrile syndrome with the greatest probability of having malaria are those from sub-Saharan Africa, those who are VFR, and those with platelet concentrations under 150.000/µL or headache. Arboviral disease was more likely in patients from Central and South America who had exanthems or joint pain.

OBJETIVO: Definir variables predictoras de malaria y arboviriasis en pacientes que consultan por síndrome febril tras la vuelta de un viaje a zonas tropicales/subtropicales. METODO: Estudio de cohortes retrospectivo. Se incluyeron variables demográficas, epidemiológicas, clínicas, analíticas y el diagnóstico final clínico y microbiológico. Se realizó un análisis multivariante y se calcularon los índices de exactitud diagnóstica (sensibilidad, especificidad, valores predictivos) y cocientes de probabilidad de la combinación de dichasvariables. RESULTADOS: Se incluyeron 291 pacientes con síndrome febril, 108 tenían malaria (37,1%), 28 arboviriasis (9,6%) y 155 otras causas de fiebre (53,3%). En el análisis multivariante, los pacientes con síndrome febril con más riesgo de padecer malaria fueron los que procedían de África subsahariana [odds ratio ajustado (ORa): 45,85; IC 95%: 9,45- 222,49], eran inmigrantes que visitan a familiares y amigos (VFA) (ORa = 3,55; IC 95%: 1,21-10,46), presentaban cifras de plaquetas 150.000/mm3 (ORa = 16,47; IC 95%: 5,46-49,70) o cefalea (ORa = 10,62; IC 95%: 3,20-35,28). La combinación de estas cuatro variables tiene un cociente de probabilidad positivo (CPP) de 23,72 (IC 95%: 5,76- 97,62). Los pacientes con síndrome febril que tienen más riesgo de padecer arboviriasis eran los que procedían de Centroamérica y Sudamérica (OR = 5,07; IC 95%: 1,73-14,92), presentaban exantema (OR = 5,10; IC 95%: 1,72- 17,02) o artromialgias (OR = 14,50; IC 95%: 3,05-68,80). La combinación de estas tres variables tiene un CPP de 20,66 (IC 95%: 7,74-55,21). CONCLUSIONES: Los pacientes con síndrome febril que tienen más riesgo de padecer malaria son los que procedían de África subsahariana, eran VFA, presentaban cifras de plaquetas 150.000/µl o cefalea, y tenían mayor riesgo de padecer arboviriasis si procedían de Centroamérica y Sudamérica, presentaban exantema o artromialgias.

Factores asociados con malaria y arboviriasis en pacientes con síndrome febril importado: estudio de cohortes retrospectivo / Factors associated with malaria and arboviral disease in patients with imported febrile syndrome: a retrospective cohort study

Objetivos: Definir variables predictoras de malaria y arboviriasis en pacientes que consultan por síndrome febril tras la vuelta de un viaje a zonas tropicales/subtropicales. Método: Estudio de cohortes retrospectivo. Se incluyeron variables demográficas, epidemiológicas, clínicas, analíticas y el diagnóstico final clínico y microbiológico. Se realizó un análisis multivariante y se calcularon los índices de exactitud diagnóstica (sensibilidad, especificidad, valores predictivos) y cocientes de probabilidad de la combinación de dichas variables. Resultados: Se incluyeron 291 pacientes con síndrome febril, 108 tenían malaria (37,1%), 28 arboviriasis (9,6%) y 155 otras causas de fiebre (53,3%). En el análisis multivariante, los pacientes con síndrome febril con más riesgo de padecer malaria fueron los que procedían de África subsahariana [odds ratio ajustado (ORa): 45,85; IC 95%: 9,45-222,49], eran inmigrantes que visitan a familiares y amigos (VFA) (ORa = 3,55; IC 95%: 1,21-10,46), presentaban cifras de plaquetas < 150.000/mm3 (ORa = 16,47; IC 95%: 5,46-49,70) o cefalea (ORa = 10,62; IC 95%: 3,20-35,28). La combinación de estas cuatro variables tiene un cociente de probabilidad positivo (CPP) de 23,72 (IC 95%: 5,76-97,62). Los pacientes con síndrome febril que tienen más riesgo de padecer arboviriasis eran los que procedían de Centroamérica y Sudamérica (OR = 5,07; IC 95%: 1,73-14,92), presentaban exantema (OR = 5,10; IC 95%: 1,72-17,02) o artromialgias (OR = 14,50; IC 95%: 3,05-68,80). La combinación de estas tres variables tiene un CPP de 20,66 (IC 95%: 7,74-55,21). Conclusiones: Los pacientes con síndrome febril que tienen más riesgo de padecer malaria son los que procedían de África subsahariana, eran VFA, presentaban cifras de plaquetas < 150.000/μl o cefalea, y tenían mayor riesgo de padecer arboviriasis si procedían de Centroamérica y Sudamérica, presentaban exantema o artromialgias. (AU)

Objective: To identify predictors of malaria and arboviral disease in patients with febrile syndrome who seek care after traveling from tropical or subtropical locations. Methods: Observational retrospective cohort study. We collected demographic, epidemiologic, and clinical data; laboratory findings; and the clinical and final microbiologic diagnoses. Multivariate analysis was used to calculate indices of diagnostic accuracy (sensitivity, specificity, and predictive values) and coefficients of probability of combinations of variables. Results: Data for 291 patients with febrile syndrome were included; 108 had malaria (37.1%), 28 had an arboviral disease (9.6%), and 155 had other causes of fever (53.3%). Multivariate analysis showed patients most likely to have malaria were those from sub-Saharan Africa, adjusted odds ratio (aOR) of 45.85 (95% CI, 9.45-222.49); immigrants who returned to visit friends and relatives (VFR), aOR of 3.55 (95% CI, 1.21-10.46); or had platelet concentrations <150 000/mm3, aORa of 16.47 (95% CI, 5.46-49.70) or headache, aOR of 10.62 (95% CI, 3.20-35.28). The combination of these 4 variables gave a positive probability coefficient (PPC) of 23.72 (95% CI, 5.76-97.62). Patients with febrile syndrome most likely to have an arboviral disease were those from Central or South America, OR 5.07 (95% CI, 1.73-14.92), and those who had exanthems, OR 5.10 (95% CI, 1.72-17.02) or joint pain, OR 14.50 (95% CI, 3.05-68.80). The combination of these 3 variables gave a PPC of 20.66 (95% CI, 7.74-55.21). Conclusions: Patients with febrile syndrome with the greatest probability of having malaria are those from sub-Saharan Africa, those who are VFR, and those with platelet concentrations under 150.000/μL or headache. Arboviral disease wasmore likely in patients from Central and South America who had exanthems or joint pain. (AU)

¿Cuánto saben los estudiantes del Grado en Medicina sobre la enfermedad de Chagas en España? / How much do Medicine students know about Chagas’ disease in Spain?

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