RESUMEN
OBJECTIVES: to evaluate the accuracy of the creatine kinase test in neonatal screening for Duchenne Muscular Dystrophy. METHODS: Investigations were carried out in six databases, gray literature and manual search. All studies and data extraction eligibility processes were carried out in paired mode. The methodological stringency of the studies was assessed using the modified QUADAS 2 tool. The performance measures of the test were calculated using the Meta-DiSc software, version 1.4. RESULTS: 11 studies were included, with a total sample of 1,416,123 newborns. The positive likelihood ratio was more than 10 in these studies. On the other hand, the negative likelihood ratio was up to 0.2 in 9/10 studies; however, when considering the upper limit of the confidence interval, only 4/10 studies showed a negative likelihood ratio less than 0.2 and in 6/11 it was greater than 0.5. Specificity was close to 1 in studies. The sensitivity was equal to or greater than 0.81 in 10/11 studies; however, when considering the reliability's minimum interval limit, only 6/10 studies showed sensitivity equal to or greater than 0.7. Ten studies were summarized on a ROC curve indicating good performance (Area under the curve = 0.9980 and Q index = 0.9846). CONCLUSIONS: the creatine kinase test showed good accuracy in screening for cases of Duchenne Muscular Dystrophy and may be a useful alternative in the early diagnosis of the disease followed by confirmatory genetic testing.
Asunto(s)
Creatina Quinasa/metabolismo , Distrofia Muscular de Duchenne , Tamizaje Neonatal , Femenino , Humanos , Recién Nacido , Masculino , Distrofia Muscular de Duchenne/diagnóstico , Distrofia Muscular de Duchenne/metabolismo , Reproducibilidad de los ResultadosRESUMEN
OBJECTIVES: To evaluate the efficacy and safety of anti-interleukin-5 class therapy agents in the treatment of eosinophilic asthma and the financial impact of these drugs on the Brazilian and Mato Grosso public health systems. METHODS: The literature review in important databases was guided by a structured research question including patient or population, intervention, comparator, outcome and type of study. The retrieved studies went through a screening, selection, data extraction, and methodological quality assessment process. A model with two scenarios, one with mepolizumab and the other with benralizumab, was created for budget impact analysis. RESULTS: Evidence indicated that anti-interleukins-5 have an acceptable safety profile and can reduce exacerbation rates by up to 50% in the population with eosinophilic asthma; however, they showed no significant difference in quality of life. The adoption of these drugs in the Brazilian health system can impact the budget from R$ 40,379,731.50 to R$ 140,301,211.34 depending on the drug incorporated, considering a time horizon of 5 years. From the perspective of the state of Mato Grosso, the budget impact may reach, in the fifth year, an amount of R$ 1,301,210.58 and R$ 2,050.687.62 for the scenarios with mepolizumab and benralizumab, respectively. CONCLUSION: Anti-interleukins-5 are promising treatments for eosinophilic asthma because they minimise exacerbations and are well tolerated and safe. The financial impact is large, implying that technology costs may be a barrier to accessing this treatment class.