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Table 3 Agreement between the classification of outcomes based on the case report meta-analysis and the SOE classification, based on the clinical study meta-analysis. Strong confirmatory method

From: Agreement between results of meta-analyses from case reports and clinical studies, regarding efficacy and safety of idursulfase therapy in patients with mucopolysaccharidosis type II (MPS-II). A new tool for evidence-based medicine in rare diseases

  Strength of evidence of clinical study meta-analysis
Number of case reports [+] for the outcome High to moderate Low to insufficient
≥ 6 [+] of 44 cases (acceptable evidence group) (True positives = 3)
-uGAGs
-Liver Volume
-Antibodies
(False positives = 0)
<  6 [+] of 44 cases (unacceptable evidence group) (False negative = 0) (True negatives = 8)
-6MWT, JROM, Growth, IRR, Pulmonary function, Cardiac, QoL, sleep apnea.
  1. Legend: The 95% confidence interval for the validity index are: positive predictive value: 100% (29 to 100%); negative predictive value: 100% (63 to 100%); sensibility: 100% (29 to 100%), and specificity: 100% (63 to 100%).
  2. 6MWT 6-min walk test, CI Confidence interval, IRR Infusion-related reaction, JROM Joint range of motion, NPV Negative predictive value, PPV Positive predictive value, QoL Quality of life, Se Sensitivity, Sp Specificity, SOE Strength of evidence, uGAGs Urinary glycosaminoglycans.