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Table 4 Sensitivity analysis based on different analysis sets

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

  Publications / Cases True (+/−); False (+/−) Accuracy% Se% Sp% PPV% NPV% Rho (95%CI)
Primary analysis seta 25 / 44 (3/8); (0/0) 100 100 100 100 100 82 (43 to 95%)
All case reportsb 38 / 56 (3/8); (0/0) 100 100 100 100 100 83 (47 to 96%)
Analyzed in Bradley study excludedc 22 / 33 (3/8); (0/0) 100 100 100 100 100 82 (43 to 95%)
Excluding congress communicationsd 21 / 36 (2/8); (0/1e) 91 67 100 100 89 82 (43 to 95%)
  1. Legend: aAll case reports of male MPS-II treated with ERT report efficacy and safety. These cases reports were written in a narrative form (results not aggregated). They were published prior to Bradley bibliographic search
  2. 6MWT 6-min walk test, CI Confidence interval, NPV Negative predictive value, PPV Positive predictive value, Rho Spearman correlation coefficient, Se Sensitivity, Sp Specificity.
  3. bAll case reports of male MPS-II treated with ERT report efficacy and safety, despite being published after the bibliographic search for Bradley (31 December 2015)
  4. cAll case reports included in the primary analysis set, excluding all studies also analyzed in the Bradley study
  5. dAll case reports included in the primary analysis set, excluding all congress communications
  6. eDevelopment of antibodies was an outcome that did not show a significant modification, although it was classified as high to moderate SOE in the Bradley study.