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

Table 4 Sensitivity analysis based on different analysis sets

	Publications / Cases	True (+/−); False (+/−)	Accuracy%	Se%	Sp%	PPV%	NPV%	Rho (95%CI)
Primary analysis set^a	25 / 44	(3/8); (0/0)	100	100	100	100	100	82 (43 to 95%)
All case reports^b	38 / 56	(3/8); (0/0)	100	100	100	100	100	83 (47 to 96%)
Analyzed in Bradley study excluded^c	22 / 33	(3/8); (0/0)	100	100	100	100	100	82 (43 to 95%)
Excluding congress communications^d	21 / 36	(2/8); (0/1^e)	91	67	100	100	89	82 (43 to 95%)

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
6MWT 6-min walk test, CI Confidence interval, NPV Negative predictive value, PPV Positive predictive value, Rho Spearman correlation coefficient, Se Sensitivity, Sp Specificity.
^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)
^cAll case reports included in the primary analysis set, excluding all studies also analyzed in the Bradley study
^dAll case reports included in the primary analysis set, excluding all congress communications
^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.

ISSN: 1750-1172