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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.
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172