Meta Agreement

Our meta-analysis of case reports took into account the same results analyzed in Bradley`s study [12]. We also evaluated each result only on the basis of the results of previous narrative case reports. We shared the number of case reports with a change for a given ERT result by the total number of case reports analyzed, and then we were measured as a percentage. It is important to keep in mind that we can only define an efficiency result as improved in a case report, if: (1) the method of evaluating the results has been described (Z.B. an abdominal ultrasound to characterize liver size was reported before the start of treatment, then after 6 months and 15 months) or (2) a quantitative measure in the clinical case (Z.B. Only infusion-related (IRR) reactions that resulted in changes in the dose of ERT were found to be relevant. This restrictive procedure for defining a result as improved (efficiency) or deteriorated (IRR) was considered a strong confirmation method. In light of this evaluation, we estimated that the higher the percentage of case reports with improvements or deficiencies (IRR) for a given outcome, the greater the degree of evidence of changes in ERT-controlled outcomes. The classification of results on the basis of the strong confirmation method obtained equivalent results compared to the SOE classification in the meta-analyses of clinical trials in all analysis sets [at least 10 results ranked below 11 (accuracy ≥91%)].

In addition, the ratio between the number of case reports with improved results and the SOE score was good (Rho > 80%). Persaud R. Misleading meta-analysis. “Fail safe N” is a useful mathematical measure for the stability of the results. Brit Med J. 1996;312 (7023):125. Kamel MG, Nam NT, Han NHB, El-Shabouny AE, Makram AM, Abd-Elhay FA, et al. Acute encephalomyelitis disseminated after dengue fever: a case report and meta-analysis. PLoS Negl Too Say. 2017;11 (6):e0005715.

Similarly, the relative match rate between the quantitative data score, based on case reports with the modified ERT results, and the SOE was good (Rho – 0.82, 95%CI: 0.43 to 0.95) when the strong confirmation method was used (Fig. 2). Conversely, analysis of the modified ERT results in case reports on the basis of the low confirmation method revealed a moderate approval rate (Rho – 0.63, 95%IC: 0.044 to 0.89) with the SOE (see additional file 1: Figure S1). For patients with MPS II treated with enzyme replacement therapy (ERT), a systematic study and meta-analysis of case reports were conducted and published until April 2018. The study is carried out in accordance with the PRISMA and MOOSE guidelines (PROSPERO CRD42018093408).