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The aim of the present study is to confirm previous results in another patient population, suffering from mucopolysaccharidosis Type II (MPS-II).<\/jats:p>\n              <\/jats:sec>\n              <jats:sec>\n                <jats:title>Methods<\/jats:title>\n                <jats:p>A systematic review and meta-analysis of case reports published by April 2018 was conducted for MPS-II patients treated with enzyme replacement therapy (ERT). The study is reported in accordance with PRISMA and MOOSE guidelines (PROSPERO database code CRD42018093408). The assessed population and outcomes were the same as previously analyzed in a meta-analysis of MPS-II clinical studies. The primary endpoint was the percent of clinical cases showing improvement in efficacy outcome, or no harm in safety outcome after ERT initiation. A restrictive procedure to aggregate case reports, by selecting standardized and well-defined outcomes, was proposed. Different sensitivity analyses were able to evaluate the robustness of results.<\/jats:p>\n              <\/jats:sec>\n              <jats:sec>\n                <jats:title>Results<\/jats:title>\n                <jats:p>Every outcome classified as \u201cacceptable evidence group\u201d in our case report meta-analysis had been graded as \u201cmoderate strength of evidence\u201d in the aforementioned meta-analysis of clinical studies. Sensitivity, specificity, and positive-negative predictive values for results of both meta-analyses reached 100%, and were deemed equivalent.<\/jats:p>\n              <\/jats:sec>\n              <jats:sec>\n                <jats:title>Conclusions<\/jats:title>\n                <jats:p>Aggregating case reports quantitatively, rather than analyzing them qualitatively, may improve conclusions in rare diseases and personalized medicine. Additionally, we propose some methods to evaluate publication bias and heterogeneity of the included studies in a meta-analysis of case reports.<\/jats:p>\n              <\/jats:sec>","DOI":"10.1186\/s13023-019-1202-6","type":"journal-article","created":{"date-parts":[[2019,10,21]],"date-time":"2019-10-21T16:20:27Z","timestamp":1571674827000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":23,"title":["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). 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In addition, Miguel Sampayo has received research funding fees from Nestle Health Science.Bernat Miguel-Huguet declares no conflict of interest.Almudena Pardo Mateos has been contracted in the past by Shire and by Genzyme.Andrea Malfettone declares no conflict of interest.Jos\u00e9 P\u00e9rez-Garc\u00eda has received consulting and advisor fees from: Roche and Eli Lilly.Antonio Llombart-Cussac has received consulting and advisor fees from Roche, GlaxoSmithKline, Novartis, Celgene, Eisai, and AstraZeneca and has stock options, patents and intellectual property from MedSIR.Javier Cort\u00e9s has received consulting and advisor fees from: Roche, Celgene, Cellestia, AstraZeneca, Biothera Pharmaceutical, Merus, Seattle Genetics, Daiichi Sankyo and Erytech. In addition, Javier Cort\u00e9s has received honorarias from: Roche, Novartis, Celgene, Eisai, Pfizer and Samsung. Add more, Javier Cort\u00e9s has received research funding fees to the institution from Roche. Finally, Javier Cort\u00e9s has stock options, patents and intellectual property from MedSIR.Marc Molt\u00f3-Abad has received research support from Shire; has received fees from Sanofi\/Genzyme, Shire and Alexion for participation in their respective registries. In addition, this work used laronidase, a product manufactured by Shire, to evaluate the efficacy of ERT in MPS II patients.Cecilia Mu\u00f1oz-Delgado as received consulting fees, fees as a speaker, and research support from Shire and Genzyme.Marta P\u00e9rez-Quintana as received consulting fees, fees as a speaker, and research support from Shire.Jordi P\u00e9rez-L\u00f3pez has received consulting fees, fees as a speaker, and research support from Shire.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}}],"article-number":"230"}}