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The Impact of Excluding Nonrandomized Studies From Systematic Reviews in Rare Diseases: "The Example of Meta-Analyses Evaluating the Efficacy and Safety of Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis"
Sampayo-Cordeiro, Miguel; Miguel-Huguet, Bernat; Malfettone, Andrea; Pérez-García, José Manuel; Llombart-Cussac, Antonio; Cortés, Javier; Pardo, Almudena; Pérez López, Jordi.
Afiliação
  • Sampayo-Cordeiro, Miguel; Medica Scientia Innovation Research (MedSIR). Barcelona. ES
  • Miguel-Huguet, Bernat; Hospital de Bellvitge. Department of Surgery. Colorectal Unit. Barcelona. ES
  • Malfettone, Andrea; Medica Scientia Innovation Research (MedSIR). Barcelona. ES
  • Pérez-García, José Manuel; Medica Scientia Innovation Research (MedSIR). Barcelona. ES
  • Llombart-Cussac, Antonio; Hospital Arnau de Vilanova. Barcelona. ES
  • Cortés, Javier; University Hospital Vall d'Hebron. Barcelona. ES
  • Pardo, Almudena; Albiotech Consultores y Redacción Científica S.L. Madrid. ES
  • Pérez López, Jordi; University Hospital Vall d'Hebron. Department of Internal Medicine. Barcelona. ES
Front. mol. biosci. ; : 690615, jun. 2021. graf, tab
Article em En | Redbvs | ID: biblio-1418391
Biblioteca responsável: BR1.1
ABSTRACT
Nonrandomized studies are usually excluded from systematic reviews. This could lead to loss of a considerable amount of information on rare diseases. In this article, we explore the impact of excluding nonrandomized studies on the generalizability of meta-analyses results on mucopolysaccharidosis (MPS) disease. A comprehensive search of systematic reviews on MPS patients up to May 2020 was carried out (CRD42020191217). The primary endpoint was the rate of patients excluded from systematic reviews if only randomized studies were considered. Secondary outcomes included the differences in patient and study characteristics between randomized and nonrandomized studies, the methods used to combine data from studies with different designs, and the number of patients excluded from systematic reviews if case reports were not considered. More than 50% of the patients analyzed have been recruited in nonrandomized studies. Patient characteristics, duration of follow-up, and the clinical outcomes evaluated differ between the randomized and nonrandomized studies. There are feasible strategies to combine the data from different randomized and nonrandomized designs. The analyses suggest the relevance of including case reports in the systematic reviews, since the smaller the number of patients in the reference population, the larger the selection bias associated to excluding case reports. Our results recommend including nonrandomized studies in the systematic reviews of MPS to increase the representativeness of the results and to avoid a selection bias. The recommendations obtained from this study should be considered when conducting systematic reviews on rare diseases
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Texto completo: 1 Coleções: 05-specialized Base de dados: Redbvs Assunto principal: Viés de Seleção / Metanálise como Assunto / Doenças Raras / Ensaios Clínicos Controlados não Aleatórios como Assunto Idioma: En Revista: Front. mol. biosci. Ano de publicação: 2021 Tipo de documento: Article País de afiliação: Espanha

Texto completo: 1 Coleções: 05-specialized Base de dados: Redbvs Assunto principal: Viés de Seleção / Metanálise como Assunto / Doenças Raras / Ensaios Clínicos Controlados não Aleatórios como Assunto Idioma: En Revista: Front. mol. biosci. Ano de publicação: 2021 Tipo de documento: Article País de afiliação: Espanha