Eficácia e segurança da terapia com idursulfase em pacientes com mucopolissacaridose tipo II, com e sem comparação com placebo: revisão sistemática e metanálise

Alegra, Taciane; Eizerik, Dauana Pitano; Cerqueira, Caio César Silva de; Pereira, Tiago; Dornelles, Alícia Dorneles; Schwartz, Ida Vanessa Döederlein

doi:10.1590/0102-311x00017613

Cited by 7 publications

(10 citation statements)

References 29 publications

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“…Furthermore, it had been recently published and developed by an independent research group. Along with the Bradley study, the efficacy and safety of ERT in patients with MPS-II had been analyzed in three previous meta-analyses: that by da Silva et al in 2016 [23], which only selected one phase II/III trial [24], that by Alegra et al in 2013 [25], which combined 2 RCTs [24, 26] and 1 open label study with the same patients of all ages [27], 1 open-label study of adults [28], and 1 cohort study of children [29], plus the one by Pérez-López et al in 2018, which analyzed adult MPS-II patients (> 16 years) [30].…”

Section: Discussionmentioning

confidence: 99%

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

Sampayo-Cordero¹,

Miguel-Huguet²,

Pardo-Mateos³

et al. 2019

Orphanet J Rare Dis

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BackgroundA preliminary exploratory study shows solid agreement between the results of case reports and clinical study meta-analyses in mucopolysaccharidosis Type I (MPS-I) adult patients. The aim of the present study is to confirm previous results in another patient population, suffering from mucopolysaccharidosis Type II (MPS-II).MethodsA 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.ResultsEvery outcome classified as “acceptable evidence group” in our case report meta-analysis had been graded as “moderate strength of evidence” 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.ConclusionsAggregating 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.

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Section: Discussionmentioning

confidence: 99%

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

Sampayo-Cordero¹,

Miguel-Huguet²,

Pardo-Mateos³

et al. 2019

Orphanet J Rare Dis

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“…They did not combine results from different study designs Kuiper et al (2019) 1) They graded individual patient data as normal (N) or abnormal (A), both at baseline and at follow-up (based on reference values as reported in the articles). Patients with an abnormal baseline and follow-up (AA) were further classified as stable, improved, or deteriorated at follow-up (AAs, AAi, and AAd) 2) They graded patient groups as improved, stable, or deteriorated based on quantitative criteria predefined in the original articles Type II Alegra et al (2013) They used GRADE criteria (Guyatt et al (2008(Guyatt et al ( ), (2011; Morgan et al (2019)) to evaluate the strength of evidence for each outcome da They selected one randomized study. They did not combine results from different study designs Almeida et al (2018) They counted the number of infusion reactions, adverse events (serious and nonserious), and deaths in each study Pérez-López et al…”

Section: Discussionmentioning

confidence: 99%

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-Cordero

Miguel-Huguet

Malfettone

et al. 2021

Front. Mol. Biosci.

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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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“…One of the aims of systematic reviews on therapeutic interventions is to summarize the state of art in this intervention [12]. However, case reports are systematically excluded in systematic reviews in rare-disease research, especially when other designs with a higher level of evidence are included [12,15,[62][63][64][65]. Therefore, important innovations, which could contribute to a better management of the patient treated with orphan drugs, are systematically excluded from systematic reviews of rare diseases [2,13].…”

Section: Discussionmentioning

confidence: 99%

The Value of Case Reports in Systematic Reviews from Rare Diseases. The Example of Enzyme Replacement Therapy (ERT) in Patients with Mucopolysaccharidosis Type II (MPS-II)

Sampayo-Cordero

Miguel-Huguet

Malfettone

et al. 2020

IJERPH

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Background: Case reports are usually excluded from systematic reviews. Patients with rare diseases are more dependent on novel individualized strategies than patients with common diseases. We reviewed and summarized the novelties reported by case reports in mucopolysaccharidosis type II (MPS-II) patients treated with enzyme replacement therapy (ERT). Methods: We selected the case reports included in a previous meta-analysis of patients with MPS-II treated with ERT. Later clinical studies evaluating the same topic of those case reports were reported. Our primary aim was to summarize novelties reported in previous case reports. Secondary objectives analyzed the number of novelties evaluated in subsequent clinical studies and the time elapsed between the publication of the case report to the publication of the clinical study. Results: We identified 11 innovative proposals in case reports that had not been previously considered in clinical studies. Only two (18.2%) were analyzed in subsequent nonrandomized cohort studies. The other nine novelties (81.8%) were analyzed in later case reports (five) or were not included in ulterior studies (four) after more than five years from their first publication. Conclusions: Case reports should be included in systematic reviews of rare disease to obtain a comprehensive summary of the state of research and offer valuable information for healthcare practitioners.

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Eficácia e segurança da terapia com idursulfase em pacientes com mucopolissacaridose tipo II, com e sem comparação com placebo: revisão sistemática e metanálise

Cited by 7 publications

References 29 publications

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

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

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”

The Value of Case Reports in Systematic Reviews from Rare Diseases. The Example of Enzyme Replacement Therapy (ERT) in Patients with Mucopolysaccharidosis Type II (MPS-II)

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