Differences between the real and the desired worlds in the results of clinical trials

Dib, Regina El; Jorge, Eliane Chaves; Kamegasawa, Amélia; Daher, Solange Ramires; Spagnuolo, Regina Stella; Silva, Marise Pereira da; Braga, Gabriel Pereira; Volpato, Enilze de Souza Nogueira; Módolo, Norma Sueli Pinheiro; Betini, Marluci; Valle, Adriana Polachini do; Corrêa, Ione; Bazan, Rodrigo; Almeida, Ricardo Augusto Monteiro de Barros; Weber, Silke Anna Theresa; Molina, Silvana A.; Yoo, Hugo Hyung Bok; Boas, Paulo José Fortes Villas; Corrente, José Eduardo; Mathew, Joseph L; Kapoor, Anil; Carvalho, Ricardo César Tavares; Vital, Roberto; Braz, Leandro Gobbo; Nascimento, Paulo do

doi:10.6061/clinics/2015(09)04

Cited by 4 publications

(2 citation statements)

References 14 publications

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“…Differences in the design (explanatory vs pragmatic), end points, and adjudication process may account for the differing results. All bAVM patients must participate in research to better understand the natural history of the disease and the proposed therapies, such as EVT, in order to resolve these discrepancies (93) (94).…”

Section: Accepted Manuscriptmentioning

confidence: 99%

“…All bAVM patients must participate in research to better understand the natural history of the disease and the proposed therapies, such as EVT, to resolve these discrepancies. 92,93 Innovation in bAVM EVT may take on a variety of forms, such as the approach, the type of LEA, and adjuvant in situ or systemic treatment. If successful, the venous approach's democratization and advancement could change the paradigm of care in view of a potential sharp rise in cure rates that exceed 90%.…”

Section: The Futurementioning

confidence: 99%

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Endovascular Management of Brain Arteriovenous Malformations

et al. 2023

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Due to the risk of cerebral hemorrhage, and its related morbidity-mortality, brain arteriovenous malformations (bAVM) are a rare and threatening disease. Concerning bAVM management, only one randomized control trial -the ARUBA trial -has been published so far. The results of the ARUBA trial favor a non-interventional approach in the case of an unruptured bAVM, however implementation of these findings is challenging in daily practice.Instead, management relies on multidisciplinary discussions that lead to patient-specific strategies based on local expertise and experience in reference centers. Given the wide patterns of presentation and multiple treatment modalities recommendations are also difficult to follow. Endovascular treatment (EVT) is a widely used modality which can be used for curative treatment, or prior to surgery or radiosurgery ("pre-EVT"). EVT practice is in constant evolution (i.e. venous approach, combination with surgery during the same anesthesia, etc.). Liquid embolic agents (LEA) such as ethylene vinyl alcohol (EVOH) copolymer and cyanoacrylates (CYA), and their method of injection to increase bAVM occlusion, have also benefited from technical evolutions such as the use of adjunctive flow arrest techniques (mini balloons, pressure cooker technique and multiple catheters). Further research is necessary to evaluate the advantages and disadvantages of EVT.

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Section: Accepted Manuscriptmentioning

confidence: 99%

Section: The Futurementioning

confidence: 99%

Endovascular Management of Brain Arteriovenous Malformations

et al. 2023

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Meta-analysis of Pragmatic and Explanatory Trials

Mbuagbaw

Aves

2021

Methods in Molecular Biology

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Generalizability of randomized controlled trials in primary health care: Applying the PRECIS‐2 tool on published protocols

Papagiannopoulou

Laiou

Tatsi³

et al. 2022

Evaluation Clinical Practice

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Rationale: Pragmatic design may facilitate the generalizability of effectiveness of randomized controlled trials (RCTs) in primary health care (PHC). Aims and Objectives:The aim of this study was to investigate whether published protocols in PHC were designed pragmatically and to explore whether specific trial characteristics may be related to a pragmatic design.Methods: Using the Pragmatic Explanatory Continuum Indicator Summary-2 (PRECIS-2), we assessed pragmatism for 123 published RCT protocols. For each domain, we calculated the mean score with the 95% confidence interval (95% CI).Interrater reliability was assessed by weighted κ-coefficient with 95% CI. We examined potential associations of published protocol characteristics with overall pragmatism by performing univariate and multivariate analyses. Results:We observed the highest score for primary analysis (4.66, 95% CI: 4.51, 4.82). The eligibility score was intermediate (3.16, 95% CI: 3.01, 3.32). Domains with scores towards the explanatory side included organization (2.50, 95% CI: 2.36, 2.63), flexibility of delivery (2.74, 95% CI: 2.60, 2.88) and flexibility of adherence (3.00, 95% CI: 2.83, 3.17). Interrater agreement was good (κ = 0.61; 95% CI: 0.34, 0.80; p < 0.001). Higher sample sizes were correlated to a pragmatic design (odds ratio: 6.86, 95% CI: 1.64, 28.75; p = 0.04). Conclusion:Most RCT protocols were rated as intermediate in the pragmaticexplanatory continuum. Future research may guide all stakeholders on how best to incorporate the level of pragmatism in the interpretation of the results so that the trials are more likely to be applicable in real-world settings.

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Differences between the real and the desired worlds in the results of clinical trials

Cited by 4 publications

References 14 publications

Endovascular Management of Brain Arteriovenous Malformations

Endovascular Management of Brain Arteriovenous Malformations

Meta-analysis of Pragmatic and Explanatory Trials

Generalizability of randomized controlled trials in primary health care: Applying the PRECIS‐2 tool on published protocols

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