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PURPOSE: We aimed to assess agreement on intravenous tissue-plasminogen activator (IV tPA) and mechanical thrombectomy (MT) management decisions in acute ischemic stroke (AIS) patients. Secondary objectives were to assess agreement on Diffusion-Weighted-Imaging-Alberta-Stroke-Program-EArly-CT-Score (DWI-ASPECTS), and clinicians' willingness to recruit patients in a randomized controlled trial (RCT) comparing medical management with or without MT. MATERIALS AND METHODS: Studies assessing agreement of IV tPA and MT were systematically reviewed. An electronic portfolio of 41 AIS patients was sent to randomly selected providers at French stroke centers. Raters were asked 4 questions for each case: (1) What is the DWI-ASPECTS? (2) Would you perform IV tPA? (3) Would you perform MT? (4) Would you include the patient in a RCT comparing standard medical therapy with or without MT? Twenty responders were randomly selected to study intrarater agreement. Agreement was assessed using Fleiss' Kappa statistics. RESULTS: The review yielded two single center studies involving 2-5 raters, with various results. The electronic survey was answered by 86 physicians (60 vascular neurologists and 26 interventional neuroradiologists). The interrater agreement was moderate for IV tPA treatment decisions (κ=0.565 [0.420-0.680]), but only fair for MT (κ=0.383 [0.289-0.491]) and for combined treatment decisions (κ=0.399 [0.320-0.486]). The intrarater agreement was at least substantial for the majority of raters. The interrater agreement for DWI-ASPECTS was fair (κ=0.325 [0.276-0.387]). Physicians were willing to include a mean of 14±9 patients (33.1%±21.7%) in a RCT. CONCLUSION: Disagreements regarding the use of IVtPA or MT in the management of AIS patients remain frequent. Further trials are needed to resolve the numerous areas of uncertainty.
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Isquemia Encefálica , Fibrinolíticos/administração & dosagem , Acidente Vascular Cerebral , Trombectomia/métodos , Terapia Trombolítica/métodos , Doença Aguda , Administração Intravenosa , Isquemia Encefálica/tratamento farmacológico , Isquemia Encefálica/patologia , Isquemia Encefálica/cirurgia , Consenso , Tomada de Decisões , Humanos , Infusões Intravenosas , Revisão por Pares , Reprodutibilidade dos Testes , Acidente Vascular Cerebral/tratamento farmacológico , Acidente Vascular Cerebral/patologia , Acidente Vascular Cerebral/cirurgiaRESUMO
The rupture of an intracranial aneurysm is a sudden, unpredictable and potentially severe event. The responsible aneurysm has to be excluded from the cerebral circulation to avoid recurrence. More and more commonly, intracranial aneurysms are detected by coincidence. How to react to these fortuitous discoveries is unclear, because the risk of rupture is difficult to estimate. We present our approach to patients facing this situation and the decision-making process.
La rupture d'un anévrisme intracrânien est un événement aigu, imprévisible et aux conséquences potentiellement gravissimes. Le traitement passe par l'exclusion de la malformation anévrismale de la circulation sanguine afin d'éviter une nouvelle rupture. De plus en plus fréquemment, en raison des progrès de l'imagerie, les anévrismes sont découverts fortuitement. L'attitude à adopter dans ce type de situation reste incertaine, car le risque de rupture est difficile à évaluer. Nous présentons notre manière de prendre en charge cette situation, notre approche du patient, les facteurs qui conditionnent notre décision, et comment nous orientons notre démarche en cas de traitement prophylactique.
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Aneurisma Intracraniano/terapia , Circulação Cerebrovascular , Embolização Terapêutica , Humanos , Aneurisma Intracraniano/diagnóstico , Aneurisma Intracraniano/patologia , Aneurisma Intracraniano/fisiopatologia , Prognóstico , Fatores de RiscoRESUMO
BACKGROUND AND PURPOSE: The best management of patients with persistent distal occlusion after mechanical thrombectomy with or without IV thrombolysis remains unknown. We sought to evaluate the variability and agreement in decision-making for persistent distal occlusions. MATERIALS AND METHODS: A portfolio of 60 cases was sent to clinicians with varying backgrounds and experience. Responders were asked whether they considered conservative management or rescue therapy (stent retriever, aspiration, or intra-arterial thrombolytics) a treatment option as well as their willingness to enroll patients in a randomized trial. Agreement was assessed using κ statistics. RESULTS: The electronic survey was answered by 31 physicians (8 vascular neurologists and 23 interventional neuroradiologists). Decisions for rescue therapies were more frequent (n = 1116/1860, 60%) than for conservative management (n = 744/1860, 40%; P < .001). Interrater agreement regarding the final management decision was "slight" (κ = 0.12; 95% CI, 0.09-0.14) and did not improve when subgroups of clinicians were studied according to background, experience, and specialty or when cases were grouped according to the level of occlusion. On delayed re-questioning, 23 of 29 respondents (79.3%) disagreed with themselves on at least 20% of cases. Respondents were willing to offer trial participation in 1295 of 1860 (69.6%) cases. CONCLUSIONS: Individuals did not agree regarding the best management of patients with persistent distal occlusion after mechanical thrombectomy and IV thrombolysis. There is sufficient uncertainty to justify a dedicated randomized trial.
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BACKGROUND: Formulating a pertinent research question is of the utmost importance in clinical research. An ill-conceived question may lead to an erroneous trial design, which may adversely affect the care of patients and provide uninformative or even misleading results. METHODS: We review the research question of a randomized trial on the timing of lumbar discectomy. We compare the resulting design with other trials, real or hypothetical, that would have been more appropriate. RESULTS: The RCT we examine randomly allocated patients to early or delayed surgery to answer a theoretical question of the effect of time on the efficacy of surgery. The trial was interpreted to have shown that early surgery was associated with better clinical and functional outcomes as compared to delayed surgery. This conclusion is clinically misleading. Valid comparisons between groups should be performed on intent-to-treat analyses and at the same time points after randomization (and not at a fixed follow-up period after surgery). The clinically pertinent comparison is not between the theoretical efficacy of surgery performed at various times, but between surgery and conservative management in patients presenting at various times. Better-designed trials on the clinical benefits of lumbar discectomy, including the treatment of chronic sciatica, have been published. CONCLUSION: Theoretical research questions inspired from observational data can lead to erroneous trial design. Prospective randomized trials impact practice immediately: they are unique occasions to address clinical problems and optimize care under uncertainty in real time. However, they require the research question to be formulated with great care.
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Deslocamento do Disco Intervertebral , Humanos , Deslocamento do Disco Intervertebral/cirurgia , Estudos Prospectivos , Vértebras Lombares/cirurgia , Discotomia/métodos , Resultado do TratamentoRESUMO
OBJECTIVES: To better understand the explanatory-pragmatic distinction in the design and interpretation of randomized controlled trials (RCTs). METHODS: We review the explanatory-pragmatic distinction in clinical trial design. We use the PRECIS-2 tool to evaluate the trial design of selected RCTs on percutaneous vertebroplasty for osteoporotic vertebral compression fractures. We discuss difficulties in the selection of criteria and in the construction of PRECIS diagrams. We also examine how inconsistency in the selection of various items of trial design can cause confusion in the interpretation of results. RESULTS: The selection of criteria and the scoring of multiple PRECIS domains were subjective and thus debatable. The pragmascope patterns of various vertebroplasty trials were heterogeneous. Many trials had both pragmatic and explanatory components. Some placebo-controlled trial goals seem to have been explanatory, but their design actually included enough pragmatic items such that the meaning of negative trial results remains ambiguous. CONCLUSION: The results of a trial cannot be interpreted without understanding the various design choices made along the explanatory-pragmatic spectrum.
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Fraturas por Compressão , Fraturas da Coluna Vertebral , Vertebroplastia , Humanos , Ensaios Clínicos Controlados Aleatórios como Assunto , Projetos de Pesquisa , Vertebroplastia/métodos , Fraturas por Compressão/cirurgia , Fraturas da Coluna Vertebral/cirurgiaRESUMO
OBJECTIVES: To better understand the choice of the comparator intervention in the design of clinical trials and its impact on the meaning of results we review randomized trials on vertebroplasty. METHODS: We conducted a systematic and narrative review of all randomized trials on vertebroplasty. Trials are categorized according to the comparator intervention (non-surgical management, placebo/sham vertebroplasty, and kyphoplasty). RESULTS: All trials were too small to show a difference in objective clinical outcomes, and 20 of 23 RCTs used mean pain scores to compare interventions. Most trials comparing vertebroplasty with non-surgical management concluded that vertebroplasty was superior. Trials comparing kyphoplasty with vertebroplasty showed similar results for both interventions. However, 4 of 5 trials comparing vertebroplasty with placebo surgery failed to show a significant difference between groups. CONCLUSION: The clinical results of an intervention cannot be interpreted without a comparison that involves a control group. The choice of comparator intervention can change the meaning of the trial. A large pragmatic trial, using hard clinical outcomes such as morbidity and mortality as a primary outcome measure, would be needed to assess the potential clinical benefits of vertebroplasty.
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Fraturas por Compressão , Cifoplastia , Fraturas por Osteoporose , Fraturas da Coluna Vertebral , Vertebroplastia , Humanos , Fraturas por Osteoporose/cirurgia , Fraturas por Compressão/cirurgia , Fraturas da Coluna Vertebral/cirurgia , Vertebroplastia/métodos , Cifoplastia/métodos , Resultado do TratamentoRESUMO
BACKGROUND: A clean-cut separation between research and care was artificially created at the time of the Belmont report more than 40 years ago. The demarcation was initially controversial but eventually was implemented for political reasons. We examine why it must be revised. METHODS: We review historical research scandals as well as the theoretical basis for the Belmont demarcation. We then discuss consequences on medical practice and propose an alternative. DISCUSSION: Most research scandals involved abusing human beings supposedly for the sake of science. Belmont commissioners were aware the research/care problem was double-headed. While research subjects should be protected from abuse in the research context, patients need to be protected from unvalidated medical and surgical interventions in the care context. For political reasons the Commission recommended the regulation of research but to leave medical practice untouched. Thus the Commission had to distinguish research from care. The notion of 'generalizable knowledge' was introduced to define and regulate research, but the inadvertent result was that by trying to protect research subjects, the regulation has not only failed to protect all other patients, but also encouraged the widespread practice of unvalidated interventions within the care context. The notion of validated care should be re-introduced into a proper analysis of the care-research demarcation, for care research is an integral ingredient of a good medical practice. CONCLUSION: The research-care demarcation should be revised to leave room for the validated/unvalidated care distinction. Care research, essential to guide medical practice, should be facilitated at all levels.
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OBJECTIVES: Randomized trials (RCTs) should include a sufficient number of patients to reduce the risk that the observed outcome is a result of chance rather than from a truly different treatment effect. Trials must be even larger to claim an absence of treatment effect in a placebo-controlled trial. To estimate the size of the trial and maximize power, it is often suggested to use a comparison between the means of a continuous variable. METHODS: We examine the RCTs that have compared vertebroplasty and placebo for patients with osteoporotic fractures. Most trials compared the means of a continuous pain score to yield implausibly small trials, as small as 24 patients per group. RESULTS: The minimally significant difference between groups has no precise clinical meaning for patients when it is based on a comparison of means of pain scores. A comparison of the proportions of patients reaching a per-patient outcome measure of treatment success is much more pertinent if the trial is to inform the care of future patients. The resulting trials will admittedly need to be larger, but they will be much less likely to fall prey to the 'evidence of absence' fallacy. Furthermore, trial size should also take into consideration harder clinical outcome measures, such as death and disability. CONCLUSION: When the goal of a trial is to inform outcome-based medical care, comparing the proportions of patients reaching a clinically pertinent outcome is more appropriate than comparing the means of a continuous variable.
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Fraturas por Compressão , Fraturas por Osteoporose , Fraturas da Coluna Vertebral , Vertebroplastia , Humanos , Fraturas por Compressão/cirurgia , Fraturas por Osteoporose/cirurgia , Fraturas por Osteoporose/etiologia , Dor/etiologia , Medição da Dor , Qualidade de Vida , Ensaios Clínicos Controlados Aleatórios como Assunto , Fraturas da Coluna Vertebral/etiologia , Resultado do Tratamento , Vertebroplastia/efeitos adversos , Vertebroplastia/métodosRESUMO
BACKGROUND: Lumbar microdiscectomy is the most frequent surgical intervention used in the treatment of sciatica from herniated lumbar discs. Many discectomy trials have been plagued with an excessive number of crossovers that have rendered results inconclusive. METHODS: We review the design and results of influential lumbar microdiscectomy trials. We also discuss the various strategies that have been used to decrease the number of crossovers or to mitigate the effects of crossovers on analyses. RESULTS: Randomized trials on lumbar discectomy were affected by crossover rates of 8% to 42%. Various strategies that have been used to decrease that number or to mitigate the effects on results include: patient selection, blinding (placebo-controlled trials), an immediate access to surgery for the surgical group (but limited access to surgery for the conservative group), shortening the follow-up period necessary to reach the primary outcome measure, postponing crossovers to surgery after determination of the primary outcome, and modifying the primary outcome measure to include treatment failures. Crossovers should be anticipated and compensated for by increasing the number of participants. CONCLUSION: Non-adherence to randomly allocated management options can deprive trials of the statistical power needed to inform clinical care. Crossovers and ways to mitigate related problems should be anticipated at the time of trial design.
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BACKGROUND: Patient understanding of care interventions, of the clinical uncertainty, and of their participation in clinical research is often poor. We hypothesized that compared to routine care, patients would better understand the prevailing uncertainty when they participated in research. METHODS: A questionnaire was administered to patients at the time they attended a follow-up neurovascular clinic 4 to 52 weeks after a care episode where they did or did not participate in a clinical trial. Patients were not reminded whether they had previously participated in a clinical trial. Questions concerned their understanding of the risks/benefits of interventions, the availability of alternative options, whether their personal opinion was taken into consideration, the reason for the final decision, their confidence at having received the best management, and whether they had been research participants. RESULTS: Between June 2019 and June 2020, 167 patients were recruited; 71 had truly been research participants, while 96 had not. A greater proportion of research patients were aware of the existence of management alternatives (65% versus 44%; P=0.008). Patients of both groups believed their personal opinion counted in the final decision (76% versus 70%), and patients were equally confident that they had received the best management (94%). Research patients believed they had participated in research 46% of the time, compared to 12% of routine care patients (P=0.003). CONCLUSION: Many patients do not recall that they participated in a clinical trial, but they have a better understanding of the clinical uncertainty and of the availability of alternative management options.
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Tomada de Decisão Clínica , Consentimento Livre e Esclarecido , Humanos , IncertezaRESUMO
BACKGROUND: The meaning of a clinical trial depends to a large extent on the choice of the primary outcome measure, which can be explanatory or pragmatic. METHODS: We review the Japanese Adult Moyamoya (JAM) trial, that compared surgical extracranial to intracranial (EC-IC) bypass and medical management of hemorrhagic moyamoya disease. We also review some principles which guide the selection of the primary trial endpoint. DISCUSSION: The main component of the primary outcome measure in JAM was rebleeding, a surrogate outcome that allowed investigators to demonstrate that surgical bypass had causal efficacy. However, the number of patients with a poor outcome, defined as those with a modified Rankin score (mRS)>2, would have been a more pragmatic choice. Unfortunately, the trial was too small to show that patients benefited from surgery. CONCLUSION: The JAM trial showed that EC-IC bypass can decrease rebleeding in moyamoya patients, but whether patients have better outcomes with surgery remains uncertain. Hard pragmatic clinical primary outcome measures are necessary to guide surgical care.
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Revascularização Cerebral , Doença de Moyamoya , Adulto , Humanos , População do Leste Asiático , Doença de Moyamoya/cirurgia , Doença de Moyamoya/etiologia , Procedimentos Neurocirúrgicos , Resultado do TratamentoRESUMO
BACKGROUND: Patients with atherosclerotic carotid or middle cerebral artery occlusions suffer ischemic events that might theoretically be preventable with a surgical extracranial-intracranial bypass, but theory by itself does not justify surgical interventions. METHODS: We review landmark randomized trials on EC-IC bypass surgery for the treatment of ischemic stroke in patients with atherosclerotic stenoses or occlusions. RESULTS: The initial EC-IC bypass trial from 1985 did not show any clinical benefit from surgery. The carotid occlusion surgery study (COSS) performed more than 20 years later included only patients highly selected to potentially benefit from bypass by using modern perfusion studies. While EC-IC bypasses were successfully created and they did improve cerebral perfusion, the COSS study also failed to show any clinical benefit to the participating patients. CONCLUSION: Neurosurgical interventions must not only work in theory; they must improve patient outcomes in real practice.
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Doenças das Artérias Carótidas , Revascularização Cerebral , AVC Isquêmico , Acidente Vascular Cerebral , Humanos , Acidente Vascular Cerebral/prevenção & controle , Acidente Vascular Cerebral/cirurgia , Doenças das Artérias Carótidas/cirurgia , Artéria Carótida Interna/cirurgia , Resultado do TratamentoRESUMO
BACKGROUND AND PURPOSE: Stent-assisted coiling may improve angiographic results of endovascular treatment of unruptured intracranial aneurysms compared with coiling alone, but this has never been shown in a randomized trial. MATERIALS AND METHODS: The Stenting in the Treatment of Aneurysm Trial was an investigator-led, parallel, randomized (1:1) trial conducted in 4 university hospitals. Patients with intracranial aneurysms at risk of recurrence, defined as large aneurysms (≥10 mm), postcoiling recurrent aneurysms, or small aneurysms with a wide neck (≥4 mm), were randomly allocated to stent-assisted coiling or coiling alone. The composite primary efficacy outcome was "treatment failure," defined as initial failure to treat the aneurysm; aneurysm rupture or retreatment during follow-up; death or dependency (mRS > 2); or an angiographic residual aneurysm adjudicated by an independent core laboratory at 12 months. The primary hypothesis (revised for slow accrual) was that stent-assisted coiling would decrease treatment failures from 33% to 15%, requiring 200 patients. Primary analyses were intent to treat. RESULTS: Of 205 patients recruited between 2011 and 2021, ninety-four were allocated to stent-assisted coiling and 111 to coiling alone. The primary outcome, ascertainable in 203 patients, was reached in 28/93 patients allocated to stent-assisted coiling (30.1%; 95% CI, 21.2%-40.6%) compared with 30/110 (27.3%; 95% CI, 19.4%-36.7%) allocated to coiling alone (relative risk = 1.10; 95% CI, 0.7-1.7; P = .66). Poor clinical outcomes (mRS >2) occurred in 8/94 patients allocated to stent-assisted coiling (8.5%; 95% CI, 4.0%-16.6%) compared with 6/111 (5.4%; 95% CI, 2.2%-11.9%) allocated to coiling alone (relative risk = 1.6; 95% CI, 0.6%-4.4%; P = .38). CONCLUSIONS: The STAT trial did not show stent-assisted coiling to be superior to coiling alone for wide-neck, large, or recurrent unruptured aneurysms.
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Embolização Terapêutica , Procedimentos Endovasculares , Aneurisma Intracraniano , Humanos , Resultado do Tratamento , Aneurisma Intracraniano/diagnóstico por imagem , Aneurisma Intracraniano/terapia , Angiografia Cerebral , Procedimentos Endovasculares/métodos , Embolização Terapêutica/métodos , Stents/efeitos adversos , Estudos RetrospectivosRESUMO
BACKGROUND AND PURPOSE: Surgical clipping and endovascular treatment are commonly used in patients with unruptured intracranial aneurysms. We compared the safety and efficacy of the 2 treatments in a randomized trial. MATERIALS AND METHODS: Clipping or endovascular treatments were randomly allocated to patients with one or more 3- to 25-mm unruptured intracranial aneurysms judged treatable both ways by participating physicians. The study hypothesized that clipping would decrease the incidence of treatment failure from 13% to 4%, a composite primary outcome defined as failure of aneurysm occlusion, intracranial hemorrhage during follow-up, or residual aneurysms at 1 year, as adjudicated by a core lab. Safety outcomes included new neurologic deficits following treatment, hospitalization of >5 days, and overall morbidity and mortality (mRS > 2) at 1 year. There was no blinding. RESULTS: Two hundred ninety-one patients were enrolled from 2010 to 2020 in 7 centers. The 1-year primary outcome, ascertainable in 290/291 (99%) patients, was reached in 13/142 (9%; 95% CI, 5%-15%) patients allocated to surgery and in 28/148 (19%; 95% CI, 13%-26%) patients allocated to endovascular treatments (relative risk: 2.07; 95% CI, 1.12-3.83; P = .021). Morbidity and mortality (mRS >2) at 1 year occurred in 3/143 and 3/148 (2%; 95% CI, 1%-6%) patients allocated to surgery and endovascular treatments, respectively. Neurologic deficits (32/143, 22%; 95% CI, 16%-30% versus 19/148, 12%; 95% CI, 8%-19%; relative risk: 1.74; 95% CI, 1.04-2.92; P = .04) and hospitalizations beyond 5 days (69/143, 48%; 95% CI, 40%-56% versus 12/148, 8%; 95% CI, 5%-14%; relative risk: 0.18; 95% CI, 0.11-0.31; P < .001) were more frequent after surgery. CONCLUSIONS: Surgical clipping is more effective than endovascular treatment of unruptured intracranial aneurysms in terms of the frequency of the primary outcome of treatment failure. Results were mainly driven by angiographic results at 1 year.
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Embolização Terapêutica , Procedimentos Endovasculares , Aneurisma Intracraniano , Humanos , Aneurisma Intracraniano/diagnóstico por imagem , Aneurisma Intracraniano/cirurgia , Resultado do Tratamento , Falha de Tratamento , Procedimentos Endovasculares/métodos , Embolização Terapêutica/métodosRESUMO
BACKGROUND: The role research can play in neurosurgical care is poorly understood. The result is that inappropriate methods are frequently used in clinical research, and unverifiable neurosurgical care is widely practised. METHODS: We review the research questions and the methods used in two landmark studies on the surgical treatment of ruptured aneurysms. We then compare the explanatory approach with the more appropriate pragmatic approach, and explain the difference by analogy with the ancient medical dispute between dogmatists and empiricists. RESULTS: The International Cooperative study on the timing of aneurysm surgery was dogmatic or rationalistic: medical care is based on reason; research aims to gain theoretical knowledge, explanations and generalizations to be used as reasons to act in practice. But practice based on case-by-case reasoning is haphazard and unverifiable. The Finnish randomized trial was more empirical in spirit: reliable knowledge is to be found in repeated clinical experience. A reliable treatment is one proven to provide verifiably better outcomes in a trial integrated into practice. CONCLUSION: Verifiable outcome-based neurosurgical care will be possible with the integration of pragmatic trials into everyday practice.
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Aneurisma Roto , Aneurisma Intracraniano , Hemorragia Subaracnóidea , Aneurisma Roto/cirurgia , Humanos , Aneurisma Intracraniano/cirurgia , Procedimentos Neurocirúrgicos/métodos , Hemorragia Subaracnóidea/cirurgia , Resultado do TratamentoRESUMO
Recent studies of the medical literature have revealed numerous and serious problems. Errors in the design, methods and interpretation of studies can frequently be identified. A huge hidden problem is publication bias, the tendency for positive articles to be published, while negative articles are either not written or submitted. This can systematically lead to an overestimation of the value of treatments, of diagnostic or prognostic studies. Even more worrisome is selective reporting: only a subset of a wide array of tested hypotheses are presented (the ones that turned out to be positive with significance testing). This is particularly true for secondary endpoints and subgroup findings, but even the primary endpoints of trials have been modified when publications are compared to protocols. The peer-review process is fallible. Even if it were strengthened, reviewers cannot examine what is not reported. Hence many problems can only be mitigated with better reporting. Numerous initiatives have proposed guidelines to promote transparent reporting, but progress is slow.
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BACKGROUND AND PURPOSE: Delayed cerebral ischemia (DCI) and angiographic vasospasm following subarachnoid hemorrhage (SAH) have been associated for more than 50years. We aimed to examine whether the knowledge gained by theoretical research on vasospasm has actually translated into better patient outcomes in practice. METHODS: This is a narrative review of the concept of vasospasm as a cause of DCI after SAH. We discuss recent studies that have assessed the accuracy and reliability of the diagnostic tests (transcranial Doppler ultrasound [TCD], CT angiography, and catheter angiography), which are used to identify SAH patients at-risk of DCI. RESULTS: Both the diagnostic accuracy of TCD and the reliability of CT angiography to identify patients in severe vasospasm are poor. For the gold standard catheter angiography, the repeatability of the diagnosis of vasospasm, made by multiple raters, is only fair. Interventions on angiographic vasospasm have never been proven to improve patient outcomes. A pragmatic trial integrating the meaning of the diagnosis of vasospasm into a study protocol that assesses the value of endovascular interventions in the prevention of DCI after SAH seems to be in order. Such a trial could provide a pragmatic definition of clinically meaningful vasospasm. CONCLUSION: We must move beyond research conceived as an enterprise aiming to acquire theoretical knowledge to one where research is integrated into clinical practice to improve clinical outcomes in real time.
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Isquemia Encefálica , Hemorragia Subaracnóidea , Vasoespasmo Intracraniano , Isquemia Encefálica/etiologia , Angiografia Cerebral , Humanos , Reprodutibilidade dos Testes , Hemorragia Subaracnóidea/complicações , Hemorragia Subaracnóidea/diagnóstico , Hemorragia Subaracnóidea/cirurgia , Vasoespasmo Intracraniano/diagnóstico , Vasoespasmo Intracraniano/etiologiaRESUMO
BACKGROUND: The reliability of outcome measures is of central importance in clinical research. Studies of reliability remain rare in the neurovascular field. METHODS: A narrative review of the history (1997-2021) of reporting angiographic results of the surgical or endovascular treatments of aneurysms serves to illustrate the importance of precisely defining outcome measures. We also review how the reliability of an angiographic classification system was studied. DISCUSSION: Outcome measures are commonly used without precise definitions. When definitions or classification systems exist, they are rarely verified for their reliability. Twenty-five years following its introduction, a classification of angiographic results of aneurysm treatments is still being studied and modified. CONCLUSION: The reliability of outcome measures should be made a research priority if we are to practice outcome-based medical or surgical care.
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Aneurisma Roto , Embolização Terapêutica , Procedimentos Endovasculares , Aneurisma Intracraniano , Aneurisma Roto/cirurgia , Angiografia Cerebral/métodos , Embolização Terapêutica/métodos , Procedimentos Endovasculares/métodos , Seguimentos , Humanos , Aneurisma Intracraniano/diagnóstico por imagem , Aneurisma Intracraniano/cirurgia , Reprodutibilidade dos Testes , Estudos Retrospectivos , Resultado do TratamentoRESUMO
BACKGROUND AND PURPOSE: The burden of proof principle is rarely discussed and poorly understood, but central to the proper design of pragmatic clinical trials. A better understanding of the principle could play an important role in the re-introduction of scientific methods within practice and in revising fundamental problems with the current research-care separation. METHODS: We analyze the design of the ARUBA trial on the management of unruptured brain arteriovenous malformations. We also review how the concept of clinical equipoise was introduced to address a misconceived problem of research ethics. RESULTS: The ARUBA trial hypothesis in favour of conservative management of brain arteriovenous malformations failed to take into account the fact that the burden of proof was on surgery, endovascular treatment or radiation therapy. Thus, results remained inconclusive and other trials are needed. The equipoise notion fails to take into account that the burden of proof is on unvalidated medical or surgical interventions, if we want to provide outcome-based medical care that patients can trust. CONCLUSION: The burden of proof principle is essential to properly design pragmatic trials. This principle also explains why in certain circumstances optimal care is a randomized care trial.
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Fístula Arteriovenosa , Malformações Arteriovenosas Intracranianas , Humanos , Malformações Arteriovenosas Intracranianas/cirurgia , Tratamento Conservador , Encéfalo , Resultado do TratamentoRESUMO
BACKGROUND: Surgical randomized trials are difficult to accomplish. One major problem is recruitment of a sufficient number of patients to address the clinical problem. METHODS: We review the various ways patient recruitment in surgical RCTs can be promoted. We examine two landmark trials on the surgical treatment of temporal lobe epilepsy (TLE), one that was successful, and one which did not attain the target number of participants. DISCUSSION: Both designs of the Canadian and American trials of surgery for TLE included a benefit to participants: the Canadian trial gave a chance to have immediate access to investigation and treatment, as compared to a 1 year delay (considered 'standard care' in that center), while the American trial offered free surgical management to both arms. Patients were recruited and treatments randomly allocated prior to knowing for certain whether they were surgical candidates or not. This design choice may have helped circumvent the 'equipoise problem'. The Canadian trial offered participation to drug-resistant patients that were already routinely referred to surgical centers, while the success of the American trial which limited recruitment to the early period of drug resistance was dependent on a change of practice of referring clinicians which did not materialize. CONCLUSION: The surgical treatment of drug-resistant temporal lobe epilepsy has been validated using RCT methods. Ways to promote participation in surgical trials should be further investigated.