Value of risk scores in the decision to palliate patients with ruptured abdominal aortic aneurysm

Background: The aim of this study was to develop a 48-h mortality risk score, which included morphology data, for patients with ruptured abdominal aortic aneurysm presenting to an emergency department, and to assess its predictive accuracy and clinical effectiveness in triaging patients to immediate aneurysm repair, transfer or palliative care. Methods: Data from patients in the IMPROVE (Immediate Management of the Patient With Ruptured Aneurysm: Open Versus Endovascular Repair) randomized trial were used to develop the risk score. Variables considered included age, sex, haemodynamic markers and aortic morphology. Backwards selection was used to identify relevant predictors. Predictive performance was assessed using calibration plots and the C-statistic. Validation of the newly developed and other previously published scores was conducted in four external populations. The net benefit of treating patients based on a risk threshold compared with treating none was quantified. Results: Data from 536 patients in the IMPROVE trial were included. The final variables retained were age, sex, haemoglobin level, serum creatinine level, systolic BP, aortic neck length and angle, and acute myocardial ischaemia. The discrimination of the score for 48-h mortality in the IMPROVE data was reasonable (C-statistic 0·710, 95 per cent c.i. 0·659 to 0·760), but varied in external populations (from 0·652 to 0·761). The new score outperformed other published risk scores in some, but not all, populations. An 8 (95 per cent c.i. 5 to 11) per cent improvement in the C-statistic was estimated compared with using age alone. Conclusion: The assessed risk scores did not have sufficient accuracy to enable potentially life-saving decisions to be made regarding intervention. Focus should therefore shift to offering repair to more patients and reducing non-intervention rates, while respecting the wishes of the patient and family

Treatment evaluation by volumetric segmentation in pediatric optic pathway glioma:evaluation of the effect of bevacizumab on intra-tumor components

Purpose: Progressive pediatric optic pathway gliomas (OPGs) are treated by diverse systemic antitumor modalities. Refined insights on the course of intra-tumoral components are limited. Methods: We performed an exploratory study on the longitudinal volumetric course of different (intra-)tumor components by manual segmentation of MRI at the start and after 3, 6 and 12 months of bevacizumab (BVZ) treatment. Results: Thirty-one patients were treated with BVZ (median 12 months, range: 2–39 months). During treatment the total tumor volume decreased with median 19.9% (range: − 62.3 to + 29.7%; n = 30) within the first 3 months, decreased 19.0% (range: − 68.8 to + 96.1%; n = 28) between start and 6 months and 27.2% (range: −73.4 to + 36.0%; n = 21) between start and 12 months. Intra-tumoral cysts were present in 12 OPGs, all showed a decrease of volume during treatment. The relative contrast enhanced volume of NF1 associated OPG (n = 11) showed an significant reduction compared to OPG with a KIAA1549-BRAF fusion (p &lt; 0.01). Three OPGs progressed during treatment, but were not preceded by an increase of relative contrast enhancement. Conclusion: Treatment with BVZ of progressive pediatric OPGs leads to a decrease of both total tumor volume and cystic volume for the majority of OPGs with emphasis on the first three months. NF1 and KIAA1549-BRAF fusion related OPGs showed a different (early) treatment effect regarding the tumor enhancing component on MRI, which did not correlate with tumor volume changes. Future research is necessary to further evaluate these findings and its relevance to clinical outcome parameters.</p

Treatment evaluation by volumetric segmentation in pediatric optic pathway glioma: evaluation of the effect of bevacizumab on intra-tumor components

Purpose: Progressive pediatric optic pathway gliomas (OPGs) are treated by diverse systemic antitumor modalities. Refined insights on the course of intra-tumoral components are limited. Methods: We performed an exploratory study on the longitudinal volumetric course of different (intra-)tumor components by manual segmentation of MRI at the start and after 3, 6 and 12 months of bevacizumab (BVZ) treatment. Results: Thirty-one patients were treated with BVZ (median 12 months, range: 2–39 months). During treatment the total tumor volume decreased with median 19.9% (range: − 62.3 to + 29.7%; n = 30) within the first 3 months, decreased 19.0% (range: − 68.8 to + 96.1%; n = 28) between start and 6 months and 27.2% (range: −73.4 to + 36.0%; n = 21) between start and 12 months. Intra-tumoral cysts were present in 12 OPGs, all showed a decrease of volume during treatment. The relative contrast enhanced volume of NF1 associated OPG (n = 11) showed an significant reduction compared to OPG with a KIAA1549-BRAF fusion (p < 0.01). Three OPGs progressed during treatment, but were not preceded by an increase of relative contrast enhancement. Conclusion: Treatment with BVZ of progressive pediatric OPGs leads to a decrease of both total tumor volume and cystic volume for the majority of OPGs with emphasis on the first three months. NF1 and KIAA1549-BRAF fusion related OPGs showed a different (early) treatment effect regarding the tumor enhancing component on MRI, which did not correlate with tumor volume changes. Future research is necessary to further evaluate these findings and its relevance to clinical outcome parameters

Amsterdam Acute Aneurysm Trial: Background, design, and methods

The objective of the Amsterdam Acute Aneurysm Trial is to study the combined outcome of conventional emergency surgery versus endovascular treatment for ruptured abdominal aortic aneurysms. The design used was a multicenter randomized clinical trial conducted in two university hospitals and one teaching hospital in the Amsterdam region. The study included all patients with a ruptured abdominal aneurysm who were eligible for endovascular and conventional surgery. The primary end points were combined mortality and severe morbidity. The secondary end points were quality of life and cost-effectiveness. The background, design, and methods of this trial are presented, and the ethical and legal issues of this type of research are discussed

Ultra-low-dose CT versus chest X-ray for patients suspected of pulmonary disease at the emergency department: a multicentre randomised clinical trial

Background: Chest CT displays chest pathology better than chest X-ray (CXR). We evaluated the effects on health outcomes of replacing CXR by ultra-low-dose chest-CT (ULDCT) in the diagnostic work-up of patients suspected of non-traumatic pulmonary disease at the emergency department. Methods: Pragmatic, multicentre, non-inferiority randomised clinical trial in patients suspected of non-traumatic pulmonary disease at the emergency department. Between 31 January 2017 and 31 May 2018, every month, participating centres were randomly allocated to using ULDCT or CXR. Primary outcome was functional health at 28 days, measured by the Short Form (SF)-12 physical component summary scale score (PCS score), non-inferiority margin was set at 1 point. Secondary outcomes included hospital admission, hospital length of stay (LOS) and patients in follow-up because of incidental findings. Results: 2418 consecutive patients (ULDCT: 1208 and CXR: 1210) were included. Mean SF-12 PCS score at 28 days was 37.0 for ULDCT and 35.9 for CXR (difference 1.1; 95% lower CI: 0.003). After ULDCT, 638/1208 (52.7%) patients were admitted (median LOS of 4.8 days; IQR 2.1-8.8) compared with 659/1210 (54.5%) patients after CXR (median LOS 4.6 days; IQR 2.1-8.8). More ULDCT patients were in follow-up because of incidental findings: 26 (2.2%) versus 4 (0.3%). Conclusions: Short-term functional health was comparable between ULDCT and CXR, as were hospital admissions and LOS, but more incidental findings were found in the ULDCT group. Our trial does not support routine use of ULDCT in the work-up of patients suspected of non-traumatic pulmonary disease at the emergency department. Trial registration number: NTR6163

Classifying the diagnosis of study participants in clinical trials: a structured and efficient approach

Background: A challenge in imaging research is a diagnostic classification of study participants. We hypothesised that a structured approach would be efficient and that classification by medical students, residents, and an expert panel whenever necessary would be as valid as classification of all patients by experts. Methods: OPTIMACT is a randomised trial designed to evaluate the effectiveness of replacing chest x-ray for ultra-low-dose chest computed tomography (CT) at the emergency department. We developed a handbook with diagnostic guidelines and randomly selected 240 cases from 2,418 participants enrolled in OPTIMACT. Each case was independently classified by two medical students and, if they disagreed, by the students and a resident in a consensus meeting. Cases without consensus and cases classified as complex were assessed by a panel of medical specialists. To evaluate the validity, 60 randomly selected cases not referred to the panel by the students and the residents were reassessed by the specialists. Results: Overall, the students and, if necessary, residents were able to assign a diagnosis in 183 of the 240 cases (76% concordance; 95% confidence interval [CI] 71–82%). We observed agreement between students and residents versus medical specialists in 50/60 cases (83% concordance; 95% CI 74–93%). Conclusions: A structured approach in which study participants are assigned diagnostic labels by assessors with increasing levels of medical experience was an efficient and valid classification method, limiting the workload for medical specialists. We presented a viable option for classifying study participants in large-scale imaging trials (Netherlands National Trial Register number NTR6163)