Everolimus for cardiac rhabdomyomas in children with tuberous sclerosis. The ORACLE study protocol (everOlimus for caRdiac rhAbdomyomas in tuberous sCLErosis): a randomised, multicentre, placebo-controlled, double-blind phase II trial.

INTRODUCTION: Tuberous sclerosis complex is a rare genetic disorder leading to the growth of hamartomas in multiple organs, including cardiac rhabdomyomas. Children with symptomatic cardiac rhabdomyoma require frequent admissions to intensive care units, have major complications, namely, arrhythmias, cardiac outflow tract obstruction and heart failure, affecting the quality of life and taking on high healthcare cost. Currently, there is no standard pharmacological treatment for this condition, and the management includes a conservative approach and supportive care. Everolimus has shown positive effects on subependymal giant cell astrocytomas, renal angiomyolipoma and refractory seizures associated with tuberous sclerosis complex. However, evidence supporting efficacy in symptomatic cardiac rhabdomyoma is limited to case reports. The ORACLE trial is the first randomised clinical trial assessing the efficacy of everolimus as a specific therapy for symptomatic cardiac rhabdomyoma. METHODS: ORACLE is a phase II, prospective, randomised, placebo-controlled, double-blind, multicentre protocol trial. A total of 40 children with symptomatic cardiac rhabdomyoma secondary to tuberous sclerosis complex will be randomised to receive oral everolimus or placebo for 3 months. The primary outcome is 50% or more reduction in the tumour size related to baseline. As secondary outcomes we include the presence of arrhythmias, pericardial effusion, intracardiac obstruction, adverse events, progression of tumour reduction and effect on heart failure. CONCLUSIONS: ORACLE protocol addresses a relevant unmet need in children with tuberous sclerosis complex and cardiac rhabdomyoma. The results of the trial will potentially support the first evidence-based therapy for this condition.

Very late hepatic arterial thrombosis manifesting as central biliary necrosis: A rare presentation.

Hepatic artery thrombosis should always be considered on a liver graft recipient with mild and nonspecific symptoms, even after a decade of the transplantation.

Accuracy of the ADNEX MR scoring system based on a simplified MRI protocol for the assessment of adnexal masses.

PURPOSE: We aimed to evaluate the ADNEX MR scoring system for the prediction of adnexal mass malignancy, using a simplified magnetic resonance imaging (MRI) protocol. METHODS: In this prospective study, 200 patients with 237 adnexal masses underwent MRI between February 2014 and February 2016 and were followed until February 2017. Two radiologists calculated ADNEX MR scores using an MRI protocol with a simplified dynamic study, not a high temporal resolution study, as originally proposed. Sensitivity, specificity, positive and negative predictive values, likelihood ratios, and the area under the receiver operating characteristic curve were calculated (cutoff for malignancy, score ≥ 4). The reference standard was histopathologic diagnosis or imaging findings during >12 months of follow-up. RESULTS: Of 237 lesions, 79 (33.3%) were malignant. The ADNEX MR scoring system, using a simplified MRI protocol, showed 94.9% (95% confidence interval [CI], 87.5%-98.6%) sensitivity and 97.5% (95% CI, 93.6%-99.3%) specificity in malignancy prediction; it was thus highly accurate, like the original system. The level of interobserver agreement on simplified scoring was high (κ = 0.91). CONCLUSION: In a tertiary cancer center, the ADNEX MR scoring system, even based on a simplified MRI protocol, performed well in the prediction of malignant adnexal masses. This scoring system may enable the standardization of MRI reporting on adnexal masses, thereby improving communication between radiologists and gynecologists.

MR imaging of hypervascular lesions in the cirrhotic liver: a diagnostic dilemma.

Cirrhosis is characterized by a spectrum of hepatocellular nodules that mark the progression from regenerative nodules to low- and high-grade dysplastic nodules, followed by small and large hepatocellular carcinomas (HCCs). Characterization of small nodules on the basis of imaging and histopathologic findings is complicated by an overlap in findings associated with each type of nodule, a reflection of their multistep transitions. Vascularity patterns change gradually as the nodules evolve, with an increasing shift from predominantly venous to predominantly arterial perfusion. Regenerative and low-grade dysplastic nodules demonstrate predominantly portal perfusion and contrast enhancement similar to that of surrounding parenchyma. Differentiation of high-grade dysplastic nodules and well-differentiated HCCs on the basis of dynamic imaging and histologic findings is challenging, with a high rate of false-negative results. Some small nodules that lack hypervascularity may be early HCCs. Progressed small and large HCCs usually present no diagnostic difficulty because of their characteristic findings. Although characterization of hypervascular lesions in the cirrhotic liver is difficult, it is a key step in disease management and is the radiologist's responsibility.

Potential role of diffusion tensor MRI in the differential diagnosis of mild cognitive impairment and Alzheimer's disease.

OBJECTIVE: The purpose of this study was to evaluate the fractional anisotropy values of several white matter tracts with the aim of differentiating a healthy population from persons with mild cognitive impairment or Alzheimer's disease. SUBJECTS AND METHODS: Seventy-nine patients with memory impairment and 16 volunteer controls participated in the study. MRI was performed with a 1.5-T system. Conventional MR images and diffusion tensor images were obtained for all participants. The diffusion tensor imaging data were postprocessed, and low b-value, fractional anisotropy, and fractional anisotropy color-coded maps were calculated. With the three maps as an anatomic reference, fractional anisotropy was measured for hippocampal formations, superior longitudinal fascicles, posterior cingulate gyri, and the splenium of the corpus callosum. Kruskal-Wallis and Steel-type multiple-comparison nonparametric tests were performed for the statistical analysis. RESULTS: The fractional anisotropy values for the splenium of the corpus callosum, bilateral posterior cingulate gyri, and bilateral superior longitudinal fascicles of patients with mild cognitive impairment and those with probable Alzheimer's disease were significantly lower than the values of controls. No differences were found in hippocampal formations in any group. No significant difference was found in fractional anisotropy values in comparisons of mild cognitive impairment versus possible Alzheimer's disease and probable Alzheimer's disease or comparisons of probable Alzheimer's disease and possible Alzheimer's disease. CONCLUSION: Diffusion tensor imaging is a promising technique for the evaluation of patients with probable mild cognitive impairment. Early detection of the disease expands the treatment options, increasing the likelihood of a good clinical response and enhancing the quality of life of patients and their relatives. Further studies with larger populations are needed to confirm the role of diffusion tensor imaging in the evaluation of memory impairment.