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BACKGROUND: The MEK inhibitor, selumetinib, reduces plexiform neurofibroma (PN) in pediatric patients with neurofibromatosis type 1 (NF1). Its safety and efficacy in adults with PN and effectiveness in other NF1manifestations (e.g., neurocognitive function, growth reduction, and café-au-lait spots) are unknown. METHODS: This open-label, phase 2 trial enrolled 90 pediatric or adult NF1 patients with inoperable, symptomatic, or potentially morbid, measurable PN (≥ 3 cm). Selumetinib was administered at doses of 20 or 25 mg/m2 or 50 mg q 12 hrs for 2 years. Pharmacokinetics, PN volume, growth parameters, neurocognitive function, café-au-lait spots, and quality of life (QoL) were evaluated. RESULTS: Fifty-nine children and 30 adults (median age, 16 years; range, 3-47) received an average of 22±5 (4-26) cycles of selumetinib. Eighty-eight (98.9%) out of 89 per-protocol patients showed volume reduction in the target PN (median, 40.8%; 4.2%-92.2%), and 81 (91%) patients showed partial response (≥ 20% volume reduction). The response lasted until cycle 26. Scores of neurocognitive functions (verbal comprehension, perceptual reasoning, processing speed, and full-scale IQ) significantly improved in both pediatric and adult patients (P <0.05). Prepubertal patients showed increases in height score and growth velocity (P <0.05). Café-au-lait spot intensity decreased significantly (P <0.05). Improvements in QoL and pain scores were observed in both children and adults. All adverse events were CTCAE grade 1 or 2 and were successfully managed without drug discontinuation. CONCLUSION: Selumetinib decrease PN volume in the majority of pediatric and adult NF1 patients while also showing efficacy in non-malignant diverse NF1 manifestations.
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Background Despite a proven role in the characterization of liver lesions, use of the gadolinium-based contrast agent (GBCA) gadoxetate disodium at MRI is limited in children due to a lack of comparative safety data. Purpose To evaluate the safety of the GBCA gadoxetate disodium (a linear ionic hepatobiliary contrast agent [HBA]) in children and adolescents, compared with extracellular contrast agents (ECA). Materials and Methods A retrospective analysis was conducted in children and adolescents aged 18 years or younger who underwent HBA-enhanced MRI at one of three tertiary hospitals from January 2010 to December 2022. The incidence of GBCA-associated acute adverse events was compared between MRI examinations with a HBA and those with ECA. Severity was categorized according to American College of Radiology guidelines (mild, moderate, or severe). (a) Propensity score matching using multivariable logistic regression models and (b) inverse probability of treatment weighting analysis based on nine covariates (age, sex, asthma, allergic rhinitis, chronic urticaria or atopy, food allergy, drug allergy, premedication, and history of GBCA-associated adverse events) were used for confounder adjustment. Results A total of 1629 MRI examinations (ECA, n = 1256; HBA, n = 373) in 1079 patients were included (mean age, 8.6 years ± 6.5; 566 girls). The per-examination incidence of GBCA-associated acute adverse events showed no evidence of a difference, with rates of 0.9% (11 of 1256 examinations) for ECA and 1.3% (five of 373 examinations) for HBA (odds ratio [OR], 1.55 [95% CI: 0.54, 4.46]; P = .42). Acute adverse events were all mild with ECA, whereas with HBA, they were mild for four patients and moderate for one patient. There was no evidence of a difference in the incidence of acute adverse events, even in propensity score matching (OR, 1.33 [95% CI: 0.30, 5.96]; P = .71) and inverse probability of treatment weighting analysis (OR, 0.84 [95% CI: 0.25, 2.86]; P = .78). Conclusion Gadoxetate disodium showed no difference in acute adverse events compared with ECA in children and adolescents, with further large-scale pediatric studies required to confirm its safety. © RSNA, 2024 Supplemental material is available for this article. See also the editorial by Otero in this issue.
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Medios de Contraste , Gadolinio DTPA , Imagen por Resonancia Magnética , Humanos , Masculino , Femenino , Niño , Adolescente , Estudios Retrospectivos , Imagen por Resonancia Magnética/métodos , Medios de Contraste/efectos adversos , Preescolar , Hígado/diagnóstico por imagen , Lactante , Hepatopatías/diagnóstico por imagenRESUMEN
Transfer learning plays a pivotal role in addressing the paucity of data, expediting training processes, and enhancing model performance. Nonetheless, the prevailing practice of transfer learning predominantly relies on pre-trained models designed for the natural image domain, which may not be well-suited for the medical image domain in grayscale. Recognizing the significance of leveraging transfer learning in medical research, we undertook the construction of class-balanced pediatric radiograph datasets collectively referred to as PedXnets, grounded in radiographic views using the pediatric radiographs collected over 24 years at Asan Medical Center. For PedXnets pre-training, approximately 70,000 X-ray images were utilized. Three different pre-training weights of PedXnet were constructed using Inception V3 for various radiation perspective classifications: Model-PedXnet-7C, Model-PedXnet-30C, and Model-PedXnet-68C. We validated the transferability and positive effects of transfer learning of PedXnets through pediatric downstream tasks including fracture classification and bone age assessment (BAA). The evaluation of transfer learning effects through classification and regression metrics showed superior performance of Model-PedXnets in quantitative assessments. Additionally, visual analyses confirmed that the Model-PedXnets were more focused on meaningful regions of interest.
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Aprendizaje Profundo , Fracturas Óseas , Humanos , Niño , Aprendizaje Automático , RadiografíaRESUMEN
PURPOSE: The aim of this study was to evaluate diagnostic accuracy and to establish computed tomography (CT) and Doppler ultrasonography (US) criteria for hepatic outflow obstruction after pediatric liver transplantation (LT) using left lobe (LL) or left lateral section (LLS) grafts. METHODS: Pediatric patients who underwent LT using LL or LLS grafts between January 1999 and December 2021 were retrospectively included. The diagnostic performance of Doppler US and CT parameters for hepatic outflow obstruction was calculated using receiver operating characteristic (ROC) curve analysis. A diagnostic decision tree model combining the imaging parameters was developed. RESULTS: In total, 288 patients (150 girls; median age at LT, 1.8 years [interquartile range, 0.9 to 3.6 years]) were included. Among the Doppler US parameters, venous pulsatility index (VPI) showed excellent diagnostic performance (area under the ROC curve [AUROC], 0.90; 95% confidence interval [CI], 0.86 to 0.93; Youden cut-off value, 0.40). Among the CT parameters, anastomotic site diameter (AUROC, 0.92; 95% CI, 0.88 to 0.95; Youden cut-off, 4.2 mm) and percentage of anastomotic site stenosis (AUROC, 0.88; 95% CI, 0.84 to 0.92; Youden cut-off, 35%) showed excellent and good diagnostic performance, respectively. A decision tree model combining the VPI, peak systolic velocity, and percentage of anastomotic site stenosis stratified patients according to the risk of hepatic outflow obstruction. CONCLUSION: VPI, anastomotic site diameter, and percentage of anastomotic site stenosis were reliable imaging parameters for diagnosing hepatic outflow obstruction after pediatric LT using LL or LLS grafts.
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OBJECTIVE: To develop a deep-learning-based bone age prediction model optimized for Korean children and adolescents and evaluate its feasibility by comparing it with a Greulich-Pyle-based deep-learning model. MATERIALS AND METHODS: A convolutional neural network was trained to predict age according to the bone development shown on a hand radiograph (bone age) using 21036 hand radiographs of Korean children and adolescents without known bone development-affecting diseases/conditions obtained between 1998 and 2019 (median age [interquartile range {IQR}], 9 [7-12] years; male:female, 11794:9242) and their chronological ages as labels (Korean model). We constructed 2 separate external datasets consisting of Korean children and adolescents with healthy bone development (Institution 1: n = 343; median age [IQR], 10 [4-15] years; male: female, 183:160; Institution 2: n = 321; median age [IQR], 9 [5-14] years; male: female, 164:157) to test the model performance. The mean absolute error (MAE), root mean square error (RMSE), and proportions of bone age predictions within 6, 12, 18, and 24 months of the reference age (chronological age) were compared between the Korean model and a commercial model (VUNO Med-BoneAge version 1.1; VUNO) trained with Greulich-Pyle-based age as the label (GP-based model). RESULTS: Compared with the GP-based model, the Korean model showed a lower RMSE (11.2 vs. 13.8 months; P = 0.004) and MAE (8.2 vs. 10.5 months; P = 0.002), a higher proportion of bone age predictions within 18 months of chronological age (88.3% vs. 82.2%; P = 0.031) for Institution 1, and a lower MAE (9.5 vs. 11.0 months; P = 0.022) and higher proportion of bone age predictions within 6 months (44.5% vs. 36.4%; P = 0.044) for Institution 2. CONCLUSION: The Korean model trained using the chronological ages of Korean children and adolescents without known bone development-affecting diseases/conditions as labels performed better in bone age assessment than the GP-based model in the Korean pediatric population. Further validation is required to confirm its accuracy.
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Inteligencia Artificial , Aprendizaje Profundo , Adolescente , Humanos , Niño , Masculino , Femenino , Lactante , Determinación de la Edad por el Esqueleto , Radiografía , República de CoreaRESUMEN
In medicine, confounding variables in a generalized linear model are often adjusted; however, these variables have not yet been exploited in a non-linear deep learning model. Sex plays important role in bone age estimation, and non-linear deep learning model reported their performances comparable to human experts. Therefore, we investigate the properties of using confounding variables in a non-linear deep learning model for bone age estimation in pediatric hand X-rays. The RSNA Pediatric Bone Age Challenge (2017) dataset is used to train deep learning models. The RSNA test dataset is used for internal validation, and 227 pediatric hand X-ray images with bone age, chronological age, and sex information from Asan Medical Center (AMC) for external validation. U-Net based autoencoder, U-Net multi-task learning (MTL), and auxiliary-accelerated MTL (AA-MTL) models are chosen. Bone age estimations adjusted by input, output prediction, and without adjusting the confounding variables are compared. Additionally, ablation studies for model size, auxiliary task hierarchy, and multiple tasks are conducted. Correlation and Bland-Altman plots between ground truth and model-predicted bone ages are evaluated. Averaged saliency maps based on image registration are superimposed on representative images according to puberty stage. In the RSNA test dataset, adjusting by input shows the best performances regardless of model size, with mean average errors (MAEs) of 5.740, 5.478, and 5.434 months for the U-Net backbone, U-Net MTL, and AA-MTL models, respectively. However, in the AMC dataset, the AA-MTL model that adjusts the confounding variable by prediction shows the best performance with an MAE of 8.190 months, whereas the other models show the best performances by adjusting the confounding variables by input. Ablation studies of task hierarchy reveal no significant differences in the results of the RSNA dataset. However, predicting the confounding variable in the second encoder layer and estimating bone age in the bottleneck layer shows the best performance in the AMC dataset. Ablations studies of multiple tasks reveal that leveraging confounding variables plays an important role regardless of multiple tasks. To estimate bone age in pediatric X-rays, the clinical setting and balance between model size, task hierarchy, and confounding adjustment method play important roles in performance and generalizability; therefore, proper adjusting methods of confounding variables to train deep learning-based models are required for improved models.
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Aprendizaje Profundo , Radiología , Humanos , Niño , Rayos X , Factores de Confusión Epidemiológicos , RadiografíaRESUMEN
Background: Surgical reconstruction of the urinary tract, anus, and vagina is the definitive treatment for cloacal malformation. However, this procedure may be technically challenging in patients with a long common channel (>3â cm), because further reconstructive procedures, such as vaginal replacement or vaginal switch maneuver, may be required. Thus, accurate determination of spatial anatomy is essential during surgical planning. Three-dimensional (3D) reconstruction using rotational fluoroscopy, computed tomography (CT), and magnetic resonance imaging (MRI) has recently been reported to help in determining the relationship between the rectum, vagina, and bladder, and provides a more accurate measurement of the channel length compared to conventional cloacography. MRI-based 3D reconstruction provides substantial information regarding soft tissue structures around the cloaca, including the pelvic floor musculature and anus. Case: A 2-year-old girl with cloacal malformation required reconstructive surgery. Colostomy and cystostomy had been performed on the first day of her life. Preoperative loopogram revealed a cloaca with a long common channel (35â mm) and short urethra (9â mm), single vaginal opening in the bladder neck, and the colon anterior to the vagina with a fistula at the vaginal neck. Because the vagina was too short to be pulled through, 3D printing based on MRI was performed to visualize structural relationships prior to surgical correction. Saline was used for cloacal visualization. Furthermore, endoscopy-assisted urogenital mobilization was performed, and vaginal substitution was performed using the rectum. No postoperative complications were observed. Conclusions: We believe this is the first report of the use of MRI-based 3D imaging and printing, with saline as a contrast agent during surgical planning for correction of cloacal malformation. MRI-based 3D printing is a potentially promising technique for surgical planning of cloacal malformation correction in patients with a long common channel, as it provides detailed information about the surrounding soft tissue structures without exposure to radiation or contrasting agents.
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PURPOSE: The aim of this study was to validate the postnatal urinary tract dilation (UTD) classification system by correlating it with the need for surgical intervention. METHODS: Young infants who underwent ultrasound (US) examinations for prenatal hydronephrosis were retrospectively identified. The kidney units (KUs; right, left, or bilateral) were graded from UTD P0 (very low risk) to P3 (high risk) based on seven US criteria from the UTD system. Surgery-free survival curves were constructed using the Kaplan-Meier method. Univariable and multivariable Cox proportional-hazards regression analysis clustered by patients was performed. Interobserver agreement was analyzed using the weighted kappa coefficient. RESULTS: In total, 504 KUs from 336 patients (mean age, 18.3±15.9 days; range, 1 to 94 days; males, n=276) were included, with a median follow-up of 24.2 months. Fifty-eight KUs underwent surgical intervention. Significant differences were observed among the Kaplan-Meier curves stratified into UTD groups (P<0.001). The presence of anterior-posterior renal pelvic diameter ≥15 mm (hazard ratio [HR], 8.602; 95% confidence interval [CI], 1.558 to 43.065), peripheral calyceal dilation (HR, 8.190; 95% CI, 1.558 to 43.065), ureteral dilation (HR, 2.619; 95% CI, 1.274 to 5.380), parenchymal thickness abnormality (HR, 3.371; 95% CI, 1.574 to 7.223), bladder abnormality (HR, 12.209; 95% CI, 3.616 to 41.225) were significantly associated with the occurrence of surgery. The interobserver agreement was moderate to almost perfect agreement for US features (κ=0.564-0.898) and substantial for final UTD grades (κ=0.716). CONCLUSION: The UTD classification system is reliable and appropriately stratifies the risk of surgical intervention.
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Intussusception involves an invagination of the proximal bowel into the distal bowel, with ileocolic intussusception being the most common type. However, a diagnostic delay can lead to intestinal ischemia, bowel infarction, or even death; therefore, its early diagnosis and management are important. The primary role of abdominal radiography is to detect pneumoperitoneum or high-grade bowel obstruction in cases of suspected intussusception, and ultrasonography is the modality of choice for its diagnosis. Nonoperative enema reduction, the treatment of choice for childhood intussusception in cases without signs of perforation or peritonitis, can be safely performed with a success rate of 82%. Enema reduction can be performed in various ways according to image guidance method (fluoroscopy or ultrasonography) and reduction medium (liquid or air). Successful enema reduction is less likely to be achieved in children with a longer symptom duration, younger age, lethargy, fever, bloody diarrhea, unfavorable radiologic findings (small bowel obstruction, trapped fluid, ascites, absence of flow in the intussusception, intussusception in the left-sided colon), and pathological lead points. This review highlights the current concepts of intussusception diagnosis, nonsurgical enema reduction, success rates, predictors of failed enema reduction, complications, and recurrence to guide general pediatricians in the management of childhood intussusception.
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Background: To demonstrate the magnetic resonance lymphangiography (MRL) imaging findings of lymphatic diseases and the clinical outcomes of lymphatic embolization in pediatric patients. Methods and Results: This retrospective study included 10 consecutive pediatric patients who underwent MRL for lymphatic diseases between June 2017 and June 2021. Nine patients underwent dynamic contrast-enhanced MRL with bilateral inguinal lymph node injection of diluted gadolinium, and one patient underwent nonenhanced MRL with a heavily T2-weighted image. The etiology of lymphatic disease was classified into three categories based on the magnetic resonance findings. The resolution of chylous fluid and weight-adjusted amounts of chylous fluid collected from a drainage tube were evaluated as outcomes. Patients were classified as postoperative lymphatic leak (n = 3), pulmonary lymphatic perfusion syndrome (n = 3), central lymphatic flow disorder (CLFD; n = 3), and primary lymphatic dysfunction (Gorham-Stout syndrome; n = 1). Three patients underwent radiological lymphatic intervention, and one CLFD patient underwent surgical intervention. In patients with postoperative lymphatic leak, the median chest tube drainage decreased significantly after the intervention [from 87.9 to 12.4 mL/(kg·d); p = 0.02]. However, in one CLFD patient, the amount of chylous fluid did not decrease until 7 days after intervention. Conclusion: The etiology of lymphatic disease in pediatrics can be recognized on MRL, and lymphatic intervention can be performed for cessation of lymphatic leak, even though the treatment outcomes may differ according to the underlying etiology. MRL can play an important role in classifying lymphatic disease, and in planning treatment on the basis of the lymphatic anatomy and underlying etiology.
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Enfermedades Linfáticas , Linfografía , Angiografía por Resonancia Magnética , Intensificación de Imagen Radiográfica , Linfografía/métodos , Angiografía por Resonancia Magnética/métodos , Enfermedades Linfáticas/diagnóstico por imagen , Estudios Retrospectivos , Intensificación de Imagen Radiográfica/métodos , Medios de Contraste , Humanos , Masculino , Femenino , Lactante , Preescolar , AdolescenteRESUMEN
OBJECTIVES: To evaluate the diagnostic performance of 2021 K-TIRADS biopsy criteria for detecting malignant thyroid nodules in a pediatric population, making comparisons with 2016 K-TIRADS. METHODS: This retrospective study included pediatric patients with histopathologically confirmed diagnoses. The diagnostic performance of 2021 K-TIRADS was compared with that of 2016 K-TIRADS. Simulation studies were performed by changing biopsy cut-off sizes for K-TIRADS 5 to 1.0 cm (K-TIRADS5-1.0cm) and 0.5 cm (K-TIRADS5-0.5cm), and for K-TIRADS 4 to 1.0 cm (K-TIRADS4-1.0cm) and 1.0-1.5 cm (K-TIRADS4-1.0~1.5cm). Subgroup analysis was performed in small (< 1.5 cm) and large nodules (≥ 1.5 cm). RESULTS: Two hundred seventy-seven thyroid nodules (54.9% malignant) from 221 pediatric patients were analyzed. All simulated 2021 K-TIRADS showed higher accuracy than 2016 K-TIRADS. Compared with 2021 K-TIRADS5-1.0cm, 2021 K-TIRADS5-0.5cm showed lower specificity (51.6% vs. 47.9%; p = 0.004) but higher sensitivity (77.2% vs. 90.3%; p < 0.001) and accuracy (62.7% vs. 68.9%; p < 0.001). Compared with 2021 K-TIRADS4-1.0cm, 2021 K-TIRADS4-1.0~1.5cm showed higher specificity (44.9% vs. 47.9%; p = 0.018) without significant difference in other diagnostic measures. Compared with 2016 K-TIRADS, 2021 K-TIRADS (biopsy cut-offs, 0.5 cm for K-TIRADS 5; 1.0-1.5 cm for K-TIRADS 4) showed higher sensitivity (34.0% vs. 67.3%; p < 0.001) while maintaining specificity (89.4% vs. 88.2%; p = 0.790) in small nodules, and higher specificity (5.9% vs. 25.4%; p < 0.001) while maintaining sensitivity (100% vs. 98.7%; p = 0.132) in large nodules. CONCLUSIONS: In pediatric patients, 2021 K-TIRADS showed superior diagnostic accuracy to 2016 K-TIRADS, especially with a biopsy cut-off of 0.5 cm for K-TIRADS 5 and 1.0-1.5 cm for K-TIRADS 4. KEY POINTS: ⢠All simulated 2021 K-TIRADS showed higher accuracy than 2016 K-TIRADS. ⢠2021 K-TIRADS with cut-off size for K-TIRADS 5 of 0.5 cm showed lower specificity but higher sensitivity and accuracy than that of 1.0 cm. ⢠Compared with 2016 K-TIRADS, 2021 K-TIRADS (biopsy cut-offs, 0.5 cm for K-TIRADS 5; 1.0-1.5 cm for K-TIRADS 4) showed higher sensitivity while maintaining specificity in small nodules, and higher specificity while maintaining sensitivity in large nodules.
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Neoplasias de la Tiroides , Nódulo Tiroideo , Humanos , Niño , Nódulo Tiroideo/diagnóstico por imagen , Estudios Retrospectivos , Neoplasias de la Tiroides/patología , Ultrasonografía/métodos , Medición de Riesgo/métodos , República de Corea/epidemiologíaRESUMEN
Background and Aims: Biliary atresia is a rare and devastating bile duct disease that occurs during the neonatal period. Timely identification and prompt surgical intervention is critical for improving the outcome. The aim of the study was to develop a new machine learning-based prediction model for the detection of biliary atresia. Methods: Neonates aged <100 days with cholestasis at least once were retrospectively screened in 2 tertiary referral hospitals between 2015 and 2020. Simple demographic data, routine laboratory indices, and imaging findings of ultrasonography and hepatobiliary scintigraphy were used as features in the multivariate analysis. The extreme gradient boosting (XGBoost) framework was used to develop prediction models according to the diagnostic steps. Results: Among 1605 enrolled neonates with all-cause cholestasis, 145 (9%) were included as having biliary atresia. Direct bilirubin, gamma-glutamyl transpeptidase, abdominal sonography, and hepatobiliary scan were the most impactful features in prediction models. The Step II XGBoost model, consisting of nonimaging inputs, showed excellent discriminatory performance (area under the curve = 0.97). The Step III and IV XGBoost models showed near-perfect performances (area under the curve = 0.998 and 0.999, respectively). In external validation (n = 912 with 118 [12.9%] biliary atresia), XGBoost-based prediction models consistently showed acceptable performances. Utilizing shapley additive explanation values also provided visualized insight and explanation of the contribution of features in detecting biliary atresia. The models were integrated into a web-based diagnostic tool for case-level application. Conclusion: We introduced a new machine learning-based prediction model for detecting biliary atresia in the largest cohorts of neonatal cholestasis.
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The Kasai portoenterostomy is the first-line treatment for the restoration of the flow of bile to the small intestine in patients with biliary atresia. Various complications can occur after Kasai portoenterostomy, including ascending cholangitis, biliary cirrhosis, and portal hypertension. Of these potential complications, ascending cholangitis in the most common. In cases of patients having uncontrolled complications due to progressive liver cirrhosis, portal hypertension, or progressive hyperbilirubinemia, liver transplantation is the indicated as treatment plan. Lifelong follow-up, particularly involving imaging studies, is important for the identification of various complications arising from biliary atresia after Kasai portoenterostomy. Additionally, imaging studies play a crucial role in the evaluation of potential liver donors and recipients. US is a key imaging modality utilized in the management of patients who undergo Kasai portoenterostomy, while CT and MRI are imperative to obtaining an accurate diagnosis.
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OBJECTIVE: To evaluate associations between postnatal imaging features and outcome of left-sided congenital diaphragmatic hernia, as defined by overall survival and a requirement for extracorporeal membrane oxygenation (ECMO). STUDY DESIGN: Newborns diagnosed prenatally with left-sided congenital diaphragmatic hernia between January 2013 and September 2021 were studied retrospectively. The esophageal deviation index was newly defined as the largest diameter from the midline to deviated gastric tube divided by the largest transverse diameter of the thoracic cavity on the radiograph. Regression analyses were performed to identify postnatal imaging features associated with overall survival and a requirement for ECMO. The predictive power (ie, area under the curve [AUC] of a time-dependent receiver operating characteristic curve) of prenatal, postnatal, and intraoperative findings for predicting survival were calculated. RESULTS: Ninety-seven patients (54 males; mean gestational age, 38.3 ± 1.9 weeks; mean birth weight, 2956.5 ± 540.0 g) were analyzed. The esophageal deviation index (adjusted hazard ratio [HR], moderate [≥0.19 to <0.24], 6.427 [P = .029]; severe [≥0.24], 33.007 [P < .001]) and right pneumothorax (adjusted HR, 8.763; P = .002) were associated with overall survival and with a requirement for ECMO. Liver herniation on postnatal ultrasound also was associated with overall survival (P < .001) and need for ECMO (P = .001). In addition, the AUC for prediction of 1-year survival from postnatal ultrasound was comparable with that of prenatally or intraoperatively detected liver herniation (0.93; 95% CI, 0.88-0.97). CONCLUSIONS: The esophageal deviation index, right pneumothorax, and liver herniation observed by postnatal imaging have prognostic value in patients with left-sided congenital diaphragmatic hernia.
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Hernias Diafragmáticas Congénitas , Neumotórax , Embarazo , Masculino , Femenino , Humanos , Recién Nacido , Lactante , Hernias Diafragmáticas Congénitas/diagnóstico por imagen , Hernias Diafragmáticas Congénitas/cirugía , Estudios Retrospectivos , Ultrasonografía Prenatal/métodos , Hígado/diagnóstico por imagenRESUMEN
Background The validation of adult-based US risk stratification systems (RSSs) in the discrimination of malignant thyroid nodules in a pediatric population remains lacking. Purpose To estimate and compare the diagnostic performance of pediatric US RSSs based on five adult-based RSSs in the discrimination of malignant thyroid nodules in a pediatric sample. Materials and methods Pediatric patients (age ≤18 years) with histopathologically confirmed US-detected thyroid nodules at a tertiary referral hospital between January 2000 and April 2020 were analyzed retrospectively. The diagnostic performance of US-based fine-needle aspiration biopsy (FNAB) criteria in thyroid cancer detection was estimated. The following sensitivity analyses were performed: (a) scenario 1: nodules smaller than 1 cm, with the highest category additionally biopsied; (b) scenario 2, application of American College of Radiology Thyroid Imaging Reporting and Data System nodule size cutoffs to other RSSs; (c) scenario 3, scenarios 1 and 2 together. Generalized estimating equations (GEEs) were used for estimation. Results A total of 277 thyroid nodules in 221 pediatric patients (median age, 16 years [interquartile range {IQR}, 13-17]; 172 female; 152 of 277 patients [55%] malignant) were analyzed. The GEE-estimated sensitivity and specificity ranged from 70% to 78% (104 to 119 of 152 patients, based on each reader's interpretation) and from 42% to 78% (49 of 124 patients to 103 of 125 patients). In scenario 1, the missed malignancy rate was reduced from 32%-38% (41 of 134 patients to 34 of 83 patients) to 15%-21% (eight of 59 patients to 28 of 127 patients). In scenario 2, the unnecessary biopsy rate was reduced from 35%-39% (60 of 176 patients to 68 of 175 patients) to 20%-34% (18 of 109 patients to 62 of 179 patients). The highest accuracy was noted in scenario 3 (range, 71%-81%; 199 of 277 patients to 216 of 262 patients). Conclusion The diagnostic performances of the fine-needle aspiration biopsy criteria of five adult-based risk stratification systems were acceptable in the pediatric population and were improved by applying the American College of Radiology Thyroid Imaging Reporting and Data System size cutoff for nodules 1 cm or larger and allowing biopsy of the highest category nodules smaller than 1 cm. © RSNA, 2022 Online supplemental material is available for this article.
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Neoplasias de la Tiroides , Nódulo Tiroideo , Adolescente , Adulto , Biopsia con Aguja Fina , Niño , Femenino , Humanos , Reproducibilidad de los Resultados , Estudios Retrospectivos , Medición de Riesgo , Neoplasias de la Tiroides/diagnóstico por imagen , Neoplasias de la Tiroides/patología , Nódulo Tiroideo/diagnóstico por imagen , Nódulo Tiroideo/patología , Ultrasonografía/métodosRESUMEN
PURPOSE: To evaluate the applicability of Greulich-Pyle (GP) standards to bone age (BA) assessment in healthy Korean children using manual and deep learning-based methods. MATERIALS AND METHODS: We collected 485 hand radiographs of healthy children aged 2-17 years (262 boys) between 2008 and 2017. Based on GP method, BA was assessed manually by two radiologists and automatically by two deep learning-based BA assessment (DLBAA), which estimated GP-assigned (original model) and optimal (modified model) BAs. Estimated BA was compared to chronological age (CA) using intraclass correlation (ICC), Bland-Altman analysis, linear regression, mean absolute error, and root mean square error. The proportion of children showing a difference >12 months between the estimated BA and CA was calculated. RESULTS: CA and all estimated BA showed excellent agreement (ICC ≥0.978, p<0.001) and significant positive linear correlations (R²≥0.935, p<0.001). The estimated BA of all methods showed systematic bias and tended to be lower than CA in younger patients, and higher than CA in older patients (regression slopes ≤-0.11, p<0.001). The mean absolute error of radiologist 1, radiologist 2, original, and modified DLBAA models were 13.09, 13.12, 11.52, and 11.31 months, respectively. The difference between estimated BA and CA was >12 months in 44.3%, 44.5%, 39.2%, and 36.1% for radiologist 1, radiologist 2, original, and modified DLBAA models, respectively. CONCLUSION: Contemporary healthy Korean children showed different rates of skeletal development than GP standard-BA, and systemic bias should be considered when determining children's skeletal maturation.
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Determinación de la Edad por el Esqueleto , Aprendizaje Profundo , Determinación de la Edad por el Esqueleto/métodos , Anciano , Pueblo Asiatico , Niño , Humanos , Masculino , Radiografía , República de CoreaRESUMEN
PURPOSE: This study evaluated the accuracy of attenuation imaging (ATI) for the assessment of hepatic steatosis in pediatric patients, in comparison with the FibroScan vibration-controlled transient elastography controlled attenuation parameter (CAP). METHODS: Consecutive pediatric patients referred for evaluation of obesity who underwent both ATI and FibroScan between February 2020 and September 2021 were included. The correlation between attenuation coefficient (AC) and CAP values was assessed using the Spearman test. The AC cutoff value for discriminating hepatic steatosis corresponding to a CAP value of 241 dB/m was calculated. Multivariable linear regression analysis was performed to estimate the strength of the association between AC and CAP. The diagnostic accuracy of AC cutoffs was estimated using the imperfect gold-standard methodology based on a two-level Bayesian latent class model. RESULTS: Seventy patients (median age, 12.5 years; interquartile range, 11.0 to 14.0 years; male:female, 58:12) were included. AC and CAP showed a moderate-to-good correlation (ρ =0.646, P<0.001). Multivariable regression analysis affirmed the significant association between AC and CAP (P<0.001). The correlation was not evident in patients with a body mass index ≥30 kg/m2 (ρ=-0.202, P=0.551). Linear regression revealed that an AC cutoff of 0.66 dB/cm/MHz corresponded to a CAP of 241 dB/m (sensitivity, 0.93; 95% confidence interval [CI], 0.85 to 0.98 and specificity, 0.87; 95% CI, 0.56 to 1.00). CONCLUSION: ATI showed an acceptable correlation with CAP values in a pediatric population, especially in patients with a body mass index <30 kg/m2. An AC cutoff of 0.66 dB/cm/MHz, corresponding to a CAP of 241 dB/m, can accurately diagnose hepatic steatosis.
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BACKGROUND AND OBJECTIVES: Although the recent approval of selumetinib is expected to transform the management of children with neurofibromatosis type 1 (NF1), particularly those with symptomatic and inoperable plexiform neurofibromas, no systematic review has summarized its efficacy and safety based on the latest studies. This study was conducted to systematically evaluate the efficacy and safety of selumetinib in children with NF1. METHODS: Original articles reporting the efficacy and safety of selumetinib in patients with NF1 were identified in PubMed and EMBASE up to January 28, 2021. The pooled objective response rates (ORRs) and disease control rates (DCRs) were calculated using the DerSimonian-Laird method based on random-effects modeling. The pooled proportion of adverse events (AEs) was also calculated. The quality of the evidence was assessed using the Grading of Recommendations, Assessment, Development and Evaluation system. RESULTS: Five studies involving 126 patients were included in our analysis. The studies had a very low to moderate quality of the evidence. The pooled ORR was 73.8% (95% CI 57.3%-85.5%) and the DCR was 92.5% (95% CI 66.5%-98.7%). The 2 most common AEs were diarrhea, which had a pooled rate of 63.8% (95% CI 52.9%-73.4%), and an increase in creatine kinase levels, which had a pooled rate of 63.3% (95% CI 35.6%-84.3%). DISCUSSION: Our results indicate that selumetinib is an effective and safe treatment for pediatric patients with symptomatic, inoperable plexiform neurofibromas. Further larger-scale randomized controlled studies are needed to confirm the long-term outcome of patients treated with this drug.
Asunto(s)
Neurofibroma Plexiforme , Neurofibromatosis 1 , Bencimidazoles/efectos adversos , Niño , Diarrea , Humanos , Neurofibromatosis 1/complicaciones , Neurofibromatosis 1/tratamiento farmacológicoRESUMEN
BACKGROUND: Neurofibromatosis type 1 (NF1) is a common human genetic disease with age-dependent phenotype progression. The overview of clinical and radiological findings evaluated by whole-body magnetic resonance imaging (WBMRI) in NF1 patients < 3 years old assessed with a genetic contribution to disease progression is presented herein. METHODS: This study included 70 clinically or genetically diagnosed NF1 patients who received WBMRI before 3 years old. Clinical, genetic, and radiologic features were collected by retrospective chart review. In NF1+, widely spread diffuse cutaneous neurofibromas, developmental delay, autism, seizure, cardiac abnormalities, hearing defect, optic pathway glioma, severe plexiform neurofibromas (> 3 cm in diameter, disfigurement, accompanying pain, bony destruction, or located para-aortic area), brain tumors, nerve root tumors, malignant peripheral nerve sheath tumors, moyamoya disease, and bony dysplasia were included. RESULTS: The age at WBMRI was 1.6 ± 0.7 years old, and NF1 mutations were found in 66 patients (94.3%). Focal areas of signal intensity (FASI) were the most common WBMRI finding (66.1%), followed by optic pathway glioma (15.7%), spine dural ectasia (12.9%), and plexiform neurofibromas (10.0%). Plexiform neurofibromas and NF1+ were more prevalent in familial case (28.7% vs 5.7%, p = 0.030; 71.4% vs 30.2%, p = 0.011). Follow-up WBMRI was conducted in 42 patients (23 girls and 19 boys) after 1.21 ± 0.50 years. FASI and radiologic progression were more frequent in patients with mutations involving GTPase activating protein-related domain (77.8% vs 52.4%, p = 0.047; 46.2% vs 7.7%, p = 0.029). CONCLUSIONS: WBMRI provides important information for the clinical care for young pediatric NF1 patients. As NF1 progresses in even these young patients, and is related to family history and the affected NF1 domains, serial evaluation with WBMRI should be assessed based on the clinical and genetic features for the patients' best care.