Longitudinal changes in magnetic resonance imaging biomarkers of the gluteal muscle groups and functional ability in Duchenne muscular dystrophy: a 12-month cohort study.

BACKGROUND: Quantitative magnetic resonance imaging (MRI) is considered an objective biomarker of Duchenne muscular dystrophy (DMD), but the longitudinal progression of MRI biomarkers in gluteal muscle groups and their predictive value for future motor function have not been described. OBJECTIVE: To explore MRI biomarkers of the gluteal muscle groups as predictors of motor function decline in DMD by characterizing the progression over 12 months. MATERIALS AND METHODS: A total of 112 participants with DMD were enrolled and underwent MRI examination of the gluteal muscles to determine fat fraction and longitudinal relaxation time (T1). Investigations were based on gluteal muscle groups including flexors, extensors, adductors, and abductors. The North Star Ambulatory Assessment and timed functional tests were performed. All participants returned for follow-up at an average of 12 months and were divided into two subgroups (functional stability/decline groups) based on changes in timed functional tests. Univariable and multivariable logistic regression methods were used to explore the risk factors associated with future motor function decline. RESULTS: For the functional decline group, all T1 values decreased, while fat fraction values increased significantly over 12 months (P<0.05). For the functional stability group, only the fat fraction of the flexors and abductors increased significantly over 12 months (P<0.05). The baseline T1 value was positively correlated with North Star Ambulatory Assessment and negatively correlated with timed functional tests at the 12-month follow-up (P<0.001), while the baseline fat fraction value was negatively correlated with North Star Ambulatory Assessment and positively correlated with timed functional tests at the 12-month follow-up (P<0.001). Multivariate regression showed that increased fat fraction of the abductors was associated with future motor function decline (model 1: odds ratio [OR]=1.104, 95% confidence interval [CI]: 1.026~1.187, P=0.008; model 2: OR=1.085, 95% CI: 1.013~1.161, P=0.019), with an area under the curve of 0.874. CONCLUSION: Fat fraction of the abductors is a powerful predictor of future motor functional decline in DMD patients at 12 months, underscoring the importance of focusing early on this parameter in patients with DMD.

Bone Mineral Density Assessment by Quantitative Computed Tomography in Glucocorticoid-Treated Boys With Duchenne Muscular Dystrophy: A Linear Mixed-Effects Modeling Approach.

Objective: Boys with Duchenne muscular dystrophy (DMD) are at risk of bone damage and low bone mineral density (BMD). The aim of the study is to examine lumbar BMD values measured by QCT and identify the factors associated with BMD loss using a multilevel mixed-effects model. Methods: Lumbar BMD was evaluated by quantitative computed tomography (QCT) at diagnosis, 1 and 2 years follow up in patients with DMD who were treated with GC. Demographic data, functional activity scores (FMSs), laboratory parameters and steroid use were recorded. A multilevel mixed-effects model was used to analyze BMD loss. Results: Nineteen patients with DMD who had a total of sixty complete records between January 2018 and October 2021 were retrospectively analyzed. At baseline, 15.8% of patients (3/19) had low lumbar BMD (Z score ≤ -2), and the mean BMD Z score on QCT was -0.85 (SD 1.32). The mean BMD Z score at 1 and 2 years postbaseline decreased to -1.56 (SD 1.62) and -2.02 (SD 1.36), respectively. In our model, BMD Z score loss was associated with age (ß=-0.358, p=0.0003) and FMS (ß=-0.454, p=0.031). Cumulative GC exposure and serum levels of calcium, phosphorus, 25(OH)-vitamin D and creatinine kinase did not independently predict BMD loss. Conclusions: This study demonstrates that in DMD patients, lumbar BMD decreased gradually and progressively. Age and FMS are the main contributors to BMD loss in boys with DMD. Early recognition of risk factors associated with BMD loss may facilitate the development of strategies to optimize bone health.

A CT-based radiomics nomogram for predicting early recurrence in patients with high-grade serous ovarian cancer.

PURPOSE: To develop and validate a radiomics nomogram for predicting early recurrence in high-grade serous ovarian cancer (HGSOC) patients. MATERIALS AND METHODS: From May 2008 to December 2019, 256 eligible HGSOC patients were enrolled and divided into training (n = 179) and test cohorts (n = 77) in a 7:3 ratio. A radiomics signature (Radscore) was selected by using recursive feature elimination based on a support vector machine (SVM-RFE) and building a radiomics model for recurrence prediction. Independent clinical risk factors were generated by univariable and multivariable Cox regression analyses. A combined model was developed based on the Radscore and independent clinical risk factors and presented as a radiomics nomogram. Its performance was assessed by AUC, Kaplan-Meier survival analysis and decision curve analysis. RESULTS: Seven radiomics features were selected. The radiomics model yielded AUCs of 0.715 (95% CI: 0.640, 0.790) and 0.717 (95% CI: 0.600, 0.834) in the training and test cohorts, respectively. The clinical model (FIGO stage and residual disease) yielded AUCs of 0.632 and 0.691 in the training and test cohorts, respectively. The combined model demonstrated AUCs of 0.749 (95% CI: 0.678, 0.821) and 0.769 (95% CI: 0.662, 0.877) in the training and test cohorts, respectively. In the combined model, PFS was significantly shorter in the high-risk group than in the low-risk group (P < 0.0001). CONCLUSIONS: The radiomics nomogram performed well for early individualized recurrence prediction in patients with HGSOC and can also be used to differentiate high-risk patients from low-risk patients.

The Development and Validation of a CT-Based Radiomics Nomogram to Preoperatively Predict Lymph Node Metastasis in High-Grade Serous Ovarian Cancer.

PURPOSE: To develop and validate a radiomics model for predicting preoperative lymph node (LN) metastasis in high-grade serous ovarian cancer (HGSOC). MATERIALS AND METHODS: From May 2008 to January 2018, a total of 256 eligible HGSOC patients who underwent tumor resection and LN dissection were divided into a training cohort (n=179) and a test cohort (n=77) in a 7:3 ratio. A Radiomics Model was developed based on a training cohort of 179 patients. A radiomics signature (defined as the Radscore) was selected by using the random forest method. Logistics regression was used as the classifier for modeling. An Integrated Model that incorporated the Radscore and CT_reported LN status (CT_LN_report) was developed and presented as a radiomics nomogram. Its performance was determined by the area under the curve (AUC), calibration, and decision curve. The radiomics nomogram was internally tested in an independent test cohort (n=77) and a CT-LN-report negative subgroup (n=179) using the formula derived from the training cohort. RESULTS: The AUC value of the CT_LN_report was 0.688 (95% CI: 0.626, 0.759) in the training cohort and 0.717 (95% CI: 0.630, 0.804) in the test cohort. The Radiomics Model yielded an AUC of 0.767 (95% CI: 0.696, 0.837) in the training cohort and 0.753 (95% CI: 0.640, 0.866) in the test. The radiomics nomogram demonstrated favorable calibration and discrimination in the training cohort (AUC=0.821), test cohort (AUC=0.843), and CT-LN-report negative subgroup (AUC=0.82), outperforming the Radiomics Model and CT_LN_report alone. CONCLUSIONS: The radiomics nomogram derived from portal phase CT images performed well in predicting LN metastasis in HGSOC and could be recommended as a new, convenient, and non-invasive method to aid in clinical decision-making.

[TW systems in estimation of carpal bone age and their potential applications in diagnosis of idiopathic precocious puberty in Chinese girls].

OBJECTIVE: To compare three assessment methods (TW2, TW2CH, and TW3) for carpal bone age and their potential applications in diagnosis of idiopathic precocious puberty (IPP) in Chinese girls. METHODS: Fifty-five (55) girls with IPP and 83 normal girls as control group were selected in this study. The X-ray films of the left hand-wrist taken at their first visit were analyzed retrospectively. Three assessment methods were used to estimate the carpal bone age with single-blinded method and percentiles were set at 5 different decision thresholds (1)>97th percentile, (2)>90 th percentile, (3)>75th percentile, (4)>50 th percentile, and (5)< or =50th percentile. RESULTS: All of the three methods showed similar high sensitivity and specificity at the threshold above 90th percentile. CONCLUSION: Our data indicate that all of the three methods for estimation of the carpal bone age are useful in diagnosis of IPP. TW2CH and TW3 methods appear to be superior to TW2 method.