Mapping function from FACT-B to EQ-5D-5 L using multiple modelling approaches: data from breast cancer patients in China.

BACKGROUND: The Functional Assessment of Cancer Therapy-Breast (FACT-B) is the most commonly used scale for assessing quality of life in patients with breast cancer. The lack of preference-based measures limits the cost-utility of breast cancer in China. The goal of this study was to explore whether a mapping function can be established from the FACT-B to the EQ-5D-5 L when the EQ-5D health-utility index is not available. METHODS: A cross-sectional survey of adults with breast cancer was conducted in China. All patients included in the study completed the EQ-5D-5 L and the disease-specific FACT-B questionnaire, and demographic and clinical data were also collected. The Chinese tariff value was used to calculate the EQ-5D-5 L utility scores. Five models were evaluated using three different modelling approaches: the ordinary least squares (OLS) model, the Tobit model and the two-part model (TPM). Total scores, domain scores, squared terms and interaction terms were introduced into models. The goodness of fit, signs of the estimated coefficients, and normality of prediction errors of the model were also assessed. The normality of the prediction error is determined by calculating the root mean squared error (RMSE), the mean absolute deviation (MAD), and the mean absolute error (MAE). Akaike information criteria (AIC) and Bayes information criteria (BIC) were also used to assess models and predictive performances. The OLS model was followed by simple linear equating to avoid regression to the mean. RESULTS: The performance of the models was improved after the introduction of the squared terms and the interaction terms. The OLS model, including the squared terms and the interaction terms, performed best for mapping the EQ-5D-5 L. The explanatory power of the OLS model was 70.0%. The AIC and BIC of this model were the smallest (AIC = -705.106, BIC = -643.601). The RMSE, MAD and MAE of the OLS model, Tobit model and TPM were similar. The MAE values of the 5-fold cross-validation of the multiple models in this study were 0.07155~0.08509; meanwhile, the MAE of the TPM was the smallest, followed by that of the OLS model. The OLS regression proved to be the most accurate for the mean, and linearly equated scores were much closer to observed scores. CONCLUSIONS: This study establishes a mapping algorithm based on the Chinese population to estimate the EQ-5D-5 L index of the FACT-B and confirms that OLS models have higher explanatory power and that TPMs have lower prediction error. Given the accuracy of the mean prediction and the simplicity of the model, we recommend using the OLS model. The algorithm can be used to calculate EQ-5D scores when EQ-5D data are not directly collected in a study.

[Precision genomic and translational medicine for acute myeloid leukemia].

Leukemia is a group of hematologic malignancy that has unfavorable prognosis and unclear mechanisms. In recent years, advances in leukemia research encompass the discovery of novel targets in acute myeloid leukemia drug resistance, epigenetic crosstalk in mixed lineage leukemia (MLL) leukemogenesis, genetic mechanisms of aggressive NK-cell leukemia, as well as the critical role of key epigenetic regulator in acute myeloid malignancy. Remarkably, researchers revealed that the histone modifying gene SETD2 as a new tumor suppressor and therapeutic target in patients with acute myeloid leukemia. Furthermore, low-dose chemotherapy as a frontline regiment in treating pediatric acute myeloid leukemia can substantially reduce the toxic side effects and treatment costs without impairing efficacy. Although advances in cancer genomics have greatly increased our understanding of the molecular characteristics in tumor biology, recent studies suggest that Darwinian evolution of intratumor heterogeneity represents a major challenge to develop therapeutic strategies to improve disease control. Researchers also dissected the distinct evolutionary dynamics under different chemotherapy regimens and the corresponding applications in the evaluation of treatment outcomes. Altogether, these efforts offered new opportunities for the development of acute myeloid leukemia diagnostics and therapeutics.

The Complementary Relationship Between Echocardiography and Multi-Slice Spiral CT Coronary Angiography in the Diagnosis of Coronary Artery Thrombosis in Children With Kawasaki Disease.

Aim: To compare the diagnostic values by using transthoracic echocardiography (ECHO) and multi-slice spiral CT coronary angiography (CTCA) for identifying coronary artery thrombosis in children with Kawasaki disease (KD). Methods: Total 97 KD children with coronary artery dilation complications in our hospital from June 2012 to December 2020 were included in the study. CTCA and ECHO were performed after over 1 month of illness. Results: Coronary artery thrombosis was found in 14 out of 97 patients. Among them, 10 were identified as positive by CTCA, 9 were identified as positive by ECHO, and 5 were identified as positive by both CTCA and ECHO. Conclusion: Both CTCA and ECHO can be used to diagnose coronary artery thrombosis. ECHO has advantage in identifying low-density thrombus, and CTCA is better for the clot in distal coronary artery. They can complement each other.

Correlation Between Arrhythmia and the Prognosis in Children With EFE/LVNC/DCM.

Aim: To explore the correlation between different phenotypes of arrhythmia and the prognosis in children with EFE/LVNC/DCM. Methods: A total of 167 children with cardiomyopathy diagnosed and treated in Shengjing Hospital between January 2010 and May 2019 were evaluated. After patient screening, 31 patients with endomyocardial fibroelastosis (EFE), left ventricular non-compaction, or dilated cardiomyopathy with significant arrhythmias were selected. In addition, 42 children with primary EFE were selected to evaluate the prognosis with or without arrhythmia. Follow-up was undertaken 0, 1, 3, 6, 9, and 12 months after treatment. Results: We revealed the outcomes for five types of cardiomyopathy: EFE patients with Wolff-Parkinson-White syndrome B and supraventricular tachycardia, intraventricular block and complete left bundle branch block recovered slower than EFE patients with atrial flutter and atrial fibrillation, even slower than EFE with ventricular tachycardia. The average recovering time for LVEF and LVED in EFE patients without arrythmia was 10 months after diagnosis, while 76.9% (3/13 cases) of those with significant arrythmia hadn't recovered until 24 months after diagnosis. Three of patients died at 6, 7, and 6 and half years after diagnosis. Conclusion: The long-term prognosis in children with cardiomyopathy is associated with the type of arrhythmia and time of intervention. The prognosis of EFE patients with arrhythmia is worse than EFE patients without arrhythmia. Patients with Wolff-Parkinson-White syndrome B, especially a significantly widen QRS complex, carry a poor prognosis if radiofrequency ablation is not undertaken. CLBBB patients have similar poor prognosis if proper pacemaker is not implanted timely.