Beijing Children's Hospital guidelines on the design and conduction of the first standardized database for medulloblastoma.

Medulloblastoma (MB) is one of the most common malignant childhood brain tumors (WHO grade IV). Its high degree of malignancy leads to an unsatisfactory prognosis, requiring more precise and personalized treatment in the near future. Multi-omics and artificial intelligence have been playing a significant role in precise medical research, but their implementation needs a large amount of clinical information and biomaterials. For these reasons, it is urgent for current MB researchers to establish a large sample-size database of MB that contains complete clinical data and sufficient biomaterials such as blood, cerebrospinal fluid (CSF), cancer tissue, and urine. Unfortunately, there are few biobanks of pediatric central nervous system (CNS) tumors throughout the world for limited specimens, scarce funds, different standards collecting methods and et cl. Even though, China falls behind western countries in this area. The present research set up a standard workflow to construct the Beijing Children's Hospital Medulloblastoma (BCH-MB) biobank. Clinical data from children with MB and for collecting and storing biomaterials, along with regular follow-up has been collected and recorded in this database. In the future, the BCH-MB biobank could make it possible to validate the promising biomarkers already identified, discover unrevealed MB biomarkers, develop novel therapies, and establish personalized prognostic models for children with MB upon the support of its sufficient data and biomaterials, laying the foundation for individualized therapies of children with MB.

Pediatric spinal cord injury with radiographic abnormality: the Beijing experience.

BACKGROUND CONTEXT: Spinal cord injury (SCI) without radiographic abnormality (SCIWORA) is a syndrome that usually occurs in children primarily because of the unique biomechanics of the pediatric spine. We recently found that the histopathological and behavioral effects of SCI with radiographic abnormality (SCIWRA) and SCIWORA are very different from each other in animal models. Although numerous studies were conducted to understand the epidemiological and clinical characteristics of the overall pediatric SCI population and the pediatric SCIWORA population, the characteristics of the pediatric SCIWRA population and their differences from those of the SCIWORA population are poorly understood. PURPOSE: To describe the epidemiology and clinical outcomes of pediatric patients with SCIWRA and their differences from those with SCIWORA. STUDY DESIGN/SETTING: Retrospective study. PATIENT SAMPLE: A total of 47 pediatric SCIWRA patients. OUTCOME MEASURES: Epidemiological characteristics, injury severities, functional deficits, and management and recovery outcomes. METHODS: Review of all cases with SCIWRA at Beijing Children's Hospital between July 2007 and December 2019 and comparison between the present data and our previous SCIWORA data. RESULTS: Of the 187 pediatric SCI patients, 47 had SCIWRA (age: 7.06 ± 3.75 years, male-to-female ratio: 3:2). Main causes of SCIWRA were fall (38%) and traffic accidents (38%). Lesions were often located at multiple levels (62%). Incubation period was 3 ± 18 hours. According to the American Spinal Injury Association impairment scale (AIS), many SCIWRA patients had incomplete impairment (AIS B, 9%; AIS C, 9%; AIS D, 32%). Specifically, many of them had abnormal upper and lower limb muscle powers (55% and 60%), upper and lower limb muscle tones (34% and 49%), sensation (38%), and knee, ankle, and abdominal reflexes (47%, 34%, and 36%). 72% of SCIWRA patients were treated with methylprednisolone, dexamethasone, or both. 81% of them showed neurological improvement before discharge. There was no association between corticosteroid therapy and neurological improvement. Moreover, functional outcomes of their upper and lower limb muscle powers were significantly associated with functional outcomes of their upper and lower limb muscle tones (p < 0.01), respectively. In comparison to the SCIWRA population, the SCIWORA population had a higher ratio of younger and female patients of sports-related thoracic injuries with long incubation period leading to lower-body deficits and complete impairment (p<0.05 or p<0.01). Despite all the differences, their neurological improvement was similar (p>0.05). CONCLUSIONS: Demographic differences exist in the SCIWRA population. Corticosteroids do not appear to be effective in the different types of pediatric SCI. Limb muscle tone may be used to evaluate the functional status of limb muscle power. The epidemiological and clinical characteristics of SCIWRA and SCIWORA are very different from each other. It is important to formulate tailor-made prevention, evaluation, and management strategies for the pediatric population to optimize the SCI outcomes.

An Immune-Related Gene Pairs Signature Predicts Prognosis and Immune Heterogeneity in Glioblastoma.

PURPOSE: Glioblastoma is one of the most aggressive nervous system neoplasms. Immunotherapy represents a hot spot and has not been included in standard treatments of glioblastoma. So in this study, we aim to filtrate an immune-related gene pairs (IRGPs) signature for predicting survival and immune heterogeneity. METHODS: We used gene expression profiles and clinical information of glioblastoma patients in the TCGA and CGGA datasets, dividing into discovery and validation cohorts. IRGPs significantly correlative with prognosis were selected to conduct an IRGPs signature. Low and high risk groups were separated by this IRGPs signature. Univariate and multivariate cox analysis were adopted to check whether risk can be a independent prognostic factor. Immune heterogeneity between different risk groups was analyzed via immune infiltration and gene set enrichment analysis (GSEA). Some different expressed genes between groups were selected to determine their relationship with immune cells and immune checkpoints. RESULTS: We found an IRGPs signature consisting of 5 IRGPs. Different risk based on IRGPs signature is a independent prognostic factor both in the discovery and validation cohorts. High risk group has some immune positive cells and more immune repressive cells than low risk group by means of immune infiltration. We discovered some pathways are more active in the high risk group, leading to immune suppression, drug resistance and tumor evasion. In two specific signaling, some genes are over expressed in high risk group and positive related to immune repressive cells and immune checkpoints, which indicate aggression and immunotherapy resistance. CONCLUSION: We identified a robust IRGPs signature to predict prognosis and immune heterogeneity in glioblastoma patients. Some potential targets and pathways need to be further researched to make different patients benefit from personalized immunotherapy.

Pediatric Spinal Cord Injury without Radiographic Abnormality: The Beijing Experience.

STUDY DESIGN: Retrospective study. OBJECTIVE: To describe the epidemiology and clinical outcomes of pediatric patients with spinal cord injury (SCI) without radiographic abnormality (SCIWORA) in mainland China for the first time. SUMMARY OF BACKGROUND DATA: SCIWORA is a syndrome that often occurs in children mainly due to the unique biomechanics of the pediatric spine. Although there have been numerous retrospective studies on pediatric SCIWORA, and mainland China has more patients with SCI than anywhere else, pediatric patients with SCIWORA in mainland China has not been described in any study. METHODS: Review of all cases with SCIWORA at Beijing Children's Hospital between July 2007 and December 2019. RESULTS: Of the 189 pediatric patients with SCI 140 had SCIWORA (age: 5.65 ±â€Š2.60 years, male-to-female ratio: 2:5). Main causes of injuries were sports (41%, mostly backbend), falls (27%), traffic accidents (10%), and violence (8%). Lesions were located at the thoracic (77%), cervical (10%), multiple (5%), and lumbar (4%) levels. Incubation period was 2 ±â€Š6 hours. Pathological characteristics of SCI were detected in 96% patients by magnetic resonance imaging (MRI). Based on the American Spinal Injury Association impairment scale (AIS), many patients had complete impairment (50% AIS A, 45% AIS B/C/D, 1% AIS E). Particularly, the five patients with normal MRI tended to have mild injury (AIS D) (P < 0.001), but they still showed abnormal reflex. In the one patient who could not be graded at all by AIS, his only functional deficits were abnormal upper and lower limb muscle tones. A total of 59% patients were treated with methylprednisolone, dexamethasone, or both. Out of 76 patients 59 showed neurological improvement before discharge. The only association among age, cause of injury, level of lesion, incubation period, AIS grade, type of corticosteroid therapy, and neurological improvement was between level of lesion and AIS grade (P < 0.001). CONCLUSION: Demographic and clinical differences exist in patients with SCIWORA. MRI and detailed neurological examinations should both be performed for proper diagnosis. There is still a need to develop better treatment strategy for these patients.Level of Evidence: 4.