1.Analysis of the safety of dinutuximab β for the treatment of neuroblastoma
Anle SHEN ; Yali HAN ; Liting YU ; An'an ZHANG ; Jie ZHAO ; Qiushi YANG ; Haonan LI ; Zhiling LI ; Yijin GAO
Journal of Chongqing Medical University 2025;50(8):1042-1046
Objective:To analyze the clinical characteristics of adverse reactions caused by dinutuximab β for the treatment of neuro-blastoma(NB)in China and to provide safety evidence for the rational use of dinutuximab β in clinical practice.Methods:Clinical data were retrospectively collected from 16 pediatric patients with NB who had been treated with dinutuximab β at Shanghai Children's Medical Center Affiliated to Shanghai Jiao Tong University School of Medicine from January 2022 to November 2023,and the adverse reactions caused by dinutuximab β were summarized and analyzed.Results:The male-to-female ratio was 5:3 among the 16 children with NB.The retroperitoneum was the main initial site of involvement,accounting for 75%.Thirteen(81.25%)patients had high-risk NB.The adverse reactions caused by dinutuximab β mainly included decreased hemoglobin,fever,vomiting,and diarrhea.The inci-dence of adverse reactions was highest in the first course of treatment,and the median time of adverse reactions was 2-5 days.Conclu-sion:Targeted monitoring should be carried out at an early stage during dinutuximab β administration.Adverse reactions should be de-tected and managed early to ensure the safety of medication for children.
2.Preliminary efficacy and safety analysis of modified SIOPEL-4 protocol for newly diagnosed pediatric hepatoblastoma with lung metastases
Jiaxin PENG ; Can HUANG ; An'an ZHANG ; Yali HAN ; Haishan RUAN ; Xiaoxia WANG ; Min XU ; Yuan XIN ; Liting YU ; Zhibao LYU ; Mingxuan FENG ; Shayi JIANG ; Yijin GAO
Chinese Journal of Pediatrics 2025;63(12):1343-1348
Objective:To assess the preliminary efficacy and safety of modified Société Internationale d′Oncologie Pédiatrique Epithelial Liver Tumor Study Group (SIOPEL)-4 protocol for pediatric hepatoblastoma (HB) with lung metastases.Methods:This prospective cohort study enrolled 27 newly diagnosed pediatric HB with lung metastases who received the modified SIOPEL-4 protocol at Shanghai Children′s Medical Center, Shanghai Jiao Tong University School of Medicine, and Shanghai Children′s Hospital between January 2020 to December 2023. Clinical characteristics, lung response rates to induction chemotherapy, treatment outcomes, prognostic factors and sever chemotherapy toxicities at different stages were analyzed. Survival analysis was performed by Kaplan-Meier method. Univariate prognostic analysis was conducted by Log-Rank test.Results:Of the 27 patients, there were 17 males and 10 females, with the age of 21 (15, 33) months. During the follow-up of 31 (12, 45) months for 17 continuous complete remission patients, 4 cases disease progression (2 cases death) and 6 cases relapse were observed. The 2-year event free survival (EFS) and overall survival (OS) rate was (58±11)% and (89±7)%, respectively. All the 27 patients had response to block 1-3 induction chemotherapy (cisplatin+doxorubicin), with 14 cases (52%) achieving complete response and 13 cases (48%) achieving partial response of lung metastatic lesions, the 2-year EFS rate was (81±12)% and (34±14)%, respectively ( χ 2=6.76, P=0.009), the 2-year OS rate was 100% and (79±13)%, respectively ( χ2=2.12, P=0.145). Patients with caudate lobe tumors or ≥10 pulmonary metastatic nodules had significantly lower EFS rates ( χ2=5.36, 7.84, P=0.021, 0.005, respectively). The incidence of grade 3/4 neutropenia after block 1-3 induction chemotherapy, CD (carboplatin+doxorubicin), and VI (vincristine+irinotecan) consolidation chemotherapy was 90% (73/81), 75% (58/77), and 31% (11/35), respectively. The incidence of grade 3/4 thrombocytopenia was 77% (62/81), 69% (53/77), and 14% (5/35), respectively. The incidence of grade 3/4 infections was 64% (52/81), 25% (19/77), and 20% (7/35), respectively. The differences between the groups were statistically significant ( χ2=43.51, 42.69, 33.00, all P<0.001). Two patients (10%) of the 20 evaluable patients for ototoxicity occurred grade 3 and higher hearing impairment, with 1 patient requiring a hearing aid. Conclusions:The modified SIOPEL-4 regimen shows good preliminary efficacy and safety in treating pediatric HB with lung metastases. The prognosis for patients with residual lesions in the lungs after induction chemotherapy needs to be improved. Attention should be given to the ototoxicity induced by high-dose cisplatin chemotherapy.
3.Progress on clinical effects and underlying mechanisms of catecholamines in neuroblastoma
Yinfeng ZHANG ; Qian LI ; Yijin GAO
International Journal of Pediatrics 2025;52(9):588-592
Neuroblastoma is a common extracranial solid tumors in children,and patients with high-risk disease have poor prognosis. Since the pathogenesis of neuroblastoma has not been fully elucidated,early diagnosis and timely,effective clinical intervention are key strategies to improve survival in affected children. As a neuroendocrine tumor,neuroblastoma is characterized by abnormal secretion of catecholamine compounds,the synthesis and release of which are closely associated with both diagnosis and prognosis. This review summarizes the biosynthetic and metabolic pathways of catecholamines and their clinical applications in neuroblastoma,and further discusses the molecular mechanisms by which catecholamines contribute to neuroblastoma progression.
4.Advances in novel therapies for children with Hodgkin lymphoma
International Journal of Pediatrics 2025;52(10):676-680
Hodgkin lymphoma(HL)is one of the common pediatric malignancies(especially in adolescents). The prognosis of pediatric patients with HL is excellent with traditional first-line treatment regimens involving short-course,high-dose combination chemotherapy and low-intensity radiotherapy,resulting in an overall response rate of 90%. However,10%-25% of pediatric HL patients are estimated to have relapsed/refractory(r/r)disease,and survivors of pediatric HL are at risk of late effects caused by traditional treatment regimens. For children with r/r HL,novel therapies,such as brentuximab vedotin,immune checkpoint inhibitors and immune cell therapy,including chimeric antigen receptor T-cell therapy and EBV-specific cytotoxic T lymphocytes are an integral part of their salvage treatment and consolidation treatment after hematopoietic stem cell transplantation. Furthermore,combining novel therapies with first-line treatment regimens for pediatric HL may help enhance therapeutic efficacy and reduce treatment toxicity. This review is aimed to describe current advances in novel therapies for pediatric HL,and serve as a reference for clinical practice.
5.Novel autosomal dominant syndromic hearing loss caused by COL4A2 -related basement membrane dysfunction of cochlear capillaries and microcirculation disturbance.
Jinyuan YANG ; Ying MA ; Xue GAO ; Shiwei QIU ; Xiaoge LI ; Weihao ZHAO ; Yijin CHEN ; Guojie DONG ; Rongfeng LIN ; Gege WEI ; Huiyi NIE ; Haifeng FENG ; Xiaoning GU ; Bo GAO ; Pu DAI ; Yongyi YUAN
Chinese Medical Journal 2025;138(15):1888-1890
6.Clinical and molecular pathological features of 17 cases of gastrointestinal leiomyo-mas with scattered expression of CD117 and DOG1
Wenjing GAO ; Zhihan ZHANG ; Haimin XU ; Yijin GU
Chinese Journal of Clinical and Experimental Pathology 2025;41(10):1318-1323
Purpose To investigate the clinical features of gastrointestinal leiomyomas(GLs)with scattered ex-pression of CD117 and DOG1,and to evaluate their biological behavior as well as their different diagnositic value from gastrointestinal stromal tumors(GISTs).Methods Clinical data from 17 cases of surgically resected GLs were col-lected.Immunohistochemistry using the EnVision method was performed to detect SMA,desmin,h-caldesmon,DOG1,CD117,and CD34.First-generation sequencing was performed to analyze exons 9-20 of the KIT gene and ex-ons 12 and 18 of the PDGFRA gene.Results Tumors occured in the stomach(8 cases)and esophagus(9 cases).Clinical manifestations included dull upper abdominal pain,dysphagia,chest pain,and fever.During a follow-up peri-od of 58-88 months,no recurrence was observed,and all patients had a favorable prognosis.Histologically,tumor cells were spindle-shaped and arranged in bundles or a woven pattern.Interstitial cells of Cajal appeared spindle-shaped or stellate with indistinct borders and dispersed chromatin.Tumor cells showed diffuse positivity for SMA,desmin,and h-caldesmon(100%).Interstitial cells of Cajal exhibited focal positive for CD117 and DOG1,with an o-verall positive rate of 9%for each marker.No pathogenic mutations of the KIT or PDGFRA genes were detected by first-generation sequencing.Conclusion Although some GLs contained interstitial cells of Cajal that showed focal pos-itivity for CD117 and DOG1,sequencing and long-term follow-up confirmed that their biological behavior differed from that of GISTs,with no malignant potential.Surgical resection remained the mainstay of treatment,and the prognosis was favorable.
7.Summary of the best evidence for cognitive function improvement in Alzheimer's disease patients
Yijin WANG ; Yanxuan ZHANG ; Ruihan LI ; Li QI ; Weixin ZHANG ; Yin GAO
Chinese Journal of Modern Nursing 2025;31(30):4152-4158
Objective:To retrieve and summarize the best evidence for cognitive function improvement in Alzheimer's disease patients, so as to provide reference for clinical practice.Methods:Following the "6S" evidence model, guidelines, expert consensus, clinical decision, and systematic reviews on cognitive function improvement in Alzheimer's disease patients were systematically retrieved from domestic and foreign databases and websites. The search keywords included "Alzheimer's disease, cognitive function, improvement". Evidence-based nursing methods were used to evaluate the quality of literature and extract evidence. The search period was from July 1, 2000 to July 31, 2024.Results:A total of 15 articles were included, including five guidelines, four expert consensus, two clinical decision, and four systematic reviews. Sixteen pieces of best evidence were summarized from four aspects of evaluation and identification, pharmacological intervention, non-pharmacological intervention, and caregiver support.Conclusions:Patients with Alzheimer's disease require timely identification of symptoms related to cognitive impairment and assessment using standardized scales. Multiple intervention methods, such as pharmacological intervention, non-pharmacological intervention, and caregiver support, should be used to enhance patients' quality of life. Evidence application should be tailored to each patient's specific circumstances through individualized selection and adjustment to ensure the effectiveness and scientific rigor of cognitive function improvement strategies, thereby facilitating the translation of optimal evidence into clinical practice.
8.Preliminary efficacy and safety analysis of modified SIOPEL-4 protocol for newly diagnosed pediatric hepatoblastoma with lung metastases
Jiaxin PENG ; Can HUANG ; An'an ZHANG ; Yali HAN ; Haishan RUAN ; Xiaoxia WANG ; Min XU ; Yuan XIN ; Liting YU ; Zhibao LYU ; Mingxuan FENG ; Shayi JIANG ; Yijin GAO
Chinese Journal of Pediatrics 2025;63(12):1343-1348
Objective:To assess the preliminary efficacy and safety of modified Société Internationale d′Oncologie Pédiatrique Epithelial Liver Tumor Study Group (SIOPEL)-4 protocol for pediatric hepatoblastoma (HB) with lung metastases.Methods:This prospective cohort study enrolled 27 newly diagnosed pediatric HB with lung metastases who received the modified SIOPEL-4 protocol at Shanghai Children′s Medical Center, Shanghai Jiao Tong University School of Medicine, and Shanghai Children′s Hospital between January 2020 to December 2023. Clinical characteristics, lung response rates to induction chemotherapy, treatment outcomes, prognostic factors and sever chemotherapy toxicities at different stages were analyzed. Survival analysis was performed by Kaplan-Meier method. Univariate prognostic analysis was conducted by Log-Rank test.Results:Of the 27 patients, there were 17 males and 10 females, with the age of 21 (15, 33) months. During the follow-up of 31 (12, 45) months for 17 continuous complete remission patients, 4 cases disease progression (2 cases death) and 6 cases relapse were observed. The 2-year event free survival (EFS) and overall survival (OS) rate was (58±11)% and (89±7)%, respectively. All the 27 patients had response to block 1-3 induction chemotherapy (cisplatin+doxorubicin), with 14 cases (52%) achieving complete response and 13 cases (48%) achieving partial response of lung metastatic lesions, the 2-year EFS rate was (81±12)% and (34±14)%, respectively ( χ 2=6.76, P=0.009), the 2-year OS rate was 100% and (79±13)%, respectively ( χ2=2.12, P=0.145). Patients with caudate lobe tumors or ≥10 pulmonary metastatic nodules had significantly lower EFS rates ( χ2=5.36, 7.84, P=0.021, 0.005, respectively). The incidence of grade 3/4 neutropenia after block 1-3 induction chemotherapy, CD (carboplatin+doxorubicin), and VI (vincristine+irinotecan) consolidation chemotherapy was 90% (73/81), 75% (58/77), and 31% (11/35), respectively. The incidence of grade 3/4 thrombocytopenia was 77% (62/81), 69% (53/77), and 14% (5/35), respectively. The incidence of grade 3/4 infections was 64% (52/81), 25% (19/77), and 20% (7/35), respectively. The differences between the groups were statistically significant ( χ2=43.51, 42.69, 33.00, all P<0.001). Two patients (10%) of the 20 evaluable patients for ototoxicity occurred grade 3 and higher hearing impairment, with 1 patient requiring a hearing aid. Conclusions:The modified SIOPEL-4 regimen shows good preliminary efficacy and safety in treating pediatric HB with lung metastases. The prognosis for patients with residual lesions in the lungs after induction chemotherapy needs to be improved. Attention should be given to the ototoxicity induced by high-dose cisplatin chemotherapy.
9.Correlations of early brain microstructural changes and cognitive function in patients with alcohol use disorder
Yijin YAN ; Chenyang WANG ; Yan GAO ; Yanchi LIU ; Jiawen DONG ; Jie LI ; Hongzeng ZHAO
Chinese Journal of Medical Imaging Technology 2025;41(2):224-228
Objective To explore the early brain microstructural changes in patients with alcohol use disorder(AUD)and the correlations with cognitive function.Methods Totally 81 AUD patients(AUD group)and 75 healthy volunteers(control group)were retrospectively enrolled.Gray matter volume(GMV)and cortical thickness of brain regions were calculated with voxel-based morphometry and surface-based morphometry,and the correlations of the above indexes of brain regions being significantly different between groups and clinical cognitive function scale scores were analyzed.Results In AUD group,GMV of bilateral hippocampus,insula,inferior parietal angular gyrus,straight gyrus,central posterior gyrus and cerebellar angle 1 area,of left medial superior frontal gyrus,precuneus,anterior cingulated cortex,middle temporal gyrus and middle occipital gyrus,as well as of right lingual gyrus reduced significantly compared with those in control group.The cortical thickness of bilateral superior frontal gyrus,middle frontal gyrus,inferior frontal gyrus,supramarginal gyrus,superior parietal lobule,inferior parietal lobule,central posterior gyrus,central anterior gyrus,temporal superior gyrus and middle temporal gyrus reduced in AUD group.In AUD group,GMV of left medial superior frontal gyrus was positively correlated with Pittsburgh sleep quality index score(r=0.301,P=0.006),while of the left and right straight gyrus were positively correlated with Hopkins verbal learning test-revised score(r=0.328,0.326;both P=0.003)but negatively correlated with trail making test-B score(r=-0.295,-0.312;P=0.008,0.005),while the cortical thickness of the left central anterior gyrus and the lower part of the right middle frontal gyrus were positively correlated with Beck anxiety inventory score(r=0.323,0.289;P=0.003,0.009).Conclusion Prefrontal cortex atrophy could be observed in early stage AUD patients,which was correlated with cognitive function impaired.
10.Summary of the best evidence for cognitive function improvement in Alzheimer's disease patients
Yijin WANG ; Yanxuan ZHANG ; Ruihan LI ; Li QI ; Weixin ZHANG ; Yin GAO
Chinese Journal of Modern Nursing 2025;31(30):4152-4158
Objective:To retrieve and summarize the best evidence for cognitive function improvement in Alzheimer's disease patients, so as to provide reference for clinical practice.Methods:Following the "6S" evidence model, guidelines, expert consensus, clinical decision, and systematic reviews on cognitive function improvement in Alzheimer's disease patients were systematically retrieved from domestic and foreign databases and websites. The search keywords included "Alzheimer's disease, cognitive function, improvement". Evidence-based nursing methods were used to evaluate the quality of literature and extract evidence. The search period was from July 1, 2000 to July 31, 2024.Results:A total of 15 articles were included, including five guidelines, four expert consensus, two clinical decision, and four systematic reviews. Sixteen pieces of best evidence were summarized from four aspects of evaluation and identification, pharmacological intervention, non-pharmacological intervention, and caregiver support.Conclusions:Patients with Alzheimer's disease require timely identification of symptoms related to cognitive impairment and assessment using standardized scales. Multiple intervention methods, such as pharmacological intervention, non-pharmacological intervention, and caregiver support, should be used to enhance patients' quality of life. Evidence application should be tailored to each patient's specific circumstances through individualized selection and adjustment to ensure the effectiveness and scientific rigor of cognitive function improvement strategies, thereby facilitating the translation of optimal evidence into clinical practice.

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