1.Clinical and Neuroelectrophysiological Features of Autoimmune Nodopathy
Hongfei TAI ; Xunyan HUANG ; Songtao NIU ; Bin CHEN ; Yuzhi SHI ; Xingao WANG ; Fan JIAN ; Hua PAN ; Zaiqiang ZHANG
JOURNAL OF RARE DISEASES 2026;5(2):191-199
To summarize the clinical characteristics, antibody spectrum and neuroelectrophysiological features of autoimmune nodopathy(AN), and to explore the phenotypic differences among different antibody-positive subgroups. The clinical and electrophysiological data of patients definitely diagnosed with AN in Beijing Tiantan Hospital, Capital Medical University, from October 2018 to January 2026 were retrospectively analyzed. A total of 33 patients with AN were included. Antibody examination results showed that, anti-neurofascin(NF)155 antibody was the most prevalent, detected in 17 patients(51.50%), followed by anti-contactin-1(CNTN1) antibody in 8 patients(24.24%). Anti-NF186 antibody(4 cases, 12.12%), anti-contactin-associated protein 1(Caspr1) antibody(2 cases, 6.06%) and dual-target antibody positivity(2 cases, 6.06%) were relatively uncommon. The main clinical manifestations of AN patients included symmetric distal paresthesia of the extremities(32 cases, 96.97%), limb weakness(31 cases, 93.93%) and sensory ataxia(25 cases, 75.76%). Different antibody-positive subgroups presented distinct phenotypic features: patients with positive anti-NF155 antibody had a relatively younger age of onset, chronic onset and a high incidence of tremor, which was dominated by immunoglobulin(Ig)G4 subclass antibodies; patients with positive anti-CNTN1 antibody had a relatively advanced age of onset, mostly presented with acute or subacute onset, and were prone to complicated nephrotic syndrome; patients with positive anti-NF186 antibody had relatively mild nerve conduction damage; patients with anti-Caspr1 antibody manifested acute or subacute onset, with relatively elevated cerebrospinal fluid protein level and 24-h intrathecal IgG synthesis rate. The prominent neuroelectrophysiological manifestations of AN included decreased motor and sensory nerve conduction velocities, prolonged distal latency, frequent non-compressive conduction block and abnormal temporal dispersion. Definite sensory nerve action potentials could not be elicited in more than half of the patients. Patients with AN show high heterogeneity in clinical and neuroelectrophysiological characteristics, and different antibody-positive subgroups correspond to specific clinical and neuroelectrophysiological phenotypes.
2.Effects of High Temperature on Antioxidant,Carbon Metabolism Physiological Indicators and Transcriptome of Mytilus coruscus
Zhi YANG ; Yuan-Yue ZHANG ; Jian-Xin WANG ; Zhi LIAO ; Qun-Ju HU ; Qiao-Mei YANG ; Xiao-Lin ZHANG ; Mei-Hua FAN
Chinese Journal of Biochemistry and Molecular Biology 2025;41(3):446-459
The ocean plays a critical role in the global carbon cycle,and base on the"dual carbon"goals,ocean carbon sinks have received widespread attention.Shellfish aquaculture is one of the most important sources of carbon sinks in fisheries,which has an important impact on the offshore carbon cy-cle.As global temperature rises and ocean acidification intensifies,the capacity of the ocean to absorb CO2 will change.However,the effects of high temperature on the physiology and transcriptome related to carbon metabolism in Mytilus coruscus are not clear enough.This study investigated the effects of high temperatures on the total carbon content,carbon metabolism,antioxidant-related enzyme activities,and the transcriptome of Mytilus coruscus.The results showed that high temperature significantly inhibited the activities of hexokinase and pyruvate kinase,increased carbonic anhydrase activity(P<0.05),de-creased the ATP content of digestive glands(P<0.05),and affected glycolysis and the tricarboxylic acid cycle,leading to a significant decrease in the mussel's ability to sequester carbon.High temperature re-sulted in significant(P<0.05)increases in the levels of reactive oxygen species and malondialdehyde,and enhanced the activities of superoxide dismutase and catalase.Observations by transmission electron microscopy showed that high temperatures damaged the subcellular structure of the digestive gland in Mytilus coruscus,resulting in the shrinkage of the nucleolus,swelling of the endoplasmic reticulum,and a significant reduction in the mitochondrial cristae.Comparative transcriptomic analysis showed that the upregulated DEGs were mainly enriched in protein processing in the endoplasmic reticulum,antigen pro-cessing and presentation,and MAPK signaling pathway.The downregulated DEGs were mainly enriched in necroptosis,DNA replication,and the NF-kappa B signaling pathway.In antioxidant-related DEGs,the upregulated DEGs include vitamin K epoxide reductase,peroxidases,heat shock protein 105 kD,heat shock protein 70 kD,and superoxide dismutase;The downregulated DEGs mainly included NADPH oxidase,glutathione reductase,cytochrome b-245,cytochrome P450,and quinone reductase.The up-regulated genes enriched in the carbon metabolism pathway included chitinase,phosphatidylinositol 4,5-bisphosphate 3-kinase,phosphoenolpyruvate carboxykinase,galactokinase,and inositol trisphosphate 3-kinase.The downregulated genes included aldose-1-epimerase,carbonic anhydrase,galactose mutaro-tase,acyl-CoA synthetase,alcohol dehydrogenase,and hexokinase.In conclusion,high temperature has an inhibitory effect on the activities of enzymes and the expression of genes related to carbon metabolism in Mytilus coruscus.The results of this study are intended to provide a scientific basis for the healthy de-velopment of mussel aquaculture and the assessment of carbon sinks.
3.The value of total volume response and total mass response in the therapeutic evaluation of lung metastasis of hepatocarcinoma
Jun-cheng WAN ; Cai-hong YU ; Chang-yu LI ; Yong-jie ZHOU ; Wei ZHANG ; Jian-hua WANG ; Zhi-ping YAN ; Guo-wei YANG ; Zhuo-yang FAN ; Xu-dong QU
Fudan University Journal of Medical Sciences 2025;52(2):201-208,231
Objective To analyze the correlation between lesion volume,lesion mass,and maximum lesion diameter in the assessment of advanced hepatocarcinoma with lung metastasis,and to evaluate the application value of total volume response and total mass response of lung metastatic lesions in efficacy assessment.Methods A retrospective analysis was conducted on the CT imaging data of 20 patients clinically confirmed with hepatocarcinoma and lung metastases,followed by subsequent follow-up to monitor their survival outcomes.Volume measurement software was used to measure the volume of lesions before and after treatment.We recored lesion diameter,volume measurements and CT values,calculated the mass of the lesions.The correlation between lesion volume,mass and diameter was analyzed,as well as the correlation between the change rates of volume,mass and lesion diameter.Additionally,the total volume and total mass of all lesions were calculated.The correlation between the change rates of total volume/total mass and the change rate of pulmonary lesion diameter under the RECIST 1.1 criteria,as well as the correlation with changes in patients'tumor markers,were analyzed.Furthermore,the overall volume response and overall mass response of lesions were evaluated based on changes in total volume and total mass,and their consistencies with the RECIST 1.1 criteria for efficacy evaluation were analyzed.Finally,univariate Cox regression analysis was performed to explore the association between these variables and patient survival outcomes.Results There was strong correlation between lesion volume,mass and tumor diameter(r=0.771,0.775),between the rate of change in mass and the rate of change in lesion diameter(r=0.846),and between the rates of change in total volume/total mass and the rate of change in pulmonary lesion diameter under the RECIST 1.1 criteria(r=0.800,0.896).The correlation between the rates of change in total volume/total mass and patients'tumor markers was not statistically significant.There was moderate correlation between the rate of change in volume and the rate of change in lesion diameter(r=0.692).The evaluation results of total volume response and total mass response for pulmonary lesions in advanced hepatocarcinoma with lung metastasis were generally consistent with the RECIST 1.1 criteria(Kappa=0.486,0.426).Univariate Cox regression analysis revealed that total lesion volume(P=0.047)and total lesion mass(P=0.049)were independent prognostic factors for survival outcomes.Conclusion Lesion volume,mass,and diameter,as well as their respective change rates,were found to be interrelated.Furthermore,total lesion volume and total lesion mass were identified as independent prognostic factors for survival outcomes.The total volume response and total mass response are promising evaluation methods in evaluating the efficacy of lung metastasis of hepatocarcinoma,which are different from the RECIST 1.1 evaluation criteria.
4.The value of total volume response and total mass response in the therapeutic evaluation of lung metastasis of hepatocarcinoma
Jun-cheng WAN ; Cai-hong YU ; Chang-yu LI ; Yong-jie ZHOU ; Wei ZHANG ; Jian-hua WANG ; Zhi-ping YAN ; Guo-wei YANG ; Zhuo-yang FAN ; Xu-dong QU
Fudan University Journal of Medical Sciences 2025;52(2):201-208,231
Objective To analyze the correlation between lesion volume,lesion mass,and maximum lesion diameter in the assessment of advanced hepatocarcinoma with lung metastasis,and to evaluate the application value of total volume response and total mass response of lung metastatic lesions in efficacy assessment.Methods A retrospective analysis was conducted on the CT imaging data of 20 patients clinically confirmed with hepatocarcinoma and lung metastases,followed by subsequent follow-up to monitor their survival outcomes.Volume measurement software was used to measure the volume of lesions before and after treatment.We recored lesion diameter,volume measurements and CT values,calculated the mass of the lesions.The correlation between lesion volume,mass and diameter was analyzed,as well as the correlation between the change rates of volume,mass and lesion diameter.Additionally,the total volume and total mass of all lesions were calculated.The correlation between the change rates of total volume/total mass and the change rate of pulmonary lesion diameter under the RECIST 1.1 criteria,as well as the correlation with changes in patients'tumor markers,were analyzed.Furthermore,the overall volume response and overall mass response of lesions were evaluated based on changes in total volume and total mass,and their consistencies with the RECIST 1.1 criteria for efficacy evaluation were analyzed.Finally,univariate Cox regression analysis was performed to explore the association between these variables and patient survival outcomes.Results There was strong correlation between lesion volume,mass and tumor diameter(r=0.771,0.775),between the rate of change in mass and the rate of change in lesion diameter(r=0.846),and between the rates of change in total volume/total mass and the rate of change in pulmonary lesion diameter under the RECIST 1.1 criteria(r=0.800,0.896).The correlation between the rates of change in total volume/total mass and patients'tumor markers was not statistically significant.There was moderate correlation between the rate of change in volume and the rate of change in lesion diameter(r=0.692).The evaluation results of total volume response and total mass response for pulmonary lesions in advanced hepatocarcinoma with lung metastasis were generally consistent with the RECIST 1.1 criteria(Kappa=0.486,0.426).Univariate Cox regression analysis revealed that total lesion volume(P=0.047)and total lesion mass(P=0.049)were independent prognostic factors for survival outcomes.Conclusion Lesion volume,mass,and diameter,as well as their respective change rates,were found to be interrelated.Furthermore,total lesion volume and total lesion mass were identified as independent prognostic factors for survival outcomes.The total volume response and total mass response are promising evaluation methods in evaluating the efficacy of lung metastasis of hepatocarcinoma,which are different from the RECIST 1.1 evaluation criteria.
5.Effects of curcumin derivative C0818 on NSCLC cells and its mechanisms
Ying-juan FAN ; Yang LIU ; Jian-hua XU
Chinese Pharmacological Bulletin 2025;41(12):2260-2268
Aim To study the effects of curcumin de-rivative C0818 on the migration,apoptosis and prolifer-ation of non-small cell lung cancer(NSCLC)cells.Methods MTT assay and CFSE were used to detect the inhibitory effect of C0818 on the proliferation of NSCLC cells NCI-H1299 and NCI-H460 in vitro.Flow cytometry was used to detect mitochondrial reactive ox-ygen species(ROS),the mitochondrial membrane po-tential(MMP),cell apoptosis,and the cell cycle.The Transwell chamber assay and scratch wound healing as-say were used to detect the effect of C0818 on the mi-gration ability of NSCLC cells.Western blot was used to detect the expression of apoptosis and migration-re-lated proteins.Results C0818 inhibited the prolifera-tion of NCI-H1299 and NCI-H460 cells in a time-and dose-dependent manner,and arrested the cell cycle at the G2/M phase.C0818 disrupted the MMP.With the increase of the concentration of C0818(0.4-1.6μmol·L-1),the apoptosis rate of cells increased sig-nificantly,accompanied by the up-regulation of pro-ap-optotic proteins Bax,cleaved PARP,and cleaved caspase-3/8,and the down-regulation of anti-apoptotic proteins Bcl-2.C0818 inhibited the migration ability of NSCLC cells and could inhibit the expression of pro-teins related to invasion and metastasis,such as N-cad-herin,β-catenin,and Vimentin.Conclusions The curcumin derivative C0818 induces caspase-mediated and ROS-dependent apoptosis,inhibits cell prolifera-tion,and has a strong anti-metastatic ability in vitro.
6.Effects of High Temperature on Antioxidant,Carbon Metabolism Physiological Indicators and Transcriptome of Mytilus coruscus
Zhi YANG ; Yuan-Yue ZHANG ; Jian-Xin WANG ; Zhi LIAO ; Qun-Ju HU ; Qiao-Mei YANG ; Xiao-Lin ZHANG ; Mei-Hua FAN
Chinese Journal of Biochemistry and Molecular Biology 2025;41(3):446-459
The ocean plays a critical role in the global carbon cycle,and base on the"dual carbon"goals,ocean carbon sinks have received widespread attention.Shellfish aquaculture is one of the most important sources of carbon sinks in fisheries,which has an important impact on the offshore carbon cy-cle.As global temperature rises and ocean acidification intensifies,the capacity of the ocean to absorb CO2 will change.However,the effects of high temperature on the physiology and transcriptome related to carbon metabolism in Mytilus coruscus are not clear enough.This study investigated the effects of high temperatures on the total carbon content,carbon metabolism,antioxidant-related enzyme activities,and the transcriptome of Mytilus coruscus.The results showed that high temperature significantly inhibited the activities of hexokinase and pyruvate kinase,increased carbonic anhydrase activity(P<0.05),de-creased the ATP content of digestive glands(P<0.05),and affected glycolysis and the tricarboxylic acid cycle,leading to a significant decrease in the mussel's ability to sequester carbon.High temperature re-sulted in significant(P<0.05)increases in the levels of reactive oxygen species and malondialdehyde,and enhanced the activities of superoxide dismutase and catalase.Observations by transmission electron microscopy showed that high temperatures damaged the subcellular structure of the digestive gland in Mytilus coruscus,resulting in the shrinkage of the nucleolus,swelling of the endoplasmic reticulum,and a significant reduction in the mitochondrial cristae.Comparative transcriptomic analysis showed that the upregulated DEGs were mainly enriched in protein processing in the endoplasmic reticulum,antigen pro-cessing and presentation,and MAPK signaling pathway.The downregulated DEGs were mainly enriched in necroptosis,DNA replication,and the NF-kappa B signaling pathway.In antioxidant-related DEGs,the upregulated DEGs include vitamin K epoxide reductase,peroxidases,heat shock protein 105 kD,heat shock protein 70 kD,and superoxide dismutase;The downregulated DEGs mainly included NADPH oxidase,glutathione reductase,cytochrome b-245,cytochrome P450,and quinone reductase.The up-regulated genes enriched in the carbon metabolism pathway included chitinase,phosphatidylinositol 4,5-bisphosphate 3-kinase,phosphoenolpyruvate carboxykinase,galactokinase,and inositol trisphosphate 3-kinase.The downregulated genes included aldose-1-epimerase,carbonic anhydrase,galactose mutaro-tase,acyl-CoA synthetase,alcohol dehydrogenase,and hexokinase.In conclusion,high temperature has an inhibitory effect on the activities of enzymes and the expression of genes related to carbon metabolism in Mytilus coruscus.The results of this study are intended to provide a scientific basis for the healthy de-velopment of mussel aquaculture and the assessment of carbon sinks.
7.Effects of curcumin derivative C0818 on NSCLC cells and its mechanisms
Ying-juan FAN ; Yang LIU ; Jian-hua XU
Chinese Pharmacological Bulletin 2025;41(12):2260-2268
Aim To study the effects of curcumin de-rivative C0818 on the migration,apoptosis and prolifer-ation of non-small cell lung cancer(NSCLC)cells.Methods MTT assay and CFSE were used to detect the inhibitory effect of C0818 on the proliferation of NSCLC cells NCI-H1299 and NCI-H460 in vitro.Flow cytometry was used to detect mitochondrial reactive ox-ygen species(ROS),the mitochondrial membrane po-tential(MMP),cell apoptosis,and the cell cycle.The Transwell chamber assay and scratch wound healing as-say were used to detect the effect of C0818 on the mi-gration ability of NSCLC cells.Western blot was used to detect the expression of apoptosis and migration-re-lated proteins.Results C0818 inhibited the prolifera-tion of NCI-H1299 and NCI-H460 cells in a time-and dose-dependent manner,and arrested the cell cycle at the G2/M phase.C0818 disrupted the MMP.With the increase of the concentration of C0818(0.4-1.6μmol·L-1),the apoptosis rate of cells increased sig-nificantly,accompanied by the up-regulation of pro-ap-optotic proteins Bax,cleaved PARP,and cleaved caspase-3/8,and the down-regulation of anti-apoptotic proteins Bcl-2.C0818 inhibited the migration ability of NSCLC cells and could inhibit the expression of pro-teins related to invasion and metastasis,such as N-cad-herin,β-catenin,and Vimentin.Conclusions The curcumin derivative C0818 induces caspase-mediated and ROS-dependent apoptosis,inhibits cell prolifera-tion,and has a strong anti-metastatic ability in vitro.
8.Feasibility study on shortening the detection time of long exercise test in the diagnosis of periodic paralysis
Shuo YANG ; Na CHEN ; Lin CHEN ; Feng CHENG ; Jingfen LI ; Lei ZHANG ; Ying WANG ; Fan JIAN ; Zaiqiang ZHANG ; Hua PAN
Chinese Journal of Neurology 2025;58(4):359-365
Objective:To explore the feasibility of shortening the time of long exercise test (LET) from 120 to 60 minutes by analyzing the positive rate within 60 minutes among periodic paralysis (PP) patients who were positive in 120-minute test.Methods:The data of patients undergoing 120-minute LET from January 2015 to October 2021 in Beijing Tiantan Hospital, Capital Medical University were retrospectively analyzed, with 30%, 33%, and 40% as diagnostic cut-off values, respectively. PP patients with positive results within 120 minutes after exercise were enrolled in the study. The positive rate within 30 minutes and 60 minutes after exercise was calculated. The change rates of compound muscle action potential (CMAP) amplitude and the sensitivity and specificity of LET at 30 minutes, 60 minutes, and 120 minutes after exercise were analyzed. The change rate of CMAP amplitude in PP patients who did not show positive results within 60 minutes was further calculated.Results:A total of 254 patients were examined, including 114 PP patients. With 30%, 33%, and 40% as diagnostic cut-off values, the results showed that there were 88, 88, and 82 positive PP patients, respectively. Under each diagnostic cut-off values, the age of positive PP patients was (32±10) years, with a male proportion of 98% (86/88), 98% (86/88), and 99% (81/82), respectively; the positive rate of PP patients within 30 minutes after exercise was 60% (53/88), 58% (51/88), and 41% (34/82), respectively; the positive rate of PP patients within 60 minutes after exercise was 91% (80/88), 86% (76/88), and 83% (68/82), respectively. At the cut-off values of 30%, 33% and 40%, the change rate of CMAP amplitude at 30 minutes [-36% (-49%, -23%), -36% (-49%, -23%), -37% (-51%, -24%)], 60 minutes [-51% (-66%, -40%), -51% (-66%, -40%), -53% (-66%, -42%)] and 120 minutes [-57% (-67%, -45%), -57% (-67%, -45%), -58% (-67%, -46%)] after exercise showed statistically significant difference among 3 time points ( H=57.764, 57.764, 59.616, respectively, all P<0.001); the further comparison between time points showed that there was statistically significant difference in the change rate of CMAP amplitude between 60 minutes ( Z=5.419, 5.419, 5.531, respectively, all P<0.001), 120 minutes ( Z=7.325, 7.325, 7.431, respectively, all P<0.001) and 30 minutes after exercise, but there was no statistically significant difference in the change rate of CMAP amplitude between 120 minutes and 60 minutes after exercise ( Z=1.906, 1.906, 1.899, respectively, all P>0.05); the sensitivity of LET for the diagnosis of PP at 60 minutes after exercise was 70.2% (80/114), 66.7% (76/114) and 59.6% (68/114), and the specificity of LET for the diagnosis of PP was 77.9% (109/140), 84.3% (118/140) and 91.4%(128/140), respectively. When 30%, 33% and 40% were used as the diagnostic cut-off values, and the change rate of CMAP amplitude at 60 minutes after exercise fell below these cut-off values but showed a decline of ≥20%, ≥22% and ≥24%, respectively, the detection time should be extended to 120 minutes. Conclusions:Whether using 30%, 33%, or 40% as diagnostic cut-off values, it is feasible to shorten the LET time from 120 minutes to 60 minutes. The 60-minute LET has good sensitivity and specificity for the diagnosis of PP. It is recommended to extend the detection time to 120 minutes for patients with a ≥20%, ≥22%, or ≥24% decline in CMAP amplitude at 60 minutes after exercise while falling short of corresponding diagnostic cut-off values when 30%, 33%, and 40% are used as diagnostic cut-off values. This method can not only improve the examination efficiency of LET, but also minimize the missed diagnosis as much as possible.
9.Expert consensus on evaluation index system construction for new traditional Chinese medicine(TCM) from TCM clinical practice in medical institutions.
Li LIU ; Lei ZHANG ; Wei-An YUAN ; Zhong-Qi YANG ; Jun-Hua ZHANG ; Bao-He WANG ; Si-Yuan HU ; Zu-Guang YE ; Ling HAN ; Yue-Hua ZHOU ; Zi-Feng YANG ; Rui GAO ; Ming YANG ; Ting WANG ; Jie-Lai XIA ; Shi-Shan YU ; Xiao-Hui FAN ; Hua HUA ; Jia HE ; Yin LU ; Zhong WANG ; Jin-Hui DOU ; Geng LI ; Yu DONG ; Hao YU ; Li-Ping QU ; Jian-Yuan TANG
China Journal of Chinese Materia Medica 2025;50(12):3474-3482
Medical institutions, with their clinical practice foundation and abundant human use experience data, have become important carriers for the inheritance and innovation of traditional Chinese medicine(TCM) and the "cradles" of the preparation of new TCM. To effectively promote the transformation of new TCM originating from the TCM clinical practice in medical institutions and establish an effective evaluation index system for the transformation of new TCM conforming to the characteristics of TCM, consensus experts adopted the literature research, questionnaire survey, Delphi method, etc. By focusing on the policy and technical evaluation of new TCM originating from the TCM clinical practice in medical institutions, a comprehensive evaluation from the dimensions of drug safety, efficacy, feasibility, and characteristic advantages was conducted, thus forming a comprehensive evaluation system with four primary indicators and 37 secondary indicators. The expert consensus reached aims to encourage medical institutions at all levels to continuously improve the high-quality research and development and transformation of new TCM originating from the TCM clinical practice in medical institutions and targeted at clinical needs, so as to provide a decision-making basis for the preparation, selection, cultivation, and transformation of new TCM for medical institutions, improve the development efficiency of new TCM, and precisely respond to the public medication needs.
Medicine, Chinese Traditional/standards*
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Humans
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Consensus
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Drugs, Chinese Herbal/therapeutic use*
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Surveys and Questionnaires
10.The efficacy of blinatumomab in the treatment of pediatric B-cell acute lymphoblastic leukemia: a multicenter study
Weiling YAN ; Jun LU ; Hua WANG ; Lihua YU ; Huidi FENG ; Bai LI ; Wenguang JIA ; Jian WANG ; Wenting HU ; Xue TANG ; Jing FAN ; Yujie GUAN ; Xiaolan LI ; Yalan YOU ; Yongmin TANG ; Xiaojun XU
Chinese Journal of Pediatrics 2025;63(11):1194-1200
Objective:To investigate the efficacy and toxicity of blinatumomab in the first-line and second-line treatment of pediatric B-cell acute lymphoblastic leukemia (B-ALL).Methods:A multi-center retrospective cohort study was conducted to analyze clinical data from 323 pediatric B-ALL patients treated with blinatumomab across 14 hospitals in China from May 2021 to July 2023. Patients were divided into four groups based on the treatment phase and disease status when blinatumomab was used: relapsed/refractory group, post-consolidation minimal residual disease (MRD)-positive group, early MRD-positive group, and MRD-negative group. Blinatumomab for the relapsed/refractory group was considered as second-line treatment, while the other 3 groups as first-line treatment. The MRD negativity rate after treatment, the survival rates and the incidence of severe adverse events were compared across these groups. Patients who received blinatumomab for more than 7 days were included in the efficacy analysis. Survival analysis was performed using the Kaplan-Meier method, and Log-Rank test was used to compare the survival rates among groups.Results:Among the 323 patients, 191 (59.1%) were male, with the age of 6.2 (3.9, 10.5) years. There were 117 patients in the relapsed/refractory group, 62 cases in the post-consolidation MRD-positive group, 43 cases in the early MRD-positive group, and 101 cases in the MRD negative group. In the relapsed/refractory group, the complete remission rate and MRD negativity rate after one course of blinatumomab were 71.4% (35/49) and 81.5% (75/92) for the 49 children without complete remission and the 92 children with flow cytometry-positive MRD, respectively. In the post-consolidation MRD-positive group, the MRD negativity rates after one course of blinatumomab were 100.0% (27/27), 12/16 and 9/19 for patients with MRD positivity detected by flow cytometry, polymerase chain reaction and next-generation sequencing, respectively. In the early MRD-positive group, the MRD negativity rates were 96.7% (29/30) and 9/9 for flow cytometry and next-generation sequencing, respectively. The 2-year overall survival rate and event-free survival rate for the 319 children evaluable for efficacy were (90.6±1.7)% and (87.6±1.9)%, respectively, with the relapsed/refractory group showing significantly lower overall survival rates and event-free survival rate compared to the other groups ( χ2=21.40, 26.21,both P<0.001). Grade 3 or higher adverse events occurred in 128 cases (39.6%), with hematological toxicity observed in 101 cases, while cytokine release syndrome (CRS), infection, and neurotoxicity occurred in 11, 26 and 8 cases, respectively. In addition, there were statistically significant differences in the grade 3 or higher CRS among the four groups ( χ2=8.03, P<0.05). Conclusion:Blinatumomab can clear MRD more effectively and achieve superior survival outcomes when used as first-line treatment for pediatric B-ALL, with less CRS.

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