Objective:To mine risk signals of adverse events (AE) in agalsidase beta therapy for Fabry disease and provide reference for the safe use in clinical practice.Methods:The AE reports with agalsidase beta as the primary suspect drug in the US Food and Drug Administration Adverse Event Reporting System (FAERS) database from the first quarter of 2004 to the third quarter of 2024 were collected and the clinical information of patients involved was analyzed descriptively. AEs were standardized and categorized using the preferred term (PT) and system organ class (SOC) in Medical Dictionary for Regulatory Activities version 26.0. Signal mining was performed using the reporting odds ratio (ROR) method and Bayesian confidence propagation neural network (BCPNN) method. A PT was defined as the risk signal if the number of reports was ≥3, the lower limit of the 95% confidence interval ( CI) of ROR was >1, and the information component minus two times standard deviation ( IC025) was >0. Descriptive statistical analysis were then conducted on the risk signals. The relative risk of target adverse events in gender subgroups was evaluated using the ROR method. Results:A total of 24 163 AE reports with agalsidase beta as the primary suspect drug were collected, involving 7 763 patients and 2 382 PTs. Severe adverse events accounted for 34.9% (2 712/7 763). A total of 121 risk signals (namely PTs) across 15 SOCs were obtained. The top 5 PTs ranked by number of reports were pyrexia (483 cases), chills (449 cases), infusion related reaction (282 cases), chest pain (221 cases), and cerebrovascular accident (218 cases). The top 5 PTs ranked by signal intensity were cornea verticillata ( ROR=56.44, IC025=4.59), drug specific antibody present ( ROR=54.13, IC025=5.46), cerebral calcification ( ROR=24.82, IC025=3.16), myocardial necrosis marker increased ( ROR=24.14, IC025=3.83), and infusion site rash ( ROR=20.41, IC025=3.46). Among the top 30 PTs ranked by number of reports and signal intensity, there were 4 and 13 PTs not documented in the drug label, respectively. The former included atrial fibrillation, cellulitis, neuralgia, and pallor, while the latter included cornea verticillate, cerebral calcification, auricular swelling, vein discoloration, aortic dilatation, bundle branch block, myocardial fibrosis, cardiac infection, vein rupture, sudden hearing loss, meningitis viral, deafness transitory, and corneal opacity. Subgroup analysis by sex showed that males had higher risks in developing chills, tremor, drug specific antibody detection, renal failure, and renal impairment, while females had higher risks in weight increase and dizziness. Conclusions:The main risk signals of adverse events of agalsidase beta include pyrexia, chills, and infusion related reactions, which are consistent with those documented in package inserts. In addition, potential new risk signals such as atrial fibrillation, cellulitis, neuralgia, cornea verticillate, auricular swelling, and sudden hearing loss were also detected. The relative risks of some target adverse events vary between genders, and it is recommended to strengthen targeted drug monitoring and individualized management in clinical practice.

Objective:To mine risk signals of adverse events (AE) in agalsidase beta therapy for Fabry disease and provide reference for the safe use in clinical practice.Methods:The AE reports with agalsidase beta as the primary suspect drug in the US Food and Drug Administration Adverse Event Reporting System (FAERS) database from the first quarter of 2004 to the third quarter of 2024 were collected and the clinical information of patients involved was analyzed descriptively. AEs were standardized and categorized using the preferred term (PT) and system organ class (SOC) in Medical Dictionary for Regulatory Activities version 26.0. Signal mining was performed using the reporting odds ratio (ROR) method and Bayesian confidence propagation neural network (BCPNN) method. A PT was defined as the risk signal if the number of reports was ≥3, the lower limit of the 95% confidence interval ( CI) of ROR was >1, and the information component minus two times standard deviation ( IC025) was >0. Descriptive statistical analysis were then conducted on the risk signals. The relative risk of target adverse events in gender subgroups was evaluated using the ROR method. Results:A total of 24 163 AE reports with agalsidase beta as the primary suspect drug were collected, involving 7 763 patients and 2 382 PTs. Severe adverse events accounted for 34.9% (2 712/7 763). A total of 121 risk signals (namely PTs) across 15 SOCs were obtained. The top 5 PTs ranked by number of reports were pyrexia (483 cases), chills (449 cases), infusion related reaction (282 cases), chest pain (221 cases), and cerebrovascular accident (218 cases). The top 5 PTs ranked by signal intensity were cornea verticillata ( ROR=56.44, IC025=4.59), drug specific antibody present ( ROR=54.13, IC025=5.46), cerebral calcification ( ROR=24.82, IC025=3.16), myocardial necrosis marker increased ( ROR=24.14, IC025=3.83), and infusion site rash ( ROR=20.41, IC025=3.46). Among the top 30 PTs ranked by number of reports and signal intensity, there were 4 and 13 PTs not documented in the drug label, respectively. The former included atrial fibrillation, cellulitis, neuralgia, and pallor, while the latter included cornea verticillate, cerebral calcification, auricular swelling, vein discoloration, aortic dilatation, bundle branch block, myocardial fibrosis, cardiac infection, vein rupture, sudden hearing loss, meningitis viral, deafness transitory, and corneal opacity. Subgroup analysis by sex showed that males had higher risks in developing chills, tremor, drug specific antibody detection, renal failure, and renal impairment, while females had higher risks in weight increase and dizziness. Conclusions:The main risk signals of adverse events of agalsidase beta include pyrexia, chills, and infusion related reactions, which are consistent with those documented in package inserts. In addition, potential new risk signals such as atrial fibrillation, cellulitis, neuralgia, cornea verticillate, auricular swelling, and sudden hearing loss were also detected. The relative risks of some target adverse events vary between genders, and it is recommended to strengthen targeted drug monitoring and individualized management in clinical practice.

OBJECTIVE To analyze the influential factors of cardioto xicity in patients with positive breast cancer of human epidermal growth factor receptor 2（HER-2）treated by trastuzumab combined with chemotherapy. METHODS From April 2017 to January 2021，200 HER-2 positive breast cancer patients receiving pirarubicin + cyclophosphamide combined with sequential paclitaxel+trastuzumab were collected from our hospital. According to the presence or absence of cardiotoxicity ，the patients were divided into cardiotoxicity group and non-cardiotoxicity group. The clinical data and echocardiographic results of the patients were collected，and the influential factors of cardiotoxicity were analyzed. RESULTS Among 200 patients，43 patients suffered from cardiotoxicity with the incidence of 21.5％. The proportion of patients with cardiotoxicity during pirarubicin+cyclophosphamide therapy accounted for 5.5％（11/200），and the proportion of patients with cardiotoxicity during sequential paclitaxel+trastuzumab therapy accounted for 20.5％（41/200）；the latter was significantly higher than the former （P＜0.01）. At the same time ，the decrease of left ventricular ejection fraction during sequential therapy of paclitaxel and trastuzumab was significantly higher than that during pirarubicin+cyclophosphamide therapy [ 14％（12％，17％）vs. 7％（3％，10％），P＜0.001]. Compared with patients without cardiotoxicity ，the proportion of patients with cardiotoxicity with a history of hyperlipidemia was significantly higher （P＜ 0.01），while the proportion of patients receiving dexrazoxane was significantly lower （P＜0.01）. Results of binary Logistic regression analysis showed that the history of hyperlipidemia [OR ＝3.672，95％ CI（1.499，8.992），P＝0.004] and the use of dextrazoxane [OR ＝0.154，95％ CI（0.072，0.330）， P＜0.001] were associated with the occurrence of cardiotoxicity. CONCLUSIONS Hyperlipidemia is an independent risk factor for cardiotoxicity induced by pirarubicin + cyclophosphamide combined with sequential paclitaxel+trastuzumab in HER 2 positive breast cancer patients ，while the use of dextrazoxane is a protective factor.

OBJECTIVE:To observe therapeutic efficacy of Rhodiola wallichiana injection for swelling and pain in fracture pa-tients during perioperative period. METHODS:120 patients with closed fresh extremity long bones fracture were randomly divided into trial group and control group,with 60 cases in each group. Both groups received open reduction and plate fixation. Before and after op-eration,control group was given routine medicine and anti-inflammatory treatment;trial group was additionally given R. wallichiana in-jection 10 ml(250 ml added into 5%Glucose injection)intravenously,qd,on the basis of control group. A treatment course lasted for 10 d. The swelling degree before operation,1 d and 3 d after operation,pain degree on the day of operation and 1,2,3 and 7 d after operation,complication and average length of stay were observed and compared between 2 groups. RESULTS:The extent of swelling and pain,the incidence of complications and average hospitalization days of trial group were lower or shorter than control group,with statistical significance (P<0.05). CONCLUSIONS:R. wallichiana injection can promote blood circulation and remove blood stasis, ease the hydropsia and pain,relieve swelling and pain,reduce the incidence of complications and shorten hospitalization day.

ObjectiveTo investigate the clinical efficacy of the 3-in-1 technique of Disc-FX system,namely:discectomy,radiofrequency ablation and annuloplasty for discogenic low back pain in the early stage.MethodsFrom February 2010 to February 2011,40 patients with the discogenic low back pain underwent discectomy,radiofrequency ablation and annuloplasty using Disc-FX system were retrospectively analyzed,including 22 males and 18 females with an average age of 38.7 years(range,32-58 years).The visual analogue scale (VAS) scoring was applied to evaluate the back and low limb pain at preoperative,postoperative 1week,3 months,6 months,and last follow-up.The Macnab score was also evaluated at last follow-up.Results All the patients were followed up for average 13.8 months (range,6-18 months).The operative time averaged 26 min(range,20-40 min).No surgical complication was found during the follow-up.The VAS of preoperative low back pain was 6.60±1.47,VAS score of low back pain at postoperative 1 week,3 months,6 months,and final follow-up were 1.05±0.68,1.15±0.70,1.00±0.62,0.95±0.63,respectively.The postoperative VAS of low back pain decreased significantly compared with that preoperative.The VAS of preoperative limb pain was 3.05±1.23,VAS score of limb pain at postoperative 1 week,3 months,6 months,and final follow-up after operation were 1.10±0.74,1.15±0.70,1.10±0.72,0.95±0.54,respectively.The postoperative VAS of limb pain decreased significantly compared with that preoperative.The evaluation of Macnab score were excellent in 20 patients,good in 17,fair in 2,and poor in 1,suggesting an effective rate of 92.5％ (37/40).Conclusion Disc-FX system,which combined three surgical technique in one procedure,provides a new choice for orthopedics to treat discogenic low back pain.It can give satisfactory clinical outcomes during a short-term followup,but the long term clinical outcomes remains unknown.

Objective To evaluate family economic burden of children with autism, or with physical disability or with intellectual disability.Methods227 parents of children with autism, children with physical disability, children with intellectual disability and normal children were interviewed for their family economic burden.ResultsThe medical cost and caring cost of children with disability were significantly more than those of normal children, and the education cost, clothes cost and amusement cost of children with disability were significantly less than those of normal children. Family income was only affected by education level of parents. Families of children with disability received more economic assistance than families of normal children except families of autistic children. More children the family had, less economic assistance the family acquired. Compared with normal children, the family economic burden of children with disability were as following, children with autism (19582.4 RMB per year), children with physical disability (16410.1 RMB per year), children with intellectual disability (6391.0 RMB per year). ConclusionFamilies of children with autism, children with physical disability and children with intellectual disability had heavier economic burden than families of normal children.

Objective To investigation the condition,expectation and support of quality of life in adult with intellectual disability.MethodsA total of 394 adult with intellectual disability,their parents and friends in Beijing were involved in the study.Their qualities of life were evaluated.ResultsAbout quality of life,the adult with intellectual disability had significant more expectation than they had gained actually;their parents and friends had significant more expectation than themselves and staff of primary rehabilitation did.ConclusionIt is necessary to provide support to improve the quality of life of adult with intellectual disability.