Objective:To explore the survival status and economic burden of disease for patients with Cryopyrin Associated Periodic Syndrome(CAPS)in China.Methods:From August 2023 to February 2024,a questionnaire survey was conducted on patients who volunteered to participate.The survey included patients'sociodemographic characteristics,current medical treatment status,disease economic burden and life quality.Results:A total of 35 valid questionnaires were collected.The average age of onset for the patients was 5.67 years,and the average duration from onset to confirmed diagnosis was 7.63 years.The average total medical cost per person in the past 12 months was 82 532.79 yuan,which is significantly higher than the national per capita disposable income of China in 2023.Conclusion:CAPS has an early onset and a long duration until diagnosis,with treatment primarily symptomatic,resulting in a heavy disease burden for patients and their families.

Aim To explore the clinical efficacy of sequential balloon pulmonary angioplasty(BPA)in the treat-ment of patients with chronic thromboembolic pulmonary hypertension(CTEPH),and analyze the safety during the periop-erative period.Methods 30 patients with CTEPH who underwent BPA treatment at Luoyang Central Hospital affiliated to Zhengzhou University from August 2018 to February 2024 were selected,30 patients with CTEPH who chose drug treatment were set as controls,the patients'gender,age,body mass index,comorbidities,plasma N-terminal pro-brain natriuretic peptide(NT-proBNP)levels,application of pulmonary hypertension targeted drugs,6-minute walking dis-tance(6-MWD),WHO cardiac function classification and cardiac ultrasound pulmonary arterial pressure related data were collected.Right heart catheterization was performed to obtain pulmonary hemodynamic parameters in the BPA group.Changes in WHO cardiac function classification,6-MWD,NT-proBNP,pulmonary vascular hemodynamic parameters and cardiac ultrasound pulmonary artery pressure data before BPA,after the final BPA and at 6-month follow-up were com-pared,as well as differences between the two groups.The occurrence and management results of surgical complications such as pulmonary artery injury,contrast nephropathy and reperfusion pulmonary edema were recorded.Results Compared with before BPA,after the last BPA,cardiac output(CO),cardiac index(CI),and mixed venous oxygen satu-ration(SvO2)increased,mean pulmonary artery pressure(mPAP)and pulmonary vascular resistance(PVR)decreased(all P＜0.05),and 6-MWD,NT-proBNP,right ventricular diameter,right atrial up-down diameter,right atrial left-right diameter,left ventricular diastolic end diameter,and tricuspid regurgitation velocity all improved(all P＜0.05).Com-pared with the control group,the surgical group showed an increase in 6-MWD,a decrease in NT-proBNP levels,a de-crease in right ventricular diameter,right atrial up-down diameter and right atrial left-right diameter,an increase in left ventricular end diastolic diameter,a decrease in tricuspid regurgitation velocity and a significant improvement in WHO car-diac function classification(all P＜0.05).Among 30 BPA patients,2 patients experienced hemoptysis during surgery,1 patient developed reperfusion pulmonary edema after surgery,and 1 patient developed contrast nephropathy.After treat-ment,all patients improved and were discharged.Conclusion Sequential BPA has good clinical efficacy and safety in the treatment of CTEPH patients,and is an effective technique for treating CTEPH,which is worth promoting in clinical practice.

Aim To explore the clinical efficacy of sequential balloon pulmonary angioplasty(BPA)in the treat-ment of patients with chronic thromboembolic pulmonary hypertension(CTEPH),and analyze the safety during the periop-erative period.Methods 30 patients with CTEPH who underwent BPA treatment at Luoyang Central Hospital affiliated to Zhengzhou University from August 2018 to February 2024 were selected,30 patients with CTEPH who chose drug treatment were set as controls,the patients'gender,age,body mass index,comorbidities,plasma N-terminal pro-brain natriuretic peptide(NT-proBNP)levels,application of pulmonary hypertension targeted drugs,6-minute walking dis-tance(6-MWD),WHO cardiac function classification and cardiac ultrasound pulmonary arterial pressure related data were collected.Right heart catheterization was performed to obtain pulmonary hemodynamic parameters in the BPA group.Changes in WHO cardiac function classification,6-MWD,NT-proBNP,pulmonary vascular hemodynamic parameters and cardiac ultrasound pulmonary artery pressure data before BPA,after the final BPA and at 6-month follow-up were com-pared,as well as differences between the two groups.The occurrence and management results of surgical complications such as pulmonary artery injury,contrast nephropathy and reperfusion pulmonary edema were recorded.Results Compared with before BPA,after the last BPA,cardiac output(CO),cardiac index(CI),and mixed venous oxygen satu-ration(SvO2)increased,mean pulmonary artery pressure(mPAP)and pulmonary vascular resistance(PVR)decreased(all P＜0.05),and 6-MWD,NT-proBNP,right ventricular diameter,right atrial up-down diameter,right atrial left-right diameter,left ventricular diastolic end diameter,and tricuspid regurgitation velocity all improved(all P＜0.05).Com-pared with the control group,the surgical group showed an increase in 6-MWD,a decrease in NT-proBNP levels,a de-crease in right ventricular diameter,right atrial up-down diameter and right atrial left-right diameter,an increase in left ventricular end diastolic diameter,a decrease in tricuspid regurgitation velocity and a significant improvement in WHO car-diac function classification(all P＜0.05).Among 30 BPA patients,2 patients experienced hemoptysis during surgery,1 patient developed reperfusion pulmonary edema after surgery,and 1 patient developed contrast nephropathy.After treat-ment,all patients improved and were discharged.Conclusion Sequential BPA has good clinical efficacy and safety in the treatment of CTEPH patients,and is an effective technique for treating CTEPH,which is worth promoting in clinical practice.

Objective To observe clinical efficacy of Danggui Yinzi prescription combined with focused ultrasound in treatment of vulvar lichen sclerosus(VLS)with blood deficiency and dryness.Methods A total of 60 patients with blood deficiency and dryness VLS hospitalized in Gansu Provincial Hospital of Traditional Chinese Medicine from September 2023 to September 2024 were selected as study subjects,they were divided into control group(n=30)and treatment group(n=30)by random number table method.Control group was treated with focused ultrasound,and treatment group was treated with oral Danggui Yinzi prescription on the basis of control group.After 1 month of treatment,symptom scores(skin lesion area,color,etc.)and clinical efficacy of two groups were compared.Results After treatment,symptom scores of two groups were lower than those before treatment,and symptom scores of treatment group were lower than those of control group.Total effective rate of treatment group was higher than that of control group(P＜0.05).Conclusion Danggui Yinzi prescription combined with focused ultrasound is effective in treatment of VLS with blood deficiency and dryness,which can significantly reduce or eliminate itching symptoms and promote skin healing.

Objective:To explore the survival status and economic burden of disease for patients with Cryopyrin Associated Periodic Syndrome(CAPS)in China.Methods:From August 2023 to February 2024,a questionnaire survey was conducted on patients who volunteered to participate.The survey included patients'sociodemographic characteristics,current medical treatment status,disease economic burden and life quality.Results:A total of 35 valid questionnaires were collected.The average age of onset for the patients was 5.67 years,and the average duration from onset to confirmed diagnosis was 7.63 years.The average total medical cost per person in the past 12 months was 82 532.79 yuan,which is significantly higher than the national per capita disposable income of China in 2023.Conclusion:CAPS has an early onset and a long duration until diagnosis,with treatment primarily symptomatic,resulting in a heavy disease burden for patients and their families.

Objective To observe clinical efficacy of Danggui Yinzi prescription combined with focused ultrasound in treatment of vulvar lichen sclerosus(VLS)with blood deficiency and dryness.Methods A total of 60 patients with blood deficiency and dryness VLS hospitalized in Gansu Provincial Hospital of Traditional Chinese Medicine from September 2023 to September 2024 were selected as study subjects,they were divided into control group(n=30)and treatment group(n=30)by random number table method.Control group was treated with focused ultrasound,and treatment group was treated with oral Danggui Yinzi prescription on the basis of control group.After 1 month of treatment,symptom scores(skin lesion area,color,etc.)and clinical efficacy of two groups were compared.Results After treatment,symptom scores of two groups were lower than those before treatment,and symptom scores of treatment group were lower than those of control group.Total effective rate of treatment group was higher than that of control group(P＜0.05).Conclusion Danggui Yinzi prescription combined with focused ultrasound is effective in treatment of VLS with blood deficiency and dryness,which can significantly reduce or eliminate itching symptoms and promote skin healing.

OBJECTIVE To evaluate the economics of trastuzumab deruxtecan versus the physician-selected chemotherapy （TPC） regimen in the second-line treatment of advanced breast cancer with epidermal growth factor receptor 2 （HER-2） low expression from the perspective of the Chinese healthcare system. METHODS Based on the data of DESTINY-Breast04 clinical trial， the dynamic Markov model was constructed. The time frame of the model simulation was 10 years， and the cycle was 3 weeks. Taking cost， quality-adjusted life year （QALY） and incremental cost-effectiveness ratio （ICER） as the model output indicators， the discount rate of 5% was applied， and 3 times China’s per capita gross domestic product （GDP） in 2023 was taken as the willingness-to-pay （WTP） threshold value. Cost-utility analysis was used to evaluate the economics of the two treatment regiments in the hormone receptor-positive cohort and all patient cohorts， and uncertainty analysis was used to verify the robustness of the basic analysis result. RESULTS The results of the basic analysis showed that compared with the TPC regimen， the ICER value of trastuzumab deruxtecan regimen edu.cn were 1 045 655.76 and 906 404.99 yuan/QALY in the hormone receptor-positive cohort and all patients， respectively， both exceeding the WTP threshold （268 074 yuan/QALY）. The results of single factor sensitivity analysis showed that progression-free survival utility value， the price of trastuzumab deruxtecan and progression disease utility had a significant influence on the model results. The results of probability sensitivity analysis showed that when the WTP threshold was 3 times China’s per capita GDP in 2023， the probability of economic viability of trastuzumab deruxtecan was 0. The results of scenario analysis showed that when the patient assistance program for trastuzumab deruxtecan was considered， the probability of trastuzumab deruxtecan regimen being economical was 0. However， when the price of trastuzumab deruxtecan was reduced by 70%， the probability of its being cost-effective was significantly increased to 82.80%. CONCLUSIONS At a WTP threshold of 3 times China’s per capita GDP in 2023， the trastuzumab deruxtecan regimen is not cost-effective compared to TPC regimen for the second-line treatment of advanced breast cancer with HER-2 low expression. Reducing the price of trastuzumab deruxtecan by region can improve its cost-effectiveness.

Objective:To retrospectively analyze the treatment status of tyrosine kinase inhibitors (TKI) in newly diagnosed patients with chronic myeloid leukemia (CML) in China.Methods:Data of chronic phase (CP) and accelerated phase (AP) CML patients diagnosed from January 2006 to December 2022 from 77 centers, ≥18 years old, and receiving initial imatinib, nilotinib, dasatinib or flumatinib-therapy within 6 months after diagnosis in China with complete data were retrospectively interrogated. The choice of initial TKI, current TKI medications, treatment switch and reasons, treatment responses and outcomes as well as the variables associated with them were analyzed.Results:6 893 patients in CP ( n=6 453, 93.6%) or AP ( n=440, 6.4%) receiving initial imatinib ( n=4 906, 71.2%), nilotinib ( n=1 157, 16.8%), dasatinib ( n=298, 4.3%) or flumatinib ( n=532, 7.2%) -therapy. With the median follow-up of 43 ( IQR 22-75) months, 1 581 (22.9%) patients switched TKI due to resistance ( n=1 055, 15.3%), intolerance ( n=248, 3.6%), pursuit of better efficacy ( n=168, 2.4%), economic or other reasons ( n=110, 1.6%). The frequency of switching TKI in AP patients was significantly-higher than that in CP patients (44.1% vs 21.5%, P<0.001), and more AP patients switched TKI due to resistance than CP patients (75.3% vs 66.1%, P=0.011). Multi-variable analyses showed that male, lower HGB concentration and ELTS intermediate/high-risk cohort were associated with lower cytogenetic and molecular responses rate and poor outcomes in CP patients; higher WBC count and initial the second-generation TKI treatment, the higher response rates; Ph + ACA at diagnosis, poor PFS. However, Sokal intermediate/high-risk cohort was only significantly-associated with lower CCyR and MMR rates and the poor PFS. Lower HGB concentration and larger spleen size were significantly-associated with the lower cytogenetic and molecular response rates in AP patients; initial the second-generation TKI treatment, the higher treatment response rates; lower PLT count, higher blasts and Ph + ACA, poorer TFS; Ph + ACA, poorer OS. Conclusion:At present, the vast majority of newly-diagnosed CML-CP or AP patients could benefit from TKI treatment in the long term with the good treatment responses and survival outcomes.

  Objective  To study the demographic and clinical characteristics, correlation of genotype and phenotype and treatment of Blau syndrome to facilitate early diagnosis and timely treatment of Blau syndrome.  Methods  Seventy-two patients with Blau syndrome from 11 centers from May 2006 to April 2022 were retrospectively analyzed, and their general information, clinical data, laboratory examination and treatment medication were collected.  Results  The distribution of patients with Blau syndrome was uniform in geographical north and south of China, and there was no obvious gender bias. The mean age of onset was (14.30±12.81) months, and the age of diagnosis was (55.18±36.22) months. 35% of patients with Blau syndrome happened before 1 year old, and all patients developed before 5 years old. 87.50% (63/72) had granulomatous arthritis, 65.28% (47/72) had rash, 36.11% (26/72) had ocular involvement, 27.78% (20/72) had fever, and 15.28% (11/72) had pulmonary involvement. Arthritic manifestations of Blau syndrome were most at risk, followed by rash, ocular involvement, and fever.The first 25 months of the disease, the risk of developing a rash was the greatest. The risk of developing arthritis was the greatest between 25 months and 84 months. The main mutations were p.R334Q and p.R334W, and patients with p.R334Q mutation had relatively high incidence of fever (35.71%[5/14] vs. 14.29%[1/7], P=0.43) and ocular involvement (42.86%[6/14]vs. 28.57%[2/7], P=0.51). There was a relatively high incidence of rash (85.71%[6/7] vs. 64.29%[9/14], P=0.59) in patients with the p.R334W mutation. Forty-five patients(62.50%)were treated with a combina-tion of glucocorticoid and methotrexate. Twenty-two patients were treated with tumor necrosis factor antagonist in addition to glucocorticoid and methotrexate.  Conclusions  The risk of different clinical manifestations of Blau syndrome from high to low was arthritis, followed by rash, ocular involvement and fever. The main treatment was glucocorticoid combined with methotrexate, to which biological agents could be added.

Objective:To explore the effects of early-life maternal deprivation on depressive-like behavior and neurogenesis in the granular layer of hippocampus in adolescent rats (6-7 weeks old).Methods:Neonatal rats were randomly divided into maternal deprivation group and control group, with 3 litters in each group.Rats in the maternal deprivation group were given maternal deprivation from 1 to 14 days after birth and rats in the control group were caged with the mother rats and raised normally.The body weight of rats at 5-6 weeks old was recorded and the increased body weight was calculated.When the rats were 6 weeks old, the sucrose preference test was carried out.Then the rats were killed and immunofluorescence histochemistry was applied to compare the expression of Ki67 and Nestin positive cells in the dentate gyrus of hippocampus.SPSS 22.0 software was used for statistical analysis.The data of the two groups were tested to conform to the normal distribution, and then t-test was carried out. Results:There was significant difference in body weight growth between the two groups at the age of 5-6 weeks.Compared with the control group, rats in the maternal deprivation group had lower body weight growth ((20.57±2.19) g, (30.57±1.25) g, t=3.96, P<0.01)) and lower sucrose preference rate((58.38±53.14)%, (73.88±3.67)%, t=3.21, P<0.01). The results of immunofluorescence showed that the number of Ki67 positive cells in the granular layer of hippocampus in the maternal deprivation group was less than that in the control group ((5.13±0.31), (7.60±0.38), t=5.09, P<0.01), and the number of Nestin immunofluorescence positive cells was more than that in the control group ((16.65±0.79), (7.64±0.70), t=8.51, P<0.01). The Nestin immunofluorescence positive cells in the maternal deprivation group had more protrusions and branches, and the morphology was similar to astrocytes, while the immunofluorescence positive cells in the control group had fewer protrusions, and the cell body was oval. Conclusions:Early-life maternal deprivation leads to depressive-like behavior in adolescent rats, which may be associated with the decrease of neurogenesis and activation of astrocytes in the dentate gyrus of the hippocampus.