OBJECTIVE To provide a basis for aligning Chinese pharmacoeconomic research on heart failure （HF） with international standards. METHODS A qualitative comparison o f domestic and global HF pharmacoeconomic studies was conducted across four dimensions： research methods and model application， research perspectives and endpoints， data sources and parameter selection， and policy translation and practical impact. RESULTS & CONCLUSIONS Global studies predominantly utilize long-term dynamic models， societal perspectives， real-world data integration， and directly inform reimbursement decisions. Conversely， domestic research often relies on short-term simplified models， a single healthcare system perspectives， literature-derived data， and individual medicine recommendations. Future domestic studies should transition to long-term dynamic modeling， develop localized disease-specific utility databases via big data， establish reimbursement-linked closed-loop mechanisms， and foster multidisciplinary collaboration to optimize healthcare resource allocation.

Objective To translate self-care in spinal cord injuries inventory(SC-SCII)into Chinese and evaluate its reliability and validity,therefore to provide medical staff with a tool for evaluation of self-care behaviour of patients.Methods Brislin translation model was adopted for forward translation,back-translation,cultural adaptation and pilot testing in the development of Chinese version of SC-SCII.Convenience sampling was used to recruit 207 patients with spinal cord injury(SCI)from three Tier-IIIA hospitals in Xi'an between January 2024 and March 2025 for the purpose of assessing the reliability and validity of the translated inventory.Results A total of 202 patients completed the study.The finalised Chinese version of SC-SCII encompassed 4 dimensions with a total of 35 items,with the same structure as the original SC-SCII.Scale-level content validity index was 0.948,and the item-level content validity indices ranged from 0.912 to 1.000.Four common factors were extracted by exploratory factor analysis:self-care maintenance(12 items),self-care monitoring(8 items),self-care management(8 items)and self-care self-efficacy(7 items),cumulatively accounting for 61.402%of the total variance.The overall Cronbach's α coefficient was 0.820 and the Cronbach's α coefficients of the four dimensions were 0.935,0.905,0.921 and 0.892,respectively.The split-half reliability was 0.798,and the two-week retest reliability was 0.875.No statistically significant difference was observed between the original and sinicised scales(paired t-test,P>0.05),indicating a high cross-time stability of the sinicised scale.Conclusion The Chinese version of SC-SCII demonstrates a good reliability and validity,making it a suitable tool in evaluation of self-care abilities in SCI patients.

Objective:To investigate the semen characteristics of male patients with autosomal dominant polycystic kidney disease (ADPKD) complicated by infertility and the impact of PKD1 mutations on their reproductive outcomes following preimplantation genetic testing for monogenic diseases (PGT-M). Methods:A retrospective cohort study was conducted to analyze the clinical data of ADPKD patients in the Clinical Center of Reproductive Medicine, the First Affiliated Hospital of Nanjing Medical University from January 2015 to December 2023. The study included two groups: observation group consisting of male infertility patients with ADPKD aged 20-45 years ( n=78), and control group comprising ADPKD patients with PKD1 mutations who demonstrated normal semen parameters ( n=5). According to the type of mutation, the patients were further divided into the PKD1 truncated mutation group and the non-truncated mutation group. Baseline data, ovulation induction outcomes and PGT-M clinical outcomes were compared between the two groups. Binary logistic regression analysis was used to assess the effects of age, family history, mutation type and mutation position on semen quality and live birth rates. Results:In the observation group, there were 28 cases of asthenozoospermia, 38 cases of oligoasthenozoospermia, and 12 cases of azoospermia. The total sperm count and forward motility ratio of patients with asthenozoospermia, oligoasthenozoospermia, and azoospermia in the observation group were significantly different from those in control group (all P<0.001). Genetic diagnosis revealed PKD1 mutations in 70 cases, PKD2 mutation in 1 case, and no known pathogenic mutations were identified in 7 cases. Among the 70 ADPKD patients with PKD1 mutations, 52 couples underwent PGT-M assisted reproduction. The blastocyst formation rate was significantly lower in the non-truncating mutation group [44.74% (51/114)] than in the truncating mutation group [58.76% (161/274), P=0.011]. No statistically significant differences were observed between the two groups in other parameters including age of both partners, semen parameters, number of oocytes retrieved, two pronuclei (2PN) rate, number of available embryos, high-quality embryo rate, clinical pregnancy rate, miscarriage rate, and live birth rate (all P>0.05). Binary logistic regression analysis showed that neither PKD1 mutation type nor PKD1 mutation site was an independent factor affecting semen parameters and live birth rate in ADPKD patients (all P>0.05). Conclusions:Asthenospermia and oligoasthenospermia are the most common semen phenotypes in ADPKD-associated male infertility. The PKD1 mutation type and location are not associated with abnormal semen parameters in ADPKD-associated infertility patients. PKD1 mutation types do not affect the outcomes of PGT-M in infertile patients with ADPKD.

Objective To translate self-care in spinal cord injuries inventory(SC-SCII)into Chinese and evaluate its reliability and validity,therefore to provide medical staff with a tool for evaluation of self-care behaviour of patients.Methods Brislin translation model was adopted for forward translation,back-translation,cultural adaptation and pilot testing in the development of Chinese version of SC-SCII.Convenience sampling was used to recruit 207 patients with spinal cord injury(SCI)from three Tier-IIIA hospitals in Xi'an between January 2024 and March 2025 for the purpose of assessing the reliability and validity of the translated inventory.Results A total of 202 patients completed the study.The finalised Chinese version of SC-SCII encompassed 4 dimensions with a total of 35 items,with the same structure as the original SC-SCII.Scale-level content validity index was 0.948,and the item-level content validity indices ranged from 0.912 to 1.000.Four common factors were extracted by exploratory factor analysis:self-care maintenance(12 items),self-care monitoring(8 items),self-care management(8 items)and self-care self-efficacy(7 items),cumulatively accounting for 61.402%of the total variance.The overall Cronbach's α coefficient was 0.820 and the Cronbach's α coefficients of the four dimensions were 0.935,0.905,0.921 and 0.892,respectively.The split-half reliability was 0.798,and the two-week retest reliability was 0.875.No statistically significant difference was observed between the original and sinicised scales(paired t-test,P>0.05),indicating a high cross-time stability of the sinicised scale.Conclusion The Chinese version of SC-SCII demonstrates a good reliability and validity,making it a suitable tool in evaluation of self-care abilities in SCI patients.

Objective·To explore the antitumor effects and potential mechanisms of combining phosphoinositide 3-kinase,FYVE-type zinc finger containing(PIKfyve)inhibitor YM201636 with the stimulator of interferon genes(STING)agonist diABZI in STING pathway-deficient melanoma.Methods·The mRNA and protein expression levels of STING in human cancer cell lines were obtained from the Cancer Cell Line Encyclopedia(CCLE)and UniProt databases.Based on median expression values,melanoma cell lines with high STING mRNA but low protein expression were identified.Quantitative real-time PCR(qRT-PCR)and Western blotting were performed to validate STING mRNA and protein expression in human melanoma cells.The murine melanoma cell line YUMM1.7,characterized by low STING protein expression,was selected through Western blotting.The ability of YM201636 to restore STING protein expression in YUMM1.7 cells was evaluated.STING agonist diABZI was then applied in combination with YM201636 to analyze the synergistic tumor cell-killing effect through CCK-8 assay.Western blotting was used to detect the phosphorylation of TANK-binding kinase 1(TBK1)and interferon regulatory factor 3(IRF3),and qRT-PCR was used to evaluate type Ⅰ interferon expression.A mouse melanoma model was established and treated with YM201636,diABZI,or their combination.Tumor volume was measured,and treatment efficacy was assessed.RNA sequencing and immunofluorescence staining were performed to analyze immune cell infiltration in the tumor microenvironment.Results·Database analyses,qRT-PCR,and Western blotting confirmed that some human melanoma cell lines exhibited high STING mRNA expression but low STING protein levels.YM201636 significantly increased STING protein expression in YUMM1.7 cells(P<0.001).Combined treatment with YM201636 and diABZI significantly enhanced phosphorylation of TBK1 and IRF3(P<0.05),indicating effective activation of the STING signaling pathway.This combination also promoted the expression of type Ⅰ interferons(P<0.001)and enhanced tumor cell killing in vitro.In vivo,the combination therapy markedly suppressed melanoma growth compared to monotherapy.Immune profiling of the tumor microenvironment revealed significantly increased infiltration of CD4? T cells and CD8? T cells in the combination treatment group(P<0.05).Conclusion·The PIKfyve inhibitor YM201636 could restore STING protein expression in STING-deficient melanoma and enhance the antitumor efficacy of the STING agonist diABZI,offering a promising therapeutic strategy for tumors with defective STING signaling.

Ionizing radiation occurs not only during nuclear and radiological terrorist attacks or nuclear accidents but also in medical applications and daily life. In recent years, the potential toxic effects of low-dose ionizing radiation on the male reproductive system have raised public health concerns. Most specifically, for the testis, a highly sensitive target organ to ionizing radiation, there is an urgent need to determine the mechanisms underlying the association between radiation-induced spermatogenic failure and male infertility. Focusing on the potential injurious effects of low-dose ionizing radiation on spermatogenesis, this review presents a summary and analysis of various underlying mechanisms including oxidative stress, apoptosis, and DNA damage repair and reviews recent domestic and international strategies for protecting against radiation-induced reproductive system damage in order to offer references for future research on the injurious effects of low-dose radiation on spermatogenesis and relevant protection.

Objective·To explore the antitumor effects and potential mechanisms of combining phosphoinositide 3-kinase,FYVE-type zinc finger containing(PIKfyve)inhibitor YM201636 with the stimulator of interferon genes(STING)agonist diABZI in STING pathway-deficient melanoma.Methods·The mRNA and protein expression levels of STING in human cancer cell lines were obtained from the Cancer Cell Line Encyclopedia(CCLE)and UniProt databases.Based on median expression values,melanoma cell lines with high STING mRNA but low protein expression were identified.Quantitative real-time PCR(qRT-PCR)and Western blotting were performed to validate STING mRNA and protein expression in human melanoma cells.The murine melanoma cell line YUMM1.7,characterized by low STING protein expression,was selected through Western blotting.The ability of YM201636 to restore STING protein expression in YUMM1.7 cells was evaluated.STING agonist diABZI was then applied in combination with YM201636 to analyze the synergistic tumor cell-killing effect through CCK-8 assay.Western blotting was used to detect the phosphorylation of TANK-binding kinase 1(TBK1)and interferon regulatory factor 3(IRF3),and qRT-PCR was used to evaluate type Ⅰ interferon expression.A mouse melanoma model was established and treated with YM201636,diABZI,or their combination.Tumor volume was measured,and treatment efficacy was assessed.RNA sequencing and immunofluorescence staining were performed to analyze immune cell infiltration in the tumor microenvironment.Results·Database analyses,qRT-PCR,and Western blotting confirmed that some human melanoma cell lines exhibited high STING mRNA expression but low STING protein levels.YM201636 significantly increased STING protein expression in YUMM1.7 cells(P<0.001).Combined treatment with YM201636 and diABZI significantly enhanced phosphorylation of TBK1 and IRF3(P<0.05),indicating effective activation of the STING signaling pathway.This combination also promoted the expression of type Ⅰ interferons(P<0.001)and enhanced tumor cell killing in vitro.In vivo,the combination therapy markedly suppressed melanoma growth compared to monotherapy.Immune profiling of the tumor microenvironment revealed significantly increased infiltration of CD4? T cells and CD8? T cells in the combination treatment group(P<0.05).Conclusion·The PIKfyve inhibitor YM201636 could restore STING protein expression in STING-deficient melanoma and enhance the antitumor efficacy of the STING agonist diABZI,offering a promising therapeutic strategy for tumors with defective STING signaling.

To report a Chinese pedigree with benign hereditary chorea (BHC) related to NKX2-1 gene mutation. The proband, a 16-year-old boy, was admitted with a 15-year history of recurrent involuntary choreiform movements, exacerbated over 2 weeks. The results of whole-exome sequencing showed that the proband and his mother both had a heterozygous mutation in exon 2 of the NKX2-1 gene (c.231_232dup:p.Pro78Hisfs *24), leading to a clinical diagnosis of BHC. No patient carrying this variant was previously reported in the literature. The clinical phenotype of NKX2-1 gene defects is complex, with BHC being a characteristic manifestation of the nervous system. This group of disorders is also referred to as NKX2-1-related disorders. This article discusses the clinical features, diagnosis and treatment points of patients with NKX2-1-related disorder with reference to the current literature, aiming to enhance clinicians′ understanding and management of this disease.

OBJECTIVE To conduct a rapid and comprehensive evaluation of commonly used oral dihydropyridine calcium channel blockers （DHP CCBs） in Guangxi， and provide scientific basis for clinical medication and drug selection in medical institutions. METHODS Based on the actual drug use data of public medical institutions at the second level and above in Guangxi Zhuang Autonomous Region， and based on the national centralized collection catalog， commonly used oral DHP CCBs were selected. The Professional Committee of Evidence-based Pharmacy of the Guangxi Pharmaceutical Association organized relevant experts from multiple medical institutions in the region to conduct a quantitative scoring of the selected oral DHP CCBs from five key dimensions of pharmaceutical characteristics， effectiveness， safety， economy， and other attributes， by referring to the Quick Guideline for Drug Evaluation and Selection in Chinese Medical Institutions （the Second Edition）， combined with the latest evidence-based medical research results， and widely soliciting suggestions from clinical and pharmaceutical experts. Finally， the Guangxi Expert Consensus on Rapid Comprehensive Evaluation of Oral Dihydropyridine Calcium Channel Blockers in the Treatment of Cardiovascular Disease was formulated. RESULTS A total of 30 commonly used oral DHP CCBs were selected. Among them， Amlodipine besylate tablets （Huizhi） demonstrated superior comprehensive performance （81.79 points）， excelling particularly in pharmaceutical properties， effectiveness and other attributes. The compound scores of Amlodipine besylate tablets （Chongqing Yaoyou） and Amlodipine besylate tablets （Suzhou Dongrui） ranked second and third respectively， with scores of 81.66 and 81.60 points. CONCLUSIONS This consensus can provide guidance and decision-making support for the rational clinical application of oral DHP CCBs in Guangxi Region and the selection of drug directories in medical institutions.

ObjectiveTo compare the contents and relative molecular weight distributions of proteins and polypeptides in Naoxuekang dropping pills， Huoxue Tongmai capsules and Maixuekang capsules of Hirudo single medicinal preparations， to evaluate the in vitro anticoagulant， antiplatelet and fibrinolytic activities of the three preparations， and to investigate the effects of temperature， pH and digestive enzymes on the anticoagulant activities of the three preparations. MethodsThe contents of soluble proteins and polypeptides in the three preparations were determined by bicinchoninic acid assay（BCA） and Bradford method， and the relative molecular weight distributions of the three preparations were determined by electrophoresis combined with gel chromatography. The antithrombin activity of the three preparations was evaluated by fibrinogen-thrombin time（Fibg-TT） method， and their anticoagulant activities were further assessed by the elongations of activated partial thromboplastin time（APTT）， prothrombin time（PT） and thrombin time（TT）. The antiplatelet aggregation activities of the three preparations were measured by turbidimetry and the fibrinolytic activities were measured by fibrin plate method. Relative TT was used as index to investigate the effects of temperature， pH and digestive enzyme buffer on anticoagulant activities of the three preparations. ResultsAt the lowest single dosage， the contents of proteins and polypeptides were in the order of Maixuekang capsules>Huoxue Tongmai capsules>Naoxuekang dropping pills. Both Huoxue Tongmai capsules and Maixuekang capsules had 11 electrophoretic bands between 4.0 kDa and 90 kDa， the bands of Maixuekang capsules were more clear in the range of >25 kDa， and there was 1 obvious band at 14 kDa for the two capsules. Huoxue Tongmai capsules had one specific band at 9.0 kDa and Maixuekang capsules had one specific band at 48.0 kDa. Naoxuekang dropping pills only had 2 electrophoretic bands at 6.5 kDa and 8.5 kDa， primarily containing peptides below 2 kDa， most of which were oligopeptides. The anticoagulant activity concentrations of the three preparations exhibited a certain dose-dependent effect. At the lowest single dosage， The anticoagulant activity concentrations were ranked as Naoxuekang dropping pills>Huoxue Tongmai capsules>Maixuekang capsules. The prolongation effect of the three preparations on coagulation time was dose-dependent. At the same concentration， the prolongation effect of Naoxuekang dropping pills and Huoxue Tongmai capsules was APTT prolongation rate>TT prolongation rate>PT prolongation rate， whereas for Maixuekang capsules， the sequence was TT prolongation rate>APTT prolongation rate>PT lengthening rate. At the single minimum dosage， the order of APTT prolongation rate was Maixuekang capsules>Huoxue Tongmai capsules≈Naoxuekang dropping pills， the order of PT prolongation rate was Naoxuekang dropping pills≈Maixuekang capsules>Huoxue Tongmai capsules， and the order of TT prolongation rate was Maixuekang capsules>Huoxue Tongmai capsules>Naoxuekang dropping pills. The three preparations showed dose-dependent effects on platelet aggregation induced by adenosine diphosphate（ADP） and arachidonic acid（AA）， and the effect induced by ADP was stronger than that induced by AA. The anti-platelet aggregation effect of Naoxuekang dropping pills was significantly stronger than that of Maixuekang capsules（P<0.01）， whereas Huoxue Tongmai capsules had the effect of promoting platelet aggregation. None of the three preparations had the ability to dissolve fibrin. The anticoagulant activity of Naoxuekang dropping pills was least affected by heating， while the activities of the two capsules decreased significantly within 5 min above 80 ℃， and continued to decrease within 2 h. Compared with pure water， the anticoagulant activities of the three preparations could be increased by 1-3 times under strong acidity（pH 1-3）. In the pepsin buffer， the anticoagulant activity of Naoxuekang dropping pills could be increased by 1-3 times， while the anticoagulant activities of Huoxue Tongmai capsules and Maxuekang capsules were significantly decreased， the lowest levels were about 60% and 20%， respectively. In trypsin buffer， the anticoagulant activities of Naoxuekang dropping pills， Huoxue Tongmai capsules and Maixuekang capsules decreased significantly， and the lowest levels decreased to about 41%， 41% and 35%， respectively. ConclusionThe contents of proteins and polypeptides and relative molecular weights of the preparations derived from lyophilized fresh Hirudo powder， dried Hirudo powder and reflux extract of Hirudo decrease sequentially， and the anticoagulant activity decrease gradually， but the anticoagulant pathway is different. And the anti-platelet aggregation activity of the reflux extract is significantly enhanced. The heat resistance and gastrointestinal stability of the three preparations increase successively， and the first two are suitable for enteric-soluble preparations， while the latter is suitable for routine oral administration. The above results can provide data reference for the rationality of different preparation methods， active substances， pharmacodynamics and mechanism of Hirudo preparations.