1.The Clinical and Genetics Characteristics of Oculopharyngodistal Myopathy
Jiaxi YU ; Zhihao QUAN ; Yilei ZHENG ; Jing AN ; Jing LIU ; Qingqing WANG ; Lingchao MENG ; Meng YU ; Zhiying XIE ; Jianwen DENG ; He LYU ; Wei ZHANG ; Yun YUAN ; Zhaoxia WANG
JOURNAL OF RARE DISEASES 2026;5(2):164-174
To analyze the clinical and genetic features of oculopharyngodistal myopathy (OPDM) patients and compare the phenotypic differences among various causative genes. A total of 65 genetically confirmed OPDM patients from 43 unrelated families, who were admitted to the Department of Neurology, Peking University First Hospital between January 2008 and December 2025, were retrospectively included.The general demographic data, clinical manifestations, laboratory/auxiliary examinations, muscle pathology, and genetic test results were systematically collected and analyzed. The clinical and pathological characteristics among different OPDM subtypes were compared. Among the 65 patients(39 male and 26 female), the mean age of onset was (31.20±10.43) years (range: 14 to 63 years). The initial symptom was predominantly distal limb weakness (67.44%), which gradually progressed to involve the extraocular muscles, pharyngeal muscles, facial muscles and proximal limb muscles. Serum creatine kinase levels were mildly to moderately elevated. Muscle pathological examinations revealed rimmed vacuoles and intranuclear inclusions (within muscle fibers). The mean duration from onset to diagnosis was (12.33±7.88) years (range: 1 to 32 years). All probands had negative results on conventional next-generation whole-exome sequencing; pathogenic variants were identified through third-generation long-read sequencing or OPDM-targeted repeat-primed polymerase chain reaction(RP-PCR). Among the 43 families, OPDM2 subtype was the most common genetic subtype ( OPDM2 was the predominant subtype in this study. All subtypes share similar age of onset and muscular pathological changes, yet exhibit distinct disease progression patterns. Future multicenter prospective cohort studies are warranted to further elucidate the clinical characteristics, pathogenetic mechanisms, and prognostic differences among OPDM subtypes.
2.Real world clinical data analysis of fuzuloparib for the treatment of ovarian epithelial cancer patients
Danhui WENG ; Jie JIANG ; Yingjie YANG ; Mingqian LU ; Jiaying BAI ; Ming LIU ; Xiaoling LI ; Jun TIAN ; Yutao GUAN ; Quan LI ; Liang CHEN ; Qiubo LYU ; Lixia MA ; Yali WANG ; Huicheng XU ; Hailong GUO ; Li SUN ; Ding MA ; Qinglei GAO
Chinese Journal of Obstetrics and Gynecology 2025;60(8):590-599
Objective:To evaluate the safety and effectiveness of fuzuloparib for the treatment of ovarian epithelial cancer patients in the real world setting.Methods:A retrospective analysis was conducted on the baseline data of 4 620 ovarian cancer patients who had received fuzuloparib monotherapy or combination therapy. Another 224 ovarian cancer patients who were willing to receive fuzuloparib monotherapy or combination therapy were prospectively enrolled, and their baseline characteristics, drug effectiveness, and safety data were analyzed.Results:(1) Among the 4 620 patients in the retrospective cohort, the median age of patients was 60 years; tumor types: 89.8% (4 149/4 620) had ovarian cancer. Among patients with clearly documented information, the vast majority had a histological type of serous carcinoma (82.9%, 3 770/4 546) and International Federation of Gynecology and Obstetrics (FIGO) staging of Ⅲ-Ⅳ (90.9%, 1 537/1 691). (2) Among the 224 patients in the prospective cohort, the median age of patients was 57 years; tumor types: 83.9% (188/224) had ovarian cancer. Among patients with clearly documented records, the predominant pathologic type was serous carcinoma (91.9%, 193/210), and FIGO stage was Ⅲ-Ⅳ in 79.9% (139/174). (3) Among the 224 prospective patients: 84 patients received first-line fluzoparib maintenance therapy, 92 patients received fluzoparib maintenance therapy after platinum-sensitive recurrence, 23 patients received direct fluzoparib treatment after platinum-sensitive recurrence, 19 patients received direct fluzoparib treatment after platinum-resistant recurrence. The median follow-up durations were 8.5, 8.7, 7.9, and 6.7 months, respectively. The median durations of fluzoparib treatment were 6.7, 4.8, 3.1, and 1.9 months, respectively. The median progression-free survival (PFS) times were not reached during follow-up, 12.6 months, not reached during follow-up, and 4.8 months, respectively. The 1-year PFS rates were 84.1%, 55.0%, 69.8%, and 45.5%, respectively. The remaining 6 patients received other fluzoparib regimens. (4) Among the 224 patients in the prospective dataset, 205 had safety data recorded. Of these, 127 patients (62.0%, 127/205) experienced treatment-related adverse events, with common events including anemia (24.4%, 50/205), thrombocytopenia (21.0%, 43/205), and leukopenia (19.5%, 40/205). Among the 205 patients, 43 (21.0%, 43/205) experienced grade 3 or higher treatment-related adverse events, with common events including anemia (8.3%, 17/205) and thrombocytopenia (8.3%, 17/205).Conclusions:The effectiveness of fuzuloparib in clinical application is generally consistent with other drugs in the same class, with good safety. This study provids new clinical evidence for the treatment of ovarian cancer with fuzuloparib.
3.Risk factors for cutout failure in geriatric intertrochanteric fracture patients after cephalomedullary nail fixation.
You-Liang HAO ; Fang ZHOU ; Hong-Quan JI ; Yun TIAN ; Zhi-Shan ZHANG ; Yan GUO ; Yang LYU ; Zhong-Wei YANG ; Guo-Jin HOU
China Journal of Orthopaedics and Traumatology 2025;38(2):141-147
OBJECTIVE:
To determine risk factors for cutout failure in geriatric intertrochanteric fracture patients after cephalomedullary nail fixation.
METHODS:
A retrospective review of 518 elderly patients who underwent cephalomedullary nail fixation for intertrochanteric fractures between January 2008 and August 2018 was conducted, including 167 males and 351 females, age from 65 to 97 years old. All patients were followed up for at least one year after surgery and divided into a healed group and a cutout group based on whether the hip screw cutout occurred. Among all patients, 10 cases experienced hip screw cutout. The general information, surgical data, and radiological data of the two groups were compared, and risk factors influencing hip screw cutout were analyzed. Propensity score matching was then performed on the cutout group based on gender, age, body mass index(BMI), and American Society of Anesthesiologists(ASA), and 40 patients from the healed group were matched at a ratio of 1∶4. Key risk factors affecting hip screw cutout were further analyzed. Multivariable logistic regression analysis was conducted to evaluate associations between variables and cutout failure.
RESULTS:
There were no statistically significant differences between the healed group and the cutout group in terms of age, gender, BMI, ASA, and AO classification. However, statistically significant differences were observed between the two groups in terms of reduction quality(P=0.003) and tip-apex distance(TAD), P<0.001. Multivariate analysis identified poor reduction quality OR=23.138, 95%CI(2.163, 247.551), P=0.009 and TAD≥25 mm OR=30.538, 95%CI(2.935, 317.770), P=0.004 as independent risk factors for cutout failure.
CONCLUSION
The present study identified poor reduction quality and TAD≥25 mm as factors for cutout failure in geriatric intertrochanteric fractures treated with cephalomedullary nails. Further studies are needed to calculate the optimal TAD for cephalomedullary nails.
Humans
;
Male
;
Female
;
Hip Fractures/surgery*
;
Aged, 80 and over
;
Aged
;
Risk Factors
;
Retrospective Studies
;
Fracture Fixation, Intramedullary/adverse effects*
;
Bone Nails
;
Bone Screws
4.Glucocorticoid Discontinuation in Patients with Rheumatoid Arthritis under Background of Chinese Medicine: Challenges and Potentials Coexist.
Chuan-Hui YAO ; Chi ZHANG ; Meng-Ge SONG ; Cong-Min XIA ; Tian CHANG ; Xie-Li MA ; Wei-Xiang LIU ; Zi-Xia LIU ; Jia-Meng LIU ; Xiao-Po TANG ; Ying LIU ; Jian LIU ; Jiang-Yun PENG ; Dong-Yi HE ; Qing-Chun HUANG ; Ming-Li GAO ; Jian-Ping YU ; Wei LIU ; Jian-Yong ZHANG ; Yue-Lan ZHU ; Xiu-Juan HOU ; Hai-Dong WANG ; Yong-Fei FANG ; Yue WANG ; Yin SU ; Xin-Ping TIAN ; Ai-Ping LYU ; Xun GONG ; Quan JIANG
Chinese journal of integrative medicine 2025;31(7):581-589
OBJECTIVE:
To evaluate the dynamic changes of glucocorticoid (GC) dose and the feasibility of GC discontinuation in rheumatoid arthritis (RA) patients under the background of Chinese medicine (CM).
METHODS:
This multicenter retrospective cohort study included 1,196 RA patients enrolled in the China Rheumatoid Arthritis Registry of Patients with Chinese Medicine (CERTAIN) from September 1, 2019 to December 4, 2023, who initiated GC therapy. Participants were divided into the Western medicine (WM) and integrative medicine (IM, combination of CM and WM) groups based on medication regimen. Follow-up was performed at least every 3 months to assess dynamic changes in GC dose. Changes in GC dose were analyzed by generalized estimator equation, the probability of GC discontinuation was assessed using Kaplan-Meier curve, and predictors of GC discontinuation were analyzed by Cox regression. Patients with <12 months of follow-up were excluded for the sensitivity analysis.
RESULTS:
Among 1,196 patients (85.4% female; median age 56.4 years), 880 (73.6%) received IM. Over a median 12-month follow-up, 34.3% (410 cases) discontinued GC, with significantly higher rates in the IM group (40.8% vs. 16.1% in WM; P<0.05). GC dose declined progressively, with IM patients demonstrating faster reductions (median 3.75 mg vs. 5.00 mg in WM at 12 months; P<0.05). Multivariate Cox analysis identified age <60 years [P<0.001, hazard ratios (HR)=2.142, 95% confidence interval (CI): 1.523-3.012], IM therapy (P=0.001, HR=2.175, 95% CI: 1.369-3.456), baseline GC dose ⩽7.5 mg (P=0.003, HR=1.637, 95% CI: 1.177-2.275), and absence of non-steroidal anti-inflammatory drugs use (P=0.001, HR=2.546, 95% CI: 1.432-4.527) as significant predictors of GC discontinuation. Sensitivity analysis (545 cases) confirmed these findings.
CONCLUSIONS
RA patients receiving CM face difficulties in following guideline-recommended GC discontinuation protocols. IM can promote GC discontinuation and is a promising strategy to reduce GC dependency in RA management. (Trial registration: ClinicalTrials.gov, No. NCT05219214).
Adult
;
Aged
;
Female
;
Humans
;
Male
;
Middle Aged
;
Arthritis, Rheumatoid/drug therapy*
;
Glucocorticoids/therapeutic use*
;
Medicine, Chinese Traditional
;
Retrospective Studies
5.Impact of two DRG performance management approaches on the operations of neurology and neuro-surgery departments
Yongji MENG ; Quan WEN ; Minlan ZHANG ; Linling QIN ; Qin LYU
Modern Hospital 2025;25(2):266-269
Objective To examine the impact of two DRG performance management approaches on the operations of neu-rology and neurosurgery departments.Methods DRG discharge case data were collected from a tertiary hospital in Laibin City between January 2022 and April 2024.The Interrupted Time Series(ITS)was used to analyze the impact of the two types of DRG performance management on financial performance,service capacity and efficiency,patient burden,and profitability of the neurology and neurosurgery departments.Heatmap clustering analysis was employed to compare the changes in disease surplus rates before and after the two management models,and non-parametric tests were conducted to analyze the impact of departmental transfers on hospitalization costs.Results The change in the ITS(Interrupted Time Series)slope coefficient for operational effi-ciency was significant in the neurology department but not in neurosurgery.The change rates of disease surplus in the two depart-ments were classified into five categories,with similar trends observed in diseases with closely related weights.Furthermore,hos-pitalization costs for certain diseases significantly increased following the transfer of patients from one department to the other(P<0.05).Conclusion Significant differences exist in the impact of different DRG(Diagnosis-Related Group)performance management approaches in the same department,and the same DRG performance management approach has varying effects on dif-ferent departments.Departmental transfer is a key factor influencing hospitalization costs.
6.Structural challenges and development pathways of the disease control supervisor system:A SWOT-CLPV integrated analysis
Yan-ling HAN ; Quan WANG ; Si-qi LIU ; Yu-meng LYU ; Yi-xin QIN ; Ying-ming SONG ; Jia-kun WANG ; Li YANG
Chinese Journal of Health Policy 2025;18(6):26-33
Objective:This study applies an integrated SWOT-CLPV framework combined with stakeholder analysis to systematically assess the strengths,weaknesses,opportunities,and threats of China's disease control inspector system,while identifying its control factors,leverage points,key problems,and vulnerabilities.Methods:Drawing on literature review,policy document analysis,and expert interviews with seven public health professionals,we extracted and categorized SWOT elements.A CLPV interaction analysis was conducted alongside stakeholder mapping to evaluate internal dynamics and systemic risks.Results:The inspector system demonstrates strengths in policy innovation and medical-public health integration,with external opportunities stemming from rising public health awareness and digital health advancements.However,the system faces weak endogenous momentum,limited leverage,and prominent control constraints and problem-prone areas,especially among grassroots institutions and inspectors themselves.Cross-sectoral coordination barriers and uneven local implementation contribute to significant institutional vulnerabilities.Conclusion:To enhance implementation and resilience,the system requires capacity building for key actors,improved governance structures,incentive and evaluation reforms,and strengthened coordination mechanisms to support the sustained and adaptive development of public health supervision.
7.Real world clinical data analysis of fuzuloparib for the treatment of ovarian epithelial cancer patients
Danhui WENG ; Jie JIANG ; Yingjie YANG ; Mingqian LU ; Jiaying BAI ; Ming LIU ; Xiaoling LI ; Jun TIAN ; Yutao GUAN ; Quan LI ; Liang CHEN ; Qiubo LYU ; Lixia MA ; Yali WANG ; Huicheng XU ; Hailong GUO ; Li SUN ; Ding MA ; Qinglei GAO
Chinese Journal of Obstetrics and Gynecology 2025;60(8):590-599
Objective:To evaluate the safety and effectiveness of fuzuloparib for the treatment of ovarian epithelial cancer patients in the real world setting.Methods:A retrospective analysis was conducted on the baseline data of 4 620 ovarian cancer patients who had received fuzuloparib monotherapy or combination therapy. Another 224 ovarian cancer patients who were willing to receive fuzuloparib monotherapy or combination therapy were prospectively enrolled, and their baseline characteristics, drug effectiveness, and safety data were analyzed.Results:(1) Among the 4 620 patients in the retrospective cohort, the median age of patients was 60 years; tumor types: 89.8% (4 149/4 620) had ovarian cancer. Among patients with clearly documented information, the vast majority had a histological type of serous carcinoma (82.9%, 3 770/4 546) and International Federation of Gynecology and Obstetrics (FIGO) staging of Ⅲ-Ⅳ (90.9%, 1 537/1 691). (2) Among the 224 patients in the prospective cohort, the median age of patients was 57 years; tumor types: 83.9% (188/224) had ovarian cancer. Among patients with clearly documented records, the predominant pathologic type was serous carcinoma (91.9%, 193/210), and FIGO stage was Ⅲ-Ⅳ in 79.9% (139/174). (3) Among the 224 prospective patients: 84 patients received first-line fluzoparib maintenance therapy, 92 patients received fluzoparib maintenance therapy after platinum-sensitive recurrence, 23 patients received direct fluzoparib treatment after platinum-sensitive recurrence, 19 patients received direct fluzoparib treatment after platinum-resistant recurrence. The median follow-up durations were 8.5, 8.7, 7.9, and 6.7 months, respectively. The median durations of fluzoparib treatment were 6.7, 4.8, 3.1, and 1.9 months, respectively. The median progression-free survival (PFS) times were not reached during follow-up, 12.6 months, not reached during follow-up, and 4.8 months, respectively. The 1-year PFS rates were 84.1%, 55.0%, 69.8%, and 45.5%, respectively. The remaining 6 patients received other fluzoparib regimens. (4) Among the 224 patients in the prospective dataset, 205 had safety data recorded. Of these, 127 patients (62.0%, 127/205) experienced treatment-related adverse events, with common events including anemia (24.4%, 50/205), thrombocytopenia (21.0%, 43/205), and leukopenia (19.5%, 40/205). Among the 205 patients, 43 (21.0%, 43/205) experienced grade 3 or higher treatment-related adverse events, with common events including anemia (8.3%, 17/205) and thrombocytopenia (8.3%, 17/205).Conclusions:The effectiveness of fuzuloparib in clinical application is generally consistent with other drugs in the same class, with good safety. This study provids new clinical evidence for the treatment of ovarian cancer with fuzuloparib.
8.Impact of two DRG performance management approaches on the operations of neurology and neuro-surgery departments
Yongji MENG ; Quan WEN ; Minlan ZHANG ; Linling QIN ; Qin LYU
Modern Hospital 2025;25(2):266-269
Objective To examine the impact of two DRG performance management approaches on the operations of neu-rology and neurosurgery departments.Methods DRG discharge case data were collected from a tertiary hospital in Laibin City between January 2022 and April 2024.The Interrupted Time Series(ITS)was used to analyze the impact of the two types of DRG performance management on financial performance,service capacity and efficiency,patient burden,and profitability of the neurology and neurosurgery departments.Heatmap clustering analysis was employed to compare the changes in disease surplus rates before and after the two management models,and non-parametric tests were conducted to analyze the impact of departmental transfers on hospitalization costs.Results The change in the ITS(Interrupted Time Series)slope coefficient for operational effi-ciency was significant in the neurology department but not in neurosurgery.The change rates of disease surplus in the two depart-ments were classified into five categories,with similar trends observed in diseases with closely related weights.Furthermore,hos-pitalization costs for certain diseases significantly increased following the transfer of patients from one department to the other(P<0.05).Conclusion Significant differences exist in the impact of different DRG(Diagnosis-Related Group)performance management approaches in the same department,and the same DRG performance management approach has varying effects on dif-ferent departments.Departmental transfer is a key factor influencing hospitalization costs.
9.Efficacy Analysis of Stanozolol Combined with Avatrombopag in the Treatment of Chemotherapy-Induced Thrombocytopenia in Relapsed/Refractory Tumors
Yan HE ; Wei-Yi LIU ; Yan-Yu ZHANG ; Yan LYU ; Shan-Shan ZHANG ; Ri-Cheng QUAN
Journal of Experimental Hematology 2025;33(4):1127-1130
Objective:To investigate the efficacy and safety of stanozolol combined with avatrombopag in the treatment of chemotherapy-induced thrombocytopenia(CIT)in patients with relapsed/refractory tumors.Methods:Twenty-five patients with relapsed/refractory CIT admitted to the Hematology Department of Xiyuan Hospital,China Academy of Chinese Medical Sciences between March 2023 to December 2023 were enrolled.These patients received a combined therapy of stanozolol and avatrombopag.The clinical efficacy,onset time,changes in platelet levels and blood cell counts before and after treatment,and adverse reactions of patients were evaluated.Results:The combination therapy demonstrated remarkable efficacy with a total effective rate of 100%.Among the 25 patients,19 achieved complete remission and 6 achieved partial remission.The median onset time was 42.5(range:35-48)days.The average platelet count of the 25 patients increased from(25.73±17.75)× 109/L before treatment to(146.4±49.59)× 109/L after 3 months of treatment,with a statistically significant difference(P<0.05).18 patients who previously required platelet transfusion were all weaned off platelet transfusion after 3 months of treatment,with a median time to be free from platelet transfusion was 26(range:18-51)days.During the treatment,both neutrophils and hemoglobin exhibited various degrees of elevation.Two patients experienced a slight increase in alanine aminotransferase(ALT)levels,which normalized after treatment with oral hepatoprotective drug.One patient had a PLT increase exceeding 350 ×109/L,and the treatment with avatrombopag was suspended,and aspirin and other drugs were given to prevent thrombosis.No thrombose events or CIT-related bleeding events were observed in all patients.Conclusion:The combination therapy of stanozolol and avatrombopag is significantly effective for treating relapsed/refractory CIT patients,with a high response rate and good safety,making it a suitable clinical treatment option.
10.Structural challenges and development pathways of the disease control supervisor system:A SWOT-CLPV integrated analysis
Yan-ling HAN ; Quan WANG ; Si-qi LIU ; Yu-meng LYU ; Yi-xin QIN ; Ying-ming SONG ; Jia-kun WANG ; Li YANG
Chinese Journal of Health Policy 2025;18(6):26-33
Objective:This study applies an integrated SWOT-CLPV framework combined with stakeholder analysis to systematically assess the strengths,weaknesses,opportunities,and threats of China's disease control inspector system,while identifying its control factors,leverage points,key problems,and vulnerabilities.Methods:Drawing on literature review,policy document analysis,and expert interviews with seven public health professionals,we extracted and categorized SWOT elements.A CLPV interaction analysis was conducted alongside stakeholder mapping to evaluate internal dynamics and systemic risks.Results:The inspector system demonstrates strengths in policy innovation and medical-public health integration,with external opportunities stemming from rising public health awareness and digital health advancements.However,the system faces weak endogenous momentum,limited leverage,and prominent control constraints and problem-prone areas,especially among grassroots institutions and inspectors themselves.Cross-sectoral coordination barriers and uneven local implementation contribute to significant institutional vulnerabilities.Conclusion:To enhance implementation and resilience,the system requires capacity building for key actors,improved governance structures,incentive and evaluation reforms,and strengthened coordination mechanisms to support the sustained and adaptive development of public health supervision.

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