1.Analysis of follow-up and prognosis in pediatric rheumatic diseases associated with pulmonary embolism
Tong YUE ; Yuchun YAN ; Min KANG ; Jia ZHU ; Yingjie XU ; Dan ZHANG ; Ming LI ; Min WEN ; Feifei WU ; Jianming LAI
Chinese Journal of Pediatrics 2026;64(1):89-94
Objective:To explore the clinical characteristics, diagnosis and treatment strategies, and prognosis of pulmonary embolism (PE) complicating childhood rheumatic diseases.Methods:A retrospective case series study was performed on the demographic data, laboratory indicators, imaging features, treatment regimens, and follow-up data of 8 children with rheumatic diseases complicated by PE who were admitted to the Department of Rheumatology and Immunology, Capital Center for Children′s Health, Capital Medical University from January 2014 to October 2023.Results:Among the 8 children, there were 4 boys and 4 girls, with an age of 12.0 (7.5, 13.0) years. Among the primary diseases, there were 3 cases of systemic lupus erythematosus, 2 cases of Beh?et′s disease, 2 cases of Takayasu arteritis, and 1 case of antiphospholipid syndrome. All children developed PE during the active phase of the primary disease. PE was detected at the onset of the primary disease in 3 cases, and the median time from the diagnosis of the primary disease to the development of PE was 10.0 (6.0, 25.0) months in the remaining 5 cases. Fever was present in all 8 children, 4 cases were accompanied by chest tightness, dyspnea, etc., and 2 cases only presented with fever. Laboratory examinations revealed the following results: erythrocyte sedimentation rate was 42.0 (17.0, 78.0) mm/1 h, high-sensitivity C-reactive protein was 12.7 (2.6, 78.7) mg/L, white blood cell count was 9.6 (7.2, 18.7)×10 9/L; D-dimer was 2.3 (0.9, 6.2) mg/L; and hemoglobin was (109±16) g/L.Imaging examinations revealed that 5 cases had involvement of the bilateral lower pulmonary arteries, 5 cases had peripheral embolism, and 3 cases had central PE. Complications included 3 cases of deep vein thrombosis, 2 cases of intracranial venous sinus thrombosis, and 1 case of mild pulmonary hypertension.In terms of treatment, 7 cases received anticoagulation with heparin followed by warfarin. Immunomodulation was mainly based on glucocorticoids combined with immunosuppressants, and 4 cases were combined with biological agents. The follow-up time of 4.17 (1.75, 7.17) years, the time for complete absorption of PE was 10.5 (6.0, 18.0) months; all 8 children had no target events, with no recurrence or chronic thromboembolic pulmonary hypertension, and the pulmonary artery remodeling was good. Conclusions:PE complicating childhood rheumatic diseases is closely related to the activity of the primary disease. The clinical manifestations are insidious, with fever as the main symptom. Imaging examination is the key to diagnosis.Early adoption of heparin followed by warfarin anticoagulation and glucocorticoids combined with immunosuppressants and (or) biological agents to control the primary disease can achieve a favorable prognosis.
2.Clinical features and molecular mechanism of infantile cholestasis caused by IFT122 gene variants
Jiaqi LI ; Yan LI ; Ruida HE ; Jiayan FENG ; Jianshe WANG
Chinese Journal of Pediatrics 2026;64(1):99-105
Objective:To investigate the clinical characteristics of infantile cholestasis caused by IFT122 gene variants and the molecular mechanism underlying its impact on primary cilia.Methods:The clinical data of an infant with cholestasis from the Children′s Hospital of Fudan University in September 2022 were retrospectively analyzed. The whole-exome sequencing was performed to identify candidate variants, which were validated by Sanger sequencing in the family. Immortalized cell lines were generated using lentiviral infection, followed by immunofluorescence staining to assess the impact of the variants on primary cilia. Intergroup comparisons were performed using the independent sample t-test and Mann-Whitney U test .Results:The proband was a 4-month-old male infant presenting with jaundice, distinctive facial features, and sagittal craniosynostosis. Blood biochemistry indicated elevated direct bilirubin, total bile acids, and transaminases, with markedly increased γ-glutamyltransferase (GGT). Liver pathology demonstrated giant cell hepatitis with cholestasis and bile duct dysplasia. Genetic analysis identified compound heterozygous variants in IFT122 (NM_052989.3) gene c.88G>C (p.Ala30Pro) and c.240G>C (p.Trp80Cys), which co-segregated with the disease in the family. Immunofluorescence analysis demonstrated that the IFT122 gene compound heterozygous missense variants not only significantly reduced the proportion of cilia-positive cells but also led to aberrant ciliary localization of ADP-ribosylation factor-like protein 13B (ARL13B).In addition, ciliary deposition with phosphatidylinositol polyphosphate 5-phosphatase type Ⅳ (INPP5E) was reduced. All differences were statistically significant (all P<0.05). Conclusion:The compound heterozygous missense variants in IFT122 gene not only impair ciliogenesis but also disrupt the ciliary localization of ARL13B and INPP5E, ultimately resulting in high-GGT infantile cholestasis.
3.Expert Consensus on Clinical Application of Qidong Yixin Oral Liquid
Changkuan FU ; Xiaochang MA ; Mingjun ZHU ; Yue DENG ; Hongxu LIU ; Mingxue ZHANG ; Ying CHEN ; Yan ZHOU ; Ling ZHANG ; Jianhua FU ; Wei YANG ; Yu'er HU ; Ming CHEN ; Yanming XIE ; Yuanyuan LI
Chinese Journal of Experimental Traditional Medical Formulae 2026;32(4):147-158
The prescription of Qidong Yixin oral liquid is derived from the experience of national medical master Ren Jixue in treating viral myocarditis (VMC). It has the functions of tonifying Qi, nourishing the heart,calming the mind, and relieving palpitations. It is used to treat VMC and angina pectoris of coronary heart disease caused by deficiency of both Qi and Yin. However,the understanding of its efficacy evidence, advantageous aspects, dosage and administration, and medication safety remains insufficient in clinical practice. Therefore,the development of the Expert Consensus on the Clinical Application of Qidong Yixin Oral Liquid (hereinafter referred to as consensus) was initiated. Consensus strictly followed the process and methods of the expert consensus on the clinical application of Chinese patent medicines of the China Association of Chinese Medicine,successively completing multiple tasks such as the consensus project initiation,determination of clinical problems,evidence search and evaluation,formation of recommendation opinions and consensus suggestions,solicitation of opinions,peer review, submission for review and release, and so on. Consensus formed a total of 10 recommendation opinions and 12 consensus suggestions,clarifying the clinical positioning,efficacy advantages,syndrome differentiation,dosage and administration,combination therapy,timing of medication,adverse reactions,contraindications, and precautions of Qidong Yixin oral liquid,indicating that it has good clinical advantages and safety in the treatment of VMC and angina pectoris of coronary heart disease,providing norms and references for physicians to safely and rationally apply Qidong Yixin oral liquid. Consensus was reviewed and approved for release by the Standardization Office of the China Association of Chinese Medicine on December 23, 2024. Standard number:GSCACM-376-2024.
4.Active Ingredients of Traditional Chinese Medicine Regulate Ferroptosis to Inhibit Breast Cancer: A Review
Yan LIU ; Zhenyao YANG ; Chengzhi WANG ; Jiuxian LI
Chinese Journal of Experimental Traditional Medical Formulae 2026;32(4):299-306
Breast cancer (BC), a common malignant tumor in women, is characterized by high incidence and mortality rates, posing a serious threat to women's life and health. Currently, the commonly used treatments for BC include surgery, radiotherapy, chemotherapy, molecular targeted therapy, and endocrine therapy. Although radiotherapy and chemotherapy can effectively kill tumor cells and inhibit the proliferation and differentiation of tumor cells, they can induce adverse reactions such as hematopoietic dysfunction and impaired immune function. The other treatment methods also have problems such as drug resistance, high recurrence rates, reduced quality of life, and poor clinical efficacy. Therefore, it is urgent to explore new drugs with better efficacy and lower toxicity. Ferroptosis is a form of iron-dependent, non-apoptotic programmed cell death triggered by lipid peroxidation. In recent years, ferroptosis has become a hot topic in the field of cancer treatment and has been gradually proven to effectively inhibit the proliferation and metastasis of BC cells, reduce the drug resistance of BC to chemotherapy drugs, and enhance the sensitivity of BC to radiotherapy. Traditional Chinese medicine (TCM), with multiple components, multiple targets, and mild side effects, is widely used in the treatment of BC. A large number of studies have shown that active ingredients of TCM, such as saponins, flavonoids, terpenoids, phenols, and polysaccharides, can inhibit the proliferation and metastasis of BC cells by modulating ferroptosis-related pathways. These include iron metabolism, lipid metabolism, cystine/glutamate antiporter system Xc-/glutathione/glutathione peroxidase 4, Specifically, these ingredients elevate the levels of lipid peroxidation, reactive oxygen species, malondialdehyde, and Fe2+ in BC cells, thereby inducing ferroptosis-mediated suppression of tumor progression. This article reviews the relevant literature at home and abroad in recent years, summarizes the mechanisms of ferroptosis in regulating BC and the research progress in the active components of TCM targeting ferroptosis in the intervention of BC, aiming to provide ideas for the development of new drugs for the treatment of BC.
5.Altered Lymphocyte Subsets in Perioperative Cancer Patients Before and After Septic Shock: Characteristics and Prognostic Implications
Miao WEI ; Lili YANG ; Xiaoyan LI ; Huifang LYU ; Yan DUAN
Medical Journal of Peking Union Medical College Hospital 2026;17(1):86-97
To investigate the changes in peripheral blood immune cells before and after the onset of septic shock in patients with malignant tumors, and to analyze the relationship between these immune cells and patient prognosis. A retrospective study was conducted, enrolling perioperative tumor patients who were transferred to the intensive care unit (ICU) due to septic shock at Shanxi Provincial Cancer Hospital between October 2018 and December 2019.Changes in lymphocyte counts and subsets were compared before and after septic shock (measured prior to septic shock onset and within 72 hours after onset).A multivariate Logistic regression model was used to analyze the relationship between these immune indicators and the 28-day mortality risk in tumor patients following septic shock. A total of 47 tumor patients transferred to the ICU due to septic shock were included.There were 32 males and 15 females, with a mean age of (63.9±11.2) years.Gastrointestinal tumors were the most common tumor type (76.60%, 36/47), and abdominal/pelvic infection (65.96%, 31/47) was the primary source of infection.Within 28 days after ICU transfer, 12 patients died and 35 survived. Compared to pre-septic shock levels, lymphocyte counts significantly decreased after septic shock[530(300, 830) cells/μL Perioperative tumor patients experience acute depletion of peripheral blood lymphocyte subsets following septic shock.Among various immune indicators, regulatory T cell count serves as an independent predictor of short-term mortality risk.Evaluating baseline immune function in such patients may help optimize treatment strategies and improve overall prognosis.
6.Explainable Machine Learning Model for Predicting Prognosis in Patients with Malignant Tumors Complicated by Acute Respiratory Failure: Based on the eICU Collaborative Research Database in the United States
Zihan NAN ; Linan HAN ; Suwei LI ; Ziyi ZHU ; Qinqin ZHU ; Yan DUAN ; Xiaoting WANG ; Lixia LIU
Medical Journal of Peking Union Medical College Hospital 2026;17(1):98-108
To develop and validate a model for predicting intensive care unit (ICU) mortality risk in patients with malignant tumors complicated by acute respiratory failure (ARF) based on an explainable machine learning framework. Clinical data of patients with malignant tumors and ARF were extracted from the eICU Collaborative Research Database in the United States, including demographic characteristics, comorbidities, vital signs, laboratory test indicators, and major interventions within the first 24 hours after ICU admission.The study outcome was ICU death.Enrolled patients were randomly divided into a training set and a validation set at a ratio of 7:3.Predictor variables were selected using least absolute shrinkage and selection operator (LASSO) regression.Five machine learning algorithms-extreme gradient boosting (XGBoost), support vector machine (SVM), Logistic regression, multilayer perceptron (MLP), and C5.0 Decision Tree-were employed to construct predictive models.Model performance was evaluated based on the area under the receiver operating characteristic curve (AUC), accuracy, sensitivity, and other metrics.The optimal model was further interpreted using the Shapley additive explanations (SHAP) algorithm. A total of 3196 patients with malignant tumors complicated by ARF were included.The training set comprised 2, 261 patients and the validation set 935 patients; 683 patients died during ICU stay, while 2513 survived.LASSO regression ultimately selected 12 variables closely associated with patient ICU outcomes, including sepsis comorbidity, use of vasoactive drugs, and within the first 24 hours after ICU admission: minimum mean arterial pressure, maximum heart rate, maximum respiratory rate, minimum oxygen saturation, minimum serum bicarbonate, minimum blood urea nitrogen, maximum white blood cell count, maximum mean corpuscular volume, maximum serum potassium, and maximum blood glucose.After model evaluation, the XGBoost model demonstrated the best performance.The AUCs for predicting ICU mortality risk in the training and validation sets were 0.940 and 0.763, respectively; accuracy was 88.3% and 81.2%;sensitivity was 98.5% and 95.9%.Its predictive performance also remained optimal in sensitivity analyses.SHAP analysis indicated that the top five variables contributing to the model's predictions were minimum oxygen saturation, minimum serum bicarbonate, minimum mean arterial pressure, use of vasoactive drugs, and maximum white blood cell count. This study successfully developed a mortality risk prediction model for ICU patients with malignant tumors complicated by ARF based on a large-scale dataset and performed explainability analysis.The model aids clinicians in early identification of high-risk patients and implementing individualized interventions.
7.Cost-utility analysis of anlotinib combined with penpulimab in first-line treatment of unresectable hepatocellular carcinoma
Wenying YAN ; Na YANG ; Ranran ZHANG ; Xinyue TAO ; Shengnan GAO ; Guoqiang LIU
China Pharmacy 2026;37(3):344-349
OBJECTIVE To evaluate the cost-effectiveness of anlotinib combined with penpulimab versus sorafenib as first- line treatment for unresectable hepatocellular carcinoma (uHCC) from the perspective of China’s healthcare system. METHODS Based on data from the APOLLO study, a partitioned survival model was established with a 21-day model cycle to simulate patient survival status over 10 years under anlotinib combined with penpulimab regimen or sorafenib monotherapy. Quality-adjusted life year (QALY) was used as the core evaluation parameter to assess the incremental cost-effectiveness ratio (ICER) of different treatment regimens. Using 3 times China’s per capita gross domestic product (GDP) in 2024 (287 247 yuan/QALY) as the willingness-to-pay (WTP) threshold, cost-utility analysis was performed to evaluate the cost-effectiveness of the treatment regimens. Sensitivity analysis was conducted to validate the robustness of the baseline analysis conclusion. Scenario analysis was performed to consider the impact of anlotinib and penpulimab assistance programs on the results; the price reduction of penpulimab to ensure the cost-effectiveness of the combination regimen was examined under varying WTP thresholds (specifically, 1, 2, and 3 times China’s per capita GDP in 2024). RESULTS The baseline analysis revealed that the ICER of anlotinib combined with penpulimab regimen relative to the sorafenib regimen was 338 611.20 yuan/QALY, which exceeded the WTP threshold set in this study. Univariate sensitivity analysis indicated that the utility value of progression free survival and penpulimab price significantly influenced the baseline analysis results. Probabilistic sensitivity analysis validated the robustness of the baseline results. The results of scenario analysis indicated that when considering the assistance programs for anlotinib and penpulimab, the obtained ICER values were all below the WTP threshold set at 3 times China’s per capita GDP in 2024. When the price of penpulimab was reduced by 58%, 35%, and 13%, the ICER values were below the WTP threshold, which was 1, 2 and 3 times the per capita GDP of China in 2024, respectively. CONCLUSIONS From the perspective of China’s healthcare system, anlotinib combined with penpulimab regimen for first-line treatment of uHCC lacks cost-effectiveness compared to sorafenib regimen. However, this conclusion would be reversed if the anlotinib and penpulimab assistance programs are taken into account or if the price of penpulimab is reduced by more than 13% and above.
8.Network meta-analysis of the efficacy and safety of immune checkpoint inhibitors in first-line treatment of advanced gastric cancer
Liyuan KE ; Yan WANG ; Anping WANG ; Danxue HUANG
China Pharmacy 2026;37(3):383-388
OBJECTIVE To evaluate the efficacy and safety of immune checkpoint inhibitors (ICIs) as first-line therapy for advanced gastric cancer. METHODS PubMed, Web of Science, Embase, The Cochrane Library, Wanfang Data, CNKI, and VIP databases were searched to collect phase Ⅲ clinical randomized controlled trials (RCTs) on ICIs as first-line therapy for advanced gastric cancer, as well as abstracts from relevant oncology academic conferences. The search period spanned from database inception to June 1, 2025. After screening literature, extracting data, and assessing quality, a network meta-analysis was performed using R software version 4.3.2. RESULTS A total of 8 studies involving 7 801 patients were included. Network meta-analysis results showed that, in terms of efficacy, compared with chemotherapy (Chemo), SHR-1701_Chemo, Cadonilimab_Chemo, Sintilimab_Chemo, Pembrolizumab_Chemo, and Tislelizumab_Chemo significantly prolonged median overall survival (OS) and median progression free survival (PFS) in patients (P<0.05); whereas Nivolumab_Chemo only significantly improved median PFS (P<0.05). Surface under the cumulative ranking curve (SUCRA) results indicated that the top 2 interventions for median OS were SHR-1701_Chemo and Cadonilimab_Chemo; for PFS, the top 2 were Cadonilimab_Chemo and SHR-1701_Chemo. For patients with combined positive score (CPS) ≥5 points for programmed death-ligand 1 (PD-L1), Cadonilimab_Chemo and SHR- 1701_Chemo also demonstrated the optimal OS and PFS benefits (P<0.05). Regarding safety, there were no statistically significant differences among the interventions in the incidence of any adverse events (AEs) or grade ≥3 AEs (P>0.05). The SUCRA ranking for the incidence of any AEs showed the top 2 were SHR-1701_Chemo and Chemo; for grade ≥3 AEs, the top 2 were Chemo and Sugemalimab_Chemo. CONCLUSIONS For patients with advanced gastric cancer, Cadonilimab_Chemo and SHR-1701_Chemo demonstrate the best benefits in terms of OS and PFS, with their advantages remaining clear in patients with PD-L1 CPS≥5 points. In terms of safety, the risk of developing any AEs and grade ≥3 AEs is relatively lowest with Chemo.
9.Research progress on the intervention of human umbilical cord mesenchymal stem cell in neurodegenerative disease
Hongcai XU ; Yumin XU ; Shiyu LIU ; Huayu YAN ; Yuan LIU ; Xin YANG ; Yabo WU
China Pharmacy 2026;37(3):395-400
Human umbilical cord mesenchymal stem cell (hUC-MSC) as a cell-based therapeutic strategy have demonstrated significant application potential in the field of intervention for neurodegenerative disease (NDD) due to their advantages such as self-renewal, multi-directional differentiation, and low immunogenicity. hUC-MSC effectively intervenes in the pathological features and neurological functions of various disease models such as Alzheimer disease, Parkinson’s disease, amyotrophic lateral sclerosis, and multiple sclerosis primarily through multiple mechanisms such as homing and differentiation, mediating paracrine actions and releasing exosomes, as well as immune regulation and anti-inflammation. Some clinical studies have also preliminarily verified their safety and effectiveness. Currently, its research still faces challenges such as immune rejection reactions requiring further observation, long-term safety needing evaluation, mechanisms of action not being fully elucidated, and slow progress in clinical trials. Future research needs to establish pharmaceutical standards for hUC-MSC, deepen their pharmacological mechanisms and clinical trials, ultimately providing new and effective drug treatment options for patients with NDD.
10.DING Ying's Clinical Experience in Treating Systemic Lupus Erythematosus Based on Differentiation of Yin Fire,Latent Fire,and Fire Toxin
Hongji WU ; Min GAO ; Yan XU ; Xuejun LI ; Jiexin SU ;
Journal of Traditional Chinese Medicine 2026;67(4):365-369
This paper summarizes Professor DING Ying's clinical experience in the treatment of systemic lupus erythematosus (SLE) through differentiation of three states, yin fire, latent fire, and fire toxin. It is proposed that fire pathogenic factors constitute a key pathological element running throughout the entire disease course of SLE. The evolution of its pathogenesis centers on these three states, spleen-kidney deficiency with the initial emergence of yin fire as the onset of disease, damage to yin by medicinal toxicity with internal blazing of latent fire as the driver of disease progression, and the interlocking of blood stasis and heat with intense scorching by fire toxin as the critical factor leading to severe and life-threatening conditions. Corresponding to these three stages, targeted prescriptions are formulated, Jiuwei Yishen Formulation (九味益肾方) to tonify the spleen and kidney, raise yang, and disperse fire; Ziyin Xiehuo Decoction (滋阴泄火汤) to nourish yin and fluids while clearing latent fire; and Santeng Changluo Jiedu Decoction (三藤畅络解毒汤) to dispel blood stasis, unblock the collaterals, detoxify, and restrain fire. This staged and integrated therapeutic strategy aims to address both root and branch and to achieve overall regulation, providing valuable guidance for the clinical differentiation and treatment of SLE.

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