QJM: An International Journal of Medicine最新文献

Background: The prophylactic effects of traditional Chinese medicine (TCM) for migraine without aura are uncertain.

Aim: This study aimed to evaluate the efficacy and safety of TCM compared with flunarizine as a prophylactic treatment for episodic migraine in adults.

Design and methods: In this randomized clinical trial (4-week treatment and 12-week follow-up), participants were allocated in a 1:1 ratio to receive either TCM (acupuncture: three sessions/week; Chinese herbal medicine: two packets per day) or flunarizine (10 mg per day). The primary outcome was the mean change from baseline in monthly migraine days (MMDs) during weeks 1-4.

Results and conclusion: Of 343 patients screened, with 212 (mean age, 46.1 [SD 8.9] years; 162 [76.4%] female) were included in the intention-to-treat analyses. Baseline characteristics were cpmparable between groups. The TCM group demonstrated a significantly greater reduction in MMDs than the flunarizine group during both weeks 1-4 and weeks 5-16, with a difference of -0.64 days (95% CI: -1.07 to -0.20; P = 0.004) during weeks 1-4. and -1.13 days (95% CI: -1.48 to -0.78; P < 0.001) during weeks 5-16. These findings indicate that TCM is effective in preventing episodic migraine, with superior efficacy to flunarizine after 4 weeks of treatment, and sustained benefits for 16 weeks. TCM may therefore be considered an optional preventive therapy for episodic migraine without aura. These results support its use in patients unwilling to take, or unresponsive to, prophylactic drugs, and suggest its consideration in future clinical guidelines.

Background: Accurate assessment of liver fibrosis is crucial for patients with autoimmune hepatitis (AIH).

Aim: We developed and validated a non-invasive explainable machine learning (ML) model for the prediction of liver fibrosis in patients with AIH.

Design: A retrospective multicenter study of patients with AIH with liver biopsy was conducted.

Methods: Patients were randomly divided into a training set and a test set. Nine ML models were built, including logistic regression, k-nearest neighbors, Support vector machine, random forest, extreme gradient boosting (XGBoost), gradient boosting, Adaboost, decision tree, and Gaussian naive bayes. The best model was compared with aminotransferase to platelet ratio index (APRI) and fibrosis index based on four factors (FIB-4) on the test set by area under receiver operating characteristic curves (AUC). Shapley additive explanation (SHAP) analysis and local interpretable model-agnostic explanations (LIME) were used for model explanation.

Results: A total of 261 patients with AIH with a median age of 54.0 (interquartile range: 47.0-62.0) years and 82.8% of female sex were included. Among nine ML models, the XGBoost model exhibited superior predictive performance. The model achieved an AUC of 0.791 (95% confidence interval [CI]: 0.668-0.890) in the test set which was higher than APRI (AUC: 0.557, 95% CI: 0.380-0.732, P < 0.001) and FIB-4 (AUC: 0.625, 95% CI: 0.452-0.789, P < 0.001). SHAP and LIME analysis revealed that platelet was the most important predictive variable of significant liver fibrosis.

Conclusions: The non-invasive interpretable XGBoost model surpasses APRI and FIB-4 for predicting significant liver fibrosis, contributing to better management of patients with AIH.

Background: Sarcopenia is a novel complication of type 2 diabetes mellitus (T2DM), the incidence of which is rapidly increasing. Therefore, early detection and diagnosis of diabetic sarcopenia is important to improve the quality of life of patients.

Methods: Differentially expressed genes (DEGs) were identified from Gene Expression Omnibus (GEO) datasets GSE76895 and GSE1428. Gene Ontology (GO) and Kyoto Encyclopedia of Genes and Genomes (KEGG) pathway analyses were conducted using DAVID and KOBAS. Hub gene of metabolic pathways were validated using gene expression and ROC curves. Hub gene expression was then validated in mouse models and human populations, and therapeutic potential was assessed.

Results: The metabolic pathway is an important pathophysiological feature of T2DM and sarcopenia, with 17 genes co-enriched in this pathway. We focused on the PGK1, stained by immunofluorescence and western blot analysis, revealed significantly lower expression of PGK1 in pancreatic islets and skeletal muscles of T2DM mice, with a negative correlation with the diabetic cycle. Similarly, serum levels of PGK1 were lower in T2DM patients than in healthy individuals. Overexpression of PGK1 alleviates metabolic stress and improves skeletal muscle function in diabetic sarcopenia, highlighting its potential as a therapeutic target.

Conclusion: These results suggest that PGK1 may serve as a novel biomarker and potential target for diabetic sarcopenia.

A raised serum ferritin (>300 µg/l in men or >200 µg/l in women) is a common finding during routine investigations in primary care. It is often non-specific since it is both an iron-storage protein and acute-phase reactant. As such, a raised ferritin level should be interpreted primarily as a context-dependent signal of disease activity rather than a direct measure of iron burden. Common causes of hyperferritinaemia include liver pathologies, metabolic syndrome and autoimmune conditions and infection. Hereditary haemochromatosis (HH) is also a cause of elevated ferritin levels but less common compared to the aforementioned causes. Appropriate investigations in someone with abnormally high ferritin levels include transferrin saturation (TSAT) and markers of infection, inflammation and liver function to rule out non-iron-overload causes. HH may be considered if the TSAT is persistently ≥50% for males and ≥40% for females in those with ferritin above the reference range. Any patient with confirmed HH should be offered iron-reduction therapy usually with therapeutic phlebotomy. Haemochromatosis UK and NHS Blood & Transplant have pathways for HH patients to donate venesected blood. Physicians should be aware of such pathways and consider referrals for eligible patients after shared decision-making.

Background: Common cold is one of the most prevalent illnesses affecting human health. Nasal symptoms are the most bothersome and characteristic manifestations, yet few safe and effective treatments are currently available.

Aim: This phase 3, randomised, double-blind, placebo-controlled trial aimed to evaluate the efficacy and safety of bencycloquidium bromide (BCQB) nasal spray in nasal symptoms of common cold.

Methods: Participants with cold symptoms for ≤48 hours and a rhinorrhea visual analogue scale (VAS) score ≥5 were enrolled. Participants were randomized 1:1 to receive either BCQB or placebo for 4 ± 1 days. Patients self-recorded VAS scores for rhinorrhea, sneezing, nasal congestion and itching daily. Efficacy and safety were assessed.

Results: Totals of 480 patients were randomized and treated. The area under the curve (AUC) for rhinorrhea VAS scores over treatment period was significantly lower in the BCQB group than in the placebo group (13.01 ± 7.19 vs. 16.01 ± 8.00; P < 0.001). Daily improvement in the average rhinorrhea VAS score was significantly greater in the BCQB group from day 2 through day 4 (P < 0.005). BCQB also demonstrated significant efficacy for nasal congestion (AUC: 10.90 ± 7.42 vs. 13.63 ± 8.64; P < 0.001) and sneezing (AUC: 7.84 ± 7.18 vs. 9.30 ± 8.64; P = 0.046). The incidence of adverse events (AEs) was similar between the two groups, and no severe AEs were reported. BCQB was well-tolerated, with no systemic anticholinergic adverse effects observed.

Conclusions: BCQB nasal spray is effective and safe for treating rhinorrhea, nasal congestion, and sneezing associated with common cold.

Trial registration: The trial was registered at chictr.org.cn (ChiCTR2500107341).