The construction of a training system for visiting physicians in the department of rare diseases in China is an important measure to improve the overall diagnosis and treatment capacity for rare diseases and address the critical challenge of insufficient knowledge and skills among clinicians in practice. This article systematically describes the visiting physician training system established by the Department of Rare Diseases at Peking Union Medical College Hospital. It summarizes the training objectives and positioning, design logic, and learning modules of the system, aiming to provide a reference for the construction of the specialized talent team for rare diseases in China.

This study aimed to design and synthesize a series of novel photocaged compounds based on the precise binding mode of cyclin-dependent kinase 2 (CDK2) inhibitor AT7519 with its target, to achieve precise optical control over molecular activity. Using AT7519 as the structural foundation, different types of photoremovable protecting groups were introduced at its key interaction sites (the NH of the pyrazole ring and the NH of the piperidine ring), resulting in the synthesis of eight dual-photocaged compounds. Their structures were confirmed by NMR and mass spectrometry, and their photochemical properties—including photolysis wavelength, photolysis time, and photolysis efficiency—were systematically evaluated. The optically controlled inhibitory effects of the compounds on HCT116 cell proliferation were assessed using MTT assay. The photoresponsive characteristics of the photocaged compounds were significantly influenced by the type of photoremovable protecting group and its introduction site. Among them, compounds 1a, 1b, and 2d exhibited excellent photolysis efficiency, each achieving a rate exceeding 90%. Particularly, compounds 1b and 2d demonstrated excellent optically controlled activity: under dark conditions, their half maximal inhibitory concentration (IC50) values were both greater than 150 µmol/L, indicating negligible cell proliferation inhibitory activity; however, upon irradiation, their cell proliferation inhibitory activity significantly increased, with IC50 values comparable to those of the positive control AT7519. The obtained compounds 1b and 2d possess excellent optically controlled properties, and can be further investigated as potential candidates for optically controlled CDK2 inhibitors.

Objective: To evaluate the efficacy of the left spermatic vein transposition to the great saphenous vein in treating left varicocele (VC) secondary to nutcracker syndrome (NCS). Methods: Clinical data of 8 patients treated during Feb.2020 and Feb.2023 in our hospital were retrospectively analyzed.A meticulous preoperative evaluation of the vascular status of the spermatic vein and the great saphenous vein was performed using color Doppler ultrasound.A spermatic vein-great saphenous vein shunt surgery was performed in patients who were strictly selected.The clinical symptoms and hemodynamics of renal vein were compared before and after operation. Results: The median age of patients was 23.5(18-33) years.There was a notable reduction in post-exercise scrotal and lower back pain in all patients，and the score of scrotal pain decreased to 0 in 7 patients. The median quantification of urinary protein was 352.8(54.4-687.3) mg prior to surgical intervention，which significantly diminished to 125.5(25.9-255.1) mg 6 months after operation.Notably，3 cases of preoperative positive urine occult blood tests were undetectable in the subsequent postoperative assessments.The median peak blood flow velocity at the site of stenosis in the left renal vein measured at 74.4(48.7-117.6) cm/s preoperatively，subsequently reduced to 45.1(25.5-61.2) cm/s postoperatively.During the 6-month follow-up，no recurrence of varicocele，vascular anastomotic stenosis or thrombosis were observed. Conclusion: Our research indicates that spermatic vein to great saphenous vein bypass is safe and feasible in the treatment of left varicocele secondary to nutcracker syndrome for strictly selected patients，which can effectively alleviate renal vein congestion without significant complications.

ObjectiveTo analyze the prevalence and influencing factors of overweight and obesity among primary school students in a community of Fengxian District, Shanghai, and to provide references for formulating prevention and control strategies against overweight and obesity. MethodsData on height and weight of all primary school students in a community in Fengxian District, Shanghai, in 2023 were obtained by physical examination, and 1 759 primary school students were included according to the entry criteria. Overweight and obesity were determined using body mass index (BMI). Additionally, a questionnaire survey was performed to 1 045 students to collect their demographic characteristics, dietary behaviors, dietary habits, sleep and physical activity information. Chi-square test and logistic regression analysis were used to analyze the influencing factors of overweight and obesity. ResultsIn 2023, among the 1 759 primary school students in the community in Fengxian District, 923 (52.47%) were male and 836 (47.53%) were female, with an overweight/obesity detection rate of 28.08%. The detection rate of overweight and obesity was 33.37% in males and 22.25% in females, which was significantly higher in males than that in females (χ²=26.845, P<0.001). Students aged 10‒12 years had a higher overweight/obesity detection rate (32.55%) than those aged 6‒<10 years (26.53%), and the difference was statistically significant (χ²=10.925, P<0.001). Logistic regression analysis revealed that being female, with young age, parental education level of bachelor’s degree and above, a high global dietary recommendation healthy (GDR-healthy) score, preference for vegetables and sweeter home-cooked meals, and a slow eating speed were negatively correlated with overweight/obesity. Whereas, parental overweight and obesity, binge eating, and a faster eating pace than same-age, same-gender peers may be positively correlated with overweight/obesity. ConclusionThe detection rate of overweight and obesity among primary school students in the community in Fengxian District of Shanghai is higher than the national level. Gender, age, parental BMI, parental education level, dietary behaviors and habits are the main influencing factors of overweight/obesity among primary school students.

BACKGROUND: Generalized pustular psoriasis (GPP), a rare and recurrent autoinflammatory disease, imposes a substantial burden on patients and society. Awareness of GPP in China remains limited. METHODS: This cross-sectional survey, conducted between September 2021 and May 2023 across 14 hospitals in China, included GPP patients of all ages and disease phases. Data collected encompassed demographics, clinical characteristics, economic impact, disease severity, quality of life, and treatment-related complications. Risk factors for GPP recurrence were analyzed. RESULTS: Among 127 patients (female/male ratio = 1.35:1), the mean age of disease onset was 25 years (1st quartile [Q1]-3rd quartile [Q3]: 11-44 years); 29.2% had experienced GPP for more than 10 years. Recurrence occurred in 75.6% of patients, and nearly half reported no identifiable triggers. Younger age at disease onset ( P  = 0.021) and transitioning to plaque psoriasis ( P  = 0.022) were associated with higher recurrence rates. The median diagnostic delay was 8 months (Q1-Q3: 2-41 months), and 32.3% of patients reported misdiagnoses. Comorbidities were present in 53.5% of patients, whereas 51.1% experienced systemic complications during treatment. Depression and anxiety affected 84.5% and 95.6% of patients, respectively. During GPP flares, the median Dermatology Life Quality Index score was 19.0 (Q1-Q3: 13.0-23.5). This score showed significant differences between patients with and without systemic symptoms; it demonstrated correlations with both depression and anxiety scores. Treatment costs caused financial hardship in 55.9% of patients, underscoring the burden associated with GPP. CONCLUSIONS The substantial disease and economic burdens among Chinese GPP patients warrant increased attention. Patients with early onset disease and those transitioning to plaque psoriasis require targeted interventions to mitigate the high recurrence risk.
Humans ; Male ; Female ; Psoriasis/pathology* ; Adult ; Cross-Sectional Studies ; Adolescent ; Child ; Young Adult ; Quality of Life ; Middle Aged ; China/epidemiology* ; Recurrence ; Risk Factors ; Surveys and Questionnaires ; East Asian People

Thyroid diseases are clinically prevalent endocrine and metabolic disorders, including various types such as Hashimoto's thyroiditis and thyroid nodules. Their pathogenesis is complex and influenced by numerous factors, potentially involving genetic factors, environmental factors, and immune dysfunction. With the continuous advancement of modern traditional Chinese medicine (TCM), TCM has established a unique theoretical system in the prevention and treatment of thyroid diseases, with increasingly rich clinical practical experience. This article reviews the recent application of TCM health status analysis, monitoring, early warning, and prevention in the field of thyroid diseases, aiming to provide useful references for in-depth study in this field.

Objective:To identify factors influencing treatment-free remission (TFR) outcomes in children and adolescent patients with chronic myeloid leukemia (CML) after imatinib (IM) discontinuation.Methods:This multicenter retrospective study analyzed 36 children and adolescent patients with CML from eight hematology centers in China (December 1, 2016, to September 27, 2024) who discontinued IM therapy with documented post-cessation outcomes. Clinical characteristics and molecular response dynamics were assessed. Univariate analysis and multivariate Cox proportional hazards regression models were employed to assess factors associated with TFR outcomes.Results:A total of 36 patients were documented, comprising 17 males and 19 females. The median ages at CML diagnosis and IM discontinuation were 11 years ( IQR: 5,16) and 20 years ( IQR: 14,25), respectively. The median time from IM initiation to first deep molecular response (DMR) was 21 months ( IQR: 13, 38). Pre-discontinuation, patients received IM for a median duration of 96 months ( IQR: 84, 121) and maintained DMR for 74 months ( IQR: 63, 89). With a median post-discontinuation follow-up of 38 months ( IQR: 15, 68), cumulative TFR rates at 6, 12, 24, and 36 months were 74.1%, 60.7%, 60.7%, and 56.0%, respectively, generating an overall TFR rate of 58.3%. Fifteen patients lost major molecular response at a median of 5 months post-discontinuation ( IQR: 3, 11). All 15 patients resumed tyrosine kinase inhibitor therapy, comprising 13 who restarted IM and 2 who switched to dasatinib. By the last follow-up, 13 (86.7% ) patients regained DMR after a median treatment duration of 5 months ( IQR: 3, 17), and no disease progression occurred in any patient. Withdrawal syndrome occurred in 2 (5.6% ) patients. Univariate analysis revealed significantly higher TFR rates in patients with pre-discontinuation IM duration of ≥100 months vs <100 months (82.4% vs 36.8%, P=0.017) and pre-discontinuation DMR duration of ≥72 months vs <72 months (84.2% vs 29.4%, P=0.003). Multivariate Cox analysis identified pre-discontinuation DMR duration as an independent protective factor for TFR ( HR=5.419, 95% CI: 1.524–19.272, P=0.009) . Conclusion:DMR duration was identified as an independent protective factor influencing TFR outcomes in children and adolescent patients with CML after IM discontinuation. Patients who maintained DMR for ≥72 months before IM discontinuation demonstrated a significantly higher TFR rate.

Objective:To explore the impact of anterior cruciate ligament(ACL) augmented repair versus autologous hamstring tendon single-bundle reconstruction on long-term joint function and radiological outcomes in patients with ACL tear.Methods:A prospective study was conducted, including medical records of 100 patients with anterior cruciate ligament (ACL) tears admitted to the 983rd Hospital of the Joint Logistic Support Force of the Chinese People′s Liberation Army from January 2020 to January 2023. There were 68 males and 32 females, who were aged from 35 to 63 years old, with a mean age of (44.73±6.31) years. The lesion was on the left side in 41 cases and on the right side in 59 cases. According to the Sherman classification, 43 cases were type Ⅰ and 57 cases were type Ⅱ. Patients were sequentially numbered according to admission order, and a random integer generator was used to assign random numbers (1 or 2). Patients assigned "1" were included in the Group 1, while those assigned "2" were allocated to the Group 2, with 50 cases in each group.The Group 1 received ACL augmented repair, while the Group 2 was treated with autologous hamstring tendon single-bundle reconstruction. After treatment, the two groups were compared in terms of knee joint function[International Knee Documentation Committee (IKDC) score], motor function(Tegner score), knee joint stability(pivot shift test) at 12 and 24 months after surgery; the incidence of active pain[visual analog scale (VAS) score] at 1-3 months, 4-6 months, 7-12 months, and 13~24 months after surgery; radiological outcomes; and the rate of secondary revision. Measurement data with normal distribution were expressed as mean ± standard deviation( ± s), and comparisons between the two groups were performed using t-test. Comparisons among multiple time points within the group were conducted using repeated measures analysis of variance. Count data were presented as cases and percentages [ n(%)], and comparisons between groups were made using the chi-square test. Ranked data were analyzed using the Wilcoxon signed-rank test. Results:As of the last follow-up, a total of 45 patients were included in Group 1 (the repair group), and 48 patients were included in Group 2 (the reconstruction group). At 12 and 24 months postoperatively, the IKDC scores in the repair group showed significant improvements compared to preoperative values ( t =11.29, P<0.05; t=14.86, P<0.05), as did the Tegner scores ( t=36.13, P<0.05; t=52.21, P<0.05). Additionally, pivot-shift test results demonstrated significant improvement ( χ2=79.33, P<0.05; χ2=90.00, P<0.05). Similarly, in the reconstruction group, both IKDC scores ( t=10.02, P<0.05; t=13.73, P<0.05) and Tegner scores ( t=44.87, P<0.05; t=60.65, P<0.05) significantly increased at 12 and 24 months postoperatively, with pivot-shift test results also showing marked improvement ( χ2=89.33, P<0.05; χ2=96.00, P<0.05). However, no statistically significant differences were observed between the repair and reconstruction groups in Tegner scores or pivot-shift test results at either time point. However, no statistically significant differences were observed between the two groups for any outcome at either time point (IKDC: t=0.93, P>0.05 at 12 months; t=0.75, P>0.05 at 24 months; Tegner: t=1.52, P>0.05; t=0.77, P>0.05; pivot-shift: χ2=1.16, P>0.05; χ2=1.34, P>0.05). The duration of postoperative activity-related pain was significantly shorter in the repair group (3.24±0.61 months) compared to the reconstruction group (4.48±0.59 months) ( t=9.95, P<0.05). During postoperative months 1-3 and 4-6, pain levels were also significantly lower in the repair group ( t=5.32, P<0.05; t=8.71, P<0.05). However, no statistically significant differences in pain levels were observed between groups at postoperative 7th to 12th months ( t=0.40, P>0.05) and 13th to 24th months ( t=0.37, P>0.05). At 24th months postoperatively, the majority of patients in both groups exhibited ligament thicknesses approaching normal levels, with fibro-osseous bridging between the ligament and bone tunnel walls. The revision rates were 2.2% (1/45) in the repair group and 2.1% (1/48) in the reconstruction group, with no statistically significant difference between groups ( χ2=0.00, P>0.05). Conclusions:The effects of ACL augmented repair for treating ACL tears on patients′ knee joint function, knee joint stability, motor function, and ligament healing are comparable to those of autologous hamstring tendon single-bundle reconstruction. However, ACL augmented repair can reduce the incidence of early active pain in patients.