ObjectiveTo explore the process and guidelines for ethical review in decentralized drug clinical trials， promote clinical trial progress， and ensure drug development progress. MethodsThe key points of the ethical review were summarized by studying the relevant laws and regulations on decentralized drug clinical trials， analyzing the advantages and challenges of decentralized drug clinical trials， and combining the experience of the ethics committee of the institution in reviewing decentralized drug clinical trials. ResultsRelevant laws and regulations were the basis for the ethical review， and the ethics committee should adopt appropriate review methods based on regulations and hospital ethical standard operating procedures. The ethics committee should focus on the feasibility， applicability， and rationality， the adequacy of informed consent， the protection of rights and interests and privacy of subjects， as well as the qualification and standard operating procedures of electronic platforms for conducting decentralized drug clinical trials. ConclusionDecentralized drug clinical trials are in their early stages and urgently require guidance from relevant laws and regulations. Ethical review is also constantly being refined through exploration. It is necessary to supervise the implementation of responsibilities by all parties， pay attention to the rights and interests of subjects， and gradually promote the implementation of decentralized drug clinical trials.

AIM:To evaluate the clinical efficacy of lamellar keratectomy and corneal collagen crosslinking(LKCCC)in treating superficial fungal keratitis.METHODS: Retrospective analysis. Totally 79 patients(79 eyes)with superficial fungal keratitis who underwent LKCCC in our hospital from January 2014 to October 2023 were included. After admission, routine antifungal drug treatment for 7 d showed no obvious improvement or progressive aggravation. The maximum diameter of corneal lesions in all patients was ≤7 mm, the maximum depth was no more than 50% of the corneal thickness at the location, and the remaining healthy corneal thickness was ≥300 μm. The follow-up time was 90 to 112 d.RESULTS:Among the included 79 eyes, the lesions were located in the central region of the cornea in 6 eyes, in the paracentral region in 61 eyes, and in the peripheral region in 12 eyes. Hypopyon was observed in 5 cases. LKCCC was successfully administered in 79 eyes, cured in 76 eyes(96%), and failed in 3 eyes(4%). The healing time of corneal epithelium in 76 cured eyes was 3-15 d, of which 51 eyes(67%)healed within 7 d and 24 eyes(32%)healed within 3 d. The uncorrected visual acuity(UCVA)and best corrected visual acuity(BCVA)of 76 eyes of cured patients were statistically significant compared with those preoperatively(P<0.0167). Two of the 3 failed eyes were located at the edge of the lesion and recovered after re-keratectomy. One eye was located in the center of the lesion and recovered after being covered by bulbar conjunctival flap. At the last follow-up, no other complications were observed in all patients except superficial cloud and thinning of cornea.CONCLUSION:LKCCC is a rapid and effective treatment for superficial fungal keratitis and can be considered a new treatment option.

Mutations in the IARS1 gene are rare in clinical practice， and up to now， only ten cases with detailed clinical and genetic data have been recorded in the literature. This article reports a case of growth retardation， intellectual developmental disorder， hypotonia， and hepatopathy （GRIDHH） associated with single heterozygous mutations in the IARS1 gene and summarizes the clinical and genetic features of GRIDHH， thereby expanding the genetic spectrum of GRIDHH.

Objective To investigate current status in knowledge,attitude and practice(KAP)of transitional care among the nurses in Shaanxi province,identify the influencing factors that hinder the transitional care and carry out training of transitional care for the nurses in different specialties in order to provide a theoretical basis for establishing a transitional care model for discharged patients.Methods A total of 2 288 nurses were selected to participate the survey from 41 hospitals in Shaanxi province by the stratified two-stage random sampling method.A self-designed questionnaire for general information,an obstacle factor scale,and a KAP of transitional care questionnaire were used as the survey instruments.The data acquired from the survey were processed by Cluster analysis to identify potential categories and the influencing factors.Results Totally 2085 nurses finished the study.Total score of KAP of transitional care among the nurses in Shaanxi province was found at(134.96±23.75),which was at a medium level.The scores of knowledge,attitude and practice were 77.24%,80.64%and 76.98%respectively.Cluster analysis showed that all the nurses could be categorised into either a lower scored group(n=1 596,76.5%)or a higher scored group(489,23.5%),with a best potential category number of 2 for the 2 085 nurses who participated the survey.There were statistically significant differences between the groups in terms of care training,telephone follow-up,network platform,outpatient service of nursing experts,family visits,patient club and hospital categories(all P＜0.05).Logistic regression analysis on even factors above showed that care training,telephone follow-up,network platform and patient club could significantly affect the potential categorisation of KAP of the nurses in transitional care(all P＜0.05).The main factors that hindered the transitional care included insufficient human resources(93.81%),insufficient supply of community services(93.57%)and lack of a standardised flowchart for transitional care(93.09%).Conclusions Levels of KAP about the transitional care are at a medium level among the nurses of 41 hospitals in Shaanxi province,which indicates that the nurses are active in attitude,but the knowledge and practice are required to be further improved and standardised.The results of cluster analysis indicate that it is necessary for nursing managers to carry out targeted trainings in transitional care for the nurses in different categories.This study provides a theoretical basis to establish a diversified transitional care mode for discharged patients in Shaanxi province.

Objective:To analyze the genetic and clinical characteristics, treatment and prognosis of patients diagnosed with maturity onset of diabetes of the young (MODY) 12 subtype.Methods:This retrospective study collected and analyzed data from 5 children with MODY12 subtype caused by ABCC8 gene variants who underwent inpatient and outpatient genetic testing at Beijing Children′s Hospital from January 2016 to December 2023. Their clinical and genetic features, treatment, and follow-up results were analyzed.Results:Among the 5 patients with MODY12 subtype, 4 were male and 1 was female, with an age of 13.4 (5.5, 14.6) years. Four of the patients were born large for gestational age, while one was born small for gestational age. Two patients were overweight or obese. Three patients exhibited typical symptoms of diabetes, while 2 were incidentally found to have elevated blood glucose level. One patient was found to have diabetic ketoacidosis at onset, who was diagnosed with congenital hyperinsulinism during the neonatal period and received diazoxide treatment, and experienced intellectual developmental delay. All 5 patients had autosomal dominant inherited diabetes within 3 generations. The fasting blood glucose at onset was 7.5 (6.5, 10.0) mmol/L, the haemoglobin A1c (HbA1c) was 11.8% (7.5%, 13.5%), and the fasting C-peptide was 1.2 (1.1, 2.2) μg/L. The duration of follow-up was 15 (9, 32) months. One patient underwent lifestyle intervention, 2 received metformin orally, 1 received insulin therapy, and the other received subcutaneous injection of insulin combined with sulfonylurea orally. At the last follow-up, the median fasting blood glucose was 6.1 (5.1, 7.0) mmol/L, the HbA1c was 5.9% (5.7%, 7.1%), and the fasting C-peptide was 1.7 (0.9, 2.9) μg/L. One patient developed diabetic retinopathy. There were 4 missense variations in ABCC8 gene and one in-frame deletion, all of which were maternally inherited heterozygotes.Conclusions:MODY12 subtype is a heterogeneous disorder with the age of onset from infancy to adolescence. It can present as mild hyperglycemia or diabetic ketoacidosis, and has a high incidence of obesity. Definitive diagnosis can be achieved through genetic test, and individualized treatment is recommended based on glucose levels.

Objective:To evaluate the effectiveness and safety of treatment with Burosumab in pediatric X-linked hypophosphatemia (XLH) patients.Methods:In this retrospective case study, 4 children with pediatric XLH, who were treated with Burosumab in Beijing Children′s Hospital, Capital Medical University and Shandong Provincial Hospital affiliated to Shandong First Medical University from July 2022 to December 2023, were selected as the study objects. We collected and analyzed their clinical characteristics, biochemical indicators, imaging results, and treatment. The children were followed up every 3 months until December 2023, and the clinical outcomes and adverse drug reactions after treatment were evaluated.Results:Of the 4 patients, 3 were males and 1 was female; they were aged 6.7, 2.9, 2.1, and 2.3 years, respectively. Three patients had previously received treatment with phosphate supplements and active vitamins, but their wadding gait and lower limb deformities did not improve significantly, neither did their imaging changes of active richets. The initial dose of Burosumab in the 4 patients was 0.8 mg/kg, administered subcutaneously every 2 weeks, with a treatment course of 0.8-1.3 years. The fasting serum phosphorus and tubular maximum reabsorption of phosphate/glomerular filtration rate (TmP/GFR) of the 4 patients before treatment were 0.72, 0.95, 0.81, 0.66 mmol/L and 0.67, 0.85, 0.87, 0.61 mmol/L, respectively. At the last follow-up, the fasting serum phosphorus and TmP/GFR levels were significantly increased (0.96, 1.09, 1.09, 0.90 mmol/L, and 0.85, 0.79, 1.03, 0.98 mmol/L, respectively). Among them, only the TmP/GFR level (1.17 mmol/L) in case 2 achieved normal values at 3 months post-therapy, while the rest did not reach the normal range for children of the same age. After treatment, the alkaline phosphatase levels of all patients gradually decreased (the values were 461, 240, 423, and 237 U/L, respectively), and the ALP levels in cases 2 and 4 returned to normal at the last visit. Case 4 showed a slight increase in parathyroid hormone (PTH) levels after 9 months of treatment, while the PTH levels in the rest 3 cases remained normal. Case 1 underwent a 6-minute walking test, and the walking distance increased from 245 m to 570 m. Abnormal gait, lower limb deformity, and the severity of rickets in the 4 patients had all improved. No adverse drug reactions such as nephrocalcinosis, local skin injection reaction, hyperphosphatemia, or vitamin D deficiency were observed.Conclusion:Burosumab can improve clinical symptoms in children with XLH with a good safety profile.

Objective:To investigate the efficacy and safety of oral testosterone therapy in individuals diagnosed with androgen insensitivity syndrome (AIS).Methods:A self-controlled study design was utilized, focusing on individuals with AIS who were genetically diagnosed at the Department of Endocrinology, Genetics, and Metabolism of Beijing Children′s Hospital between 2009 and 2021. These patients underwent treatment involving the administration of testosterone. The primary observed indexes include the measurement of penis length, which should meet the minimal surgical standard (penis length≥2.5 cm) or greater than or equal to -2.5 s (lower limit of normal). Secondary observed indexes include penile length standard deviation score (PL-SDS), an increase in penis longitude (ΔPL), medication dosage, the course of therapy, and safety indicators, among others. There were 4 courses of treatment. After each course, patients were evaluated to determine whether termination of treatment was appropriate. Patients who exhibited inadequate post-treatment penile length growth were advised to continue with further treatment. The statistical methodology included t-test, and a Wilcoxon rank sum test to describe efficacy and safety. The patients were followed up until 2023. Results:The study comprised a total of 51 individuals with AIS, comprising 33 males and 18 females (gender of registered permanent residence). Among these patients, 10 were diagnosed with complete androgen insensitivity syndrome (CAIS) and 41 were diagnosed with partial androgen insensitive syndrome (PAIS). There were 2 children with CAIS were diagnosed by doctors and prescribed testosterone undecanoate, but the children did not really take medicine.The penile length of CAIS patients could not be measured (penile length<0.5 cm) before and after treatment. For PAIS patients, baseline penile length and PL-SDS were (2.3±0.6) cm and -3.7±1.3, respectively. The measurements for penile length and PL-SDS after each treatment course were recorded as follows: (2.7±0.8), (2.8±0.6), (2.6±0.4), (2.6±0.4) cm and -2.8±1.6, 2.5±1.6, 2.9±1.2, -3.2±0.9, respectively. Both penile length and PL-SDS interventions showed statistically significant gains when compared to the baseline performance of the 4 courses ( t=4.05、3.56、2.55、2.23 and 3.88、3.50、2.50、2.19, all P<0.05). Before treatment, 13 PAIS patients (32%) reached 2.5 cm and seven (17%) reached greater than or equal to -2.5 s. Following the initial, subsequent, third, and fourth therapeutic interventions, 18 cases (44%), 24 cases (59%), 25 cases (61%), and 26 cases (63%) reached 2.5 cm, respectively. Additionally, A total of 12 cases (29%), 15 cases (37%), 20 cases (49%), and 21 cases (51%), respectively, were found to reach greater than or equal to -2.5 s. The study involved the longitudinal monitoring of patients with the highest recorded age being 13.7 years. The weight, height, body mass index, bone age/age, cholesterol, hemoglobin and so on were all within the normal range and the difference were not statistically significant (all P>0.05). All 49 patients were no abnormalities in blood electrolyte, liver and kidney function and thyroid function and no changes in precocious puberty, pubic hair growth, aggressive behavior, vulvar skin darkening, diarrhea or other conditions. Conclusions:Testosterone undecanote in children with CAIS was no effective. The initial course of treatment for patients with PAIS demonstrates observable enhancements in penile length and PL-SDS. For patients with inadequate penile length growth, continued treatment in subsequent courses (such as the second, third, and fourth courses) is recommended toenhance outcomes gradually. Testosterone undecanoate was safe and effective for the majority of individuals with PAIS patients, with few adverse effects and good treatment tolerance.

Graves′ disease (GD) is the most prevalent cause of hyperthyroidism in children, posing a significant threat to their normal growth and development.The incidence of GD is increasing year by year.Antithyroid drugs are the preferred treatment for GD in children, which can alleviate immune disorders and normalize thyroid function in a short period of time.However, there are still numerous unsolved problems and controversies surrounding this treatment, and the challenges that need to be overcome include the low remission rate, high recurrence rate and high incidence of adverse reactions.The key challenges facing antithyroid drug therapy are how to normalize thyroid function quickly, maintain therapeutic efficacy and reduce recurrence rate, and further studies are needed to improve the effectiveness and safety of the therapy.This article summarizes and reviews the therapeutic strategies, controversies and adverse reactions of antithyroid drugs for GD in children and adolescents.

Objective:The classification model of prognosis of elderly cancer patients in rural areas of Shaanxi province was constructed based on machine learning algorithm, and its effectiveness was verified, providing reference for early prognosis recognition and intervention treatment.Methods:Using a multicenter, cross-sectional survey method and convenience sampling method, 597 elderly cancer patients in rural areas hospitalized in 9 different medical institutions (Shaanxi Cancer Hospital and its member units of specialty alliance) in Shaanxi Province from July to August 2022 were selected as the research objects, and a variable database of "basic information", "self-care ability", "symptoms", "comprehensive needs" and "quality of life" of elderly cancer patients in rural areas was established. Machine learning and statistical analysis were carried out to explore the important prognostic characteristics of elderly cancer patients in rural areas, and a prognostic classification model for elderly cancer patients in rural areas was constructed and verified.Results:The 597 valid questionnaires were ultimately collected.Among the 597 elderly cancer patients in rural areas, 207 were males and 390 were females, aged (69.56 ± 8.84) years. The results of cluster exploration showed that the prognosis of elderly cancer patients in rural areas was divided into three categories: good, medium and poor. The areas under the working characteristic curves of the subjects in good, medium and poor were 0.84, 0.79 and 0.69, respectively. The results of characteristic exploration showed that 10 indicators of "whether accompanied by metastasis", "distress", "sadness", "numbness", "eating", "walking", "fatigue", "forgetfulness", "fun of life" and "relationship with others" were important characteristic indicators of prognosis evaluation of elderly cancer patients in rural areas. There were statistically significant differences among the 10 important characteristic indicators in the three categories of prognosis of rural elderly cancer patients ( χ2=21.07, H values were 18.51-144.38, all P<0.01). There were statistically significant differences in the three categories of "comprehensive needs", "quality of life", "self-care ability" and "symptoms" ( H values were 519.40, 40.80, 103.69, all P<0.01). Conclusions:The construction and verification of a prognostic classification model for elderly cancer patients in rural areas based on machine learning algorithm can effectively explore the important characteristic indicators and prognostic classification of elderly cancer patients in rural areas, and provide basis and guidance for clinical medical staff to make individualized plans.

Objective:To establish HPLC fingerprints of Aristolochia contorta and honey-processed Aristolochia contorta; To analyze the changes of chemical components before and after honey processing with multivariate statistics; To provide a reference for the study on the toxicity reduction of Aristolochia contorta.Methods:The fingerprints of 11 batches of Aristolochia contorta and honey-processed Aristolochia contorta were established through HPLC. Clustering analysis (HCA), principal component analysis (PCA), orthogonal partial least squares discriminant analysis (OPLS-DA) and independent sample t-test were used to compare the changes of chemical components of Aristolochia contorta before and after honey processing.Results:The results showed that there were 14 common peaks in the fingerprints of Aristolochia contorta and Aristolochia contorta. 7 common peaks were identified. Both HCA and PCA could clearly distinguish the samples of Aristolochia contorta before and after honey processing. OPLS-DA found and screened 7 differential markers, and the order of difference significance was peak 3 > peak 7 (7-hydroxy aristolochic acid A) > peak 5 (aristolochic acid C)> peak 8 (aristolochic acid D) > peak 6 > peak 2 (Magnolia alkaloid) > peak 14 (aristolochic acid Ⅰ). After honey processing, the content of chemical components represented by peaks 2, 3, 5, 6, 7, 8 and 14 decreased ( P<0.05). Conclusion:This method is simple and specific, which can be used for the fingerprint analysis of Aristolochia contorta and honey-processed Aristolochia contorta, and can effectively distinguish Aristolochia contorta and honey-processed Aristolochia contorta, and provide a reference for the processing research of toxicity reduction of Aristolochia contorta honey processing.