Rare diseases are a collective term for diseases with extremely low prevalence and incidence rates.Up to now,China has released two lists identifying a total of 207 rare diseases.Given that most rare diseases do not have drugs with corresponding indications,physicians frequently resort to using off-label drugs when treating patients with rare diseases.However,there is currently no systematic guideline or expert consensus for the use of off-label medications in China.To comprehensively collect existing evidence of off-label drug use for rare diseases,fully analyze and evaluate the rationality of off-label drug use for rare diseases,and standardize the management of off-label drug use for rare diseases,the Rare Disease Branch of Beijing Medical Association,Chinese Pharmaceutical Association,Beijing Pharmaceutical Association,and the School of Public Health,Peking University have jointly initiated the drafting of the Expert Consensus on Off-label Use of Drugs for Rare Diseases.This consensus refer to the WHO Handbook for Guideline Development,the Guidelines for Developing/Revising Clinical Diagnostic and Treatment Guidelines in China(2022 Edition),the AGREE Ⅱ and the STAR tools.This protocol outlines the background and purpose of consensus,as well as the comprehensive framework for consensus development,encompassing panel formation,clinical issue identification,evidence retrieval,data extraction,and evidence-based recommendation formulation.

Objective:To explore the feasibility and efficacy of left ventricular decompression in pediatric patients supported by extracorporeal membrane oxygenation（ECMO）.Methods:The clinical data of 15 children who underwent left ventricular decompression during ECMO in the cardiac intensive care unit（CICU）of Guangzhou Women and Children's Medical Center from February 2021 to August 2023 were retrospectively analyzed.Results:Among the 15 patients，there were 8 males and 7 females,with a median age of 1.1 years old（20 days to 15 years old）and a median weight of 8.5（3.1 to 38.0）kg. Cardiac pathologies varied，including hypertrophic obstructive cardiomyopathy(1 case),left coronary artery abnormalities originating from the pulmonary artery（3 cases），aortic coarctation with ventricular septal defect（1 case），complete transposition of great arteries with ventricular septal defect（1 case），ventricular septal defect with severe mitral valve insufficiency（1 case），fulminant myocarditis（5 cases），tetralogy of Fallot（1 case），ascending aortic aneurysm with aortic valve insufficiency（1 case），and Kawasaki disease complicated with giant aneurysm（1 case）. The venous-arterial ECMO mode was used in all 15 children,9 cases were intubated centrally and 6 cases were intubated peripherally. The reasons for ECMO：low cardiac output（8 cases），cardiogenic shock（5 cases），and frequent ventricular tachycardia and ventricular fibrillation（2 cases）. Extracorporeal cardiopulmonary resuscitation was performed in 3 cases. The median duration of mechanical ventilation was 248 (142-590) h,the median CICU stay was 18 (7-26) d,and the median hospital stay was 28 (7-37) d. Of the 15 children,7 cases underwent emergency left ventricular decompression through a small sternal incision,with a mean decompression time of ECMO-assisted 12.2(9-22) h，and 8 cases underwent selective left ventricular decompression through a median sternal incision. Left ventricular systolic function improved significantly after decompression in all 15 patients，of whom 12 cases were successfully withdrew off ECMO and 10 cases of them survived.Conclusion:Left ventricular decompression can improve the cardiac dysfunction in children with ECMO，reduce the occurrence of complications，and increase the success rate and survival rate of ECMO.

Rare diseases are a collective term for diseases with extremely low prevalence and incidence rates.Up to now,China has released two lists identifying a total of 207 rare diseases.Given that most rare diseases do not have drugs with corresponding indications,physicians frequently resort to using off-label drugs when treating patients with rare diseases.However,there is currently no systematic guideline or expert consensus for the use of off-label medications in China.To comprehensively collect existing evidence of off-label drug use for rare diseases,fully analyze and evaluate the rationality of off-label drug use for rare diseases,and standardize the management of off-label drug use for rare diseases,the Rare Disease Branch of Beijing Medical Association,Chinese Pharmaceutical Association,Beijing Pharmaceutical Association,and the School of Public Health,Peking University have jointly initiated the drafting of the Expert Consensus on Off-label Use of Drugs for Rare Diseases.This consensus refer to the WHO Handbook for Guideline Development,the Guidelines for Developing/Revising Clinical Diagnostic and Treatment Guidelines in China(2022 Edition),the AGREE Ⅱ and the STAR tools.This protocol outlines the background and purpose of consensus,as well as the comprehensive framework for consensus development,encompassing panel formation,clinical issue identification,evidence retrieval,data extraction,and evidence-based recommendation formulation.

The prevalence of Class III malocclusion varies among different countries and regions. The populations from Southeast Asian countries (Chinese and Malaysian) showed the highest prevalence rate of 15.8%, which can seriously affect oral function, facial appearance, and mental health. As anterior crossbite tends to worsen with growth, early orthodontic treatment can harness growth potential to normalize maxillofacial development or reduce skeletal malformation severity, thereby reducing the difficulty and shortening the treatment cycle of later-stage treatment. This is beneficial for the physical and mental growth of children. Therefore, early orthodontic treatment for Class III malocclusion is particularly important. Determining the optimal timing for early orthodontic treatment requires a comprehensive assessment of clinical manifestations, dental age, and skeletal age, and can lead to better results with less effort. Currently, standardized treatment guidelines for early orthodontic treatment of Class III malocclusion are lacking. This review provides a comprehensive summary of the etiology, clinical manifestations, classification, and early orthodontic techniques for Class III malocclusion, along with systematic discussions on selecting early treatment plans. The purpose of this expert consensus is to standardize clinical practices and improve the treatment outcomes of Class III malocclusion through early orthodontic treatment.
Humans ; Malocclusion, Angle Class III/classification* ; Orthodontics, Corrective/methods* ; Consensus ; Child

This consensus was developed by the Orthodontic Society of the Chinese Stomatological Association to provide a systematic, scientific, and practical guideline for informed consent in orthodontic care. Orthodontic treatment is typically lengthy, highly individualized, and involves multiple factors such as growth and development, occlusal function, and facial esthetics. Rapid technological advances and diverse risk profiles make the traditional reliance on orthodontist experience or institutional templates insufficient to ensure patients′ full understanding and autonomous decision-making. To address this, the expert panel conducted extensive reviews of domestic and international guidelines, analyzed representative dispute cases, and performed multicenter patient-clinician surveys. Using a multi-round Delphi method, the group established a standardized informed consent framework covering the initial consultation, treatment, and retention phases. The consensus emphasizes that informed consent is not only a fundamental legal and ethical requirement but also a key step in building trust, improving patient compliance, and enhancing treatment satisfaction. Orthodontists should clearly and comprehensively explain treatment plans, potential risks, uncertainties, and associated costs, while respecting the autonomy of patients or guardians, and maintain continuous communication and dynamic evaluation throughout the treatment process. The release of this consensus provides unified and authoritative guidance for clinical orthodontics, helping to standardize informed consent, enhance its transparency, safeguard patient rights, reduce medical risks, and promote high-quality, sustainable development of orthodontic practice.

This consensus was developed by the Orthodontic Society of the Chinese Stomatological Association to provide a systematic, scientific, and practical guideline for informed consent in orthodontic care. Orthodontic treatment is typically lengthy, highly individualized, and involves multiple factors such as growth and development, occlusal function, and facial esthetics. Rapid technological advances and diverse risk profiles make the traditional reliance on orthodontist experience or institutional templates insufficient to ensure patients′ full understanding and autonomous decision-making. To address this, the expert panel conducted extensive reviews of domestic and international guidelines, analyzed representative dispute cases, and performed multicenter patient-clinician surveys. Using a multi-round Delphi method, the group established a standardized informed consent framework covering the initial consultation, treatment, and retention phases. The consensus emphasizes that informed consent is not only a fundamental legal and ethical requirement but also a key step in building trust, improving patient compliance, and enhancing treatment satisfaction. Orthodontists should clearly and comprehensively explain treatment plans, potential risks, uncertainties, and associated costs, while respecting the autonomy of patients or guardians, and maintain continuous communication and dynamic evaluation throughout the treatment process. The release of this consensus provides unified and authoritative guidance for clinical orthodontics, helping to standardize informed consent, enhance its transparency, safeguard patient rights, reduce medical risks, and promote high-quality, sustainable development of orthodontic practice.

Objective:To explore the feasibility and efficacy of left ventricular decompression in pediatric patients supported by extracorporeal membrane oxygenation（ECMO）.Methods:The clinical data of 15 children who underwent left ventricular decompression during ECMO in the cardiac intensive care unit（CICU）of Guangzhou Women and Children's Medical Center from February 2021 to August 2023 were retrospectively analyzed.Results:Among the 15 patients，there were 8 males and 7 females,with a median age of 1.1 years old（20 days to 15 years old）and a median weight of 8.5（3.1 to 38.0）kg. Cardiac pathologies varied，including hypertrophic obstructive cardiomyopathy(1 case),left coronary artery abnormalities originating from the pulmonary artery（3 cases），aortic coarctation with ventricular septal defect（1 case），complete transposition of great arteries with ventricular septal defect（1 case），ventricular septal defect with severe mitral valve insufficiency（1 case），fulminant myocarditis（5 cases），tetralogy of Fallot（1 case），ascending aortic aneurysm with aortic valve insufficiency（1 case），and Kawasaki disease complicated with giant aneurysm（1 case）. The venous-arterial ECMO mode was used in all 15 children,9 cases were intubated centrally and 6 cases were intubated peripherally. The reasons for ECMO：low cardiac output（8 cases），cardiogenic shock（5 cases），and frequent ventricular tachycardia and ventricular fibrillation（2 cases）. Extracorporeal cardiopulmonary resuscitation was performed in 3 cases. The median duration of mechanical ventilation was 248 (142-590) h,the median CICU stay was 18 (7-26) d,and the median hospital stay was 28 (7-37) d. Of the 15 children,7 cases underwent emergency left ventricular decompression through a small sternal incision,with a mean decompression time of ECMO-assisted 12.2(9-22) h，and 8 cases underwent selective left ventricular decompression through a median sternal incision. Left ventricular systolic function improved significantly after decompression in all 15 patients，of whom 12 cases were successfully withdrew off ECMO and 10 cases of them survived.Conclusion:Left ventricular decompression can improve the cardiac dysfunction in children with ECMO，reduce the occurrence of complications，and increase the success rate and survival rate of ECMO.

Objective:To examine the effect of short-term regular aerobic exercise on physical fitness of children with pulmonary with atresia ventricular septal defect after radical biventricular treatment.Methods:This was a prospective self pre-and post-control observation study. The subjects performed regular aerobic exercise for 10 days according to the exercise prescription. Body composition measurement and cardiopulmonary exercise test[lung ventilation function, maximum oxygen uptake(VO 2max), maximum oxygen pulse(O 2/HR max), ventilation oxygen uptake efficiency(OUES), exercise load time], 6 min walking distance(6MWD), sports psychometric test, motor function screening test and fitness test, were collected. The changes of test parameters and scale scoring before and after exercise were analyzed and compared. Results:A total of 7 children with PA/VSD after biventricular surgery were enrolled. The age ranged 8.2-16.2 years old, and there were 2 males and 5 females. VO 2max[(1 196.71±395.31)ml/min vs.(1 297.43±425.73)ml/min, P=0.031], O 2/HRmax[(82.43±7.53)ml/beat vs.(91.57±6.95)ml/beat, P<0.001]increased after exercise. The exercise load time was significantly increased compared with that before intervention[(476.43±35.73)s vs.(531.43±45.76)s, P=0.002]. Resting heart rate before exercise( P=0.013) and peak respiration exchange ratio(PeakRER, P=0.021) were significantly lower. Body composition tests suggest weight, intracellular water, protein and muscle content of lower limb were higher( P<0.05). The motor function score was higher than before( P=0.015); the score of sports fear was lower than before( P=0.009). There was no significant difference in lung capacity and 6-minute walking distance before and after exercise( P>0.05). There were no cardiovascular events during the study period. Conclusion:Short-term regular aerobic exercise for children with PA/VSD after biventricular surgery can improve exercise tolerance, increase lower limb muscle content, improve exercise fear and exercise function, and has good safety and feasibility.

Objective:To study the growth trajectory and the incidence of malnutrition and clinical events in infants with congenital heart disease in 1 year after surgery.Methods:Children at the age of 1 year or younger who were diagnosed with congenital heart disease and underwent surgery at Guangzhou Women and Children's Medical Center affiliated to Guangzhou Medical University from January 2018 to January 2019 were included. The age, gender, birth weight and length, and parental height and weight were collected, and the occurrence of clinical events of interest and the children's health as evaluated by caregiver within 1 year after surgery were followed up through questionnaire survey. Malnutrition was defined as a weight-for-age z-score (WAZ) ≤-2 at 1 year after surgery and a WAZ>-2 was defined as non-malnourished.Results:Among the 502 children, 301 were boys and 201 were girls, aged 4.1 (range: 2.0 to 6.8) months, of whom 64.7% were with simple congenital heart disease and 35.3% complex congenital heart disease. The children were generally with mild malnutrition (WAZ<-1 and >-2) before surgery. At 3 months and 6 months after surgery, the children showed a rapid growth catch-up, although failing to reach the normal level, and the trend plateaued at 1 year after surgery. The proportion of children with malnutrition decreased gradually within 1 year after surgery. 47.0% of included children had malnutrition before surgery, and the proportion decreased significantly at 3 months and 6 months, to 17.9% at 1 year after surgery. After discharge, these children suffered from upper respiratory infection most commonly (74.5%), followed by pneumonia (41.2%) and diarrhea (36.7%), and vomiting and constipation (22.1%). In terms of children's health status as evaluated by parents, about 32.0% of families considered their children in poor health before surgery, and the proportion decreased to 6.9% within 1 year after surgery.Conclusion:Infants with congenital heart disease continued to grow and catch up within 1 year after surgery, showing significantly improved nutrition status, but some children still experienced malnutrition at 1 year after surgery.

Objective:To summarize the results of surgical treatment for complete atrioventricular septal defect(CAVSD) in early and middle stages.Methods:147 children with CAVSD in Guangzhou Women and Children’s Medical Center from January 2010 to December 2019 were selected, Males 85, females 62, median age of surgery 5 months(1 months-10 years old), median body mass 5.5 kg(2.4-20.9 kg). Complete atrioventricular septal defect was diagnosed by ultrasonic cardiogram before surgery. All the children underwent atrial ventricular valve formation and underwent simultaneous repair.Outpatient follow-up was planned.Ultrasonic cardiogram and electrocardiogram were performed. SPSS 22 statistical software was used for data analysis.Results:All 147 CAVSD patients underwent one-time surgical correction.Early postoperative death occurred in 7 cases(4.76%). The causes of death were: 3 cases of pulmonary hypertension crisis, 3 cases of severe mitral insufficiency(MI), 1 case of postoperative malignant arrhythmia, and the rest of the children were cured and discharged. Permanent pacemaker was installed in 3 patients due to atrioventricular block(AVB). The follow-up time was 1-10 years old, and 2 patients died in late stage: 1 patient did not seek medical treatment in time due to infection, and 1 patient had unknown cause. Five patients underwent secondary surgery: 4 due to severe mitral/tricuspid insufficiency(MI/TI) and 1 due to delayed AVB. The mid-term follow-up showed 9 cases of severe MI and 4 cases of severe TI. Compared with children with surgical age<3 months and ≥3 months, there were statistically significant differences in postoperative ventilator-assisted ventilation time, severe MI before postoperative discharge and total mortality between the two groups( P<0.05). Mid-term follow-up results showed no difference between the two groups. There were statistically significant differences in surgical age, postoperative CICU stay time and total hospital stay between the children with trisomy 21-syndrome and those without trisomy 21-syndrome( P<0.05), and there was no difference between the two groups in mid-term follow-up results. Residual shunt of 1-3 mm VSD was found in 29 cases, 26 cases were closed during follow-up, and 3 cases had smaller residual shunt. Conclusion:Modified single patch technique treatment of CAVSD has good effect, low mortality and low re-operation rate. But age <3 months group, infant mortality was significantly increased, the duration of postoperative mechanical assisted ventilation was prolonged, and the proportion of early postoperative severe MI was high.Severe MI and TI is easy to occur after CAVSD, which requires long-term follow-up and timely treatment. The children with trisomy 21-syndrome were similar to those with normal chromosome except for longer stay in ICU and total hospital stay.