OBJECTIVES: To investigate the efficacy and safety of empagliflozin in patients with glycogen storage disease (GSD)-associated inflammatory bowel disease (IBD). METHODS: A cross-sectional study was conducted, enrolling 25 patients with GSD-associated IBD who received empagliflozin treatment. General data, details of empagliflozin use, and adverse events were collected. Clinical symptoms and biochemical parameters before and after empagliflozin therapy were compared. RESULTS: Twenty-five patients with GSD-associated IBD were included, with a median age at diagnosis of 0.7 years, and a mean age at initiation of empagliflozin therapy of (11 ± 6) years. The initial dose of empagliflozin was (0.30 ± 0.13) mg/(kg·d), with a maintenance dose of (0.40 ± 0.21) mg/(kg·d), and a treatment duration of (34 ± 6) months. Seventy-eight percent (18/23) of patients' parents reported that empagliflozin therapy reduced the frequency of infections and oral ulcers, and increased neutrophil counts. Clinically, the number of patients with anorexia decreased from 12 to 5 after treatment, and 30% showed improved appetite (P<0.05). The numbers of patients with diarrhea, mucus/bloody stools, perianal disease, and oral ulcers decreased from 19, 9, 11, and 21 before treatment to 7, 1, 0, and 10 after treatment, respectively (P<0.05). Laboratory findings showed that absolute neutrophil counts increased, while platelet counts, lactate, and uric acid levels decreased significantly after empagliflozin treatment (P<0.05). Adverse reactions occurred in 7 patients (28%) during empagliflozin treatment. Two cases occurred in the treatment initiation phase, presenting as hypotension or profuse sweating with dehydration, along with urinary tract infections (UTIs); empagliflozin was discontinued in both cases. During the maintenance phase, 3 cases of UTIs and 2 cases of hypoglycemia (one with profuse sweating) were reported. CONCLUSIONS Empagliflozin therapy can increase neutrophil counts, reduce the incidence of infections and oral ulcers, alleviate diarrhea and abdominal pain, improve appetite, and ameliorate platelet count, lactate, and uric acid levels in patients with GSD-associated IBD, demonstrating significant clinical benefit. UTIs, hypoglycemia, hypotension, profuse sweating, and dehydration may be potential adverse reactions associated with empagliflozin therapy.
Humans ; Benzhydryl Compounds/adverse effects* ; Male ; Female ; Glucosides/adverse effects* ; Inflammatory Bowel Diseases/etiology* ; Child ; Child, Preschool ; Cross-Sectional Studies ; Adolescent ; Glycogen Storage Disease/drug therapy* ; Infant

Objective: To analyze the short-time efficacy of empagliflozin in the treatment of glycogen storage disease type Ⅰb (GSD Ⅰb). Methods: In this prospective open-label single-arm study, the data of 4 patients were collected from the pediatric department in Peking Union Medical College Hospital from December 2020 to December 2022. All of them were diagnosed by gene sequencing and had neutropenia. These patients received empagliflozin treatment. Their clinical symptoms such as height and weight increase, abdominal pain, diarrhea, oral ulcer, infection times, and drug applications were recorded at 2 weeks, 1 month, 2 months, 3 months, 6 months, 9 months, 12 months, and 15 months after treatment to assess the therapeutic effect. The liquid chromatography-tandem mass spectrometry method was used to monitor the changes in 1, 5-anhydroglucitol (1, 5AG) concentration in plasma. At the same time, adverse reactions such as hypoglycemia and urinary tract infection were closely followed up and monitored. Results: The 4 patients with GSD Ⅰb were 15, 14, 4 and 14 years old, respectively at the beginning of empagliflozin treatment, and were followed up for 15, 15, 12 and 6 months, respectively. Maintenance dose range of empagliflozin was 0.24-0.39 mg/(kg·d). The frequency of diarrhea and abdominal pain decreased in cases 2, 3, and 4 at 1, 2 and 3 months of treatment, respectively. Their height and weight increased at different degrees.The absolute count of neutrophils increased from 0.84×10⁹, 0.50×10⁹, 0.48×10⁹, 0.48×10⁹/L to 1.48×10⁹, 3.04×10⁹, 1.10×10⁹, 0.73×10⁹/L, respectively. Granulocyte colony-stimulating factor was gradually reduced in 1 patients and stopped in 3 patient. Plasma 1, 5 AG levels in 2 children were significantly decreased after administration of empagliflozin (from 46.3 mg/L to 9.6 mg/L in case 2, and from 56.1 mg/L to 15.0 mg/L in case 3). All 4 patients had no adverse reactions such as hypoglycemia, abnormal liver or kidney function, or urinary system infection. Conclusion: In short-term observation, empagliflozin can improve the symptoms of GSD Ⅰb oral ulcers, abdominal pain, diarrhea, and recurrent infection, also can alleviate neutropenia and decrease 1, 5AG concentration in plasma, with favorable safety.
Humans ; Child ; Child, Preschool ; Adolescent ; Prospective Studies ; Glycogen Storage Disease Type I/drug therapy* ; Neutropenia ; Abdominal Pain ; Diarrhea/drug therapy* ; Hypoglycemia

Pompe disease, also known as glycogen storage disease type Ⅱ, is a rare autosomal recessive disease. With the application of enzyme replacement therapy, more and more patients with Pompe disease can survive to adulthood, and nervous system-related clinical manifestations gradually emerge. Nervous system involvement seriously affects the quality of life of patients with Pompe disease, and a systematic understanding of the clinical manifestations, imaging features and pathological changes of nervous system injury in Pompe disease is of great significance for the early identification and intervention of Pompe disease. This article reviews the research progress of neurological damage in Pompe disease.
Humans ; Glycogen Storage Disease Type II/drug therapy* ; alpha-Glucosidases ; Quality of Life ; Enzyme Replacement Therapy

The cold chain safety of vaccines is a global issue. The electronic vaccine vial monitor (eVVM) label can monitor the temperature of vaccines in real time and provide "early warning" prompts. In order to comprehensively evaluate the monitoring efficiency of eVVM, this study selected 75 eVVM labels and distributed them with a total of 600 vaccine vial monitor (VVM) labels of four different types in different experimental environment (2-8℃, -20℃ and 40℃), and used a temperature recorder as "gold standard". The results showed that the accuracy of the eVVM labels and VVM labels in high temperature environment was as same as that of the temperature recorder (
Drug Storage ; Electronics ; Refrigeration ; Temperature ; Vaccines

The flower color of Dendrobium catenatum(D. officinale) tends to fade during storage. In order to clarify the influence of storage conditions on the pigment components in flowers, two conditions were applied:temperature and illumination. The contents of pigments in the D. catenatum flower were determined by UV-Vis spectrophotometry and HPLC, and the changes of them during storage were analyzed. The results showed that illumination and temperature had an effect on the pigments of D. catenatum flower during sto-rage. Illumination significantly promoted the degradation of pigments. The contents of total chlorophyll, carotenoids and anthocyanins in the light samples were significantly lower than those in the dark. The total chlorophyll, carotenoids and anthocyanins in the light samples were decreased by 46.5%, 63.4%, and 69.2% respectively. Illumination had a greater effect on fat-soluble pigments than water-soluble pigments. Among the three temperature treatments, the contents of chlorophyll, carotenoid and anthocyanin were as follows:-20 ℃>4 ℃>room temperature, it is indicated that-20 ℃ was the best temperature to maintain the stability of pigment composition. The contents of chlorophyll a, chlorophyll b, β-carotene, lutein and zeaxanthin in the light samples decreased by 34.8%, 69.0%, 72.5%, 61.6%, 36.1%, respectively. After storage for 5 months, the contents of chlorophyll, carotenoid and anthocyanin constituent at-20 ℃ was significantly higher than those at 4 ℃ and room temperature. The results show that light avoiding and low-temperature can effectively slow down the degradation of pigment components. Therefore, it is suggested that D. catenatum flower should be stored in light avoiding and low-temperature conditions in actual production and processing, which can prolong the usable time.
Anthocyanins/analysis* ; Carotenoids/analysis* ; Chlorophyll/analysis* ; Chromatography, High Pressure Liquid ; Dendrobium/chemistry* ; Drug Storage ; Flowers/chemistry* ; Light ; Pigments, Biological/analysis* ; Plants, Medicinal/chemistry* ; Spectrophotometry ; Temperature

BACKGROUND: The National Health Insurance Service (NHIS) established a healthcare claim database for all Korean citizens. This study aimed to analyze the NHIS data and investigate the patterns of breast cancer treatments. METHODS: We constructed a retrospective female breast cancer cohort by analyzing annual incident cases. The annual number of newly diagnosed female breast cancer was compared between the NHIS data and Korea National Cancer Incidence Database (KNCIDB). The annual treatment patterns including surgery, chemotherapy, radiation therapy, endocrine therapy and targeted therapy were analyzed. RESULTS: A total of 148,322 women with newly diagnosed invasive breast cancer during 2006–2014 was identified. The numbers of newly diagnosed invasive breast cancer cases were similar between the NHIS data and KNCIDB, which demonstrated a strong correlation (r = 0.995; P < 0.001). The age distribution of the breast cancer cases in the NHIS data and KNCIDB also showed a strong correlation (r = 1.000; P < 0.001). About 85% of newly diagnosed breast cancer patients underwent operations. Although the proportions of chemotherapy use have not changed during 2006–2014, the total number of chemotherapy prescriptions sharply increased during this period. The proportions of radiotherapy and anti-hormonal therapy increased. Among the anti-hormonal agents, tamoxifen was the most frequently prescribed medication, and letrozole was the most preferred endocrine treatment in patients aged ≥ 50 years. CONCLUSION: Along with the increased breast cancer incidence in Korea, the frequencies of breast cancer treatments have increased. The NHIS data can be a feasible data source for future research.
Age Distribution ; Breast Neoplasms* ; Breast* ; Cohort Studies ; Delivery of Health Care ; Drug Therapy ; Epidemiology ; Female ; Humans ; Incidence ; Information Storage and Retrieval ; Korea ; National Health Programs* ; Prescriptions ; Radiotherapy ; Retrospective Studies ; Survivors* ; Tamoxifen

To study the effect of different storage time on the chemical compositions and sulfur dioxide residues of sulfur-fumigated Gastrodiae Rhizoma (GR), and provide scientific basis for solving the quality and safety issues of sulfur-fumigated traditional Chinese medicinal materials. GR, sulfur-fumigated GR and its medicinal slices were stored under the same conditions, and then 8 active ingredients and sulfur dioxide residues were measured respectively. The results showed that the content of gastrodins in sulfur-fumigated GR and its medicinal slices was significantly lower than that in the non-fumigated GR. Moreover, the content of sulfur dioxide residue in sulfur-fumigated GR was significantly higher than that in its medicinal slices. That is to say, sulfur fumigation degree had significantly higher effect on GR quality as compared with its medicinal slices. During the whole storage time (8 months), the content of the eight chemical components in GR was not changed greatly in general. However, after the storage for 4 months, the content of 8 components and sulfur dioxide residues in all of GR samples were significantly changed. In particular, the content of sulfur dioxide residue in GR medicinal materials decreased up to 50% or more.
Drug Storage ; Drugs, Chinese Herbal ; chemistry ; Fumigation ; Gastrodia ; chemistry ; Rhizome ; chemistry ; Sulfur ; Sulfur Dioxide ; analysis

OBJECTIVES: The objective of this investigation was to demonstrate the possibility of the construction of a real-time prescription drug monitoring system (PDMOS) using data from the nationwide Drug Utilization Review (DUR) system in Korea. METHODS: The DUR system collects information on drug prescriptions issued by healthcare practitioners and on drugs dispensed by pharmacies. PDMOS was constructed using this data. The screen of PDMOS is designed to exhibit the number of drug prescriptions, the number of prescriptions dispensed by pharmacies, and the dispensed prescription drug costs on a daily and weekly basis. Data was sourced from the DUR system between June 1, 2016 and July 18, 2016. The TOGA solution developed by the EYEQMC Co. Ltd. of Seoul, Korea was used to produce the screen shots. RESULTS: Prescription numbers by medical facilities were more numerous than the number of prescriptions dispensed by pharmacies, as expected. The number of prescriptions per day was between 2 to 3 million. The prescriptions issued by primary care clinics were most numerous, at 75% of the total number of prescriptions. Daily prescription drug costs were found to be approximately US $50 million. The prescription drug costs were highest on Mondays and were reduced towards the end of the week. Prescriptions and dispensed prescriptions numbered approximately 1,200 and 1,000 million, respectively. CONCLUSIONS: The construction of a real-time PDMOS has been successful to provide daily and weekly information. There was a lag time of only one day at the national level in terms of information extraction, and scarcely any time was required to load the data. Therefore, this study highlights the potential of constructing a PDMOS to monitor the estimate the number of prescriptions and the resulting expenditures from prescriptions.
Delivery of Health Care ; Drug Costs ; Drug Monitoring* ; Drug Prescriptions ; Drug Utilization ; Drug Utilization Review ; Health Expenditures ; Information Storage and Retrieval ; Korea ; Pharmacies ; Prescription Drugs ; Prescriptions* ; Primary Health Care ; Seoul

Wilms' tumor is the most common malignant renal tumor in childhood. The brain metastasis of a Wilms' tumor with anaplastic histopathology is rare. We present the case of an 8-year-old girl with Wilms' tumor, who presented with multiple brain metastases 5 years after her primary diagnosis. The brain masses were diagnosed after a generalized tonic-clonic seizure attack. The big solid mass in the cerebellum was resected, and whole-brain radiotherapy was performed, after which, she succumbed to her disease. In the case of clinical suspicion, cranial surveillance should be included in the routine clinical work-up for Wilms' tumor. Combined aggressive therapy (surgery+radiotherapy+chemotherapy) should be applied whenever possible, for both better survival and palliative aspects.
Brain* ; Cerebellum ; Child ; Diagnosis ; Drug Therapy ; Female ; Glycogen Storage Disease Type VI ; Humans ; Neoplasm Metastasis* ; Radiotherapy ; Seizures ; Wilms Tumor*

Although the importance of clinical data is drawing more attention in drug development in China, the clinical data management is not good enough in the clinical trials right now. With the development of internet and progress of information technology, especially with the setup of the state innovation strategy for drug development, it is necessary and urgent to improve the clinical data quality. Good data quality is the primary basis of technical evaluation of drug at the marketing authorization. So Center for Drug Evaluation of CFDA has made some endeavors to enhance data management in the clinical trials in recent years. This article is focused on these aspects of data managment.
China ; Data Collection ; standards ; Drug Evaluation ; standards ; Information Storage and Retrieval ; standards ; Internet