[Short-term efficacy of empagliflozin in children with glycogen storage disease type â b].

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.

[The influence of two kinds of transnasal endoscopic surgery on the outcome status of sinus cavity in patients with eosinophilic chronic rhinosinusitis with nasal polyps].

Objective: To investigate the trend of postoperative cavity status in patients with eosinophilic chronic sinusitis with nasal polyps (eCRSwNP) who underwent total nasalization surgery and partial reboot surgery. And to discuss the relationship between tissue eosinophil counts and status of postoperative cavity. Methods: Patients with eCRSwNP in four tertiary medical centers (Longgang ENT Hospital, Xiamen Humanity Hospital, Guangdong Clifford Hospital and the First Affiliated Hospital of Sun Yat-Sen University) from March 2018 to October 2021 were divided into 2 groups. The group without previous surgery history was performed for the nasalization surgery, and another group with previous surgery history underwent the part-reboot surgery. The follow-up time after operation was defined as the following 5 stages: 6, 12, 20-24, 36 and more than 42 months. According to FESS-95 Guangzhou standard, status of sinus cavity was assessed and classified into 3 categories: good, better and bad. The association between the sinus cavity status and tissue eosinophil counts in the above 5 stages was analyzed by one-way ANOVA, and P<0.05 was considered statistically significant. Results: A total of 72 eCRSwNP patients finished the follow-up in this study. There were 47 males and 25 females in these patients, aged from 11 to 67 years. A total of 50 cases underwent nasalization surgery and 22 cases underwent partial reboot surgery. With the follow-up time from 6 to 48 months, there were 72 cases (100.0%) who completed 6 months and 12 months follow up, 46 cases (63.9%) for 20-24 months, 36 cases (50.0%) for 32-36 months and 16 cases (22.2%) with the follow-up time more than 42 months. No matter what kind of surgery, there was no "bad" situation of the surgical cavity status 6 months after the operation, and the differentiation gradually occurred more than 12 months after the surgery. Moreover, the rates of "good" cavity status for the 5 stages in the group of nasalization surgery were 78.0%, 66.0%, 56.7%, 47.6% and 42.9%, and were 63.6%, 45.5%, 25.0%, 20.0% and 11.1% in the partial reboot surgery group, respectively, suggesting that the status of nasal cavity in nasalization surgery group was always better than that in partial reboot surgery group in every period. In addition, the "bad" rate was 0, 8.0%, 10.0%, 14.3% and 28.6% in the group of nasalization surgery, and was 0, 27.3%, 18.8%, 33.3% and 55.6% in the partial reboot surgery group, respectively. The average percentage of tissue eosinophil counts in the 72 cases was 42.1%, which had no obvious effect on the status of the surgical cavity (P>0.05). Conclusions: For eCRSwNP patients, the operative cavity status in the patients without previous operation history treated with nasalization surgery is good. The time of 1-2 years after surgery is the main period for sinus lesions. The counts of tissue eosinophils has no significant influence on surgical sinus cavity status in the eCRSwNP patients.

Utilizing integrating network pharmacological approaches to investigate the potential mechanism of Ma Xing Shi Gan Decoction in treating COVID-19.

Beginning in December 2019, coronavirus disease 2019 (COVID-19), due to 2019-nCoV infection, emerged in Wuhan and spread rapidly throughout China and even worldwide. Employing combined therapy of modern medicine and traditional Chinese medicine has been proposed, in which Ma Xing Shi Gan Decoction (MXSGD) was recommended as a basic prescription and applied widely in the clinical treatment of COVID-19. We investigated the underlying mechanism of MXSGD in treating COVID-19 utilizing the approaches of integrating network pharmacology. A total of 97 active ingredients of MXSGD were screened out, and 169 targets were predicted. The protein-protein interaction network exhibited hub targets of MXSGD, such as Heat shock protein 90, RAC-alpha serine/threonine-protein kinase, Transcription factor AP-1, Mitogen-activated protein kinase 1, Cellular tumor antigen p53, Vascular endothelial growth factor A, and Tumour necrosis factor. Gene Ontology functional enrichment analysis demonstrated that the biological processes altered within the body after taking MXSGD were closely related to the regulation of such processes as the acute inflammatory response, chemokine production, vascular permeability, response to oxygen radicals, oxidative stress-induced apoptosis, T cell differentiation involved in the immune response, immunoglobulin secretion, and extracellular matrix disassembly. KEGG enrichment analysis indicated that the targets of MXSGD were significantly enriched in inflammation-related pathways, immunomodulation-related pathways, and viral infection-related pathways. The therapeutic mechanisms of MXSGD on COVID-19 may primarily involve the following effects: reducing inflammation, suppressing cytokine storm, protecting the pulmonary alveolar-capillary barrier, alleviating pulmonary edema, regulating the immune response, and decreasing fever.

[Reliability and validity of the simplified Chinese version of the fecal incontinence quality of life questionnaire in the patients with fecal incontinence].

Objective: To develop the Chinese version of the fecal incontinence quality of life (FIQL) questionnaire, and to validate them in Chinese population. Methods: Our study included 52 cases recruiting from outpatients diagnosed with fecal incontinence in Peking Union Medical College Hospital, fecal incontinence in Beijing City nursing homes for the elderly, and patients received perineal repair surgeries who suffered from perineal laceration and laceration in in Peking Union Medical College Hospital from October 2013 to 2016 December.The Original English Fecal Incontinence Quality of Life scale questionnaires were translated into Chinese and they were linguistically validated following the Cross-cultural adaptation of health-related quality of life measures.The reliability and validity of the Chinese version of FIQL questionnaires in Chinese population were evaluated. Results: Fifty-two patients completed the FIQL and the SF-12 questionnaires, as well as Cleveland Clinic Incontinence Score.The Cronbach's alpha of FIQL was 0.949, test-retest reliability ICC was 0.437-0.866. FIQL scores were positively correlated with SF-12 (Spearman correlation coefficient was 0.588, P<0.01) and negatively correlated with Cleveland Clinic Incontinence Score (Spearman correlation coefficient was -0.617, P<0.01). Factor analysis showed that there were 6 common factors in FIQL. Conclusions: Chinese version of FIQL have high reliability and validity in Chinese population, there fore it is suitable for clinic and research.

[Study on antibiotic resistance of Escherichia coli and Enterococcus colonized in intestine of neonates from neonatal intensive care unit].

Objective: To understand the antibiotic resistance of bacteria colonized in intestine of the neonates from neonatal intensive care unit (NICU) and provide evidence to guide clinical antibiotic treatment. Methods: From May, 2014 to May, 2015, a total of 572 stool samples were collected from the neonates of NICU in our hospital. Escherichia coli and Enterococcus were detected with VITEK-2 system. Results: A total of 328 strains of E. coli and 243 strains of Enterococcus were isolated respectively in this study. The 199 strains of E. coli selected for drug susceptibility test showed lower resistant rate to imipenem, ertapenem, amikacin, nitrofurantoin, ranging from 0.50% to 3.52% and showed higher resistant rate to ampicillin, tetracycline, trimethoprim/sulfamethoxazole and cefazolin, ranging from 54.27% to 84.92%. No meropenem resistant strainsere were found. The percentage of ESBLs production strains was 45%. The multi drug resistance test showed that 34.6% of the strains were resistant to four antibiotics. Three strains were resistant to seven antibiotics. The 243 strains of Enterococcus showed lower resistant rate to quinupristin/dalfopristin, nitrofurantoin, streptomycin, ranging from 0.41% to 4.53% and showed higher resistant rate to ampicillin, benzylpenicillin, ciprofloxacin, tetracycline, gentamicin and erythromycin, ranging from 70.78% to 91.77%. No strains which were resistant to tigecycline, vancomycin, rina thiazole amine/ketone were found. The multi drug-resistance test showed that 86.5% of the strains were resistant to five antibiotics. Conclusions: According to the analysis of the 199 strains of E. coli and 243 strains of Enterococcus isolated from the neonates, we found that the resistance of intestinal bacteria in the neonates was very serious, showing multi drug resistance. It is necessary to use antibiotics according to the drug susceptibility test results in clinical treatment.

[Clinical analysis of 12 patients with pediatric antiphospholipid syndrome with pulmonary embolism].

Objective: To identify the clinical and immunological characteristics of pediatric antiphospholipid syndrome (APS) patients with pulmonary embolism. Method: Among 47 pediatric APS patients from Peking Union Medical College Hospital during the year of 2000 to 2015, 12 patients were diagnosed of pulmonary embolism, who were investigated and compared with APS patients without pulmonary embolism. Result: Twelve patients (among whom 6 cases were primary and the other 6 were secondary APS)had pulmonary embolism and all of them were non-shock type, which was the first presenting manifestation in 6 of them.Eight cases were misdiagnosed as infection, while 3 cases were missed.Among patients with pulmonary embolism, 10 patients suffered from deep vein thrombosis at the same time, mainly in lower extremities.2 cases had thrombotic recurrence, which happened only in primary APS patients, because of irregular monitoring of International Normalized Ratio, or not taking aspirin after quitting warfarin.Positive anticardiolipin (ACL) and lupus anticoagulant (LA) were found in 10 and 9 patients respectively.Four primary APS patients had positive anti-nuclear antibodies (ANA). During follow-up of 3-100 months (median 23 months) of primary APS, no one had evolved manifestations of systemic lupus erythematosus.Primary APS was more often seen in males (M∶F 5∶1 vs. 0∶6) and the patients were much younger ((15±1) vs. (17±0) years old) than those with secondary APS.Besides that, no statistically significant difference was seen between primary and secondary APS (P all>0.05). Compared with APS patients without pulmonary embolism, pulmonary hypertension was more common in patients suffered from pulmonary embolism (3/12 vs. 0, P<0.05). Conclusion: Pulmonary embolism can be the first symptom in pediatric APS patients and all of them are non-shock type, which tends to be misdiagnosed or missed. A majority of them suffer from deep vein thrombosis in the lower extremities.Rethrombosis takes place when the anticoagulant therapy is irregular.Positive anti-nuclear antibodies can be seen in primary APS patients, but no manifestations of lupus come out during follow-up.There is no significant difference between primary APS and secondary APS.Pulmonary hypertension is more common in APS patients suffered from pulmonary embolism.

[Alterations of glial fibrillary acidic protein in rat brain after gamma knife irradiation].

OBJECTIVE: To study glial fibrillary acidic protein (GFAP) immunoreactivity in different time and water content of the rat brain treated with gamma knife radiotherapy and to understand the alteration course of the brain lesion after a single high dose radiosurgical treatment. METHODS: In the brains of the normal rats were irradiated by gamma knife with 160 Gy-high dose. The irradiated rats were then killed on the 1st day, 7th day, 14th day, and 28th day after radiotherapy, respectively. The positive cells of GFAP in brain tissue were detected by immunostaining; the water content of the brain tissue was measured by microgravimetry. The histological study of the irradiated brain tissue was performed with H.E. and examined under light microscope. RESULTS: The numbers of GFAP-positive astrocytes began to increase on the 1st day after gamma knife irradiation. It was enlarged markedly in the number and size of GFAP-stained astrocytes over the irradiated areas. Up to the 28th day, circumscribed necrosis foci (4 mm in diameter) was seen in the central area of the target. In the brain tissue around the necrosis, GFAP-positive astrocytes significantly increased (P < 0.01, compared with the control group). The swelling of cells in irradiated region was observed on the 1st day; after irradiation endothelial cells degenerated and red blood cells escaped from blood vessel on the 7th day; leakage of Evans blue dye was observed in the target region on the 14th day. There was a significant decrease of specific gravity in the irradiated brain tissue the 14th and 28th day after irradiation. CONCLUSION: The results suggest that GFAP can be used as a marker for the radiation-induced brain injury. The brain edema and disruption of brain-blood barrier can be occurred during the acute stage after irradiation.