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Objective: To explore the clinical characteristics and risk factors of pediatric patients with Wiskott-Aldrich syndrome (WAS). Methods: This was a case-control study. Clinical data of 165 cases of pediatric patients with WAS, who visited the Department of Rheumatology, Children's Hospital of Chongqing Medical University between January 2007 and August 2020 were retrospectively analyzed and divided into death group and survival group (control group) according to the prognosis in the follow-up. Two independent samples t -test, Welch approximate t -test, Mann-Whitney U test, Pearson χ² test, Yates corrected χ² test, or Fisher exact probability test were used for comparison between groups. Risk factors were analyzed by multivariate Logistic regression analysis. Results: A total of 165 patients with Wiskott-Aldrich syndrome were enrolled in this study, including 40 cases in the death group and 125 cases in the survival group. The WAS score was (4.1±0.8) score in the death group and (3.1±1.2) score in the survival group. The age was 19 (9, 28) months in the death group and 60 (36,86) in the survival group. The episode rates of recurrent infection and (or) severe infection, intracranial hemorrhage and eczema in the death group were significantly higher than those in the survival group (95.0% (38/40) vs. 32.0% (40/125),25.0% (10/40) vs. 2.4% (3/125), 90.0% (36/40) vs. 72.0% (90/125), χ²=48.253, 18.325, 5.440, all P <0.05). Infection (22 cases, 55.0%) and intracerebral hemorrhage (15 cases, 37.5%) were the main causes of death, 3 cases (7.5%) died of severe graft-versus-host disease after transplantation. The Logistic regression model indicated that repeated infection and (or) severe infection and non-use of intravenous immunoglobulin (IVIG) replacement therapy were risk factors for death in Chinese WAS patients ( OR values were 8.999 and 2.860, 95% CI were (2.041-39.667) and (1.375-5.950), respectively, all P <0.05). Conclusions: Recurrent and (or) severe infection is the main risk factor of death for WAS patietns. Regular IVIG treatment can improve the survival rate of patients with WAS.

Objective: To assess the efficacy of metformin in megestrol acetate (MA)-based fertility-sparing treatment for patients with atypical endometrial hyperplasia (AEH) and endometrioid endometrial cancer (EEC)., Design: A randomised, single-centre, open-label, controlled trial conducted between October 2013 and December 2017., Setting: Shanghai OBGYN Hospital of Fudan University, China., Population: A total of 150 patients (18-45 years old) with primary AEH or well-differentiated EEC were randomised into an MA group (n = 74) and an MA plus metformin group (n = 76)., Methods: Patients with AEH or EEC were firstly stratified, then randomised to receive MA (160 mg orally, daily) or MA (160 mg orally, daily) plus metformin (500 mg orally, three times a day)., Main Outcomes and Measures: The primary efficacy parameter was the cumulate complete response (CR) rate within 16 weeks of treatment (16w-CR rate); the secondary efficacy parameters were 30w-CR rate and adverse events., Results: The 16w-CR rate was higher in the metformin plus MA group than in the MA-only group (34.3 versus 20.7%, odds ratio [OR] 2.0, 95% confidence interval [CI] 0.89-4.51, P = 0.09) but the difference was more significant in 102 AEH patients (39.6 versus 20.4%, OR 2.56, 95% CI 1.06-6.21, P = 0.04). This effect of metformin was also significant in non-obese (51.4 versus 24.3%, OR 3.28, 95% CI 1.22-8.84, P = 0.02) and insulin-sensitive (54.8 versus 28.6%, OR 3.04, 95% CI 1.03-8.97, P = 0.04) subgroups of AEH women. No significant result was found in secondary endpoints., Conclusion: As a fertility-sparing treatment, metformin plus MA was associated with a higher early CR rate compared with MA alone in AEH patients., Tweetable Abstract: For AEH patients, metformin plus MA might be a better fertility-sparing treatment to achieve a higher early CR rate compared with MA alone., (© 2020 Royal College of Obstetricians and Gynaecologists.)

Objective: Nestin has been found to be overexpressed in several human malignancies. The primary goal of this research was to investigate whether nestin expression correlates with the occurrence and development of ovarian cancer., Patients and Methods: Expression of nestin was evaluated by immunohistochemistry in 315 cases of various epithelial ovarian lesions as well as 52 cases of normal ovarian epithelia. The association between nestin expression and various clinical pathological parameters was analysed., Results: Immunohistochemical results showed that the positive correlation of nestin expression gradually increased from benign and borderline to malignant ovarian tumours. Nestin overexpression was associated with more advanced International Federation of Gynaecology and Obstetrics stage and higher histological grade in serous adenocarcinoma. Nestin expression was significantly associated with chemoresistance. However, there was no significant correlation between nestin expression and the age and level of CA125 in patients., Conclusions: We propose that nestin expression might contribute to the initiation, promotion, and progression of serous ovarian carcinoma; nestin expression is associated with poor chemoresponse. It could be a potential therapeutic target in patients with epithelial ovarian carcinoma.

Objective: To study the chemical constituents in the volatile oil from fruits of Alpinia oxyphylla., Method: Using GC-MS to identify the constituents., Result and Conclusion: Sixty-four compounds were identified on the basis of GC-MS, the main ones being p-cymene, valence, linalool, myrtenal, alpha-pinene, beta-pinene, furopelargone A and terpinen-4-ol. Three sesquiterpenes valencene, nootkanone and nootkanol have been isolated from the CHCl3 extract as check, of these 64 identified compounds linalyl oxide, valencene, bakkenolide A, furopelargone A and 3-hydroxycalamenene are reported for the first time.

Aim: To study the chemical constituents of the seeds from Artabotrys hexapetalus (L.f.) Bhandari (Annonaceae)., Methods: Various chromatographic techniques were used to separate and purify the constituents. Their structures were elucidated on the physico-chemical properties and spectral data., Results: Eight compounds were isolated from the seeds of A. hexapetalus. They were identified as four neolignans: isoamericanin A (1), isoamericanol A (2), americanin B (3) and artabotrycinol (4), a semiterpenoid: (R)-artabotriol (5) and others: palmitic acid (6), beta-sitosterol (7) and daucosterol (8)., Conclusion: Artabotrycinol (4) and (R)-artabotriol (5) are new compounds. Three other neolignans were isolated from this plant for the first time.

Muricatenol (1) is a new C37 non-THF ring acetogenin with four hydroxyls and one isolated double bond in the long aliphatic chain. 2,4-cis-Gigantetrocinone (2) and 2,4-trans-gigantetrocinone (3) have been isolated as their acetates by preparative TLC. 2,4-trans-Isoannonacin-10-one (4) and 2,4-trans-isoannonacin (5) have been isolated as only 2,4-trans-form for the first time (no cis-form). Also four known acetogenins, gigantetrocin-A (6), gigantetrocin-B (7), annomontacin (8), gigantetronenin (9) and a mixture of N-fatty acyl tryptamines have been isolated (10). Their structures have been established on the basis of spectral analyses. The CHCl3 fraction of the seeds showed strong antitumor activities.

Aim: To study the chemical constituents of the rhizomes of Kaempferia galanga L. (Zingiberaceae)., Methods: Various chromatographic techniques were used to separate and purify. Their physico-chemical properties and spectral data were used to elucidate the structures., Results: Six compounds were isolated from the hexane extract of K. galanga. Their structures were identified as marginatol (1), 8 (14), 15-sanderacopimaradiene-1 alpha, 9 alpha-diol (2), 8 (14), 15-sanderacopimaradiene-1 alpha, 6 beta, 9 alpha-triol (3), germacrone (4), trans-ethyl p-methoxycinnamate (5) and n-pentadecane (6) on the basis of spectral data (IR, MS, 1H-1H, 13C-1H NMR, NOEs and HMBC)., Conclusion: Marginatol (1) is a new isopimarene diterpenoid and was elucidated as 8(14), 15-isopimaradiene-6 alpha-ol. Compounds 2, 3 and 4 were isolated for the first time from this plant.

Routine targeting of neurones for expression of exogenous genes would facilitate our ability to manipulate their internal milieu or functions, providing insight into physiology of neurones. The magnocellular neurones of the paraventricular and supraoptic nuclei of the hypothalamus have been the objects of limited success by this approach. Here we report on the placement of the enhanced green fluorescent protein (eGFP) coding sequence at various locations within an oxytocin transgene. Placement within the first exon yielded little to no expression, whereas placement in the third exon (as an in-frame fusion with the carboxyl terminus of the oxytocin preprohormone) resulted in cell-specific expression of eGFP in oxytocin neurones. Furthermore, placement of the eGFP sequence downstream of a picornavirus internal ribosomal entry site (IRES), also in the third exon, allowed expression of the eGFP as a separate protein. Other coding sequences should now be amenable to expression within oxytocin neurones to study their physiology.

Annonaceous acetogenin (or polyketide) is a kind of potential antineoplastic agents from Annonaceae plants. Two new acetogenins, Muricatalicin (I) and muricatalin (VI), a mesitoate of a new acetogenin, annonacin-B mesitoate (Vb), and three known acetogenins, annonacin (II), annonacin-A (III) and annonacin-10-one (IV) have been isolated from Annona muricata L. The structures and relative stereochemistry of I, VI and Vb were elucidated on the basis of spectral analysis and examination of their acetates and/or mesitoate.

Thirty children with daytime urinary frequency (DUFC) were randomly divided into two groups. The first group was given oral indomethacin, 1 mg/kg per dose, three times a day for 7 days. The frequency of urination fell from 26.3 +/- 2.3 to 11.7 +/- 2.8 at the end of the treatment period. The second group was treated with oral chloramphenicol, 30 mg/kg per day, in four divided doses for 7 days. Their urinary frequency did not change. They were then treated with a 1-week course of indomethacin and responded in a similar manner to group 1 patients. The use of indomethacin was accompanied by significant increases in plasma carbon dioxide-combining power, urine osmolality and urine pH; urine sodium and potassium levels decreased. These results show that a short course of indomethacin, in low dose, improves DUFC. We propose that it acts by inhibiting prostaglandin synthesis which modifies tubular function and increases urinary pH.

Giant-cell tumor of the sacrum is rare. There were only a few reports of large series in the literature. From 1960-1988 a total of ten cases giant-cell tumor of the sacrum were treated in our Department. The average age of the patients was 34 years (with range of 19-49 years). The lesions located in the proximal parts of the sacrums in 8 cases, and in S3-5 in another 2 cases. of 10, 7 were treated with en bloc resection or subtotal resection. of the sacrum, and followed by irradiation in 5 of 7 cases (with range of 2000r-4500r). All the 7 cases were followed up without any evidence of local recurrence or distant metastases. The average follow-up period was 10.3 years (with range of 1.5-27.5 years). The rectal, bladder and sexual function were normal or restored. The other 3 cases, biopsied in 2, partially resected in 1, died less than one year after diagnosis. Comparing the died group with the survival group, we concluded that early diagnosis and thorough excision of the tumor are the two decisive factors for a favorable prognosis.