Objective:To investigate the clinical experience and efficacy of unrelated umbilical cord blood hematopoietic stem cell transplantation (HSCT) for the treatment of congenital amegakaryocytic thrombocytopenia (CAMT).Methods:A case summary was conducted.The clinical data of 2 children with CAMT who were finally cured by unrelated umbilical cord blood HSCT in the Department of Pediatric Medicine, the First Affiliated Hospital of Zhengzhou University from March 2020 to August 2023 were retrospectively analyzed.Related studies were retrieved from databases CNKI, Wanfang and PubMed using search terms including " congenital amegakaryocytic thrombocytopenia" and " hematopoietic stem cell transplantation" from the inception to July 2024.The clinical characteristics, diagnosis and treatment processes, and prognosis of CAMT patients treated by HSCT were then summarized.Results:Both cases exhibited scattered skin haemorrhages throughout the body and carried 2 compound heterozygous mutations with pathogenicity in the MPL gene.Both patients were finally diagnosed with CAMT.Case 1 was a girl aged 3 at the time of transplantation, and case 2 was also a girl, who aged 5 at the time of transplantation.Both of them received unrelated umbilical cord blood HSCT and hematopoietic reconstruction was achieved.The time of neutrophil and platelet implantation was 21 and 40 days after transplantation in case 1, and 20 and 31 days in case 2, respectively.The chimerism rate of neutrophil implantation in both children was complete chimerism of donor cells.Implantation syndrome was detected in case 1 following transplantation.Case 2 suffered implantation syndrome, hypertensive encephalopathy, and cytomegalovirus infection following transplantation.Both children showed no graft-versus-host disease (GVHD).Both children had hematopoietic and immune reconstruction after transplantation and their primary diseases were cured.Cases 1 and 2 were followed up for more than 14 and 17 months, respectively.Both of them achieved disease-free survival during the follow up.Literature review of 26 cases with CAMT treated by HSCT, including the above-mentioned 2 cases was conducted, with an overall disease-free survival rate of 92.3%(24/26).Of 12 cases with CAMT typing, 10 were type Ⅰ and 2 were type Ⅱ.Of the 26 cases treated by HSCT, 17 had bone marrow HSCT, with an overall survival rate of 88.2%(15/17), and 2 had peripheral blood HSCT.Seven cases had umbilical cord blood HSCT (6 cases receiving unrelated umbilical cord blood HSCT and 1 case receiving related umbilical cord blood HSCT), with an overall survival rate of 100%.Unlike bone marrow and peripheral blood HSCT, unrelated umbilical cord blood HSCT did not result in 3-4 grade GVHD. Conclusions:Unrelated umbilical cord blood HSCT can achieve good therapeutic effects in CAMT patients when there is no suitable donor.Myeloablative pretreatment is conducive to CAMT patients.

Objective:To investigate the clinical experience and efficacy of unrelated umbilical cord blood hematopoietic stem cell transplantation (HSCT) for the treatment of congenital amegakaryocytic thrombocytopenia (CAMT).Methods:A case summary was conducted.The clinical data of 2 children with CAMT who were finally cured by unrelated umbilical cord blood HSCT in the Department of Pediatric Medicine, the First Affiliated Hospital of Zhengzhou University from March 2020 to August 2023 were retrospectively analyzed.Related studies were retrieved from databases CNKI, Wanfang and PubMed using search terms including " congenital amegakaryocytic thrombocytopenia" and " hematopoietic stem cell transplantation" from the inception to July 2024.The clinical characteristics, diagnosis and treatment processes, and prognosis of CAMT patients treated by HSCT were then summarized.Results:Both cases exhibited scattered skin haemorrhages throughout the body and carried 2 compound heterozygous mutations with pathogenicity in the MPL gene.Both patients were finally diagnosed with CAMT.Case 1 was a girl aged 3 at the time of transplantation, and case 2 was also a girl, who aged 5 at the time of transplantation.Both of them received unrelated umbilical cord blood HSCT and hematopoietic reconstruction was achieved.The time of neutrophil and platelet implantation was 21 and 40 days after transplantation in case 1, and 20 and 31 days in case 2, respectively.The chimerism rate of neutrophil implantation in both children was complete chimerism of donor cells.Implantation syndrome was detected in case 1 following transplantation.Case 2 suffered implantation syndrome, hypertensive encephalopathy, and cytomegalovirus infection following transplantation.Both children showed no graft-versus-host disease (GVHD).Both children had hematopoietic and immune reconstruction after transplantation and their primary diseases were cured.Cases 1 and 2 were followed up for more than 14 and 17 months, respectively.Both of them achieved disease-free survival during the follow up.Literature review of 26 cases with CAMT treated by HSCT, including the above-mentioned 2 cases was conducted, with an overall disease-free survival rate of 92.3%(24/26).Of 12 cases with CAMT typing, 10 were type Ⅰ and 2 were type Ⅱ.Of the 26 cases treated by HSCT, 17 had bone marrow HSCT, with an overall survival rate of 88.2%(15/17), and 2 had peripheral blood HSCT.Seven cases had umbilical cord blood HSCT (6 cases receiving unrelated umbilical cord blood HSCT and 1 case receiving related umbilical cord blood HSCT), with an overall survival rate of 100%.Unlike bone marrow and peripheral blood HSCT, unrelated umbilical cord blood HSCT did not result in 3-4 grade GVHD. Conclusions:Unrelated umbilical cord blood HSCT can achieve good therapeutic effects in CAMT patients when there is no suitable donor.Myeloablative pretreatment is conducive to CAMT patients.

Objective Polysaccharide is a major active component in Lycium barbarum.the structure and content of polysaccharide are different among different producing districts.At present,most of the quality control methods of Lycium barbarum polysaccharide construct fingerprint from the aspect of monosaccharide composition,but the quality control of Lycium barbarum polysaccharide only by analyzing monosaccharide composition can not fully explain the relationship between structure and Lycium barbarum polysaccharide.Therefore,we established the Needs methylation method of Lycium barbarum polysaccharide fingerprint to achieve quality control of Lycium barbarum from different places by determining the sugar residue linkage mode of Lycium barbarum polysaccharide on the basis of monosaccharide composition.Methods Lycium barbarum polysaccharide was extracted through water extraction followed by alcohol precipitation,and the Needs methylation,complete acid hydrolysis,reduction by NaBH4,acetylation and other methods combined with GC-MS to determine the sugar residue linkage motif of Lycium barbarum polysaccharide.Result The chromatograms of 18 batches of Lycium barbarum polysaccharides were imported into the Chinese Medicine Chromatographic Fingerprint Similarity Evaluation System(2004 edition),combined with chemometrics to evaluate the differences of Lycium barbarum polysaccharide from different origin.The result of similarity showed that the similarity of three batches of Lycium barbarum polysaccharide from Tibet ranged from 0.551 to 0.569,others were more than 0.929.16 common peaks were marked in chromatogram of sugar residue linkage mode,and 10 of them was identified,they are T-Arap,T-Araf,T-Xylp,1,2-Arap,1,3-Rhap,1,5-Araf,T-Glcp,T-Galp,1,4-Glcp,1,6-Galp.The results of HCA,PCA and PLS-DA analysis divided 18 bathes of Lycium barbarum polysaccharide were divided into three categories,and three signature components were screened,which were T-Araf,1,5-Araf and T-Glcp,to judge the differences of Lycium barbarum polysaccharides from different places.Conclusion The Needs methylation fingerprint of 18 batches of Lycium barbarum polysaccharide was established for the first time.The establishment of the fingerprint can provide experimental data reference for the quality control of Lycium barbarum polysaccharide,and further prove the role of polysaccharide in the quality control of Traditional Chinese Medicine.

OBJECTIVE To study the sedative and hypnotic effects of zolpidem and the content of amino acid neurotransmitters in the thalamus and hypothalamus after treatment with zolpidem.METHODS Experiments on the loss of righting reflex(LORR)induced by the upper-threshold dose pentobarbital sodium(50 mg·kg-1,ip)were conducted to establish a hypoxic insomnia model in mice by simulating an altitude of 5500 m.Based on this model,the synergistic effect of zolpidem(0.33,1,3,9 and 27 mg·kg-1,ip)and the subthreshold(20 mg·kg-1,ip)and upper-threshold pentobarbital sodium,as well as the sedative hypnotic effect of zolpidem(10,13,17,20,23,30 and 40 mg·kg-1,ip)were evaluated via the LORR in normoxic and hypoxic environments.One hour after ip given zolpidem,the levels of glutamic acid(Glu)and γ-aminobutyric acid(GABA)in the thalamus and hypothalamus of mice in either environment were determined by the high-performance liquid chromatography(HPLC)with fluorescence detection.RESULTS One-day treatment with hypoxia significantly shortened the duration of LORR induced by the upper-threshold dose pentobarbital sodium.Compared with normoxia vehicle and hypoxia induced insomnia vehicle groups,zolpidem 9 and 27 mg·kg-1 significantly shortened the latency to LORR(P<0.01,P<0.05)and prolonged duration of LORR induced by subthreshold and upper-threshold pentobarbital sodi-um(P<0.01,P<0.05).The median effective dose(ED50)of LORR induced by zolpidem was 16.21 and 20.55 mg·kg-1 in normoxic and hypoxic environments,respectively.The results of neurotransmitter level detection showed that Glu contents in the thalamus and hypothalamus and the ratio of Glu/GABA in the hypothalamus were decreased after treatment with zolpidem 40 mg·kg-1 in a normoxic environment(P<0.01,P<0.05).Compared with the normoxia control group,Glu content and the ratio of Glu/GABA in the hypothalamus were significantly increased after treatment with hypoxia(P<0.01,P<0.05),and zolpidem 40 mg·kg-1 could reverse their elevation.CONCLUSION The sedative-hypnotic effect of zolpidem is weakened in a hypoxic environment,and the effect of zolpidem on the levels of Glu and GABA in the hypothalamus may play an important role in the sedative-hypnotic effect of zolpidem.

Objective: Danhong injection (DHI) is commonly used in China to treat chronic stable angina (CSA), demonstrating significant clinical efficacy and safety. The present study aimed to estimate the cost-effectiveness of DHI combined with standard treatment (ST) compared with ST alone in patients with CSA. Methods: A Markov model with a 3-month cycle and a 1-year time horizon was used to assess the cost-effectiveness of DHI + ST for CSA. Health outcomes were measured as quality-adjusted life years (QALYs), and societal perspective costs were collected in China. Cost-effectiveness was evaluated using the incremental cost-effectiveness ratio (ICER). Sensitivity analysis was performed to test the robustness of results to parameter uncertainty. Results: Analysis demonstrated DHI + ST provided 0.8190 QALYs at an average cost of $3644.24, while ST yielded 0.7884 QALYs costing $2739.54 over 1 year. The incremental analysis indicated DHI + ST dominance, with an ICER of $29,588.91 per QALY gained compared with ST. One-way sensitivity analysis confirmed the robustness of base case findings, and probabilistic sensitivity analysis suggested a 61.66% probability of DHI + ST being cost-effective over ST at a $37,663.26 per QALY threshold. Conclusion: From a societal perspective in China, DHI + ST appears cost-effective for the treatment of CSA compared with ST alone. These findings support recommending DHI as a clinically valuable and cost-effective option in CSA treatment guidelines.

Objective:To investigate the changes of intestinal mucosal barrier function related indexes [diamine oxidase and secretory immunoglobulin A (sIgA)] in peripheral blood of patients with rheumatoid arthritis (RA) and their correlation with peripheral immune function.Methods:A total of 40 patients with RA who admitted to the Rheumatology and Immunology department of the Second Hospital of the Shanxi Medical University were enrolled. We collected their clinical and laboratory data, and selected 20 age and gender matched people as the control group. Enzyme-linked immunosorbnent assay (ELISA) was used to detect the level of DAO and sIgA in the peripheral blood, the lymphocytes and CD4 + T subsets were detected by flow cytometry. Then t-test, rank sum test and correlation analysis were conducted for statistical analysis. Results:① The level of DAO in patients with RA was higher than that of healthy controls [205(164, 251) ng/ml vs 364 (276, 483) ng/ml, Z=-4.48, P<0.001], while the level of sIgA was decreased [3.64 (2.76, 4.83)×10 5 ng/ml vs 6.83 (4.80, 9.44)×10 5, Z=-3.84, P<0.001]. ② The absolute number of B and CD4 + T cells were increased in RA, the difference were statistically significant, but the absolute number of T, natural killer cells (NK) and CD8 + T cells were not significantly different between the two groups. For CD4 + T subsets, the absolute number of T helper cells (Th)1 and Treg cells in RA group were significantly decreased than healthy controls, but there were no statistical significant difference in the number of Th2 and Th17 cells. ③ The level of DAO was positively correlated with absolute number of Th17 cells in patients with RA ( r=0.36 P=0.038), and positively correlated with age and white blood cell count ( r=0.40, P=0.021; r=0.40, P=0.020), but no significant correlation among other indicators were found. ④ The serum sIgA level of RA patients in antimutated citrullinated vimentin antibody (MCV), antiperinuclear factor (APF) and antikeratin antibody (AKA) positive group were higher than those in the negative group [3.99(2.99, 5.58)×10 5 ng/ml vs 2.73(2.29, 3.05)×10 5 ng/ml, Z=-2.55, P=0.011; 5.49 (3.26, 5.70)×10 5 ng/ml vs 3.12 (2.29, 4.04)×10 5 ng/ml, Z=-2.28, P=0.023; 4.07 (3.19, 5.65)×10 5 ng/ml vs 2.88 (2.24, 3.86)×10 5 ng/ml, Z=-2.42, P=0.016], while there was no significant difference in DAO level between groups. ⑤ The DAO level of RA patients with pulmonary interstitial fibrosis was significantly higher than that in the group without pulmonary interstitial fibrosis [421 (216, 528) ng/ml vs 191 (150, 223) ng/ml, Z=-2.81, P=0.005], while there were no significant differences in DAO and sIgA levels among other groups. Conclusion:In RA patients with inte-stinal mucosal barrier impairment, the DAO level is increased, while the sIgA is decreased, and in addition, elevated peripheral blood Th17 may be involved in the process of intestinal mucosal barrier impairment.

The correlation between transient receptor potential vanilloid-1 (TRPV1) receptor and peripheral nervous system diseases has been confirmed. In recent years, studies have shown that TRPV1 plays an important role in the central nervous system; its wide distribution in the hippocampus, cortex, and midbrain bridges the correlation between TRPV1 receptor and cognitive learning and memorial function. This paper reviews the biological role of TRPV1 receptor in the nervous system at home and abroad and concludes the relations of TRPV1 receptor with cognitive associated diseases, aiming at providing theory basis for whether TRPV1 receptors can be used as therapeutic drug targets for cognitive associated diseases.

Objective To explore the efficacy and prognostic factors of chemotherapy regimens including decitabine in treatment of elderly patients newly diagnosed with acute myeloid leukemia (AML). Methods The clinical data of 47 elderly patients newly diagnosed with AML (except M3) who received chemotherapy regimens including decitabine in the First Hospital of Jilin University from February 2013 to November 2017 were retrospectively analyzed, including 11 patients treated with single decitabine and 36 patients treated with decitabine combined with low_dose chemotherapy group. The treatment outcome and the impact of different factors on the prognosis were also analyzed. Results Of 47 patients, there were 15 males and 32 females, and the median age was 65 years old (60-83 years old). The overall response rate of decitabine plus low_dose chemotherapy group for 1 course was higher than that of single decitabine group [80.6% (26/36) vs. 27.3% (3/11), χ 2 ＝ 8.693, P＝ 0.003], and the former showed less courses to acquire remission than the latter (u＝ 3.133, P＝ 0.002); however, there was no significant difference in the median overall survival (OS) time between the two groups (14 months vs. 12 months, P＝ 0.950). Univariate analysis indicated that the median OS time in the complete remission (CR) group was longer than that in the non_CR group (17 months vs. 5 months, P <0.01). The median OS time of the elderly patients with primary AML was longer than that of the patients with secondary AML (16 months vs. 6 months, P＝ 0.01). Cox multifactor analysis showed that failing to achieve CR was identified as an independent adverse influencing factor ( HR＝0.180, 95% CI 0.085-0.382, P< 0.01). The incidence of neutropenia with fever in the patients treated with decitabine plus low_dose chemotherapy group was higher than that in single decitabine group [69.4% (25/36) vs. 36.4% (4/11), χ2＝3.902, P＝0.048]. Conclusion For newly elderly AML patients, chemotherapy regimens including decitabine are safe and effective.

Objective Toinvestigatetheclinicalvalueofreadoutsegmentationoflongvariableecho-trainsdiffusion-weightedimaging (RESOLVE-DWI)inthediagnosisandstagingofthyroid-associatedophthalmopathy(TAO).Methods Atotalof30consecutivepatientswith TAOand30healthycontrols(HCs)whounderwentRESOLVE-DWIwereenrolledinourstudy.ADCvaluesofextraocularmuscles (superiorrectus,inferiorrectus,medialrectusandlateralrectus)were measuredandcomparedbetween TAOsand HCs,active TAOsandinactiveTAOs,orinactiveTAOsandHCs.ROCanalysiswasperformedtoevaluatethediagnosticvalueofsignificantparametersfor discriminatingactivefrominactiveTAOs.Results TheADCvaluesofallextraocularmusclesinTAOsweresignificantlyhigherthan thoseinHCs(P<0.05).Meanwhile,alltheextraocularmusclesinactiveTAOsshowedsignificantlyhigherADCvaluesthanthose ininactiveTAOs(P<0.05),exceptlateralrectus(P=0.267).WhilstnosignificantdifferenceswerefoundontheADCvaluesofall extraocularmusclesbetweeninactiveTAOsandHCs(P>0.05).ROCanalysisresultsindicatedthattheADCvalueofmedialrectus showedtheoptimalstagingefficacy(cutoffvalue,1.40×10-3 mm2/s;AUC,0.766;sensitivity,92.1%;specificity,59.1%).Conclusion RESOLVE-DWIanditsderivedADCvaluesofextraocularmusclescanassistinthediagnosisofTAO.TheADCvalueofmedial rectushastheoptimalefficacyontheevaluationofitsclinicalactivity.

Objective To analyze the relationship of neurocognitive impairments and 1 H MRS changes of the thalamus in patients with chronic hepatitis B virus related cirrhosis (HBV-RC).Methods Totally 28 patients with HBV-RC (cirrhosis group) and 28 well-matched healthy subjects (control group) were enrolled.All subjects underwent number connection test A (NCT-A) and the digit symbol test (DST) before MRS scanning.The ratios of peak area to each metabolite,including N-acetylaminosuccinic acid (NAA),choline (Cho),glutamine and glutamate (Glx),myoinositol (mI) and creatine (Cr) were calculated,respectively.Results Compared with control group,patients in cirrhosis group showed lower Cho/Cr and mI/Cr,higher Glx/Cr,prolonged NCT-A time and decreased DST scores (all P＜0.001).NCT-A completion time was negatively correlated with Cho/Cr and mI/Cr (r=-0.477,P =0.001;r=-0.695,P＜0.001) and positively correlated with Glx/Cr (r=0.665,P＜0.001).DST scores were positively correlated with Cho/Cr and mI/Cr (r =0.478,P =0.001;r=0.632,P＜0.001),and negatively correlated with Glx/Cr (r=-0.572,P＜0.001).Conclusion The neurocognitive impairments may be related to metabolic changes of the thalamus in patients with HBV RC.