Therapeutic efficacy of hematopoietic stem cell transplantation for Wiskott-Aldrich syndrome in 60 children / 中华儿科杂志

Objective: To evaluate the therapeutic efficacy of hematopoietic stem cell transplantation (HSCT) for Wiskott-Aldrich syndrome (WAS), and to analyze the factors related to the outcomes. Methods: The clinical data of 60 children with WAS received HSCT in Shanghai Children's Medical Center from January 2006 to December 2020 were retrospectively analyzed. All cases were treated with a myeloablative conditioning regimen with busulfan and cyclophosphamide, and a graft-versus-host disease (GVHD) prevention regimen based on cyclosporine and methotrexate. Implantation, GVHD, transplant-related complications, immune reconstitution and survival rate were observed. Survival analysis was performed by Kaplan-Meier method, and Log-Rank method was used for univariate comparison. Results: The 60 male patients had main clinical features as infection and bleeding. The age at diagnosis was 0.4 (0.3, 0.8) years, and the age at transplantation was 1.1 (0.6, 2.1) years. There were 20 cases of human leukocyte antigen matched transplantation and 40 mismatched transplantation; 35 patients received peripheral blood HSCT, and 25 cord blood HSCT. All cases were fully implanted. The incidence of acute GVHD (aGVHD) was 48% (29/60) and only 2 (7%) developed aGVHD of grade Ⅲ; the incidence of chronic GVHD (cGVHD) was 23% (13/56), and all cases were limited. The incidence of CMV and EBV infection was 35% (21/60) and 33% (20/60) respectively; and 7 patients developed CMV retinitis. The incidence of sinus obstruction syndrome was 8% (5/60), of whom 2 patients died. There were 7 cases (12%) of autoimmune hemocytopenia after transplantation. Natural killer cells were the earliest to recover after transplantation, and B cells and CD4+T cells returned to normal at about 180 days post HSCT. The 5-year overall survival rate (OS) of this group was 93% (95%CI 86%-99%), and the event free survial rate (EFS) was 87% (95%CI 78%-95%). EFS of non-CMV reactivation group is higher than that of CMV reactivation group (95% (37/39) vs.71% (15/21), χ2=5.22, P=0.022). Conclusions: The therapeutic efficacy of HSCT for WAS is satisfying, and the early application of HSCT in typical cases can achieve better outcome. CMV infection is the main factor affecting disease-free survival rate, which can be improved by strengthening the management of complications.

Cell Apoptosis Mechanism and Traditional Chinese Medicine Intervention in Diabetic Peripheral Neuropathy： A Review / 中国实验方剂学杂志

Diabetic peripheral neuropathy is a common complication of diabetes， and its pathogenesis is complex. Its high morbidity can result in disability， teratogenesis， and death in diabetic patients. At present， the pathogenesis of diabetic peripheral neuropathy has not been clearly elucidated， which may be related to oxidative stress， inflammatory response， microcirculation dysfunction， metabolic abnormalities， etc. Recent studies have found that apoptosis plays an important role in the pathogenesis of diabetic peripheral neuropathy. The three pathways， i.e.， mitochondrial pathway， death receptor pathway， and endoplasmic reticulum pathway， jointly regulate the cell apoptosis in the body. Traditional Chinese medicine， with definite efficacies in the treatment of diabetic peripheral neuropathy， is advantageous in overall regulation and multi-target and multi-pathway treatment. As reported， the active ingredients in Chinese medicine and Chinese medicinal compounds can alleviate diabetic peripheral neuropathy by regulating apoptosis signaling pathways. Furthermore， apoptosis pathways are expected to be potential targets for new drugs against diabetic peripheral neuropathy following oxidative stress. Therefore， this paper， taking apoptosis as the entry point， reviewed the research progress on TCM intervention in diabetic peripheral neuropathy in recent years to provide references for the clinical prevention and treatment of diabetic peripheral neuropathy and the development of new drugs.

Regulatory Effect of Quyu Huatan Tongmai Prescription on Intestinal Microflora in Golden Hamster with Hyperlipidemia / 中国实验方剂学杂志

ObjectiveTo explore the regulatory effect of Quyu Huatan Tongmai prescription on intestinal mircoflora of hyperlipidemia golden hamster and scientific evidence for the compatibility. MethodSyrian golden hamsters were randomized into normal， model， prescription， stasis-dispelling （Quyu）， phlegm-dissolving （Huatan）， and detoxification （Jiedu） groups， with 8 in each group. Hyperlipidemia in golden hamsters was induced by high-fat diet （4 weeks）. Then hamsters in the Quyu group （1.11 g·kg-1）， Huatan group （0.39 g·kg-1）， Jiedu group （0.07 g·kg-1）， and prescription group （1.42 g·kg-1） were given （ig） corresponding drugs and those in the normal group and the model group received （ig） distilled water of equivalent volume， once a day for 6 weeks. Serum lipids were determined， and hematoxylin-eosin （HE） staining was used to observe the pathological morphology of the liver. Feces were collected for 16S rRNA gene high-throughput sequencing of intestinal flora. ResultCompared with normal group， the model group demonstrated increase in body weight （P<0.05， P<0.01） and blood lipids （P<0.01）， decrease in intestinal flora diversity （P<0.05， P<0.01）， and variation of the relative abundance of intestinal flora at phylum， family， and genus levels （P<0.05， P<0.01）. Compared with the model group， Quyu Huatan Tongmai prescription controlled the body weight change， reduced the serum triglyceride （TG）， total cholesterol （TC）， and low density lipoprotein cholesterol/high density lipoprotein cholesterol ratio （LDL-C/HDL-C） （P<0.05， P<0.01）， improved the structure of intestinal flora， decreased the ratio of Firmicutes to Bacteroides （P<0.01）， raised the abundance of Bacteroidaceae， Porphyromonadaceae， Rikenellaceae， and Pasteurella （P<0.05， P<0.01）， and lowered the relative abundance of Coriobacterium （P<0.05） in hyperlipidemia golden hamsters. All the split prescriptions improved blood lipids and intestinal flora of the hamsters and particularly， the lipids-lowering effect of the Jiedu group and the regulation of flora in the Huatan group were closer to those of the prescription group. ConclusionQuyu Huatan Tongmai prescription and the split prescriptions all alleviated the hyperlipidemia of golden hamsters to different degrees possibly by regulating intestinal flora structure and improving intestinal microecology. The effect of the prescription group was most significant， and coming in second was the Huatan group. This study also provides scientific evidence for the effect of Quyu Huatan Tongmai prescription.

Quantitative analysis of macular structure and microvascular changes in patients with diabetic retinopathy at different stages / 国际眼科杂志(Guoji Yanke Zazhi)

@#AIM: To observe and quantitatively analyze the thickness of macular ganglion cell inner plexiform layer(GCIPL)and the characteristics of superficial retinal capillaries vessel density in different stages of diabetic retinopathy(DR)by optical coherence tomography(OCT)and optical coherence tomography angiography(OCTA).<p>METHODS: A retrospective case-control study. Thirty-three patients with diabetic(54 eyes)were selected as the DR group from December 2019 to May 2020. Among them, six patients(8 eyes)as non-diabetic retinopathy(NDR)group, eighteen patients(28 eyes)as non-proliferative diabetic retinopathy(NPDR)group and nine patients(18 eyes)as proliferative diabetic retinopathy(PDR)group according to fundus conditions. Eighteen healthy volunteers(26 eyes)without eye disease were selected as the normal group. The macular GCIPL thickness and the values of vascular linear density(vascular density, VD)and density of vascular perfusion(perfusion density, PD)in the superficial retinal capillaries vessels in each quadrant of macular region were observed and quantitatively analyzed in DR patients with different stages. <p>RESULTS: The VD, PD and minimum thickness of GCIPL in each quadrant of DR group was lower than that of the healthy control group(<i>P</i><0.05). The minimum thickness of GCIPL in macular area and the VD of superficial retinal capillaries in each quadrant decreased significantly in patients with different stages of diabetic retinopathy(<i>P</i><0.01). The inferior VD of superficial retinal capillaries vessels had the highest diagnostic value for DR(AUC=0.807, optimal diagnostic threshold value of 18.60 mm-1, sensitivity of 0.923, specificity of 0.648). The minimum thickness of GCIPL in macular area of DR patients was positively correlated with VD of superficial retinal capillaries vessels in each quadrant(<i>r</i>=0.342, 0.480, 0.384, 0.342, all <i>P</i><0.05). <p>CONCLUSION: OCT combined with OCTA can provide repeatable and quantifiable detection methods and monitoring indicators for early assessment and regular follow-up of DR progress.

Efficacy and safety of Shenyankangfu Tablet, a Chinese patent medicine, for primary glomerulonephritis: A multicenter randomized controlled trial / 中西医结合学报

BACKGROUND@#Shenyankangfu Tablet (SYKFT) is a Chinese patent medicine that has been used widely to decrease proteinuria and the progression of chronic kidney disease.@*OBJECTIVE@#This trial compared the efficacy and safety of SYKFT, for the control of proteinuria in primary glomerulonephritis patients, against the standard drug, losartan potassium.@*DESIGN, SETTING, PARTICIPANTS AND INTERVENTION@#This was a multicenter, double-blind, randomized, controlled clinical trial. Primary glomerulonephritis patients, aged 18-70 years, with blood pressure ≤ 140/90 mmHg, estimated glomerular filtration rate (eGFR) ≥ 45 mL/min per 1.73 m@*MAIN OUTCOME MEASURES@#The primary outcome was change in the 24-hour proteinuria level, after 48 weeks of treatment.@*RESULTS@#A total of 735 participants were enrolled. The percent decline of urine protein quantification in the SYKFT group after 48 weeks was 8.78% ± 2.56% (P = 0.006) more than that in the losartan 50 mg group, which was 0.51% ± 2.54% (P = 1.000) less than that in the losartan 100 mg group. Compared with the losartan potassium 50 mg group, the SYKFT plus losartan potassium 50 mg group had a 13.39% ± 2.49% (P < 0.001) greater reduction in urine protein level. Compared with the losartan potassium 100 mg group, the SYKFT plus losartan potassium 100 mg group had a 9.77% ± 2.52% (P = 0.001) greater reduction in urine protein. With a superiority threshold of 15%, neither was statistically significant. eGFR, serum creatinine and serum albumin from the baseline did not change statistically significant. The average change in TCM syndrome score between the patients who took SYKFT (-3.00 [-6.00, -2.00]) and who did not take SYKFT (-2.00 [-5.00, 0]) was statistically significant (P = 0.003). No obvious adverse reactions were observed in any group.@*CONCLUSION@#SYKFT decreased the proteinuria and improved the TCM syndrome scores of primary glomerulonephritis patients, with no change in the rate of decrease in the eGFR. SYKFT plus losartan potassium therapy decreased proteinuria more than losartan potassium therapy alone.@*TRIAL REGISTRATION NUMBER@#NCT02063100 on ClinicalTrials.gov.

Application value of magnetic resonance hydrography of the inner ear in cochlear implantation

SUMMARY OBJECTIVE This study aims to investigate the application value of magnetic resonance (MR) hydrography of the inner ear in cochlear implantation. METHODS 146 patients were enrolled. MR hydrography and spiral CT examinations for the intracranial auditory canal were performed before surgery, and all imaging results were statistically analyzed in order to explore the application value of MR hydrography of the inner ear in cochlear implantation. RESULTS 146 patients (292 ears) were examined. Among these patients, 13 were diagnosed with abnormal vestibular aqueducts (20 ears) by MR hydrography, while five were diagnosed with this disease by CT; 15 patients were diagnosed with inner ear malformation (19 ears) by MR hydrography, while 11 were diagnosed by CT (four were misdiagnosed); five patients were diagnosed with internal acoustic canal stenosis (eight ears) by MR hydrography, while two were diagnosed by CT (three were misdiagnosed); and four patients were diagnosed with cochlear fibrosis (five ears) by MR hydrography, while four were diagnosed by CT (four ears). The correct rate of diagnosis was 77.40% (113/146) based on CT, while the rate was 93.84% (137/146) based on MR hydrography. CONCLUSIONS MR hydrography imaging technique can be applied to the preoperative evaluation of cochlear implantation, providing accurate and reliable anatomic information on the inner membranous labyrinth and nerves in the internal acoustic canal and an accurate basis for the diagnosis of cochlear fibrosis and nerve development. This has a guiding significance for the selection of treatment schemes.

RESUMO OBJETIVO Este estudo visa investigar o valor da aplicação da hidrografia por ressonância magnética (RM) do ouvido interno no implante coclear. MÉTODOS Cento e quarenta e seis pacientes foram inscritos. Os exames da hidrografia por RM e do CT espiral para o canal auditivo intracraniano foram executados antes da cirurgia, e todos os resultados da imagem foram analisados estatisticamente, a fim de explorar o valor da aplicação da hidrografia por RM do ouvido interno no implante coclear. RESULTADOS Centro e quarenta e seis pacientes (292 ouvidos) foram examinados. Dentre esses pacientes, 13 foram diagnosticados com aquedutos vestibulares anormais (20 ouvidos) pela hidrografia por RM, enquanto cinco pacientes foram diagnosticados com esta doença pelo CT; 15 pacientes foram diagnosticados com malformação do ouvido interno (19 ouvidos) pela hidrografia por RM, enquanto 11 pacientes foram diagnosticados por CT (quatro foram diagnosticados erroneamente); cinco pacientes foram diagnosticados com estenose de canal acústico interno (oito ouvidos) pela hidrografia por RM, enquanto dois pacientes foram diagnosticados por CT (três foram diagnosticados erroneamente); e quatro pacientes foram diagnosticados com fibrose coclear (cinco ouvidos) pela hidrografia por RM, enquanto quatro foram diagnosticados por CT (quatro ouvidos). A taxa correta de diagnóstico foi de 77,40% (113/146) com base no CT, enquanto a taxa foi de 93,84% (137/146) com base na hidrografia por RM. CONCLUSÕES A técnica de imagem da hidrografia por RM pode ser aplicada à avaliação pré-operatória do implante coclear, que pode fornecer informações anatômicas precisas e confiáveis sobre o labirinto membranoso interno e os nervos no canal acústico interno, além de uma base exata para o diagnóstico da fibrose coclear e do desenvolvimento do nervo. Isso tem um significado orientador para a seleção de esquemas de tratamento.

Investigation on Community-based Rehabilitation Resources in Shanghai, China / 中国康复理论与实践

Objective:To investigate the rehabilitation resources of community health centers in Shanghai. Methods:December, 2018, all 247 community health centers in 16 districts of Shanghai were investigated with Health Institution Questionnaires, including the number of rehabilitation personnel (physiatrician, physical therapists) and the number of rehabilitation beds, etc. Results:There were 152 physiatrician and 597 physical therapists in community health centers in Shanghai. The numbers of physiatrician and physical therapists per 1000 registered population were 0.01 and 0.04, and were 0.03 and 0.12 for per 1000 registered elderly population. There were 17 484 beds in community health centers, in which the rehabilitation beds were 1425, accounting for 8.15%. Besides, the numbers of rehabilitation beds per 1000 registered population and registered elderly population in Shanghai were 0.10 and 0.30. Conclusion:The development of community-based rehabilitation in Shanghai is unbalanced. There is lack of community-based rehabilitation resources, the supply of service is insufficient, and there is gap between supply and demand. It is suggested to strengthen the support to community-based rehabilitation, and improve the supply of community-based rehabilitation resources.

Alternative donor HSCT for 109 children with acquired severe aplastic anemia: a single center retrospective analysis / 中华血液学杂志

Objective: To investigate the efficacy of alternative donor (AD) in the treatment of aplastic anemia (AA) in children. Methods: The clinical data of AA children who received AD HSCT in our center from Apr. 2010 to Dec. 2016 were retrospectively analyzed. The overall survival (OS) rate, implant success rate, incidence of acute and chronic graft-versus-host disease (GVHD) were statistically analyzed. Results: A total of 109 children with acquired AA, including 64 severe AA (SAA) , 32 very severe AA (VSAA) and 13 transfusion dependent non-severe AA (NSAA) , were recruited in this retrospective AD HSCT study, the median age was 6 (0.8-18) years old. Of them, 44 patients with 10/10 matched unrelated donor (MUD) , 44 patients with mismatched unrelated donor (MMUD) and 21 patients with mismatched related donor (MMRD) . All patients did not receive ATG before HSCT and the active infection was excluded. Except 3 patients suffered from a second graft failure (2 of them rescued by second HSCT) , 106/109 (97.2%) were engrafted with neutrophil and platelet recovery occurring at a median of 13 days (range, 9-19) and 16 days (range, 10-81) post-transplant. Until day 100 post transplantation, the incidence was 74.3% (81/109) for acute GVHD (aGVHD) and 39.4% (43/109) for grade Ⅱ-Ⅳ aGVHD, 30.7% (31/101) and 9.9% (10/101) for overall chronic GVHD (cGVHD) and moderate cGVHD, respectively, and nobody developed an extend cGVHD. After median follow up of 39 (0.7-103) months for all patients, 13 of 109 patients died. The estimated 5-year overall survival (OS) of the entire cohort was 88.1% (95%CI 81.1%-91.4%) with no difference among the MUD, MMUD and MMRD cohort (93.2%, 84.1% and 85.7%, respectively, P=0.361) . Conclusion: These excellent outcomes suggest that unmanipulated AD PBSC is a good HSCT source for children with SAA. It's reasonable to consider AD HSCT as first line therapy for SAA children without matched sibling donor. Better strategies are required to prevent GVHD.

Metabonomics Analysis of Sailuotong Capsules in Treatment of Acute Cerebral Ischemia in Rats / 中国实验方剂学杂志

Objective:To explore the mechanism of Sailuotong capsules in treating acute cerebral ischemia from the perspective of metabonomics. Method:A total of 24 SD rats were randomly divided into 3 groups, including sham-operated group, model group and Sailuotong group (33 mg·kg-1). The rat model of acute multiple cerebral infarction was established by injecting fluorescent microspheres into internal carotid artery. After the successful operation, rats in Sailuotong group were administered by duodenal injection immediately, and the dosage volume was 2 mL·kg-1. Endogenous metabolites in rat brain tissues of each group were determined by UPLC-Q-TOF-MS. The relevant data and biomarkers were analyzed by principal component analysis (PCA) and partial least squares-discriminant analysis (PLS-DA). Result:The analysis of pattern recognition indicated that the metabolite profiles in model group and sham-operated group were separated obviously, and ten biomarkers related to acute cerebral ischemia were also identified. Compared with the sham-operated group, contents of N-acetylaspartate (NAA), fumaric acid, glutathione, dehydroascorbic acid, aspartic acid and S-adenosylhomocysteine were decreased, while the contents of arginine, citrulline, saccharopine and hydantoin-5-propionic acid were increased in the model group. Meanwhile, the ten abnormal biomarkers mentioned above got restoration in Sailuotong group. Conclusion:The main regulated metabolic pathways of Sailuotong capsules are NAA metabolism, arginine metabolism, energy metabolism, oxidative stress, etc.

Predictive value of rs2200733 polymorphism for atrial fibrillation recurrence after radiofrequency catheter ablation / 医学研究生学报

Objective The recurrence rate of atrial fibrillation (AF) after radiofrequency catheter ablation (RFCA) remains relatively high. The aim of this study was to investigate the predictive value of rs2200733 polymorphism for AF recurrence after RFCA. Methods Fifty-three AF patients underwent RFCA guided by the magnetic navigation system between July 2015 and September 2016 in Wuxi People’s Hospital. We obtained the baseline data on the patients, conducted genotyping for rs2200733 variants, and followed up the patients for symptoms and complications by electrocardiography (ECG) and dynamic ECG. Using Cox survival analysis, we determined the independent predictors of AF recurrence after RFCA and the sensibility and specificity of predicting AF recurrence at 12 and 24 months post-operatively. Results All the patients were Han Chinese, followed-up for 21.6 ± 9.5 months, and 25 (47.2%) of them experienced AF recurrence at 6.6 ± 5.3 months after RFCA. Kaplan-Meier survival analysis revealed a significant association between rs2200733 polymorphism and AF recurrence in the additive and recessive models (P < 0.001), and multivariate Cox analysis showed the rs2200733 polymorphism (recessive model) to be an independent predictor of post-RFCA AF recurrence (OR = 3.184, 95% CI: 1.378-7.357, P = 0.007). The sensitivity, specificity, positive predictive value, negative predictive value, and accuracy of rs2200733 TT in predicting AF recurrence at 12 months were 64%, 81%, 70%, 76% and 74%, and those at 24 months were 60%, 82%, 75%, 70%, and 72%, respectively. Conclusion The rs2200733 polymorphism is an independent predictor of AF recurrence after RFCA, and its high specificity indicates that it could be used as a tool for screening Han Chinese patients with AF for RFCA.

Health economics evaluation on rehabilitation mode of integrated traditional Chinese and western medicine applied in community / 预防医学

Objective To observe the advantages and disadvantages the combination of traditional Chinese medicine and western medicine in community health service centers through cost-effectiveness and cost-utility analysis.Methods Take community common diseases as an example, Knee Osteoarthritis (KOA), Cervical Spondylosis (CS) and Lumbar Disc Herniation (LDH) were used. A total of 200 cases were selected for each disease, and 100 cases in each one group.In the treatment group, the rehabilitation mode was used.In the control group, comprehensive treatment of traditional Chinese medicine was adopted. We analyzed the cost-effectiveness and cost-utility, with VAS as the effect indicator and QALYs as the utility indicator. Results The VAS values of the knee osteoarthritis, cervical spondylosis and lumbar disc herniation in the treatment group were lower than those in the control group after 14 days, 30 days and 6 months (P<0.05) . The QALYs values of them in the treatment group were lower than those in the control group after 14 days, 30 days and 6 months (P<0.05) . The treatment group has a higher increase cost-effectiveness ratio (ICER) . Each get a unit effect can pay 222.56, 297.13 and 1178.59 Yuan less . The treatment group has a higher increase cost-utility ratio (ICUR) . Each get a unit effect can pay 741.86, 3178.60 and 2862.29 Yuan less . Conclusion Community application in rehabilitation mode of integrated traditional Chinese and western medicine can improve the treatment effect, and can reduce the cost of treatment and the loss of QALYs.

Pharmacokinetics research of Tianlongtongxin formula in rat / 药学学报

Tianlongtongxin (TLTX) formula is composed of six Chinese herbs including Rhodiola rosea L., Salviae Miltiorrhizae Radix et Rhizoma, Chuanxiong Rhizoma and so on. It has been mainly used in the treatment of chest-Bi syndrome in the clinics. To investigate the material foundation and provide reference for clinical dosage regimen, the pharmacokinetics of seven components in the rat plasma were studied after oral administration of TLTX. A high sensitive method was established to determine the seven active components from TLTX in rat plasma based on the LC-MS/MS technique. The method met the requirements of preclinical pharmacokinetic study, through the investigation of linearity, specificity, recovery, accuracy, precision and stability. After administration of TLTX at 4.5 g·kg-1 dose, all of the components were detectable in the plasma after 5 min. The concentration peaks were observed at 0.11-4.67 h respectively after administration with great difference in levels. The AUC of salidroside was significantly higher than other components, suggesting it as a main active component in TLTX formula. The observations provide scientific evidence for the rationality of salidroside as monarch drug in the formula.

Lyme Borreliosis-associated Risk Factors in Residents of Beijing Suburbs: a Preliminary Case-control Study / 生物医学与环境科学（英文）

A population-based case-control study was conducted to evaluate the relative factors in the environments, agricultural works, outdoor activities, and the effectiveness of Lyme borreliosis (LB)- associated personal protective measures in Beijing. Thirty-four cases and 272 controls were personally interviewed by well-trained interviewers. Venous blood samples were taken from each subject. Sowing or harvesting in summer (OR=2.571, 95% CI: 1.109-5.962), living in house with weeding in the yard (OR=2.247, 95% CI: 1.062-4.755), and residence at the plain area (OR=2.630, 95% CI: 1.050-6.588) were the independent relative factors for seropositive LB. Wearing long pants and clothes with cuffs was the only protective behavior against tick bite (OR=0.186, 95% CI: 0.041-0.846). The findings showed that local farmers were easily infected with LB and almost no protective measure was taken against LB infection. Infection with LB was easier in residents of plain regions. Pets raising and outdoor activities were not the risk factors for infection with LB. Further studies are needed to fully understand the risk of infection with LB in China.

Analysis of effect of allogeneic hematopoietic stem cell transplantation for children with myelodysplastic syndrome / 中华实用儿科临床杂志

Objective To analyze the clinic efficacy of allogeneic hematopoietic stem cell transplantation(alloHSCT) for children with myelodysplastic syndromes(MDS) and to investigate the possible prognostic factors.Methods Eighteen children with MDS who underwent allo-HSCT at Shanghai Children's Medical Center Affiliated to Medical School of Shanghai Jiaotong University between Nov.2007 and Aug.2011 were retrospectively reviewed.Seven cases received transplantation before Dec.2009 and 11 cases later.Results Up to the follow-up end point,6 cases died,and among them 3 cases died of relapse and 3 cases died of related treatment after transplantation.The 2-year disease free survival(DFS) rate of all patients was(65.0 ± 11.7)％.The 2-year DFS rate was significantly different between the group receiving transplantation before Dec.2009 and the group receiving transplantation later[(28.6 ± 17.1)％ vs (90.9 ± 8.7) ％,P =0.01].Patients treated by regimen of busulfan/cyclophosphamide (BUCy) had a significant higher 2-year DFS rate compared with other treatment regimens [(83.3 ± 10.8) ％ vs (25 ± 20.4) ％,P =0.035].The selected pretreatment regimen showed a significant difference between 2 groups in the different transplantation periods,and the pretreatment regimen of BUCy was more frequently used in both groups after Dec.2009 (P =0.004).Conclusions Allo-HSCT is an effective treatment strategy for children with MDS and the pretreatment regimen of BUCy is suitable for those patients.The experience accumulation about transplantation and the improvement of supportive medical care have substantially improved HSCT outcome and reduced the treatment-related mortality.

Efficacy and safety of Changfu peritoneal dialysis solution: a multi-center prospective randomized controlled trial / 中华医学杂志(英文版)

<p><b>BACKGROUND</b>A multi-center large scale study is needed to confirm the efficacy and safety of domestic peritoneal dialysis (PD) solutions. Some researchers believe that 6 L/d is enough for adequate dialysis, but there is no multi-center prospective study on Chinese population to confirm this. In this study, we evaluated the efficacy and safety of domestic PD solution (Changfu) and its difference between 6 L and 8 L dosage.</p><p><b>METHODS</b>Adult PD patients who had taken PD therapy for at least one month were selected and divided into four groups according to two dialysis solution brands and two dialysis dosages, i.e., 6 L dose with Changfu dialysis solution, 6 L dose with Baxter dialysis solution, 8 L dose with Changfu dialysis solution, and 8 L dose with Baxter dialysis solution. After 48 weeks, the changes of primary and secondary efficacy indices were compared between different types and different dosages. We also analyzed the changes of safety indices.</p><p><b>RESULTS</b>Changes of Kt/V from baseline to 48 weeks between Changfu and Baxter showed no statistical differences; so did those of creatinine clearance rate (Ccr). Normalized protein catabolic rate (nPCR) from baseline to 48 weeks between Changfu and Baxter showed no statistical differences; so did those of net ultrafiltration volume (nUF) and estimated glomerular filtration rate (eGFR). Changes of nPCR from baseline to 48 weeks between 6 L and 8 L showed no statistical differences; so did those of nUF and eGFR. The decline of Kt/V from baseline to 48 weeks in 6 L group was more than that in 8 L group. Change of Ccr was similar. During the 48-week period, the mean Kt/V was above 1.7/w, and mean Ccr was above 50 L×1.73 m(-2)×w(-1). More adverse events were found in Changfu group before Changfu Corporation commenced technology optimization, and the statistical differences disappeared after that.</p><p><b>CONCLUSIONS</b>The domestic PD solution (Changfu) was proven to be as effective as Baxter dialysis solution. During 48-week period, a dosage of 6 L/d was enough for these patients to reach adequate PD. Clinical study promotes technological optimization, further helps to improve the safety indices of the medical products.</p>

Long-term outcomes of childhood lymphoblastic lymphoma: report of 70 cases / 中华血液学杂志

<p><b>OBJECTIVE</b>To summarize long-term outcomes of childhood lymphoblastic lymphoma (LBL) with protocol CCCG-97 and -2002.</p><p><b>METHODS</b>From November 1998 to October 2010, 70 consecutive newly diagnosed childhood LBL (5 B-LBL and 65 T-LBL) were enrolled in this study, in which 22 received CCCG-97 and 48 CCCG-2002 protocols. St.Jude staging system was adopted. Patients were divided into three risk groups based on clinical stage and serum LDH, and received chemotherapy with different intensity. The factors, which were possibly associated with the prognosis, were analyzed. The survival rates were evaluated by Kaplan-Meier analysis.</p><p><b>RESULTS</b>The patients were 1.5 to 14 years old with the median age of 8 years old. They were evaluated as stage I-II for 6 , stage III41, and stage IV23 (15 were BM positive and 8 multiple bone metastases). Until Dec.31th, 2011,the mean follow-up was 62.5 months (range, 14 to 161 months) with the median follow-up of 48 months. 1-year overall survival (OS) was 74.3%, and 5- year event-free survival (EFS) 64.1% (abundance as event). Thirteen patients were complicated with serious condition during chemotherapy and 1 died of complication. Univariate analysis indicated that delayed and/or non-completed response on days 33 and 63 of induction was the unfavorable prognostic factor.</p><p><b>CONCLUSION</b>Primary LBL usually located in the mediastinum. 90% of the patients was at advanced stage III-IV at first presentation. The 5-year EFS was 64.1%. Patients not achieved CR at days 33 and 63 at the end of induction was a poor prognostic factor.</p>

The impact of cardiac resynchronization therapy on left ventricular diastolic function evaluated by speckle tracking imaging in patients with dilated cardiomyopathy / 中华心血管病杂志

<p><b>OBJECTIVE</b>To investigate the effects of cardiac resynchronization therapy (CRT) on left ventricular (LV) diastolic function measured by speckle tracking imaging (STI) in patients with dilated cardiomyopathy (DCM).</p><p><b>METHODS</b>CRT was performed in 21 DCM patients [15 male, mean age: 61.2 ± 11.2 (49-82) years].LV synchronization, LV systolic function and LV diastolic function were evaluated with conventional echocardiography, tissue Doppler imaging and STI before and 6 months after CRT.NYHA heart function was also assessed. Clinic Response to CRT was defined as improvement of more than 1 NYHA class.Response to CRT in echocardiography was defined as ≥ 15% reduction in LV end systolic volume at 6 months post CRT.</p><p><b>RESULTS</b>There were 16 responders and 5 non-responders at 6 months post CRT.In terms of diastolic function, conventional echocardiography derived deceleration time was both prolonged in non-responders and responders. At 6 months post CRT, STI derived LV isovolumetric diastolic strain rate [(0.19 ± 0.11) /s vs.(0.14 ± 0.09)/s, P < 0.001] was significantly increased while early diastolic mitral valve blood flow velocity/left ventricular isovolumetric diastolic strain rate (680 ± 600 vs.787 ± 690, P < 0.04) was significantly reduced in responder group while remained unchanged in non-responder group.Furthermore, left ventricular isovolumetric diastolic strain rate negatively correlated with plasma brain natriuretic peptide level (r = -0.68, P < 0.05).</p><p><b>CONCLUSION</b>In CRT responders of DCM patients, LV diastolic function is significantly improved and this change could be detected more effectively by STI derived LV diastolic function parameters.</p>

Long-term follow-up of childhood low-risk ALL patients treated with SCMC-ALL-2005 protocol / 中华血液学杂志

<p><b>OBJECTIVE</b>To evaluate the long-term efficacy of SCMC-ALL-2005 protocol in treatment of low-risk childhood acute lymphoblastic leukemia (ALL).</p><p><b>METHODS</b>From May 1, 2005 to April 30, 2009, 387 patients enrolled into SCMC-ALL-2005 protocol. Based on the characteristics of cell morphology, immunology, cytogenetics and molecular biology and treatment response, 158 patients were fit into the low-risk treatment group. All the cases were registered in pediatric oncology network database (POND). The clinical characteristics and outcome were analyzed.</p><p><b>RESULTS</b>Until December 31, 2012, the 5-year event free survival (EFS) and overall survival (OS) is (77.76±3.37)% and (89.55±2.83)%, respectively. Median follow-up time is 5.33 y (3.75-7.70 y). Five patients (3.16%) died of complication, all of them were severe infections. Twenty-seven patients (17.09%) relapsed, including 13 bone marrow relapse (8.23%), 5 testis relapse (5.32% of boys, 2 of unilateral and 3 bilateral), 6 central nerve system relapse (CNS, 3.80%), 1 relapse in both bone marrow and CNS, 1 relapse in both bone marrow and testis, and 1 right ovary and fallopian tube relapse. Relapse is related to positive minimal residual disease. Two cases (1.27%) occurred second tumors, 4 patients (2.53%) gave up treatment in complete remission without special reasons.</p><p><b>CONCLUSION</b>The EFS and life quality of SCMC-ALL-2005 protocol in the treatment of childhood low-risk ALL is satisfactory. The treatment-related mortality rate is lower, and the long-term EFS is higher than that of XH-99 protocol.</p>

Treatment of chronic primary glomerulopathy patients of Shen deficiency and dampness heat syndrome by yishen qingli granule combined low-dose Tripterygium wilfordii multiglycoside tablet: a clinical efficacy observation / 中国中西医结合杂志

<p><b>OBJECTIVE</b>To evaluate the clinical efficacy and safety of treatment of chronic primary glomerulopathy (CPG) patients of Shen deficiency and dampness heat syndrome (SDDHS) by Yishen Qingli Granule (YQG) combined with low-dose Tripterygium Wilfordii multiglycoside Tablet (TWT).</p><p><b>METHODS</b>Totally 231 CPG patients of SDDHS were enrolled in this study (including 60 patients from First Affiliated Hospital of Nanjing University of Chinese Medicine, 58 from First Affiliated Hospital of Nanjing Medical University, 46 from Xinqiao Hospital of Third Military Medical University, 35 from First Affiliated Hospital of Guangzhou University of Chinese Medicine, 14 from First Affiliated Hospital of Soochow University, and 18 from Wuxi Affiliated Hospital of Nanjing University of Chinese Medicine). They were randomly assigned to the control group (116 cases) and the trial group (115 cases) according to block group method. There were 217 cases in the safety analysis set (109 cases in the trial group vs 108 cases in the control group), and 203 cases in the full analysis set (99 cases in the trial group vs 104 cases in the control group). All patients received basic treatment such as ACEI/ARB. Furthermore, YQG (consisting of raw astragalus 10 g, prepared Polygonum Multiflorum 10 g, Pyrrosia 10 g, 1.5 g each package, containing 10 g of crude drugs) was additionally given to patients in the trial group, each package, twice daily. The TWT (10 mg) was given, twice a day. The TWT dose was adjusted according to 24 h urinary total protein (UTP). The placebos of YQG and TWT were administered to those in the control group. The treatment course consisted of 24 weeks and the follow-up visit lasted for 24 weeks. The biochemical indices were observed before and after treatment including 24 h UTP, urine red cell count (U(RBC)), renal functions (BUN, SCr), blood routine test (WBC), and liver functions (SGPT, SGOT). Reverse reactions such as gastrointestinal discomfort, skin rash, and irregular menstruation were also observed.</p><p><b>RESULTS</b>Compared with the control group, the total effective rate was better in the trial group (82.83% vs 61.54%, P < 0.01). Results of stratified comparison of UTP showed better efficacy in the trial group (0.8-3.0 g/24 h, P < 0.01). The UTP decline occurred in the trial group after 8 weeks of treatment, with stable action, showing statistical difference when compared with the control group (P < 0.01). In the trial group, U(RBC) level decreased after treatment but changed more significantly. But there was no statistical difference in the changes when compared with the control group (P > 0.05). After treatment, there were no statistical difference in safety indicators such as WBC, SGPT, and SGOT between the two groups after treatment (P > 0.05).</p><p><b>CONCLUSION</b>On the basis of basic treatment such as ACEI/ARB, application of YQG combined with low-dose TWT had better effect in controlling proteinuria of CPG patients, and could help stabilizing their conditions with less adverse reactions.</p>

Therapeutic efficacy of allogeneic hematopoietic stem cell transplantation in children with chronic myelogenous leukemia / 中国当代儿科杂志

<p><b>OBJECTIVE</b>To investigate the therapeutic efficacy of allogeneic hematopoietic stem cell transplantation (allo-HSCT) in children with chronic myelogenous leukemia (CML), and to analyze the possible prognostic factors.</p><p><b>METHODS</b>The clinical data of 20 children with CML who had received allo-HSCT was analyzed retrospectively to investigate possible prognostic factors, including age, sex, interval between diagnosis and transplantation, HLA matching between donors and recipients, illness status on transplantation and acute and chronic graft-versus-host disease (GVHD).</p><p><b>RESULTS</b>At the end of follow-up, 13 of the 20 treated children had disease-free survival (DFS) and the rest (7 cases) died. Four died of severe acute GVHD, two of chronic GVHD and its complications, and one of relapse after transplantation. The three-year DFS was (64.6±1.1%). As shown by the univariate analysis, age was the most important prognostic factor in children with CML who had received allo-HSCT (P<0.05), and in children over 10 years, the prognosis was poor. No other of the above factors had a significant impact on prognosis (P>0.05). The multivariate logistic regression analysis also confirmed age as the only prognostic factor (P<0.01). Severe acute and/or chronic GVHD was the most important cause of patient death. 10/10 HLA-matched donors could improve the transplantation outcome.</p><p><b>CONCLUSIONS</b>Allo-HSCT is an effective treatment for children with CML. To improve the prognosis and treatment outcome, children with CML aged over 10 years should receive allo-HSCT as early as possible. 10/10 HLA-matched donors are preferred in allo-HSCT and GVHD should be prevented.</p>