Objective To develop a new statistical method for comparing effectiveness of two treatments for the same disease indication. A statistical method needs to be developed to compare effectiveness of different treatments by using existing clinical data when lacking of evidence from randomized controlled trials. Methods Datasets including an outcome variable and a confounder for two treatments were simulated. Subjects in each dataset were randomly divided into two arms for 100 times, and the means were compared by using student-t tests and Per-protocol analysis strategy was applied. Subjects with assigned treatment by randomization that was contradictory to the actual received treatment were considered drop-out of conventional RCTs, and were excluded from comparisons of means. The ratio of the frequency of hypothesis tests that rejected H0 to the frequency of hypothesis tests that did not reject H0, called odds, was used to predict the probability of significant tests for group difference. To document the consistency and reliability of the method, the distribution of odds and its 95% CI, and the confounding effect were described in simulated datasets with various between-group mean differences and statistical power (ranging from 0.5 to 0.85) with varied sample sizes (n=50, 100, 500, and 1 000). Stata 11.0 was used to program and perform the analysis. Results The odds and its 95%CI of simulated RCTs were perfectly and linearly correlated with the change of between-group mean differences and statistical power, by difference sample size. A conclusion could be made based on the hypothesis of the simulated randomized controlled trials (sRCT). The probability of loss of balance of confounding was over 95% for equal and unequal sample size of two arms after excluding misclassified subjects. Conclusions The proposed novel analytical method, simulated RCTs based on real clinical treatment data, can be used to compare effectiveness of two treatments when evidence from RCTs is unavailable.
Objective To study the clinical efficacy and safety of transplacental digoxin therapy to fetal heart failure, and to investigate the significance of cardiovascular profile score (CVPS) and myocardial performance index (Tei index) in assessment and evaluating treatment efficacy of fetal heart failure. Methods According to the research protocol, with written informed consent, fetal heart failure cases, diagnosed in West China Second University Hospital of Sichuan University from May 2008 to December 2011, were consecutively recruited. Based on the parents′ decisions, the included subjects received transplacental digoxin therapy or short-term clinical observation only. During the clinical course, fetal CVPS and ventricular Tei index were dynamically monitored, pregnant outcomes were observed and recorded. A number of healthy pregnant women and fetuses were enrolled to provide reference levels of cardiac function, who were registered in West China Second University Hospital for their routine antenatal care, the fetal CVPS and right ventricular Tei index were dynamically measured and recorded at the time points of 20, 24, 28, 32, 36 gestational weeks and until delivery. Results ① Twelve cases with fetal heart failure including 6 cases of fetal atrial flutter (AF), 3 cases of fetal supraventricular tachycardia (SVT), 2 cases of fetal anemia (MA) and 1 case of fetal dilated cardiomyopathy (DCM), received digoxin treatment. Seven subjects including 4 AF, 3 SVT and 2 MA were not treated with digoxin and were treated as controls. After a consultation the individual protocol of transplacental digoxin therapy was determined. Fetal heart failure was gradually controlled in AF, SVT and MA fetuses with transplacental digoxin therapy, and achieved uneventful post-therapy pregnancy and full-term delivery. CVPS increased gradually, at or near the 10 points, left and right ventricular Tei index decreased gradually, closed to normal range. After 10 to 55 months follow-ups, evaluation from Bayley Scale of Infant Development（BSID）revealed the normal growth and development of physique and mentality in all the enrolled children. ② The 7 subjects in control group manifested as gradually decreased CVPS and increased Tei index, and had pregnancy termination finally after short-term clinical observation. Thirty healthy pregnant women and their healthy fetuses were enrolled to provide 10 time points active comparable reference values for CVPS and Tei index measurements. With the gestational age progression, fetal right ventricular Tei index decreased gradually, the value was 0.48±0.05 at 20 gestational weeks and decreased to 0.38±0.04 before delivery. Only gastrointestinal reaction was reported in one case from digoxin group and disappeared after reduction of the does. Conclusions Digoxin stood first on the list of medicaments has important clinical value in clinical treatment for fetal heart failure. With the alleviating of fetal heart failure, CVPS increases gradually, and ventricular Tei index decreases, with the negative correlation between them. CVPS and Tei index can effectively guide the prenatal transplacental digoxin therapy for fetal heart failure. Timely and effective prenatal intervention can significantly improve the prognosis of the suffered fetuses.
Objective To evaluate the value of bronchoscopy in children with different types of respiratory failure. Methods The clinical data of cases with respiratory failure from March 2011 to June 2012 were collected retrospectively.Changes of arterial blood gas, lung imaging and fiberoptic bronchoscopic findings were analysed. Results Finally, the results of the 60 patients were analyzed. Based on the blood gas level children were divided into group A (ARDS Group, 11 cases)and group B (non-ARDS Group ,49 cases). Group B was divided into three subgroups: typeⅠrespiratory failure(11 cases), type Ⅱ respiratory failure(17 cases) and hypercapnia alone (21cases). ① Under bronchoscope rough mucosa and edema could be found in all 60 patients. In 8/11 cases (72.7%) more secretion could be found in ARDS group and in 19/49 cases (38.8%) in non-ARDS group. Tracheostenosis could be seen in 16 cases in non-ARDS group. Hypoventilation could be seen in 2/11 cases (182%) in ARDS group and in 7/49 cases (14.3%) in non-ARDS group. Mucosal erosion could be found in 5/11 cases (454%) in ARDS group and in 2/49 cases (4.1%) in non-ARDS gropup. ② Etilogical diagnosis was made with bronchoscopy in 26/60 (43.3%), 3/11 (27.3%) and 23/49 (46.9%) cases in ARDS and non-ARDS groups respectively. ③ Chest X-ray (CT) pre- and post-bronchoscopic lavage showed in ARDS group consolidation shadow, grind glass shadow and brochial aeration were the main findings, while in non-ARDS group consolidation shadow, fronchial aeration and insufficient aeration of lung were the main presentation. The efficacy rates of imaging were 87.5% (33/40) and 0% in non-ARDS and ARDS groups respectively. ④ Blood gas analysis showed that improvement was found in 3/11 (27.3%) and 38/47 (80.8%) cases pre- and post-bronchoscopy in ARDS and non-ARDS groups respectively. ⑤ Total efficacy rates were 27.3% (3/11) and 93.9% (46/49) in ARDS and non-ARDS groups, and there was significant difference between two groups (P=0.000). Total efficacy rates were 90.9% (10/11), 94.1% (16/17) and 95.2% (20/21) in type Ⅰ, type Ⅱ respiratory failure and hypercapnia groups respectively, and there all were significant differences compared to that of ARDS group (P all <0.05). Conclusions ①For children with ARDS, bronchoscopy should not be used.②Bronchial lavage treatment for non-mechanical obstruction respiratory failure in non-ARDS children was effective. ③When the etiology of respiratory failure in children is unknown, bronchoscopy may be helpful for etiological diagnosis.
Objective To analyze the clinical features and gene mutation of 4 Chinese children with Alport syndrome． Methods The clinical and laboratory data of 4 children with Alport syndrome were summarized. COL4A5, COL4A4 and COL4A3 genes were analyzed using next-generation sequencing. Results Four patients all presented an atypical clinical picture with onset of hematuria accompanied with different degrees of proteinuria . Ophthalmology, otolaryngology and renal function studies showed no abnormalities, except for a sensorineural hearing loss detected in case 2. Characteristic ultrastructual changes of the glomerular basement membrane were found in all patients. Four pathogenic mutations were identified in COL4A5 gene in the 4 families, including Gly132Glu, Gly1238Arg, Gly267Arg and Gly1033Ser. The 4 mutations were not reported previously, two of which were the novel mutations. Conclusions The 4 novel mutations in coding DNA of COL4A5 gene identified in our study contribute to further study of pathogenesis of Alport syndrome , genetic counselling and prenatal diagnosis in Chinese population.
Objective To conduct a systematic review to identify the diagnostic value of C-reactive protein for detecting serious bacterial infections in children with fever without apparent causes. Methods Medline, EMBASE, Cochrane Library, VIP Information, China National Knowledge Infrastructure, Wanfang Chinese Periodical Database and Chinese Biomedical Database were searched. QUADAS was used for the quality assessment of included studies, and software MetaDisc 1.4 was used for the test of heterogeneity and meta-analysis. Results Seven studies (n=2 179) were included for the review. Six of them were prospective studies, while one was a retrospective study. None of the studies was assessed with high methodological quality. The prevalence of serious bacterial infections ranged from 1.7% to 29.3%. Multivariate logistic analysis conducted in four studies showed that C-reactive protein was an independent predictor. The sensitivities and specificities of the C-reactive protein with different optimal cut-offs were ranged from 33% to 89%, and 75% to 97% respectively. Random effects meta-analysis yielded a pooled positive likelihood ratio of 4.43 (95%CI:3.49-5.63), and a negative likelihood ratio of 0.39 (95%CI:0.29-0.54). The area under the summary receiver operating characteristic curve was 0.85, and Q index was 0.78. Conclusions C-reactive protein provides a good diagnostic value in infants and young children with fever without apparent causes, it should be applied in combination with clinical features and other tests.
Objective To investigate the overall contribution of perinatal environmental effects on poor response to HBV vaccination in Chinese infants. Methods Case-control study was used, one-year-old healthy twins and unrelated singletons were recruited from child care settings of 5 hospitals. Parental characteristics, neonates′ condition at birth, postnatal infant feeding history, and growth during the 1st year of life were investigated by interview and medical record review. HBV related markers were examined for infants at one year of age. The role of environmental factors on poor vaccination response (anti-HBs<100 mIU·mL-1) was analyzed using XTGEE programs for twins and logistic and tobit regression programs in singletons. Results Overall, 71（19.2%）of 370 twins and 35 (11.7%) of 300 singletons had poor responses to HBV vaccine. In twins, paternal smoking and low birth weight were associated with an increased risk of poor response to HBV vaccination (OR=4.50, 95%CI: 2.52-8.03 and OR= 2.55, 95%CI: 1.33-4.87, respectively). Higher Apgar score and gaining more body weight in the 1st year of life reduced this risk. No environmental factors were related to poor response to vaccination in the singleton samples. Conclusions After three HBV vaccinations, low birth weight, father smoking, Apgar scores and weight gain in 1st year were associated with poor antibody response to infant hepatitis B vaccination.
Objective To identify risk factors for preterm birth less than 34 weeks gestation based on placental histopathology. Methods Clinical characteristics and placental histological results were collected from 216 singleton pregnancies. According to gestational ages, the subjects were divided into 2 groups, group 1 with 27+0～31+6 weeks gestation group (n=106) and group 2 with 32+0～33+6 weeks gestation group (n=110). Results There was no difference in maternal age, cesarean section and antenatal steroid between two groups. The placental histopathological results showed that intrauterine inflammation response was present in 104 of 216 cases (48.1%), and of 104 cases, in 53 cases (51.0%) the inflammation involved maternal side only （MIR+FIR-）and in 51 cases (49.0%) the inflammation involved both maternal and fetal sides（MIR+FIR+）. There was no case with fetal inflammation response only（MIR-FIR+）. The risk factors included premature preterm rupture of membranes (n=105), labor (n=115), hypertensive disorders in pregnancy(n=52), fetal distress (n=51), antepartum hemorrhage(n=47), placentaprevia(n=21), history of abortion(n=108) , irregular antenatal attendance(n=46), medical disorders(n=23) and intrauterine growth retardation(n=10) and so on. In the 27+0-31+6 weeks gestation group, MIR+FIR- or MIR+FIR+ was more frequently associated with premature preterm rupture of membranes and labor compared with MIR-FIR-（64.3%, 83.9% vs 21.3%，P=0.000；78.6%, 67.7% vs 46.8%，P=0.016）, and less frequently associated with hypertensive disorders in pregnancy compared with MIR-FIR- (0, 6.5% vs 40.4%，P=0.000); MIR+FIR+ was less frequently associated with antepartum hemorrhage compared with MIR-FIR- or MIR+FIR-（6.5% vs 29.8%,32.1%，P=0.027）. In the 32+0-33+6 weeks gestation group, MIR+FIR- or MIR+FIR+ was more frequently associated with premature preterm rupture of membranes compared with MIR-FIR-（52.0%, 90.0% vs 30.8%，P=0.000）. Under the condition of MIR-, the risk factors were similar between 27+0-31+6 weeks and 32+0-33+6 weeks gestation groups except for labor （46.8% vs 20.0%，P=0.003）, and under the condition of MIR+, the risk factors were similar between two groups. Conclusions Under the condition of intrauterine inflammation response positive, the preterm delivery is mostly spontaneous. However, under the condition of intrauterine inflammation response negative, the preterm delivery mostly indicates to be with various risk factors. The intrauterine inflammation response does not increase the risk of fetal distress and placental abruption.
Objective To analyze the clinical characteristics and body height of 153 pediatric patients with disorders of sex development (DSD) without known causes. Methods Clinical data of DSD patients, including age, bone age, gender and family history were collected. Categories were definited and body height, body weight, penis length and testis volume were measured and deformation was described. The levels of sex hormones were determined. HCG test, B-ultrasonography and chromosomes examination were performed. The growth profile of DSD patients and the relevant factors were analyzed. Results A total of 153 DSD patients were enrolled including 128 social sex males and 25 females aged from 42 days to16 years and 10 months (4.6±4.2 years). There were 121（90.3%） with 46,XY DSD, 3(2.2%) 46,XX DSD and 10(7.5%) chromosome DSD. Among 46,XY DSD, 39 cases(32.2%) were diagnosed as hypospadias combined with micropenis, 19(15.7%) were micropenis with testis abnormality and 18 cases(14.9%) were simple micropenis. 15（9.8%）cases had DSD family histories and 19（12.4%）patients′ mother had taken progesterone in early pregnant stage to avoid threatened abortion. The proportion of body height shorter than 25 percentile and 50 percentile was higher than normal population, P=0.039 and 0.056 respectively. 125/153 DSD were performed HCG test, height＞P50 was 60.3% among 78 who had normal testosterone response, while it was only 18.2% height＞P50 among 33 cases with abnormal testosterone response (P=0.000). Conclusions The major patients of unknown cause DSD were 46,XY DSD, the most of them were hypospadias combined with micropenis, and followed by micropenis. Some patients had DSD family histories and some patients whose mothers had threatened abortion and took progesterone during pregnancy. Patients with DSD may have both disorders of sex development and short statures. Most patients with DSD were shorter than normal population and testicular development was related to short statures.
Objective In China, Beckwith-Wiedemann syndrome (BWS), a rare pediatric congenital overgrowth disorder, associated with abnormal regulation of gene transcription in an imprinted domain located at chromosome 11p15.5, is still poorly recognized. The purpose of this study was to investigate the clinical features, genetic abnormality, and clinical management of BWS. Methods Clinical manifestations, laboratory examinations and genetic testing of one case of BWS were presented, analyzed and discussed. The related literatures were reviewed. Results The case was female, aged 5 months, presenting typical BWS clinical features, including macroglossia, visceromegaly, ear pits, transient neonatal hypoglycemia, facial nevus flammeus, infraorbital creases, etc. A hypomethylation at IC2 of CDKNIC domain was identified and confirmed by MS-MLPA. Conclusions This case is the first report of BWS in China with epigenetic confirmation, and has typical maternal dysmethylation at ICs. The diagnosis of BWS relies on a combination of clinical features and gene-based tests. The BWS patients need to be long term followed-up.
Objective Wolman disease (WD) is a rare autosomal recessive lysosomal storage disease, caused by deficiency of lysosomal acid lipase (LAL). In China, WD, the same type disorder as cholesteryl ester storage disease (CESD), but with severe clinical manifestations, is still poorly recognized. The purpose of this study was to investigate the clinical features, genetic abnormality of WD. Methods Clinical manifestations, laboratory examinations, and genetic testing of one case of WD were presented, analyzed, and discussed. The related literatures were reviewed. Results A approximately 5monthold female infant presented typical WD clinical features, including massive hepatosplenomegaly, remarkable jaundice, liver failure, severe coagulopathy, depressed bone marrow function, bilateral calcified adrenal glands, and in bone marrow aspirate, multiple foamy, vacuolated cytoplasm, seablue histiocytes with negative periodic acid schiff staining were observed. Sequencing of the LIPA gene revealed that she was a homozygote with the nonsense mutation (c.796G>T) in exon 7, resulting in the change of amino acid 266 from Gly to termination codon (GGATGA). This mutation finally result in a synthesis of the truncated protein (p.G266*). Conclusions The case presented here is the second WD case reported in mainland China. Based on the size of our population, WD and CESD are both underestimated. With the progress in DNA sequencing technique, and recognition the nature of WD and CESD, hopefully more cases will be diagnosed and recognized.
Objective To investigate epidemiological and clinical features of childhood solid tumor in our hospital from Jan. 2009 to Dec. 2011. Methods Clinical and pathological data were retrospectively reviewed and analyzed. Tumor classification was performed according to WHO tumor classification and diagnostic criteria. Results In total, 983 cases of childhood solid tumor were enrolled into the study, including 721 （73.35%）benign tumors and 262 malignant tumors. There were 548 boys(55.7%) and 435 girls, aged from 8 days to 14 years (median age was 4 years old). ① Sort by the pathological type: The top five benign tumors were vascular tumor, calcifying epithelioma, chondroma, fibroma and mature teratoma, while the top three malignant tumors were neuroblastoma, lymphoma and Wilm′s tumor. ② Sort by the original site:The top three benign tumors were skin and soft tissue tumors,craniofacial and neck tumors and bone tumors, while the top three malignant tumors were abdominal tumors,intracranial tumors and craniofacial and neck tumors. ③ Most common malignant tumors were embryonal tumors (171 cases, 65.3%), followed by tumors containing similar ingredients (57 cases, 21.8%) and lymphoma (34 cases, 13.0%). ④ Solid tumors were much more prevalent in infants and toddlers, showing a characteristic single peak pattern. ⑤ 29/262 cases（11.1%） were massive malignant tumor and 26 of them（89.7%）were from abdomen. Conclusions Benign tumor remains the main type of solid tumor in children. Childhood malignant tumors commonly occur in the abdominal cavity with embryonic tumors as the predominant histological types. Neuroblastoma and Wilm′s tumor are the primary tumors with huge tumor mass in children under 4 years old.
Abstract Objective To explore the clinical curative effects in the treatment of children glottis and subglottic disease.Methods etrospective analysis of 4 cases admitted in our hospital since 2011 after the using of argon plasma coagulation via tracheal endoscopic .Results 3 cases of subglottic disease were not found granulation tissue proferation again and trachebronchial stenosis after sugery.They were resore normal breathing after unplugging tracheotomy cannula and endotracheal intubation.1 case of larynx papilloma was not found tumor recurrence after surgery 5 months later .Then we unplugged the tracheotomy cannula.Conclusion The imaging of electronic tracheal endoscopic is clear.It can freely prospective change the angle and positioning accuracy.The nozzle dimeter of argon plasma coagulation is small.It can enter the children’s glottis smoothly.It can make operation more precise and reduce operative time.It shoule be widely applied in the he treatment of children glottis and subglottic disease.
