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@#In this study, different functional layer formulations and process parameters were used to prepare the levomilnacipran hydrochloride sustained-release capsules, the influence of functional layer formulation and process factors on dose dumping was studied by comparing their release curves in 40% ethanol; and the risk of dose dumping of the self-developed drug was evaluated by the similar factors of the release curve of the self-developed drug and the reference drug.The results showed that as the coating weight increased, the degree of dose dumping decreased; when the concentration of ethanol in the coating liquid solvent was less than 80%, the dose dumping increased; as the atomization pressure and maturation time increase, the dose dumping became more serious. In 0% ethanol (purified water), 5% ethanol, 20% ethanol and 40% ethanol media, the self-developed and reference preparations had the same degree of dose dumping within the specified time, and rotation speed had no significant effect on the release of metformin in vitro. In summary, formulation factors such as coating weight gain, ethanol concentration in the coating solution solvent, and process factors such as atomization pressure and curing time have a serious impact on the dose dumping of sustained-release capsules.Under the optimal functional layer formulation and process, special attention should be paid to the control of risk of self-developed dose dumping.
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Objective:To investigate the short-term therapeutic effect and long-term survival of multiple myeloma patients with extramedullary disease (EMD) in the new drug era.Methods:The data of 74 patients with multiple myeloma diagnosed and treated in Anhui Wanbei Coal and Electricity Group General Hospital from January 2015 to January 2020 were retrospectively analyzed, including 17 patients with soft tissue infiltration (EM-S), 9 patients with bone infiltration (EM-B), and 48 patients without EMD (No-EMD). The short-term efficacy, the 4-year progression-free survival (PFS) rate and overall survival (OS) rate, and their influencing factors in three groups of patients after receiving bortezomib regimen were analyzed.Results:After 3-4 courses of early induction therapy of bortezomib regimen, the overall response rate of patients in the EM-S group was lower than that in the No-EMD group and the EM-B group [58.8% (10/17) vs. 85.4% (41/48), 100.0% (9/9)], and the differences were statistically significant ( χ2 = 13.7, P = 0.036; χ2 = 26.5, P = 0.003), while the difference between No-EMD group and EM-B group was not statistically significant ( χ2 = 12.7, P = 0.211). Survival analysis showed that the 4-year PFS rate of No-EMD group was higher than that of the EM-S group and EM-B group (41.0% vs. 7.6%, 0), and the differences were statistically significant ( χ2 = 10.835, P < 0.01; χ2 = 8.276, P = 0.004). Meanwhile, the 4-year OS rate of EM-S group was lower than that of the No-EMD group and EM-B group (16.5% vs. 54.3%, 59.3%), and the differences were statistically significant ( χ2 = 9.146, P = 0.002; χ2 = 4.066, P = 0.044). Conclusion:The early treatment effect of bortezomib regimen, PFS and OS in multiple myeloma patients with EM-S are poor, while the EM-B has no effect on OS.
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Objective@#To investigate the clinical characteristics, diagnostic approach, and treatment of infantile lactose intolerance, and to evaluate the diagnostic value of fecal pH.@*Methods@#The feces and clinical data of all infants(less than 1 year old) diagnosed with simple diarrhea but without signs of infection were collected, who were treated at 4 Grade Ⅲ-Class-A hospitals within the period of June 2012 to June 2015 in Beijing were collected.Lactose intolerance was diagnosed based on urine galactose level, lactose tolerance test, and fecal pH.The clinical characteristics, therapeutic effects and detection methods of lactose tolerance group and intolerance group were analyzed.Then the diagnostic value of fecal pH was evaluated.@*Results@#A total of 217 infants were enrolled in the study, consisting of 113 boys and 104 girls.Their age ranged from 3 to 330 days, 174 infants (80.2%) were less than 6 months old.Among them, 156 infants were diagnosed with lactose intolerance (71.9%), their median age 90.0 days (3-330 days), while the median age of the lactose tolerant group was 51.5 days (3-300 days). The incidence of lactose intolerance in infants less than 6 months old was 70.6%, and 76.7% in those older than 6 months.Clinical characteristics showed that more infants in the lactose intolerant group were breastfed compared with the lactose tolerant group [73/156 cases(46.8%) vs.16/61 cases(26.2%), χ2=7.666, P<0.05], the occurrence of loose foamy feces was higher in the lactose intolerant group [67/156 cases(42.9%) vs.15/61 cases (24.6%), χ2=6.287, P<0.05], the median duration of diarrhea in the lactose intolerant group was 30 days (1-210 days), and that in the lactose tolerant group was 30 days (1-300 days). The incidence of diarrhea more frequent than 10 times per day in the lactose intolerant group was 9.0% (14/156 cases), while that in the lactose tolerant group was 6.6% (4/61 cases). The rate of infection in the lactose intolerant group was similar to that in the lactose tolerant group [32/165 cases((20.5%) vs.17/61 cases(27.9%)]. The median time of recovery by feeding lactose-free formula milk was 2 days (1-60 days), recovery by feeding lactase lasted a median of 4 days (2-25 days), while recovery by using pro-biotics and dioctahedral smectite lasted a median of 2 days (1-5 days). The characteristics of fecal pH showed that mean pH value of fecal and the incidence of fecal pH<5.5 had no statistical significance between the lactose intolerant and tolerant group, between infants ≤6 months and infants>6 months, between the breastfeeding group and formula-feeding group, or between those with infection and those without infection (all P>0.05).@*Conclusions@#Infants with lactose intolerance often manifest foamy feces and tend to be breastfed.Lactose-free treatment efficacy was better than that with lactase.Clinical symptoms when combined with urine galactose level and lactose tolerance test can help diagnosis.However, the decrease of fecal pH proves to be unhelpful in aiding diagnosis.
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Objective@#To explore the clinical and genetic characteristics of infantile nephrotic syndrome caused by COQ2 variants.@*Methods@#The clinical and genetic data of a patient with nephrotic syndrome caused by COQ2 variants diagnosed at pediatric department of Peking University First Hospital from February 2018 to March 2018 were retrospectively analyzed. Related literature retrieved from PubMed, CNKI and Wanfang databases were searched to date (up to July 2018) with "COQ2 gene" or "primary coenzyme Q10 deficiency" and "nephrotic syndrome" or "nephropathy" as key words.@*Results@#A 14-month-old male, presented to local hospital at 11 months of age with edema and severe proteinuria, without hematuria, hypertension or renal dysfunction. He did not have infection or seizure in the course of the disease. He had no response to a more than four-week full-dose prednisone treatment. He had normal birth, mild motor development retardation and moderate language retardation. He was born to non-consanguineous healthy parents. He had two unaffected older sisters and one older sister died of "nephropathy" at one year of age. Genetic testing identified compound heterozygous variants in COQ2 gene: c.518G>A and c.973A>G, both could be predicted by in silico tools to be deleterious in protein function. These variants are not single nucleotide polymorphism and rare in normal populations. Both variants have previously been reported as pathogenic. These missense mutations were inherited from parents in autosomal recessive manner tested by Sanger sequencing. The patient was supplemented with high-dose of coenzyme Q10, at 30 mg/(kg·day) and glucocorticoid was withdrawn. Within three weeks of high dose coenzyme Q10 treatment, the edema disappeared. After seven weeks of high dose coenzyme Q10 treatment, the patient had decreased proteinuria and improved serum albumin levels. The urine protein to creatinine ratio decreased from 22.87 mg/mg to 1.98 mg/mg; Serum albumin increased from 14.2 g/L to 39.9 g/L, with normal kidney function and improved motor development. Primary CoQ10 deficiency is reported to be a rare autosomal recessive mitochondrial disorder with heterogeneous renal, neurologic, and muscular manifestations. To date, COQ2 variants have been reported in 14 children with glomerular involvement. Their age at onset ranged from neonatal period to 10-year-old (8 patients within the first year of life). Steroid resistant nephrotic syndrome (SRNS) is the most common phenotype. Some of these children also had progressing encephalopathy and myopathy, and seizures. Patients with COQ2 variants might show clinical improvement with early high-dose oral CoQ10 supplementation. Literature review revealed two Chinese articles, mainly about adults with neurologic symptoms. SRNS was previously not reported in Chinese pediatric patients.@*Conclusions@#It is necessary to carry out genetic testing for infant with SRNS. The coexistence of some degree of encephalomyopathy, such as development retardation, should raise suspicion of a mitochondrial defect caused by COQ2 variants. Timely diagnostic genetic testing and early high dose of coenzyme Q10 supplement could significantly improve their prognosis.
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Objective To study the ultrasonographic features and differential diagnosis of fetal penoscrotal transposition.Two dimensional and three dimensional ultrasound were applied in the diagnosis of fetal penoscrotal transpositionto improve the detection rate. Methods Twenty cases of suspected penile scrotal transposition of the fetus in Shengjing Hospital affiliated to China Medical University fromJanuary 2015 to February 2017were included in present study. The ultrasound findings, fetal chromosome examination and clinical follow-up outcome were retrospectively summarized. Results Among the 20 suspected cases of penile scrotal transposition, 17 cases were diagnosed correctly. All the 17 cases were partial type of penile scrotal transposition. In the remaining 3 cases, 2 caseswere hermaphroditism with the karyotype of 46-XX, and the other 1 case was confirmed as normal female fetusesby clinical follow-up after birth. The ″tulip″signwas the typical ultrasonographic features offetal penoscrotal transposition. Conclusion 2D combined with 3D ultraosound is useful in diagnosis and differential diagnosis of fetal penile scrotal transposition.
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Purpose To discuss the ultrasonic cardiogram of cardiac rhabdomyoma,and to analyze the change trend of single and multiple rhabdomyoma before and 6 months after childbirth.Materials and Methods Eleven fetuses diagnosed as cardiac rhabdomyoma prenatally from January 2014 to January 2016 were analyzed retrospectively,and the tumor size,number and anatomical location were recorded by ultrasound.The 11 fetuses were divided into single group and multiple group,and both groups were reviewed the changes to tumors during pregnancy and within postnatal 6 months.Results For 11 cases with rhabdomyoma,6 cases were in the multiple group,with tumors growing in 5 cases,and tumors unchanged in 1 case,and 5 cases were in the single group,with all tumors unchanged.The prenatal difference between the two groups was statistically significant (P＜0.05).After six months follow-up of 11 fetuses with cardiac rhabdomyoma,tumor regression occurred in 2 cases of the multiple group,tumors were unchanged in 4 cases,tumor regression occurred in 2 cases of the single group,tumors were unchanged in 3 cases,and there was no significant difference between the fetuses in the two groups within postnatal 6 months (P＞0.05).Conclusion The cardic rhabdomyoma has a tendency to increase in the prenatal period,and the increase may be significant in the multiple group.The rhabdomyoma is relatively stable within 6 months follow-up after birth,and there is a partial regression trend,without significant difference between single and multiple groups.
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Objective To study the clinical value of extensively whey protein hydrolyzed formula and partially whey protein hydrolyzed formula as an adjunctive therapy in treating infantile eczema.Methods Totally 59 bottle-feeding babies with infantile eczema were divided into three groups depending on different formula feeding:extensively hydrolyzed formula feeding group (eHF group,n =18),partially hydrolyzed formula feeding group (pHF group,n =22),and conventional cow's milk formula feeding group (CMF group,n =19).Meanwhile,all patients received the same drug treatment.The effective rate and remission rate were evaluated using the SCORing Atopic Dermatitis (SCORAD) index.Results After 2 weeks of treatment,the effective rate was 38.9％ and the remission rate was 50.0％ in the eHF group,which were significantly different from those in the CMF group (5.3％ and 31.6％) (x2 =12.225,P =0.002).The effective rate was 9.1％ and the remission rate was 40.9％ in the pHF group,showing no significant difference when compared with the CMF group (x2 =0.761,P =0.683).After 8 weeks of treatment,the effective rate was 55.6％ and the remission rate was 38.9％ in the eHF group,comparing to 15.8％ and 47.4％ in the CMF group,the difference was statistically significant (x2 =8.498,P =0.014).The effective rate was 31.8 ％ and the remission rate was 59.1％ in the pHF group,a trend towards higher than that of the CMF group,but the difference was not statistically significant (x2 =4.912,P =0.086).Before (F =0.773,P =0.466) and after (F =1.313,P =0.277) the treatment,the growth and development data in different groups showed no significance differences.Conclusion Both the extensively and partially whey protein hydrolyzed formula play an adjunctive role in treating infantile eczema,although the treatment effectiveness of the extensively hydrolyzed formula appears earlier than that of the partially hydrolyzed formula.