Background Recombinant human growth hormone(rhGH)therapy has shown to improve height and body composition in children with Prader–Willi syndrome(PWS),the evidence of early rhGH treatment on motor and mental developme...Background Recombinant human growth hormone(rhGH)therapy has shown to improve height and body composition in children with Prader–Willi syndrome(PWS),the evidence of early rhGH treatment on motor and mental development is still accumulating.This study explored the time effect on psychomotor development,anthropometric indexes,and safety for infants and young children with PWS.Methods A phase 3,single-arm,multicenter,self-controlled study was conducted in six sites.Patients received rhGH at 0.5 mg/m2/day for first four weeks,and 1 mg/m2/day thereafter for up to 52 weeks.Motor development was measured using Peabody Developmental Motor Scales-second edition,mental development using Griffiths Development Scales-Chinese(GDS-C).Height standard deviation score(SDS),body weight SDS,and body mass index(BMI)SDS were also assessed.Results Thirty-five patients were enrolled totally.Significant improvements were observed in height,body weight,and BMI SDS at week 52;GDS-C score showed significant improvement in general quotient(GQ)and sub-quotients.In a linear regression analysis,total motor quotient(TMQ),gross motor quotient(GMQ),and fine motor quotient were negatively correlated with age;however,treatment may attenuate deterioration of TMQ and GMQ.Changes in GQ and locomotor sub-quotient in<9-month group were significantly higher than≥9-month group.Mild to moderate severity adverse drug reactions were reported in six patients.Conclusion Fifty-two-week treatment with rhGH improved growth,BMI,mental development,and lessened the deterioration of motor function in infants and young children with PWS.Improved mental development was more pronounced when instituted in patients<9 months old.展开更多
Background The real-world exposure levels of non-therapeutic antibiotics and neonicotinoids in type 1 diabetes(T1D)children and their associations as environmental triggers through gut microbiota shifts remained unkno...Background The real-world exposure levels of non-therapeutic antibiotics and neonicotinoids in type 1 diabetes(T1D)children and their associations as environmental triggers through gut microbiota shifts remained unknown.We thus investigated the antibiotics and neonicotinoids’exposure levels and their associations with gut microbiota in pediatric T1D.Methods Fifty-one newly onset T1D children along with 67 age-matched healthy controls were recruited.Urine concentrations of 28 antibiotics and 12 neonicotinoids were measured by mass spectrometry.Children were grouped according to the kinds of antibiotics’and neonicotinoids’exposures,respectively.The 16S rRNA of fecal gut microbiota was sequenced,and the correlation with urine antibiotics and neonicotinoids’concentrations was analyzed.Results The overall detection rates of antibiotics were 72.5%and 61.2%among T1D and healthy children,whereas the neonicotinoids detection rates were 70.6%and 52.2%(P=0.044).Children exposed to one kind of antibiotic or two or more kinds of neonicotinoids had higher risk of T1D,with the odd ratios of 2.579 and 3.911.Furthermore,co-exposure to antibiotics and neonicotinoids was associated with T1D,with the odd ratio of 4.924.Antibiotics or neonicotinoids exposure did not affect overall richness and diversity of gut microbiota.However,children who were exposed to neither antibiotics nor neonicotinoids had higher abundance of Lachnospiraceae than children who were exposed to antibiotics and neonicotinoids alone or together.Conclusion High antibiotics and neonicotinoids exposures were found in T1D children,and they were associated with changes in gut microbiota featured with lower abundance of butyrate-producing genera,which might increase the risk of T1D.展开更多
文摘Background Recombinant human growth hormone(rhGH)therapy has shown to improve height and body composition in children with Prader–Willi syndrome(PWS),the evidence of early rhGH treatment on motor and mental development is still accumulating.This study explored the time effect on psychomotor development,anthropometric indexes,and safety for infants and young children with PWS.Methods A phase 3,single-arm,multicenter,self-controlled study was conducted in six sites.Patients received rhGH at 0.5 mg/m2/day for first four weeks,and 1 mg/m2/day thereafter for up to 52 weeks.Motor development was measured using Peabody Developmental Motor Scales-second edition,mental development using Griffiths Development Scales-Chinese(GDS-C).Height standard deviation score(SDS),body weight SDS,and body mass index(BMI)SDS were also assessed.Results Thirty-five patients were enrolled totally.Significant improvements were observed in height,body weight,and BMI SDS at week 52;GDS-C score showed significant improvement in general quotient(GQ)and sub-quotients.In a linear regression analysis,total motor quotient(TMQ),gross motor quotient(GMQ),and fine motor quotient were negatively correlated with age;however,treatment may attenuate deterioration of TMQ and GMQ.Changes in GQ and locomotor sub-quotient in<9-month group were significantly higher than≥9-month group.Mild to moderate severity adverse drug reactions were reported in six patients.Conclusion Fifty-two-week treatment with rhGH improved growth,BMI,mental development,and lessened the deterioration of motor function in infants and young children with PWS.Improved mental development was more pronounced when instituted in patients<9 months old.
基金supported by the National Key Research and Development Program of China(2016YFC1305302)the Clinical special project of integrated traditional Chinese and Western medicine in 2019,Shanghai Municipal Health Commission,Shanghai Municipal Administrator of Traditional Chinese Medicine.
文摘Background The real-world exposure levels of non-therapeutic antibiotics and neonicotinoids in type 1 diabetes(T1D)children and their associations as environmental triggers through gut microbiota shifts remained unknown.We thus investigated the antibiotics and neonicotinoids’exposure levels and their associations with gut microbiota in pediatric T1D.Methods Fifty-one newly onset T1D children along with 67 age-matched healthy controls were recruited.Urine concentrations of 28 antibiotics and 12 neonicotinoids were measured by mass spectrometry.Children were grouped according to the kinds of antibiotics’and neonicotinoids’exposures,respectively.The 16S rRNA of fecal gut microbiota was sequenced,and the correlation with urine antibiotics and neonicotinoids’concentrations was analyzed.Results The overall detection rates of antibiotics were 72.5%and 61.2%among T1D and healthy children,whereas the neonicotinoids detection rates were 70.6%and 52.2%(P=0.044).Children exposed to one kind of antibiotic or two or more kinds of neonicotinoids had higher risk of T1D,with the odd ratios of 2.579 and 3.911.Furthermore,co-exposure to antibiotics and neonicotinoids was associated with T1D,with the odd ratio of 4.924.Antibiotics or neonicotinoids exposure did not affect overall richness and diversity of gut microbiota.However,children who were exposed to neither antibiotics nor neonicotinoids had higher abundance of Lachnospiraceae than children who were exposed to antibiotics and neonicotinoids alone or together.Conclusion High antibiotics and neonicotinoids exposures were found in T1D children,and they were associated with changes in gut microbiota featured with lower abundance of butyrate-producing genera,which might increase the risk of T1D.