Objective Little is known about the association between whole-blood nicotinamide adenine dinucleotide(NAD^(+))levels and nabothian cysts.This study aimed to assess the association between NAD^(+)levels and nabothian c...Objective Little is known about the association between whole-blood nicotinamide adenine dinucleotide(NAD^(+))levels and nabothian cysts.This study aimed to assess the association between NAD^(+)levels and nabothian cysts in healthy Chinese women.Methods Multivariate logistic regression analysis was performed to analyze the association between NAD^(+)levels and nabothian cysts.Results The mean age was 43.0±11.5 years,and the mean level of NAD^(+)was 31.3±5.3μmol/L.Nabothian cysts occurred in 184(27.7%)participants,with single and multiple cysts in 100(15.0%)and84(12.6%)participants,respectively.The total nabothian cyst prevalence gradually decreased from37.4%to 21.6%from Q1 to Q4 of NAD^(+)and the prevalence of single and multiple nabothian cysts also decreased across the NAD^(+)quartiles.As compared with the highest NAD^(+)quartile(≥34.4μmol/L),the adjusted odds ratios with 95%confidence interval of the NAD^(+)Q1 was 1.89(1.14–3.14)for total nabothian cysts.The risk of total and single nabothian cysts linearly decreased with increasing NAD^(+)levels,while the risk of multiple nabothian cysts decreased more rapidly at NAD^(+)levels of 28.0 to35.0μmol/L.Conclusion:Low NAD^(+)levels were associated with an increased risk of total and multiple nabothian cysts.展开更多
AIM:To assess the application of multiple planar volume reconstruction(MPVR) and three-dimensional (3D) transparency lung volume rendering(TL-VR) with 64-row multidetector-row computed tomography (MDCT) in neonates wi...AIM:To assess the application of multiple planar volume reconstruction(MPVR) and three-dimensional (3D) transparency lung volume rendering(TL-VR) with 64-row multidetector-row computed tomography (MDCT) in neonates with congenital esophageal atresia (EA) and distal tracheoesophageal fistula(TEF).METHODS:Twenty neonates(17 boys,3 girls) with EA and distal TEF at a mean age of 4.6 d(range 1-16 d) were enrolled in this study.A helical scan of 64-row MDCT was performed at the 64 mm×0.625 mm collimation.EA and TEF were reconstructed with MPVR and TL-VR,respectively.Initial diagnosis of EA was made by chest radiography showing the inserted catheter in the proximal blind-ended esophageal pouch.Manifestations of MDCT images were compared with the findings at surgery.RESULTS:MDCT showed the proximal and distal esophageal pouches in 20 cases.No significant difference was observed in gaps between the proximal and distal esophageal pouches detected by MPVR and TLVR.The lengths of gaps between the proximal and distal esophageal pouches detected by MPVR and TL-VR correlated well with the findings at surgery(R=0.87,P<0.001).The images of MPVR revealed the orifice of TEF in 13 cases,while TL-VR images showed the orifice of TEF in 4 cases.CONCLUSION:EA and distal TEF can be reconstructed using MPVR and TL-VR of 64-row MDCT,which is a noninvasive technique to demonstrate the distal esophageal pouches and inter-pouch distance in neonates with EA and distal TEF.展开更多
BACKGROUND Children with congenital glaucoma are often accompanied by acquired epiblepharon in the lower eyelid,which causes entropion of the lower eyelid and damages the cornea.AIM To infer the possible causes of low...BACKGROUND Children with congenital glaucoma are often accompanied by acquired epiblepharon in the lower eyelid,which causes entropion of the lower eyelid and damages the cornea.AIM To infer the possible causes of lower eyelid entropion by comparing the difference of ocular axis and corneal diameter between inverted and non-inverted ciliary eyes in children with congenital glaucoma.METHODS A total of 15 patients(11 males and 4 females)diagnosed with congenital glaucoma between July 2016 and January 2019 at Tongren Hospital were included.Five patients had bilateral glaucoma,and ten had unilateral glaucoma.Each patient had only one eye with lower eyelid entropion which is associated with congenital glaucoma.All the patients had no entropion in another eye.The clinical data were collected.Main outcome measures were the ocular axis and corneal diameter.RESULTS The average age of the 15 patients was 1.85±0.49 years.Paired t-test showed that the average ocular axis of congenital glaucoma eyes with lower eyelid entropion(24.86±3.44 mm)was significantly longer than that of congenital glaucoma eyes without lower eyelid entropion(20.79±1.34 mm;P<0.001).The average corneal diameter of congenital glaucoma eyes with lower eyelid entropion(13.61±0.88 mm)was also significantly greater than that of congenital glaucoma eyes without lower eyelid entropion(11.63±0.48;P<0.001).CONCLUSION The rapid growth of the ocular axis and corneal diameter may be the main cause of congenital glaucoma with acquired lower eyelid entropion.Therefore,children with poor control of intraocular pressure and excessive growth of ocular axis and corneal diameter must be observed for the existence of acquired epiblepharon.展开更多
It is well known that the taste of sweet solutions produces a morphine-like analgesia in both rats and human infants, and under certain conditions, possibly in human adults. To further explore whether ingestion analge...It is well known that the taste of sweet solutions produces a morphine-like analgesia in both rats and human infants, and under certain conditions, possibly in human adults. To further explore whether ingestion analgesia persists into human adulthood, the present study was the first to utilize contact heat, a method of pain induction used commonly in both behavioural and pharmacological studies with laboratory animals. Left arms of 120 university undergraduates were exposed to a hot-plate, with pain responsivity assessed both before and after consuming either nothing (control group), or foods that they rated previously as unpalatable (e.g., black olives), neutral (e.g., rice cakes), or palatable (e.g., chocolate-chip cookies). Pain responsivity was assessed with four pain measures: pain threshold, pain tolerance, and visual analogue scale (VAS) ratings of pain intensity and unpleasantness. Between-groups comparisons in 2 separate experiments revealed that women (but not men) who consumed a palatable food showed increased pain tolerance, relative to the nothing, unpalatable, or neutral groups. Collectively, these data support our previous findings that “palatability-induced analgesia” exists in human adults, at least in females. Moreover, the findings support contact heat as a suitable method for assessing ingestion analgesia to experimental pain with human adults.展开更多
BACKGROUND Anaplastic lymphoma kinase-positive(ALK+)large B-cell lymphoma(LBCL)is a rare type of lymphoma with high invasiveness and rapid progression.It occurs in all age groups,but is extremely rare in children.The ...BACKGROUND Anaplastic lymphoma kinase-positive(ALK+)large B-cell lymphoma(LBCL)is a rare type of lymphoma with high invasiveness and rapid progression.It occurs in all age groups,but is extremely rare in children.The lesions mainly involve the lymph nodes and may present with extra-nodal involvement.Response to conventional chemotherapies and local radiotherapy is poor,with a 5-year overall survival of less than 40%.Recently,the use of ALK inhibitors for the treatment of this disease has been reported.CASE SUMMARY We present a case of a 12-year-old boy diagnosed with ALK+LBCL.The patient had a 2-mo medical history of a calvarial mass,extensive systemic involvement,and positive bone marrow clathrin heavy chain(CLTC)-ALK fusion gene.Complete remission 1(CR1)was achieved using the modified LMB89 Group C regimen followed by autologous stem cell transplantation.The patient relapsed 3 mo later.He then achieved CR2 with three short courses of chemotherapy(COP,reduceddose ICE,low-dose Ara-c+VP16)and continuous alectinib targeted therapy.Afterward,allogeneic hematopoietic stem cell transplantation(allo-HSCT)was performed.At 16 mo after the allo-HSCT,the patient was still in CR2.CONCLUSION The modified LMB89 Group C regimen and ALK inhibitors are effective.Allo-HSCT should be performed after remission.展开更多
Mycoplasma pneumoniae pneumonia (MPP) becomes one of the most important health problems in China recently. Date for MPP inChinais scarce. Although macrolides and/or cortical steroids had been reported to be effective ...Mycoplasma pneumoniae pneumonia (MPP) becomes one of the most important health problems in China recently. Date for MPP inChinais scarce. Although macrolides and/or cortical steroids had been reported to be effective treatment for MPP, the long-term outcome remained uncertain. A study on status of MPP in China was conducted via a systematic review of published studies which have the Chinese data and collected from published PubMed and core journals of China Knowledge Resource Integrated Database (CNKI). The analysis was based on epidemiology, clinical characteristics, treatment, drug resistance and prognosis. Twenty five articles concerned about MPP in Chinese children and adolescent were enrolled, including 11 studies on epidemiology/etiology, 11 studies on clinical characteristics, 7 studies on drug resistance, 5 studies on treatment from China's Mainland respectively. The overall incidence of MPP ranged from 7.1% to 54.4%. Fever and cough were most frequently identified in manifestations. Drug resistance to macrolides ranged from 18.9% to 90%. The outcome of treatment in patients who received combined treatment of macrolides, cephalosporin antibiotics and/or cortical steroid seems to be better than those who received macrolides only. Macrolide combined with cephalosporin or cortical steroid both may decrease the severity of MPP in the past decade. There was not enough evidence to suggest that cortical steroid can decrease the mortality of MPP in children. And a multi-center, randomized double blind research on the effect of cortical steroid was encouraged.展开更多
The global burden of respiratory diseases is a significant and increasing threat to individuals worldwide.In 2017,there were 544.9 million cases of chronic respiratory diseases,a 39.8%increase since 1990(1).These dise...The global burden of respiratory diseases is a significant and increasing threat to individuals worldwide.In 2017,there were 544.9 million cases of chronic respiratory diseases,a 39.8%increase since 1990(1).These diseases were the third leading cause of global mortality in 2017,accounting for 7.0%of all deaths,an 18.0%increase compared to 1990.展开更多
To the Editor:Burkitt’s lymphoma(BL)is the most common subtype of pediatric non-Hodgkin lymphoma(NHL),accounting for nearly 40%of cases.[1,2]The 5-year overall survival(OS)rate is nearly 90%owing to the use of existi...To the Editor:Burkitt’s lymphoma(BL)is the most common subtype of pediatric non-Hodgkin lymphoma(NHL),accounting for nearly 40%of cases.[1,2]The 5-year overall survival(OS)rate is nearly 90%owing to the use of existing standard chemotherapy regimens in conjunction with rituximab.[2]Approximately 25%patients with pediatric BL present with central nervous system(CNS)disease and a poor prognosis.[1]The significance of the expressions of T helper 1/T helper 2(Th1/Th2)cytokines and lymphocyte subsets in the occurrence,development,and prognosis of adult malignant lymphoma has been reported in the literature,but data on pediatric BL are limited.This study aimed to investigate the characteristics of cytokines and lymphocyte subset expression in patients with pediatric BL,identify the differences between the CNS-positive and CNS-negative groups,and determine the risk factors in the CNS-positive group.展开更多
BACKGROUND The aberrant expression of the anaplastic lymphoma kinase(ALK)gene in ALKpositive(ALK+)anaplastic large cell lymphoma(ALCL)is usually due to t(2;5)/NPM-ALK.However,rarely,aberrant ALK expression can also re...BACKGROUND The aberrant expression of the anaplastic lymphoma kinase(ALK)gene in ALKpositive(ALK+)anaplastic large cell lymphoma(ALCL)is usually due to t(2;5)/NPM-ALK.However,rarely,aberrant ALK expression can also result from a rearrangement of the ALK gene with various partner genes.Central nervous system(CNS)metastasis is very rare in ALK+ALCL.Patients with CNS involvement show an inferior prognosis.CASE SUMMARY Here,we present the case of an 8-year-old girl diagnosed with ALK+ALCL.She presented with fever,skin nodules,leg swelling,and abdominal pain over the preceding 6 mo.She had extensive involvement and showed an extraordinary rare translocation,t(2;17)/CLTC-ALK,as demonstrated by RNA-seq.She underwent chemotherapy as per ALCL99,followed by vinblastine(VBL)maintenance treatment,and achieved complete remission.However,she developed CNS relapse during VBL monotherapy.The patient achieved a durable second remission with high-dose chemotherapy(including methotrexate 8 g/m2)and continuous treatment with alectinib and VBL.CONCLUSION Alectinib showed significant and durable CNS effects in this patient.However,more cases are needed to prove the efficacy and safety of alectinib for pediatric ALK+ALCL patients.展开更多
Simulation code for a model of the adaptive immune response seen in flavivirus infections is used to explain the immunopathological consequences seen in West Nile Virus virus (WNV) infections. We use a model that spec...Simulation code for a model of the adaptive immune response seen in flavivirus infections is used to explain the immunopathological consequences seen in West Nile Virus virus (WNV) infections. We use a model that specifically handles the differences in how the virus infects resting cells, the G0 state, versus dividing cells, the G1 state, which includes vastly increased MHC-I upregulation for resting cells over dividing cells. The simulation suggests how the infection progresses in a one host model and the results shed insight into the unusual survival curve data obtained for this infection: there is an increase in health even though viral load has increased.展开更多
Tumor is one of the leading causes of death in children(0 to 14-year-old)and adolescents(15 to 19-year-old)worldwide.Unlike adult tumors,childhood and adolescent tumors are unique in their type,molecular characteristi...Tumor is one of the leading causes of death in children(0 to 14-year-old)and adolescents(15 to 19-year-old)worldwide.Unlike adult tumors,childhood and adolescent tumors are unique in their type,molecular characteristics,and pathogenesis,and their treatment involves many challenges.In recent years,with the development of a large number of clinical studies,the survival rate of children and adolescents with tumors has improved significantly.The extensive research and application of optimized treatment regimens and new targeted drugs have led to new hope for the treatment of childhood and adolescent tumors.This article reviews the clinical and basic research and treatment of childhood and adolescent tumors and provides new ideas for the future development of precise treatment of childhood and adolescent tumors.展开更多
Importance:It remained unclear that the efficacy comparison between low-dose immune tolerance induction(LD-ITI)incorporating immunosuppressants(IS)when severe hemophilia A(SHA)patients had inhibitor-titer≥200 Bethesd...Importance:It remained unclear that the efficacy comparison between low-dose immune tolerance induction(LD-ITI)incorporating immunosuppressants(IS)when severe hemophilia A(SHA)patients had inhibitor-titer≥200 Bethesda Units(BU)/mL(LD-ITI-IS^(200) regimen)and LD-ITI combining with IS when SHA patients had inhibitor-titer≥40 BU/mL(LD-ITI-IS^(40) regimen).Objective:To compare the efficacy of the LD-ITI-IS^(200) regimen with that of the LD-ITI-IS^(40) regimen for SHA patients with high-titer inhibitors.Methods:A prospective cohort study on patients receiving LD-ITI-IS^(200) compared to those receiving LD-ITI-IS^(40) from January 2021 to December 2023.Both received LD-ITI[FVIII 50 IU/kg every other day].IS(rituximab+prednisone)was added when peak inhibitor tier≥200 BU/mL in the LD-ITI-IS^(200) regimen and≥40 BU/mL in the LD-ITI-IS^(40) regimen.Success is defined as a negative inhibitor plus FVIII recovery≥66%of the expected.Results:We enrolled 30 patients on LD-ITI-IS^(200) and 64 patients on LD-ITI-IS^(40),with similar baseline clinical characteristics.A lower IS-use rate was discovered in the LD-ITI-IS^(200) regimen compared to the LD-ITI-IS^(40) regimen(30.0%vs.62.5%).The two regimens(LD-ITI-IS^(200) vs.LD-ITI-IS^(40))had similar success rate(70.0%vs.79.7%),median time to success(9.4 vs.10.6 months),and annualized bleeding rate during ITI(3.7 vs.2.8).The cost to success was lower for LD-ITI-IS^(200) than for LD-ITI-IS^(40)(2107 vs.3256 US Dollar/kg).Among patients with peak inhibitor-titer 40-199 BU/mL,10 non-IS-using(on LD-ITI-IS^(200) regimen)and 28 IS-using(on LD-ITI-IS^(40) regimen)had similar success rates(70.0%vs.78.6%)and time to success(9.0 vs.8.8 months).Interpretation:In LD-ITI,IS are not necessary for inhibitor titer<200 BU/mL.展开更多
Background:Acute leukemia is the most common pediatric hematological malignancy.Bloodstream infections (BSIs) are severe complications in these patients during chemotherapy.This study aimed to explore the clinical ...Background:Acute leukemia is the most common pediatric hematological malignancy.Bloodstream infections (BSIs) are severe complications in these patients during chemotherapy.This study aimed to explore the clinical presentation and etiology of BSI,as well as the common sites of infection,and to provide a basis for the rational regarding antibiotic use.Methods:We performed a retrospective chart review of all pediatric patients who had acute leukemia accompanied by a BSI in our hospital from December 2011 to September 2015.All patients were selected based on clinical presentation and had to have at least one positive blood culture for inclusion.The basic clinical characteristics,blood culture results,and antimicrobial susceptibilities were analyzed.Results:All 231 patients had a fever;of them,12 patients continued to have a fever.Twenty-five patients had nonremitting (NR) leukemia,and 206 patients achieved complete remission (CR).Differences in the duration of fever between the NR and CR groups were significant (9.6 &#177; 7.9 vs.5.1 &#177; 3.8 days,P =0.016).One hundred and eighty patients had agranulocytosis.Differences in fever duration between the agranulocytosis and nonagranulocytosis groups were significant (6.2 &#177; 5.1 vs.4.1 &#177; 2.6 days,P =0.001).The other sites of infection in these 231 patients were the lung,mouth,digestive tract,and rectum.Blood culture comprised 2635 samples.There were 619 samples,which were positive.Of the 619 positive blood culture samples,59.9% had Gram-negative bacteria,39.3% had Gram-positive bacteria,and 0.8% had fungus.The primary pathogens were Pseudomonas aeruginosa,Enterobactercloacae,Escherichia coli,and Klebsiella pneumoniae.Of these 231 patients,217 patients were cured.The effective treatment ratio was 94%.Conclusions:Gram-negative bacteria were the main pathogenic bacteria in patients with acute leukemia in our center.NR primary illness,agranulocytosis,and drug-resistant pathogenic bacteria were all risk factors for poor prognosis.展开更多
Background Primary Ewing's sarcoma/primitive neuroectodermal tumor (ES/PNET) of urogenital tract is a rare condition with non-specific clinical presentations, which can make it difficult to diagnose. In this study,...Background Primary Ewing's sarcoma/primitive neuroectodermal tumor (ES/PNET) of urogenital tract is a rare condition with non-specific clinical presentations, which can make it difficult to diagnose. In this study, we summarize the clinical presentation, pathological features, therapeutic strategies, and prognosis of ES/PNET.展开更多
Background:Endobronchial tuberculosis (EBTB) is the most frequent complication of primary pulmonary tuberculosis (PTB) in children.The aim of the study was to analyze characteristics and clinical role of bronchoscopy ...Background:Endobronchial tuberculosis (EBTB) is the most frequent complication of primary pulmonary tuberculosis (PTB) in children.The aim of the study was to analyze characteristics and clinical role of bronchoscopy in diagnosis of childhood EBTB.Methods:A retrospective,descriptive study was undertaken in 157 children with EBTB undergone flexible bronchoscopy (FB) between January 2006 and June 2014.Results:The median age of the enrolled patients was 3.4 years,with 73.2% of patients under five years old.The most common subtype was tumorous type (145/157,92.4%).If only involved bronchus were considered,the common affected sites were right middle lobe bronchus (49/228,21.5%),left upper lobe bronchus (41/228,18.0%),right upper lobe bronchus (41/228,18.0%),right main bronchus (35/228,15.4%),respectively.Children younger than five years old were at higher risk to have multiple endobronchial lesions (P=0.044),with an odds ratio of 2.313 (95% confidence interval:1.009-5.299).Before the bronchoscopy,only 16 (10.2%)patients were highly suspected of EBTB,while the others were diagnosed as PTB without EBTB (69.4%),or misdiagnosed as pneumonia or foreign body aspiration (20.4%) on admission.Conclusions:The patients under five years old are at high risk to progress to EBTB and have multiple endobronchial lesions.The most frequent subtype of EBTB in children is tumorous type.The lesions are seen in the right bronchial system more frequently.FB should be performed to detect the endobronchial lesions in suspected patients as soon as possible.展开更多
Background Resistance to thyroid hormone (RTH) is a dominant inherited syndrome of reduced tissue responsiveness to thyroid hormone. It is usually due to mutations located at the ligand-binding domain and adjacent h...Background Resistance to thyroid hormone (RTH) is a dominant inherited syndrome of reduced tissue responsiveness to thyroid hormone. It is usually due to mutations located at the ligand-binding domain and adjacent hinge region of the thyroid hormone receptor β(TRβ). We report the clinical and laboratory characteristics and the genetic analysis of a patient with this rare disorder and his family members. Methods The clinical presentations and changes of thyroid function tests (TFTs) including magnetic resonance imaging (MRI) of pituitary and other laboratory tests were analysed. TFTs of his family's members were detected as well. Direct DNA sequencing of the TRβ gene was done for those with abnormal TFTs. Results The RTH child had goiter, irritability, aggressiveness, and sudoresis. His TFTs showed high levels of circulating free thyroid hormones (FT4 and FT3) and normal thyroid-stimulating hormone (TSH) concentrations. He felt worse when treated as hyperthyroidism (Grave disease) with thiamazole and his clinical presentations got improved obviously when treated as RTH with bromocriptine without obvious advert effect. We identified a novel missense mutation, A317D, located in exon 9 of the gene of this boy and his mother. His mother had not any clinical presentation, but having abnormal TFTs results. Conclusions This patient reported here was concordant with the criteria of RTH. The feature is dysfunction of hypothalamus-pituitary-thyroid axis. A novel mutation was found in the TRβ, A317D, of this family. This research verified the phenomena that there is a clinical heterogeneity within the same mutation of different RTH patients.展开更多
Background: Although much attention has been paid to the pharmacokinetics (PKs) of different factor VIII (FVIlI) concentrates in persons with hemophilia A (HA), limited information is available in young boys wi...Background: Although much attention has been paid to the pharmacokinetics (PKs) of different factor VIII (FVIlI) concentrates in persons with hemophilia A (HA), limited information is available in young boys with severe HA. In this study, we aimed to assess the PK parameters of FVIII products in boys with severe HA in China. Methods: A total of 36 boys (plasma-derived [pd]-FVIII, n= 15; recombinant [r] FVIII, n = 21) were enrolled between January 2015 and May 2016 in Beijing Children's Hospital. PK characteristics of FVIII products were studied according to a reduced 4-sampling time point design (1 h, 9 h, 24 h, and 48 h postinfusion). Results: The mean FVIII half-life (t1/2) was 10.99 ± 3.45 h (range 5.52-20.02 h), the mean in vivo recovery (IVR) was 2.01 ± 0.42 IU/dl per IU/kg (range 1.24-3.02 IU/dl per IU/kg) and mean clearance (CL) of FVIII is 4.34 ± 1.58 ml·kg^-1·h ^-1 (range 2.29-7.90 ml·kg^-1·h·^-1). We also analyzed the influence of several parameters that potentially modulate FVIII PK. The age was closely associated with FVIII half-life (R^2 = 0.32, P 〈 0.01 ). The t1/2 of FVIII increased by 0.59 h per year. Besides age, yon Willebrand factor antigen (VWF:Ag) also was associated with FVIII half-life (R^2 = 0.52, P 〈 0.01). Patients with blood Group O had a shorter FVIII halt-life than patients with non-O blood group (9.40 ± 0.68 h vs. 12.3 ± 0.79 h, t = 2.70, P = 0.01). The FVIII IVR correlated with age (R^2 =0.21, P 〈 0.01) and VWF:Ag level (R^2 = 0.28, P 〈 0.01 ). CL rates were taster in young patients and in those with low-VWF:Ag levels. CL rates of FVIII are higher in blood Group O versus non-blood Group O persons (5.02 ± 0.38 vs. 4.00 ± 0.32 ml·kg^-1·h^-1 , t = 2.53, P = 0.02). Conclusions: Chinese boys with severe HA have similar PK values to other ethnic groups and large differences in FVIII PK between individual patients. Age, blood group, and VWF:Ag levels are important determining factors for FVlll CL.展开更多
Background:Infections remain a major cause of therapy-associated morbidity and mortality in children with acute lymphoblastic leukemia (ALL).Methods:We retrospectively analyzed the medical charts of 256 children t...Background:Infections remain a major cause of therapy-associated morbidity and mortality in children with acute lymphoblastic leukemia (ALL).Methods:We retrospectively analyzed the medical charts of 256 children treated for ALL under the CCLG-2008 protocol in Beijing Children's Hospital.Results:There were 65 infectious complications in 50 patients during vincristine,daunorubicin,L-asparaginase and dexamethasone induction therapy,including microbiologically documented infections (n =12; 18.5%),clinically documented infections (n =23; 35.3%) and fever of unknown origin (n =30; 46.2%).Neutropenia was present in 83.1% of the infectious episodes.In all,most infections occurred around the 15t1h day of induction treatment (n =28),and no patients died of infection-associated complications.Conclusions:The infections in this study was independent of treatment response,minimal residual diseases at the end of induction therapy,gender,immunophenotype,infection at first visit,risk stratification at diagnosis,unfavorable karyotypes at diagnosis and morphologic type.The infection rate of CCLG-2008 induction therapy is low,and the outcome of patients is favorable.展开更多
Background This study aimed to evaluate the feasibility and clinical effect of haploidentical hematopoietic stem cell transplantation(haplo-HSCT)for the treatment of pediatric patients with chronic active Epstein-Barr...Background This study aimed to evaluate the feasibility and clinical effect of haploidentical hematopoietic stem cell transplantation(haplo-HSCT)for the treatment of pediatric patients with chronic active Epstein-Barr virus infection(CAEBV).Methods Children with CAEBV who did not have matched donors and underwent haplo-HSCT in Beijing Children's Hospital,Capital Medical University,from October 2016 to June 2020 were analyzed retrospectively.Data relating to the clinical manifestations,engraftment,and prognosis of the children were extracted from medical records.Results Twenty-five patients,including 16 males and 9 females,with an onset age of 5.0±2.6 years and a transplantation age of 6.9±2.9 years,were enrolled irnhis study.The mean time from diagnosis to transplantation was 3.8(2.0-40.2)months.The mean observation time was 19.0±12.0 months.Three patients received the reduced intensity conditioning regimen,and the remaining patients all received the modified myeloablative conditioning regimen.By the end of the follow-up,23 patients were characterized by disease-free survival(DFS),22 were characterized by event-free survival(EFS).and two died.One of the patients died of thrombotic microangiopathy(TMA),and another died of graft versus host disease(GVHD);this patient discontinued the treatment for economic reasons.The 3-year overall survival(OS)rate was estimated to be 92.0%±5.4%,and the 3-year EFS rate was estimated to be 87.4%±6.8%.All active patients survived after HSCT event-free.Acute GVHD degrees 1-3 were observed in ten patients(40.0%),and degree IV was observed in six(24.0%),who were all cured except for one patient.Chronic GVHD was observed in nine(36.0%),and most of these cases were mild.The incidence of TMA and veno-occlusive disease(VOD)was 28.0%and 4.0%.Conclusions Haploidentical hematopoietic stem cell transplantation is safe and effective in the treatment of pediatric CAEBV and can be used as an alternative therapy without matched donors or emergency transplantation.Patients with active disease before HSCT also benefited from haplo-HSCT.Haplo-HSCT requires careful monitoring for complications,such as GVHD and TMA.Early detection of TMA and timely treatment can reduce mortality and can improve the survival rate.展开更多
Staphylococcus aureus is associated with a variety of invasive infection; typically, these infections occur as sepsis, osteomyelitis, endocarditis, and arthritis. China has a relatively high incidence of invasive S. a...Staphylococcus aureus is associated with a variety of invasive infection; typically, these infections occur as sepsis, osteomyelitis, endocarditis, and arthritis. China has a relatively high incidence of invasive S. aureus disease in children.[1] The present study aimed to provide the demographics, clones, and the antimicrobial susceptibility of S. aureus that cause invasive infection in Chinese children.展开更多
基金supported by grants from the NSFC-Regional Innovation and Development Joint Fund(No.U22A20364)the National Key R&D Program of China(No.2021YFC2500500)the National Natural Science Foundation of China(No.81973112,No.92049302)。
文摘Objective Little is known about the association between whole-blood nicotinamide adenine dinucleotide(NAD^(+))levels and nabothian cysts.This study aimed to assess the association between NAD^(+)levels and nabothian cysts in healthy Chinese women.Methods Multivariate logistic regression analysis was performed to analyze the association between NAD^(+)levels and nabothian cysts.Results The mean age was 43.0±11.5 years,and the mean level of NAD^(+)was 31.3±5.3μmol/L.Nabothian cysts occurred in 184(27.7%)participants,with single and multiple cysts in 100(15.0%)and84(12.6%)participants,respectively.The total nabothian cyst prevalence gradually decreased from37.4%to 21.6%from Q1 to Q4 of NAD^(+)and the prevalence of single and multiple nabothian cysts also decreased across the NAD^(+)quartiles.As compared with the highest NAD^(+)quartile(≥34.4μmol/L),the adjusted odds ratios with 95%confidence interval of the NAD^(+)Q1 was 1.89(1.14–3.14)for total nabothian cysts.The risk of total and single nabothian cysts linearly decreased with increasing NAD^(+)levels,while the risk of multiple nabothian cysts decreased more rapidly at NAD^(+)levels of 28.0 to35.0μmol/L.Conclusion:Low NAD^(+)levels were associated with an increased risk of total and multiple nabothian cysts.
文摘AIM:To assess the application of multiple planar volume reconstruction(MPVR) and three-dimensional (3D) transparency lung volume rendering(TL-VR) with 64-row multidetector-row computed tomography (MDCT) in neonates with congenital esophageal atresia (EA) and distal tracheoesophageal fistula(TEF).METHODS:Twenty neonates(17 boys,3 girls) with EA and distal TEF at a mean age of 4.6 d(range 1-16 d) were enrolled in this study.A helical scan of 64-row MDCT was performed at the 64 mm×0.625 mm collimation.EA and TEF were reconstructed with MPVR and TL-VR,respectively.Initial diagnosis of EA was made by chest radiography showing the inserted catheter in the proximal blind-ended esophageal pouch.Manifestations of MDCT images were compared with the findings at surgery.RESULTS:MDCT showed the proximal and distal esophageal pouches in 20 cases.No significant difference was observed in gaps between the proximal and distal esophageal pouches detected by MPVR and TLVR.The lengths of gaps between the proximal and distal esophageal pouches detected by MPVR and TL-VR correlated well with the findings at surgery(R=0.87,P<0.001).The images of MPVR revealed the orifice of TEF in 13 cases,while TL-VR images showed the orifice of TEF in 4 cases.CONCLUSION:EA and distal TEF can be reconstructed using MPVR and TL-VR of 64-row MDCT,which is a noninvasive technique to demonstrate the distal esophageal pouches and inter-pouch distance in neonates with EA and distal TEF.
文摘BACKGROUND Children with congenital glaucoma are often accompanied by acquired epiblepharon in the lower eyelid,which causes entropion of the lower eyelid and damages the cornea.AIM To infer the possible causes of lower eyelid entropion by comparing the difference of ocular axis and corneal diameter between inverted and non-inverted ciliary eyes in children with congenital glaucoma.METHODS A total of 15 patients(11 males and 4 females)diagnosed with congenital glaucoma between July 2016 and January 2019 at Tongren Hospital were included.Five patients had bilateral glaucoma,and ten had unilateral glaucoma.Each patient had only one eye with lower eyelid entropion which is associated with congenital glaucoma.All the patients had no entropion in another eye.The clinical data were collected.Main outcome measures were the ocular axis and corneal diameter.RESULTS The average age of the 15 patients was 1.85±0.49 years.Paired t-test showed that the average ocular axis of congenital glaucoma eyes with lower eyelid entropion(24.86±3.44 mm)was significantly longer than that of congenital glaucoma eyes without lower eyelid entropion(20.79±1.34 mm;P<0.001).The average corneal diameter of congenital glaucoma eyes with lower eyelid entropion(13.61±0.88 mm)was also significantly greater than that of congenital glaucoma eyes without lower eyelid entropion(11.63±0.48;P<0.001).CONCLUSION The rapid growth of the ocular axis and corneal diameter may be the main cause of congenital glaucoma with acquired lower eyelid entropion.Therefore,children with poor control of intraocular pressure and excessive growth of ocular axis and corneal diameter must be observed for the existence of acquired epiblepharon.
文摘It is well known that the taste of sweet solutions produces a morphine-like analgesia in both rats and human infants, and under certain conditions, possibly in human adults. To further explore whether ingestion analgesia persists into human adulthood, the present study was the first to utilize contact heat, a method of pain induction used commonly in both behavioural and pharmacological studies with laboratory animals. Left arms of 120 university undergraduates were exposed to a hot-plate, with pain responsivity assessed both before and after consuming either nothing (control group), or foods that they rated previously as unpalatable (e.g., black olives), neutral (e.g., rice cakes), or palatable (e.g., chocolate-chip cookies). Pain responsivity was assessed with four pain measures: pain threshold, pain tolerance, and visual analogue scale (VAS) ratings of pain intensity and unpleasantness. Between-groups comparisons in 2 separate experiments revealed that women (but not men) who consumed a palatable food showed increased pain tolerance, relative to the nothing, unpalatable, or neutral groups. Collectively, these data support our previous findings that “palatability-induced analgesia” exists in human adults, at least in females. Moreover, the findings support contact heat as a suitable method for assessing ingestion analgesia to experimental pain with human adults.
基金The Special Fund of the Pediatric Medical Coordinated Development Center of Beijing Hospitals Authority,No.XTZD20180204.
文摘BACKGROUND Anaplastic lymphoma kinase-positive(ALK+)large B-cell lymphoma(LBCL)is a rare type of lymphoma with high invasiveness and rapid progression.It occurs in all age groups,but is extremely rare in children.The lesions mainly involve the lymph nodes and may present with extra-nodal involvement.Response to conventional chemotherapies and local radiotherapy is poor,with a 5-year overall survival of less than 40%.Recently,the use of ALK inhibitors for the treatment of this disease has been reported.CASE SUMMARY We present a case of a 12-year-old boy diagnosed with ALK+LBCL.The patient had a 2-mo medical history of a calvarial mass,extensive systemic involvement,and positive bone marrow clathrin heavy chain(CLTC)-ALK fusion gene.Complete remission 1(CR1)was achieved using the modified LMB89 Group C regimen followed by autologous stem cell transplantation.The patient relapsed 3 mo later.He then achieved CR2 with three short courses of chemotherapy(COP,reduceddose ICE,low-dose Ara-c+VP16)and continuous alectinib targeted therapy.Afterward,allogeneic hematopoietic stem cell transplantation(allo-HSCT)was performed.At 16 mo after the allo-HSCT,the patient was still in CR2.CONCLUSION The modified LMB89 Group C regimen and ALK inhibitors are effective.Allo-HSCT should be performed after remission.
文摘Mycoplasma pneumoniae pneumonia (MPP) becomes one of the most important health problems in China recently. Date for MPP inChinais scarce. Although macrolides and/or cortical steroids had been reported to be effective treatment for MPP, the long-term outcome remained uncertain. A study on status of MPP in China was conducted via a systematic review of published studies which have the Chinese data and collected from published PubMed and core journals of China Knowledge Resource Integrated Database (CNKI). The analysis was based on epidemiology, clinical characteristics, treatment, drug resistance and prognosis. Twenty five articles concerned about MPP in Chinese children and adolescent were enrolled, including 11 studies on epidemiology/etiology, 11 studies on clinical characteristics, 7 studies on drug resistance, 5 studies on treatment from China's Mainland respectively. The overall incidence of MPP ranged from 7.1% to 54.4%. Fever and cough were most frequently identified in manifestations. Drug resistance to macrolides ranged from 18.9% to 90%. The outcome of treatment in patients who received combined treatment of macrolides, cephalosporin antibiotics and/or cortical steroid seems to be better than those who received macrolides only. Macrolide combined with cephalosporin or cortical steroid both may decrease the severity of MPP in the past decade. There was not enough evidence to suggest that cortical steroid can decrease the mortality of MPP in children. And a multi-center, randomized double blind research on the effect of cortical steroid was encouraged.
基金Supported by the National Natural Science Foundation of China(Grant Numbers:72122001 and 72211540398)Chinese Academy of Medical Sciences Innovation Fund for Medical Sciences(2022-I2M-CoV19-0062021-I2M-1-044).
文摘The global burden of respiratory diseases is a significant and increasing threat to individuals worldwide.In 2017,there were 544.9 million cases of chronic respiratory diseases,a 39.8%increase since 1990(1).These diseases were the third leading cause of global mortality in 2017,accounting for 7.0%of all deaths,an 18.0%increase compared to 1990.
基金supported by the Special Fund of the Pediatric Medical Coordinated Development Center of the Beijing Children’s Hospital(No.XTZD20180204)
文摘To the Editor:Burkitt’s lymphoma(BL)is the most common subtype of pediatric non-Hodgkin lymphoma(NHL),accounting for nearly 40%of cases.[1,2]The 5-year overall survival(OS)rate is nearly 90%owing to the use of existing standard chemotherapy regimens in conjunction with rituximab.[2]Approximately 25%patients with pediatric BL present with central nervous system(CNS)disease and a poor prognosis.[1]The significance of the expressions of T helper 1/T helper 2(Th1/Th2)cytokines and lymphocyte subsets in the occurrence,development,and prognosis of adult malignant lymphoma has been reported in the literature,but data on pediatric BL are limited.This study aimed to investigate the characteristics of cytokines and lymphocyte subset expression in patients with pediatric BL,identify the differences between the CNS-positive and CNS-negative groups,and determine the risk factors in the CNS-positive group.
基金Supported by the Special Fund of the Pediatric Medical Coordinated Development Center of Beijing Hospitals Authority,No.XTZD20180204。
文摘BACKGROUND The aberrant expression of the anaplastic lymphoma kinase(ALK)gene in ALKpositive(ALK+)anaplastic large cell lymphoma(ALCL)is usually due to t(2;5)/NPM-ALK.However,rarely,aberrant ALK expression can also result from a rearrangement of the ALK gene with various partner genes.Central nervous system(CNS)metastasis is very rare in ALK+ALCL.Patients with CNS involvement show an inferior prognosis.CASE SUMMARY Here,we present the case of an 8-year-old girl diagnosed with ALK+ALCL.She presented with fever,skin nodules,leg swelling,and abdominal pain over the preceding 6 mo.She had extensive involvement and showed an extraordinary rare translocation,t(2;17)/CLTC-ALK,as demonstrated by RNA-seq.She underwent chemotherapy as per ALCL99,followed by vinblastine(VBL)maintenance treatment,and achieved complete remission.However,she developed CNS relapse during VBL monotherapy.The patient achieved a durable second remission with high-dose chemotherapy(including methotrexate 8 g/m2)and continuous treatment with alectinib and VBL.CONCLUSION Alectinib showed significant and durable CNS effects in this patient.However,more cases are needed to prove the efficacy and safety of alectinib for pediatric ALK+ALCL patients.
文摘Simulation code for a model of the adaptive immune response seen in flavivirus infections is used to explain the immunopathological consequences seen in West Nile Virus virus (WNV) infections. We use a model that specifically handles the differences in how the virus infects resting cells, the G0 state, versus dividing cells, the G1 state, which includes vastly increased MHC-I upregulation for resting cells over dividing cells. The simulation suggests how the infection progresses in a one host model and the results shed insight into the unusual survival curve data obtained for this infection: there is an increase in health even though viral load has increased.
文摘Tumor is one of the leading causes of death in children(0 to 14-year-old)and adolescents(15 to 19-year-old)worldwide.Unlike adult tumors,childhood and adolescent tumors are unique in their type,molecular characteristics,and pathogenesis,and their treatment involves many challenges.In recent years,with the development of a large number of clinical studies,the survival rate of children and adolescents with tumors has improved significantly.The extensive research and application of optimized treatment regimens and new targeted drugs have led to new hope for the treatment of childhood and adolescent tumors.This article reviews the clinical and basic research and treatment of childhood and adolescent tumors and provides new ideas for the future development of precise treatment of childhood and adolescent tumors.
基金Capital Health Development Research Project,Grant/Award Number:2022-2-2093Beijing Research Ward Construction Demonstration Unit Project,Grant/Award Number:BCRW202101+1 种基金National Natural Science Foundation of China,Grant/Award Number:82270133Beijing Municipal Scienceand Technology Commission,Grant/Award Number:Z221100007422067。
文摘Importance:It remained unclear that the efficacy comparison between low-dose immune tolerance induction(LD-ITI)incorporating immunosuppressants(IS)when severe hemophilia A(SHA)patients had inhibitor-titer≥200 Bethesda Units(BU)/mL(LD-ITI-IS^(200) regimen)and LD-ITI combining with IS when SHA patients had inhibitor-titer≥40 BU/mL(LD-ITI-IS^(40) regimen).Objective:To compare the efficacy of the LD-ITI-IS^(200) regimen with that of the LD-ITI-IS^(40) regimen for SHA patients with high-titer inhibitors.Methods:A prospective cohort study on patients receiving LD-ITI-IS^(200) compared to those receiving LD-ITI-IS^(40) from January 2021 to December 2023.Both received LD-ITI[FVIII 50 IU/kg every other day].IS(rituximab+prednisone)was added when peak inhibitor tier≥200 BU/mL in the LD-ITI-IS^(200) regimen and≥40 BU/mL in the LD-ITI-IS^(40) regimen.Success is defined as a negative inhibitor plus FVIII recovery≥66%of the expected.Results:We enrolled 30 patients on LD-ITI-IS^(200) and 64 patients on LD-ITI-IS^(40),with similar baseline clinical characteristics.A lower IS-use rate was discovered in the LD-ITI-IS^(200) regimen compared to the LD-ITI-IS^(40) regimen(30.0%vs.62.5%).The two regimens(LD-ITI-IS^(200) vs.LD-ITI-IS^(40))had similar success rate(70.0%vs.79.7%),median time to success(9.4 vs.10.6 months),and annualized bleeding rate during ITI(3.7 vs.2.8).The cost to success was lower for LD-ITI-IS^(200) than for LD-ITI-IS^(40)(2107 vs.3256 US Dollar/kg).Among patients with peak inhibitor-titer 40-199 BU/mL,10 non-IS-using(on LD-ITI-IS^(200) regimen)and 28 IS-using(on LD-ITI-IS^(40) regimen)had similar success rates(70.0%vs.78.6%)and time to success(9.0 vs.8.8 months).Interpretation:In LD-ITI,IS are not necessary for inhibitor titer<200 BU/mL.
文摘Background:Acute leukemia is the most common pediatric hematological malignancy.Bloodstream infections (BSIs) are severe complications in these patients during chemotherapy.This study aimed to explore the clinical presentation and etiology of BSI,as well as the common sites of infection,and to provide a basis for the rational regarding antibiotic use.Methods:We performed a retrospective chart review of all pediatric patients who had acute leukemia accompanied by a BSI in our hospital from December 2011 to September 2015.All patients were selected based on clinical presentation and had to have at least one positive blood culture for inclusion.The basic clinical characteristics,blood culture results,and antimicrobial susceptibilities were analyzed.Results:All 231 patients had a fever;of them,12 patients continued to have a fever.Twenty-five patients had nonremitting (NR) leukemia,and 206 patients achieved complete remission (CR).Differences in the duration of fever between the NR and CR groups were significant (9.6 &#177; 7.9 vs.5.1 &#177; 3.8 days,P =0.016).One hundred and eighty patients had agranulocytosis.Differences in fever duration between the agranulocytosis and nonagranulocytosis groups were significant (6.2 &#177; 5.1 vs.4.1 &#177; 2.6 days,P =0.001).The other sites of infection in these 231 patients were the lung,mouth,digestive tract,and rectum.Blood culture comprised 2635 samples.There were 619 samples,which were positive.Of the 619 positive blood culture samples,59.9% had Gram-negative bacteria,39.3% had Gram-positive bacteria,and 0.8% had fungus.The primary pathogens were Pseudomonas aeruginosa,Enterobactercloacae,Escherichia coli,and Klebsiella pneumoniae.Of these 231 patients,217 patients were cured.The effective treatment ratio was 94%.Conclusions:Gram-negative bacteria were the main pathogenic bacteria in patients with acute leukemia in our center.NR primary illness,agranulocytosis,and drug-resistant pathogenic bacteria were all risk factors for poor prognosis.
文摘Background Primary Ewing's sarcoma/primitive neuroectodermal tumor (ES/PNET) of urogenital tract is a rare condition with non-specific clinical presentations, which can make it difficult to diagnose. In this study, we summarize the clinical presentation, pathological features, therapeutic strategies, and prognosis of ES/PNET.
文摘Background:Endobronchial tuberculosis (EBTB) is the most frequent complication of primary pulmonary tuberculosis (PTB) in children.The aim of the study was to analyze characteristics and clinical role of bronchoscopy in diagnosis of childhood EBTB.Methods:A retrospective,descriptive study was undertaken in 157 children with EBTB undergone flexible bronchoscopy (FB) between January 2006 and June 2014.Results:The median age of the enrolled patients was 3.4 years,with 73.2% of patients under five years old.The most common subtype was tumorous type (145/157,92.4%).If only involved bronchus were considered,the common affected sites were right middle lobe bronchus (49/228,21.5%),left upper lobe bronchus (41/228,18.0%),right upper lobe bronchus (41/228,18.0%),right main bronchus (35/228,15.4%),respectively.Children younger than five years old were at higher risk to have multiple endobronchial lesions (P=0.044),with an odds ratio of 2.313 (95% confidence interval:1.009-5.299).Before the bronchoscopy,only 16 (10.2%)patients were highly suspected of EBTB,while the others were diagnosed as PTB without EBTB (69.4%),or misdiagnosed as pneumonia or foreign body aspiration (20.4%) on admission.Conclusions:The patients under five years old are at high risk to progress to EBTB and have multiple endobronchial lesions.The most frequent subtype of EBTB in children is tumorous type.The lesions are seen in the right bronchial system more frequently.FB should be performed to detect the endobronchial lesions in suspected patients as soon as possible.
文摘Background Resistance to thyroid hormone (RTH) is a dominant inherited syndrome of reduced tissue responsiveness to thyroid hormone. It is usually due to mutations located at the ligand-binding domain and adjacent hinge region of the thyroid hormone receptor β(TRβ). We report the clinical and laboratory characteristics and the genetic analysis of a patient with this rare disorder and his family members. Methods The clinical presentations and changes of thyroid function tests (TFTs) including magnetic resonance imaging (MRI) of pituitary and other laboratory tests were analysed. TFTs of his family's members were detected as well. Direct DNA sequencing of the TRβ gene was done for those with abnormal TFTs. Results The RTH child had goiter, irritability, aggressiveness, and sudoresis. His TFTs showed high levels of circulating free thyroid hormones (FT4 and FT3) and normal thyroid-stimulating hormone (TSH) concentrations. He felt worse when treated as hyperthyroidism (Grave disease) with thiamazole and his clinical presentations got improved obviously when treated as RTH with bromocriptine without obvious advert effect. We identified a novel missense mutation, A317D, located in exon 9 of the gene of this boy and his mother. His mother had not any clinical presentation, but having abnormal TFTs results. Conclusions This patient reported here was concordant with the criteria of RTH. The feature is dysfunction of hypothalamus-pituitary-thyroid axis. A novel mutation was found in the TRβ, A317D, of this family. This research verified the phenomena that there is a clinical heterogeneity within the same mutation of different RTH patients.
文摘Background: Although much attention has been paid to the pharmacokinetics (PKs) of different factor VIII (FVIlI) concentrates in persons with hemophilia A (HA), limited information is available in young boys with severe HA. In this study, we aimed to assess the PK parameters of FVIII products in boys with severe HA in China. Methods: A total of 36 boys (plasma-derived [pd]-FVIII, n= 15; recombinant [r] FVIII, n = 21) were enrolled between January 2015 and May 2016 in Beijing Children's Hospital. PK characteristics of FVIII products were studied according to a reduced 4-sampling time point design (1 h, 9 h, 24 h, and 48 h postinfusion). Results: The mean FVIII half-life (t1/2) was 10.99 ± 3.45 h (range 5.52-20.02 h), the mean in vivo recovery (IVR) was 2.01 ± 0.42 IU/dl per IU/kg (range 1.24-3.02 IU/dl per IU/kg) and mean clearance (CL) of FVIII is 4.34 ± 1.58 ml·kg^-1·h ^-1 (range 2.29-7.90 ml·kg^-1·h·^-1). We also analyzed the influence of several parameters that potentially modulate FVIII PK. The age was closely associated with FVIII half-life (R^2 = 0.32, P 〈 0.01 ). The t1/2 of FVIII increased by 0.59 h per year. Besides age, yon Willebrand factor antigen (VWF:Ag) also was associated with FVIII half-life (R^2 = 0.52, P 〈 0.01). Patients with blood Group O had a shorter FVIII halt-life than patients with non-O blood group (9.40 ± 0.68 h vs. 12.3 ± 0.79 h, t = 2.70, P = 0.01). The FVIII IVR correlated with age (R^2 =0.21, P 〈 0.01) and VWF:Ag level (R^2 = 0.28, P 〈 0.01 ). CL rates were taster in young patients and in those with low-VWF:Ag levels. CL rates of FVIII are higher in blood Group O versus non-blood Group O persons (5.02 ± 0.38 vs. 4.00 ± 0.32 ml·kg^-1·h^-1 , t = 2.53, P = 0.02). Conclusions: Chinese boys with severe HA have similar PK values to other ethnic groups and large differences in FVIII PK between individual patients. Age, blood group, and VWF:Ag levels are important determining factors for FVlll CL.
基金grants from Beijing Municipal Administration of Hospitals Clinical medicine Development of special funding support,the National Natural Science Foundation of China,Beijing Municipal Natural Science Foundation,Specialized Research Fund for the Doctoral Program of Higher Education,BCH Young Investigator Program
文摘Background:Infections remain a major cause of therapy-associated morbidity and mortality in children with acute lymphoblastic leukemia (ALL).Methods:We retrospectively analyzed the medical charts of 256 children treated for ALL under the CCLG-2008 protocol in Beijing Children's Hospital.Results:There were 65 infectious complications in 50 patients during vincristine,daunorubicin,L-asparaginase and dexamethasone induction therapy,including microbiologically documented infections (n =12; 18.5%),clinically documented infections (n =23; 35.3%) and fever of unknown origin (n =30; 46.2%).Neutropenia was present in 83.1% of the infectious episodes.In all,most infections occurred around the 15t1h day of induction treatment (n =28),and no patients died of infection-associated complications.Conclusions:The infections in this study was independent of treatment response,minimal residual diseases at the end of induction therapy,gender,immunophenotype,infection at first visit,risk stratification at diagnosis,unfavorable karyotypes at diagnosis and morphologic type.The infection rate of CCLG-2008 induction therapy is low,and the outcome of patients is favorable.
文摘Background This study aimed to evaluate the feasibility and clinical effect of haploidentical hematopoietic stem cell transplantation(haplo-HSCT)for the treatment of pediatric patients with chronic active Epstein-Barr virus infection(CAEBV).Methods Children with CAEBV who did not have matched donors and underwent haplo-HSCT in Beijing Children's Hospital,Capital Medical University,from October 2016 to June 2020 were analyzed retrospectively.Data relating to the clinical manifestations,engraftment,and prognosis of the children were extracted from medical records.Results Twenty-five patients,including 16 males and 9 females,with an onset age of 5.0±2.6 years and a transplantation age of 6.9±2.9 years,were enrolled irnhis study.The mean time from diagnosis to transplantation was 3.8(2.0-40.2)months.The mean observation time was 19.0±12.0 months.Three patients received the reduced intensity conditioning regimen,and the remaining patients all received the modified myeloablative conditioning regimen.By the end of the follow-up,23 patients were characterized by disease-free survival(DFS),22 were characterized by event-free survival(EFS).and two died.One of the patients died of thrombotic microangiopathy(TMA),and another died of graft versus host disease(GVHD);this patient discontinued the treatment for economic reasons.The 3-year overall survival(OS)rate was estimated to be 92.0%±5.4%,and the 3-year EFS rate was estimated to be 87.4%±6.8%.All active patients survived after HSCT event-free.Acute GVHD degrees 1-3 were observed in ten patients(40.0%),and degree IV was observed in six(24.0%),who were all cured except for one patient.Chronic GVHD was observed in nine(36.0%),and most of these cases were mild.The incidence of TMA and veno-occlusive disease(VOD)was 28.0%and 4.0%.Conclusions Haploidentical hematopoietic stem cell transplantation is safe and effective in the treatment of pediatric CAEBV and can be used as an alternative therapy without matched donors or emergency transplantation.Patients with active disease before HSCT also benefited from haplo-HSCT.Haplo-HSCT requires careful monitoring for complications,such as GVHD and TMA.Early detection of TMA and timely treatment can reduce mortality and can improve the survival rate.
基金This work was supported by grants from the National Natural Science Foundation of China (No. 81571948) and Beijing Natural Science Foundation (No. 7172075).
文摘Staphylococcus aureus is associated with a variety of invasive infection; typically, these infections occur as sepsis, osteomyelitis, endocarditis, and arthritis. China has a relatively high incidence of invasive S. aureus disease in children.[1] The present study aimed to provide the demographics, clones, and the antimicrobial susceptibility of S. aureus that cause invasive infection in Chinese children.