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Beijing Children's Hospital affiliated to the Capital Medical University set up a three-tiered system for hemophilia and explore solutions for local and remote referral modes.Through the establishment of electronic database,improving drug management,assisting medical insurance reimbursement and other auxiliary measures,it smooths the two-way referral path of children with hemophilia.It can rationally allocate medical resources,promote the development of local professional medical ability,make rational use of rare disease drugs,improve the efficiency of medical insurance funds,strengthen the service capability of children with rare diseases through the joint reform of three medical systems,and ensure the treatment of difficult and severe child patients,improve the accessibility of high-quality diagnosis and treatment,effectively reduce the financial burden of patients.
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Being a hereditary bleeding disorder, hemophilia is characterized by spontaneous bleeding, especially joint bleeding. The treatment outcome is a comprehensive evaluation system of the following three aspects: bleeding, musculoskeletal structure (imaging), and function-activity participation. Multidisciplinary testing and corresponding scales are needed in the assessment. Among them, the quality of life assessment of hemophilia patients is particularly important, through general questionnaires and hemophilia-specific questionnaires. Canadian hemophilia outcomes-Kids' life assessment tool(CHO-KLAT), a special quality of life assessment tool for children with hemophilia, is the most widely used. This paper briefly describes the development and application of comprehensive evaluation system of the children with the disease.
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Hereditary thrombotic thrombocytopenic purpura (hTTP) in children is a rare but severe and fatal thrombotic microangiopathy. The etiology of the disease is the persistent severe deficiency of the enzyme ADAMTS13 gene mutation, resulting in microangiopathic hemolytic anemia, thrombocytopenia, neuropsychiatric symptoms, fever, and renal involvement. Different from adults, children with hTTP present earlier onset of the disease and are more likely to develop long-term complications in brain and kidney, so that the need for preventive replacement therapy is more urgent. This article reviews the research progress of hTTP in children.
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A 6-year-old girl presented with recurrent skin rash at the initial stage, recent joint pain, and neutrophilia was found during a routine blood test. After a multidisciplinary case discussion, she was diagnosed with chronic neutrophil leukemia, and the symptoms were relieved after hydroxyurea and luxolitinib treatment. She received the allogeneic hematopoietic stem cell transplantation subsequently. At present, she is in stable condition and under follow-up. Chronic neutrophil leukemia is a rare disease, which rarely occurs in children. It is more difficult to diagnose in patients with skin rash as the first manifestation. The diagnosis and treatment of this case reflects the important role of multidisciplinary cooperation in the diagnosis and treatment of difficult and rare diseases.
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Immune thrombocytopenia is a common bleeding disease characterized by isolated thrombocytopenia.Some patients last for more than 12 months and suffer from chronic immune thrombocytopenia(CITP). The pathogenesis of CITP is complex, and the traditional first-line has little improvement.In recent years, researches on second-line treatments(thrombopoietin and its receptor agonists, rituximab and splenectomy), immunosuppressive agents, all-trans retinoic acid, atorvastatin, and hematopoietic stem cell transplantation have provided new ideas for the treatment of CITP.This review summarizes the recent progress in the treatments of CITP and will be helpful for individualized treatment.
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Hereditary thrombocytopenia(HT)is a hemorrhagic disease characterized by thrombocytopenia caused by genetic variation.HT can be manifested as simple thrombocytopenia or combined syndrome, and its clinical manifestations are complex.It often occurs in children.The unique clinical characteristics of HT are platelet dysfunction, unstable course of the disease and susceptibility to other diseases.Due to different pathogenic genes, the treatment and prognosis of HT are diverse.The evaluation of hemorrhage in the clinical management of HT children is very important.In addition, platelet transfusion, thrombopoietin receptor agonists, hematopoietic stem cell transplantation and gene therapy also supply new ideas for HT treatment.This review summarized the current research progress on HT, in order to help clinicians comprehensively identify HT and take active and effective treatment programs.
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Objective:To analyze the clinical manifestations, genetic variations, diagnosis and treatment of children with inherited thrombophilia(IT).Methods:Retrospective study.Children with IT treated in Department of Respiratory Diseases 1 of Beijing Children′s Hospital, Capital Medical University from October 2016 to August 2021 were included in the study and followed up.Results:A total of 5 children met the inclusion criteria, with 3 boys and 2 girls; the age of diagnosis ranged from 7 years to 13 years and 6 months.There were 2 cases of protein C deficiency, 1 case of congenital protein S deficiency, 1 case of activated protein C resistance and 1 case of congenital afibrinogenemia.All 5 cases had pulmonary embolism, 2 cases had deep venous thrombosis of lower limbs, and 1 case had cardiac thrombosis and arterial embolism.The level of protein C was significantly decreased in 1 case, and the level of protein S in 1 case was significantly decreased in the laboratory test of thrombophilia; 2 cases were positive for antiphospholipid antibodies in the acute phase, but negative after 3-6 months of re-examination.Genetic analysis showed 2 cases of PROC gene mutation, 1 case of PROSI gene mutation, 1 case of F5 gene mutation, and 1 case of FGA gene mutation.All children were treated with anticoagulation drugs for long-term, including 4 patients with Warfarin and 1 patient with Rivaroxaban.The follow-up time ranged from 3 months to 5 years.During the follow-up, 1 patient experienced thrombosis recurrence due to infection incentives 1 month after discontinuing anticoagulant drugs on his own. Conclusions:The clinical manifestations of children with IT are the same as those of adults, mainly including venous thromboembolism(VTE); there are limitations in laboratory detection of thrombophilia, and gene analysis is of great significance.Children diagnosed with IT need long-term anticoagulant therapy to reduce the recurrence of VTE.
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The clinical and laboratory data of a patient with chronic refractory immune thrombocytopenia (ITP) who had a significant increase in the proportion of inhibitory T cells in the hematological oncology center of Beijing Children′s Hospital Affiliated to Capital Medical University in February 2018 and regularly followed up in the outpatient department received a retrospective analysis.This 8-year-old patient′s clinical features were 6 years of skin and mucous membrane bleeding spots and petechiae recurring, with occasionally nasal epistaxis.Physical examination: cardiopulmonary abdomen and nervous system examinations are normal, and no superficial lymphadenopathy is touched.Blood routine indicated that the platelets were 2.00×10 9/L, and white blood cell count and hemoglobin level were normal.Bone marrow suggested that hyperplasia was significantly active, with more than 300 megakaryocytes.The patient was diagnosed with ITP, and he was treated with first-line treatment with gamma globulin and oral Corticosteroids.The first-line treatment with high-dose Dexamethasone therapy was repeated, and the second-line treatment was low-dose Rituximab combined with high-dose Dexamethasone.Evaluations had been conducted with every relapsed course, and Cyclosporine was administered orally on the basis of the highly suppressive T cells.After half a month, the child achieved partial remission and continued for 3 months, and then reached a complete remission of 6 months (till this paper). It is suggested that, with ITP as an immunological disease with high heterogeneity, the immune abnormality index is expected to become a breakthrough in the development of precise treatment.
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Objective:To analyze the clinical characteristics and diagnosis and treatment of children with Kasabach-Merritt phenomenon (KMP).Methods:A retrospective analysis was conducted on the clinical data and follow-up data of 8 patients diagnosed KMP in Beijing Children′s Hospital, Capital Medical University from January 2016 to January 2019.The clinical data included laboratory examination, diagnosis, treatment and prognosis.Results:Among the 8 children with KMP, 6 cases were male and 2 cases were female.The median onset age was 4 (0-17) months, 2 cases of neonatal onset.The median onset to the diagnosis time was 59 (34-140) days; 6 cases with bone destruction; 6 cases had misdiagnosis and mistreatment history, they were misdiagnosed as idiopathic thrombocytopenic purpura, Evans syndrome, abnormal bone and joint development; 4 cases were Kaposiform hemangioendothelioma; 8 cases were used alone or combined with the application of hormones, Sirolimus, and Vincristine, 7 patients underwent interventional therapy.All patients survived with a median follow-up period of 487 (112-1 033) days.Median time of platelet count returned to normal was 24.5 (7-60) days, and median time of fibrinogen returned to normal was 20 (7-30) days.Median time of D-dimer dropped to a normal was 105 (40-240) days.Conclusions:Children with concurrent platelet count and coagulation abnormalities should be considered with KMP.Doctors need to identify the potential visceral vascular lesions.Early diagnosis and treatment are important, which can improve the clinical prognosis of patients.
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Objective:To understand the relationship between joint bleeding and joint disease in hemophilia children, and to provide a theoretical basis for clinical treatment and prognosis.Methods:The patients with severe hemophilia A between 1 and 7 years old and with relevant nodal bleeding records were selected.All the patients admitted in Beijing Children′s Hospital, Capital Medical University, and Chengdu New Century Women′s and Children′s Hospital since June 2016 to January 2017.All the joint bleeding of each child was taken as the study joint, and the joint bleeding was collected during the last 3 months.The joints were assessed by using ultrasound, X-ray, magnetic resonance imaging(MRI) and Hemophilia Joint Health Score (HJHS) scoring systems.The correlation analysis was conducted between the joint bleeding, ultrasound, X-ray, MRI and HJHS scores.The correlation analysis was conducted for baseline ultrasound, X-ray, MRI and HJHS scores.Results:(1) There were 18 patients enrolled.The mean age was (5.6±1.8) years old.There were 30 joints bleeding in the observation period in total, with the annul median joint bleeding times of 4 (4-16 times), and the annul median target joint bleeding times of 8 (4-16 times). (2) Joint bleeding times of was correlated with ultrasound and X-ray evaluation ( r=0.390, P=0.033; r=0.517, P=0.008), and not correlated with HJHS or MRI(all P>0.05). (3) There was significantly positive correlation among ultrasound, X-ray, HJHS and MRI [ r=0.815(ultrasound vs.X-ray), r=0.510(ultrasound vs.HJHS), r=0.812(ultrasound vs.MRI), r=0.666(X-ray vs.HJHS), r=0.911(X-ray vs.MRI), r=0.781(HJHS vs.MRI), all P<0.01]. (4) There was no correlation between times and assessment for joints whose ultrasound and /or MRI in joints with abnormal ultrasound and /or MRI evaluation( P>0.05). Conclusions:The results of joint bleeding and joint evaluation are inconsistent.Joint bleeding can not truly reflect the situation of joint diseases.The assessment of hemophilia should include comprehensive evaluation of joint structure, function, activity ability and other aspects to guide the treatment of haemophi-lia children.
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Objective:To investigate the related factors of the serum sickness morbidity in the treatment of children with acquired aplastic anemia (AA) by rabbit antithymosinglobulin (ATG), summarize the clinical characte-ristics of serum sickness and evaluate the influence of serum sickness on the prognosis of AA.Methods:The data of patients diagnosed as AA after treated with immunosuppressive therapy (IST) in Beijing Children′s Hospital, Capital Medical University, from March 2016 to December 2018 were collected, and the onset time, clinical manifestations, treatment, and prognosis of serum sickness were analyzed.Results:A total of 48 cases were enrolled, with the median age of 5 years and 5 months (ranging from 2 years and 1 month to 15 years and 6 months), and the proportion of male to female was 1.4∶1.0, 75.0% of the patients(36/48 cases) developed serum sickness.The median onset time was the 11 th day and 72.2% of the patients (26/48 cases) occurred from the 7 th to the 14 th day during IST.The 3 main clinical manifestations included arthralgia (63.9%, 23 cases), fever (52.7%, 19 cases) and rash (52.7%, 19 cases). There was no significant difference in peripheral blood leukocytes, neutrophils and lymphocytes between the patients with serum sickness and patients without serum sickness before IST and during serum sickness (all P>0.05). The incidence of serum sickness in children who received continuous glucocorticoid prophylaxis after IST (2/12 cases, 16.6%) was lower than that of those who did not (34/36 cases, 94.4%), and the difference was significant ( χ2=29.037, P<0.001). The symptoms of serum sickness improved after glucocorticoid therapy [Methylprednisolone 2-4 mg/(kg·d)]. Among 37 children who were followed up for 6 months or more after IST treatment, 25 patients had serum sickness and 12 patients did not have serum sickness.Nineteen patients with serum sickness and 10 patients without serum sickness were cured or markedly improved; 6 patients with serum sickness and 2 patients without serum sickness were not cured.No significant difference in the prognosis between 2 groups was observed ( P>0.05). Conclusion:Children with AA are prone to develop serum sickness after IST treatment.The peak period of incidence of serum sickness is the second week during IST, and the main clinical manifestations of serum sickness include arthralgia, fever, and rash.There is no correlation between the incidence of serum sickness and the blood routine test before IST and during serum sickness.The incidence of serum sickness can be reduced by giving glucocorticoid prophylaxis, and glucocorticoid is still effective after the onset of the serum sickness.There is no correlation between the morbidity of serum sickness and the prognosis of AA treated with IST.
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Objective@#To discuss the clinical characteristics and management approaches to hepatitis associated aplastic anemia (HAAA) presenting as hemophagocytic lymphohistiocytosis (HLH) at onset.@*Methods@#The clinical data and laboratory results of hospitalized 5 HAAA patients presenting as HLH at onset in Beijing Children′s Hospital from January 2017 to May 2019 were analyzed retrospectively.@*Results@#Among 5 cases, there were 4 males and 1 female. The age of onset was 6.0 (2.7-12.7) years. All patients presented with high fever, hepatomegaly, hepatic dysfunction (aspartate aminotransferase 1 716 (1 409-2 570) U/L, alanine aminotransferase 1 699 (937-2 540) U/L) at onset. After admission, the laboratory results showed pancytopenia (white blood cell 1.2 (0.6-6.7) ×109/L, haemoglobin 94 (65-111) g/L, blood platelet 29 (10-41) ×109/L), decreased fibrinogen (1.3 (1.1-2.5) g/L), significantly elevated triglyceride (4.0 (2.8-5.1) mmol/L), ferritin (1 766 (399-5 253) μg/L) and soluble CD25 (27 457 (9 625-44 000) ng/L). Hemophagocytosis was found in the bone marrow smears of all 5 patients. The diagnosis of acute hepatitis and HLH was confirmed. During the treatment of HLH, the blood cells remain below normal level and the further biopsy of bone marrow (iliac bone) indicated low myeloproliferation. After exclusion of congenital bone marrow failure syndromes and other pancytopenic diseases, HAAA was confirmed. After the diagnosis of HAAA, 1 patient received antithymocyte globulin (ATG) and cyclosporin treatment in our hospital, 1 patient received allogeneic stem cell transplantation (HSCT) in other hospital, 2 patients received ATG in other hospitals. Only 1 patient died of severe infection.@*Conclusions@#HAAA can present as HLH at onset. It is mainly manifested by high fever, acute severe hepatitis, pancytopenia, elevated ferritin and hemophagocytosis in the bone marrow. The diagnosis of HAAA should be considered whenever cytopenia could not completely corrected while apparent improvement of HLH and hepatitis related complications were improved after immunosuppressive therapy. ATG or HSCT treatment should be performed as soon as the diagnosis of severe or transfusion dependent aplastic anemia is confirmed.
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To analyze respectively a case,presented with recurrent petechial and epistaxis after a 2 years history of diabetes mellitus (DM),who was hospitalized in Beijing Children's Hospital Affiliated to Capital Medical University.The clinical manifestation,examination,diagnosis and treatment were recorded.The patient was diagnosed with immune thrombocytopenia (ITP) and DM at the first admission.The initial therapy with gamma globulin didn't show ideal effect.The pediatric specialists from the department of ENT,Hematology/Oncology,Endocrinology,Pharmacy and Immunodeficiency Clinic were invited to discuss the case.The final diagnosis of autoimmune polyglandular syndrome (APS) was made and supplementary steroid treatment was started.But the response of the steroid therapy was poor.Once again with the multidisciplinary consultation,the patient received several schemes of Rituximab under the informed consent.This treatment reached a stable condition for almost 7 years.APS should be considered when DM patient showed the manifestation of other immune organ damages.Rituximab immunosuppressive therapy should be tried when the response to first-line treatment was poor.
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Objective To compare the efficacy and safety of short course and high-dose Dexamethasone (HDD) and conventional Prednisone as first-line strategy for children newly diagnosed as primary immune thrombocytopenia (ITP).Methods This study analyzed pre-experimental data of a single center prospective randomized controlled clinical trial.Newly diagnosed but untreated ITP patients enrolled at the Department of Blood and Cancer Center,Beijing Children's Hospital,Capital Medical University from November 2016 to May 2017 were randomized into HDD group[Dexamethasone 0.6 mg/(kg · d),intravenous injection for 4 days] and Prednisone group [Prednisone 2 mg/(kg · d) for 14-28 days and then tapered within 1-2 months,the course of treatment less than 3 months].Initial response,sustained response and adverse effects after therapy were observed in 2 groups.Results Sixty-six children with ITP were included in the study:32 patients were in the HDD group and 34 patients were in the Prednisone group.Two groups were matched in the baseline characteristics including gender,age,platelet counts and disease course before therapy and bleeding assessment (all P > 0.05).The initial response (the response of HDD group within 10 days of treatment and Prednisone group within 28 days of treatment):overall initial response had no statistical difference between the HDD group and the Prednisone group[90.6% (29/32 cases) vs.100.0% (34/34 cases),x2 =1.528,P > 0.05].HDD group had a lower incidence of complete response compared with that in the Prednisone group [54.4% (19/32 cases) vs.94.1% (32/34 cases),x2 =11.330,P =0.001];median time of response in two groups showed no statistically difference (2 d vs.1 d,Z =-0.149,P > 0.05).There was no significant difference in the recovery of skin and mucosal bleeding after treatment between the Dexamethasone group and the Prednisone group (Z =-1.413,-1.031,all P > 0.05).The sustained response (the response lasted for up to 6 months and above):overall and complete sustained response had no statistically difference between the HDD group and the Prednisone group [92.9% (26/ 28 cases) vs.85.3% (29/34 cases),P =0.594;78.9% (15/19 cases) vs.81.3% (26/32 cases),P=1.000].Log-rank test showed no significant difference in the duration of response between 2 groups (P =0.341).The side effects in the Prednisone group included weight gain or Cushing sign (94.1%) and mental and emotional changes (23.5%);in the HDD group 15.6% of children had infection,without other glucocorticoid-related side effects.There was no significant difference in the incidence of infection between two groups[15.6% (5/32 cases) vs.26.5 % (9/34 cases),P =0.281].All of the above infections were of respiratory tract infections and mild gastrointestinal infections.Conclusions Efficacy of the HDD group in the initial and sustained responses is similar,but side effects were apparently lower compared with that in the Prednisone group.However,a large multicenter randomized controlled clinical study is necessary to confirm this result.
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Objective To analyze the practical value of D-dimer in diseases condition judgment and prognosis evaluation of childhood Mycoplasma pneumoniae pneumonia (MPP).Methods Retrospective analysis was performed on clinical data of 606 MPP at Department of Respiratory,Beijing Children's Hospital,Capital Medical University from January 2009 to July 2017,and the subjects were divided a severe group (298 cases) and a moderate group (308 cases) according to severity.By comparing clinical characteristics,laboratory tests and imaging findings,multivariate Logistic regression analysis for significant single factors was accomplished,which was to find out the independent factors affecting the severity of childhood MPP in acute phase.Receiver operating characteristic (ROC) curves were drawn in the area under the curve (AUC) and the diagnosis threshold value was calculated,which could be used to judge the predicators affecting the severity judgment of childhood MPP in acute phase.And the prognosis was judged according to the convalescent fiberoptic bronchoscopic indicators in recovery phase.Results The levels of white blood cells (WBC) [(10.25 ± 3.76) × 109/L],neutrophil (Neu) [(7.31 ± 3.76) × 109/L],platelet (PLT) [(334.66 ± 143.80) × 109/L],C-reactive protein (CRP) [(69.00 ± 80.50) mg/L],erythrocyte sedimentation (ESR) [(39.38 ± 26.29) mm/1 h],lactate dehydrogenase (LDH) [(436.61 ± 248.96) IU/L],fibrinogen (Fib) [(4.61 ± 1.36) g/L] and D-dimer [(2.09 ± 1.66) mg/L] in the severe group were higher than those in the moderate group [(7.55 ±3.14) × 109/L,(4.77 ±2.54) × 109/L,(291.60 ± 109.19) × 109/L,(23.40 ±42.50) mg/L,(30.25 ± 16.18) mm/1 h,(318.05 ± 116.97) IU/L,(4.18 ±0.88) g,(0.58 ±0.72) mg/L],and the differences were statistically significant (all P < 0.01).The levels of Neu,PLT,CRP,LDH and D-dimer were independent and relevant factors for the severity of acute MPP.The area under each ROC curve was Neu 0.719,PLT 0.592,LDH 0.675,CRP 0.749,D-dimer 0.848,and each diagnostic threshold was 6.5 × 109/L,265.5 × 109/L,417.5 IU/L,28.9 mg/L,0.73 mg/L,respectively.Obviously,D-dimer had the highest sensitivity and specificity for the severe MPP.There was a significant difference in D-dimer level between the endobronchial inflammation group and the subbronchial stenosis,poor ventilation and occlusion group of fiberoptic bronchoscopy [(1.11 ± 0.26) mg/L vs.(2.14 ± 1.84) mg/L,t =-5.870,P < 0.05].Conclusion D-dimer levels can be used as one of the most sensitive indicator for determining the severity and prognosis of MPP.
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Objective To know the detection rate of hereditary thrombocytopenia (HT) in children with chronic thrombocytopenia and its clinical and laboratory characteristics for an early clinical identification and diagnosis of HT in future.Methods Data of the children with thrombocytopenia,who had been treated in Beijing Children's Hospital from April 2016 to May 2018 and whose present history lasted for more than 1 year and had poor response to immunotherapy were retrospectively collected.HT was screened in these patients by adopting next generation sequencing (NGS).Finally,clinical and laboratory characteristics of these children with HT were summarized and analyzed.Results A total of 161 children with chronic thrombocytopenia were included.Forty-three cases (26.7%) were found to have gene mutations.The genetic rules of the mutant gene,the family verification and the clinical manifestations of the proband and some related laboratory tests were analyzed and 24 cases (14.9%) can be diagnosed as HT.Among the HT patients,the proportion of males and females was 15 ∶ 9,and the median onset of age was 0.58 years,which was significantly lower than that of non-HT cases (the median onset of age was 4.36 years),and the difference was statistically significant (P < 0.001);the proportion of mucosal hemorrhage and visceral hemorrhage (31.8% and 13.7%) of HT was significantly higher than non-HT cases (15.3% and 0.6%),and the difference was statistically significant (P < 0.001).Fifty percent of (12/24 cases) cases of HT had positive family history;according to the average platelet volume and platelet morphology in peripheral blood smear,HT could be divided into small platelet HT,positive platelet HT and large platelet HT.Some cases had well response to immunotherapy but seemed easy to relapse during the withdrawal period,while the others responded poorly to therapy.Different clinical manifestations of HT suggest different pathogenesis,which can be divided into the related types of megakaryocyte differentiation defect,megakaryocyte maturation defect,platelet release defect and platelet survival time shortening.Conclusions The pathogenesis and clinical phenotype of HT was different.Some of them were effective for immunotherapy,which were easily confused with immune thrombocy-topenla(ITP).It is clinically necessary to perform NGS in children with thrombocytopenia at early onset,abnormal platelet morphology,prolonged disease course or severe mucosal/visceral hemorrhage,in order to recognize HT timely to avoid delay in diagnosis and poor prognosis.
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Objective@#To know the detection rate of hereditary thrombocytopenia (HT) in children with chronic thrombocytopenia and its clinical and laboratory characteristics for an early clinical identification and diagnosis of HT in future.@*Methods@#Data of the children with thrombocytopenia, who had been treated in Beijing Children′s Hospital from April 2016 to May 2018 and whose present history lasted for more than 1 year and had poor response to immunotherapy were retrospectively collected.HT was screened in these patients by adopting next generation sequencing (NGS). Finally, clinical and laboratory characteristics of these children with HT were summarized and analyzed.@*Results@#A total of 161 children with chronic thrombocytopenia were included.Forty-three cases (26.7%) were found to have gene mutations.The genetic rules of the mutant gene, the family verification and the clinical manifestations of the proband and some related laboratory tests were analyzed and 24 cases (14.9%) can be diagnosed as HT.Among the HT patients, the proportion of males and females was 159, and the median onset of age was 0.58 years, which was significantly lower than that of non-HT cases (the median onset of age was 4.36 years), and the difference was statistically significant (P<0.001); the proportion of mucosal hemorrhage and visceral hemorrhage (31.8% and 13.7%) of HT was significantly higher than non-HT cases (15.3% and 0.6%), and the difference was statistically significant (P<0.001). Fifty percent of (12/24 cases) cases of HT had positive family history; according to the ave-rage platelet volume and platelet morphology in peripheral blood smear, HT could be divided into small platelet HT, po-sitive platelet HT and large platelet HT.Some cases had well response to immunotherapy but seemed easy to relapse du-ring the withdrawal period, while the others responded poorly to therapy.Different clinical manifestations of HT suggest different pathogenesis, which can be divided into the related types of megakaryocyte differentiation defect, megakaryocyte maturation defect, platelet release defect and platelet survival time shortening.@*Conclusions@#The pathogenesis and cli-nical phenotype of HT was different.Some of them were effective for immunotherapy, which were easily confused with immune thrombocy-topenla(ITP). It is clinically necessary to perform NGS in children with thrombocytopenia at early onset, abnormal platelet morphology, prolonged disease course or severe mucosal/visceral hemorrhage, in order to recognize HT timely to avoid delay in diagnosis and poor prognosis.
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Objective@#To compare the efficacy and safety of short course and high-dose Dexamethasone (HDD) and conventional Prednisone as first-line strategy for children newly diagnosed as primary immune thrombocytopenia (ITP).@*Methods@#This study analyzed pre-experimental data of a single center prospective randomized controlled clinical trial.Newly diagnosed but untreated ITP patients enrolled at the Department of Blood and Cancer Center, Beijing Children's Hospital, Capital Medical University from November 2016 to May 2017 were randomized into HDD group[Dexamethasone 0.6 mg/(kg·d), intravenous injection for 4 days]and Prednisone group[Prednisone 2 mg/(kg·d) for 14-28 days and then tapered within 1-2 months, the course of treatment less than 3 months]. Initial response, sustained response and adverse effects after therapy were observed in 2 groups.@*Results@#Sixty-six children with ITP were included in the study: 32 patients were in the HDD group and 34 patients were in the Prednisone group.Two groups were matched in the baseline characteristics including gender, age, platelet counts and disease course before therapy and bleeding assessment (all P>0.05). The initial response (the response of HDD group within 10 days of treatment and Prednisone group within 28 days of treatment): overall initial response had no statistical difference between the HDD group and the Prednisone group[90.6%(29/32 cases) vs.100.0%(34/34 cases), χ2=1.528, P>0.05]. HDD group had a lower incidence of complete response compared with that in the Prednisone group[54.4%(19/32 cases) vs.94.1%(32/34 cases), χ2=11.330, P=0.001]; median time of response in two groups showed no statistically difference (2 d vs.1 d, Z=-0.149, P>0.05). There was no significant difference in the recovery of skin and mucosal bleeding after treatment between the Dexamethasone group and the Prednisone group (Z=-1.413, -1.031, all P>0.05). The sustained response (the response lasted for up to 6 months and above): overall and complete sustained response had no statistically difference between the HDD group and the Prednisone group [92.9%(26/28 cases) vs.85.3%(29/34 cases), P=0.594; 78.9% (15/19 cases) vs.81.3%(26/32 cases), P=1.000]. Log-rank test showed no significant difference in the duration of response between 2 groups (P=0.341). The side effects in the Prednisone group included weight gain or Cushing sign (94.1%) and mental and emotional changes (23.5%); in the HDD group 15.6% of children had infection, without other glucocorticoid-related side effects.There was no significant difference in the incidence of infection between two groups[15.6%(5/32 cases) vs.26.5%(9/34 cases), P=0.281]. All of the above infections were of respiratory tract infections and mild gastrointestinal infections.@*Conclusions@#Efficacy of the HDD group in the initial and sustained responses is similar, but side effects were apparently lower compared with that in the Prednisone group.However, a large multicenter randomized controlled clinical study is necessary to confirm this result.
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Objective@#To analyze the practical value of D-dimer in diseases condition judgment and prognosis evaluation of childhood Mycoplasma pneumoniae pneumonia (MPP).@*Methods@#Retrospective analysis was performed on clinical data of 606 MPP at Department of Respiratory, Beijing Children′s Hospital, Capital Medical University from January 2009 to July 2017, and the subjects were divided a severe group (298 cases) and a moderate group (308 cases) according to severity.By comparing clinical characteristics, laboratory tests and imaging findings, multivariate Logistic regression analysis for significant single factors was accomplished, which was to find out the independent factors affecting the severity of childhood MPP in acute phase.Receiver operating characteristic (ROC) curves were drawn in the area under the curve (AUC) and the diagnosis threshold value was calculated, which could be used to judge the predicators affecting the severity judgment of childhood MPP in acute phase.And the prognosis was judged according to the convalescent fiberoptic bronchoscopic indicators in recovery phase.@*Results@#The levels of white blood cells (WBC)[(10.25±3.76)×109/L], neutrophil(Neu)[(7.31±3.76)×109/L], platelet (PLT)[(334.66±143.80)×109/L], C-reactive protein(CRP)[(69.00±80.50) mg/L], erythrocyte sedimentation (ESR)[(39.38±26.29) mm/1h], lactate dehydrogenase (LDH)[(436.61±248.96) IU/L], fibrinogen(Fib)[(4.61±1.36) g/L] and D-dimer [(2.09±1.66) mg/L]in the severe group were higher than those in the moderate group[(7.55±3.14)×109/L, (4.77±2.54)×109/L, (291.60±109.19)×109/L, (23.40±42.50) mg/L, (30.25±16.18) mm/1 h, (318.05±116.97) IU/L, (4.18±0.88) g, (0.58±0.72) mg/L], and the differences were statistically significant (all P<0.01). The levels of Neu, PLT, CRP, LDH and D-dimer were independent and relevant factors for the severity of acute MPP.The area under each ROC curve was Neu 0.719, PLT 0.592, LDH 0.675, CRP 0.749, D-dimer 0.848, and each diagnostic threshold was 6.5× 109/L, 265.5×109/L, 417.5 IU/L, 28.9 mg/L, 0.73 mg/L, respectively.Obviously, D-dimer had the highest sensitivity and specificity for the severe MPP.There was a significant difference in D-dimer level between the endobronchial inflammation group and the subbronchial stenosis, poor ventilation and occlusion group of fiberoptic bronchoscopy [(1.11±0.26) mg/L vs.(2.14±1.84) mg/L, t=-5.870, P<0.05].@*Conclusion@#D-dimer levels can be used as one of the most sensitive indicator for determining the severity and prognosis of MPP.
ABSTRACT
Objective@#To evaluate the efficacy and safety of imatinib in the treatment of newly diagnosed chronic myeloid leukemia during chronic phase (CML-CP) in children and to analyze the difference of the efficacy and safety between imported original imatinib (Gleevec) and domestic generic imatinib (Xinwei).@*Methods@#Clinical data of 35 children with newly diagnosed CML-CP in Beijing Children′s Hospital from January 2014 to January 2018 were collected, among which 15 cases were treated with the imported original imatinib (original drug group) and 20 cases were treated with the domestic generic imatinib (generic drug group). The hematological, cytogenetic and molecular reactions and safety of the treatments were monitored at months 3, 6 and 12. Chi square test or rank sum test was used for the comparison between two groups.@*Results@#A total of 35 cases were treated for over 3 months, 31 cases were treated for over 6 months and 25 cases were treated for over 12 months. At 3 months, main cytogenetic response was obtained in 15 (100%) cases in the original drug group and 16 (80%) cases in the generic drug group respectively (χ2=3.387, P=0.119). At 6 months, complete cytogenetic response was obtained in 12 (80%) cases in the original drug group and 10 (63%) cases in the generic drug group (χ2=1.435, P=0.390). At 12 months, BCR-ABLIS ≤ 0.1% was obtained in 11 (92%) cases in the original drug group and 10 (77%) cases in the generic drug group (χ2=1.009, P=0.593). There was no significant difference at all stages (all P>0.05). Hematologic toxicity occurred in 7(20%) cases. The non-hematologic adverse reactions include nausea in 8 (23%) cases, pain in 8 (23%) cases, edema in 6 (17%) cases, emesis in 2 (6%) cases, fever in 2 (6%) cases, weakness in 1 (3%) case, rash in 1 (3%) case. The adverse reactions were easy to control and no drug toxicity related deaths occurred. There was no significant difference in the adverse reactions between original drug group and generic drug group (P>0.05).@*Conclusions@#Imatinib had a good efficacy and safety in the early treatment of newly diagnosed CML-CP in children. The efficacy and safety of generic imatinib is similar to that of imported imatinib.