Objective To investigate the clinical features and prognostic factors in children with idi-opathic pulmonary arterial hypertension(IPAH). Methods The data of children with IPAH diagnosed in Beijing Children′s Hospital from January 2006 to December 2015 were collected. The cases were divided into survival group and death group according to the prognosis. The risk factors influencing prognosis in children with IPAH were identified by Logistic regression analysis. Results Total 26 patients were enrolled in this study,and 17 of them survived and 9 of them were dead. IPAH was found to be more common in school-age children. The average age of the patients was(6. 6 ± 3. 6)years and the male to female ratio was 1. 17∶1. All of the children had the symptoms with decreased activity tolerance and fatigue. The incidence of syncope and dyspnea and edema of lower extremity were 34. 6%,23. 1%. Accentuated pulmonic second sound(P2) was detected in 92. 3% of patients during physical examination, which was also the most common sign. About 50. 0% patients were functional class Ⅲ-Ⅳ. Echocardiography showed that all childrens′ mean pulmonary artery systolic pressure(PASP) was(79. 0 ± 20. 3) mmHg(1 mmHg=0. 133 kPa) and 16 of them with right ventricular dilatation. Electrocardiogram with ST-T changes and right ventricular high voltage were common. The average plasma level of brain natriuretic peptide(BNP) in all cases was(870. 4 ± 720. 9) pg/ml. The single factor analysis illustrated that syncope(OR=26. 25,95%CI 3. 04 -226. 60,P=0. 003),heart func-tional class Ⅲ-Ⅳ(OR=19. 199,95%CI 1. 88 -196. 53, P=0. 0128), PASP≥70 mmHg(OR =9. 936, 95%CI 1. 81-∞,P=0. 005),BNP≥850 pg/ml(OR=59. 991,95%CI 4. 69-767. 62,P=0. 002) indica-ted the worse outcome. Conclusion IPAH in children is rare and the prognosis is poor. Syncope, cardiac function grade,PASP and the levels of BNP are the main important factors affecting the prognosis of children with IPAH. BNP has important clinical significance for the evaluation of IPAH and the prognosis of the disease.

Objective To evaluate the effect of different doses of Astragalus membranaceus on the levels of vascular endothelial growth factor ( VEGF) and basic fibroblast growth factor ( bFGF) in serum and lung tissues of rats with pulmonary embolism. Methods Seventy-six clean-grade healthy male Sprague-Dawley rats, aged 8-9 weeks, weighing 140-170 g, were assigned to control group ( group C, n=11) and experimental group ( group E, n=65) by a random number table method. The rats with pulmonary em-bolism in group E were further divided into 4 subgroups using a random number table method: pulmonary embolism group (group P), low-dose Astragalus membranaceus group (group H1), median-dose Astraga-lus membranaceus group ( group H2 ) and high-dose Astragalus membranaceus group ( group H3 ) . The model of pulmonary embolism was established by injecting autologous blood clots into the right jugular vein. At 1 h and 1, 2, 3, 4, 5 and 6 days after successful establishment of the model, Astragalus membrana-ceus 20, 40 and 60 g∕kg were injected intraperitoneally in H1-3 groups, respectively, while the equal vol-ume of normal saline was given instead in group P. The chest was opened after anesthesia on day 7, and blood samples were collected from cardiac chambers for determination of concentrations of serum VEGF and bFGF by enzyme-linked immunosorbent assay. The pulmonary specimens were obtained from the upper lobe of right lungs for determination of the expression of VEGF and bFGF mRNA ( using real-time polymerase chain reaction) . Results Compared with group C, the concentrations of serum VEGF and bFGF were sig-nificantly increased, and the expression of VEGF and bFGF mRNA in lung tissues was up-regulated in the other four groups (P<0. 05). Compared with group P, the serum bFGF concentration was significantly in-creased, and the expression of VEGF and bFGF mRNA in lung tissues was up-regulated in H1-3 groups ( P<0. 05) . Compared with group H1, the serum bFGF concentration was significantly increased, the ex-pression of VEGF mRNA and bFGF mRNA in lung tissues was up-regulated in H2 and H3 groups ( P<0. 05) . Compared with group H2, the expression of VEGF and bFGF mRNA in lung tissues was significant-ly up-regulated in group H3 ( P<0. 05 ) . Conclusion Astragalus membranaceus can up-regulate the ex-pression of VEGF and bFGF in lung tissues in a dose-dependent manner, thus improving pulmonary embol-ism in rats.

Objective:To summarize the clinical and genetic characteristics of catecholaminergic polymorphic ventricular tachycardia (CPVT) in children caused by CASQ2 gene variants. Methods:The clinical data of 8 children (4 males and females, respectively) with CPVT caused by CASQ2 gene variants admitted to Beijing Children′s Hospital, Capital Medical University from January 2017 to November 2018 were retrospectively analyzed.The targeted next generation sequencing was employed to identify CASQ2 variants and Sanger sequencing was conducted to conform the candidate variants and determine the parental origin. Results:As for 8 children in this study, the average age of onset was 6.4 years, the mean age at diagnosis was 9.4 years, and the average interval from onset to diagnosis was 3 years.Only 2 cases had clearly diagnosis at onset, other 6 cases had a delay to diagnosis and 3 cases of them were diagnosed at other hospitals as having epilepsy and did not respond to anti-epileptic therapy.During physical activity and/or emotional stress, 8 cases presented with recurrent syncope and were able to regain consciousness after a few minutes.They had no a history of sudden cardiac death or family history.There was no abnormality on resting electrocardiogram during the paroxysmal interval in 6 cases and mild sinus bradycardia in 2 cases.Typical bidirectional ventricular tachycardia (VT) and/or polymorphic VT were detected in 8/8 cases and 5/5 cases, respectively, based on Holter electrocardiography and cardiac stress test.The CASQ2 gene variant was found in all children, with 6 cases carrying compound heterozygous variants and 2 cases carrying homozygous variants.A total of 9 different CASQ2 variants were detected in 8 cases, of which 5 had not been previously reported.According to the family-line verification, all of them had a familial variant, with no novel variants.All 8 cases were treated orally with β-blockers, with asymptomatically recurrent episodes, with a mean follow-up of 1.5 years, during which implantable cardioverter defibrillation was performed in 1 case owing to severe sinus bradycardia.There was no death case among them. Conclusions:CPVT with CASQ2 variants is characterized by early onset before preschool age, recurrent syncope after exercise or emotional stress and bidirectional/polymorphic VT.Early diagnosis of CPVT remains challenging due to delayed diagnosis or misdiagnosis.Treatment with β-blockers can achieve favorable effectiveness and safety.Five novel variants in this study would further expand the database of CASQ2 genes.

OBJECTIVE：To learn from the experience of foreign listed chimeric antigen receptor T lymphocyte （CAR-T） products in signing risk sharing agreements in medical insurance access ，so as to provide references for relevant decisions of medical insurance departments in China. METHODS ：Taking 9 risk sharing agreements of CAR-T products marketed in the United Kingdom，France，Italy and Germany as samples ，the international experience of medical insurance payment of CAR-T products were analyzed from six dimensions ，such as agreement types ，monitoring indicators ，data collection metho ds，agreement periods ， payment conditions and payment methods. Some suggestions were put forward for the medical insurance access of these products in China. RESULTS & CONCLUSIONS ：Four sample countries generally signed risk sharing agreements of medical insurance access （financial agreement and performance-based agreement ）with pharmaceutical enterprises ；the indicators such as progressive disease and progression-free survival were collected by using data collection system or clinical research data ，so as to monitor the efficacy and safety of CAR-T products. The agreement periods and payment conditions were determined according to different agreement types；“medical insurance advance payment ”or“pharmaceutical enterprise advance payment ”combined with “staged payments ” were adopted for risk control. Solving the risk of medical insurance funds caused by “efficacy uncertainty ”is the core issue of CAR-T product access. The induction of risk sharing agreements may be the way to solve this problem ，and the scientific design of the various elements of risk sharing agreements is a prerequisite to ensure that the agreement is operational.