Polycentric analysis of current situation of diagnosis of haemophilia in Chinese children / 中华实用儿科临床杂志

Objective To analyze the diagnostic status of haemophilia in Chinese children in recent years,and to provide information for increasing the life quality of children with haemophilia in China.Methods The pediatric haemophilia cases registration data were collected and analyzed by using questionnaire,from January 1,2008 to March 30,2014 in 13 haemophilia treatment centers of haemophilia treatment center collaboration network of China pediatric group.These centers were from 12 provinces / municipalities.Results A total of 554 cases were collected;median age was 7.0 years old(0.1 to 17.9 years old);among them,481 cases(86.8％) were hemophilia A,and 73 cases were hemophilia B (13.2％);55 mild cases (9.9％),290 moderate cases (52.4％),and 209 severe cases (37.7％);162 cases(29.2％) had family history,the other 392 cases(70.8％) had no family history.The diagnosis was made at a median age of 12.0 month-old(0 to 180.0 months);the diagnosis time was 0.5 months(0 to 144.0 months) after the first bleeding;diagnosis timing with short interval was in 356 cases(64.3％),long interval was in 198 cases(35.7％).The diagnostic timing was not correlated with disease severity (P =0.812) or the family history (P =0.243);but correlated with the severity of first bleeding(P =0.027) and per capita gross domestic product (P ＜ 0.01) in patients' residence.From 1996 to 2013,the annual number of newly diagnosed cases was increasing year by year,with a higher proportion of short interval of diagnose timing.Conclusions With development of hemophilia work in China,the number of diagnosis of haemophilia children is increasing year by year.Moderate and severe hemorrhage are both taken seriously and diagnosed timely.But the diagnosis is delayed in some children.Chinese haemophilia work still need to be strengthen and the propaganda and diagnostic technology are to be popularized.

Progress on novel agents treating childhood primary immune thrombocytopenia / 中华实用儿科临床杂志

The mechanisms of childhood primary immune thrombocytopenia(ITP) are the destruction of platelet by platelet antibody and cytotoxic T cells,which also influence the function of megakaryocyte and cause the reduction of platelet.Novel agents targeted to the immune abnormalities of ITP includes 4 types:(1) CD40-CD154 pathway blocker,such as humanized CD154 antibody Toralizumab,which is monoclonal antibody and modify Fc,hopefully to be used in treatment of ITP.(2) The drugs which block the binding between Fc receptor and anti-platelet antibody such as Rozrolimupab,which is the combination of 25 kinds of anti-RHD and presents 62％ efficacy but has severe hemolysis as adverse effects which restricts the application on ITP.Soluble FcγRⅡb(SM101) is also effective on ITP and it conquer the shortcome of activating of B cells to induce autoimmune diseases.(3) The drugs which block the key molecular Syk of activation the platelet phagocytosis by microphage such as R788,with 44％ effectiveness.(4)The drugs which stimulate thrombopoiesis such as Avatrombopag et al.

Diagnosis and treatment of rare hereditary bleeding disorders / 中国小儿急救医学

In this feature article,we focused on rare hereditary bleeding disorders with the exception of hemophilia,hereditary factor Ⅺ deficiency and von Willebrand's disease.In order to raise the awareness and promote the diagnosis rate of the rare hereditary bleeding disorders,we discussed the classification according the bleeding mechanism,clinical features and therapeutic advance.