Objective To investigate the physical growth status of pediatric patients with transfusion-dependent thalassemia(TDT)and analyze the effects of treatmentrelated and socioeconomic factors on physical growth.Methods Bas...Objective To investigate the physical growth status of pediatric patients with transfusion-dependent thalassemia(TDT)and analyze the effects of treatmentrelated and socioeconomic factors on physical growth.Methods Based on the specialized thalassemia database from gene therapy clinical research at the Institute of Hematology&Hospital of Blood Diseases,Chinese Academy of Medical Sciences&Peking Union Medical College,we collected data on height and weight development,familyy economic status,and medical records of 338 pediatric patients with TDT from October 2023 to May 2024.展开更多
In a recent article in The Lancet,an international team led by JL Kwiatkowski,F Locatelli and AA Thompson presented their successful multicenter phase 3 clinical trial(HGB-212,NCT03207009)that used gene therapy to tre...In a recent article in The Lancet,an international team led by JL Kwiatkowski,F Locatelli and AA Thompson presented their successful multicenter phase 3 clinical trial(HGB-212,NCT03207009)that used gene therapy to treat severe genetic forms of transfusion-dependentβ-thalassemia.展开更多
Objective： The aims of this study were to assess the prognostic significance of WHO-based Prognostic Scoring System （WPSS） in myelodysplastic syndrome （MDS） from a single center institute and to compare WPSS with...Objective： The aims of this study were to assess the prognostic significance of WHO-based Prognostic Scoring System （WPSS） in myelodysplastic syndrome （MDS） from a single center institute and to compare WPSS with the international prognostic scoring system （IPSS）. Methods： A total of 100 cases with de novo MDS were reviewed and their karyotypes were detected. All of them were followed up and classified according to IPSS and WPSS risk groups. SPSS 13.0 software was applied to deal with all the data. The statistical methods included Kaplan - Meier, Log-rank test and cox regression. Results： Multivariate cox regression analysis indicated that WHO Classification （P=0.0190）, karyotype abnormalities categorized according to IPSS （P=0.0159） and red blood cell （RBC） transfusion （P=0.0009） were the three most important independent factors for predicting overall survival （OS） of MDS. WPSS and IPSS both had great capacity in predicting the OS of MDS at the time of diagnosis （P〈0.0001）. In time-dependent analysis, WPSS can predict the OS accurately in the following three years after diagnosis （P〈0.0001）, while IPSS failed to predict the OS 24 months after diagnosis （P=0.1094）. Conclusion： Our single center results proved that WPSS is a dynamic prognostic system which can predict the OS of MDS patients at any time during the course of their disease. This time-dependent prognostic scoring system may replace the IPSS in the near future.展开更多
文摘Objective To investigate the physical growth status of pediatric patients with transfusion-dependent thalassemia(TDT)and analyze the effects of treatmentrelated and socioeconomic factors on physical growth.Methods Based on the specialized thalassemia database from gene therapy clinical research at the Institute of Hematology&Hospital of Blood Diseases,Chinese Academy of Medical Sciences&Peking Union Medical College,we collected data on height and weight development,familyy economic status,and medical records of 338 pediatric patients with TDT from October 2023 to May 2024.
文摘In a recent article in The Lancet,an international team led by JL Kwiatkowski,F Locatelli and AA Thompson presented their successful multicenter phase 3 clinical trial(HGB-212,NCT03207009)that used gene therapy to treat severe genetic forms of transfusion-dependentβ-thalassemia.
文摘Objective： The aims of this study were to assess the prognostic significance of WHO-based Prognostic Scoring System （WPSS） in myelodysplastic syndrome （MDS） from a single center institute and to compare WPSS with the international prognostic scoring system （IPSS）. Methods： A total of 100 cases with de novo MDS were reviewed and their karyotypes were detected. All of them were followed up and classified according to IPSS and WPSS risk groups. SPSS 13.0 software was applied to deal with all the data. The statistical methods included Kaplan - Meier, Log-rank test and cox regression. Results： Multivariate cox regression analysis indicated that WHO Classification （P=0.0190）, karyotype abnormalities categorized according to IPSS （P=0.0159） and red blood cell （RBC） transfusion （P=0.0009） were the three most important independent factors for predicting overall survival （OS） of MDS. WPSS and IPSS both had great capacity in predicting the OS of MDS at the time of diagnosis （P〈0.0001）. In time-dependent analysis, WPSS can predict the OS accurately in the following three years after diagnosis （P〈0.0001）, while IPSS failed to predict the OS 24 months after diagnosis （P=0.1094）. Conclusion： Our single center results proved that WPSS is a dynamic prognostic system which can predict the OS of MDS patients at any time during the course of their disease. This time-dependent prognostic scoring system may replace the IPSS in the near future.
