A5100432005- Hemophilia Treatment and Research
- Platelet Disorders and Treatments
- Myeloproliferative Neoplasms: Diagnosis and Treatment
- Chronic Myeloid Leukemia Treatments
- Blood groups and transfusion
- Coagulation, Bradykinin, Polyphosphates, and Angioedema
- Blood Coagulation and Thrombosis Mechanisms
- Kruppel-like factors research
- Cancer-related gene regulation
- Acute Myeloid Leukemia Research
- Blood properties and coagulation
- Autoimmune Bullous Skin Diseases
- Hematopoietic Stem Cell Transplantation
- Eosinophilic Disorders and Syndromes
- Dermatological and Skeletal Disorders
- Hemostasis and retained surgical items
- Multiple Myeloma Research and Treatments
- Lymphoma Diagnosis and Treatment
- Chronic Lymphocytic Leukemia Research
- Acute Lymphoblastic Leukemia research
- Pluripotent Stem Cells Research
- CAR-T cell therapy research
- Mesenchymal stem cell research
- Immunodeficiency and Autoimmune Disorders
- CRISPR and Genetic Engineering
Chinese Academy of Medical Sciences & Peking Union Medical College
2015-2025
Institute of Hematology & Blood Diseases Hospital
2015-2025
Peking Union Medical College Hospital
2014-2025
Beijing Tian Tan Hospital
2025
Capital Medical University
2025
Peking University First Hospital
2017-2024
Peking University
2017-2024
China Shenhua Energy (China)
2024
CHN Energy (China)
2024
Aerospace Center Hospital
2023
Replacement therapy for hemophilia remains a lifelong treatment. Only gene can cure at fundamental level. The clustered regularly interspaced short palindromic repeats–CRISPR associated nuclease 9 (CRISPR-Cas9) system is versatile and convenient genome editing tool which be applied to hemophilia. A patient's induced pluripotent stem cells (iPSCs) were generated from their peripheral blood mononuclear (PBMNCs) using episomal vectors. AAVS1-Cas9-sgRNA plasmid targets the AAVS1 locus...
Patients with refractory immune thrombocytopenia (ITP) frequently encounter substantial bleeding risks and demonstrate limited responsiveness to existing therapies. Umbilical cord-derived mesenchymal stem cells (UC-MSCs) present a promising alternative, capitalizing on their low immunogenicity potent immunomodulatory effects for treating diverse autoimmune disorders. This prospective phase I trial enrolled eighteen eligible patients explore the safety efficacy of UC-MSCs in ITP. The research...
Choledochal cysts are most common in Asian populations. In addition, the incidence of biliary tract malignancies from choledochal is increasing, but risk carcinogenesis still unclear.Clinical data 214 congenital cyst cases 1968 to 2013 were retrospectively analyzed.Todani type I was more (139, 65.0%) than IVa (53, 24.8%) or V (17, 7.9%) these patients. Biliary malignant tumors occurred gallbladder (2, 13.3%), bile duct (10, 66.7%), and intrahepatic (3, 20%) 15 patients (7.0%), including one...
Background: The radiation oncology clinical practice involves many steps relying on the dynamic interplay of abundant text data. Large language models have displayed remarkable capabilities in processing complex information. But their direct applications specific fields like remain underexplored. Purpose: This study aims to investigate whether fine-tuning LLMs with domain knowledge can improve performance Task (1) treatment regimen generation, (2) modality selection (photon, proton,...
Purpose Atypical teratoid/rhabdoid tumors (ATRTs) are very rare, highly malignant embryonal neoplasms in central nervous system. The aim of this study was to conduct a retrospective analysis ATRT patient survival and investigate the prognostic factors associated with ATRT. Methods A conducted using information patients who received treatment between 2016 2021 Beijing Tiantan Hospital. Kaplan–Meier curves were used for overall (OS) analysis. Univariate multivariate COX analyses applied OS...
This study aimed to summarize the intestinal ultrasound (IUS) and computed tomography enterography (CTE) features of cryptogenic multifocal ulcerous stenosing enteritis (CMUSE) compare performance IUS CTE in evaluation CMUSE a single tertiary center. Clinically or pathologically confirmed patients between December 2009 April 2023 were recruited. Imaging who underwent both summarized retrospectively. Twenty-nine included. All found have ileum involvement, with majority (96.6%, 28/29) showing...
Objective: Our previous studies have indicated potentially higher proliferative activity of tumor cells in Chinese patients with mantle-cell lymphoma (MCL) than those Western. Given the success and tolerability R-DA-EDOCH immunochemotherapy treating aggressive B-cell lymphomas, we designed a prospective, phase 3 trial to explore efficacy safety alternating R-DA-EDOCH/R-DHAP induction therapy for young newly diagnosed MCL. The primary endpoint was complete remission rate (CRR) at end (EOI)....
<i>Aim:</i> This multicenter, open-label study determined safety and efficacy of once-every-other-week administration darbepoetin alfa for anemia chronic kidney disease in erythropoietin-naive patients not on dialysis. <i>Methods:</i> Participants with hemoglobin levels <11.0 g/dl at baseline were administered an initial dosage 0.75 µg/kg once every other week. The dose was titrated to achieve maintain a response, defined as range between 11.0 13.0 up 24 weeks....
Abstract Acquired hemophilia A (AHA) is a rare but serious bleeding disorder. Randomized controlled trial (RCT) comparing the efficacy of immunosuppression therapy for AHA lacks. We conducted first multicenter RCT aiming to establish whether single‐dose rituximab combination regimen was noninferior cyclophosphamide regimen. From 2017 2022, 63 patients with newly diagnosed from five centers were randomly assigned 1:1 receive glucocorticoid (methylprednisolone 0.8 mg/kg per day 3 weeks and...
Abstract Nectin‐like molecule 1 (NECL1)/CADM3/IGSF4B/TSLL1/SynCAM3 is a neural tissue‐specific immunoglobulin‐like cell–cell adhesion downregulated at the mRNA level in 12 human glioma cell lines. Here we found that expression of NECL1 was lost six lines and 15 primary tissues both RNA protein levels. Re‐expression into line U251 would repress proliferation vitro by inducing cycle arrest. And also could decrease growth rate tumors nude mice vivo . To further investigate mechanism why...
Bleeding episodes in hemophiliacs with inhibitors are difficult to control. Staidson protein-0601 (STSP-0601), a specific factor (F)X activator purified from the venom of Daboia russelii siamensis, has been developed.We aimed investigate efficacy and safety STSP-0601 preclinical clinical studies.In vitro vivo studies were performed. A phase 1, first-in-human, multicenter, open-label trial was conducted. The study divided into parts B. Hemophiliacs eligible for this study. Patients received...
Reduced doses of emicizumab improve the affordability among patients in developing countries. However, relationship between variant dose selection and efficacy real world China is still unclear.
Abstract Introduction The management of haemophilia is critical to minimize the risk disability and reduce burden on China's healthcare system. Aim This study was based a single centre in China conducted understand evolution real‐world care over past 15 years. Methods We retrospectively analysed clinical characteristics, diagnosis, treatment medical expenditures 428 patients with from January 2004 December 2018 Institute Hematology & Blood Diseases Hospital Tianjin, China. Results...
The development of inhibitors against factor FIX (FIX) is the most serious complication replacement therapy in haemophilia B (HB) patients. Currently, only few cohorts HB inhibitor patients have been reported worldwide.This Chinese nationwide study explored their risk factors for and experience on management.We retrospectively analysed patient characteristics, F9 genotypes, treatment strategies outcomes registered to National Registry Patient Organization Registry.Forty-four unique were...
SUMMARY: Aim: Darbepoetin alfa, an erythropoiesis‐stimulating protein, has a longer serum half‐life than recombinant human erythropoietin, allowing less‐frequent administration. This study aimed to demonstrate that once‐monthly (QM) darbepoetin alfa administration would maintain haemoglobin (Hb) concentrations in subjects with chronic kidney disease (CKD) not receiving dialysis who had previously been administered every 2 weeks (Q2W). Methods: was multicentre which CKD stable Q2W doses and...
Congenital factor VII (FVII) deficiency is a rare bleeding disorder characterised by wide molecular and clinical heterogeneity. We investigated the phenotype of 193 patients F7 genotype 55/193 with FVII throughout China showed their current status management. The most frequent symptoms were epistaxis (44.6%), cutaneous (38.9%), oral cavity (40.4%) menorrhagia (44.3% females reproductive age). Fatal central nervous system disabling joint occurred in three each. majority (89.6%) had activity...
To the Editor: Male sex has been confirmed to be an independent risk factor for survival in patients with essential thrombocythemia (ET).[1,2] Sexual dimorphism significantly contributes patient heterogeneity, and adopting a sex-informed perspective emerged as pioneering paradigm field of precision medicine. Investigating spectrum disparities among diagnosed ET holds substantial scientific justification. Furthermore, influences presentation, disease phenotypes, symptom expression, clinical...