Changcheng Zheng
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View article: Factor IX-Padua AAV gene therapy in hemophilia B: phases 1/2 and 3 trials
Factor IX-Padua AAV gene therapy in hemophilia B: phases 1/2 and 3 trials Open
Gene therapy for hemophilia B with adeno-associated virus (AAV) vector has achieved great advances over the last decade. We previously conducted a pilot study demonstrating the safety and efficacy of AAV-factor IX (FIX) Padua gene therapy …
View article: Chinese guidance for the clinical application of adeno-associated virus vector-based gene therapy for hemophilia B (2025)
Chinese guidance for the clinical application of adeno-associated virus vector-based gene therapy for hemophilia B (2025) Open
Currently, factor IX replacement is the mainstay treatment for hemophilia B in clinical practice. However, since the disease cannot be cured, lifelong treatment and frequent infusion is required. In recent years, hemophilia B gene therapy …
View article: Efficacy and safety of Factor IX Padua gene transfer via BBM-H901 among Chinese patients with haemophilia B: A multicentre, single-arm, phase 1/2 and 3 study and long-term follow-up of a phase 1 pilot study
Efficacy and safety of Factor IX Padua gene transfer via BBM-H901 among Chinese patients with haemophilia B: A multicentre, single-arm, phase 1/2 and 3 study and long-term follow-up of a phase 1 pilot study Open
Building on promising phase 1 pilot results demonstrating safety and efficacy of BBM-H901 (AAV-mediated FIX Padua gene therapy) in restoring factor IX activity (FIX:C) among 10 patients with haemophilia B, we conducted current studies to v…
View article: Real-World Analysis of Clinical Characteristics, Treatment Outcomes, and the Novel Predictive Model for Patients with Thrombotic Thrombocytopenic Purpura (TTP) and TTP-Like Syndrome
Real-World Analysis of Clinical Characteristics, Treatment Outcomes, and the Novel Predictive Model for Patients with Thrombotic Thrombocytopenic Purpura (TTP) and TTP-Like Syndrome Open
This study demonstrates that TTP and TTP-like syndrome are two distinct types of diseases. The new predictive model has shown good efficacy in distinguishing TTP and TTP-like syndrome. Plasma exchange significantly improves survival in TTP…
View article: Detection of nontoxic BoNT/A levels in post-facial Botox injection breastmilk
Detection of nontoxic BoNT/A levels in post-facial Botox injection breastmilk Open
The use of cosmetic Botox (BoNT/A) has become increasingly prevalent among women, even during the post-pregnancy breastfeeding period. However, there is currently a limited understanding of the extent Botox enters breastmilk and its potent…
View article: Neutralizing antibody test supports booster strategy for young individuals after SARS-CoV-2 Omicron breakthrough
Neutralizing antibody test supports booster strategy for young individuals after SARS-CoV-2 Omicron breakthrough Open
View article: Protocol to measure cell avidity between cord blood-derived NK cells and leukemia cell line KG-1a
Protocol to measure cell avidity between cord blood-derived NK cells and leukemia cell line KG-1a Open
View article: Successfully treatment of PLZF-RARα positive acute promyelocytic leukemia by Venetoclax combining with decitabine: a case report and literature review
Successfully treatment of PLZF-RARα positive acute promyelocytic leukemia by Venetoclax combining with decitabine: a case report and literature review Open
Venetoclax combining with decitabine can be used as an effective therapy in the PLZF-RARα positive APL.
View article: Highly Curative Treatment of High-Risk Acute Promyelocytic Leukemia: Induction and Consolidation with ATRA+ATO+anthracyclines and Maintenance with ATRA+RIF
Highly Curative Treatment of High-Risk Acute Promyelocytic Leukemia: Induction and Consolidation with ATRA+ATO+anthracyclines and Maintenance with ATRA+RIF Open
For newly diagnosed high-risk APL patients, induction and consolidation with ATRA+ATO+anthracyclines and maintenance with ATRA+RIF is a highly curative treatment approach.
View article: Detection of Nontoxic BoNT/A Levels in Post-Facial Botox Injection Breastmilk using a Multi-technique Approach
Detection of Nontoxic BoNT/A Levels in Post-Facial Botox Injection Breastmilk using a Multi-technique Approach Open
Background The use of cosmetic Botox (Botulinum Neurotoxin Type A, BoNT/A ) has become increasingly prevalent. Particularly after pregnancy, postpartum depression represents one major factor motivating women to use Botox even during the la…
View article: Venetoclax acts as an immunometabolic modulator to potentiate adoptive NK cell immunotherapy against leukemia
Venetoclax acts as an immunometabolic modulator to potentiate adoptive NK cell immunotherapy against leukemia Open
Natural killer (NK) cell-based immunotherapy holds promise for cancer treatment; however, its efficacy remains limited, necessitating the development of alternative strategies. Here, we report that venetoclax, an FDA-approved BCL-2 inhibit…
View article: Can COVID-19 Increase Platelet in Adult Immune Thrombocytopenia During the TPO-RA Administration? A Real-World Observational Study
Can COVID-19 Increase Platelet in Adult Immune Thrombocytopenia During the TPO-RA Administration? A Real-World Observational Study Open
This study showed that some adult ITP patients had an increase in platelet count after COVID-19 infection, and this phenomenon was strongly associated with the use of TPO-RA at the time of infection. Although no thrombotic events were obse…
View article: Safety and infection risk factors in elderly acute myeloid leukemia patients undergoing induction therapy with venetoclax combined with hypomethylating agents
Safety and infection risk factors in elderly acute myeloid leukemia patients undergoing induction therapy with venetoclax combined with hypomethylating agents Open
Venetoclax combined with hypomethylating agents shows a favorable safety profile and reduced infection risk in the treatment of AML in the elderly patients. Meanwhile, nontargeted therapies, a prolonged duration of neutropenia, and a prolo…
View article: Efficacy and safety of QL0911 in adult patients with chronic primary immune thrombocytopenia: A multicenter, randomized, double-blind, placebo-controlled, phase III trial
Efficacy and safety of QL0911 in adult patients with chronic primary immune thrombocytopenia: A multicenter, randomized, double-blind, placebo-controlled, phase III trial Open
Objective QL0911, a recombinant human thrombopoietin mimetic peptide-Fc fusion protein, is a romiplostim (Nplate ® ) biosimilar used to treat primary immune thrombocytopenia (ITP). This phase III study aimed to assess the efficacy and safe…
View article: Selinexor Synergistically Promotes the Antileukemia Activity of Venetoclax in Acute Myeloid Leukemia by Inhibiting Glycolytic Function and Downregulating the Expression of DNA Replication Genes
Selinexor Synergistically Promotes the Antileukemia Activity of Venetoclax in Acute Myeloid Leukemia by Inhibiting Glycolytic Function and Downregulating the Expression of DNA Replication Genes Open
This study indicated that selinexor can synergistically enhance the antileukemia activity of venetoclax in AML cells in vitro by inhibiting glycolytic function and downregulating DNA replication-related genes. Based on our experimental dat…
View article: Treatment of immune thrombocytopenia with hetrombopag olamine tablets in a Kabuki syndrome patient with new KMT2D mutations
Treatment of immune thrombocytopenia with hetrombopag olamine tablets in a Kabuki syndrome patient with new KMT2D mutations Open
Kabuki syndrome (KS) is a rare multisystem-affecting genetic disorder, and usually accompanied with autoimmune disorders such as immune thrombocytopenic purpura (ITP). Here, we report a 16-year-old patient with Kabuki syndrome with ITP and…
View article: A survey and antibody test following the surge of SARS-CoV-2 Omicron infection in China
A survey and antibody test following the surge of SARS-CoV-2 Omicron infection in China Open
The surge of SARS-CoV-2 Omicron infection in most Chinese residents at the end of 2022 provided a unique opportunity to understand how the immune system responds to the Omicron infection in a population with limited contact to prior SARS-C…
View article: High-dose cytarabine monotherapy is superior to standard-dose cytarabine- based multiagent sequential treatment cycle for consolidation treatment in adult (14-59 years) AML patients according to European Leukemia Net 2022 risk stratification
High-dose cytarabine monotherapy is superior to standard-dose cytarabine- based multiagent sequential treatment cycle for consolidation treatment in adult (14-59 years) AML patients according to European Leukemia Net 2022 risk stratification Open
Introduction We firstly investigate based on 2022 European Leukemia Net (ELN) risk stratification, whether standard-dose cytarabine based multiagent sequential chemotherapy (SDMSC) is more beneficial than high-dose cytarabine (HDAC) monoth…
View article: The Addition of Hypomethylating Agents to Low-Intensity Induction Chemotherapy Does Not Improve Outcomes in Elderly Acute Myeloid Leukemia Patients: A Single-Center Retrospective Study
The Addition of Hypomethylating Agents to Low-Intensity Induction Chemotherapy Does Not Improve Outcomes in Elderly Acute Myeloid Leukemia Patients: A Single-Center Retrospective Study Open
Background and Objectives: This study aimed to evaluate whether the addition of hypomethylating agents (HMA) to low-intensity chemotherapy can enhance the clinical efficacy of induction treatment for elderly acute myeloid leukemia (AML) pa…
View article: Cytomegalovirus-Specific T Cells from Third-Party Donors Successfully Treated Refractory Cytomegalovirus Retinitis after Unrelated Umbilical Cord Blood Transplantation
Cytomegalovirus-Specific T Cells from Third-Party Donors Successfully Treated Refractory Cytomegalovirus Retinitis after Unrelated Umbilical Cord Blood Transplantation Open
Umbilical cord blood (UCB) transplants (UCBTs) are becoming increasingly common in the treatment of a variety of hematologic and nonhematologic conditions. The T cells from UCB are naïve T cells, which have not yet been exposed to antigens…
View article: Standard-Intensity Induction and Intermediate/High-Dose Cytarabine Consolidation Can Improve Survival for Elderly Patients with Newly Diagnosed Acute Myeloid Leukemia: A Retrospective Cohort Study
Standard-Intensity Induction and Intermediate/High-Dose Cytarabine Consolidation Can Improve Survival for Elderly Patients with Newly Diagnosed Acute Myeloid Leukemia: A Retrospective Cohort Study Open
Li Wang, Na Zhao, Li Zhou, Juan Tong, Lei Xue, Lei Zhang, Yongsheng Han, Xingbing Wang, Liangquan Geng, Baolin Tang, Huilan Liu, Weibo Zhu, Xiaoyan Cai, Xin Liu, Xiaoyu Zhu, Zimin Sun, Changcheng Zheng Department of Hematology, The First A…
View article: Dose tapering to withdrawal stage and long‐term efficacy and safety of hetrombopag for the treatment of immune thrombocytopenia: Results from an open‐label extension study
Dose tapering to withdrawal stage and long‐term efficacy and safety of hetrombopag for the treatment of immune thrombocytopenia: Results from an open‐label extension study Open
View article: [Unrelated cord blood stem cell transplantation for high-risk/refractory childhood acute myeloid leukemia: a clinical analysis of 160 cases].
[Unrelated cord blood stem cell transplantation for high-risk/refractory childhood acute myeloid leukemia: a clinical analysis of 160 cases]. Open
Objective: To retrospectively analyze the clinical outcomes of single unrelated cord blood transplantation (UCBT) in children with high risk and refractory acute myeloid leukemia (AML) . Methods: Between June 2008 and Decembe…
View article: Efficacy of Venetoclax Combined with Decitabine-Based Treatment for Heavily Pre-Treated Relapsed or Refractory AML Patients in a Real-World Setting
Efficacy of Venetoclax Combined with Decitabine-Based Treatment for Heavily Pre-Treated Relapsed or Refractory AML Patients in a Real-World Setting Open
The efficacy of venetoclax plus decitabine-based treatment in the real-world treatment of heavily pre-treated RR-AML is similar to that in clinical trials, and the side effects are controllable.
View article: Clinical efficacy of high-dose dexamethasone with sequential prednisone maintenance therapy for newly diagnosed adult immune thrombocytopenia in a real-world setting
Clinical efficacy of high-dose dexamethasone with sequential prednisone maintenance therapy for newly diagnosed adult immune thrombocytopenia in a real-world setting Open
Objective As first-line treatments for newly diagnosed adult immune thrombocytopenia (ITP), high-dose dexamethasone (HD-DXM) and conventional-dose prednisone achieve good initial responses, but their long-term efficacy is poor. To improve …
View article: Imbalance of T Lymphocyte Subsets in Adult Immune Thrombocytopenia
Imbalance of T Lymphocyte Subsets in Adult Immune Thrombocytopenia Open
Our results suggest that T lymphocyte immune dysfunction does exist in adult ITP patients and plays an important role in the pathogenesis of ITP.
View article: A multicenter, randomized phase III trial of hetrombopag: a novel thrombopoietin receptor agonist for the treatment of immune thrombocytopenia
A multicenter, randomized phase III trial of hetrombopag: a novel thrombopoietin receptor agonist for the treatment of immune thrombocytopenia Open
Background Hetrombopag, a novel thrombopoietin receptor agonist, has been found in phase I studies to increase platelet counts and reduce bleeding risks in adults with immune thrombocytopenia (ITP). This phase III study aimed to evaluate t…
View article: All-trans retinoic acid 45 mg/m2 is superior to 25 mg/m2 as the first induction regimen for the treatment of acute promyelocytic leukaemia: a retrospective analysis in a real-world clinical setting
All-trans retinoic acid 45 mg/m2 is superior to 25 mg/m2 as the first induction regimen for the treatment of acute promyelocytic leukaemia: a retrospective analysis in a real-world clinical setting Open
View article: Umbilical cord blood transplantation can overcome the poor prognosis of KMT2A-MLLT3 acute myeloid leukemia and can lead to good GVHD-free/relapse-free survival
Umbilical cord blood transplantation can overcome the poor prognosis of KMT2A-MLLT3 acute myeloid leukemia and can lead to good GVHD-free/relapse-free survival Open
View article: The Cytokine Profiles and Immune Response Are Increased in COVID-19 Patients with Type 2 Diabetes Mellitus
The Cytokine Profiles and Immune Response Are Increased in COVID-19 Patients with Type 2 Diabetes Mellitus Open
The induction of inflammation and cytokine storm was proposed to play a critical role in COVID-19. This study is aimed at investigating the relationship between glucose metabolism and the inflammatory state of inpatients with COVID-19. 71 …