Mankai Ju
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View article: Real-world clinical trial of hetrombopag (TPO-RA) in the treatment of primary immune thrombocytopenia (ITP)
Real-world clinical trial of hetrombopag (TPO-RA) in the treatment of primary immune thrombocytopenia (ITP) Open
Background: Primary immune Thrombocytopenia (ITP) is an acquired autoimmune hemorrhagic disorder characterized by isolated peripheral thrombocytopenia without identifiable causes. Its primary pathogenesis involves increased platelet destru…
View article: Prophylaxis CTLA4-ig reduces anti-AAV neutralizing antibodies in AAV gene therapy for hemophilia
Prophylaxis CTLA4-ig reduces anti-AAV neutralizing antibodies in AAV gene therapy for hemophilia Open
Objective: This study aimed to investigate the inhibitory effect of CTLA4-Ig on anti-AAV neutralizing antibody (Nab) development after AAV gene therapy in hemophilia patients, providing an immunomodulatory strategy for potential re-treatme…
View article: Anti-CD38 monoclonal antibody CM313 for primary immune thrombocytopenia: multicentre, randomised, placebo controlled, phase 2 trial
Anti-CD38 monoclonal antibody CM313 for primary immune thrombocytopenia: multicentre, randomised, placebo controlled, phase 2 trial Open
Objective To assess the efficacy and safety of CM313, an anti-CD38 monoclonal antibody, in adults with persistent or chronic primary immune thrombocytopenia. Design Multicentre, randomised, placebo controlled, phase 2 trial. Setting Five h…
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: Current progress on pathogenicity-related genes in Fusarium oxysporum f. sp. cubense tropical race 4
Current progress on pathogenicity-related genes in Fusarium oxysporum f. sp. cubense tropical race 4 Open
Vascular wilt, a disease caused by Fusarium oxysporum f. sp. cubense tropical race 4 (Foc TR4), is highly destructive to bananas. Identifying genes that contribute to the fungus's virulence is crucial for understanding its pathogenesis. In…
View article: Integrating chemokines and machine learning algorithms for diagnosis and bleeding assessment in primary immune thrombocytopenia: A prospective cohort study
Integrating chemokines and machine learning algorithms for diagnosis and bleeding assessment in primary immune thrombocytopenia: A prospective cohort study Open
Summary Primary immune thrombocytopenia (ITP) is an autoimmune bleeding disorder, and chemokines have been shown to be dysregulated in autoimmune disorders. We conducted a prospective analysis to identify potential chemokines that could en…
View article: A single-center study of patients with rare isolated acquired clotting factor deficiencies other than acquired hemophilia A
A single-center study of patients with rare isolated acquired clotting factor deficiencies other than acquired hemophilia A Open
Rare isolated ACFDs are underestimated, associated with delayed diagnosis, and lack effective therapy. Clinicians should raise awareness for recognizing and managing rare isolated ACFD patients to avoid morbidity and mortality.
View article: Efficacy and safety of human umbilical cord-derived mesenchymal stem cells in the treatment of refractory immune thrombocytopenia: a prospective, single arm, phase I trial
Efficacy and safety of human umbilical cord-derived mesenchymal stem cells in the treatment of refractory immune thrombocytopenia: a prospective, single arm, phase I trial Open
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 promisin…
View article: Sex-specific impact on disease outcome and the mutational landscape in essential thrombocythemia: A retrospective cohort study
Sex-specific impact on disease outcome and the mutational landscape in essential thrombocythemia: A retrospective cohort study Open
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 to patient heterogeneity, and adopting a sex-informe…
View article: Proteomics landscape and machine learning prediction of long‐term response to splenectomy in primary immune thrombocytopenia
Proteomics landscape and machine learning prediction of long‐term response to splenectomy in primary immune thrombocytopenia Open
Summary This study aimed to identify key proteomic analytes correlated with response to splenectomy in primary immune thrombocytopenia (ITP). Thirty‐four patients were retrospectively collected in the training cohort and 26 were prospectiv…
View article: Genetically Determined Telomere Length and Risk for Haematologic Diseases: Results from Large Prospective Cohorts and Mendelian Randomization Analysis
Genetically Determined Telomere Length and Risk for Haematologic Diseases: Results from Large Prospective Cohorts and Mendelian Randomization Analysis Open
The causal direction and extent of the link between telomere length, epigenetic age acceleration (EAA), and the occurrence of haematological malignancies and benign haematological disorders remain uncertain because of the inherent suscepti…
View article: Acquired hemophilia A: a single-center study of 165 patients
Acquired hemophilia A: a single-center study of 165 patients Open
Immunosuppressive therapies yield remarkably high response rates, with a CR rate exceeding 80%; notably, the regimen containing rituximab exhibits a CR rate of approximately 90%. FVIII inhibitor titer of ≥5 BU/mL and BS of ≥6 were poor pre…
View article: Efficacy and safety of eltrombopag in the treatment of immune thrombocytopenia patients with hepatitis B virus infection: A prospective, multicenter, single arm, phase <scp>II</scp> trial
Efficacy and safety of eltrombopag in the treatment of immune thrombocytopenia patients with hepatitis B virus infection: A prospective, multicenter, single arm, phase <span>II</span> trial Open
Efficacy and safety of eltrombopag in the treatment of immune thrombocytopenia patients with hepatitis B virus infection: A prospective, multicenter, single arm, phase II trialTo the Editor: Hepatitis B virus (HBV) infection is the most co…
View article: Single‐dose rituximab plus glucocorticoid versus cyclophosphamide plus glucocorticoid in patients with newly diagnosed acquired hemophilia A: A multicenter, open‐label, randomized noninferiority trial
Single‐dose rituximab plus glucocorticoid versus cyclophosphamide plus glucocorticoid in patients with newly diagnosed acquired hemophilia A: A multicenter, open‐label, randomized noninferiority trial Open
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 the first multicenter RCT aiming to establish whether th…
View article: Longitudinal assessment of fatigue and health-related quality of life in adults with immune thrombocytopenia in China
Longitudinal assessment of fatigue and health-related quality of life in adults with immune thrombocytopenia in China Open
Background Currently, there are few studies focusing on fatigue and health-related quality of life (HRQoL) longitudinally among immune thrombocytopenia (ITP) adults, especially in China. We aimed to evaluate changes in fatigue and HRQoL an…
View article: Hematopoietic stem cell heterogeneity is linked to the initiation and therapeutic response of myeloproliferative neoplasms
Hematopoietic stem cell heterogeneity is linked to the initiation and therapeutic response of myeloproliferative neoplasms Open
(Cell Stem Cell 28, 502–513.e1–e6; March 4, 2021) In the originally published version of our manuscript, we indicated the wrong accession number for our single-cell RNA-seq data both in the Deposited data subsection of the Key resources ta…
View article: [Gene mutation spectrum and clinical characteristics analysis of 178 patients with essential thrombocytosis].
[Gene mutation spectrum and clinical characteristics analysis of 178 patients with essential thrombocytosis]. Open
Objective: To analyze the gene mutation spectrum, clinical features, and the factors of disease progression and prognosis in patients with essential thrombocytosis (ET) . Methods: A retrospective analysis was conducted on 178 newly diagnos…
View article: Multilevel defects in the hematopoietic niche in essential thrombocythemia
Multilevel defects in the hematopoietic niche in essential thrombocythemia Open
The role of the bone marrow niche in essential thrombocythemia (ET) remains unclear. Here, we observed multilevel defects in the hematopoietic niche of patients with JAK2V617F-positive ET, including functional deficiency in mesenchymal str…
View article: [The value of platelet count in predicting the efficacy of rituximab treatment in adult patients with chronic primary immune thrombocytopenia].
[The value of platelet count in predicting the efficacy of rituximab treatment in adult patients with chronic primary immune thrombocytopenia]. Open
Objective: To investigate the value of platelet count in predicting the efficacy of rituximab treatment in chronic primary immune thrombocytopenia (ITP). Methods: A retrospective study was conducted in 103 chronic ITP patients hospitalized…
View article: ASXL1 mutations in Chinese patients with essential thrombocythemia
ASXL1 mutations in Chinese patients with essential thrombocythemia Open
Essential thrombocythemia (ET) is characterized by thrombotic and hemorrhagic events. The association of clinical characteristics of Chinese ET patients and additional sex combs like 1 (ASXL1) mutations in these patients has remained to be…