Pub Date : 2025-10-14DOI: 10.3760/cma.j.cn121090-20250519-00235
M Gao, Y Xie, Z Y Liu, P Q Liang, L M Liu, J Yin, D Wang, B Han, H Y Qiu, J H Fu, D P Wu
This report presents the management of a critically ill 36-year-old woman diagnosed with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph(+)ALL) at 28 weeks of gestation. The patient rapidly deteriorated, developing disseminated intravascular coagulation (DIC) , diffuse alveolar hemorrhage (DAH) , septic shock, and multi-organ dysfunction, necessitating admission to the hematological intensive care unit. Given her critical condition and advanced pregnancy, a chemotherapy-free induction regimen comprising imatinib and dexamethasone was initiated, alongside comprehensive supportive measures, including mechanical ventilation, continuous renal replacement therapy (CRRT) , broad-spectrum antibiotics, and high-dose corticosteroids. During treatment, intrauterine fetal demise occurred, and a stillborn was delivered following obstetric intervention. With aggressive treatment, the patient's respiratory failure, DIC, and DAH gradually resolved, and she achieved complete remission. She subsequently received consolidation chemotherapy, CAR-T cell therapy, and allogeneic hematopoietic stem cell transplantation, achieving sustained complete molecular remission on long-term follow-up. This case demonstrates that for critically ill pregnant patients with Ph(+) ALL, a chemotherapy-free regimen of targeted therapy and corticosteroids, when combined with intensive supportive care, is a safe and effective approach that may offer a therapeutic option for similar cases.
{"title":"[Chemotherapy-free induction therapy for a critically ill pregnant woman with Philadelphia chromosome-positive acute lymphoblastic leukemia: a case report and literature review].","authors":"M Gao, Y Xie, Z Y Liu, P Q Liang, L M Liu, J Yin, D Wang, B Han, H Y Qiu, J H Fu, D P Wu","doi":"10.3760/cma.j.cn121090-20250519-00235","DOIUrl":"10.3760/cma.j.cn121090-20250519-00235","url":null,"abstract":"<p><p>This report presents the management of a critically ill 36-year-old woman diagnosed with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph(+)ALL) at 28 weeks of gestation. The patient rapidly deteriorated, developing disseminated intravascular coagulation (DIC) , diffuse alveolar hemorrhage (DAH) , septic shock, and multi-organ dysfunction, necessitating admission to the hematological intensive care unit. Given her critical condition and advanced pregnancy, a chemotherapy-free induction regimen comprising imatinib and dexamethasone was initiated, alongside comprehensive supportive measures, including mechanical ventilation, continuous renal replacement therapy (CRRT) , broad-spectrum antibiotics, and high-dose corticosteroids. During treatment, intrauterine fetal demise occurred, and a stillborn was delivered following obstetric intervention. With aggressive treatment, the patient's respiratory failure, DIC, and DAH gradually resolved, and she achieved complete remission. She subsequently received consolidation chemotherapy, CAR-T cell therapy, and allogeneic hematopoietic stem cell transplantation, achieving sustained complete molecular remission on long-term follow-up. This case demonstrates that for critically ill pregnant patients with Ph(+) ALL, a chemotherapy-free regimen of targeted therapy and corticosteroids, when combined with intensive supportive care, is a safe and effective approach that may offer a therapeutic option for similar cases.</p>","PeriodicalId":24016,"journal":{"name":"Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi","volume":"46 10","pages":"967-971"},"PeriodicalIF":0.0,"publicationDate":"2025-10-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12665469/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145670095","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-10-14DOI: 10.3760/cma.j.cn121090-20250702-00309
L L Fu, S Sun, D D Wang, J N Li
{"title":"[Systemic necrotizing lymphadenitis: a case report].","authors":"L L Fu, S Sun, D D Wang, J N Li","doi":"10.3760/cma.j.cn121090-20250702-00309","DOIUrl":"10.3760/cma.j.cn121090-20250702-00309","url":null,"abstract":"","PeriodicalId":24016,"journal":{"name":"Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi","volume":"46 10","pages":"979"},"PeriodicalIF":0.0,"publicationDate":"2025-10-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12665464/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145670163","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-10-14DOI: 10.3760/cma.j.cn121090-20250120-00035
X X Shen, J P Yu, R Guo, Y Xu, Y Y Jin, Q L Shi, L J Chen
To retrospectively analyze the clinical data of 465 newly diagnosed patients with multiple myeloma (NDMM) admitted to the First Affiliated Hospital of Nanjing Medical University from December 2016 to December 2024, and compare the prognostic value of high-risk cytogenetic abnormalities (HRCAs) in NDMM patients under mSMART 3.0 and mSMART 4.0 risk stratification systems. The results showed that in both stratification systems, the prognosis of high-risk patients was worse than that of standard-risk patients. Moreover, a higher number of HRCAs was associated with a worse prognosis. The mSMART 4.0 system, which considers the coexistence of various cytogenetic abnormalities, provides a more precise definition of HRCA than mSMART 3.0. It demonstrates a superior ability to differentiate between different categories of cytogenetic risk.
{"title":"[Prognostic value of high-risk cytogenetic abnormalities inmultiple myeloma].","authors":"X X Shen, J P Yu, R Guo, Y Xu, Y Y Jin, Q L Shi, L J Chen","doi":"10.3760/cma.j.cn121090-20250120-00035","DOIUrl":"10.3760/cma.j.cn121090-20250120-00035","url":null,"abstract":"<p><p>To retrospectively analyze the clinical data of 465 newly diagnosed patients with multiple myeloma (NDMM) admitted to the First Affiliated Hospital of Nanjing Medical University from December 2016 to December 2024, and compare the prognostic value of high-risk cytogenetic abnormalities (HRCAs) in NDMM patients under mSMART 3.0 and mSMART 4.0 risk stratification systems. The results showed that in both stratification systems, the prognosis of high-risk patients was worse than that of standard-risk patients. Moreover, a higher number of HRCAs was associated with a worse prognosis. The mSMART 4.0 system, which considers the coexistence of various cytogenetic abnormalities, provides a more precise definition of HRCA than mSMART 3.0. It demonstrates a superior ability to differentiate between different categories of cytogenetic risk.</p>","PeriodicalId":24016,"journal":{"name":"Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi","volume":"46 10","pages":"958-962"},"PeriodicalIF":0.0,"publicationDate":"2025-10-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12665476/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145670137","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-10-14DOI: 10.3760/cma.j.cn121090-20250126-00045
L Wang, M M Li, J J Bai, C X Deng, P Wu, C W Luo, P L Lai, J Y Weng, X Du
Objective: To evaluate the clinical value of peripheral blood monocyte subset analysis in the diagnosis and treatment of chronic myelomonocytic leukemia (CMML) . Method: We retrospectively enrolled 51 patients newly diagnosed with CMML at Guangdong Provincial People's Hospital between June 1, 2020, and December 31, 2024, according to the WHO 2022 diagnostic criteria. Twenty-three patients with other myeloid neoplasms (excluding CMML) and peripheral monocytosis (absolute count ≥0.5×10(9)/L and percentage ≥10%) were included as the control group. All patients underwent bone marrow aspiration for examinations including bone marrow smears, biopsies, cytogenetics, and gene mutation analysis to establish a definitive diagnosis. Concurrently, flow cytometry was used to determine the proportions of peripheral blood monocyte subsets: classical (MO1, CD14(+)CD16(-)) , intermediate (MO2, CD14(+)CD16(+)) , and non-classical (MO3, CD14(low)CD16(+)) . Differences between the groups were compared, and diagnostic efficacy was evaluated using receiver operating characteristic (ROC) curves. Result: Among the 51 CMML patients, the proportion of the peripheral blood MO1 subset was significantly higher than that in patients with other myeloid neoplasms (P=0.027) , whereas there were no significant differences in the MO2 and MO3 subsets (all P>0.05) . Further analysis revealed that 43 (84.31%) of the CMML patients met the WHO diagnostic threshold for the MO1 subset (≥94%) , while the remaining 8 patients did not; 46 patients (90.20%) had MO3 subset proportions below the threshold proposed by Hudson (≤1.13%) , while the remaining 5 patients were above this threshold. In-depth analysis showed that among the 8 patients who did not meet the WHO criteria, 7 were experiencing inflammation. Similarly, all 5 patients who did not meet the Hudson criteria were in an inflammatory state. Subsequent ROC curve analysis of this cohort identified a cut-off value for the MO1 subset of 97.55% [Area Under the Curve (AUC) =0.661, P=0.027], which aligns with the WHO criteria. Conclusion: Peripheral blood monocyte subset analysis, particularly MO1 subset analysis, can effectively assist in CMML diagnosis, but exclusion of inflammatory conditions is required.
{"title":"[Analysis of peripheral blood monocyte subsets in chronic myelomonocytic leukemia: a single-center study].","authors":"L Wang, M M Li, J J Bai, C X Deng, P Wu, C W Luo, P L Lai, J Y Weng, X Du","doi":"10.3760/cma.j.cn121090-20250126-00045","DOIUrl":"10.3760/cma.j.cn121090-20250126-00045","url":null,"abstract":"<p><p><b>Objective:</b> To evaluate the clinical value of peripheral blood monocyte subset analysis in the diagnosis and treatment of chronic myelomonocytic leukemia (CMML) . <b>Method:</b> We retrospectively enrolled 51 patients newly diagnosed with CMML at Guangdong Provincial People's Hospital between June 1, 2020, and December 31, 2024, according to the WHO 2022 diagnostic criteria. Twenty-three patients with other myeloid neoplasms (excluding CMML) and peripheral monocytosis (absolute count ≥0.5×10(9)/L and percentage ≥10%) were included as the control group. All patients underwent bone marrow aspiration for examinations including bone marrow smears, biopsies, cytogenetics, and gene mutation analysis to establish a definitive diagnosis. Concurrently, flow cytometry was used to determine the proportions of peripheral blood monocyte subsets: classical (MO1, CD14(+)CD16(-)) , intermediate (MO2, CD14(+)CD16(+)) , and non-classical (MO3, CD14(low)CD16(+)) . Differences between the groups were compared, and diagnostic efficacy was evaluated using receiver operating characteristic (ROC) curves. <b>Result:</b> Among the 51 CMML patients, the proportion of the peripheral blood MO1 subset was significantly higher than that in patients with other myeloid neoplasms (<i>P</i>=0.027) , whereas there were no significant differences in the MO2 and MO3 subsets (all <i>P</i>>0.05) . Further analysis revealed that 43 (84.31%) of the CMML patients met the WHO diagnostic threshold for the MO1 subset (≥94%) , while the remaining 8 patients did not; 46 patients (90.20%) had MO3 subset proportions below the threshold proposed by Hudson (≤1.13%) , while the remaining 5 patients were above this threshold. In-depth analysis showed that among the 8 patients who did not meet the WHO criteria, 7 were experiencing inflammation. Similarly, all 5 patients who did not meet the Hudson criteria were in an inflammatory state. Subsequent ROC curve analysis of this cohort identified a cut-off value for the MO1 subset of 97.55% [Area Under the Curve (AUC) =0.661, <i>P</i>=0.027], which aligns with the WHO criteria. <b>Conclusion:</b> Peripheral blood monocyte subset analysis, particularly MO1 subset analysis, can effectively assist in CMML diagnosis, but exclusion of inflammatory conditions is required.</p>","PeriodicalId":24016,"journal":{"name":"Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi","volume":"46 10","pages":"952-957"},"PeriodicalIF":0.0,"publicationDate":"2025-10-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12665471/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145670073","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-10-14DOI: 10.3760/cma.j.cn121090-20241214-00569
S Y Zhao, B Li, Z F Xu, T J Qin, S Q Qu, L J Pan, M Jiao, Q Y Gao, H J Wang, Q Sun, Y J Jia, Y R Yan, J Y Gong, F H Li, X Wang, Z J Xiao
<p><p><b>Objective:</b> To explore the clinical features and molecular characteristics of myeloproliferative neoplasms (MPNs) patients with NFE2 gene mutations. <b>Methods:</b> Gene targeted sequencing was used to detect NFE2 gene mutation in 723 patients diagnosed with MPNs who were admitted to Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College between April 2021 and June 2023. The association between NFE2 gene mutations and clinical features and molecular characteristics of MPNs patients were retrospectively analyzed. <b>Results:</b> Among 723 patients with MPNs, NFE2 gene mutations were found in 41 cases (5.7%) . NFE2 gene mutations were predominantly frameshift mutations (44.4%) , followed by nonsense mutations (33.3%) . The median number of mutations in patients with NFE2 gene mutations (4 [2,5]) was higher compared to the group without NFE2 gene mutations (2, [1,3]) (<i>P</i><0.001) . NFE2 gene mutations frequently co-occurred with mutations in MPL, ATM, PPM1D, and TET1. NFE2 gene mutations were mostly sub-clonal events, with 80.5% occurring after MPNs driver mutations (JAK2, CALR, or MPL) . NFE2 mutations were correlated with older age [median age: 60 (54, 67) years <i>vs</i> 54 (41, 63) years, <i>P</i>=0.001]. Patients with NFE2 gene mutations had a higher incidence of pre-diagnosis thrombosis (39.0% <i>vs</i> 22.0%, <i>P</i>=0.012) and pre-diagnosis arterial thrombosis (36.6% <i>vs</i> 20.4%, <i>P</i>=0.014) . Using a logistic regression analysis model adjusting for age and comorbidities (including chronic infections, malignancies, and autoimmune diseases) , NFE2 gene mutation was identified as an independent determinant of elevated tumor necrosis factor-alpha (TNF-α) (<i>OR</i>=2.747, 95%<i>CI</i>: 1.143-6.605, <i>P</i>=0.024) , interferon-gamma (IFN-γ) (<i>OR</i>=2.689, 95%<i>CI</i>: 1.191-6.076, <i>P</i>=0.017) , IL-10 (<i>OR</i>=3.219, 95% <i>CI</i>: 1.343-7.717, <i>P</i>=0.009) , IL-12P70 (<i>OR</i>=3.397, 95%<i>CI</i>:1.003-11.508, <i>P</i>=0.049) , IL-17 (<i>OR</i>=2.284, 95%<i>CI</i>: 1.017-5.127, <i>P</i>=0.045) . In polycythaemia vera (PV) patients with the NFE2 gene mutation, the proportion of those classified as high-risk is notably higher in both the IWG-PV and mutation-enhanced international prognostic systems for PV (MIPSS-PV) (66.7% <i>vs</i> 25.3% for IWG-PV, <i>P</i>=0.033; 22.2% <i>vs</i> 2.0% for MIPSS-PV, <i>P</i>=0.013) . Similarly, for essential thrombocythaemia (ET) patients, the proportion in the high-risk group of the mutation-enhanced international prognostic systems for ET (MIPSS-ET) is significantly higher (15.4% <i>vs</i> 6.1%, <i>P</i>=0.021) . No statistically significant differences were observed in overall survival or cumulative incidence of thrombosis between NFE2-mutated (38 cases) and non-mutated MPNs patients (671 cases, <i>P</i>>0.05) . <b>Conclusion:</b> NFE2 gene mutations in MPNs were predominantly frameshift mutations. NFE2 gene
目的:探讨骨髓增生性肿瘤(mpn) NFE2基因突变患者的临床特点及分子特征。方法:采用基因靶向测序检测2021年4月至2023年6月在中国医学科学院血液学血液病研究所和北京协和医学院住院的723例MPNs患者的NFE2基因突变。回顾性分析NFE2基因突变与MPNs患者临床特征及分子特征的关系。结果:723例MPNs患者中,NFE2基因突变41例(5.7%)。NFE2基因突变以移码突变为主(44.4%),其次是无义突变(33.3%)。NFE2基因突变患者的中位突变数(4[2,5])高于无NFE2基因突变组(2,[1,3])(Pvs 54(41,63)年,P=0.001)。NFE2基因突变患者的诊断前血栓发生率(39.0% vs 22.0%, P=0.012)和诊断前动脉血栓发生率(36.6% vs 20.4%, P=0.014)较高。通过调整年龄和合共病(包括慢性感染、恶性肿瘤和自身免疫性疾病)的logistic回归分析模型,确定NFE2基因突变是肿瘤坏死因子-α (TNF-α) (OR=2.747, 95%CI: 1.143-6.605, P=0.024)、干扰素-γ (IFN-γ) (OR=2.689, 95%CI: 1.191-6.076, P=0.017)、IL-10 (OR=3.219, 95%CI: 1.343-7.717, P=0.009)、IL-12P70 (OR=3.397, 95%CI:1.003-11.508, P=0.049)、IL-17 (OR=2.284, 95%CI: P=0.049)升高的独立决定因素。1.017-5.127, p =0.045)。在NFE2基因突变的真性红细胞增多症(PV)患者中,在IWG-PV和突变增强的PV国际预后系统(MIPSS-PV)中,被归类为高风险的比例明显更高(IWG-PV为66.7% vs 25.3%, P=0.033; MIPSS-PV为22.2% vs 2.0%, P=0.013)。同样,对于原发性血小板增多症(ET)患者,突变增强的ET国际预后系统(MIPSS-ET)在高危人群中的比例明显更高(15.4% vs 6.1%, P=0.021)。nfe2突变(38例)与非突变(671例,P < 0.05) mpn患者的总生存期和累计血栓发生率无统计学差异。结论:NFE2基因在mpn中以移码突变为主。NFE2基因突变与年龄增大、多种炎症因子(TNF-α、IFN-γ、IL-10、IL-12P70、IL-17)水平升高相关,且多发生在mpn晚期。
{"title":"[Clinical and molecular characteristics of myeloproliferative neoplasms patients with NFE2 gene mutations].","authors":"S Y Zhao, B Li, Z F Xu, T J Qin, S Q Qu, L J Pan, M Jiao, Q Y Gao, H J Wang, Q Sun, Y J Jia, Y R Yan, J Y Gong, F H Li, X Wang, Z J Xiao","doi":"10.3760/cma.j.cn121090-20241214-00569","DOIUrl":"10.3760/cma.j.cn121090-20241214-00569","url":null,"abstract":"<p><p><b>Objective:</b> To explore the clinical features and molecular characteristics of myeloproliferative neoplasms (MPNs) patients with NFE2 gene mutations. <b>Methods:</b> Gene targeted sequencing was used to detect NFE2 gene mutation in 723 patients diagnosed with MPNs who were admitted to Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College between April 2021 and June 2023. The association between NFE2 gene mutations and clinical features and molecular characteristics of MPNs patients were retrospectively analyzed. <b>Results:</b> Among 723 patients with MPNs, NFE2 gene mutations were found in 41 cases (5.7%) . NFE2 gene mutations were predominantly frameshift mutations (44.4%) , followed by nonsense mutations (33.3%) . The median number of mutations in patients with NFE2 gene mutations (4 [2,5]) was higher compared to the group without NFE2 gene mutations (2, [1,3]) (<i>P</i><0.001) . NFE2 gene mutations frequently co-occurred with mutations in MPL, ATM, PPM1D, and TET1. NFE2 gene mutations were mostly sub-clonal events, with 80.5% occurring after MPNs driver mutations (JAK2, CALR, or MPL) . NFE2 mutations were correlated with older age [median age: 60 (54, 67) years <i>vs</i> 54 (41, 63) years, <i>P</i>=0.001]. Patients with NFE2 gene mutations had a higher incidence of pre-diagnosis thrombosis (39.0% <i>vs</i> 22.0%, <i>P</i>=0.012) and pre-diagnosis arterial thrombosis (36.6% <i>vs</i> 20.4%, <i>P</i>=0.014) . Using a logistic regression analysis model adjusting for age and comorbidities (including chronic infections, malignancies, and autoimmune diseases) , NFE2 gene mutation was identified as an independent determinant of elevated tumor necrosis factor-alpha (TNF-α) (<i>OR</i>=2.747, 95%<i>CI</i>: 1.143-6.605, <i>P</i>=0.024) , interferon-gamma (IFN-γ) (<i>OR</i>=2.689, 95%<i>CI</i>: 1.191-6.076, <i>P</i>=0.017) , IL-10 (<i>OR</i>=3.219, 95% <i>CI</i>: 1.343-7.717, <i>P</i>=0.009) , IL-12P70 (<i>OR</i>=3.397, 95%<i>CI</i>:1.003-11.508, <i>P</i>=0.049) , IL-17 (<i>OR</i>=2.284, 95%<i>CI</i>: 1.017-5.127, <i>P</i>=0.045) . In polycythaemia vera (PV) patients with the NFE2 gene mutation, the proportion of those classified as high-risk is notably higher in both the IWG-PV and mutation-enhanced international prognostic systems for PV (MIPSS-PV) (66.7% <i>vs</i> 25.3% for IWG-PV, <i>P</i>=0.033; 22.2% <i>vs</i> 2.0% for MIPSS-PV, <i>P</i>=0.013) . Similarly, for essential thrombocythaemia (ET) patients, the proportion in the high-risk group of the mutation-enhanced international prognostic systems for ET (MIPSS-ET) is significantly higher (15.4% <i>vs</i> 6.1%, <i>P</i>=0.021) . No statistically significant differences were observed in overall survival or cumulative incidence of thrombosis between NFE2-mutated (38 cases) and non-mutated MPNs patients (671 cases, <i>P</i>>0.05) . <b>Conclusion:</b> NFE2 gene mutations in MPNs were predominantly frameshift mutations. NFE2 gene ","PeriodicalId":24016,"journal":{"name":"Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi","volume":"46 10","pages":"943-951"},"PeriodicalIF":0.0,"publicationDate":"2025-10-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12665466/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145670026","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-10-14DOI: 10.3760/cma.j.cn121090-20241205-00536
X Ye, Z X Sun, X Y Qu, L Fan
Multiple myeloma (MM) is a malignant disease characterized by clonal plasma cell proliferation, with metabolic reprogramming. Abnormal metabolism of amino acids, glucose, lipids and nucleotides plays a crucial role in various pathological processes in MM, such as proliferation, metastasis, immune escaping and drug resistance. This review summarized the application of metabolomics technology in identifying biomarkers for MM and targeting related metabolic pathways. The objective is to guide further investigations into the metabolic mechanisms underlying MM and encourage innovation in clinical diagnosis and treatment strategies for this disease.
{"title":"[Progress in metabolomics of multiple myeloma].","authors":"X Ye, Z X Sun, X Y Qu, L Fan","doi":"10.3760/cma.j.cn121090-20241205-00536","DOIUrl":"10.3760/cma.j.cn121090-20241205-00536","url":null,"abstract":"<p><p>Multiple myeloma (MM) is a malignant disease characterized by clonal plasma cell proliferation, with metabolic reprogramming. Abnormal metabolism of amino acids, glucose, lipids and nucleotides plays a crucial role in various pathological processes in MM, such as proliferation, metastasis, immune escaping and drug resistance. This review summarized the application of metabolomics technology in identifying biomarkers for MM and targeting related metabolic pathways. The objective is to guide further investigations into the metabolic mechanisms underlying MM and encourage innovation in clinical diagnosis and treatment strategies for this disease.</p>","PeriodicalId":24016,"journal":{"name":"Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi","volume":"46 10","pages":"980-984"},"PeriodicalIF":0.0,"publicationDate":"2025-10-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12665467/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145670126","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-10-14DOI: 10.3760/cma.j.cn121090-20250731-00356
J J Wu, J F Wang
Objective: Using the impact of chronic graft-versus-host disease (cGVHD) on overall survival after hematopoietic stem cell transplantation as an example, this study aims to introduces and critically appraises five statistical approaches for handling time-dependent events in survival analysis. Methods: This study was based on data from the Center for International Blood and Marrow Transplant Research (CIBMTR) GV18-03 study. A total of 4361 patients with acute myeloid leukemia or myelodysplastic syndrome were included, who were aged ≥40 years and had received an HLA 8/8 matched sibling donor transplant between 2008 and 2017. Five analytical approaches were used, treating cGVHD as: ① a baseline fixed covariate, ② a time-dependent covariate, ③ a time-dependent event via landmarking analysis, ④ a transitional event in a multi-state model, and ⑤ an exposure in the parametric g-formula. For each approach, we presented its core concept and results, and elaborated on its rationale, strengths, limitations, and applicable scenarios. Results: The five approaches showed significant differences in bias control, modeling flexibility, and clinical interpretability. Method 1 was prone to immortal time bias. Method 2 partially corrected this bias but was vulnerable to informative censoring and estimation instability due to insufficient sample sizes in the early-onset cGVHD group. Method 3 was straightforward but dependent on a predefined landmark time point and unable to account for cGVHD occurring after this point. Method 4 allowed for a comprehensive description of the impact of time-dependent events on survival prognosis by modeling dynamic clinical transitions. Method 5 could simultaneously handle both time-dependent events and confounders, making it suitable for estimating population-level effects of specific exposure strategies. Conclusion: Researchers should select the appropriate method based on their specific study objectives to ensure that the estimation of the impact of time-dependent events on outcomes is both statistically valid and clinically meaningful.
目的:以慢性移植物抗宿主病(chronic graft- anti -host disease, cGVHD)对造血干细胞移植后总生存率的影响为例,介绍并批判性评价在生存分析中处理时间依赖性事件的五种统计方法。方法:本研究基于国际血液和骨髓移植研究中心(CIBMTR) GV18-03研究的数据。共纳入4361例急性髓性白血病或骨髓增生异常综合征患者,年龄≥40岁,在2008年至2017年期间接受了HLA 8/8匹配的兄弟姐妹供体移植。采用五种分析方法,将cGVHD视为:①基线固定协变量,②时间相关协变量,③通过地标分析的时间相关事件,④多状态模型中的过渡事件,以及⑤参数g公式中的暴露。对于每种方法,我们都介绍了其核心概念和结果,并详细阐述了其基本原理、优势、局限性和适用场景。结果:五种方法在偏倚控制、建模灵活性和临床可解释性方面存在显著差异。方法一易出现不朽时间偏差。方法2部分纠正了这一偏差,但由于早发性cGVHD组的样本量不足,容易受到信息审查和估计不稳定的影响。方法3是直接的,但依赖于一个预定义的里程碑时间点,无法解释cGVHD发生在这一点之后。方法4允许通过模拟动态临床转变来全面描述时间依赖性事件对生存预后的影响。方法5可以同时处理时间相关事件和混杂因素,使其适合于估计特定暴露策略的人群水平效应。结论:研究者应根据其具体的研究目的选择合适的方法,以确保对时间相关事件对结果影响的估计在统计上有效且具有临床意义。
{"title":"[Methodological appraisal of statistical approaches to evaluating the impact of chronic graft-versus-host disease on mortality after hematopoietic stem cell transplantation].","authors":"J J Wu, J F Wang","doi":"10.3760/cma.j.cn121090-20250731-00356","DOIUrl":"10.3760/cma.j.cn121090-20250731-00356","url":null,"abstract":"<p><p><b>Objective:</b> Using the impact of chronic graft-versus-host disease (cGVHD) on overall survival after hematopoietic stem cell transplantation as an example, this study aims to introduces and critically appraises five statistical approaches for handling time-dependent events in survival analysis. <b>Methods:</b> This study was based on data from the Center for International Blood and Marrow Transplant Research (CIBMTR) GV18-03 study. A total of 4361 patients with acute myeloid leukemia or myelodysplastic syndrome were included, who were aged ≥40 years and had received an HLA 8/8 matched sibling donor transplant between 2008 and 2017. Five analytical approaches were used, treating cGVHD as: ① a baseline fixed covariate, ② a time-dependent covariate, ③ a time-dependent event via landmarking analysis, ④ a transitional event in a multi-state model, and ⑤ an exposure in the parametric g-formula. For each approach, we presented its core concept and results, and elaborated on its rationale, strengths, limitations, and applicable scenarios. <b>Results:</b> The five approaches showed significant differences in bias control, modeling flexibility, and clinical interpretability. Method 1 was prone to immortal time bias. Method 2 partially corrected this bias but was vulnerable to informative censoring and estimation instability due to insufficient sample sizes in the early-onset cGVHD group. Method 3 was straightforward but dependent on a predefined landmark time point and unable to account for cGVHD occurring after this point. Method 4 allowed for a comprehensive description of the impact of time-dependent events on survival prognosis by modeling dynamic clinical transitions. Method 5 could simultaneously handle both time-dependent events and confounders, making it suitable for estimating population-level effects of specific exposure strategies. <b>Conclusion:</b> Researchers should select the appropriate method based on their specific study objectives to ensure that the estimation of the impact of time-dependent events on outcomes is both statistically valid and clinically meaningful.</p>","PeriodicalId":24016,"journal":{"name":"Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi","volume":"46 10","pages":"905-913"},"PeriodicalIF":0.0,"publicationDate":"2025-10-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12665468/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145670171","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-10-14DOI: 10.3760/cma.j.cn121090-20241111-00443
H Ai, Q Wang, H F Wu, Q S Yin
<p><p><b>Objective:</b> To compare the efficacy and safety of two induction regimens, homoharringtonine plus venetoclax and azacitidine (VHA) versus venetoclax and azacitidine (VA) , for newly diagnosed acute myeloid leukemia (AML) patients who are elderly or ineligible for intensive chemotherapy. <b>Methods:</b> We retrospectively analyzed the clinical data of 59 newly diagnosed AML patients treated with the VHA or VA regimen at Zhengzhou University Affiliated Cancer Hospital from September 2018 to July 2021. The cohort included 25 males and 34 females, with a median age of 63 years. The overall response rate (ORR) , composite complete remission (CRc) rate [CR+CR with incomplete hematologic recovery (CRi) ], minimal residual disease (MRD) negativity rate, overall survival (OS) , relapse-free survival (RFS) , and adverse events were compared between the two groups. Survival was estimated by the Kaplan-Meier method, and prognostic factors were evaluated using univariable and multivariable Cox regression. <b>Results:</b> At the end of the treatment, the ORR was 88.4% (23/26) in the VHA group [21 CR, 2 partial remissions (PR) ] and 90.9% (30/33) in the VA group (25 CR, 5 PR) , with no significant difference between the groups (<i>P</i>=0.458) . The MRD negativity rates after one cycle of induction were 73.1% (19/26) in the VHA group and 60.6% (20/33) in the VA group, respectively (<i>P</i>=0.315) . In the high-risk subgroup, the composite remission rates after one cycle were 78.6% (11/14) with VHA and 50.0% (5/10) with VA (<i>P</i>=0.143) ; MRD negativity rates were 64.3% (9/14) and 20.0% (2/10) , respectively (<i>P</i>=0.032) . The main adverse events were myelosuppression, gastrointestinal reactions, and infections during neutropenia. Rates of grade 3-4 neutropenia and decreased hemoglobin were similar between groups, whereas grade 3-4 thrombocytopenia was more frequent with VHA than with VA (76.9% <i>vs</i> 45.5%, <i>P</i>=0.015) . With a median follow-up of 13 months (range, 1-59) , 1 year RFS was 69.9% (95% <i>CI</i>: 53.1%-92.2%) with VHA and 55.6% (95% <i>CI</i>: 40.1%-77.1%) with VA (<i>P</i>=0.305) . The 1 year OS rates were 91.7% (95% <i>CI</i>: 77.3%-100.0%) and 58.2% (95% <i>CI</i>: 41.7%-81.4%) , respectively (<i>P</i>=0.024) . Among high risk patients, 1 year RFS and OS were higher with VHA than with VA (RFS: 66.2% <i>vs</i> 37.5%, <i>P</i>=0.046; OS: 85.7% <i>vs</i> 48.0%, <i>P</i>=0.011) . Undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT) significantly improved RFS and OS (<i>P</i>=0.027 and 0.047, respectively) . On multivariable analysis, ELN risk classification and MRD negativity after the first cycle were independent prognostic factors for RFS. Treatment regimen (VHA <i>vs</i> VA) , MRD negativity after the first cycle, and receipt of transplantation were independent prognostic factors for OS. <b>Conclusion:</b> VHA provides clinical benefit in newly diagnosed AML patients who are unfit for intensive chemo
{"title":"[A preliminary analysis of the efficacy and safety of homoharringtonine, venetoclax, and azacitidine for newly diagnosed acute myeloid leukemia].","authors":"H Ai, Q Wang, H F Wu, Q S Yin","doi":"10.3760/cma.j.cn121090-20241111-00443","DOIUrl":"10.3760/cma.j.cn121090-20241111-00443","url":null,"abstract":"<p><p><b>Objective:</b> To compare the efficacy and safety of two induction regimens, homoharringtonine plus venetoclax and azacitidine (VHA) versus venetoclax and azacitidine (VA) , for newly diagnosed acute myeloid leukemia (AML) patients who are elderly or ineligible for intensive chemotherapy. <b>Methods:</b> We retrospectively analyzed the clinical data of 59 newly diagnosed AML patients treated with the VHA or VA regimen at Zhengzhou University Affiliated Cancer Hospital from September 2018 to July 2021. The cohort included 25 males and 34 females, with a median age of 63 years. The overall response rate (ORR) , composite complete remission (CRc) rate [CR+CR with incomplete hematologic recovery (CRi) ], minimal residual disease (MRD) negativity rate, overall survival (OS) , relapse-free survival (RFS) , and adverse events were compared between the two groups. Survival was estimated by the Kaplan-Meier method, and prognostic factors were evaluated using univariable and multivariable Cox regression. <b>Results:</b> At the end of the treatment, the ORR was 88.4% (23/26) in the VHA group [21 CR, 2 partial remissions (PR) ] and 90.9% (30/33) in the VA group (25 CR, 5 PR) , with no significant difference between the groups (<i>P</i>=0.458) . The MRD negativity rates after one cycle of induction were 73.1% (19/26) in the VHA group and 60.6% (20/33) in the VA group, respectively (<i>P</i>=0.315) . In the high-risk subgroup, the composite remission rates after one cycle were 78.6% (11/14) with VHA and 50.0% (5/10) with VA (<i>P</i>=0.143) ; MRD negativity rates were 64.3% (9/14) and 20.0% (2/10) , respectively (<i>P</i>=0.032) . The main adverse events were myelosuppression, gastrointestinal reactions, and infections during neutropenia. Rates of grade 3-4 neutropenia and decreased hemoglobin were similar between groups, whereas grade 3-4 thrombocytopenia was more frequent with VHA than with VA (76.9% <i>vs</i> 45.5%, <i>P</i>=0.015) . With a median follow-up of 13 months (range, 1-59) , 1 year RFS was 69.9% (95% <i>CI</i>: 53.1%-92.2%) with VHA and 55.6% (95% <i>CI</i>: 40.1%-77.1%) with VA (<i>P</i>=0.305) . The 1 year OS rates were 91.7% (95% <i>CI</i>: 77.3%-100.0%) and 58.2% (95% <i>CI</i>: 41.7%-81.4%) , respectively (<i>P</i>=0.024) . Among high risk patients, 1 year RFS and OS were higher with VHA than with VA (RFS: 66.2% <i>vs</i> 37.5%, <i>P</i>=0.046; OS: 85.7% <i>vs</i> 48.0%, <i>P</i>=0.011) . Undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT) significantly improved RFS and OS (<i>P</i>=0.027 and 0.047, respectively) . On multivariable analysis, ELN risk classification and MRD negativity after the first cycle were independent prognostic factors for RFS. Treatment regimen (VHA <i>vs</i> VA) , MRD negativity after the first cycle, and receipt of transplantation were independent prognostic factors for OS. <b>Conclusion:</b> VHA provides clinical benefit in newly diagnosed AML patients who are unfit for intensive chemo","PeriodicalId":24016,"journal":{"name":"Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi","volume":"46 10","pages":"937-942"},"PeriodicalIF":0.0,"publicationDate":"2025-10-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12665462/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145670001","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-10-14DOI: 10.3760/cma.j.cn121090-20250612-00273
Marginal zone lymphoma (MZL) is an indolent B-cell lymphoma arising from marginal zone B cells within lymphoid follicles, accounting for approximately 5%-15% of non-Hodgkin lymphomas. MZL is characterized by diagnostic challenges and diverse treatment options, making it a challenging subtype in clinical practice. In recent years, with the rapid progress in lymphoma research, the understanding of MZL has improved significantly worldwide. To further standardize the diagnosis and treatment of MZL in China, the Chinese Workshop of Indolent Lymphomas convened a panel of national experts. Based on thorough discussion and consensus, this guideline was developed to provide a reference for medical practitioners.
{"title":"[The guideline for the diagnosis and treatment of marginal zone lymphoma in China (2025)].","authors":"","doi":"10.3760/cma.j.cn121090-20250612-00273","DOIUrl":"10.3760/cma.j.cn121090-20250612-00273","url":null,"abstract":"<p><p>Marginal zone lymphoma (MZL) is an indolent B-cell lymphoma arising from marginal zone B cells within lymphoid follicles, accounting for approximately 5%-15% of non-Hodgkin lymphomas. MZL is characterized by diagnostic challenges and diverse treatment options, making it a challenging subtype in clinical practice. In recent years, with the rapid progress in lymphoma research, the understanding of MZL has improved significantly worldwide. To further standardize the diagnosis and treatment of MZL in China, the Chinese Workshop of Indolent Lymphomas convened a panel of national experts. Based on thorough discussion and consensus, this guideline was developed to provide a reference for medical practitioners.</p>","PeriodicalId":24016,"journal":{"name":"Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi","volume":"46 10","pages":"881-890"},"PeriodicalIF":0.0,"publicationDate":"2025-10-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12665474/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145670168","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-10-14DOI: 10.3760/cma.j.cn121090-20250225-00094
Y X Zhou, Y Y Wei, Z W Liu, C Yang, M Chen, B Han
This study retrospectively analyzed 12 patients with transfusion-dependent, non-severe aplastic anemia (TD-NSAA) refractory to cyclosporine A (CsA) , who were treated with lusutrombopag monotherapy. These patients either had a variety of chronic comorbidities or medication-related risks, or they were unresponsive to or intolerant of other thrombopoietin receptor agonists (TPO-RA) . The median treatment duration with lusutrombopag was 4 months (range: 3-11 months) , while the median follow-up period was 8 months (range: 6-11 months) . The overall response (OR) rates at months 3, 6, and the end of follow-up were 50.0%, 58.3%, and 50.0%, respectively, with a median time to OR of 2 months (range: 1-4 months) . Complete response (CR) rates were 8.3%, 16.7%, and 16.7% at the same time points, with a median time to CR of 4 months (range: 2-5 months) . Adverse events were all Grade 1, with an incidence rate of 25.0%. During follow-up, one patient experienced a loss of OR after discontinuing treatment, with a relapse rate of 14.3%; no clonal evolution or mortality was observed. These findings suggest that lusutrombopag is both effective and well-tolerated in CsA-refractory TD-NSAA patients and represents a promising therapeutic option for those with poor treatment tolerability.
{"title":"[Efficacy and safety of lusutrombopag monotherapy for cyclosporine A-refractory, transfusion-dependent non-severe aplastic anemia].","authors":"Y X Zhou, Y Y Wei, Z W Liu, C Yang, M Chen, B Han","doi":"10.3760/cma.j.cn121090-20250225-00094","DOIUrl":"10.3760/cma.j.cn121090-20250225-00094","url":null,"abstract":"<p><p>This study retrospectively analyzed 12 patients with transfusion-dependent, non-severe aplastic anemia (TD-NSAA) refractory to cyclosporine A (CsA) , who were treated with lusutrombopag monotherapy. These patients either had a variety of chronic comorbidities or medication-related risks, or they were unresponsive to or intolerant of other thrombopoietin receptor agonists (TPO-RA) . The median treatment duration with lusutrombopag was 4 months (range: 3-11 months) , while the median follow-up period was 8 months (range: 6-11 months) . The overall response (OR) rates at months 3, 6, and the end of follow-up were 50.0%, 58.3%, and 50.0%, respectively, with a median time to OR of 2 months (range: 1-4 months) . Complete response (CR) rates were 8.3%, 16.7%, and 16.7% at the same time points, with a median time to CR of 4 months (range: 2-5 months) . Adverse events were all Grade 1, with an incidence rate of 25.0%. During follow-up, one patient experienced a loss of OR after discontinuing treatment, with a relapse rate of 14.3%; no clonal evolution or mortality was observed. These findings suggest that lusutrombopag is both effective and well-tolerated in CsA-refractory TD-NSAA patients and represents a promising therapeutic option for those with poor treatment tolerability.</p>","PeriodicalId":24016,"journal":{"name":"Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi","volume":"46 10","pages":"963-966"},"PeriodicalIF":0.0,"publicationDate":"2025-10-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12665463/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145670104","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}