To compare the efficacy, safety, and pharmacoeconomic differences between Mecapeg-filgrastim and recombinant human granulocyte colony-stimulating factor （rhG-CSF） in mobilizing peripheral blood stem cells （PBSCs） from healthy donors. A total of 100 healthy donors who underwent PBSC mobilization at our center between January 2022 and June 2025 were included and assigned to the Mecapegfilgrastim group or the rhG-CSF group. Successful mobilization was defined as CD34+cells ≥2×106/kg, and optimal mobilization as ≥5×106/kg. The mobilization efficacy, adverse events, and medical costs between the two groups were compared. Outcomes of the corresponding recipients were evaluated, including the dose of infused cells, the time to hematopoietic recovery, and the incidence of acute graft-versus-host disease（aGVHD）. The mobilization success rate was 100% in both groups. The optimal mobilization rate （98.0% vs. 80.0%, P=0.004） and median number of mobilized CD34+ cells ［7.84（4.86-20.86） vs. 5.85（2.99-15.20）×106/kg, P<0.001］ in the mecapeg-filgrastim group were significantly higher than that in the rhG-CSF group, while the number of mononuclear cells was lower ［10.13（5.38-24.80） vs. 12.16（4.99-28.38） × 108/kg, P=0.049］. The engraftment rates of the two groups of recipients （88% vs. 90%） and aGVHD incidence rates （52.3% vs. 48.9%） showed no significant differences（P>0.05）. However, compared with the rhG-CSF group, the Mecapegfilgrastim group showed significantly shorter neutrophil （14 vs. 16 days） and platelet （13 vs. 22 days） engraftment time （both P<0.001）. The incidence and spectrum of adverse events were comparable between the two groups, with no statistically significant difference（32.0% vs.30.0%, P>0.05）. Additionally, compared with the rhG-CSF group, the Mecapegfilgrastim group had a shorter donor hospitalization length ［2（2-3） vs. 6（6-7）days］ and lower mobilization costs（both P<0.001）. In healthy donor PBSC mobilization, Mecapegfilgrastim is non-inferior to rhG-CSF, offers superior promotion of hematopoietic recovery in recipients, and provides significant advantages in reducing hospital stay and medical costs with a comparable safety profile. Mecapegfilgrastim thus represents a more cost-effective mobilization strategy. 硫培非格司亭与rhG-CSF在健康供者外周血造血 干细胞动员中的疗效与安全性对比研究. 探讨硫培非格司亭与重组人粒细胞集落刺激因子（rhG-CSF）在健康供者外周血造血干细胞（PBSC）动员中的疗效、安全性及药物经济学差异. 纳入2022年1月至2025年6月在本院行PBSC动员的100例健康供者，将其分为硫培非格司亭组与rhG-CSF组。设定成功动员标准为CD34+细胞≥2×106/kg，CD34+细胞≥5×106/kg为优良动员标准。比较两组供者的动员效果、不良反应与医疗费用，并评估相应受者的细胞输注量、造血重建时间及急性移植物抗宿主病（aGVHD）的发生率. 两组的动员成功率均为100%。硫培非格司亭组在优良动员率（98.0% vs. 80.0%, P=0.004）及动员的CD34+细胞数量中位数［7.84（4.86-20.86） vs. 5.85（2.99-15.20）×106/kg, P<0.001］方面均显著高于rhG-CSF组，而单个核细胞数低于rhG-CSF组［10.13（5.38-24.80） vs. 12.16（4.99-28.38）×108/kg, P=0.049］。两组受者的植入率（88% vs. 90%）及aGVHD发生率（52.3% vs. 48.9%）无显著差异（P>0.05）；但硫培非格司亭组受者的中性粒细胞（14 vs. 16 d）与血小板（13 vs. 22 d）植入时间较rhG-CSF组均显著缩短（均P<0.001）。两组的不良反应发生率（32.0% vs. 30.0%）及症状谱相似（P>0.05）。此外，硫培非格司亭组供者的住院时长［2（2-3） vs. 6（6-7） d］及动员总费用均显著低于rhG-CSF组（P<0.001）. 在健康供者PBSC动员中，硫培非格司亭的疗效不劣于rhG-CSF，且在促进受者造血重建方面更具优势，同时能缩短住院时间并降低医疗成本，安全性相当，是一种具有更高成本效益的动员方案.
To analyze and explore the clinical characteristics and prognostic factors of chronic myelomonocytic leukemia (CMML), a rare hematological malignancy, thereby providing insights for basic research and clinical management in hematology. Retrospective analysis was conducted on the clinical data of 64 newly diagnosed CMML patients from three hospitals between January 2018 and January 2025. Clinical features, treatment outcomes and prognosis were summarized. Kaplan-Meier survival analysis, Log-rank tests, and Cox proportional hazards regression models were employed to evaluate prognostic factors. A total of 64 patients were included: 36 males (56.25%) and 28 females (43.75%). The median age at onset was 64 years (range, 28-87). According to the French-American-British (FAB) classification, 19 cases were MD-CMML and 45 were MP-CMML. Based on the World Health Organization (WHO) classification,14 cases were CMML-0, 19 CMML-1, and 31 CMML-2. Bone marrow morphology revealed dysplasia in 46 cases: 19 with single-lineage, 16 with bilineage, and 11 with trilineage involvement. Bone marrow biopsy showed active hyperplasia in 37 cases, decreased hyperplasia in 27, and concurrent myelofibrosis in 18. Cytogenetic analysis was performed in 56 patients, with abnormalities detected in 7 (12.5%). Molecular testing was performed in 45 patients, and gene mutations were identified in 20 (44.4%). The primary first-line treatment for the included patients with CMML was chemotherapy, 17 patients received only symptomatic and supportive care. Among 47 patients with evaluable treatment responses, 23 achieved complete remission, 15 achieved partial remission, 8 had stable disease, and 1 had progressive disease. As of May 1, 2025, 37 patients (57.8%) were alive, 24 (37.5%) had died, and 3 (4.69%) were lost to follow-up. The median overall survival (OS) was 23 months. Low hemoglobin levels, lactate dehydrogenase (LDH) ≥250 U/L, and a peripheral blast count (PBC) ≥5% were significantly associated with inferior OS (P<0.05). PBC≥5% was identified as an independent risk factor affecting OS in CMML patients. CMML lacks distinctive clinical features and is often accompanied by pathological hematopoiesis and molecular biological abnormalities, with a poor prognosis. PBC≥5% is an independent risk factor for OS in patients with CMML. 慢性粒－单核细胞白血病的临床特征及预后分析. 分析与探讨慢性粒-单核细胞白血病（CMML）患者的临床特征及其预后因素，旨在为血液病学领域有关CMML的基础研究与临床诊疗提供借鉴和参考. 收集整理2018年1月至2025年1月来自国内3家三甲医院共64例CMML患者的数据资料，总结并分析CMML患者的临床特征、诊治经过以及预后等，同时利用Kaplan-Meier法、Log-rank检验与Cox比例风险回归模型等进行预后分析. 共纳入64例患者，男性36例（56.25%），女性28例（43.75%），中位发病年龄为64（28-87）岁。根据FAB分型，MD-CMML 19例，MP-CMML 45例；依照WHO分型，14例CMML-0，19例CMML-1，31例CMML-2。骨髓分析结果提示46例伴随病态造血（DBM），其中，19例为单系DBM，16 例两系DBM，11例存在三系DBM。骨髓活检提示37例增生活跃，27例增生减低，18例患者同时存在骨髓纤维化。共56例患者检测了染色体，7例（12.50％）存在细胞遗传学异常；共45例患者进行了分子生物学检测，20例（44.44％）患者存在基因突变。纳入本研究的CMML患者一线治疗以化疗为主，17例患者仅采用对症支持治疗，跟踪病程且可评估疗效的患者47例，其中，23例完全缓解，15例部分缓解，8例病情稳定，1例出现病情进展。随访截至2025年5月1日，64例CMML患者中，存活37例（57.81％），死亡24 例（37.50％），失访3例（4.69％），中位总生存期（OS）为23个月。此外，HGB减低、LDH异常升高、外周血原始细胞比例（PBC）≥5%与不良OS有关（P<0.05），而外周血PBC≥5%是影响CMML患者OS的独立危险因素. CMML临床缺乏特异性，常伴病态造血与分子生物学异常，预后较差，外周血PBC≥5%是CMML患者OS的独立危险因素.
To explore the genetic characteristics and clinical efficacy of pediatric acute myeloid leukemia (AML) with NUP98∷KDM5A fusion gene positive. The laboratory and clinical characteristics of 10 NUP98∷KDM5A-positive pediatric AML patients identified by transcriptome sequencing (RNA-seq) were retrospectively analyzed during the period from March 2018 to March 2024 at Hebei Yanda Ludaopei Hospital and Beijing Ludaopei Hospital. Survival curves were plotted using the Kaplan-Meier method, and the 1-year overall survival and cumulative recurrence rates were calculated. The median onset age of the 10 patients was 2(1-4) years, with a male to female ratio of 3∶7. All patients presented with thrombocytopenia, skin ecchymosis, and/or scattered petechiae as the main clinical manifestations. According to the FAB classification, 7 cases were diagnosed as AML-M7, 2 as AML-M5, and 1 as AML-M2. Cytogenetic analysis showed that 8 pediatric patients exhibited structural abnormalities involving chromosome 13 at initial diagnosis, relapse, or post-transplant relapse, and mainly manifested as del(13q). RNA-seq results showed that 9 patients had a fusion of NUP98 exon 13 with KDM5A exon 27, and 1 patient had a fusion of NUP98 exon 13 with KDM5A exon 25. Expression levels of MECOM and PRDM16 genes in the patient group were significantly higher than in the normal control group (P <0.05). Among co-occurring genetic mutations, JAK2 gene mutations exhibited the highest frequency of occurrence (40%). All patients underwent allogeneic hematopoietic stem cell transplantation (allo-HSCT), with 7 in complete remission (CR) and 3 in partial remission (PR)/non-remission (NR) before transplantation. The median follow-up time was 5.8 (2.5-23.7) months. During the follow-up period, 3 patients survived and 7 died, among which 6 died from relapse and 1 died from acute graft-versus-host disease of the gut post-transplantation. The median survival time post-transplantation was 7.8 months (95%CI : 0.8-14.8 months), the median relapse time was 5.5 months (95%CI : 0-11.7 months), the 1-year overall survival rate was 34.3%, and cumulative relapse rate was 55%. Pediatric AML with the NUP98∷KDM5A fusion gene is more common in the M7 subtype, predominantly in females, and is often associated with chromosome 13 abnormalities and a higher rate of JAK2 mutations. Allo-HSCT can partially improve the prognosis of pediatric AML positive for NUP98∷KDM5A, but the relapse rate is high, and relapse is a significant factor affecting patient survival. NUP98∷KDM5A融合基因阳性儿童急性髓系白血病的遗传学特征及临床疗效分析. 探讨NUP98∷KDM5A 融合基因阳性儿童急性髓系白血病（AML）的遗传学特征及临床疗效。. 回顾分析2018年3月至2024年3月在河北燕达陆道培医院和北京陆道培医院经转录组测序(RNA-seq)鉴定的10例NUP98∷KDM5A阳性儿童AML患者的实验室及临床特征，采用Kaplan-Meier法绘制生存曲线，计算总生存率与复发率。. 10例患者中位发病年龄2(1-4)岁，男∶女=3∶7。所有患者均以血小板减低、皮肤瘀斑和/或散在出血点为主要临床表现。根据FAB分型，7例诊断为AML-M7、2例AML-M5、1例AML-M2。染色体核型分析显示，8例患者在初诊、疾病复发或移植后复发时均累及13号染色体异常，主要表现为del(13q)。RNA-seq结果显示9例患者为NUP98 exon 13与KDM5A exon 27融合，1例为NUP98 exon 13与KDM5A exon 25融合。患者组MECOM、PRDM16基因表达量显著高于正常对照组（P < 0.05）。伴随基因突变中AK2基因突变发生率最高（40%）。所有患者均接受异基因造血干细胞移植(allo-HSCT)，移植前7例为完全缓解(CR)状态，3例为部分缓解(PR)/未缓解(NR)。中位随访时间5.8(2.5-23.7)个月。随访期间3例存活，7例死亡，其中6例因复发死亡，1例死于移植后肠道急性移植物抗宿主病。移植后中位生存时间为7.8个月(95%CI ：0.8-14.8个月)，中位复发时间为5.5个月(95%CI ：0-11.7个月)，1年总生存率为34.3%，1年累积复发率为55%。. 伴NUP98∷KDM5A融合基因的儿童AML多见于M7型，主要见于女性，易伴13号染色体异常及较高的JAK2突变率。Allo-HSCT可部分改善NUP98∷KDM5A阳性儿童AML的预后，但复发率较高，复发是影响患者生存的重要因素。.
Acute myeloid leukemia (AML) can be transformed by the malignant transformation of hematopoietic progenitor cells at different stages of normal myeloid cell differentiation and development, thus leading to infection, anemia, bleeding symptoms. It is a common highly heterogeneous hematologic malignancy, the incidence of which is on the rise year by year. The treatment of AML includes chemotherapy, hematopoietic stem cell transplantation and targeted therapy, etc. However, due to the highly heterogeneous nature of AML and the easy recurrence, the treatment is difficult and the cure rate is low. In recent years, combination therapy based on venetoclax has become one of the important research directions for the treatment of AML, and its efficacy has gradually received the attention of clinical researchers. This paper reviews the mechanism of action of venetoclax and its application in the current treatment of AML. Through an in-depth discussion of venetoclax and its combination therapy AML related clinical trials, the emergence of resistance mechanisms to venetoclax and potential strategies to overcome its resistance are summarized. 以维奈克拉为基础的联合治疗在急性髓系白血病中 的研究进展. 急性髓系白血病（AML）可以由正常髓系细胞分化发育过程中不同阶段的造血祖细胞恶性变转化而来，从而导致感染、贫血、出血等症状，是一种常见的高度异质性血液系统恶性肿瘤，发病率呈逐年上升趋势。治疗AML的方法包括化疗、造血干细胞移植和靶向治疗等，但由于AML的高度异质性和易复发等特点，治疗难度较大，治愈率较低。近年来，以维奈克拉为基础的联合治疗成为治疗AML的重要研究方向之一，其疗效逐渐受到临床研究者的关注和重视。本文综述了维奈克拉的作用机制、在当前AML治疗中的应用，通过深入讨论维奈克拉及其联合治疗AML的相关临床试验，对维奈克拉耐药机制的出现和克服其耐药的潜在策略进行了归纳总结.
To analyze a difficult to match blood recipient who tested negative for irregular antibody screening and positive for anti-"Mur" antibodies, and to screen the distribution frequency of Mur blood type antigens and anti-"Mur" antibodies in patients in Guiyang, Guizhou. Blood type serological tests were used to identify the blood type, screen and identify antibodies, determine antibody titers, and perform cross matching on the blood recipient. Mur blood group antigen and anti-"Mur" antibody were screened by microcolumn gel method. Anti-"Mur" (IgM titer 16, IgG titer 8) was detected in the plasma of the blood recipient. Blood with no agglutination in both the primary and secondary sides was selected. The blood recipient had no adverse reactions after transfusion, indicating effective transfusion. Among patients seeking medical care in the Guiyang area, 46 cases (6.62%, 46/695) were identified as Mur blood group antigen positive, and 4 cases (0.58%, 4/695) were anti-"Mur" antibody positive. Blind matching method can be used to screen for cross matched blood transfusions, but specific antibody identification should be carried in the future. The screening of Mur blood type antigens and anti-"Mur" antibodies has important clinical significance in the Guiyang area. To avoid the missed detection of irregular antibodies, antibody screening cells with positive Mur blood type antigens should be selected for irregular antibody screening. 抗-“Mur”疑难配血及贵州贵阳地区Mur血型抗原和抗-“Mur”抗体的筛查研究. 分析1例不规则抗体筛查阴性抗-“Mur”抗体阳性的疑难配血受血者，对贵州贵阳地区就诊患者进行Mur血型抗原和抗-“Mur”抗体分布频率筛查. 采用血型血清学试验对受血者进行血型鉴定、抗体筛查鉴定、抗体效价测定及交叉配血；采用微柱凝胶法筛查本院就诊患者Mur血型抗原和抗-“Mur”抗体. 受血者血浆中检测出抗-“Mur”，其中IgM型效价16，IgG型效价8；选择主侧和次侧均无凝集的血液，受血者输注后无不良反应，输血有效。在贵阳地区就诊患者中筛选出Mur血型抗原阳性46例（6.62%，46/695），抗-“Mur”抗体阳性4例（0.58%，4/695）。. 可用盲配法筛选交叉配血相合的血液输血，但后续应进行特异性抗体鉴定；Mur血型抗原和抗-“Mur”抗体筛查在贵阳地区具有重要的临床意义，为避免不规则抗体漏检，应选择Mur血型抗原阳性的抗筛细胞进行不规则抗体筛查工作.
To analyze the clinical characteristics of patients with multiple myeloma (MM) complicated with light chain amyloidosis (AL), particularly those with cardiac amyloidosis (CA), in order to provide evidence for early identification. Clinical data were retrospectively collected from 359 newly diagnosed MM patients at the First Affiliated Hospital of Soochow University from August 2017 to December 2023. Based on histopathological results, patients were grouped to compare the clinical characteristics between the multiple myeloma with amyloid light-chain (MM-AL) group and the multiple myeloma without amyloid light-chain (MM without AL) group, as well as between the cardiac involvement and non-cardiac involvement subgroups within the MM-AL cohort. MM-AL patients accounted for 19.5% (70/359), of whom 30.0% had cardiac involvement. Compared with the MM without AL group, the MM-AL group had a higher proportion of λ light chain type (65.7% vs. 45.0%), a higher proportion of frail patients (45.7% vs. 29.1%), a lower proportion of DS stage III (84.3% vs. 93.4%), fewer patients presenting with bone pain as the initial symptom (45.7% vs. 69.2%), and higher proportions of patients with heart failure (12.8% vs. 4.2%) and non-hypoproteinemia polyserositis (27.2% vs. 5.9%) (all P < 0.05). Electrocardiogram abnormalities (low voltage, pseudo-infarction) were observed only in the MM-AL group. Compared with those without cardiac involvement, MM-CA patients had a higher proportion of light chain type (47.6% vs. 18.4%), a higher proportion of congestive heart-failure at onset (33.3% vs. 4.1%), a higher incidence of major adverse cardiovascular events during treatment (33.3% vs. 2.0%), and significantly elevated levels of NT-proBNP and hs-TnT, as well as a higher incidence of electrocardiogram abnormalities (all P < 0.05). Multivariate analysis showed that non-hypoproteinemia polyserositis (OR =7.66, P < 0.001) and λ light chain type (OR =2.40, P =0.017) were independent risk factors for MM complicated with AL. In terms of cytogenetics, the proportion of high-risk cytogenetic abnormalities was lower in MM-CA patients compared with those without cardiac involvement (14.3% vs. 36.7%, P =0.047). Patients with MM complicated by AL present with distinct clinical and cytogenetic features. The presence of λ light chain type and non-hypoproteinemic polyserous effusions are independent risk factors. Electrocardiographic findings of low voltage and pseudoinfarction patterns are suggestive of early recognition of AL and cardiac involvement. 初诊多发性骨髓瘤合并轻链型淀粉样变性的临床特征分析. 分析多发性骨髓瘤（MM）合并轻链型淀粉样变性（AL）患者的临床特征，尤其是心脏淀粉样变（CA）患者，为早期识别提供依据。. 收集2017年8月至2023年12月在苏州大学第一附属医院新诊断的359例MM患者的临床资料，根据组织病理结果进行分组，比较MM-AL组与MM未合并AL组、MM-AL组中累及心脏与未累及心脏亚组的临床特征差异。. MM-AL患者占19.5%（70/359），其中30.0%的患者累及心脏。与MM未合并AL组相比，MM-AL组λ轻链型比例更高（65.7% vs. 45.0%）、虚弱人群比例更高（45.7% vs. 29.1%）、DS分期III期比例更低（84.3% vs. 93.4%），且以骨痛为首发症状的患者比例较低（45.7% vs. 69.2%），而合并心力衰竭（12.8% vs. 4.2%）及非低蛋白血症性多浆膜腔积液的比例更高（27.2% vs. 5.9%）（均P < 0.05）。心电图异常（低电压、假性心肌梗死）仅见于MM-AL患者。与未累及心脏者相比，MM-CA患者轻链型比例更高（47.6% vs. 18.4%），起病时充血性心力衰竭比例更高（33.3% vs. 4.1%），治疗期间主要心血管不良事件发生率更高（33.3% vs. 2.0%），且NT-proBNP、hs-TnT水平显著升高，心电图异常发生率更高（均P < 0.05）。多因素分析显示，非低蛋白血症性多浆膜腔积液（OR =7.66，P < 0.001）和λ轻链（OR =2.40，P =0.017）是MM合并AL的独立危险因素。细胞遗传学方面，MM-CA患者高危细胞遗传学异常比例低于未累及心脏者（14.3% vs. 36.7%，P =0.047）。. MM合并AL患者具有独特的临床及细胞遗传学特征，λ轻链与非低蛋白血症性多浆膜腔积液是其独立危险因素，心电图低电压和假性心肌梗死对早期识别AL和心脏受累具有提示意义。.
To investigate the gene mutations in tumor tissues of patients with primary gastrointestinal diffuse large B-cell lymphoma (PGI-DLBCL), and analyze its relationship with clinical features and prognosis. A total of 31 newly diagnosed PGI-DLBCL patients treated in the People's Hospital of Xinjiang Uygur Autonomous Region from March 2009 to March 2021 and 81 nodal DLBCL patients matching gender, age, and ethnic group during the same period were collected. The sequencing of patients' tumor tissues were targeted by a panel of 475 lymphoma-related genes. The differences of mutational profiles and biological signaling pathway between PGI-DLBCL and nodal DLBCL were analyzed. The relationship between mutated genes and age, lactate dehydrogenase (LDH) level, Lugano stage, IPI score, cell origin typing, overall survival (OS) and progression-free survival (PFS) of PGI-DLBCL patients were investigated. A total of 50 high frequency mutated genes (number of gene mutations ≥3, mutation frequency ≥10%) were detected in PGI-DLBCL. The mutation rates of GNA13, EZH2, and FBXO11 genes in PGI-DLBCL patients were significantly higher than those in nodal DLBCL patients. BTG2 and CD79B mutations were closely associated with high-risk of IPI scores and the elderly. MYD88 mutations were more easily detected in the elderly patients. And mutations of the P2RY8 and KMT2D gene were related to early stage of the disease and high LDH, respectively. Only GNA13 mutations were detected in GCB subtype, while KMT2C, IRF4 and ID3 mutations were most frequent in non-GCB subtype. Further studies found that patients with CARD11, FANCA and ID3 mutations showed lower 5-year OS rate (all P < 0.05), while ID3 and NFKBIE mutations were associated with poorer PFS (both P < 0.05). The results of multivariate survival analysis suggested that ID3 mutation was an independent adverse prognostic factor for OS (HR=6.213, 95%CI : 1.215-31.770, P =0.028) and PFS (HR=0.060, 95%CI : 0.012-0.307, P =0.001) in PGI-DLBCL patients. PGI-DLBCL has a characteristic gene mutation spectrum, which is different from the molecular mechanism of nodal DLBCL development. ID3 gene mutation is an independent prognostic factor for predicting poor prognosis of PGI-DLBCL. 应用二代测序技术分析胃肠道弥漫大B细胞淋巴瘤患者基因突变分布特点及其与预后关系. 探讨胃肠道弥漫大B细胞淋巴瘤（PGI-DLBCL）患者肿瘤组织中基因突变情况，分析其与临床特征及预后的相关性。. 收集2009年3月至2021年3月在新疆维吾尔自治区人民医院诊治的初诊PGI-DLBCL患者31例，及同期性别、年龄、民族相匹配的结内DLBCL患者81例，使用475个淋巴瘤相关基因组对患者肿瘤组织进行靶向测序，分析PGI-DLBCL及结内DLBCL患者突变图谱及生物信号转导通路差异，探讨PGI-DLBCL中突变基因与年龄、乳酸脱氢酶水平、Lugano分期、IPI评分、细胞起源分型及患者总生存期（OS）和无进展生存期（PFS）间的关系。. 在PGI-DLBCL中共检测到50个高频突变基因（基因突变个数≥3个，突变频率≥10%）。与结内DLBCL相比，PGI-DLBCL患者中 GNA13, EZH2以及 FBXO11基因突变率显著更高。BTG2和CD79B 基因突变与IPI评分高危及高龄密切相关，老年患者中更易检测到 MYD88基因突变，P2RY8、KMT2D基因突变分别与疾病早期和高乳酸脱氢酶相关。仅在GCB亚型中检测到 GNA13基因发生突变，而KMT2C、IRF4、ID3 基因在non-GCB亚型中突变更为频繁。进一步研究发现，CARD11、FANCA、ID3 基因突变的患者表现出更低的5年OS率（均P < 0.05）， ID3和NFKBIE基因突变与较差的PFS相关（均P < 0.05），多因素生存分析结果提示 ID3基因突变是影响PGI-DLBCL患者OS（HR=6.213，95%CI ：1.215-31.770，P =0.028）及PFS（HR=0.060，95%CI ：0.012-0.307，P =0.001）的独立不良预后因素。. PGI-DLBCL具有特征性的基因突变谱，与结内DLBCL发生发展分子机制存在差异。ID3 基因突变是预测PGI-DLBCL预后不良的独立预后因子。.
Acute myeloid leukemia (AML) is the most common hematological malignancies in adults. TP53, as a tumor suppressor gene, has a mutation rate of about 5%-10% in AML patients. In 2017, the European Leukemia Net first classified AML with TP53 mutations as an unfavorable risk group, highlighting its importance of its importance in predicting prognosis. Currently, the treatment for TP53-mutated AML mainly includes intensive chemotherapy, demethylating therapy, and allogeneic hematopoietic stem cell transplantation. In recent years, with the application of new treatment methods such as immunotherapy and targeted drugs, the survival rate of patients has been improved to some extent. Overall, the efficacy and prognosis of TP53-mutated AML are poor, but there is a certain heterogeneity in the efficacy and survival of these patients. This article reviews the main pathogenesis and the latest treatment progress of AML with TP53 gene mutations, aiming to provide reference for clinical diagnosis and treatment. 伴 TP53突变的急性髓系白血病的治疗进展. 急性髓系白血病（AML）是成人最常见的血液系统恶性肿瘤。 TP53作为一种肿瘤抑制基因，在AML患者中的突变率约为5%-10%。2017年欧洲白血病网首次将 TP53突变的AML归类为不利风险，突显了其对预测预后的重要 性。目前，针对 TP53突变AML的治疗主要包括强化化疗、去甲基化治疗以及异基因造血干细胞移植。近年来，随着免疫治疗和靶向药物等新型治疗手段的应用，患者的生存率得到了一定程度的提高。总体而言， TP53突变AML的疗效不佳、预后极差，但这类患者的疗效和生存存在一定的异质性。本文综述了伴有 TP53基因突变的AML的主要发病机制及最新治疗进展，旨在为临床诊疗提供参考.
To explore the correlation between CD269 expression patterns in multiple myeloma (MM) cells and the expression of other antigens and molecular cytogenetics. Flow cytometry was used to detect the expression level of CD269 in bone marrow plasma cells of 115 newly diagnosed MM patients. The patients were subsequently divided into CD269- group (<20%), CD269 partial expression group (20%-80%), and CD269 high expression group (>80%). The CD269 partial expression group and high expression group were combined as the CD269+ group. The immunophenotype of bone marrow plasma cells was analyzed by flow cytometry. Molecular cytogenetic analysis was performed using combined probe fluorescence in situ hybridization technology. There were 27 patients (23.5%) in the CD269- group, 42 patients (36.5%) in the CD269 partial expression group, and 46 patients (40.0%) in the CD269 high expression group. The expression rate of CD56 antigen was 72.7% in the CD269+ group, which was higher than 48.1% in the CD269- group (P <0.05). The expression rates of CD56 and CD117 antigen in the CD269 high expression group were 76.1% and 47.8%, respectively, which were higher than 69.0% and 16.7% in the CD269 partial expression group (both P <0.05). The CD56/CD117 co-expression rate in the CD269 high expression group was 39.1%, which was higher than 14.8% in the CD269- group and 14.3% in the CD269 partial expression group (both P <0.05). The positive rate of IgH/CCND1 gene fusion in the CD269- group was 63.0%, which was higher than 31.0% in the CD269 partial expression group, 19.6% in the CD269 high expression group, and 25.0% in the CD269+ group (all P <0.05). The IgH deletion rate was 13.0% in the CD269 high expression group, while no IgH deletion was observed in the CD269 partial expression group, with a statistically significant difference between the two groups (P <0.05). The expression of CD269 on bone marrow plasma cells in MM patients is significantly correlated with the expression of CD56 and CD117, as well as IgH/CCND1 gene fusion and IgH deletion. The high expression of CD269 is associated with better prognosis related biological indicators. 多发性骨髓瘤细胞CD269表达模式与抗原表达和分子细胞遗传学异常的相关性. 探索多发性骨髓瘤（MM）细胞CD269表达模式与其他抗原表达和分子细胞遗传学的相关性。. 采用流式细胞术检测115例初诊MM患者骨髓浆细胞CD269表达水平，并据此将患者分为CD269阴性组（<20%）、部分表达组（20%-80%）和高表达组（>80%），部分表达和高表达之和为阳性组。流式细胞术检测患者骨髓浆细胞免疫表型，组合探针荧光原位杂交技术进行分子细胞遗传学分析。. CD269阴性组患者27例（23.5%），部分表达组患者42例（36.5%），高表达组患者46例（40.0%）。CD269阳性表达组患者CD56抗原的表达率为72.7%，高于CD269阴性表达组的48.1%（P <0.05）。CD269高表达组患者CD56抗原和CD117抗原的表达率分别为76.1%和47.8%，高于CD269部分表达组患者的69.0%和16.7%（均P <0.05）。CD269高表达组患者CD56/CD117共表达率为39.1%，高于CD269阴性表达组的14.8%及部分表达组的14.3%（均P <0.05）。CD269阴性组 IgH/CCND1阳性率为63.0%，高于CD269部分表达组的31.0%、高表达组的19.6%和阳性组的25.0%（均P <0.05）。CD269高表达组IgH 缺失率为13.0%，而CD269部分表达组未见IgH 缺失，两组比较差异有统计学意义（P <0.05）。. MM患者骨髓浆细胞CD269的表达与CD56、CD117的表达以及 IgH/CCND1基因融合、IgH 缺失有显著的相关性，CD269高表达与预后较好的生物学指标相关。.
Chimeric antigen receptor (CAR) T cell immunotherapy has achieved significant clinical efficacy in lymphatic malignancies, and CAR-T therapy for acute myeloid leukemia (AML) has become a research hotspot in recent years. At present, there is no CAR-T target as specific and effective as CD19 in lymphatic system tumors in AML. However, there are more and more new single target or modified CAR-T therapies for AML.The research on combination targeting (dual target) for AML has improved the long-term control problem of AML that cannot be achieved by single spectrum targeted CAR-T in the past. Both single target and dual target CAR-T can improve efficacy and reduce toxicity. This article reviews the latest research progress of CAR-T cell therapy in AML treatment. 嵌合抗原受体T细胞治疗急性髓系白血病研究进展. 嵌合抗原受体（CAR）T细胞免疫治疗在淋巴系统恶性肿瘤中已取得非常可观的临床疗效，急性髓系白血病（AML）的CAR-T治疗是近年来的研究热点。目前AML中尚无如淋巴系统肿瘤中CD19一样特异且有效的CAR-T靶点，但是AML中诸多新的单靶点或既往靶点改造后的CAR-T治疗研究越来越多，而针对AML进行组合靶向（双靶点）的研究改善了既往单一谱系靶向CAR-T无法实现的对AML的长期控制问题，单靶点及双靶点CAR-T均可提高疗效、减轻毒性。本文就CAR-T细胞疗法在AML治疗领域的最新研究进展作一综述.
To investigate the effect of miR-100-5p on the biological behaviors of pediatric acute myeloid leukemia (AML) cells by targeting insulin-like growth factor 1 receptor (IGF1R ). QRT-PCR was used to detect the expression levels of miR-100-5p and IGF1R mRNA in AML cell lines (THP-1, TF-1a, HL-60) and serum of children with AML. The dual-luciferase reporter gene assay was applied to verify the targeting relationship between miR-100-5p and IGF1R . THP-1 cells were assigned into Control group, miR-NC group, miR-100-5p mimics group, miR-100-5p mimics+pcDNA group, and miR-100-5p mimics+IGF1R group. The colony formation assay, scratch healing assay, and transwell assay were used to detect the proliferation, migration, and invasion abilities of THP-1 cells in each group, and Western blot assay was used to detect the protein expression of IGF1R, PCNA, MMP-2, MMP-9, E-cadherin, N-cadherin, and Vimentin. The expression level of IGF1R mRNA was significantly increased, while the expression level of miR-100-5p was significantly decreased in both the serum of pediatric AML patients and THP-1 cells (P < 0.001). Analysis via the StarBase database predicted the presence of potential binding sites between miR-100-5p and IGF1R . Compared with the miR-NC group, in the miR-100-5p mimics group, the number of colonies formed, scratch healing rate, and number of invasive cells of THP-1 cells were significantly decreased; the protein expression levels of IGF1R, PCNA, MMP-2, MMP-9, N-cadherin, and Vimentin were significantly decreased, while the expression level of miR-100-5p and the protein expression level of E-cadherin were significantly increased (all P < 0.05). Compared with the miR-100-5p mimics+pcDNA group, in the miR-100-5p mimics+IGF1R group, the number of colonies formed, scratch healing rate, and number of invasive cells were significantly increased; the protein expression levels of IGF1R, PCNA, MMP-2, MMP-9, N-cadherin, and Vimentin were significantly increased, while the protein expression level of E-cadherin was significantly decreased (all P < 0.05). Overexpression of miR-100-5p can targetedly inhibit IGF1R , thereby suppressing the proliferation, migration, invasion, and epithelial-mesenchymal transition (EMT) of AML cells. miR-100-5p靶向IGF1R 对小儿急性髓系白血病细胞生物学行为的影响. 探究miR-100-5p靶向胰岛素样生长因子1受体（IGF1R ）对小儿急性髓系白血病（AML）细胞生物学行为的影响。. 采用qRT-PCR法检测AML细胞系（THP-1、TF-1a、HL-60）和AML患儿血清中miR-100-5p、IGF1R mRNA表达水平；采用双荧光素酶报告基因实验验证miR-100-5p与IGF1R 的靶向关系。将THP-1细胞分为Control组、miR-NC组、miR-100-5p mimics组、miR-100-5p mimics+pcDNA组、miR-100-5p mimics+IGF1R组。采用平板克隆、划痕愈合、Transwell实验检测各组THP-1细胞的增殖、迁移、侵袭能力，采用免疫印迹实验检测IGF1R、PCNA、MMP-2、MMP-9、E-cadherin、N-cadherin、Vimentin蛋白表达。. AML患儿血清以及THP-1细胞中IGF1R mRNA表达水平明显升高，miR-100-5p表达水平明显降低（P < 0.001）。通过StarBase数据库分析，预测miR-100-5p与IGF1R 之间存在靶向结合位点。与miR-NC组相比，miR-100-5p mimics组THP-1细胞的克隆形成数、划痕愈合率、侵袭细胞数目明显降低，IGF1R、PCNA、MMP-2、MMP-9、N-cadherin、Vimentin蛋白表达水平明显下降，miR-100-5p、E-cadherin蛋白表达水平明显上升（均P < 0.05）；与miR-100-5p mimics+pcDNA组相比，miR-100-5p mimics+IGF1R组克隆形成数、划痕愈合率、侵袭细胞数目明显升高，IGF1R、PCNA、MMP-2、MMP-9、N-cadherin、Vimentin蛋白表达水平明显升高，E-cadherin表达水平明显降低（均P < 0.05）。. 过表达miR-100-5p能够靶向抑制IGF1R ，进而抑制AML细胞增殖、迁移、侵袭和上皮-间质转化（EMT）。.
To establish a doxorubicin (DOX)-resistant acute myeloid leukemia (AML) cell line and explore the mechanisms of its drug resistance. A DOX-resistant THP1 cell line (THP1-Rdox) was established using a low-dose, concentration-gradient intermittent induction method. The resistance effect was evaluated by calculating the resistance index (RI). The expression levels of resistance-related proteins ［P-glycoprotein (P-gp) and lung resistance-related protein (LRP)］ and cell cycle-related proteins (Cyclin A2, Cyclin B1 and Cyclin D1) were examined by Western blot. Intracellular DOX accumulation was observed using confocal laser scanning microscopy. Apoptosis rate and cell cycle distribution were analyzed by flow cytometry. RNA sequencing (RNA-seq) was performed to compare the differential gene expression profiles between DOX-sensitive parental THP1 cells and DOX-resistant THP1-Rdox cells. The DOX-resistant cell line THP1-Rdox was successfully established. The THP1-Rdox cells could stably proliferate at a DOX concentration of 500 ng/ml with a resistance index as high as 198.7. The THP1-Rdox cells exhibited cross-resistance to homoharringtonine (HHT) and paclitaxel (PTX) but no significant resistance to cytarabine (Ara-C). Compared with parental THP1 cells, the expression levels of drug resistance-related proteins P-gp and LRP in THP1-Rdox cells were significantly upregulated. Additionally, THP1-Rdox cells showed decreased uptake and increased efflux of DOX. Notably, after treatment with verapamil (Ver), a specific inhibitor of P-gp, intracellular DOX accumulation was significantly increased in THP1-Rdox cells. Compared with parental THP1 cells, the THP1-Rdox cells exhibited a significantly decreased apoptosis rate, a reduced proportion of cells in the S phase, an increased proportion of cells in the G1 phase, and a marked upregulation in the expression level of the cell cycle regulatory protein Cyclin D1. RNA-seq analysis showed that among the differentially expressed genes between THP1-Rdox and parental THP1 cells, the top 10 most significantly upregulated genes in THP1-Rdox cells were ABCB1, HNRNPA1P9, SEMA3E, MICB, JAML, FGL2, VSIG1, CLEC1B, DLGAP1-AS3, and HOOK1; the top 10 most significantly downregulated genes were MAGEB2, NPTX2, CGREF1, CDO1, GBP5, ZNF595, ZNF630, DTX3, KLHL4 and ZBED6CL. Low-dose, concentration-gradient intermittent induction method can successfully establish a DOX-resistant AML cell line, THP1-Rdox. The drug resistance of this cell line is likely attributed to enhanced drug efflux mediated by the elevated expression of P-gp and LRP, as well as cell cycle dysregulation resulting from the upregulation of Cyclin D1 protein. 急性髓系白血病多柔比星耐药细胞株的建立及其耐药机制研究. 构建耐多柔比星（DOX）的急性髓系白血病（AML）细胞株，并对其耐药机制进行初步探讨。. 采用小剂量DOX浓度递增间歇诱导法建立DOX耐药的THP1细胞株（THP1-Rdox），通过计算耐药指数（RI）评估细胞的耐药效果；采用Western blot法检测耐药相关蛋白P-gp（P-糖蛋白）、LRP（肺耐药相关蛋白）以及细胞周期相关蛋白Cyclin A2、Cyclin B1、Cyclin D1的表达水平；利用激光共聚焦显微镜观察DOX在细胞内的蓄积情况；通过流式细胞术检测细胞凋亡率及细胞周期变化；采用RNA测序技术分析DOX敏感的亲本THP1细胞和耐药的THP1-Rdox细胞之间的差异基因表达谱。. 成功构建了DOX耐药细胞株THP1-Rdox，可在500 ng/ml DOX浓度下稳定增殖，耐药指数高达198.7，且对高三尖杉酯碱、紫杉醇表现出交叉耐药性，但对阿糖胞苷无明显耐药性。与亲本THP1细胞相比，THP1-Rdox细胞中耐药相关蛋白P-gp和LRP蛋白表达水平明显上调；对DOX的摄取减少而外排增多；经P-gp特异性抑制剂维拉帕米处理后，THP1-Rdox细胞内DOX蓄积量明显增加。与亲本THP1细胞相比，THP1-Rdox细胞凋亡率明显下降，S期细胞比例减少，G1期细胞比例升高，且细胞周期调控蛋白Cyclin D1表达水平明显增加。RNA测序分析显示，THP1-Rdox与亲本细胞间差异表达基因中，上调最为显著的前10个基因分别为ABCB1、HNRNPA1P9、SEMA3E、MICB、JAML、FGL2、VSIG1、CLEC1B、DLGAP1-AS3、HOOK1；下调最为显著的前10个基因分别为MAGEB2、NPTX2、CGREF1、CDO1、GBP5、ZNF595、ZNF630、DTX3、KLHL4、ZBED6CL. 采用小剂量DOX浓度递增间歇诱导法可成功构建耐DOX的AML细胞株THP1-Rdox，该细胞株的耐药可能与P-gp、LRP高表达介导的药物外排增强，以及Cyclin D1蛋白上调导致的细胞周期紊乱相关。.
To validate the predictive ability of the International Myeloma Working Group (IMWG) venous thromboembolism (VTE) scoring system, the IMPEDE VTE scoring system, and the SAVED scoring system for VTE in Chinese multiple myeloma (MM) patients. A total of 415 patients with MM who visited The First Affiliated Hospital of Soochow University from January 1, 2019 to June 30, 2023 were included in the research cohort, and a retrospective analysis of their data was conducted. These patients were scored according to the IMWG, IMPEDE, and SAVED thrombosis risk models, respectively, and the predictive value of these three scoring models for VTE events was evaluated. The cumulative incidence of VTE events was 7.0% in the 415 patients. Both the IMWG score and the IMPEDE score demonstrated predictive value for VTE events. Each 1-point increase in the IMWG score was significantly associated with VTE events after treatment initiation (HR=3.41, 95%CI : 2.48-4.69, P < 0.001). Similarly, each 1-point increase in the IMPEDE score was also significantly associated with VTE events after treatment initiation (HR=2.46, 95%CI : 1.95-3.10, P < 0.001). In contrast, the SAVED score did not show significant predi- ctive value for VTE events in Chinese MM patients (P =0.693). However, according to the IMWG risk stratification, the vast majority of patients (97.3%) were classified into the high-risk group; in contrast, only 1 case (0.2%) was categorized into the high-risk group based on the IMPEDE risk stratification, indicating that these two risk stratification criteria are not suitable for Chinese patients. Therefore, patients were regrouped according to the IMWG score: ≤2 points as low-risk (163 cases), 3-4 points as intermediate-risk (214 cases), and ≥5 points as high-risk (38 cases); The 6-month cumulative incidence of VTE events in the high-risk group was 36.8%, which was significantly higher than that in the intermediate- and low-risk groups (P < 0.001, log-rank test). Patients were regrouped according to the IMPEDE score: ≤3 points as low-risk (265 cases), 4-5 points as intermediate-risk (131 cases), and ≥6 points as high-risk (19 cases); The 6-month cumulative incidence of VTE events in the high-risk group was 42.1%, which was significantly higher than that in the intermediate- and low-risk groups (P < 0.001, log-rank test). The IMWG score and the IMPEDE score have predictive value for VTE events in Chinese patients with MM, but restratification of patients based on these scores is required, while the SAVED score is not applicable to Chinese MM patients. IMWG、IMPEDE、SAVED血栓风险分层对中国多发性骨髓瘤患者静脉血栓栓塞症的预测能力验证. 验证国际骨髓瘤工作组（IMWG）静脉血栓栓塞（VTE)评分系统、IMPEDE VTE评分系统和SAVED评分系统对中国多发性骨髓瘤（MM）患者VTE的预测能力。. 纳入2019年1月1日至2023年6月30日于苏州大学附属第一医院就诊的415例MM患者作为研究对象，对患者资料进行回顾性分析。将这些患者分别根据IMWG、IMPEDE、SAVED血栓风险模型进行评分，并评估这3个评分模型对VTE事件的预测价值。. 415例患者VTE事件累积发生率为7.0％。IMWG评分与IMPEDE评分对于VTE事件有预测作用，IMWG评分每增加1分，与治疗开始后发生VTE事件显著相关（HR =3.41，95%CI ：2.48-4.69，P <0.001）；IMPEDE评分每增加1分，也与治疗开始后发生VTE事件显著相关（ HR =2.46，95%CI ：1.95-3.10，P <0.001）；SAVED评分对中国MM患者的VTE事件无显著预测意义（P =0.693）。然而，按IMWG风险分层，绝大部分(97.3%)患者被分为高危组；按IMPEDE风险分层，仅1例（0.2%）患者被分为高危组，这两种风险分层标准不适合中国患者。因此，将患者按IMWG评分重新分组，≤2分为低危（163例），3-4分为中危（214例），≥5分为高危（38例），高危组患者6个月VTE事件累计发生率为30.8％，明显高于中危组和低危组（P <0.001，log-rank）；将患者按IMPEDE评分重新分组，≤3分为低危（265例），4-5分为中危（131例），≥6分为高危（19例），高危组患者6个月VTE事件累积发生率为42.1％，明显高于中危组和低危组（P <0.001，log-rank）。. IMWG评分与IMPEDE评分对于中国MM患者VTE事件有预测作用，但需重新进行分组，而SAVED评分不适用于中国MM患者。.
Acute myeloid leukemia (AML) is a highly heterogeneous disease. This heterogeneity often leads to treatment failure or unsustainable efficacy, and high relapse rates as well as short progression-free survival (PFS) are closely associated with poor prognosis. Cyclin-dependent kinases (CDKs) play a central role in cell cycle regulation, transcription regulation, and metabolic processes, and their aberrant expression or dysfunction is considered as one of the key drivers of AML progression. Recent studies have increasingly focused on CDK inhibitors, aiming to address drug resistance and improve prognosis. Although some CDK inhibitors have shown promising anti-AML potential, challenges such as off-target effects and systemic toxicity remain daunting. This review systematically summarized the research progress of CDK inhibitors in the treatment of AML, with a particular focus on the results of preclinical studies and clinical trials targeting CDKs in AML, such as CDK2, CDK4/6, CDK7, and CDK9. In addition, the limitations of current therapeutic applications of CDK inhibitors were discussed, and potential strategies to overcome these challenges were explored, aiming to provide a reference for optimizing AML treatment regimens. CDK抑制剂在急性髓系白血病中的研究进展. 急性髓系白血病（AML）因其高度异质性而易导致治疗失败或疗效难以维持，且高复发率和较短的无进展生存时间与不良预后密切相关。细胞周期蛋白依赖性激酶（CDK）在细胞周期调控、转录调节和代谢过程中发挥核心作用，其异常表达或功能紊乱被认为是AML发生和进展的关键驱动因素之一。近年来，越来越多的研究聚焦于CDK抑制剂，试图通过解决治疗耐药问题进一步改善预后。尽管部分CDK抑制剂显示出显著的抗AML潜力，但目前仍面临诸多挑战，如脱靶效应显著和多系统毒性等问题。本文系统综述了CDK抑制剂在AML治疗领域的研究进展，重点讨论了CDK2、CDK4/6、CDK7及CDK9等靶点在AML中的基础研究及临床试验结果，探讨了CDK抑制剂在当前AML治疗中的应用局限性与潜在策略，旨在为优化AML的治疗方案提供参考。.
To explore the infection characteristics and survival status of lymphoma patients who received intravenous immunoglobulin (IVIG) after autologous hematopoietic stem cell transplantation (AHSCT). The clinical data of 121 lymphoma patients who underwent AHSCT from September 2019 to September 2023 were retrospectively analyzed. A Cox proportional hazards model was used to identify risk factors for post-transplant infection-free survival. The patients were divided into IVIG and non-IVIG group according to whether they received IVIG after transplantation, and their infection and survival outcomes were compared. Within 1 year after transplantation, bacterial, viral, and fungal infections occurred in 37 (30.58%), 18 (14.88%), and 3 (2.48%) of the 121 lymphoma patients, respectively. The 1-year overall infection rate after transplantation was 38.1% in the IVIG group (42 patients) and 44.3% in the non-IVIG group (79 patients). In the IVIG group, median infection time was +55 (9-233) days, with 7 cases of early infection, 5 cases of blood stream infection, and 6 cases of respiratory tract infection. In the non-IVIG group, median infection time was +8 (4-122) days, with 27 cases of early infection, 20 cases of bloodstream infection, and 4 cases of respiratory tract infection. IVIG group had fewer early and bloodstream infections, more respiratory tract infections, and a delayed median infection time compared to non-IVIG group. Multivariate Cox regression analysis indicated that pre-transplant serum IgG< 7 g/L, graft MNC< 8.1×108/kg and CD34+ cells≤2.8×106/kg served as independent risk factors for infection-free duration in AHSCT-treated lymphoma patients. The 3-year progression-free survival rates of the IVIG group and non-IVIG group were 52.3% and 48.4%, respectively, and 3-year overall survival rates were 79.4% and 83.0%. At 1 month post-transplant, 69 patients showed a decline in immunoglobulin levels, and 12 patients developed severe hypogamaglobulinemia (HG) (IgG< 4 g/L), with 4 cases (15.4%) in IVIG group and 8 cases (18.6%) in non-IVIG group. One month after transplantation, patients with serum IgG< 4 g/L had a significantly lower survival rate than those with IgG≥4 g/L (55.0% vs. 83.0%). In lymphoma patients, post-AHSCT early stage sees mainly bacterial infections. Some develop severe HG. The utilization of IVIG is capable of reducing the incidence of early post-transplant infections and severe HG. 淋巴瘤患者自体造血干细胞移植后应用静脉注射免疫球蛋白的感染特征及生存情况. 探讨淋巴瘤患者自体造血干细胞移植（AHSCT）后应用静脉注射免疫球蛋白（IVIG）的感染特征及生存情况。. 回顾性分析2019年9月至2023年9月121例行AHSCT的淋巴瘤患者的临床资料，采用Cox比例风险模型分析影响移植后无感染持续时间的危险因素。根据移植后是否使用IVIG将患者分为IVIG组和non-IVIG组，对比分析两组患者的感染和生存情况。. 121例淋巴瘤患者移植后1年内37例（30.58%）发生细菌感染，18例 （14.88%）发生病毒感染，3例（2.48%）发生真菌感染。IVIG组42例患者与non-IVIG组79例患者移植后1年内总感染发生率分别为38.1%、44.3%。IVIG组患者中位感染时间+55（9-233）d，早期感染7例，血流感染5例，呼吸道感染6例；non-IVIG组患者中位感染时间为+8（4-122）d，早期感染27例，血流感染20例，呼吸道感染4例。与non-IVIG组相比，IVIG组早期感染较少，血流感染减少，呼吸道感染增多，感染中位时间延迟。多因素Cox回归分析结果显示，移植前血清IgG< 7 g/L、移植物MNC计数< 8.1×108/kg、CD34+细胞计数≤2.8×106/kg可作为影响AHSCT治疗淋巴瘤患者无感染持续时间的独立危险因素。IVIG组与non-IVIG组患者3年PFS率分别为52.3%、48.4%，3年OS率分别为79.4%、83.0%。移植后1个月，69例患者免疫球蛋白水平出现不同程度下降，12例患者出现重度低丙种球蛋白血症（IgG< 4 g/L），IVIG组与non-IVIG组分别为4例（15.4%）、8例（18.6%）。移植后1个月血清免疫球蛋白IgG< 4 g/L组较IgG≥4 g/L组患者3年OS率显著降低（55.0% vs. 83.0%）。. 淋巴瘤患者AHSCT后早期以细菌感染为主，部分患者出现重度低丙种球蛋白血症。应用IVIG有助于降低移植后早期感染以及重度低丙种球蛋白血症发生率。.
To retrospectively analyze the early death of patients with newly diagnosed multiple myeloma (NDMM) treated with daratumumab, build a risk warning model and verify its clinical decision-making benefits. The clinical data of 112 NDMM patients treated with daratumumab combination therapy in Tangshan Gongren Hospital from June 2018 to June 2022 were retrospectively collected as the training set, and the clinical data of 78 NDMM patients who received daratumumab combination therapy in the same period were collected as the validation set. According to whether early death occurred during regular follow-up (OS <24 months), the patients were divided into early death group (26 cases) and non-early death group (86 cases). The differences of clinical data between the two groups were analyzed, and Kaplan-Meier survival curves were used to analyze the survival difference of patients with different efficacy. Univariate and multivariate Cox regression analysis were used to analyze the independent risk factors affecting early death of NDMM patients treated with daratumumab. A warning nomogram model for the risk of early death was established, and the predictive performance was analyzed by receiver operating characteristic (ROC) curve and verified internally. According to whether the efficacy of daratumumab treatment achieved partial response (PR), the patients were divided into <PR group (32 cases) and ≥PR group (80 cases). Kaplan-Meier analysis found that the median OS of both groups were not reached, while the OS of patients with efficacy ≥PR was significantly longer than that of patients with efficacy <PR (log-rank χ2=14.225, P <0.001). Multivariate Cox regression analysis showed that older age, R-ISS stage Ⅲ, elevated hs-CRP, and efficacy <PR were independent risk factors for early death in NDMM patients (all P <0.05), and a nomogram model for early death risk in NDMM patients was constructed. ROC analysis and DeLong test showed that the AUC of the nomogram model was 0.894(95%CI : 0.828-0.959), which was higher than that of each individual model, and the differences were statistically significant (all P <0.05). Internal and external validation showed that the nomogram model was stable and had a positive net benefit. The OS of NDMM patients who did not reach PR after daratumumab treatment can be affected. Daratumumab treatment early death risk warning model for NDMM has good efficacy, and can be targeted at high-risk population for intensive treatment to improve prognosis. 达雷妥尤单抗治疗新诊断多发性骨髓瘤患者早期死亡风险预警多模型研究及临床决策分析. 分析达雷妥尤单抗治疗新诊断多发性骨髓瘤（NDMM）患者早期死亡情况，构建发生风险预警模型并验证其临床决策效益。. 回顾性收集2018年6月至2022年6月于唐山市工人医院接受含达雷妥尤单抗联合方案治疗的112例NDMM患者临床资料，设为训练集；收集同期接受含达雷妥尤单抗联合方案治疗的78例NDMM患者临床资料，作为验证集。依据定期随访中是否发生早期死亡（总生存期OS <24个月），将患者划分为发生早期死亡组（26例）与未发生早期死亡组（86例）。对两组患者临床相关资料行差异性分析，Kaplan-Meier生存曲线分析不同疗效患者生存期差异。单因素和多因素Cox回归分析NDMM达雷妥尤单抗治疗早期死亡的独立危险因素。建立早期死亡发生风险预警列线图模型，通过ROC曲线分析预测效能并进行内部验证。. 根据治疗疗效是否达到部分缓解（PR），将所有患者分为<PR组（32例）与≥PR组（80例）。Kaplan-Meier分析结果显示，两组患者的中位OS均未达到，而≥PR患者OS明显长于<PR患者（log-rank χ2=14.225，P <0.001）。多因素Cox回归分析结果显示，年龄越大、R-ISS分期Ⅲ期、hs-CRP升高、疗效未达到PR为NDMM早期死亡的独立危险因素（均P <0.05），并构建NDMM患者早期死亡风险预警列线图模型。ROC分析和DeLong法检验结果显示，列线图模型的AUC值为0.894(95%CI :0.828-0.959)，高于各单个变量的AUC，比较差异有统计学意义（均P <0.05）。内部及外部验证结果显示，该列线图模型较为稳定，且有正向净收益率。. 达雷妥尤单抗治疗未达PR的NDMM可影响患者OS。NDMM达雷妥尤单抗治疗早期死亡风险预警模型效能良好，可针对高风险人群进行强化治疗以改善预后。.
To analyze the expression level of heterogeneous nuclear ribonucleoprotein U (hnRNP U) and its correlation with prognosis in patients with multiple myeloma (MM), and explore the functional role as well as molecular mechanism of hnRNP U, thereby providing a theoretical basis for development of hnRNP U as a novel therapeutic target for MM. Based on Gene Expression Omnibus (GEO) database, the expression of hnRNP U in plasma cell diseases and healthy controls was compared. Based on the GSE9782 dataset (n =264), patients were divided into high expression group (n =114) and low expression group (n =150) according to the median expression level of hnRNP U . Overall survival (OS) between the two groups was compared. RPMI 8226, NCI-H929 and MM.1S cell lines were selected as tool cell lines. Following knockdown of hnRNP U by shRNA, the cell proliferation was detected by CCK-8. The apoptosis of MM cells was analyzed by Annexin V/7-AAD staining, and cell cycle was detected by BrdU/DAPI staining followed by flow cytometric analysis. The effect of hnRNP U on the biological characteristics of human MM cells was explored. The effect of knockdown of hnRNP U on DNA damage response pathways was analyzed by Western blot. Analysis of GSE5900 and GSE2113 datasets showed that the mRNA level of hnRNP U rose with the increased degree of malignancy of plasma cell diseases. Survival curve analysis of GSE9782 dataset showed that the high expression group of hnRNP U had a shorter OS than that of the low expression group. Down-regulation of hnRNP U in MM cell lines RPMI 8226, NCI-H929 and MM.1S could inhibit the cell proliferation, promote cell apoptosis and arrest the cell cycle. After knocking down hnRNP U, the expression levels of cleaved PARP and p-H2A.X, as the important markers of activated DNA damage pathway, were significantly increased in MM cells. hnRNP U is highly expressed in several plasma cell diseases including MM, and the high expression of hnRNP U in MM patients predicts poor prognosis. Knocking down hnRNP U can inhibit the malignant progression of MM cells, which is possibly associated with aggravated DNA damage. hnRNP U对多发性骨髓瘤的临床意义及体外实验研究. 研究异质核糖核蛋白U（hnRNP U）在多发性骨髓瘤（MM）患者中的表达水平及与预后相关性，探索hnRNP U在人MM细胞中的功能性作用及分子作用机制，从而为开发hnRNP U作为MM治疗的新靶点提供理论依据。. 基于GEO数据库比较hnRNP U 在几种浆细胞疾病及健康对照中的表达差异；下载GSE9782数据集（n =264），按照hnRNP U 表达水平中位值分为高表达组（n =114）和低表达组（n =150），分析比较两组间总体生存期的差异；选择RPMI 8226、NCI-H929和MM.1S作为工具细胞系，利用shRNA载体敲低hnRNP U后，再通过CCK-8检测细胞增殖能力，利用Annexin V/7-AAD双染法检测细胞凋亡，BrdU/DAPI流式检测细胞周期，探究hnRNP U对人MM肿瘤细胞生物学特性的影响；利用蛋白免疫印迹实验研究敲低hnRNP U后对DNA损伤应答通路的影响。. 数据集GSE5900和GSE2113的分析结果显示，hnRNP U mRNA含量随着浆细胞疾病恶性化（风险）程度上升而升高，数据集GSE9782生存曲线分析显示hnRNP U 高表达组较低表达组具有更短的总体生存期；在MM细胞系RPMI 8226、NCI-H929和MM.1S中敲低hnRNP U后，可抑制MM细胞增殖，促进细胞凋亡和细胞周期发生阻滞；敲低hnRNP U后，MM细胞中DNA损伤应答通路活化的关键标志物PARP剪切体和p-H2A.X的含量明显升高。. hnRNP U在包括MM等多种浆细胞疾病中高表达，并且高表达hnRNP U的MM患者预后较差。敲低hnRNP U后可抑制MM细胞的恶性化进展，其分子机制可能是抑制hnRNP U后DNA损伤加重。.
To summarize the clinicopathological characteristics and survival outcomes of pediatric non-Hodgkin lymphoma (NHL) in Fujian Province. Clinical data of 294 newly diagnosed pediatric NHL patients treated at multiple centers in Fujian Province from January 2011 to December 2023 were collected. The characteristics of different pathological subtypes were summarized, Kaplan-Meier survival analysis were performed and Cox proportional hazards regression model was used for prognostic analysis. A total of 294 pediatric NHL patients were included in this study, with a male-to-female ratio of 3.03∶1 and a median age of 7 years (range, 0.9-14 years). The most common subtype was mature B-cell lymphoma, accounting for 59.2% of cases. The majority of patients were diagnosed at stage III/IV (86.2%), with 32 cases (10.9%) involving central nervous system (CNS) infiltration and 89 cases (30.3%) showing bone marrow involvement. The rate of voluntary abandonment significantly decreased after 2018 (abandonment rates before and after 2018: 6/110 (5.45%) vs. 1/184 (0.54%), P =0.012). Furthermore, excluding cases of voluntary abandonment, the 5-year EFS and OS of newly diagnosed pediatric NHL patients from 2018 to 2023 were still significantly higher than those diagnosed from 2011 to 2017 (EFS: 79.3%±3.7% vs. 70.2%±4.5%, P =0.032; OS: 87.7%±2.6% vs. 70.2%±4.5%, P < 0.001). OS improvements after 2018 were significant in patients with BL and LBL (BL: 89.3%±3.6% vs. 73.5%±7.6%, P =0.033; LBL: 89.3%±5.3% vs. 56.5%±10.3%, P =0.001). However, there were no statistically significant differences in EFS or OS for patients with ALCL or DLBCL (all P >0.05). Multivariate survival analysis identified concurrent hemophagocytic lymphohistiocytosis syndrome was an independent risk factors for both EFS and OS in pediatric NHL patients. Over the past six years, OS and EFS in children with NHL in Fujian Province have improved markedly, with more pronounced gains in BL and LBL. This trend may be related to the combined effects of reduced voluntary treatment abandonment, more standardized diagnostic and therapeutic pathways, treatment optimization, and updated protocols. HLH at initial diagnosis is an independent risk factor for poor prognosis in pediatric NHL, while remission after two chemotherapy cycles suggests a favorable outcome. 福建省儿童非霍奇金淋巴瘤的临床特征及生存分析. 总结福建省儿童非霍奇金淋巴瘤（NHL）的临床病理特征及生存情况。. 收集2011年1月-2023年12月期间福建省多中心诊治的294例初发NHL患儿临床资料，总结不同病理亚型患儿的临床特征，采用Kaplan-Meier进行生存分析，采用Cox比例风险回归模型进行预后分析。. 共纳入294例NHL患儿，男女比例为3.03 ∶1，中位年龄为7（0.9-14）岁，以成熟B细胞淋巴瘤最常见（59.2%）。绝大多数在诊断时处于Ⅲ/Ⅳ期（86.2%），32例（10.9%）合并有中枢神经系统浸润，89例（30.3%）伴有骨髓浸润。总体5年无事件生存（EFS）率和总生存（OS）率分别为74.0%±2.7% 和79.1%±2.5%。2018年后主动放弃率显著降低［2018年前后放弃率分别为6/110（5.45%）、1/184（0.54%）, P =0.012］，而且剔除主动放弃病例后，2018-2023年期间初诊NHL患儿5年EFS、OS仍均显著高于2011-2017年初诊患儿（EFS：79.3%±3.7% 对 70.2%±4.5%，P =0.032；OS：87.7%±2.6% 对 70.2%±4.5%， P < 0.001），其中以伯基特淋巴瘤（BL）和淋巴母细胞淋巴瘤（LBL）最为显著（BL：89.3%±3.6% 对 73.5%±7.6%，P =0.033；LBL：89.3%±5.3% 对 56.5%±10.3%，P =0.001），而ALCL及DLBCL患儿的EFS及OS差异无统计学意义（均P >0.05）。多因素生存分析结果显示，合并噬血细胞综合征是NHL患儿EFS和OS的独立危险因素，化疗2疗程后达到缓解则是EFS和OS预后良好因素。. 近6年来，福建省儿童NHL的OS及EFS明显改善，尤以BL和LBL更为突出。这一趋势可能与主动放弃率下降、诊疗流程规范化、治疗优化以及方案更新等多因素共同作用相关。初诊合并HLH是儿童NHL不良预后的独立危险因素，而2疗程后达到缓解则提示预后良好。.
To analyze the efficacy and safety of anti-thymocyte globulin (ATG), anti-CD25 monoclonal antibody (CD25 mAb), and maraviroc (MVC) as graft-versus-host disease (GVHD) prophylaxis regimens in patients with high-risk acute myeloid leukemia (AML) or high-risk myelodysplastic syndromes (MDS) undergoing unrelated donor hematopoietic stem cell transplantation (unrelated-HSCT). The clinical characteristics of 193 high-risk AML/MDS patients who underwent unrelated-HSCT at the Fifth Medical Center of Chinese PLA General Hospital from January 2011 to December 2018 were retrospectively analyzed. Patients were divided into three groups based on the GVHD prophylaxis: ATG group (n=46), CD25 mAb group (n=126), and MVC group (n=21). The cumulative incidences of acute GVHD (aGVHD), chronic GVHD (cGVHD), relapse and non-relapse mortality (NRM), as well as disease-free survival (DFS), overall survival (OS) were analyzed. The ATG, CD25 mAb and MVC groups showed no statistically significant differences in hematopoietic reconstitution (P>0.05). The cumulative incidence of 100 d grade Ⅱ-Ⅳ aGVHD in CD25 mAb, ATG and MVC group was 42.1%, 24.0% and 19.0% respectively, with a statistically significant difference (P=0.020). Multivariate Cox regression analysis showed that ATG (HR=0.47，P=0.028) and MVC (HR=0.36，P=0.045) were independent protective factors for reducing the incidence of grade Ⅱ-Ⅳ aGVHD. There were no significant differences in 100d grade Ⅲ-Ⅳ aGVHD (P=0.239), 5-year cGVHD (P=0.123), moderate-severe cGVHD (P=0.100), DFS (P=0.130), OS (P=0.271), and NRM (P=0.949). The 5-year cumulative incidence of relapse in the ATG, CD25 mAb and MVC group was 13.3% 35.9% and 38.1%, respectively, with a statistically significant difference (P=0.011). Multivariate Cox regression analysis showed that ATG was an independent protective factor for reducing recurrence rate (HR=0.34，P=0.016). The 5-year GRFS in the ATG, CD25 mAb and MVC group was 38.0%, 21.0% and 9.5%, respectively, with a statistically significant difference (P=0.034). Multivariate Cox regression analysis showed that ATG was an independent protective factor for improving GRFS (HR=0.54，P=0.008). This study concludes that among high-risk AML/MDS patients receiving unrelated-HSCT, an ATG-based regimen for GVHD prophylaxis can effectively reduce the incidence and recurrence rate of acute GVHD, improve GRFS, and should be considered as the preferred strategy. 不同GVHD预防方案对非血缘造血干细胞移植疗效的 影响：一项真实世界长期回顾性研究. 比较抗胸腺细胞球蛋白（ATG）、抗CD25单抗和马拉韦罗（MVC）三种移植物抗宿主病（GVHD）预防方案在非血缘造血干细胞移植（unrelated-HSCT）治疗高危急性髓系白血病（AML）和高危骨髓增生异常综合征（MDS）患者中的长期疗效. 回顾性分析2011年1月至2018年12月解放军总医院第五医学中心连续接受unrelated-HSCT的193例高危AML/MDS患者的临床资料。根据GVHD预防方案分为ATG组（46例）、CD25单抗组（126例）和MVC组（21例）。比较三组患者的造血重建、急慢性GVHD（aGVHD、cGVHD）、无病生存（DFS）、总生存（OS）、复发率和非复发死亡率（NRM）. ATG组、CD25单抗组和MVC组患者中性粒细胞和血小板植入时间无显著差异（P>0.05）。CD25单抗组、ATG组和MVC组100天内Ⅱ-Ⅳ度aGVHD累积发生率分别为42.1%、24.0%、19.0%，差异有统计学意义（P=0.020）。多因素Cox回归分析显示，ATG与MVC是降低Ⅱ-Ⅳ度aGVHD发生率的独立保护因素。3组100 d内Ⅲ-Ⅳ度aGVHD、5年cGVHD和中重度cGVHD累积发生率比较均无统计学差异（P值分别为0.239、0.123、0.100），3组间5年DFS、OS和NRM比较均无统计学差异（P值分别为0.130、0.271、0.949）。ATG组、CD25单抗组和MVC组5年累积复发率分别为13.3%、35.9%和38.1%，差异有统计学意义（P=0.011）。多因素Cox回归分析显示，ATG是降低复发率的独立保护因素（HR=0.34，P=0.016）。ATG组、CD25单抗组与MVC组的5年GRFS分别为38.0%、21.0%和9.5%，差异有统计学意义（P=0.034）。多因素Cox回归分析显示ATG是提高GRFS的独立保护因素（HR=0.54，P=0.008）. 在unrelated-HSCT治疗高危AML/MDS患者中，以ATG为主的GVHD预防方案能有效降低急性GVHD发生率和复发率，并提高GRFS，可作为unrelated-HSCT中GVHD预防的优选方案.
To investigate the expression and methylation alterations of ARHGAP10 in AML and to further explore its clinical significance. ARHGAP10 expression was measured by RT-qPCR in 18 controls and 68 AML patients. ARHGAP10 methylation was assessed using targeted sulfite sequencing in 25 controls and 102 AML patients. The clinical significance of ARHGAP10 expression and methylation changes in AML was further analyzed. Analysis of publicly available datasets identified increased expression of ARHGAP10 in AML patients, which was further validated in our hospital cohort. Among the FAB subtypes, ARHGAP10 expression was significantly higher in the M2 and M5 subgroups compared to the control group. ROC curve analysis indicated that ARHGAP10 expression could be a potential diagnostic biomarker for AML. Kaplan-Meier analysis showed that high ARHGAP10expression and ARHGAP10 hypomethylation were associated with shorter overall survival. Furthermore, ARHGAP10 hypomethylation was observed in AML patients, and further analysis revealed a negative correlation between ARHGAP10 methylation level and expression level. ARHGAP10 exhibits high expression and hypomethylation changes in AML, and these alterations may serve as potential indicators of poor prognosis in AML patients. 急性髓系白血病中ARHGAP10基因改变及其预后意义. 探讨ARHGAP10在AML患者中的表达和甲基化改变及其临床意义。. 采用实时荧光定量PCR检测18例对照者及68例初诊AML患者中ARHGAP10的表达水平；通过靶向亚硫酸盐测序检测25例对照者和102 例AML患者中ARHGAP10的甲基化水平，并进一步分析ARHGAP10表达和甲基化状态与预后的关系。. 公共数据库分析发现ARHGAP10AML患者中高表达，本研究结果同样证实其在AML中呈高表达，且在 FAB亚型的M2、M5组中明显高于对照组。ROC曲线分析表明，ARHGAP10表达可能作为鉴别AML的潜在生物标志物。Kaplan-Meier生存分析显示ARHGAP10高表达组以及ARHGAP10低甲基化组患者的总生存时间均较短。此外，AML患者中ARHGAP10呈低甲基化状态，进一步分析表明，ARHGAP10甲基化水平与表达水平呈负相关。. ARHGAP10在AML中呈现高表达和低甲基化改变，该改变可以作为AML预后不良的潜在指标之一。.