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Clinical trials in early-stage CLL: what has been learned and what's next? 早期CLL的临床试验:已经学到了什么?下一步是什么?
IF 2.2 4区 医学 Q3 HEMATOLOGY Pub Date : 2025-03-01 Epub Date: 2024-11-29 DOI: 10.1080/10428194.2024.2422839
Manuela A Hoechstetter, Clemens-Martin Wendtner

More than 80% of newly diagnosed chronic lymphocytic leukemia (CLL) patients present with asymptomatic, early-stage CLL. Of these, only 30-50% progress to advanced stage with reduced survival, while the rest may never require treatment. According to the 2018 International Workshop on CLL (iwCLL) guidelines, patients who do not meet the criteria for treatment initiation should only be treated within the context of clinical trials, as data demonstrating an overall survival benefit in early-stage CLL are still awaited. Risk stratification through continually advancing prognostic models can assist in identifying high-risk patients for early, risk-adapted treatment within clinical trials. Currently, new targeted therapies with high efficacy and lower toxicity are available in early intervention trials. This review (1) explores the development of prognostic models for identifying high-risk patients, (2) examines the design of early intervention trial, (3) summarizes the outcomes of early intervention trials, particularly in the context of targeted therapies, and (4) highlights ongoing clinical trials involving targeted treatments.

超过80%的新诊断慢性淋巴细胞白血病(CLL)患者表现为无症状的早期CLL。其中,只有30-50%进展到晚期,生存期缩短,而其余的可能永远不需要治疗。根据2018年CLL国际研讨会(iwCLL)指南,不符合开始治疗标准的患者只能在临床试验的背景下进行治疗,因为早期CLL的总体生存获益数据仍在等待中。通过不断推进的预后模型进行风险分层,可以在临床试验中帮助识别高危患者,以便进行早期、适应风险的治疗。目前,在早期干预试验中出现了新的高效、低毒的靶向治疗方法。本综述(1)探讨了识别高危患者的预后模型的发展,(2)检查了早期干预试验的设计,(3)总结了早期干预试验的结果,特别是在靶向治疗的背景下,(4)重点介绍了正在进行的涉及靶向治疗的临床试验。
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引用次数: 0
Ixazomib-induced thrombotic microangiopathy resolving without complement pathway inhibition: a case report. 伊沙佐米诱发的血栓性微血管病无需补体途径抑制即可缓解:一份病例报告。
IF 2.2 4区 医学 Q3 HEMATOLOGY Pub Date : 2025-03-01 Epub Date: 2024-11-20 DOI: 10.1080/10428194.2024.2428360
Tanguy Lafont, Stephen Booth, Harry Wakefield
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引用次数: 0
Impact of the addition of rituximab in adults with newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia. 利妥昔单抗对新诊断费城染色体阳性急性淋巴细胞白血病的影响
IF 2.2 4区 医学 Q3 HEMATOLOGY Pub Date : 2025-03-01 Epub Date: 2024-12-01 DOI: 10.1080/10428194.2024.2432582
Fausto Alfredo Rios-Olais, Sergio Rodriguez-Rodriguez, Analy Mora-Cañas, Christian Omar Ramos-Peñafiel, Victor Itaí Urbalejo-Ceniceros, Yadith Karina Lopez-Garcia, Andres Gomez-De Leon, Nancy Delgado-Lopez, Mistral Castellanos-Mares, Patricia Carolina Figueroa-Hernandez, Roberta Demichelis-Gomez
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引用次数: 0
Clinical outcomes of patients diagnosed with SETBP1 mutated myeloid neoplasms. 确诊为 SETBP1 基因突变骨髓性肿瘤患者的临床疗效。
IF 2.2 4区 医学 Q3 HEMATOLOGY Pub Date : 2025-03-01 Epub Date: 2024-11-06 DOI: 10.1080/10428194.2024.2425048
Yazan Jabban, Mahmoud Yacout, Anmol Baranwal, Rong He, David Viswanatha, Patricia Greipp, Dragan Jevremovic, Kurt Bessonen, James Foran, Jeanne Palmer, Antoine N Saliba, Mehrdad Hefazi-Torghabeh, Kebede Begna, William J Hogan, Abhishek Mangaonkar, Mrinal Patnaik, Mithun Shah, Hassan Alkhateeb, Aref Al-Kali

SETBP1 mutations (m) have been previously reported in myeloid neoplasms and are associated with poor prognostic co-mutations and cytogenetic abnormalities. We retrospectively analyzed the charts of 113 patients diagnosed with myeloid neoplasms with SETBP1m. The most common diagnosis was MDS (31%). Cytogenetics were abnormal in 51 cases (46.4%), with monosomy 7 being the most common (41.1%). The most frequent co-mutations were ASXL1 (71.7%), SRSF2 (46.9%), TET2 (20.4%). Higher SETBP1m VAF was associated with proliferative features (p < 0.05). Most SETBP1m (96.5%) were in one of three hotspots (Asp868, Gly870, Ile871), with Asp868m being most frequent (51.3%). Patients with Ile871m had higher number of co-mutations (median= 4) compared to Asp868m and Gly870m (p = 0.07). On multivariate analysis, age ≥ 70 years (p = 0.004) and higher peripheral blood blasts (p = 0.02) had worse OS. Patients with Ile871m had lower OS when compared with Asp868m and Gly870m (5.5 months vs. 17.4 and 17 months, respectively, p = 0.1).

以前曾报道过骨髓性肿瘤中的 SETBP1 基因突变(m)与预后不良的共突变和细胞遗传学异常有关。我们回顾性分析了 113 例被诊断为 SETBP1m 的骨髓性肿瘤患者的病历。最常见的诊断是 MDS(31%)。51例(46.4%)细胞遗传学异常,其中单体7型最为常见(41.1%)。最常见的共突变是ASXL1(71.7%)、SRSF2(46.9%)和TET2(20.4%)。较高的 SETBP1m VAF 与增殖特征相关(P = 0.07)。在多变量分析中,年龄≥70岁(p = 0.004)和外周血血细胞较高(p = 0.02)的患者OS较差。与Asp868m和Gly870m相比,Ile871m患者的OS较低(分别为5.5个月 vs. 17.4个月和17个月,p = 0.1)。
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引用次数: 0
Clinical and biological advances of critical complications in acute myeloid leukemia. 急性髓性白血病严重并发症的临床和生物学进展。
IF 2.2 4区 医学 Q3 HEMATOLOGY Pub Date : 2025-03-01 Epub Date: 2024-11-24 DOI: 10.1080/10428194.2024.2425051
Alessandro Costa, Emilia Scalzulli, Ida Carmosino, Claudia Ielo, Maria Laura Bisegna, Maurizio Martelli, Massimo Breccia

Managing acute myeloid leukemia (AML) and its critical complications requires understanding the complex interplay between disease biology, treatment strategies, and patient characteristics. Complications like sepsis, acute respiratory failure (ARF), hyperleukocytosis, coagulopathy, tumor lysis syndrome (TLS) and central nervous system (CNS) involvement present unique challenges needing precise evaluation and tailored interventions. Venetoclax-induced TLS and differentiation syndrome (DS) from IDH1/IDH2 or menin inhibitors highlight the need for ongoing research and innovative approaches. As the microbiological landscape evolves and new therapeutic agents emerge, adapting strategies to mitigate harmful pharmacological interactions is crucial. Advances in understanding the genetic profiles of patients with hyperleukocytosis contribute to better-targeted therapeutic strategies. Effective AML management relies on collaborative efforts from hematologists, specialized services, and intensive care units (ICUs). This review analyzes recent data on critical AML complications, identifies areas for further investigation, and proposes ways to advance clinical research and enhance patient care strategies.

治疗急性髓性白血病(AML)及其严重并发症需要了解疾病生物学、治疗策略和患者特征之间复杂的相互作用。脓毒症、急性呼吸衰竭(ARF)、高白细胞血症、凝血功能障碍、肿瘤溶解综合征(TLS)和中枢神经系统(CNS)受累等并发症提出了独特的挑战,需要精确的评估和量身定制的干预措施。IDH1/IDH2或menin抑制剂引起的Venetoclax诱导的TLS和分化综合征(DS)凸显了持续研究和创新方法的必要性。随着微生物环境的演变和新治疗药物的出现,调整策略以减轻有害的药理相互作用至关重要。在了解高白细胞症患者的遗传特征方面取得的进展有助于制定更有针对性的治疗策略。有效的急性髓细胞白血病管理有赖于血液科医生、专业服务部门和重症监护室(ICU)的通力合作。本综述分析了急性髓细胞性白血病重要并发症的最新数据,确定了需要进一步研究的领域,并提出了推进临床研究和加强患者护理策略的方法。
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引用次数: 0
Oral azacitidine maintenance after intensive chemotherapy versus venetoclax and azacitidine: real world outcomes in newly diagnosed acute myeloid leukemia. 强化化疗后口服阿扎胞苷维持治疗与 Venetoclax 和阿扎胞苷:新诊断急性髓性白血病的实际疗效。
IF 2.2 4区 医学 Q3 HEMATOLOGY Pub Date : 2025-03-01 Epub Date: 2024-11-28 DOI: 10.1080/10428194.2024.2425792
Alice Mims, Zhuoer Xie, Ravi Potluri, David Rotter, Manoj Chevli, Thomas Prebet, Lona Gaugler, Maria Strocchia, Alberto Vasconcelos, Jan Sieluk

Oral azacitidine (Oral-AZA) is recommended as maintenance therapy for patients with newly diagnosed acute myeloid leukemia (ND AML) achieving remission with intensive chemotherapy (IC) but not transplant candidates; venetoclax plus injectable azacitidine (VEN-AZA) is recommended for patients ineligible for IC. Some patients may be considered candidates for either regimen. This retrospective study used Flatiron Health's database to compare treatment patterns and clinical outcomes with Oral-AZA maintenance after IC (IC🡪Oral-AZA) versus frontline VEN-AZA. Relapse-free survival (RFS) and overall survival (OS) were analyzed at 4 different time points, including from Oral-AZA initiation (IC🡪Oral-AZA cohort) or from remission (VEN-AZA cohort) in the Core Analysis. Median RFS was 14.9 and 8.1 months for IC🡪Oral-AZA and VEN-AZA propensity score-matched cohorts, in the Core Analysis (n = 32 in each; p = 0.027); median OS was 18.7 and 15.2 months (p = 0.034). In patients with AML, IC🡪Oral-AZA significantly improved RFS and OS compared with VEN-AZA.

新确诊的急性髓性白血病(ND AML)患者在接受强化化疗(IC)后病情得到缓解,但不适合接受移植治疗,建议将口服阿扎胞苷(Oral-AZA)作为维持治疗方案;不适合接受IC治疗的患者建议使用venetoclax加注射用阿扎胞苷(VEN-AZA)。有些患者可能被认为适合这两种方案。这项回顾性研究利用 Flatiron Health 的数据库,比较了 IC(ICᾆOral-AZA)后口服阿扎胞苷维持治疗与一线 VEN-AZA 的治疗模式和临床结果。在4个不同的时间点分析了无复发生存期(RFS)和总生存期(OS),包括核心分析中从口服AZA开始(ICᾆOral-AZA队列)或从缓解开始(VEN-AZA队列)。在核心分析中,ICᾆOral-AZA和VEN-AZA倾向评分匹配队列的中位RFS分别为14.9个月和8.1个月(各32人;p=0.027);中位OS分别为18.7个月和15.2个月(p=0.034)。与 VEN-AZA 相比,ICᾆOral-AZA 能显著改善 AML 患者的 RFS 和 OS。
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引用次数: 0
Extramedullary immunoprivileged sites as a niche for residual and relapsed FLT3-ITD mutated AML: an unmet clinical need. 髓外免疫特权位点作为残留和复发的FLT3-ITD突变AML的利基:未满足的临床需求。
IF 2.2 4区 医学 Q3 HEMATOLOGY Pub Date : 2025-03-01 Epub Date: 2024-11-28 DOI: 10.1080/10428194.2024.2431880
Philip D Tracy, Ruben Delgado, Ahmad Al-Akdi, Monika Pilichowska, Zheng Zhou
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引用次数: 0
A case of acute macular neuroretinopathy as atipycal ICANS manifestation after CAR-T cell infusion. CAR-T细胞输注后急性黄斑神经视网膜病变非典型ICANS表现1例。
IF 2.2 4区 医学 Q3 HEMATOLOGY Pub Date : 2025-03-01 Epub Date: 2024-11-12 DOI: 10.1080/10428194.2024.2426056
A Soravia, F Menchini, F Di Bin, G Facchin, P Lanzetta, R Fanin, F Patriarca
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引用次数: 0
Severe toxicity, but long-term persistence of CAR T cells after immune checkpoint inhibitors in large B-cell lymphoma. 大 B 细胞淋巴瘤患者使用免疫检查点抑制剂后,CAR T 细胞毒性严重,但可长期存活。
IF 2.2 4区 医学 Q3 HEMATOLOGY Pub Date : 2025-03-01 Epub Date: 2024-11-20 DOI: 10.1080/10428194.2024.2430703
Xavier Deschênes-Simard, Martina Pennisi, Miguel-Angel Perales, Gunjan L Shah, Andrew D Zelenetz, Joachim Yahalom, Brandon S Imber, Bianca D Santomasso, Parastoo B Dahi
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引用次数: 0
Arsenic trioxide neurotoxicity in acute promyelocytic leukemia patients: a single center experience. 急性早幼粒细胞白血病患者的三氧化二砷神经毒性:单中心经验。
IF 2.2 4区 医学 Q3 HEMATOLOGY Pub Date : 2025-03-01 Epub Date: 2024-11-08 DOI: 10.1080/10428194.2024.2427266
G Arrigo, M Scaldaferri, E Audisio, E Boscaro, F Catania, F Cattel, L Celona, M Cerrano, R Freilone, V Giai, I Urbino, S D'Ardia, C Frairia
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引用次数: 0
期刊
Leukemia & Lymphoma
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