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Corrigendum to “First-line therapy with daratumumab, lenalidomide and dexamethasone for patient with POEMS syndrome: A case report” [Leukemia Research Reports (2024) Volume 22, 100491] “用达拉单抗、来那度胺和地塞米松治疗POEMS综合征患者的一线治疗:一个病例报告”的勘误表[白血病研究报告(2024)卷22,100491]
IF 0.7 Q4 HEMATOLOGY Pub Date : 2025-01-01 Epub Date: 2025-01-20 DOI: 10.1016/j.lrr.2025.100499
E. Amabile, F. Fazio, M. Martelli, M.T. Petrucci
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引用次数: 0
Venetoclax combination with Cladribine, idarubicin, Cytarabine for relapsed T-Cell acute lymphoblastic leukemia/lymphoblastic lymphoma treatment: A case report and literature review Venetoclax联合克拉德滨、依达柔比星、阿糖胞苷治疗复发性t细胞急性淋巴细胞白血病/淋巴细胞淋巴瘤1例报告并文献复习
IF 0.7 Q4 HEMATOLOGY Pub Date : 2025-01-01 Epub Date: 2025-03-14 DOI: 10.1016/j.lrr.2025.100506
Alaa Eldein Yahia , Ibrahim Motabi , Abdullah A. Alsakkaf , Kamal Alzahrani , Laila M. Alsuhaibani , Bilal Albtoosh , Abdullah Khaled AlBathi , Abdullah M. Alrajhi
Acute lymphoblastic leukemia (ALL) represents only 20 % of adult acute leukemias, while Lymphoblastic lymphoma is even rarer, accounting for 2 % of adult non-Hodgkin lymphomas. T-acute lymphoblastic leukemia (T-ALL) and T-lymphoblastic lymphoma (T-LBL) are neoplasms characterized by the presence of immature T-cell precursors or lymphoblasts. Relapsed T-ALL or LBL is associated with a very poor prognosis, necessitating the exploration of novel therapeutic approaches. This case report describes the use of Venetoclax in combination with Cladribine, Idarubicin, and Cytarabine (CLIA) as salvage therapy for relapsed T-ALL/T-LBL. The treatment regimen resulted in remission and negative minimal residual disease. However, it was accompanied by delayed count recovery, febrile neutropenia, and Central Line-Associated Bloodstream Infection. The management of central nervous system involvement was challenging due to low platelet counts requiring transfusion support. The findings highlight the need for further investigation into the efficacy and optimal therapeutic regimen for relapsed T-ALL/T-LBL. Additionally, the case emphasizes the importance of early salvage therapy and potentially consolidative hematopoietic stem cell transplantation for improved survival outcomes in relapsed T-ALL/T-LBL patients.
急性淋巴母细胞白血病(ALL)仅占成人急性白血病的20%,而淋巴母细胞淋巴瘤更罕见,占成人非霍奇金淋巴瘤的2%。t急性淋巴母细胞白血病(T-ALL)和t淋巴母细胞淋巴瘤(T-LBL)是一种以未成熟t细胞前体或淋巴母细胞存在为特征的肿瘤。复发的T-ALL或LBL预后非常差,需要探索新的治疗方法。本病例报告描述了使用Venetoclax联合cladriine, Idarubicin和Cytarabine (CLIA)作为复发T-ALL/T-LBL的补救性治疗。治疗方案导致缓解和阴性最小残留疾病。然而,它伴随着计数恢复延迟、发热性中性粒细胞减少和中央线相关血流感染。由于血小板计数低,需要输血支持,中枢神经系统受累的管理具有挑战性。研究结果强调需要进一步研究复发T-ALL/T-LBL的疗效和最佳治疗方案。此外,该病例强调了早期补救性治疗和潜在的巩固性造血干细胞移植对改善复发T-ALL/T-LBL患者生存结果的重要性。
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引用次数: 0
Selinexor combination with decitabine and half-dose CMG (Cytarabine+ Mitoxantrone+G-CSF) in Patients with refractory/relapsed acute myeloid leukemia (AML) previously exposed to Venetoclax: a single-center retrospective study Selinexor联合地西他滨和半剂量CMG(阿糖胞苷+米托蒽醌+G-CSF)治疗先前暴露于Venetoclax的难治性/复发性急性髓性白血病(AML)患者:一项单中心回顾性研究
IF 0.7 Q4 HEMATOLOGY Pub Date : 2025-01-01 Epub Date: 2025-06-19 DOI: 10.1016/j.lrr.2025.100523
Zi-Yu Zhao, Xiao-Lei Zhang, Ying Zhan, Ya-Bei Zuo, Jin-Ao Li, Jin-Hai Ren, Xiao-Ling Guo, Zi-Yuan Nie
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引用次数: 0
Retrospective claims data analysis of ASCT characteristics and costs for working-age, multiple myeloma patients in the US, 2017–2019 2017-2019年美国工龄多发性骨髓瘤患者ASCT特征和费用的回顾性索赔数据分析。
IF 0.7 Q4 HEMATOLOGY Pub Date : 2025-01-01 Epub Date: 2024-12-18 DOI: 10.1016/j.lrr.2024.100496
Whanhui Chi , Juhyeon Song , Tyler J. Varisco
Multiple myeloma (MM) is a rare hematologic malignancy with a 5-year survival rate of 52 %. For transplant-eligible MM patients, high-dose chemotherapy followed by autologous stem cell transplant (ASCT) is recommended. Given the complexities of the ASCT procedure, understanding patient-specific factors and their impact on treatment decisions is essential.
Our study examines patient characteristics and patterns of health resource utilization associated with ASCT receipt in patients with MM.
This retrospective study used the Merative™ MarketScan® database from 2017 to 2019 to analyze working-aged adults (18–65 years) with MM. We categorized 643 ASCT recipients by demographic characteristics (age, sex, region, employment status, year of ASCT procedure) and clinical factors (Charlson Comorbidity Index score). We assessed health resource utilization, focusing on ASCT-related costs, including total payments and hospitalization duration. Descriptive statistics were calculated for all variables, with means, medians, standard deviations for continuous variables, and frequencies for categorical variables. Pearson correlation assessed the relationship between total payment and hospitalization duration.
Over 80 % of patients were over 50, highlighting the need for age-specific clinical strategies. Most patients had CCI scores of 2–4, indicating a moderate comorbidity burden. The mean hospitalization duration was 21.71 days, with average ASCT costs totaling $166,235.99. The correlation coefficient of 0.21 indicated that total payments also increase as the number of hospitalization days increases.
These findings highlight the need for tailored care approaches and resource allocation in ASCT, informing future research and clinical decision-making.
多发性骨髓瘤(MM)是一种罕见的血液系统恶性肿瘤,5年生存率为52%。对于符合移植条件的MM患者,推荐高剂量化疗后进行自体干细胞移植(ASCT)。鉴于ASCT程序的复杂性,了解患者特异性因素及其对治疗决策的影响至关重要。我们的研究检查了MM患者接受ASCT相关的患者特征和健康资源利用模式。这项回顾性研究使用了2017年至2019年Merative™MarketScan®数据库,分析了患有MM的工作年龄成年人(18-65岁)。我们根据人口统计学特征(年龄、性别、地区、就业状况、ASCT手术年份)和临床因素(Charlson共病指数评分)对643名ASCT接受者进行了分类。我们评估了卫生资源利用情况,重点关注与asct相关的费用,包括总费用和住院时间。对所有变量进行描述性统计,包括连续变量的均值、中位数、标准差和分类变量的频率。Pearson相关性评估总付款额与住院时间的关系。超过80%的患者超过50岁,这突出了针对年龄的临床策略的必要性。大多数患者的CCI评分为2-4,表明有中度合并症负担。平均住院时间为21.71天,平均ASCT费用总计为166,235.99美元。相关系数为0.21,表明随着住院天数的增加,支付总额也随之增加。这些发现强调了在ASCT中定制护理方法和资源分配的必要性,为未来的研究和临床决策提供了信息。
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引用次数: 0
Intercurrent presentation of hairy cell leukemia and diffuse large B-cell lymphoma with hemophagocytic lymphohistiocytosis: a case report 毛细胞白血病和弥漫性大b细胞淋巴瘤并发噬血细胞性淋巴组织细胞增多症1例
IF 0.9 Q4 HEMATOLOGY Pub Date : 2025-01-01 Epub Date: 2025-08-07 DOI: 10.1016/j.lrr.2025.100535
Tiffany Nong , Xiaoqiong Wang , Wenhui Li , Ellen Madarang , Justin Watts , Justin Taylor
Hairy cell leukemia (HCL) is a rare, indolent B-cell lymphoma that is highly responsive to the purine analog cladribine. A defining feature of classical HCL is the presence of the BRAF V600E mutation, which is almost universally present. The transformation of HCL to an aggressive large B-cell lymphoma is extremely rare. Here, we report a case of intercurrent presentation of worsening classical HCL with a rapidly evolving BRAF V600E-negative aggressive large B-cell lymphoma and associated hemophagocytic lymphohistiocytosis (HLH). The contiguous presentation of these three entities posed significant diagnostic and therapeutic challenges. Despite treatment with cladribine, the patient’s condition deteriorated, leading to a palliative focus. This case underscores the challenges of managing multiple intercurrent hematologic malignancies manifesting with overlapping symptoms and sites of organ involvement but non-overlapping treatments. The presence of HLH as a complication in patients with malignancy further complicates the clinical picture and requires early recognition and prompt intervention. Further research is needed to better understand the pathophysiological links between HCL, large cell transformation, and HLH. Additionally, registry or other cross-sectional studies with larger numbers of HCL patients could clarify the exact frequency of these rare but fatal complications.
毛细胞白血病(HCL)是一种罕见的惰性b细胞淋巴瘤,对嘌呤类似物克拉德里滨高度敏感。经典HCL的一个决定性特征是BRAF V600E突变的存在,这种突变几乎普遍存在。HCL向侵袭性大b细胞淋巴瘤的转变极为罕见。在这里,我们报告了一例恶化的经典HCL与快速发展的BRAF v600e阴性侵袭性大b细胞淋巴瘤和相关的噬血细胞性淋巴组织细胞增多症(HLH)并存的病例。这三种实体的连续表现构成了重大的诊断和治疗挑战。尽管用克拉宾治疗,病人的病情恶化,导致姑息治疗的焦点。本病例强调了管理多重并发的血液恶性肿瘤的挑战,表现为重叠的症状和器官受累部位,但不重叠的治疗。作为恶性肿瘤患者的并发症,HLH的存在进一步使临床情况复杂化,需要早期识别和及时干预。需要进一步的研究来更好地了解HCL、大细胞转化和HLH之间的病理生理联系。此外,大量HCL患者的登记或其他横断面研究可以明确这些罕见但致命的并发症的确切频率。
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引用次数: 0
AL amyloidosis with elevated peripheral blood cell counts – A frequent association with liver involvement. A single-center retrospective study AL淀粉样变伴外周血细胞计数升高-常与肝脏受累有关。单中心回顾性研究
IF 0.7 Q4 HEMATOLOGY Pub Date : 2025-01-01 Epub Date: 2025-04-12 DOI: 10.1016/j.lrr.2025.100509
Mateusz Ziarkiewicz , Justyna Szczygieł , Marta Legatowicz-Koprowska , Joanna Drozd-Sokołowska , Piotr Boguradzki , Krzysztof Jamroziak , Grzegorz Basak

Background

AL amyloidosis is a systemic protein misfolding disorder characterized by organ deposition of monoclonal immunoglobulin fragments, with insidious onset and progressive course. The plasma cell clone in the bone marrow is relatively small and typically does not impair hematopoiesis, in contrast to multiple myeloma. Herein we present a novel observation of increased thrombocyte, leukocyte and erythrocyte counts in a subset of AL amyloidosis patients.

Material and Methods

We performed a retrospective analysis of medical records of all consecutive patients diagnosed with AL amyloidosis at the Medical University of Warsaw in years 2001–2022, which included clinical, pathological and laboratory data, as well as treatment protocols and outcomes.

Results

Twenty-three patients out of 124 (18.4 %) included had elevated blood counts: 17 (13.6 %) had leukocytosis with neutrophilia, 7 (5.6 %) had thrombocytosis, whereas 2 (1.6 %) had erythrocytosis. In comparison to the remaining AL population this subgroup was characterized by younger age (median 57 vs 62 years, p = 0.018), higher frequency of hepatomegaly (42.9 % vs.14.7 %, p = 0.004), higher median alkaline phosphatase concentration (129 U/L vs 93 U/L, p = 0.006) and more frequent hepatic amyloidosis (34.8 % vs 10.3 %, p = 0.003). None of the patients had definite features of a myeloproliferative neoplasm, although genetic testing was available in 5 out of 9 cases with thrombocytosis or erythrocytosis. There were no significant differences in terms of survival between patients with elevated cell counts and non-polycythemic patients (median overall survival 2.9 vs 6.6 years, p = 0.51, median event-free survival 0.7 vs 1.8 years, p = 0.29, respectively).

Conclusions

Elevated peripheral blood counts in a subset of patients with AL amyloidosis constitute a rare but significant phenomenon and appear to be associated with frequent hepatic involvement. We hypothesize that cytokine deregulation and hyposplenism may belong to its pathomechanisms.
背景:淀粉样变性是一种以单克隆免疫球蛋白片段器官沉积为特征的全身性蛋白质错误折叠疾病,发病隐匿,病程渐进。与多发性骨髓瘤相比,骨髓中的浆细胞克隆相对较小,通常不会损害造血功能。在这里,我们提出了一个新的观察增加的血小板,白细胞和红细胞计数在一个子集AL淀粉样变性患者。材料和方法我们对2001-2022年在华沙医科大学诊断为AL淀粉样变性的所有连续患者的医疗记录进行了回顾性分析,包括临床、病理和实验室数据,以及治疗方案和结果。结果124例患者中23例(18.4%)出现血球计数增高,其中白细胞增多伴中性粒细胞增多17例(13.6%),血小板增多7例(5.6%),红细胞增多2例(1.6%)。与其他AL人群相比,该亚组的特点是年龄更年轻(中位57岁对62岁,p = 0.018),肝肥大发生率更高(42.9%对14.7%,p = 0.004),碱性磷酸酶中位浓度更高(129 U/L对93 U/L, p = 0.006),肝淀粉样变发生率更高(34.8%对10.3%,p = 0.003)。虽然9例有血小板增多或红细胞增多的患者中有5例进行了基因检测,但没有一例患者具有骨髓增生性肿瘤的明确特征。细胞计数升高的患者和非红细胞增多症患者的生存期没有显著差异(中位总生存期为2.9年vs 6.6年,p = 0.51,中位无事件生存期为0.7年vs 1.8年,p = 0.29)。结论:AL淀粉样变患者的外周血计数升高是一种罕见但重要的现象,似乎与频繁的肝脏受累有关。我们推测细胞因子失调和脾功能减退可能属于其病理机制。
{"title":"AL amyloidosis with elevated peripheral blood cell counts – A frequent association with liver involvement. A single-center retrospective study","authors":"Mateusz Ziarkiewicz ,&nbsp;Justyna Szczygieł ,&nbsp;Marta Legatowicz-Koprowska ,&nbsp;Joanna Drozd-Sokołowska ,&nbsp;Piotr Boguradzki ,&nbsp;Krzysztof Jamroziak ,&nbsp;Grzegorz Basak","doi":"10.1016/j.lrr.2025.100509","DOIUrl":"10.1016/j.lrr.2025.100509","url":null,"abstract":"<div><h3>Background</h3><div>AL amyloidosis is a systemic protein misfolding disorder characterized by organ deposition of monoclonal immunoglobulin fragments, with insidious onset and progressive course. The plasma cell clone in the bone marrow is relatively small and typically does not impair hematopoiesis, in contrast to multiple myeloma. Herein we present a novel observation of increased thrombocyte, leukocyte and erythrocyte counts in a subset of AL amyloidosis patients.</div></div><div><h3>Material and Methods</h3><div>We performed a retrospective analysis of medical records of all consecutive patients diagnosed with AL amyloidosis at the Medical University of Warsaw in years 2001–2022, which included clinical, pathological and laboratory data, as well as treatment protocols and outcomes.</div></div><div><h3>Results</h3><div>Twenty-three patients out of 124 (18.4 %) included had elevated blood counts: 17 (13.6 %) had leukocytosis with neutrophilia, 7 (5.6 %) had thrombocytosis, whereas 2 (1.6 %) had erythrocytosis. In comparison to the remaining AL population this subgroup was characterized by younger age (median 57 vs 62 years, <em>p</em> = 0.018), higher frequency of hepatomegaly (42.9 % vs.14.7 %, <em>p</em> = 0.004), higher median alkaline phosphatase concentration (129 U/L vs 93 U/L, <em>p</em> = 0.006) and more frequent hepatic amyloidosis (34.8 % vs 10.3 %, <em>p</em> = 0.003). None of the patients had definite features of a myeloproliferative neoplasm, although genetic testing was available in 5 out of 9 cases with thrombocytosis or erythrocytosis. There were no significant differences in terms of survival between patients with elevated cell counts and non-polycythemic patients (median overall survival 2.9 vs 6.6 years, <em>p</em> = 0.51, median event-free survival 0.7 vs 1.8 years, <em>p</em> = 0.29, respectively).</div></div><div><h3>Conclusions</h3><div>Elevated peripheral blood counts in a subset of patients with AL amyloidosis constitute a rare but significant phenomenon and appear to be associated with frequent hepatic involvement. We hypothesize that cytokine deregulation and hyposplenism may belong to its pathomechanisms.</div></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"23 ","pages":"Article 100509"},"PeriodicalIF":0.7,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143843498","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Treatment outcomes and survival analysis of pediatric mature B-Cell non-Hodgkin lymphoma: A retrospective study comparing LMB96 and R-CHOP regimens 儿童成熟b细胞非霍奇金淋巴瘤的治疗结果和生存分析:一项比较LMB96和R-CHOP方案的回顾性研究
IF 0.9 Q4 HEMATOLOGY Pub Date : 2025-01-01 Epub Date: 2025-07-23 DOI: 10.1016/j.lrr.2025.100531
Hamid D. Habeeb Rjeib , Usama Al-Jumaily , Inas Muayad Mohammed Ali , Safa Faraj , Mohammed Fawzi Al-Qanbar , Dheyaa Aldeen Al-Khateeb , Shaima Jabbar

Background

Mature B-cell non-Hodgkin lymphoma (B-NHL) is a prevalent pediatric malignancy with significant treatment advancements. This study retrospectively analyzes clinical characteristics, treatment outcomes, and survival rates of children and adolescents diagnosed with B-NHL at Al-Hasan Al-Mojtaba Hospital between January 2014 and December 2024. A comparative analysis was performed between the LMB96 and R-CHOP regimens.

Methods

Patients with confirmed diagnoses of Large B-cell lymphoma or Burkitt’s lymphoma, based on WHO classification criteria, were included. Staging was conducted using the St. Jude system, and risk classification followed the FAB/LMB criteria. Treatment involved a modified LMB96 regimen, later replaced by R-CHOP in the last two years of the study. Event-free survival (EFS) was analyzed using Kaplan-Meier survival curves, with stratifications by staging, risk group, and gender.

Results

A total of 66 patients were included (median age: 5.8 years; 69.7 % male). Burkitt’s lymphoma was the predominant histology (86.3 %). The abdomen was the most common primary site (84.8 %). The majority of patients (72.7 %) presented with advanced-stage disease (Stages III and IV). Risk group classification identified 62.1 % of patients in Group B and 28.8 % in Group C. Kaplan-Meier survival analysis revealed Group A had the most favorable prognosis (EFS ∼100 %), followed by Group B (∼75 %), and Group C (∼50 %). Disease stage significantly influenced survival (p = 0.021), with Stage IV patients demonstrating the poorest outcomes. While female patients exhibited higher EFS than males, the difference was not statistically significant (p = 0.27). By the end of follow-up, 28.8 % of patients had experienced a fatal outcome.

Conclusion

Advanced-stage B-NHL remains prevalent, with significant survival differences based on staging and risk classification. The transition from LMB96 to R-CHOP warrants further evaluation to optimize pediatric treatment strategies. Larger studies are needed to validate observed gender-based survival trends.
成熟b细胞非霍奇金淋巴瘤(B-NHL)是一种常见的儿科恶性肿瘤,治疗进展显著。本研究回顾性分析了2014年1月至2024年12月在Al-Hasan Al-Mojtaba医院诊断为B-NHL的儿童和青少年的临床特征、治疗结果和生存率。对LMB96和R-CHOP方案进行了比较分析。方法根据WHO的分类标准,纳入确诊为大b细胞淋巴瘤或伯基特淋巴瘤的患者。采用St. Jude系统进行分期,并按照FAB/LMB标准进行风险分类。治疗包括改良的LMB96方案,后来在研究的最后两年被R-CHOP取代。使用Kaplan-Meier生存曲线分析无事件生存(EFS),并按分期、风险组和性别进行分层。结果共纳入66例患者(中位年龄:5.8岁;69.7%男性)。Burkitt淋巴瘤为主要组织学类型(86.3%)。腹部是最常见的原发部位(84.8%)。大多数患者(72.7%)表现为晚期疾病(III期和IV期)。风险组分类确定了B组62.1%的患者和C组28.8%的患者。Kaplan-Meier生存分析显示,A组预后最佳(EFS ~ 100%),其次是B组(~ 75%)和C组(~ 50%)。疾病分期显著影响生存率(p = 0.021),其中IV期患者预后最差。女性患者的EFS高于男性,但差异无统计学意义(p = 0.27)。到随访结束时,28.8%的患者经历了致命的结果。结论晚期B-NHL仍然普遍存在,根据分期和风险分类存在显著的生存差异。从LMB96到R-CHOP的过渡值得进一步评估,以优化儿科治疗策略。需要更大规模的研究来验证观察到的基于性别的生存趋势。
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引用次数: 0
Effective treatment of peritoneal myeloid sarcoma with decitabine and venetoclax: A case report 地西他滨联合维妥乐治疗腹膜髓系肉瘤1例
IF 0.9 Q4 HEMATOLOGY Pub Date : 2025-01-01 Epub Date: 2025-08-08 DOI: 10.1016/j.lrr.2025.100534
Kavya Sudireddy , Minorvi Amin , Rafy Odeh , Patrick Svrcek , Salwa Khedr , Lloyd Hutchinson , Shyam A. Patel , Jan Cerny , Laurie Pearson
Myeloid sarcomas (MS) are rare extramedullary manifestations of myeloid progenitor cells occurring with or without acute myeloid leukemia. Peritoneal MS is exceptionally uncommon, with no established treatment guidelines, but treatment has historically consisted of cytotoxic chemotherapy. We present the case of a 78-year-old female with 60% CD34+, HLA-DR+, and CD33+ myeloblasts in ascitic fluid, while bone marrow biopsy demonstrated only molecular evidence of the leukemic clone with FLT3-ITD, ASXL1, and TET2 mutations. Findings were consistent with primary peritoneal myeloid sarcoma. Due to chemotherapy ineligibility, the patient was treated with decitabine and venetoclax. After nine cycles, she demonstrated a complete radiographic response. To our knowledge, this is the first case report of a patient with primary peritoneal MS treated with a combination of a hypomethylating agent and venetoclax.
髓系肉瘤(MS)是罕见的髓系祖细胞髓外表现,伴或不伴急性髓系白血病发生。腹膜性多发性硬化症非常罕见,没有既定的治疗指南,但治疗历史上包括细胞毒性化疗。我们报告了一例78岁的女性患者,腹水中有60%的CD34+、HLA-DR+和CD33+的髓母细胞,而骨髓活检仅显示了带有FLT3-ITD、ASXL1和TET2突变的白血病克隆的分子证据。结果与原发性腹膜髓系肉瘤一致。由于不适合化疗,患者接受地西他滨和维妥乐治疗。9个周期后,她表现出完全的x线片反应。据我们所知,这是第一例用低甲基化药物和venetoclax联合治疗原发性腹膜MS的病例报告。
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引用次数: 0
Tagraxofusp maintenance post-hematopoietic stem cell transplantation provides long-term survival and manageable safety for a patient with blastic plasmacytoid dendritic cell neoplasm Tagraxofusp维持后造血干细胞移植为母细胞浆细胞样树突状细胞肿瘤患者提供了长期生存和可管理的安全性
IF 0.7 Q4 HEMATOLOGY Pub Date : 2025-01-01 Epub Date: 2025-06-12 DOI: 10.1016/j.lrr.2025.100518
Qaiser Bashir , Marina Konopleva , Glorette Abueg , Jeremy Ramdial , Chitra Hosing , Samer A. Srour , Amin Alousi , Uday R. Popat , Yago Nieto , Gheath Alatrash , Richard E. Champlin , Elizabeth J. Shpall , Muzaffar Qazilbash , Naveen Pemmaraju
Presented here is the case of a 68-year-old woman with blastic plasmacytoid dendritic cell neoplasm (BPDCN) treated with tagraxofusp (TAG) maintenance therapy post-allogeneic hematopoietic stem cell transplantation (allo-HCT). Prior to allo-HCT, the patient was treated with hydroxyurea and mini-CVD (cyclophosphamide, vincristine, and dexamethasone alternating with methotrexate (Methotrexate) and cytarabine) + venetoclax + TAG for 5 cycles, which induced morphologic complete remission with minimal residual disease. After allo-HCT, the patient had persistent cytogenic abnormalities 45,XX,der(7)add(7)(p13)del(7)(q11.2q22)add(7)(q32),add(12)(p13),-15,del(16)(q23),-17,+22,+2mar[1]/46,XX[19], and was then treated with TAG maintenance therapy at 9 mg/kg on a 28-day cycle for 16 cycles. At mid-treatment (cycle 6 of 16 cycles of TAG) and prior to TAG discontinuation (due to travel burden), the patient was negative for minimal residual disease and was in complete remission. There was no high-grade toxicity or capillary leak syndrome. The patient remains in complete remission as of present day (>17 months CR). This is the first case study illustrating the feasibility of long-term TAG maintenance post-allo-HCT to control BPDCN.
本文报告一位68岁的女性,她患有母浆细胞样树突状细胞肿瘤(BPDCN),同种异体造血干细胞移植(alloo - hct)后接受tagraxofusp (TAG)维持治疗。在alloo - hct之前,患者接受羟基脲和mini-CVD(环磷酰胺、新碱和地塞米松与甲氨蝶呤(甲氨蝶呤)和阿糖胞苷交替治疗)+ venetoclax + TAG治疗5个周期,诱导形态完全缓解,残余疾病极小。同种异体hct后,患者持续出现细胞遗传学异常45、XX、der(7)add(7)(p13)del(7)(q11.2q22)add(7)(q32)、add(12)(p13)、-15、del(16)(q23)、-17、+22、+2mar[1]/46、XX[19],然后给予TAG维持治疗,剂量为9 mg/kg, 28天为一个周期,共16个周期。在治疗中期(16个TAG周期中的第6周期)和TAG停止之前(由于旅行负担),患者的微小残留疾病呈阴性,并且完全缓解。无严重毒性或毛细血管渗漏综合征。截至目前,患者仍处于完全缓解期(17个月CR)。这是第一个案例研究,说明了在允许hct后长期维持TAG以控制BPDCN的可行性。
{"title":"Tagraxofusp maintenance post-hematopoietic stem cell transplantation provides long-term survival and manageable safety for a patient with blastic plasmacytoid dendritic cell neoplasm","authors":"Qaiser Bashir ,&nbsp;Marina Konopleva ,&nbsp;Glorette Abueg ,&nbsp;Jeremy Ramdial ,&nbsp;Chitra Hosing ,&nbsp;Samer A. Srour ,&nbsp;Amin Alousi ,&nbsp;Uday R. Popat ,&nbsp;Yago Nieto ,&nbsp;Gheath Alatrash ,&nbsp;Richard E. Champlin ,&nbsp;Elizabeth J. Shpall ,&nbsp;Muzaffar Qazilbash ,&nbsp;Naveen Pemmaraju","doi":"10.1016/j.lrr.2025.100518","DOIUrl":"10.1016/j.lrr.2025.100518","url":null,"abstract":"<div><div>Presented here is the case of a 68-year-old woman with blastic plasmacytoid dendritic cell neoplasm (BPDCN) treated with tagraxofusp (TAG) maintenance therapy post-allogeneic hematopoietic stem cell transplantation (allo-HCT). Prior to allo-HCT, the patient was treated with hydroxyurea and mini-CVD (cyclophosphamide, vincristine, and dexamethasone alternating with methotrexate (Methotrexate) and cytarabine) + venetoclax + TAG for 5 cycles, which induced morphologic complete remission with minimal residual disease. After allo-HCT, the patient had persistent cytogenic abnormalities 45,XX,der(7)add(7)(p13)del(7)(q11.2q22)add(7)(q32),add(12)(p13),-15,del(16)(q23),-17,+22,+2mar[1]/46,XX[19], and was then treated with TAG maintenance therapy at 9 mg/kg on a 28-day cycle for 16 cycles. At mid-treatment (cycle 6 of 16 cycles of TAG) and prior to TAG discontinuation (due to travel burden), the patient was negative for minimal residual disease and was in complete remission. There was no high-grade toxicity or capillary leak syndrome. The patient remains in complete remission as of present day (&gt;17 months CR). This is the first case study illustrating the feasibility of long-term TAG maintenance post-allo-HCT to control BPDCN.</div></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"24 ","pages":"Article 100518"},"PeriodicalIF":0.7,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144306788","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Corrigendum to “Unveiling prognosis in patients with acute myeloid leukemia treated with hypomethylating agents and venetoclax through novel risk stratification” [Leukemia Research Reports 24 (2025) 100550] “通过新的风险分层揭示低甲基化药物和venetoclax治疗急性髓系白血病患者的预后”的勘误表[白血病研究报告24 (2025)100550]
IF 0.9 Q4 HEMATOLOGY Pub Date : 2025-01-01 Epub Date: 2025-10-30 DOI: 10.1016/j.lrr.2025.100551
Jiajia Sun, Zhiping Guo
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引用次数: 0
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Leukemia Research Reports
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