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Treatment of Vietnamese patients diagnosed with myelodysplastic neoplasms: Practical experience in a developing country 越南骨髓增生异常肿瘤患者的治疗:在发展中国家的实践经验
IF 0.7 Q4 HEMATOLOGY Pub Date : 2025-01-01 Epub Date: 2024-11-24 DOI: 10.1016/j.lrr.2024.100490
Quang Hao Nguyen , Minh Phuong Vu , Ha Trang Kieu , Duc Binh Vu , Ha Thanh Nguyen , Quoc Khanh Bach

Background

Treatment of patients diagnosed with myelodysplastic neoplasms (MDS) is difficult and the outcome is still limited, especially in developing countries. We conducted this study in order to share some experience in treating patients diagnosed with MDS in developing countries.

Methods

This was a retrospective study that included 32 patients with newly MDS. 13 lower-risk patients, including 2 patients with MDS 5q- were treated with erythropoiesis stimulating agent (ESA). 19 patients with higher risk were treated with hypomethylating agent (HMA), which was decitabine.

Results

In the ESA treatment group, the rate of hematologic improvement-erythroid was 69.2 %, the rate of total hematologic improvement (with 3 lineages improvement) was 61.5 %. In the HMA treatment group, the overall response rate was 52.6 %. The follow-up times were 42 months. The overall survival (OS), leukemic transformation-free survival (LFS), and progression-free survival (PFS) of the ESA treatment group were 30.44, 28.91, and 28.29 months; respectively. The OS, LFS, and PFS of the HMA treatment group were 34.27, 31.45, and 26.83 months; respectively.

Conclusions

Patients with lower risk MDS, including MDS 5q-, may benefit from treatment with erythropoiesis stimulating agent (ESA). Patients with higher risk MDS may have a favorable outcome with decitabine (HMA) treatment.
背景骨髓增生异常肿瘤(MDS)患者的治疗是困难的,结果仍然有限,特别是在发展中国家。我们进行这项研究是为了分享在发展中国家治疗MDS患者的一些经验。方法回顾性研究32例新发MDS患者。13例低危患者,包括2例MDS 5q-患者接受促红细胞生成剂(ESA)治疗。19例高危患者采用低甲基化剂(HMA)治疗,即地西他滨。结果ESA治疗组血液学改善率为69.2%,总血液学改善率为61.5%(其中3个系改善)。HMA治疗组总有效率为52.6%。随访42个月。ESA治疗组总生存期(OS)、无白血病转化生存期(LFS)、无进展生存期(PFS)分别为30.44个月、28.91个月和28.29个月;分别。HMA治疗组的OS、LFS、PFS分别为34.27、31.45、26.83个月;分别。结论低危MDS(包括MDS 5q-)患者可从促红细胞生成剂(ESA)治疗中获益。高风险MDS患者使用地西他滨(HMA)治疗可能有良好的结果。
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引用次数: 0
A rare case of CD38-negative abdominal multiple extramedullary plasmacytoma and literature review cd38阴性腹腔多发髓外浆细胞瘤1例并文献复习。
IF 0.7 Q4 HEMATOLOGY Pub Date : 2025-01-01 Epub Date: 2024-12-21 DOI: 10.1016/j.lrr.2024.100493
XS Bao, DH Gong, KG Zhou, W Huang
Abdominal multiple extramedullary plasmacytoma (EMP) is a rare disease. CD38-negative relapsed/refractory EMP after treatment with daratumumab has never been reported. In 2020, a patient with jaundice was diagnosed with plasmacytoma in another hospital, which progressed one year after receiving multiline therapy. In July 2021, he was admitted to our hospital and showed CD38-pogative plasmacytoma. The patient received 2 cycles of treatment including daratumumab, venetoclax and DCEP chemotherapy and achieved partial remission. However, he developed ascites and eventually died. Our case indicated that multiple EMP has much lower incidence and far worse prognosis than solitary EMP.
摘要腹腔多发髓外浆细胞瘤是一种罕见的疾病。经达拉单抗治疗后cd38阴性的复发/难治性EMP从未报道过。2020年,一名黄疸患者在另一家医院被诊断为浆细胞瘤,在接受多线治疗一年后病情恶化。2021年7月入院,cd38阳性浆细胞瘤。患者接受达拉单抗、venetoclax和DCEP化疗2个周期治疗,部分缓解。然而,他患上了腹水,最终死亡。我们的病例显示多发性EMP的发生率比单发EMP低得多,预后也差得多。
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引用次数: 0
Management of relapsed acute lymphoblastic leukemia in a patient with down syndrome: A case report 唐氏综合征患者复发性急性淋巴细胞白血病的治疗:1例报告
IF 0.9 Q4 HEMATOLOGY Pub Date : 2025-01-01 Epub Date: 2025-09-03 DOI: 10.1016/j.lrr.2025.100543
Eman Almatar, Sondus Alsharidah, Omnia A. Hashem
Children with Down syndrome (DS) have a 10–20-fold increased risk of acute lymphoblastic leukemia (ALL) and heightened chemotherapy toxicity. Blinatumomab, a bispecific CD19 × CD3 T-cell engager, offers targeted immunotherapy with reduced myelotoxicity. We describe a 9-year-old girl with DS diagnosed with B-cell precursor ALL in early 2021 who relapsed during maintenance in September 2023. After reinduction, she received two 28-day blinatumomab cycles: the first resulted in <5 % blasts and undetectable minimal residual disease (MRD), and the second was well tolerated. She remains in remission pending allogeneic hematopoietic stem cell transplantation, highlighting blinatumomab’s efficacy and safety as a bridge to transplantation.
患有唐氏综合症(DS)的儿童患急性淋巴细胞白血病(ALL)的风险增加10 - 20倍,化疗毒性增加。Blinatumomab是一种双特异性CD19 × CD3 t细胞参与剂,提供降低骨髓毒性的靶向免疫治疗。我们描述了一名患有DS的9岁女孩,她在2021年初被诊断为b细胞前体ALL,并于2023年9月在维持期间复发。再诱导后,她接受了两个28天的blinatumumab周期:第一个周期导致5%的细胞和无法检测到的微小残留病(MRD),第二个周期耐受性良好。她仍处于缓解期,等待异体造血干细胞移植,这突出了blinatumomab作为移植桥梁的有效性和安全性。
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引用次数: 0
Real-world management of blastic plasmacytoid dendritic cell neoplasm at an academic center with a broad regional referral base 在一个具有广泛区域转诊基础的学术中心,母浆细胞样树突状细胞肿瘤的实际管理
IF 0.9 Q4 HEMATOLOGY Pub Date : 2025-01-01 Epub Date: 2025-08-25 DOI: 10.1016/j.lrr.2025.100541
Nathan M. Krah , Rasmus Hoeg , Paul J. Shami , Shannon E. Elf , Lauren E. Lee , Naveen Pemmaraju , Ami B. Patel
BPDCN is an aggressive myeloid malignancy characterized by unique expression of CD123, CD4, CD56, CD303, CD304, TCL1 and TCF4. The development of tagraxofusp, a CD123-directed cytotoxin, has revolutionized BPDCN treatment, especially for patients unfit for chemotherapy. While most patients respond to frontline tagraxofusp, there are challenges associated with treatment. In this case series, we describe our institutional experience treating BPDCN both pre- and post-tagraxofusp approval. We summarize six cases, the majority of which occurred in elderly patients, and highlight unique challenges of treating BPDCN at a center that serves a large rural/frontier area with variable access to specialty care.
BPDCN是一种侵袭性髓系恶性肿瘤,其特征是CD123、CD4、CD56、CD303、CD304、TCL1和TCF4的独特表达。tagraxofusp是一种cd123导向的细胞毒素,它的开发已经彻底改变了BPDCN的治疗,特别是对于不适合化疗的患者。虽然大多数患者对一线tagraxofusp有反应,但与治疗相关的挑战仍然存在。在本案例系列中,我们描述了我们在fda批准前和批准后处理BPDCN的机构经验。我们总结了6例病例,其中大多数发生在老年患者中,并强调了在一个服务于广大农村/边境地区的中心治疗BPDCN的独特挑战,这些地区有不同的专科护理机会。
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引用次数: 0
Isolated bone marrow gamma/delta T-cell lymphoma: A difficult case to classify according to the current WHO classification of lymphoid malignancies 孤立骨髓γ / δ t细胞淋巴瘤:根据目前世界卫生组织对淋巴细胞恶性肿瘤的分类,这是一个难以分类的病例
IF 0.9 Q4 HEMATOLOGY Pub Date : 2025-01-01 Epub Date: 2025-08-08 DOI: 10.1016/j.lrr.2025.100539
Hideto Hyuuga , Naoki Oishi , Takuma Kumagai , Minori Matuura , Ayato Nakadate , Yuma Sakamoto , Jun Suzuki , Megumi Suzuki , Megumi Koshiisi , Ichiro Kawashima , Takeo Yamamoto , Kei Nakajima , Masaru Tanaka , Tetuso Kondo , Keita Kirito
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引用次数: 0
BCR::ABL1 kinase domain mutations and their predictive value for treatment outcomes in patients with chronic myeloid leukemia treated with first-line imatinib in a low-income setting: Experience from Côte d’Ivoire BCR: ABL1激酶结构域突变及其对低收入环境中一线伊马替尼治疗的慢性髓性白血病患者治疗结果的预测价值:来自Côte科特迪瓦的经验
IF 0.9 Q4 HEMATOLOGY Pub Date : 2025-01-01 Epub Date: 2025-09-06 DOI: 10.1016/j.lrr.2025.100544
Kouassi Gustave Koffi , Sara Akou Bognini , Dohoma Alexis Silué , Ismael Kamara , Ines Kouakou , Ruth Dieket , Emeraude N’dhatz , Boidy Kouakou , Danho Clotaire Nanho , Yannick Kouassi , David Tea Okou

Background

We aimed to analyse the incidence of BCR::ABL1 kinase domain mutations in patients newly diagnosed with or undergoing treatment for chronic myeloid leukemia (CML) in Côte d’Ivoire, as well as to evaluate the predictive factors associated with treatment outcomes.

Methods

We evaluated 42 patients with suboptimal molecular response who underwent BCR::ABL1 kinase domain mutation screening. Sequencing was performed in collaboration with the Fred Hutchinson Cancer Research Center, Seattle, USA.

Results

Among the 42 patients, 16 (38.1%) were found to have known BCR::ABL1 point mutations. A total of nine distinct mutations were identified, with T315I being the most common (5). Three patients harbored compound mutations: one had T315I + M244V, another had G250E + E459K, and the third had H396P + E459K. The 5-year overall survival (OS) rate was significantly lower in patients with BCR::ABL1 mutations (85%; 95 % CI: 51.0%–96.1%) compared to those without mutations (100%). However, there was no statistically significant difference in OS between patients with T315I mutations (83.3 %; 95 % CI: 27.4%–97.5%) and those without T315I (83.3%; 95% CI: 5.9%–98.8%). Being in the chronic phase of the disease and having a low-risk ELTS score were identified as protective factors against the development of mutations.

Conclusion

Our findings support the recommendation for BCR::ABL1 mutation screening in CML patients with an inadequate initial response or evidence of loss of response. Screening is also advised at progression to the accelerated or blast phase, and in patients with high-risk ELTS scores, and mutation profiles may serve as important prognostic indicators.
背景:我们旨在分析Côte科特迪瓦新诊断或正在接受慢性髓性白血病(CML)治疗的患者中BCR::ABL1激酶结构域突变的发生率,并评估与治疗结果相关的预测因素。方法对42例分子反应欠佳的患者进行BCR::ABL1激酶结构域突变筛查。测序是与美国西雅图的Fred Hutchinson癌症研究中心合作进行的。结果42例患者中,已知BCR::ABL1点突变16例(38.1%)。共鉴定出9种不同的突变,其中T315I最为常见(5)。3例患者携带复合突变:1例为T315I + M244V, 1例为G250E + E459K, 3例为H396P + E459K。BCR::ABL1突变患者的5年总生存率(OS)明显低于无突变患者(100%)(85%;95% CI: 51.0%-96.1%)。而T315I突变患者(83.3%,95% CI: 27.4% ~ 97.5%)与无T315I突变患者(83.3%,95% CI: 5.9% ~ 98.8%)的OS差异无统计学意义。处于疾病的慢性期和具有低风险的ELTS评分被确定为防止突变发展的保护因素。结论:我们的研究结果支持对初始反应不足或无反应证据的CML患者进行BCR::ABL1突变筛查的建议。筛查也建议在进展到加速期或爆炸期时,以及在具有高风险ELTS评分的患者中进行筛查,突变谱可作为重要的预后指标。
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引用次数: 0
Catastrophic asparaginase-induced cerebral and systemic thrombosis in a young female with T-cell lymphoblastic lymphoma: A case report & literature review 灾难性天冬酰胺酶诱发的年轻女性t细胞淋巴母细胞淋巴瘤脑及全身血栓:1例报告及文献复习
IF 0.7 Q4 HEMATOLOGY Pub Date : 2025-01-01 Epub Date: 2025-07-04 DOI: 10.1016/j.lrr.2025.100526
Shihab Ahmed Ibrahim Ahmed, Amr Suliman AlHanbali
Asparaginase is a cornerstone in the treatment of acute lymphoblastic leukemia (ALL) and its variant, T-cell lymphoblastic lymphoma (T-LBL), particularly in adolescents and young adults (AYA). However, its use is associated with a significant risk of thrombotic complications due to its profound effects on coagulation pathways. We report a catastrophic case of asparaginase-induced cerebral and systemic thrombosis in a previously healthy 20-year-old female with T-LBL. Despite prophylactic anticoagulation, the patient developed extensive cerebral venous sinus thrombosis, deep vein thrombosis, and pulmonary embolism, necessitating mechanical thrombectomy, Argatroban therapy, and long-term anticoagulation. This case underscores the multifactorial pathophysiology of asparaginase-associated thrombosis and highlights the need for individualized risk assessment, vigilant monitoring, and dynamic anticoagulation strategies. A comprehensive literature review is provided to contextualize the epidemiology, mechanisms, risk factors, prevention, and management of this life-threatening complication, with practical recommendations for optimizing care in high-risk patients.
天冬酰胺酶是治疗急性淋巴细胞白血病(ALL)及其变种t细胞淋巴母细胞淋巴瘤(T-LBL)的基石,特别是在青少年和年轻人(AYA)中。然而,由于其对凝血途径的深远影响,其使用与血栓并发症的显著风险相关。我们报告一个灾难性的病例天冬酰胺酶诱导的脑和全身血栓形成在一个以前健康的20岁女性与T-LBL。尽管进行了预防性抗凝治疗,但患者出现了广泛的脑静脉窦血栓、深静脉血栓和肺栓塞,需要机械取栓、阿加曲班治疗和长期抗凝。该病例强调了天冬酰胺酶相关血栓形成的多因素病理生理学,并强调了个体化风险评估、警惕监测和动态抗凝策略的必要性。本文对这一危及生命的并发症的流行病学、机制、危险因素、预防和管理进行了全面的文献综述,并提出了优化高危患者护理的实用建议。
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引用次数: 0
Pediatric chronic myeloid leukemia: A decade of clinical experience at the NBK specialized hospital for children 儿童慢性髓性白血病:在NBK儿童专科医院的十年临床经验
IF 0.9 Q4 HEMATOLOGY Pub Date : 2025-01-01 Epub Date: 2025-10-10 DOI: 10.1016/j.lrr.2025.100549
Maha Bourusly , Mohammad Adil Obaid , Nashwa Farag , Mona Bourhama , Sundos Alsharidah , Eman Almatar
Pediatric chronic myeloid leukemia is rare and differs from adult disease. We retrospectively reviewed 12 children (median age 8.4 years, six males) treated with imatinib (340 mg/m²/day) from 2010 to 2020; dasatinib was used for intolerance. All presented with leukocytosis (median WBC, 358 × 109/L) and splenomegaly. Imatinib achieved complete hematologic response in 92% by 3 months and 100% by 6 months; major molecular response was observed in all evaluable cases at 12 months. Three who lost response switched to dasatinib and maintained remission; two attained treatment-free remission. TKIs are effective; prospective studies should optimize risk stratification and discontinuation.
小儿慢性髓性白血病是罕见的,不同于成人疾病。我们回顾性分析了2010年至2020年接受伊马替尼(340 mg/m²/天)治疗的12名儿童(中位年龄8.4岁,6名男性);达沙替尼用于治疗不耐受。所有患者均表现为白细胞增多(白细胞中位数为358 × 109/L)和脾肿大。伊马替尼在3个月和6个月达到了92%和100%的完全血液学缓解;在12个月时,所有可评估的病例均观察到主要的分子反应。3名失去反应的患者改用达沙替尼并维持缓解;两名患者获得了无治疗缓解。tki是有效的;前瞻性研究应优化风险分层和停药。
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引用次数: 0
Management of hemolytic transfusion reactions in a patient with chronic myelomonocytic leukemia and rare antibodies: A case report 慢性髓单细胞白血病合并罕见抗体患者的溶血性输血反应的处理:1例报告
IF 0.7 Q4 HEMATOLOGY Pub Date : 2025-01-01 Epub Date: 2024-11-28 DOI: 10.1016/j.lrr.2024.100485
Grace S. Park , Himachandana Atluri , Courtney D. DiNardo , Bryan Guillroy , Jean Horak , Effrosyni Apostolidou , Maryam Buni , Guillermo Montalban Bravo , Naveen Pemmaraju
Delayed hemolytic transfusion reaction (DHTR) poses a significant challenge in patients receiving blood transfusions. This case report highlights the complexities of managing DHTR in a newly diagnosed chronic myelomonocytic leukemia (CMML) patient with clinically significant JKa and little c antibodies during induction chemotherapy. A 46-year-old woman with CMML-2 who presented for induction chemotherapy was found to have hemolytic anemia. Due to presence of JKa and little c antibodies, she required intensive monitoring and supportive care measures. The coexistence of JKa and little c antibodies complicates transfusion management and chemotherapy tolerance in CMML patients.
延迟溶血性输血反应(DHTR)对接受输血的患者提出了重大挑战。本病例报告强调了新诊断的慢性髓细胞白血病(CMML)患者在诱导化疗期间治疗DHTR的复杂性,这些患者临床上有明显的JKa和少量c抗体。一名46岁CMML-2患者接受诱导化疗,发现有溶血性贫血。由于存在JKa和少量c抗体,她需要加强监测和支持性护理措施。JKa和little c抗体的共存使CMML患者的输血管理和化疗耐受性复杂化。
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引用次数: 0
Analysis of atypical clinical manifestations in eight patients with AIDS complicated by lymphoma 艾滋病合并淋巴瘤8例不典型临床表现分析
IF 0.9 Q4 HEMATOLOGY Pub Date : 2025-01-01 Epub Date: 2025-08-25 DOI: 10.1016/j.lrr.2025.100540
Peng fei Tao, Jincheng Lou, Xicheng Wang

Objective

Acquired Immunodeficiency Syndrome (AIDS)-related lymphoma has diverse clinical manifestations, and its diagnosis is often challenging. Misdiagnosis can delay treatment and affect patient prognosis. We assessed the clinical data of eight patients with atypical clinical manifestations of AIDS-related lymphoma, to enhance physicians' understanding of these patients, and reduce the potential for misdiagnosis.

Methods

A retrospective analysis was conducted on eight patients with atypical manifestations of AIDS-related lymphoma admitted to the Department of Infectious Diseases of Yunnan Provincial Hospital between May 2017 and May 2023. They were initially misdiagnosed with opportunistic infections, and were later diagnosed with lymphoma.

Results

The patients comprised five males and three females. Cluster of Differentiation 4 (CD4) counts were lower than 200/μl for all patients, and inflammatory marker levels were elevated to varying degrees. Four patients had recurrent fever, one had bleeding, one had pulmonary infection, one had long-term diarrhoea, and one had visual impairment as the primary symptoms. Six patients were diagnosed through bone marrow cytology and biopsy, one through colonoscopy and pathological biopsy, and one through computed tomography-guided percutaneous lung biopsy. All patients had extranodal involvement, including one case in the intestine, one in the lung, and six in the bone marrow. All patients were at lymphoma stage IV, with four in Group A and four in group B.

Conclusion

In patients with AIDS, particularly those with low CD4 counts and unexplained fever, atypical lymphoma should be considered, and tissue biopsy should be performed to further confirm the diagnosis.
目的获得性免疫缺陷综合征(AIDS)相关淋巴瘤具有多种临床表现,其诊断往往具有挑战性。误诊会延误治疗,影响患者预后。我们对8例临床表现不典型的艾滋病相关淋巴瘤患者的临床资料进行分析,以提高医生对这些患者的认识,减少误诊的可能性。方法对2017年5月至2023年5月云南省医院感染性疾病科收治的8例非典型表现艾滋病相关淋巴瘤患者进行回顾性分析。他们最初被误诊为机会性感染,后来被诊断为淋巴瘤。结果男性5例,女性3例。所有患者CD4细胞计数均低于200/μl,炎症标志物水平均不同程度升高。4例反复发热,1例出血,1例肺部感染,1例长期腹泻,1例以视力受损为主要症状。6例患者通过骨髓细胞学和活检诊断,1例通过结肠镜检查和病理活检诊断,1例通过计算机断层扫描引导下的经皮肺活检诊断。所有患者均有结外受累,包括1例肠、1例肺和6例骨髓。所有患者均为淋巴瘤IV期,其中A组4例,b组4例。结论艾滋病患者,特别是CD4计数低、不明原因发热者,应考虑非典型淋巴瘤,并行组织活检进一步确诊。
{"title":"Analysis of atypical clinical manifestations in eight patients with AIDS complicated by lymphoma","authors":"Peng fei Tao,&nbsp;Jincheng Lou,&nbsp;Xicheng Wang","doi":"10.1016/j.lrr.2025.100540","DOIUrl":"10.1016/j.lrr.2025.100540","url":null,"abstract":"<div><h3>Objective</h3><div>Acquired Immunodeficiency Syndrome (AIDS)-related lymphoma has diverse clinical manifestations, and its diagnosis is often challenging. Misdiagnosis can delay treatment and affect patient prognosis. We assessed the clinical data of eight patients with atypical clinical manifestations of AIDS-related lymphoma, to enhance physicians' understanding of these patients, and reduce the potential for misdiagnosis.</div></div><div><h3>Methods</h3><div>A retrospective analysis was conducted on eight patients with atypical manifestations of AIDS-related lymphoma admitted to the Department of Infectious Diseases of Yunnan Provincial Hospital between May 2017 and May 2023. They were initially misdiagnosed with opportunistic infections, and were later diagnosed with lymphoma.</div></div><div><h3>Results</h3><div>The patients comprised five males and three females. Cluster of Differentiation 4 (CD<sub>4</sub>) counts were lower than 200/μl for all patients, and inflammatory marker levels were elevated to varying degrees. Four patients had recurrent fever, one had bleeding, one had pulmonary infection, one had long-term diarrhoea, and one had visual impairment as the primary symptoms. Six patients were diagnosed through bone marrow cytology and biopsy, one through colonoscopy and pathological biopsy, and one through computed tomography-guided percutaneous lung biopsy. All patients had extranodal involvement, including one case in the intestine, one in the lung, and six in the bone marrow. All patients were at lymphoma stage IV, with four in Group A and four in group B.</div></div><div><h3>Conclusion</h3><div>In patients with AIDS, particularly those with low CD4 counts and unexplained fever, atypical lymphoma should be considered, and tissue biopsy should be performed to further confirm the diagnosis.</div></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"24 ","pages":"Article 100540"},"PeriodicalIF":0.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144914017","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
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Leukemia Research Reports
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