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Clinical Signs and Treatment of New-Onset Bone Marrow Failure Associated SARS-CoV-2 Infection in Children: A Single Institution Prospective Cohort Study. 与 SARS-CoV-2 感染相关的儿童新发骨髓衰竭的临床症状和治疗:单机构前瞻性队列研究。
IF 3.2 4区 医学 Q3 HEMATOLOGY Pub Date : 2024-03-01 eCollection Date: 2024-01-01 DOI: 10.4084/MJHID.2024.034
Mervat A M Youssef, Ebtisam Shawky Ahmed, Dalia Tarik Kamal, Khalid I Elsayh, Mai A Abdelfattah, Hyam Hassan Mahran, Mostafa M Embaby

Background: Viral infections can cause direct and indirect damage to hematopoietic stem cells. The objectives of this study were to identify the frequency and severity of aplastic anemia in children infected with severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) as well as recognize the response to treatment.

Methodology: 13 children with newly diagnosed severe aplastic anemia were enrolled in this prospective clinical trial. Blood samples were obtained from all patients to detect SARS-CoV-2 antibodies, and nasopharyngeal swabs were collected for reverse-transcription Polymerase Chain Reaction to detect SARS-CoV-2 viruses. According to the laboratory results, patients were classified as having SARS-CoV-2 positive antibodies and SARS-CoV-2 negative antibodies. Both groups received combined cyclosporine (CsA) + Eltrombopag (E-PAG). The hematological response, either complete response (CR) or partial response (PR), no response (NR), and overall response (OR) rates of combined E-PAG + CsA treatment after 6 months were evaluated.

Results: Four children were recognized to have aplastic anemia and SARS-CoV-2 positive antibodies. Two patients fulfilled the hematological criteria for CR and no longer required transfusion of packed red blood cells (PRBCs) or platelets, and one had PR and was still PRBC transfusion-dependent but no longer required platelet transfusion. The remaining patient showed NR, and he had died before reaching the top of the HSCT waiting list. Moreover, six patients in the SARS-CoV-2 negative antibodies group had CR, while three patients had PR. The difference in ANC, Hg, and platelet counts between both groups was not significant.

Conclusion: The SARS-CoV-2 virus is added to several viral infections known to be implicated in the pathogenesis of aplastic anemia. Studies are needed to establish a definitive association and determine whether the response of bone marrow failure to standard therapy differs from that of idiopathic cases.

背景:病毒感染可对造血干细胞造成直接或间接损害。本研究的目的是确定感染严重急性呼吸系统综合征冠状病毒 2(SARS-CoV-2)的儿童发生再生障碍性贫血的频率和严重程度,并识别对治疗的反应。方法:13 名新确诊的重型再生障碍性贫血患儿参加了这项前瞻性临床试验,所有患者均采集了血样以检测 SARS-CoV-2 抗体,并采集鼻咽拭子进行反转录聚合酶链反应以检测 SARS-CoV-2 病毒。根据化验结果,患者被分为 SARS-CoV-2 抗体阳性组和 SARS-CoV-2 抗体阴性组。两组患者均接受环孢素(CsA)+艾曲波帕(E-PAG)联合治疗。结果:4名患儿被确诊为浆细胞性白血病(SARS-CoV-2)抗体阳性,1名患儿为SARS-CoV-2抗体阴性;两组患儿均接受了环孢素(CsA)+艾曲波帕(E-PAG)联合治疗:结果:4名儿童被确认为再生障碍性贫血和SARS-CoV-2抗体阳性。两名患者符合 CR 的血液学标准,不再需要输注包装红细胞(PRBC)或血小板;一名患者出现 PR,仍需输注 PRBC,但不再需要输注血小板。剩下的一名患者显示为 NR,但在进入造血干细胞移植候选名单前已经死亡。此外,SARS-CoV-2 阴性抗体组中有 6 名患者出现 CR,3 名患者出现 PR。两组患者的ANC、Hg和血小板计数差异不显著:结论:SARS-CoV-2 病毒是多种已知与再生障碍性贫血发病机制有关的病毒感染的补充。需要进行研究以确定两者之间的明确联系,并确定骨髓衰竭对标准疗法的反应是否与特发性病例不同。
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引用次数: 0
Comparison of Infectious Complications in Patients Receiving High-Dose Cyclophosphamide as GvHD Prophylaxis After Transplantation From A 9/10 HLA-Matched Unrelated Donor with Standard GvHD Prophylaxis After Transplant From A Fully Matched Related Donor. 从 9/10 HLA 匹配的非亲缘供体进行移植后接受大剂量环磷酰胺作为 GvHD 预防措施的患者与从完全匹配的亲缘供体进行移植后接受标准 GvHD 预防措施的患者的感染并发症比较。
IF 3.2 4区 医学 Q3 HEMATOLOGY Pub Date : 2024-03-01 eCollection Date: 2024-01-01 DOI: 10.4084/MJHID.2024.016
Selim Sayýn, Murat Yýldýrým, Melda Cömert, Bilge Uğur, Esra Şafak Yýlmaz, Ferit Avcu, Ali Uğur Ural, Meltem Aylı

Background: The aim of this study was to evaluate whether cyclophosphamide administered after allogeneic stem cell transplantation (ASCT) from 9/10 HLA-Matched Unrelated Donors (MMUD) increases the rates of bacterial, fungal, viral infections, complications (hemorrhagic cystitis (HC)), and infection-related mortality compared to allogeneic stem cell transplantation from matched related donors (MRD).

Methods: This is a retrospective multicenter study. 45 MMUD ASCT patients who received posttransplant cyclophosphamide+methotrexate+calcineurin inhibitor compared with 45 MRD ASCT patients who received methotrexate+calcineurin inhibitor.

Results: Although there was a statistically significant prolongation of neutrophil engraftment time in the PTCy arm, there was no statistically significant difference in bacterial infection frequencies between the groups (PTCy; 9 (20%), control; 8 (17.8%), p=0.778). The distribution of CMV infection in the first 100 days was similar (p=0.827), but the distribution of CMV infection rate between the 100th and 365th days was observed more frequently in the control group (p=0.005). HC rates and their grades were similar in both groups (PTCy; 4 (8.8%), control; 6 (13.3%) p=0.502). The rates of VZV infection and invasive aspergillosis were similar in the PTCy and control groups (13.3% in the PTCy and 17.8% in the control group p=0.561). There is also no statistically significant difference in survival analysis (OS, LFS, GRFS, RI, IRM, NRM) between groups. However, the incidence of cGVHD was significantly higher in the control group (P=0.035).

Conclusions: The addition of PTCy to standard GvHD prophylaxis in MMUD ASCT does not lead to an increase in CMV reactivation, bacterial infections, invasive fungal infection, viral hemorrhagic cystitis, or mortality.

背景:本研究旨在评估与配型相合的亲属供者(MRD)异基因干细胞移植相比,9/10 HLA配型相合的非亲属供者(MMUD)异基因干细胞移植(ASCT)后给予环磷酰胺是否会增加细菌、真菌、病毒感染、并发症(出血性膀胱炎(HC))和感染相关死亡率:这是一项回顾性多中心研究。45名接受环磷酰胺+甲氨蝶呤+钙调素抑制剂移植后的MMUD ASCT患者与45名接受甲氨蝶呤+钙调素抑制剂移植后的MRD ASCT患者进行了比较:虽然PTCy组的中性粒细胞移植时间明显延长,但两组间的细菌感染率差异无统计学意义(PTCy组9例(20%),对照组8例(17.8%),P=0.778)。前 100 天的 CMV 感染分布情况相似(P=0.827),但对照组在第 100 天和第 365 天之间的 CMV 感染率分布更频繁(P=0.005)。两组的 HC 感染率及其等级相似(PTCy:4(8.8%),对照组:6(13.3%),P=0.502)。PTCy 组和对照组的 VZV 感染率和侵袭性曲霉菌病发生率相似(PTCy 组为 13.3%,对照组为 17.8%,P=0.561)。各组间的生存分析(OS、LFS、GRFS、RI、IRM、NRM)也无统计学差异。然而,对照组的 cGVHD 发生率明显更高(P=0.035):结论:在MMUD ASCT中,在标准GvHD预防措施的基础上增加PTCy不会导致CMV再激活、细菌感染、侵袭性真菌感染、病毒性出血性膀胱炎或死亡率的增加。
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引用次数: 0
Antibody Response to Breakthrough SARS-CoV-2 Infection in "Booster" Vaccinated Patients with Multiple Myeloma According to B/T/NK Lymphocyte Absolute Counts and anti-CD38 Treatments. 根据 B/T/NK 淋巴细胞绝对数和抗 CD38 治疗,多发性骨髓瘤 "增效 "疫苗接种患者对突破性 SARS-CoV-2 感染的抗体反应。
IF 3.2 4区 医学 Q3 HEMATOLOGY Pub Date : 2024-03-01 eCollection Date: 2024-01-01 DOI: 10.4084/MJHID.2024.022
Nicola Sgherza, Anna Mestice, Angela Maria Vittoria Larocca, Pellegrino Musto
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引用次数: 0
Probable Autoimmune Lymphoproliferative Syndrome with Monogenic Lupus Due to KRAS Mutation. KRAS突变导致的伴有单基因狼疮的自身免疫性淋巴细胞增生综合征(Probable Autoimmune Lymphoproliferative Syndrome with Monogenic Lupus)。
IF 3.2 4区 医学 Q3 HEMATOLOGY Pub Date : 2024-03-01 eCollection Date: 2024-01-01 DOI: 10.4084/MJHID.2024.033
Amiya Nayak, Pratyusha Gudapati, Swapnil Tripathi, Jasmita Dass, Mukul Aggarwal, Pradeep Kumar
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引用次数: 0
Successful Treatment of De Novo Acute Myeloid Leukemia-Associated Aortitis by Induction Chemotherapy Alone. 单用诱导化疗成功治疗新发急性髓性白血病相关性大动脉炎
IF 3.2 4区 医学 Q3 HEMATOLOGY Pub Date : 2024-03-01 eCollection Date: 2024-01-01 DOI: 10.4084/MJHID.2024.025
Urbain Tauveron-Jalenques, Vincent Grobost, Benoît Magnin, Cécile Moluçon-Chabrot, Jacques-Olivier Bay, Olivier Tournilhac, Romain Guièze
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引用次数: 0
Internal Medicine Ward with Hematological Skills for the Treatment of Complications Suffered by Hematological Patients on Therapy: Experience of Villa Betania Hospital in Rome. 内科病房利用血液病治疗技能治疗血液病患者的并发症:罗马贝塔尼亚别墅医院的经验。
IF 3.2 4区 医学 Q3 HEMATOLOGY Pub Date : 2024-03-01 eCollection Date: 2024-01-01 DOI: 10.4084/MJHID.2024.030
A Andriani, L Marchetti, F Rossi, M A Perretti, S Raja, U Recine
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引用次数: 0
Plasmablastic Lymphoma. A State-of-the-Art Review: Part 2-Focus on Therapy. 浆细胞性淋巴瘤。最新进展回顾:第二部分--聚焦治疗。
IF 2 4区 医学 Q3 HEMATOLOGY Pub Date : 2024-03-01 eCollection Date: 2024-01-01 DOI: 10.4084/MJHID.2024.015
Michele Bibas

The objective of this two-part review is to present a current and comprehensive understanding of the diagnosis and management of plasmablastic lymphoma. The first part, which was published previously, focused on the study of epidemiology, etiology, clinicopathological characteristics, differential diagnosis, prognostic variables, and the impact of plasmablastic lymphoma on specific populations. This second part addresses the difficult topic of the treatment of plasmablastic lymphoma, specifically examining both the conventional, consolidated approach and the novel therapeutic strategy.

本综述由两部分组成,旨在介绍目前对浆细胞性淋巴瘤诊断和管理的全面认识。第一部分已经发表,重点研究流行病学、病因学、临床病理特征、鉴别诊断、预后变量以及浆细胞性淋巴瘤对特定人群的影响。第二部分探讨了浆细胞性淋巴瘤的治疗这一难题,特别研究了传统的综合治疗方法和新型治疗策略。
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引用次数: 0
Secondary Prophylaxis of Venous Thromboembolism (VTE) with Low Dose Apixaban or Rivaroxaban: Results from a Patient Population with More than 2 Years of Median Follow-up. 使用小剂量阿哌沙班或利伐沙班对静脉血栓栓塞症(VTE)进行二级预防:中位随访时间超过 2 年的患者群体的研究结果。
IF 3.2 4区 医学 Q3 HEMATOLOGY Pub Date : 2024-03-01 eCollection Date: 2024-01-01 DOI: 10.4084/MJHID.2024.020
Alessandro Laganà, Giovanni Manfredi Assanto, Chiara Masucci, Mauro Passucci, Livia Donzelli, Alessandra Serrao, Erminia Baldacci, Cristina Santoro, Antonio Chistolini

Background: Direct oral anticoagulants (DOACs) are widely used for the treatment and secondary prophylaxis of venous thromboembolism (VTE). Nowadays, DOACs represent the gold standard for long-term anticoagulation, with low-intensity DOACs administration becoming increasingly used worldwide in such scenario. Albeit low-intensity apixaban and rivaroxaban are approved for clinical usage as secondary VTE prophylaxis, there are few literature data regarding their efficacy and safety with a long follow-up.

Objectives: The aim of our study was to evaluate the efficacy and safety of low-dose DOACs for VTE secondary prophylaxis in patients at high risk of VTE recurrence.

Methods: We retrospectively evaluated patients who required long-term anticoagulant secondary prophylaxis to prevent recurrent VTE, treated with apixaban 2.5 mg BID or rivaroxaban 10 mg daily with a follow-up ≥ 12 months.

Results: The examined patients were 323. The median low-dose DOAC administration time was 25.40 months (IQR 13.93-45.90). Twelve (3.7%) VTE recurrences were observed; 21 bleeding events were registered (6.5%), including one episode of Major bleeding (MB) (0.3%), 8 Clinically relevant nonmajor bleeding (CRNMB) (2.5%) and 12 minor bleeding (3.7%). No statistically significant difference in the rate of VTE recurrence and/or bleeding events emerged between the rivaroxaban and apixaban groups. Patients included in the study for multiple episodes of VTE presented a significantly higher risk of a new VTE recurrence during low-intensity DOAC.

Conclusions: Our data suggest that low-dose DOACs may be effective and safe in secondary VTE prophylaxis in patients at high risk of VTE recurrence; however, attention might be needed in their choice in such a scenario for patients who experienced multiple episodes of VTE.

背景:直接口服抗凝剂(DOACs)被广泛用于静脉血栓栓塞症(VTE)的治疗和二级预防。如今,DOACs 已成为长期抗凝治疗的黄金标准,而低强度 DOACs 的使用在全球范围内也日益增多。尽管低强度的阿哌沙班和利伐沙班已被批准用于二级 VTE 预防的临床治疗,但有关其长期随访疗效和安全性的文献数据却很少:我们的研究旨在评估低剂量 DOACs 在 VTE 复发高风险患者中用于 VTE 二级预防的有效性和安全性:我们对需要长期抗凝剂二级预防以防止VTE复发的患者进行了回顾性评估,这些患者接受了阿哌沙班2.5毫克/日或利伐沙班10毫克/日的治疗,随访时间≥12个月:接受检查的患者有 323 人。低剂量 DOAC 的中位用药时间为 25.40 个月(IQR 13.93-45.90)。观察到12例(3.7%)VTE复发;21例出血事件(6.5%),包括1例大出血(MB)(0.3%)、8例临床相关性非大出血(CRNMB)(2.5%)和12例轻微出血(3.7%)。利伐沙班组和阿哌沙班组的 VTE 复发率和/或出血事件发生率在统计学上没有明显差异。因多次VTE发作而被纳入研究的患者在服用低剂量DOAC期间出现新的VTE复发的风险明显更高:我们的数据表明,低剂量 DOAC 在 VTE 复发高风险患者的二次 VTE 预防中可能是有效且安全的;然而,在这种情况下,需要注意为多次发作 VTE 的患者选择 DOAC。
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引用次数: 0
Clinical Characteristics and Treatment Response of a Novel ELANE Gene Mutation (c.295_303del) in Congenital Neutropenia. 先天性中性粒细胞减少症中新型 ELANE 基因突变(c.295_303del)的临床特征和治疗反应。
IF 3.2 4区 医学 Q3 HEMATOLOGY Pub Date : 2024-03-01 eCollection Date: 2024-01-01 DOI: 10.4084/MJHID.2024.024
Junjie Ning
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引用次数: 0
CAR-T Cell Therapy for T-Cell Malignancies. 治疗 T 细胞恶性肿瘤的 CAR-T 细胞疗法。
IF 3.2 4区 医学 Q3 HEMATOLOGY Pub Date : 2024-03-01 eCollection Date: 2024-01-01 DOI: 10.4084/MJHID.2024.031
Ugo Testa, Patrizia Chiusolo, Elvira Pelosi, Germana Castelli, Giuseppe Leone

Chimeric antigen receptor T-cell (CAR-T) therapy has revolutionized the treatment of B-cell lymphoid neoplasia and, in some instances, improved disease outcomes. Thus, six FDA-approved commercial CAR-T cell products that target antigens preferentially expressed on malignant B-cells or plasma cells have been introduced in the therapy of B-cell lymphomas, B-ALLs, and multiple myeloma. These therapeutic successes have triggered the application of CAR-T cell therapy to other hematologic tumors, including T-cell malignancies. However, the success of CAR-T cell therapies in T-cell neoplasms was considerably more limited due to the existence of some limiting factors, such as: 1) the sharing of mutual antigens between normal T-cells and CAR-T cells and malignant cells, determining fratricide events and severe T-cell aplasia; 2) the contamination of CAR-T cells used for CAR transduction with malignant T-cells. Allogeneic CAR-T products can avoid tumor contamination but raise other problems related to immunological incompatibility. In spite of these limitations, there has been significant progress in CD7- and CD5-targeted CAR-T cell therapy of T-cell malignancies in the last few years.

嵌合抗原受体 T 细胞(CAR-T)疗法彻底改变了 B 细胞淋巴肿瘤的治疗方法,并在某些情况下改善了疾病的预后。因此,在治疗 B 细胞淋巴瘤、B-ALL 和多发性骨髓瘤方面,已有六种针对恶性 B 细胞或浆细胞上优先表达的抗原的 CAR-T 细胞产品获得了美国食品及药物管理局(FDA)的批准。这些治疗上的成功引发了 CAR-T 细胞疗法在其他血液肿瘤(包括 T 细胞恶性肿瘤)上的应用。然而,由于存在一些限制因素,CAR-T 细胞疗法在 T 细胞肿瘤中的成功受到了相当大的限制,这些限制因素包括1)正常T细胞和CAR-T细胞与恶性细胞之间共享抗原,决定了自相残杀事件和严重的T细胞增生;2)用于CAR转导的CAR-T细胞被恶性T细胞污染。同种异体 CAR-T 产品可以避免肿瘤污染,但会引发其他与免疫不相容有关的问题。尽管存在这些局限性,但在过去几年中,CD7 和 CD5 靶向 CAR-T 细胞治疗 T 细胞恶性肿瘤取得了重大进展。
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引用次数: 0
期刊
Mediterranean Journal of Hematology and Infectious Diseases
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