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Efficacy and safety of primary letermovir prophylaxis for cytomegalovirus infection in paediatric patients undergoing allogeneic transplantation: a single-centre, retrospective, real-world analysis.
Q4 Health Professions Pub Date : 2025-03-14 eCollection Date: 2025-01-01 DOI: 10.46989/001c.131683
Xin Wang, Chaoqian Jiang, Lipeng Liu, Xia Chen, Yuanyuan Ren, Yang Wan, Aoli Zhang, Xiaoyan Zhang, Yue Shang, Yao Zou, Xiaojuan Chen, Fang Liu, Wenyu Yang, Xiaofan Zhu, Ye Guo

Background: Cytomegalovirus (CMV) infection is a common and life-threatening complication following allogeneic haematopoietic stem cell transplantation (allo-HSCT). Letermovir (LET) has been the standard prophylaxis for adult recipients, but studies in children remain limited.

Methods: We retrospectively analyzed children with or without LET prophylaxis after haploidentical donor (HID) for the Beijing protocol or unrelated cord blood (UCB) transplantation.

Results: Of the 151 patients, 67 received LET, including 35 HID recipients and 32 UCB recipients. During the 180 days after transplantation, we found that the LET group had a lower incidence of clinically significant CMV infection (csCMVi) than the non-LET group (13.4% vs. 56.0%, P<0.001). In the LET group, later LET administration was identified as a risk factor for the occurrence of csCMVi (HR: 1.07, 95% CI: 1.01 - 1.14, P=0.029). Further, the HID subgroup had a lower incidence of csCMVi during follow-up than the UCB subgroup (2.9% vs. 25.0%, P=0.009). In terms of safety, the incidence and severity of adverse events, overall survival, cumulative incidence of relapse, relapse free survival, nonrelapse mortality and graft versus host disease-free, relapse-free survival were similar between the two groups.

Conclusion: LET is effective and safe in preventing csCMVi among Chinese children undergoing allo-HSCT. Compared to UCB recipients, children undergoing HID transplantation for the Beijing protocol develop less scCMVi up to 180 days post-HSCT.

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引用次数: 0
Therapeutics of acute myeloid leukemia with central nervous system involvement.
Q4 Health Professions Pub Date : 2025-03-11 eCollection Date: 2025-01-01 DOI: 10.46989/001c.131722
Edwin U Suárez, Tamara Castaño-Bonilla, Rocio Salgado, Laura Solán, Alberto Lázaro-García, Juan M Alonso-Domínguez

FLT3-mutated acute myeloid leukemia (AML) with central nervous system (CNS) involvement poses therapeutic challenges. We describe two cases and performed a systematic review evaluating the efficacy of therapeutic strategies in CNS involvement for both FLT3-mutated and wild-type (WT) AML. A MEDLINE, EMBASE, and Cochrane literature search identified relevant studies. Although CNS involvement in AML is associated with poor prognosis, routine CNS prophylaxis is not standard. Due to the uncertainty regarding the effect of intermediate doses of cytarabine on CNS involvement, we support a diagnostic lumbar puncture (LP) after achieving complete remission in patients with risk factors for CNS infiltration. Consolidation management should be modified depending on the result of the LP. The impact of total body irradiation (TBI) as a conditioning regimen in allogeneic stem cell transplantation on CNS AML outcomes remains ambiguous. Routine craniospinal irradiation is not recommended due to its associated higher morbidity rates, while cranial radiotherapy is preferred, particularly when combined with TBI. Fortunately, currently we can employ a FLT3 inhibitor with CNS penetrance in FLT3-mutated (either gilteritinib or sorafenib) or FLT3-WT (sorafenib) AML patients.

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引用次数: 0
Frontline management of multiple myeloma patients: optimizing treatment for patients in the Gulf region. 多发性骨髓瘤患者的一线治疗:优化海湾地区患者的治疗。
Q4 Health Professions Pub Date : 2025-02-11 eCollection Date: 2025-01-01 DOI: 10.46989/001c.128113
Mahmoud Marashi, Khalil Al Farsi, Hussni Al Hateeti, Ahmad Alhuraiji, Hesham Elsabah, Honar Cherif, Anas Hamad, Kayane Mheidly, Hani Osman, Mohamad Mohty

Treatment options for newly diagnosed multiple myeloma (NDMM) have expanded dramatically over the last two decades, resulting in remarkable improvements in response rates and median survival times. In eligible patients, autologous stem cell transplant plays the central role of an overall treatment strategy comprising induction, transplantation, consolidation, and maintenance. In this article, we draw from our own collective clinical experience of treating patients with NDMM in the Gulf region to discuss treatment strategies in both transplant-eligible and -ineligible patients, as well as in high-risk patients. We present position statements for these distinct patient populations specifically for treatment in the Gulf region, where patients with NDMM have a younger median age than and different comorbidity profile from Western populations. We discuss how the introduction of anti-CD38 agents, including daratumumab and isatuximab, have had a major impact on the frontline treatment landscape in MM, with daratumumab-based quadruplet and triplet regimens emerging as the new standard of care in transplant-eligible and -ineligible patients, respectively. In addition, we advocate aggressive quadruplet treatment of high-risk patients with NDMM, as part of a strategy including single or tandem transplant when eligible. Finally, we discuss the clinical and practical rationale behind our statements, which is intended to serve as a useful reference for hematologists treating physicians within the Gulf region and beyond.

在过去二十年里,新诊断的多发性骨髓瘤(NDMM)的治疗方案急剧增加,反应率和中位生存时间显著改善。对于符合条件的患者,自体干细胞移植在包括诱导、移植、巩固和维持在内的整体治疗策略中发挥着核心作用。在这篇文章中,我们从海湾地区治疗NDMM患者的临床经验出发,讨论了符合移植条件和不符合移植条件的患者以及高危患者的治疗策略。在海湾地区,NDMM 患者的中位年龄比西方人小,合并症情况也与西方人不同。我们讨论了抗 CD38 药物(包括达拉土单抗和伊沙妥昔单抗)的引入如何对 MM 的一线治疗格局产生重大影响,基于达拉土单抗的四联疗法和三联疗法分别成为符合移植条件和不符合移植条件患者的新治疗标准。此外,我们还提倡对高风险的NDMM患者进行积极的四联疗法,作为包括符合条件的单次或串联移植在内的策略的一部分。最后,我们讨论了我们声明背后的临床和实用原理,希望能为海湾地区内外的血液科医生提供有益的参考。
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引用次数: 0
How to perform a high-quality peer review.
Q4 Health Professions Pub Date : 2025-02-03 eCollection Date: 2025-01-01 DOI: 10.46989/001c.128601
Mohamad Mohty, Junia V Melo

High-quality peer review is a cornerstone of credible and impactful scientific and medical publishing. This manuscript provides a comprehensive overview of the best practices, responsibilities, and evaluation criteria for peer reviewers in clinical and translational research. By adhering to high standards of objectivity, rigor and professionalism, peer reviewers support the integrity of scientific research and contribute to the evolution of evidence-based medicine. We elaborate on the principles and structured processes that ensure a thorough, impartial review, aiming to guide reviewers in producing evaluations that enrich the scientific discourse and foster innovation in clinical practice.

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引用次数: 0
Delivering bad news in clinical hematology: a personal perspective. 在临床血液学中传递坏消息:个人观点。
Q4 Health Professions Pub Date : 2024-12-21 eCollection Date: 2024-01-01 DOI: 10.46989/001c.126214
Mohamad Mohty, Finn Bo Petersen, Didier Blaise
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引用次数: 0
Role of cytomegalovirus reactivation on relapse after allogeneic hematopoietic stem cell transplantation in acute lymphoblastic leukemia. 巨细胞病毒再激活在异基因造血干细胞移植后急性淋巴细胞白血病复发中的作用。
Q4 Health Professions Pub Date : 2024-12-05 eCollection Date: 2024-01-01 DOI: 10.46989/001c.125912
Sabrine Mekni, Nour Ben Abdeljelil, Rihab Ouerghi, Rimmel Yosra Kanoun, Siwar Frigui, Dorra Belloumi, Insaf Ben Yaiche, Ines Turki, Anna Chabaane, Lamia Torjemane, Tarek Ben Othman

Cytomegalovirus reactivation (CMV-R) is a frequent complication post-allogeneic hematopoietic stem cell transplantation (allo-HSCT), associated with poor outcomes. Previous studies have demonstrated the protective effect of CMV-R against relapse after allo-HSCT for acute myeloblastic leukemia (AML). However, this impact remains unclear in acute lymphoblastic leukemia (ALL). We conducted a retrospective study on 81 patients with ALL who received allo-HSCT after myeloablative conditioning regimen from matched sibling donors between 2016 and 2022. All patients underwent weekly monitoring for CMV-R by quantitative polymerase chain reaction assay from engraftment until day +100 post allo-HSCT, and received antiviral prophylaxis with acyclovir from day +1 to 6 months after allo-HSCT. Preemptive treatment was initiated when a viremia was higher than 150 copies/mL. The median age was 20 years (range, 5-50 years). After allo-HSCT, 35% of patients developed CMV-R after a median of 39 days (range, 19-100 days). After a median follow-up of 30 months (range, 1-93 months), CMV-R was an independent factor associated with lower cumulative incidence of relapse (CIR) (OR: 0.17; 95% CI [0.03 - 0.98], p = 0.04) without survival benefit. Further studies are needed to validate the protective effect of CMV-R on ALL relapse.

巨细胞病毒再激活(CMV-R)是同种异体造血干细胞移植(alloo - hsct)后常见的并发症,与不良预后相关。先前的研究已经证明CMV-R对急性髓母细胞白血病(AML)的同种异体造血干细胞移植后复发具有保护作用。然而,这种影响在急性淋巴细胞白血病(ALL)中尚不清楚。我们对2016年至2022年间来自匹配的兄弟姐妹供体的81例ALL患者进行了回顾性研究,这些患者在清骨髓调节方案后接受了同种异体造血干细胞移植。所有患者从移植到移植后第100天,通过定量聚合酶链反应法每周监测CMV-R,并在移植后第1天至第6个月接受阿昔洛韦抗病毒预防治疗。当病毒血症高于150拷贝/mL时,开始先发制人的治疗。中位年龄为20岁(范围5-50岁)。同种异体造血干细胞移植后,35%的患者在中位39天(范围19-100天)后出现CMV-R。中位随访30个月(范围1-93个月)后,CMV-R是与较低的累积复发发生率(CIR)相关的独立因素(OR: 0.17;95% CI [0.03 - 0.98], p = 0.04),无生存获益。需要进一步的研究来验证CMV-R对ALL复发的保护作用。
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引用次数: 0
Social media in medicine: a personal perspective about opportunities, challenges, and best practice. 医学社交媒体:从个人角度看机遇、挑战和最佳实践。
Q4 Health Professions Pub Date : 2024-11-23 eCollection Date: 2024-01-01 DOI: 10.46989/001c.125931
Mohamad Mohty, Bipin Savani
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引用次数: 0
Nurses' reported training needs for advanced cell therapies: a survey on behalf of the Nurses Group of the EBMT. 护士报告的高级细胞疗法培训需求:代表 EBMT 护士小组进行的调查。
Q4 Health Professions Pub Date : 2024-11-15 eCollection Date: 2024-01-01 DOI: 10.46989/001c.124593
Michelle Kenyon, Sarah Jayne Liptrott, Annika Kisch, Jarl Mooyaart, Brian Piepenbroek, Daphna Hutt, Isabel Salcedo, Annalisa Ruggeri, Cristian Chabannon, Rose Ellard, John Murray

Background: Advanced Therapy Medicinal Products (ATMPs) for human use have advanced globally with the rapid adoption of Chimeric Antigen Receptor T-cell (CAR-T) therapies in haemato-oncology. CAR-T cell therapy and ATMPs have unique, significant acute and chronic toxicities, and appropriate patient care is crucial. Significant challenges, including the need for nurse education and training, accompany optimal patient success and benefits.

Objectives: This study aimed to describe nurses' training needs in relation to ATMP management and patient care.

Methods: A cross-sectional online survey was performed by the European Society for Blood and Marrow Transplantation, based on a previously tested questionnaire developed in the UK.

Findings: 109 complete responses from 86 different centers from 24 countries were returned (1207 distributed). Over 1/3 reported experience delivering licensed ATMPs (CAR-T). High-priority training areas included a general introduction to ATMPs, toxicity management, product-specific information, and regulatory frameworks for ATMPs. A clear need for ATMP-specific training exists and is regarded as important. Training prior to implementation is key and should be supported by ongoing competency maintenance. Counseling, patient support, and long-term follow-up are identified for future training and opportunities for nurse experience sharing in this rapidly evolving field.

背景:随着嵌合抗原受体 T 细胞(CAR-T)疗法在血液肿瘤学领域的快速应用,人类使用的先进治疗药物产品(ATMP)在全球范围内取得了进展。CAR-T 细胞疗法和 ATMP 具有独特、显著的急性和慢性毒性,因此适当的患者护理至关重要。在为患者带来最佳疗效和益处的同时,也面临着巨大的挑战,包括需要对护士进行教育和培训:本研究旨在描述护士在 ATMP 管理和患者护理方面的培训需求:方法:欧洲血液与骨髓移植学会根据之前在英国开发的测试问卷进行了一项横断面在线调查:共收到来自 24 个国家 86 个不同中心的 109 份完整回复(共发放 1207 份)。超过 1/3 的人报告说他们有提供获得许可的 ATMP(CAR-T)的经验。优先培训领域包括 ATMP 的一般介绍、毒性管理、特定产品信息和 ATMP 的监管框架。对ATMP特定培训的明显需求存在,并被视为非常重要。实施前的培训是关键,并应辅以持续的能力维护。咨询、患者支持和长期随访是未来培训的重点,也是护士在这一快速发展的领域分享经验的机会。
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引用次数: 0
Untangling the clonal architecture in a case of acute myeloid leukaemia with multiple cytogenetically unrelated clones. 解开一例急性髓性白血病患者的细胞遗传无关克隆结构。
Q4 Health Professions Pub Date : 2024-11-12 eCollection Date: 2024-01-01 DOI: 10.46989/001c.124931
Helen Cashman, Andrew J Wilson, Ke Xu, Elisabeth Nacheva, Robert Baker, Rajeev Gupta
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引用次数: 0
Management of multiple myeloma presenting as malignant spinal cord compression during pregnancy: a case report. 妊娠期多发性骨髓瘤恶性脊髓压迫的处理:病例报告。
Q4 Health Professions Pub Date : 2024-10-23 eCollection Date: 2024-01-01 DOI: 10.46989/001c.124664
Khushnuma Mullanfiroze, Charlotte Jones, David Williams, Matias Vieira, Ashutosh Wechalekar, Xenofon Papanikolaou, Rakesh Popat, Charalampia Kyriakou, Ke Xu

not included as this is letter to the editor.

由于这是一封致编辑的信,因此不包括在内。
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引用次数: 0
期刊
Clinical hematology international
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