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What should be the optimal dose of post-transplantation cyclophosphamide for GVHD prophylaxis in allogeneic stem cell transplantation? 同种异体干细胞移植预防GVHD的移植后环磷酰胺的最佳剂量是多少?
IF 1.4 4区 医学 Q4 HEMATOLOGY Pub Date : 2024-12-17 DOI: 10.1016/j.transci.2024.104058
Turgay Ulas, Sinem Namdaroglu, Ipek Yonal Hindilerden, Mehmet Ali Erkurt, Kerim Erer, Tugce Nur Yigenoglu, Tarik Onur Tiryaki, Emine Hidayet, Serdal Korkmaz, Bahar Uncu Ulu, Seda Yilmaz, Emin Kaya, Mehmet Sezgin Pepeler, Abdulkadir Basturk, Mehmet Sinan Dal, Fevzi Altuntas

Objectives: In this study, we aimed to compare the engraftment days, graft versus host disease (GVHD) development, relapse and overall survival (OS) rates in patients using variable intensity conditioning regimens with two different post-transplant cyclophosphamide (PTCy) doses for hematological malignancies.

Material and methods: We retrospectively analyzed 162 patients who have had PTCy at a dose of 25 mg/kg × 2 and 50 mg/kg × 2 between 2018 and 2024. Patients were divided in 2 groups; PTCy dose with 25 mg/kg × 2 (Group 1, n = 45) and PTCy dose with 50 mg/kg × 2 (Group 2, n = 117). The engraftment days, GVHD, relapse and OS rates were compared across groups.

Results: All patients had myeloablative conditioning regimens and peripheral stem cell collected transplantation. 61.1 % of patients (n = 99) were alive at the end of the study (60 % (n = 27) in Group1 and 61.5 % (n = 72) in Group 2). In Group 1 the median follow-up was 6.9 months and in Group 2 this was 7 months; the median OS was 15.5 months in Group 1 and 49.5 months in Group 2 but this is not statistically significant (Log rank = 0.796). In Group 1, the engraftment times for platelets was 13 days, for neutrophils 17 days; in Group 2, for platelet this was 18 days; and for neutrophils 17 days; this was statistically significant for platelets but not for neutrophil engraftment (p: < 0.001 and p:0.839, respectively). Eighteen patients (40 %) in Group 1 and twenty-seven (23 %) patients in group 2 had acute GVHD (aGVHD). In Group 1 aGVHD rates were higher than Group 2 (p = 0.031). Seven patients (15.5 %) in Group 1 and 6 (5.12 %) patients in group 2 had chronic GVHD (cGVHD). In Group 1 cGVHD rates were also higher than Group 2 (p = 0.048). Twenty-five patients (55.6 %) in Group 1 and 19 patients (16.2 %) in Group 2 had relapsed disease (p < 0.001).

Conclusion: Our study showed that there were no differences in survival across the groups. The platelet engraftment time was shorter for the PTCy 25 mg/kg × 2 doses compared to the post-transplantation 50 mg/kg × 2 doses. Both aGVHD and cGVHD rates were higher in 25 mg/kg × 2 dose treated patients. Relapses occurred more commonly with 25 mg/kg × 2 PTCy dose.

目的:在这项研究中,我们旨在比较使用两种不同剂量环磷酰胺(PTCy)的可变强度调节方案治疗血液系统恶性肿瘤患者的移植天数、移植物抗宿主病(GVHD)的发展、复发和总生存率。材料和方法:我们回顾性分析了2018年至2024年间接受PTCy剂量为25 mg/kg × 2和50 mg/kg × 2的162例患者。患者分为两组;PTCy剂量为25 mg/kg × 2(第一组,n = 45)和50 mg/kg × 2(第二组,n = 117)。比较各组移植时间、GVHD、复发率和OS率。结果:所有患者均行清髓调理方案和外周血干细胞收集移植。61.1 %的患者(n = 99)还活着在研究结束时(60 % (n = 27)在Group1和61.5 %组(n = 72)2)。在组1组2中的平均随访6.9个月,这是7个月;第1组中位OS为15.5个月,第2组中位OS为49.5个月,但差异无统计学意义(Log rank = 0.796)。1组血小板移植时间为13 d,中性粒细胞移植时间为17 d;在组2中,血小板为18天;中性粒细胞17天;这在血小板中有统计学意义,但在中性粒细胞移植中无统计学意义(p分别< 0.001和p:0.839)。1组18例(40 %),2组27例(23 %)出现急性GVHD (aGVHD)。1组aGVHD发生率高于2组(p = 0.031)。1组7例(15.5 %),2组6例(5.12 %)存在慢性GVHD (cGVHD)。1组cGVHD发生率高于2组(p = 0.048)。第1组25例(55.6% %)和第2组19例(16.2% %)复发(p )。结论:本研究显示两组患者的生存率无差异。血小板植入时间在PTCy 25 mg/kg × 2剂量组较移植后50 mg/kg × 2剂量组短。25 mg/kg × 2剂量组aGVHD和cGVHD发生率均较高。当PTCy剂量为25 mg/kg × 2时,复发更为常见。
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引用次数: 0
Outcomes in patients with thrombotic microangiopathy associated with a trigger following plasma exchange: A systematic literature review. 血浆置换后与触发相关的血栓性微血管病患者的预后:系统文献综述。
IF 1.4 4区 医学 Q4 HEMATOLOGY Pub Date : 2024-12-17 DOI: 10.1016/j.transci.2024.104048
Shinya Kaname, Moh-Lim Ong, Jonathan Mathias, Francesca Gatta, Lisa Law, Yan Wang

Plasma exchange (PE) outcomes in patients with trigger-associated thrombotic microangiopathy (TMA) have not been comprehensively reviewed. Embase and MEDLINE® were searched on 03/14/2022 for English language articles published after 2007, alongside a congress materials search (2019-2022; PROSPERO: CRD42022325170). Studies with patients with trigger-associated TMA (excluding thrombotic thrombocytopenic purpura, 'typical' hemolytic uremic syndrome caused by Shiga toxin-producing Escherichia coli, post-partum TMA, and TMAs with known genetic cause) who received PE or plasma infusion (PI) and reported treatment response (including measures), safety, patient-/caregiver-reported outcomes, or economic burden data were examined. The NICE quality appraisal checklist assessed bias risk. After screening 695 articles, 49 PE or PI studies were identified, of which 42 reported PE exclusively; most were retrospective observational studies (n = 37). The most common TMA trigger was transplantation (n = 12). The median number of PE sessions was 3.5-25.0. Outcomes following PE varied by trigger type. Treatment response rates and definitions varied (0-100 %; 24 studies); in studies of > 25 patients, response rates were 5-63 %. TMA relapse rates were 0-67 % (7 studies). Mortality was 10-91 % (23 studies). Progression to chronic kidney disease (CKD; 5 studies) and end-stage renal disease (ESRD; 6 studies) occurred in 0-93 % and 17-100 % of patients, respectively. Two serious adverse events were identified (transfusion-related injury, acute lung injury; 10 studies; 231 patients). Patients with trigger-associated TMA may experience a substantial burden in terms of mortality, relapse, and progression to CKD and ESRD following PE, leading to increased healthcare resource utilization. Additional interventions may be required.

触发相关血栓性微血管病(TMA)患者的血浆交换(PE)结果尚未全面审查。我们于2022年3月14日检索了Embase和MEDLINE®,检索了2007年以后发表的英语文章,同时检索了会议资料(2019-2022;普洛斯彼罗:CRD42022325170)。对接受PE或血浆输注(PI)并报告治疗反应(包括措施)、安全性、患者/护理人员报告的结果或经济负担数据的诱发性TMA患者(不包括血栓性血小板减少性紫癜、由产志贺毒素大肠杆菌引起的“典型”溶血性尿毒症综合征、产后TMA和已知遗传原因的TMA)进行研究。NICE质量评估清单评估偏倚风险。在筛选695篇文章后,鉴定出49篇PE或PI研究,其中42篇专门报道PE;大多数为回顾性观察性研究(n = 37)。最常见的TMA触发因素是移植(n = 12)。体育课程的中位数为3.5-25.0次。PE后的结果因触发类型而异。治疗反应率和定义各不相同(0-100 %;24个研究);在bb0 25例患者的研究中,有效率为5-63 %。TMA复发率为0-67 %(7项研究)。死亡率为10-91 %(23项研究)。进展为慢性肾脏疾病(CKD);5项研究)和终末期肾病(ESRD;6项研究)分别发生在0-93 %和17-100 %的患者中。发现了两个严重不良事件(输血相关损伤、急性肺损伤;10的研究;231名患者)。与触发相关的TMA患者在PE后的死亡率、复发和CKD和ESRD进展方面可能会经历巨大的负担,导致医疗资源利用率增加。可能需要额外的干预措施。
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引用次数: 0
A Comprehensive SWOT and TOWS analysis of transfusion medicine: Indian Perspective. 输血医学的SWOT和TOWS综合分析:印度视角。
IF 1.4 4区 医学 Q4 HEMATOLOGY Pub Date : 2024-12-17 DOI: 10.1016/j.transci.2024.104061
Akshay Chopra, Shamee Shastry, Ganesh Mohan, Chenna Deepika, Vinu Rajendran

Background and objective: SWOT and TOWS analysis is a strategic planning tool for identifying internal and external factors influencing a field. It helps to identify areas for improvement and growth by maximizing strengths and opportunities while minimizing weaknesses and threats. Transfusion medicine specialists can better understand the field's current status and plan potential growth using SWOT and TWOS strategic tools.

Methods: A cross-sectional questionnaire-based study using Google Forms, was conducted among Transfusion Medicine specialists in India. The authors formulated five open-ended questions for each of the S, W, O, and T categories to address and analyze the main aspects of Strengths, Weaknesses, Opportunities, and Threats. Descriptive statistics analyzed demographic details and keywords, extracting factors for SWOT and TOWS analysis to develop strategies.

Results: 71 specialists, across India: Central (8), East (7), North (18), South (31), and West (7) responded, achieving a 48 % response rate. Strengths focused on Immunohematology and blood safety. Conversely, weaknesses highlighted were limited clinical exposure and insufficient recognition within the medical fraternity. Fostering interdisciplinary collaboration and integrating emerging advancements were key opportunities identified. The prevailing concerns encompassed two primary threats: the potential overlap with related branches like pathology, and the emergence of artificial blood products. The SO, ST, WO, and WT strategies focused on apheresis, recruitment policy, clinical exposure, and newer developments, respectively.

Conclusion: This study highlights the application of SWOT and TWOS analysis as a valuable strategic tool for Transfusion Medicine specialists to address these factors proactively and to enhance the field's trajectory.

背景和目的:SWOT 和 TOWS 分析是一种战略规划工具,用于确定影响某一领域的内部和外部因素。它通过最大限度地利用优势和机会,同时最大限度地减少劣势和威胁,帮助确定需要改进和发展的领域。输血医学专家可以利用 SWOT 和 TWOS 战略工具更好地了解该领域的现状并规划潜在的发展:使用谷歌表格对印度输血医学专家进行了横向问卷调查。作者分别针对 S、W、O 和 T 类别拟定了五个开放式问题,以解决和分析优势、劣势、机会和威胁的主要方面。描述性统计分析了人口详情和关键字,提取了用于 SWOT 和 TOWS 分析的因素,以制定战略:71 名专家来自印度各地:结果:印度各地的 71 名专家:中部(8 名)、东部(7 名)、北部(18 名)、南部(31 名)和西部(7 名)做出了回应,回应率为 48%。优势集中在免疫血液学和血液安全方面。反之,不足之处则是临床接触有限,在医学界的认可度不够。促进跨学科合作和整合新兴技术是已确定的主要机遇。普遍关注的问题包括两个主要威胁:与病理学等相关分支的潜在重叠,以及人造血液制品的出现。SO、ST、WO 和 WT 战略分别侧重于无细胞疗法、招聘政策、临床接触和最新发展:本研究强调了 SWOT 分析和 TWOS 分析的应用,它们是输血医学专家积极应对这些因素并改善该领域发展轨迹的重要战略工具。
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引用次数: 0
Message from the new WAA President. 来自WAA新主席的信息。
IF 1.4 4区 医学 Q4 HEMATOLOGY Pub Date : 2024-12-17 DOI: 10.1016/j.transci.2024.104047
Hans Vrielink, Joseph Schwartz
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引用次数: 0
Emerging trends and controversies in GVHD management: Bridging research and clinical practice. GVHD管理的新趋势和争议:衔接研究和临床实践。
IF 1.4 4区 医学 Q4 HEMATOLOGY Pub Date : 2024-12-17 DOI: 10.1016/j.transci.2024.104049
Fevzi Altuntas
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引用次数: 0
Biographies of the guest editors for the theme papers on graft-versus-host disease (GVHD). 关于移植物抗宿主病(GVHD)主题论文的客座编辑传记。
IF 1.4 4区 医学 Q4 HEMATOLOGY Pub Date : 2024-12-17 DOI: 10.1016/j.transci.2024.104050
Serdal Korkmaz, Fevzi Altuntas
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引用次数: 0
Efficacy of Ruxolitinib in the management of chronic GVHD. 鲁索利替尼治疗慢性GVHD的疗效观察。
IF 1.4 4区 医学 Q4 HEMATOLOGY Pub Date : 2024-12-17 DOI: 10.1016/j.transci.2024.104053
Asli Odabasi Giden, Mehmet Ali Erkurt, Ipek Yonal Hindilerden, Emine Hidayet, Ilhami Berber, Tarik Onur Tiryaki, Tugba Zorlu, Sinem Namdaroglu, Ahmet Sarici, Elif Aksoy, Esra Yildizhan, Muruvvet Seda Aydin, Serdal Korkmaz, Mehmet Sinan Dal, Turgay Ulas, Fevzi Altuntas

Objectives: Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is a potentially curative treatment for hematological diseases, with success rates improving due to advancements in conditioning regimens and new anti-graft versus host disease (GVHD) drugs. Ruxolitinib, an oral selective Janus kinase (JAK) 1 and 2 inhibitor has been used to mitigate the effects of various inflammatory and myeloproliferative syndromes, given the JAK kinase pathway's central role in cytokine signaling during inflammatory and immune processes. In this study we aimed to assess ruxolitinib's efficacy in patients with chronic GVHD (cGVHD).

Material and methods: This retrospective observational multi-center study involved 50 patients diagnosed with cGVHD after allo-HSCT in Turkey, who were treated with ruxolitinib between April 2018 and March 2024.

Results: At the time of initiation of ruxolitinib treatment, most patients had severe cGVHD (n = 29, 58 %). The overall response rate at 6 months of ruxolitinib treatment was observed in 34 patients (68 %), including 6 patients (12 %) with complete responses and 28 patients (56 %) with partial responses, while 7 patients (14 %) experienced treatment failure. ECOG (2-4) performance status was established as an independent risk factor for adverse outcomes [p = 0.029, HR 3.492 (95 % CI: 1.139-10.705)]. At the two-year follow-up, the estimated survival rate was 52 %.

Conclusion: Ruxolitinib is safe and effective in the real-world setting for treating cGVHD, showing remission rates comparable to clinical trials. Further research with extended follow-up is necessary to confirm these findings, optimize dosing, and establish the best tapering strategies for responders.

目的:同种异体造血干细胞移植(Allogeneic hematopoietic stem cell transplantation, alloo - hsct)是一种治疗血液病的潜在治疗方法,由于调节方案的进步和新的抗移植物抗宿主病(GVHD)药物,其成功率不断提高。Ruxolitinib是一种口服选择性Janus激酶(JAK) 1和2抑制剂,已被用于减轻各种炎症和骨髓增生综合征的影响,因为JAK激酶途径在炎症和免疫过程中细胞因子信号传导中起核心作用。在这项研究中,我们旨在评估ruxolitinib对慢性GVHD (cGVHD)患者的疗效。材料和方法:这项回顾性观察性多中心研究纳入了50例在土耳其接受同种异体造血干细胞移植后诊断为cGVHD的患者,这些患者在2018年4月至2024年3月期间接受了鲁索利替尼治疗。结果:ruxolitinib治疗开始时,大多数患者为重度cGVHD (n = 29,58 %)。34例患者(68 %)在ruxolitinib治疗6个月时观察到总缓解率,其中6例患者(12 %)完全缓解,28例患者(56 %)部分缓解,7例患者(14 %)治疗失败。ECOG(2-4)表现状态被确定为不良结局的独立危险因素[p = 0.029,HR 3.492(95 % CI: 1.139-10.705)]。在两年的随访中,估计存活率为52% %。结论:Ruxolitinib在治疗cGVHD的现实环境中是安全有效的,其缓解率与临床试验相当。有必要进行进一步的研究,延长随访时间,以证实这些发现,优化剂量,并为应答者建立最佳的逐渐减少策略。
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引用次数: 0
Platelet proteomics: Clinical implications - Decoding the black box! 血小板蛋白质组学:临床意义-解码黑盒子!
IF 1.4 4区 医学 Q4 HEMATOLOGY Pub Date : 2024-12-16 DOI: 10.1016/j.transci.2024.104060
Hadi Goubran, Shahid Ahmed, Gaafar Ragab, Jerard Seghatchian, Thierry Burnouf

Platelets are anucleate blood cells traditionally associated with hemostasis but now increasingly recognized for their multifaceted roles in immunity, inflammation, and tissue repair. Advances in platelet proteomics, employing high-throughput techniques such as mass spectrometry, have significantly enhanced our understanding of platelet biology and its clinical implications in transfusion medicine. Platelet proteomics offers a retrospective view of physiological and pathological changes over the platelet's 7-10-day lifespan, making it a unique tool for studying cumulative biological events. Recent applications include the identification of biomarkers for cardiovascular, infectious, autoimmune diseases and cancer. In neurodegeneration and aging, platelets have been explored for their shared molecular pathways with neurons, with findings implicating Tau, amyloid-beta, and alpha-synuclein as potential biomarkers. Proteomics is also emerging as an important factor in the development of evidence-based, tailor-made platelet-derived therapies. While promising, platelet proteomics requires further standardization and computational advances to support transitioning from research to routine clinical practice.

血小板是一种无核血细胞,传统上与止血有关,但现在越来越多地认识到其在免疫、炎症和组织修复中的多方面作用。血小板蛋白质组学的进步,采用高通量技术,如质谱,大大提高了我们对血小板生物学及其在输血医学中的临床意义的理解。血小板蛋白质组学提供了血小板在7-10天生命周期内的生理和病理变化的回顾性视图,使其成为研究累积生物学事件的独特工具。最近的应用包括心血管、传染病、自身免疫性疾病和癌症的生物标志物鉴定。在神经退行性变和衰老中,血小板与神经元共享的分子通路已被探索,发现Tau、淀粉样蛋白- β和α -突触核蛋白是潜在的生物标志物。蛋白质组学也正在成为基于证据、量身定制的血小板衍生疗法发展的重要因素。虽然有希望,血小板蛋白质组学需要进一步的标准化和计算进步,以支持从研究到常规临床实践的转变。
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引用次数: 0
Is there a relationship between vitamin D levels and graft versus host disease? 维生素D水平与移植物抗宿主病之间是否存在关系?
IF 1.4 4区 医学 Q4 HEMATOLOGY Pub Date : 2024-12-16 DOI: 10.1016/j.transci.2024.104054
Tugce Nur Yigenoglu, Bahar Uncu Ulu, Sinem Namdaroglu, Mehmet Ali Erkurt, Rasim Sahin, Nazik Okumus, Seda Yilmaz, Funda Ceran, Mehmet Koca, Ugur Hatipoglu, Mehmet Sinan Dal, Serdal Korkmaz, Turgay Ulas, Fevzi Altuntas

Objective: Vitamin D deficiency is common in adult patients undergoing allogenic hematopoietic stem cell transplantation (allo-HSCT). Since vitamin D is an important regulatory factor for the immune system, vitamin D deficiency may have effects on antitumor activity, relapse rates, graft versus host disease (GVHD) occurrence and infection rates in allo-HSCT. We aimed to investigate the effects of vitamin D levels on the outcome of allo-HSCT.

Material and methods: This study included 211 patients who underwent allo-HSCT at seven transplant centers in Türkiye. The impact of pretransplant vitamin D level on overall survival (OS), relapse rate, GVHD occurrence and engraftment times was analyzed retrospectively RESULTS: Pretransplant vitamin D levels were not related to the neutrophil engraftment day (p: 0.887), relapse rate (p: 0.433) and GVHD occurrence (p: 0.391). At a median follow-up of 14 months, OS was 84.8 % and median OS was not reached. Univariate Cox Regression analysis showed that higher levels of vitamin D (>12 ng/mL) affected the survival rates (p = 0.029) (HR: 0.392: 95 % CI: 10.170-0.907).

Conclusion: In our study, pretransplant vitamin D levels were not related to GVHD occurrence, relapse rate and engraftment times. However, we found that higher levels of pretransplant vitamin D levels (threshold is 12 ng/mL) were associated with increased survival. Further studies with a larger population are necessary to reveal the role of vitamin D in patients undergoing allo-HSCT.

目的:维生素D缺乏在接受同种异体造血干细胞移植(alloo - hsct)的成人患者中很常见。由于维生素D是免疫系统的重要调节因子,维生素D缺乏可能影响同种异体造血干细胞移植的抗肿瘤活性、复发率、移植物抗宿主病(GVHD)的发生和感染率。我们的目的是研究维生素D水平对同种异体造血干细胞移植结果的影响。材料和方法:本研究纳入了211例在泰国7个移植中心接受同种异体造血干细胞移植的患者。结果:移植前维生素D水平与中性粒细胞移植天数(p: 0.887)、复发率(p: 0.433)、GVHD发生(p: 0.391)无关。在中位随访14个月时,OS为84.8 %,中位OS未达到。单因素Cox回归分析显示,较高水平的维生素D(>12 ng/mL)影响存活率(p = 0.029)(HR: 0.392: 95 % CI: 10.170 ~ 0.907)。结论:在我们的研究中,移植前维生素D水平与GVHD的发生、复发率和移植次数无关。然而,我们发现移植前较高水平的维生素D水平(阈值为12 ng/mL)与生存率增加有关。需要在更大的人群中进行进一步的研究,以揭示维生素D在接受同种异体造血干细胞移植患者中的作用。
{"title":"Is there a relationship between vitamin D levels and graft versus host disease?","authors":"Tugce Nur Yigenoglu, Bahar Uncu Ulu, Sinem Namdaroglu, Mehmet Ali Erkurt, Rasim Sahin, Nazik Okumus, Seda Yilmaz, Funda Ceran, Mehmet Koca, Ugur Hatipoglu, Mehmet Sinan Dal, Serdal Korkmaz, Turgay Ulas, Fevzi Altuntas","doi":"10.1016/j.transci.2024.104054","DOIUrl":"https://doi.org/10.1016/j.transci.2024.104054","url":null,"abstract":"<p><strong>Objective: </strong>Vitamin D deficiency is common in adult patients undergoing allogenic hematopoietic stem cell transplantation (allo-HSCT). Since vitamin D is an important regulatory factor for the immune system, vitamin D deficiency may have effects on antitumor activity, relapse rates, graft versus host disease (GVHD) occurrence and infection rates in allo-HSCT. We aimed to investigate the effects of vitamin D levels on the outcome of allo-HSCT.</p><p><strong>Material and methods: </strong>This study included 211 patients who underwent allo-HSCT at seven transplant centers in Türkiye. The impact of pretransplant vitamin D level on overall survival (OS), relapse rate, GVHD occurrence and engraftment times was analyzed retrospectively RESULTS: Pretransplant vitamin D levels were not related to the neutrophil engraftment day (p: 0.887), relapse rate (p: 0.433) and GVHD occurrence (p: 0.391). At a median follow-up of 14 months, OS was 84.8 % and median OS was not reached. Univariate Cox Regression analysis showed that higher levels of vitamin D (>12 ng/mL) affected the survival rates (p = 0.029) (HR: 0.392: 95 % CI: 10.170-0.907).</p><p><strong>Conclusion: </strong>In our study, pretransplant vitamin D levels were not related to GVHD occurrence, relapse rate and engraftment times. However, we found that higher levels of pretransplant vitamin D levels (threshold is 12 ng/mL) were associated with increased survival. Further studies with a larger population are necessary to reveal the role of vitamin D in patients undergoing allo-HSCT.</p>","PeriodicalId":49422,"journal":{"name":"Transfusion and Apheresis Science","volume":"64 1","pages":"104054"},"PeriodicalIF":1.4,"publicationDate":"2024-12-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142878416","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
What is the role of alpha-1 antitrypsin in the management of acute graft versus host disease? α -1抗胰蛋白酶在急性移植物抗宿主病治疗中的作用是什么?
IF 1.4 4区 医学 Q4 HEMATOLOGY Pub Date : 2024-12-16 DOI: 10.1016/j.transci.2024.104057
Tugce Nur Yigenoglu, Mehmet Ali Erkurt, Simten Dagdas, Bahar Uncu Ulu, Irfan Kuku, Ali Durdu, Mehmet Sezgin Pepeler, Sinem Kul, Mehmet Sinan Dal, Emin Kaya, Serdal Korkmaz, Turgay Ulaş, Fevzi Altuntas

Objective: Acute graft versus host disease (GVHD) occurs in 20-80 % of patients undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT). Of these patients, 40 % will be resistant to steroids, which is the standard first-line approach. There is no standard second line treatment approach for patients with steroid refractory acute GVHD (SR-aGVHD). Alpha-1 antitrypsin is a protease inhibitor and has anti-inflammatory and immune regulatory properties. Here we report the outcomes and safety data of 17 patients treated with alpha-1 antitrypsin for SR-aGVHD.

Material and methods: Patients who received at least 2 lines of alpha-1 antitrypsin treatment for SR-aGVHD at five transplant centers in Türkiye were included in this retrospective study.

Results: The median number of alpha-1 antitrypsin treatment line patients received was 4 (range, 2-5). The median time between alpha-1 antitrypsin administration and response was 65 days (range, 10-138 days). Overall response rate was 70.6 %. When the first- and second-month response rates were compared according to GVHD organ involvement, we found that the response rates were similar in skin, liver and gastrointestinal system involvement (p = 0.281 and p = 0.305, respectively). No grade 3-4 anemia, thrombocytopenia or neutropenia was observed after alpha-1 antitrypsin treatment. Two patients had cytomegalovirus infection and 1 patient had pneumonia. At a median follow-up of 7 months, overall survival was 70.6 % and median overall survival was not reached.

Conclusion: In conclusion, alpha-1 antitrypsin is an effective and safe treatment option in patients with SR-aGVHD, with response rates of up to 70 % in patients with skin, liver and gastrointestinal system involvement. Larger studies are needed to establish a standard second and subsequent treatment approach in patients with SR-aGVHD.

目的:急性移植物抗宿主病(GVHD)发生在20- 80% %接受同种异体造血干细胞移植(alloo - hsct)的患者中。在这些患者中,40% %将对类固醇产生耐药性,这是标准的一线治疗方法。对于类固醇难治性急性GVHD (SR-aGVHD)患者,没有标准的二线治疗方法。α -1抗胰蛋白酶是一种蛋白酶抑制剂,具有抗炎和免疫调节特性。在这里,我们报告了17例使用α -1抗胰蛋白酶治疗SR-aGVHD的患者的结果和安全性数据。材料和方法:在日本5个移植中心接受至少2行α -1抗胰蛋白酶治疗的SR-aGVHD患者被纳入本回顾性研究。结果:α -1抗胰蛋白酶治疗系患者接受治疗的中位数为4例(范围2-5)。从α -1抗胰蛋白酶给药到缓解的中位时间为65天(范围10-138天)。总有效率为70.6 %。当根据GVHD受累器官比较第一个月和第二个月的反应率时,我们发现皮肤、肝脏和胃肠道系统受累的反应率相似(p = 0.281和p = 0.305)。α -1抗胰蛋白酶治疗后未见3-4级贫血、血小板减少或中性粒细胞减少。巨细胞病毒感染2例,肺炎1例。在中位随访7个月时,总生存率为70.6 %,中位总生存率未达到。结论:α -1抗胰蛋白酶是SR-aGVHD患者有效且安全的治疗选择,在累及皮肤、肝脏和胃肠道系统的患者中,其有效率高达70% %。需要更大规模的研究来确定SR-aGVHD患者的标准第二次和后续治疗方法。
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