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Hematopoietic cell transplantation in CR1 may help mitigate the adverse outcomes of Ph-like B-ALL in adult patients CR1患者的造血细胞移植可能有助于减轻成人患者ph样B-ALL的不良后果。
IF 3.6 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-12-01 DOI: 10.1016/j.jtct.2024.11.007
John C. Molina
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引用次数: 0
Risk-Based Qualification of Cellular Starting Material Collection Sites: Tools to Determine Scope of Manufacturer Audits and Reduce Duplicative Efforts 基于风险的细胞起始材料收集点资格认证:确定制造商审核范围和减少重复工作的工具。
IF 3.6 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-12-01 DOI: 10.1016/j.jtct.2024.09.006
Peter Holman , Kara Wacker , Linda Barnes , Joan Myers , Tracey Hlucky , Victoria Walters , Christina Anderson , William Shingler , Karishma Contractor
Cellular and Gene Therapy product (CGT product) manufacturers are required by regulations to qualify sites performing cell collection and processing as part of their manufacturing processes. The use of audits to qualify a site is part of the traditional supplier quality models for drug products. Due to the rapid growth of the CGT industry in recent years, healthcare institutions and manufacturers are finding it difficult to manage the increasing workload and resources needed to support audits when they are applied to the provision of Cellular Starting Material (CSM) collection from patients/subjects for the purpose of manufacturing. To alleviate this audit burden, several manufacturers have applied risk-based approaches to determine the needs and scope of audits. The authors of this commentary recommend that all manufacturers utilize a risk-based assessment program when appropriate and explain the use of tools created to facilitate a risk-based approach to streamline and reduce duplicative audits. This approach and tools, created by The NextGen Industry Working Group (IWG) site certification workstream with representation from pharmaceutical and biotech companies, health care institutions, accrediting organizations, and other stakeholders, is aligned with proposals from other multistakeholder groups, including the ASTCT 80/20 Task Force. The tools aim to streamline the site qualification processes performed by each manufacturer, offering a standardized approach to audits or gap analysis assessments. Offering an abbreviated audit model for sites that have already attained accreditation through agencies such as the AABB (Association for the Advancement of Blood & Biotherapies) and FACT (Foundation for the Accreditation of Cellular Therapy), helping the manufacturer to focus on product-specific requirements rather than re-evaluating systems that have already been audited in great detail by other parties.
根据法规要求,细胞和基因治疗产品(CGT 产品)生产商必须对进行细胞采集和处理的场所进行资格审查,这是生产过程的一部分。使用审核对生产基地进行资格认证是药品传统供应商质量模式的一部分。由于近年来 CGT 行业的快速发展,医疗机构和制造商发现,当审核适用于从患者/受试者处采集细胞起始材料 (CSM) 用于生产时,很难管理日益增加的工作量和所需资源。为了减轻审核负担,一些生产商采用了基于风险的方法来确定审核的需求和范围。本评论的作者建议所有生产商在适当的时候采用基于风险的评估计划,并解释了如何使用为促进基于风险的方法而创建的工具来简化和减少重复审核。这种方法和工具由 NextGen 行业工作组 (IWG) 现场认证工作流创建,其代表来自制药和生物技术公司、医疗机构、认证组织和其他利益相关者,与其他多利益相关者团体(包括 ASTCT 80/20 工作组)的建议相一致。这些工具旨在简化各生产商执行的生产基地资格认证流程,提供标准化的审核或差距分析评估方法。为已通过 AABB(血液与生物治疗促进协会)和 FACT(细胞治疗认证基金会)等机构认证的研究机构提供简短的审核模式,帮助制造商专注于特定产品的要求,而不是重新评估已通过其他机构详细审核的系统。
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引用次数: 0
Immune checkpoint inhibition and CAR T-cells: no longer exhausted? 免疫检查点抑制和CAR - t细胞:不再耗尽?
IF 3.6 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-12-01 DOI: 10.1016/j.jtct.2024.11.008
Janna Minehart , Elise A. Chong
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引用次数: 0
Officers and Directors of ASTCT
IF 3.6 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-12-01 DOI: 10.1016/S2666-6367(24)00755-3
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引用次数: 0
Prospective Assessment of Quality of Life and Patient-Reported Toxicities Over the First Year After Chimeric Antigen Receptor T-Cell Therapy 对 CAR T 细胞疗法后第一年的生活质量和患者报告的毒性进行前瞻性评估。
IF 3.6 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-12-01 DOI: 10.1016/j.jtct.2024.09.013
Aasha I. Hoogland , Anna Barata , Xiaoyin Li , Nathaly Irizarry-Arroyo , Michael D. Jain , Taylor Welniak , Yvelise Rodriguez , Laura B. Oswald , Lisa M. Gudenkauf , Julio C. Chavez , Farhad Khimani , Aleksandr Lazaryan , Hien D. Liu , Taiga Nishihori , Javier Pinilla-Ibarz , Bijal D. Shah , Sylvia L. Crowder , Nathan H. Parker , Tiffany L. Carson , Christine E. Vinci , Heather S.L. Jim
Chimeric antigen receptor (CAR) T-cell therapy has transformed survival outcomes in patients with relapsed and refractory large B-cell lymphoma (LBCL), but it is associated with a variety of side effects. This study examined changes in patient-reported quality of life (QoL) and toxicities, as well as risk factors for worse QoL and toxicities, in the first year after treatment. Patients with LBCL completed questionnaires assessing QoL and toxicity severity before infusion, and 90, 180, and 360 days after infusion. Mixed models were used to examine changes in QoL and toxicities over time, and clinical moderators of change in QoL and toxicities. Patients reported improvements in physical functioning and fatigue in the year after treatment (P values <.01), but there were no changes in pain, anxiety, or depression over time. Patients with active disease at day 90 reported more physical dysfunction at all postinfusion timepoints (Ps ≤ .01) compared to patients who responded to treatment. Similarly, patients with active disease at day 90 reported worsening depression over time, such that at day 360, depressive symptoms were worse for patients with active disease than patients without active disease (P = .02). Patients treated with 4+ lines of prior therapy reported worsening pain and anxiety over time, such that at day 360, both pain and anxiety were significantly worse for patients previously treated with 4 of more lines of therapy than patients treated with fewer lines of therapy (Ps ≤ .01). Regarding toxicities, patients reported decreasing overall toxicity burden up to day 180, with subsequent worsening at day 360 (P = .02). Most patients reported at least one or two grade 2 toxicities at each timepoint. Patients demonstrated unchanging or improved QoL after treatment with CAR T-cell therapy, but active disease and greater prior lines of therapy were associated with worse QoL outcomes over time. Toxicity severity also improved during the first 6 months post-treatment, but worsened thereafter, particularly among patients with active disease after treatment.
背景嵌合抗原受体(CAR)T细胞疗法改变了复发和难治性大B细胞淋巴瘤患者的生存结果,但它也与各种副作用有关 目标: :本研究调查了治疗后第一年患者报告的生活质量(QoL)和毒性的变化,以及导致生活质量和毒性恶化的风险因素:大B细胞淋巴瘤患者在输液前、输液后90天、180天和360天填写问卷,评估生活质量和毒性严重程度。采用混合模型研究随时间变化的生活质量和毒性变化,以及生活质量和毒性变化的临床调节因素 结果:............:治疗后一年内,患者的身体机能和疲劳感均有所改善(P值
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引用次数: 0
Healthcare Resource Utilization and Associated Costs in Patients With Chronic Graft-Versus-Host Disease Post-Allogeneic Hematopoietic Stem Cell Transplantation in England 英国异基因造血干细胞移植后慢性移植物抗宿主疾病患者的医疗资源利用率和相关费用:英国异基因造血干细胞移植后慢性移植物抗宿主疾病患者的医疗资源利用率和相关费用:HCRU 和相关费用。
IF 3.6 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-12-01 DOI: 10.1016/j.jtct.2024.10.002
Daniele Avenoso , Jennifer A. Davidson , Harriet Larvin , Hannah R. Brewer , Caoimhe T. Rice , Katharina Ecsy , Arunesh Sil , Luke Skinner , Richard D.A. Hudson
<div><div>Limited evidence suggests chronic graft-versus-host disease (cGvHD) following allogeneic hematopoietic stem cell transplantation (allo-HSCT) increases healthcare resource utilization (HCRU) and costs. However, this burden has not been well characterized in England. This study assesses secondary care HCRU and costs for patients following allo HSCT in England with cGvHD and patients who did not develop graft-versus-host disease (GvHD). Further stratification was performed among patients who did or did not subsequently receive high-cost therapies for the treatment of cGvHD. This descriptive, retrospective cohort study used Hospital Episode Statistics (HES) data from April 2017 to March 2022. HES data captures information on reimbursed diagnoses and procedures from all National Health Service (NHS) secondary care admissions and attendances in England. High-cost drugs as defined by NHS England are recorded in HES, these drugs and other procedures including plasma exchange, were used to identify patients with cGvHD who were in receipt of high-cost therapies. HCRU and costs were described for patients with cGvHD following allo-HSCT (<em>n</em> = 721) and were matched with patients with no evidence of GvHD following allo-HSCT (<em>n</em> = 718). HCRU and costs were also described for the subset of patients with cGvHD (<em>n</em> = 198) following receipt of high-cost therapies and patients with cGvHD prior to or without such therapies (<em>n</em> = 523). A higher proportion of patients with cGvHD had at least one inpatient or intensive care unit (ICU) admission or emergency care attendance than patients without GvHD (inpatient: 74.6% versus 66.6%; emergency care: 39.3% versus 30.5%; ICU: 7.4% versus 4.7%; respectively); whilst the proportion of patients with an outpatient attendance were similar for both groups (outpatient: 80.3% versus 84.1%; respectively). The cost across all secondary care settings was higher for patients with cGvHD than patients without GvHD, with a mean cost of inpatient admissions of £17,339 per patient-year for those with cGvHD versus £8548 per patient-year in patients without GvHD. A higher proportion of patients who received high-cost therapies for the treatment of cGvHD had at least one secondary care admission or attendance, than patients who did not (inpatient: 85.4% versus 66.4%; ICU: 7.1% versus 5.4%; outpatient: 87.9% versus 76.7%; emergency care: 44.4% versus 36.5%; respectively). Patients who were treated with high-cost therapies for the treatment of cGvHD had a greater mean number (14.6 versus 8.2 per patient-year, respectively) for all-cause inpatient admissions after treatment than patients who did not. In all secondary care settings, the total cost per patient-year was higher for patients who received high-cost therapies for the treatment of cGvHD, than for those who did not. Patients who were treated with high-cost therapies for the treatment of cGvHD had a greater mean cost (£21,137 versus £15,956 per pati
背景:有限的证据表明,异基因造血干细胞移植(allo-HSCT)后的慢性移植物抗宿主疾病(cGvHD)会增加医疗资源利用率(HCRU)和成本。然而,在英格兰,这种负担还没有得到很好的描述:本研究评估了在英国接受异体造血干细胞移植后,发生移植物抗宿主疾病(GvHD)的患者和未发生移植物抗宿主疾病(GvHD)的患者的二级护理HCRU和费用。对随后接受或未接受高成本疗法治疗 cGvHD 的患者进行了进一步分层:这项描述性、回顾性队列研究使用了2017年4月至2022年3月的医院病例统计(Hospital Episode Statistics,HES)数据。HES 数据收集了英格兰所有国民健康服务(NHS)二级医疗机构入院和就诊的有偿诊断和手术信息。HES 中记录了英格兰国家医疗服务体系定义的高成本药物,这些药物和包括血浆置换在内的其他程序被用来识别接受高成本疗法的 cGvHD 患者。对接受异体造血干细胞移植(allo-HSCT)后出现 cGvHD 的患者(人数=721)的 HCRU 和费用进行了描述,并与接受异体造血干细胞移植后未出现 GvHD 的患者(人数=718)进行了比对。此外,还对接受高成本疗法后出现 cGvHD 的患者(n=198)和接受或未接受此类疗法前出现 cGvHD 的患者(n=523)的 HCRU 和费用进行了描述:与未接受高成本疗法的患者相比,接受过至少一次住院治疗或重症监护室(ICU)治疗或急诊治疗的cGvHD患者比例更高(住院患者:74.6%对66.6%;急诊患者:39.3%对30.5%):39.3%对30.5%;重症监护室:7.4%对4.7%);而两组患者的门诊就诊比例相似(门诊:80.3%对84.1%)。cGvHD 患者在所有二级医疗机构的费用均高于非 GvHD 患者,cGvHD 患者的平均住院费用为每年 17,339 英镑,而非 GvHD 患者的平均住院费用为每年 8,548 英镑。在接受高成本疗法治疗 cGvHD 的患者中,至少接受过一次二级护理或就诊的患者比例高于未接受二级护理或就诊的患者(住院患者:85.4% 对 66.4%;重症监护室:7.1% 对 5.4%;门诊患者:87.9% 对 76.7%;急诊患者:44.4% 对 36.5%):分别为 44.4% vs 36.5%)。接受高成本疗法治疗 cGvHD 的患者在治疗后因各种原因住院的平均人数(分别为 14.6 人对 8.2 人)高于未接受治疗的患者。在所有二级医疗机构中,接受高成本疗法治疗 cGvHD 的患者的总成本ppy均高于未接受治疗的患者。与未接受高成本疗法治疗的患者相比,接受高成本疗法治疗的 cGvHD 患者因各种原因住院的平均费用更高(分别为 21,137 英镑 vs 15,956 英镑/年):本研究表明,在英格兰的各种二级医疗机构中,cGvHD 和使用相关高成本疗法对医疗活动和成本的影响要大于未接受 GvHD 治疗的患者和未接受高成本疗法的 cGvHD 患者。
{"title":"Healthcare Resource Utilization and Associated Costs in Patients With Chronic Graft-Versus-Host Disease Post-Allogeneic Hematopoietic Stem Cell Transplantation in England","authors":"Daniele Avenoso ,&nbsp;Jennifer A. Davidson ,&nbsp;Harriet Larvin ,&nbsp;Hannah R. Brewer ,&nbsp;Caoimhe T. Rice ,&nbsp;Katharina Ecsy ,&nbsp;Arunesh Sil ,&nbsp;Luke Skinner ,&nbsp;Richard D.A. Hudson","doi":"10.1016/j.jtct.2024.10.002","DOIUrl":"10.1016/j.jtct.2024.10.002","url":null,"abstract":"&lt;div&gt;&lt;div&gt;Limited evidence suggests chronic graft-versus-host disease (cGvHD) following allogeneic hematopoietic stem cell transplantation (allo-HSCT) increases healthcare resource utilization (HCRU) and costs. However, this burden has not been well characterized in England. This study assesses secondary care HCRU and costs for patients following allo HSCT in England with cGvHD and patients who did not develop graft-versus-host disease (GvHD). Further stratification was performed among patients who did or did not subsequently receive high-cost therapies for the treatment of cGvHD. This descriptive, retrospective cohort study used Hospital Episode Statistics (HES) data from April 2017 to March 2022. HES data captures information on reimbursed diagnoses and procedures from all National Health Service (NHS) secondary care admissions and attendances in England. High-cost drugs as defined by NHS England are recorded in HES, these drugs and other procedures including plasma exchange, were used to identify patients with cGvHD who were in receipt of high-cost therapies. HCRU and costs were described for patients with cGvHD following allo-HSCT (&lt;em&gt;n&lt;/em&gt; = 721) and were matched with patients with no evidence of GvHD following allo-HSCT (&lt;em&gt;n&lt;/em&gt; = 718). HCRU and costs were also described for the subset of patients with cGvHD (&lt;em&gt;n&lt;/em&gt; = 198) following receipt of high-cost therapies and patients with cGvHD prior to or without such therapies (&lt;em&gt;n&lt;/em&gt; = 523). A higher proportion of patients with cGvHD had at least one inpatient or intensive care unit (ICU) admission or emergency care attendance than patients without GvHD (inpatient: 74.6% versus 66.6%; emergency care: 39.3% versus 30.5%; ICU: 7.4% versus 4.7%; respectively); whilst the proportion of patients with an outpatient attendance were similar for both groups (outpatient: 80.3% versus 84.1%; respectively). The cost across all secondary care settings was higher for patients with cGvHD than patients without GvHD, with a mean cost of inpatient admissions of £17,339 per patient-year for those with cGvHD versus £8548 per patient-year in patients without GvHD. A higher proportion of patients who received high-cost therapies for the treatment of cGvHD had at least one secondary care admission or attendance, than patients who did not (inpatient: 85.4% versus 66.4%; ICU: 7.1% versus 5.4%; outpatient: 87.9% versus 76.7%; emergency care: 44.4% versus 36.5%; respectively). Patients who were treated with high-cost therapies for the treatment of cGvHD had a greater mean number (14.6 versus 8.2 per patient-year, respectively) for all-cause inpatient admissions after treatment than patients who did not. In all secondary care settings, the total cost per patient-year was higher for patients who received high-cost therapies for the treatment of cGvHD, than for those who did not. Patients who were treated with high-cost therapies for the treatment of cGvHD had a greater mean cost (£21,137 versus £15,956 per pati","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"30 12","pages":"Pages 1207.e1-1207.e11"},"PeriodicalIF":3.6,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142401428","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
ASTCT Consensus Recommendations on Testing and Treatment of Patients with Donor-specific Anti-HLA Antibodies ASTCT 关于检测和治疗捐献者特异性抗 HLA 抗体患者的共识建议。
IF 3.6 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-12-01 DOI: 10.1016/j.jtct.2024.09.005
Piyanuch Kongtim , Pongthep Vittayawacharin , Jun Zou , Samer Srour , Brian Shaffer , Roman M. Shapiro , Ankur Varma , Joseph McGuirk , Bhagirathbhai R. Dholaria , Shannon R. McCurdy , Amy E. DeZern , Nelli Bejanyan , Asad Bashey , Sabine Furst , Luca Castagna , Jacopo Mariotti , Annalisa Ruggeri , Rebeca Bailen , Takanori Teshima , Huang Xiao-Jun , Stefan O. Ciurea
Donor-specific anti-HLA antibodies (DSA) are an important cause of engraftment failure and may negatively impact survival outcomes of patients receiving allogeneic hematopoietic stem cell transplantation (HSCT) using an HLA-mismatched allograft. The incidence of DSA varies across studies, depending on individual factors, detection or identification methods and thresholds considered clinically relevant. Although DSA testing by multiplex bead arrays remains semiquantitative, it has been widely adopted as a standard test in most transplant centers. Additional testing to determine risk of allograft rejection may include assays with HLA antigens in natural conformation, such as flow cytometric crossmatch, and/or antibody binding assays, such as C1q testing. Patients with low level of DSA (<2,000 mean fluorescence intensity; MFI) may not require treatment, while others with very high level of DSA (>20,000 MFI) may be at very high-risk for engraftment failure despite current therapies. By contrast, in patients with moderate or high level of DSA, desensitization therapy can successfully mitigate DSA levels and improve donor cell engraftment rate, with comparable outcomes to patients without DSA. Treatment is largely empirical and multimodal, involving the removal, neutralization, and blocking of antibodies, as well as inhibition of antibody production to prevent activation of the complement cascade. Desensitization protocols are based on accumulated multicenter experience, while prospective multicenter studies remain lacking. Most patients require a full intensity protocol that includes plasma exchange, while protocols relying only on rituximab and intravenous immunoglobulin may be sufficient for patients with lower DSA levels and negative C1q and/or flow cytometric crossmatch. Monitoring DSA levels before and after HSCT could guide preemptive treatment when high levels persist after stem cell infusion. This paper aims to standardize current evidence-based practice and formulate future directions to improve upon current knowledge and advance treatment for this relatively rare, but potentially serious complication in allogeneic HSCT recipients.
捐献者特异性抗-HLA抗体(DSA)是导致移植失败的重要原因,可能会对使用HLA不匹配异体移植物接受异基因造血干细胞移植(HSCT)患者的生存结果产生负面影响。不同研究的DSA发生率各不相同,取决于个体因素、检测或鉴定方法以及临床相关阈值。尽管通过多重微珠阵列进行的 DSA 检测仍是半定量的,但它已被大多数移植中心广泛采用为标准检测方法。确定同种异体排斥风险的其他检测可包括自然构型的 HLA 抗原检测(如流式细胞交叉配型)和/或抗体结合检测(如 C1q 检测)。DSA水平较低(20,000 MFI)的患者可能面临很高的移植失败风险,尽管目前有各种疗法。相比之下,对于中度或高度 DSA 患者,脱敏疗法可成功降低 DSA 水平并提高供体细胞移植率,其疗效与无 DSA 患者相当。治疗方法主要是经验性的多模式治疗,包括清除、中和和阻断抗体,以及抑制抗体产生以防止补体级联激活。脱敏方案是根据多中心积累的经验制定的,但目前仍缺乏前瞻性多中心研究。大多数患者需要包括血浆置换在内的全强度方案,而对于DSA水平较低、C1q和/或流式细胞术交叉配型阴性的患者,仅依靠利妥昔单抗和静脉注射免疫球蛋白的方案可能就足够了。在造血干细胞移植前后监测DSA水平,可在干细胞输注后DSA水平持续较高时指导先期治疗。本文旨在规范目前的循证实践,并制定未来发展方向,以改善现有知识,推进异基因造血干细胞移植受者中这种相对罕见但潜在严重并发症的治疗。
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引用次数: 0
Allogeneic Hematopoietic Cell Transplantation for the Treatment of Severe Aplastic Anemia: Evidence-Based Guidelines From the American Society for Transplantation and Cellular Therapy 异体造血细胞移植治疗重型再生障碍性贫血:美国移植与细胞治疗学会的循证指南》(Evidence-Based Guidelines from the American Society for Transplantation and Cellular Therapy)。
IF 3.6 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-12-01 DOI: 10.1016/j.jtct.2024.09.017
Raheel Iftikhar , Zachariah DeFilipp , Amy E. DeZern , Michael A. Pulsipher , Nelli Bejanyan , Lauri M. Burroughs , Mohamed A. Kharfan-Dabaja , Sally Arai , Adetola Kassim , Ryotaro Nakamura , Blachy J. Dávila Saldaña , Mahmoud Aljurf , Mehdi Hamadani , Paul A. Carpenter , Joseph H. Antin
Allogeneic hematopoietic cell transplantation (HCT) is a potentially curative treatment for severe aplastic anemia (SAA). Existing guidance about HCT in SAA is primarily derived from expert reviews, registry data and societal guidelines; however, transplant-specific guidelines for SAA are lacking. A panel of SAA experts, both pediatric and adult transplant physicians, developed consensus recommendations using Grading of Recommendation, Assessment, Development, and Evaluation (GRADE) methodology employing a GRADE guideline development tool. The panel agrees with previous recommendations for the preferential use of bone marrow as a graft source and the use of rabbit over horse antithymocyte globulin (ATG) for HCT conditioning. Fludarabine containing regimens are preferred for patients at high risk of graft failure and those receiving matched unrelated or haploidentical donor transplant. Given advancements in HCT, the panel does not endorse the historical 40-year age cut-off for considering upfront HCT in adults, acknowledging that fit older patients may also benefit from HCT. The panel also endorses increased utilization of HCT by prioritizing matched unrelated or haploidentical donor HCT over immunosuppressive therapy in children and adults who lack a matched related donor. Finally, the panel suggests either calcineurin inhibitor plus methotrexate or post-transplant cyclophosphamide-based graft-versus-host disease (GVHD) prophylaxis for matched related or matched unrelated donor recipients. These recommendations reflect a significant advancement in transplant strategies for SAA and highlight the importance of ongoing and further research to revisit current evidence in terms of donor choice, conditioning chemotherapy, GVHD prophylaxis and post-transplant immunosuppression.
异基因造血细胞移植(HCT)是一种可能治愈重型再生障碍性贫血(SAA)的治疗方法。现有的 SAA 造血干细胞移植指南主要来自专家评论、登记数据和社会指南,但缺乏针对 SAA 的移植指南。一个由儿科和成人移植医师组成的 SAA 专家小组采用建议、评估、发展和评价分级(GRADE)方法,利用 GRADE 指南开发工具制定了共识建议。专家小组同意之前的建议,即优先使用骨髓作为移植物来源,以及在 HCT 调理中使用兔抗胸腺细胞球蛋白 (ATG) 而非马抗胸腺细胞球蛋白 (ATG)。对于移植失败风险较高的患者和接受配型非血缘关系或单倍体供体移植的患者来说,含氟达拉滨的方案是首选。鉴于造血干细胞移植技术的进步,专家小组不赞成在考虑成人前期造血干细胞移植时采用 40 岁这一历史分界线,并承认适合的老年患者也可从造血干细胞移植中获益。专家小组还赞同提高造血干细胞的利用率,在缺乏匹配的亲属供体的儿童和成人中,优先考虑匹配的非亲属或单倍体供体造血干细胞,而不是免疫抑制疗法。最后,专家小组建议对配型相关或配型无关的供体受者采用钙神经蛋白抑制剂加甲氨蝶呤或移植后环磷酰胺为基础的移植物抗宿主病(GVHD)预防疗法。这些建议反映了 SAA 移植策略的重大进展,并强调了持续开展进一步研究的重要性,以重新审视在供体选择、条件化疗、移植物抗宿主病(GVHD)预防和移植后免疫抑制方面的现有证据。
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引用次数: 0
Comparison of Thiotepa-based Conditioning Regimens for Older Adults with Primary Diffuse Large B-cell Lymphoma of the Central Nervous System Undergoing Autologous Hematopoietic Cell Transplantation 中枢神经系统原发性弥漫性大 B 细胞淋巴瘤老年患者接受自体造血细胞移植的噻替帕治疗方案比较。
IF 3.6 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-12-01 DOI: 10.1016/j.jtct.2024.09.015
Othman S. Akhtar , Shanze Arshad , Qinghua Lian , Kwang W. Ahn , Anita D'Souza , Binod Dhakal , Meera Mohan , Marcelo Pasquini , Walter Longo , Nirav N. Shah , Timothy S. Fenske , Mehdi Hamadani
In this study, we compare outcomes of older patients with primary diffuse large B-cell lymphoma of the central nervous system (PCNSL) undergoing autologous hematopoietic cell transplantation (autoHCT) with either thiotepa/carmustine (BCNU/Thio) or thiotepa/busulfan/cyclophosphamide (TBC) conditioning. We used a postpublication dataset made available by the Center for International Blood and Marrow Transplantation Research including patients who were ≥65 years in age with PCNSL and underwent autoHCT as consolidation with TBC or BCNU/Thio conditioning. Out of 147 patients; n = 84 received BCNU/Thio and n = 63 received TBC. The 1-year NRM in the BCNU/Thio group was 10% versus 22% in the TBC group (P = .05) and the 2-year relapse rate was 5% versus 5%, respectively (P = 1.00). The 2-year progression-free survival (PFS) in the BCNU/Thio group was 85% versus 71% in the TBC group (P = .05) and 2-year overall survival (OS) was 86% versus 74% (P = .08). In a multivariable regression model, BCNU/Thio was associated with a lower risk for NRM (hazard ratio [HR], 0.33, P = .009), improved PFS (HR, 0.41, P = .008) and OS (HR, 0.37, P = .007), but there was no association with relapse risk. We found that in older adults with PCNSL undergoing consolidation with autoHCT, BCNU/Thio conditioning is associated with lower NRM and improved OS compared to TBC.
研究背景在这项研究中,我们比较了中枢神经系统原发性弥漫大B细胞淋巴瘤(PCNSL)老年患者在噻替派/卡莫司汀(BCNU/Thio)或噻替派/布磺安/环磷酰胺(TBC)治疗条件下接受自体造血细胞移植(autoHCT)的疗效:我们使用了国际血液与骨髓研究中心(CIBMTR)公布的数据集,该数据集包括年龄≥65岁的PCNSL患者,他们接受了自体血细胞移植,作为TBC或BCNU/Thio治疗的巩固治疗:在147名患者中,84人接受了BCNU/硫氧嘧啶治疗,63人接受了TBC治疗。BCNU/硫氧嘧啶组的1年NRM为10%,而TBC组为22%(P=0.05);2年复发率分别为5%和5%(P=1.00)。BCNU/硫氧嘧啶组的2年PFS为85%,而TBC组为71%(P=0.05),2年OS为86%,而TBC组为74%(P=0.08)。在多变量回归模型中,BCNU/硫氧嘧啶与较低的NRM风险相关[危险比(HR),0.33,p=0.009],改善了PFS(HR,0.41,p=0.008)和OS(HR,0.37,p=0.007),但与复发风险无关:我们发现,对于接受自体血细胞移植巩固治疗的老年 PCNSL 患者,与 TBC 相比,BCNU/硫氧嘧啶治疗可降低 NRM 并改善 OS。
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引用次数: 0
Proceedings of the 2024 Third Annual ASTCT-NMDP ACCESS Initiative Workshop 2024 年第三届 ASTCT-NMDP ACCESS 计划研讨会论文集。
IF 3.6 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-12-01 DOI: 10.1016/j.jtct.2024.09.004
Jeffery J. Auletta , Jennifer Holter-Chakrabarty , Pashna Munshi , Sarah Wall , Nandita Khera , Jess Knutson , Alexandra Gomez-Arteaga , Anurekha G. Hall , Jackie Foster , Amber Ruffin , Delilah Robb , Eneida Nemecek , Rayne Rouce , Stella M. Davies , ACCESS Workshop Team Members
The Third Annual Workshop of the American Society for Transplantation and Cellular Therapy (ASTCT) and National Marrow Donor Program (NMDP) ACCESS Initiative occurred on July 23 and 24, 2024. Content from the workshop is provided to inform the hematopoietic cell transplantation (HCT) and cellular therapy (CT) ecosystem about progress and direction of the collaborative. Highlights from the meeting are reviewed, including the inaugural Corporate Roundtable and Advocacy Day, new partnerships with non-profit organizations, and updates on projects from the Awareness, Poverty and Race and Ethnicity Inequity Committees. In addition, the Junior Faculty and Trainee Immersion Program–sponsored efforts in workforce diversity and physician advocacy are presented. Lastly, continued education was provided on patient and caregiver participation as well as community engagement. As it enters its third year, the ASTCT-NMDP ACCESS Initiative will transition from foundation building as a grass roots collaborative to intentional impact in reducing barriers and improving outcome disparities for all patients in need of HCT/CT. Enthusiasm for and participation in the ACCESS Initiative remain high. Both are needed to sustain progress in achieving its goal in enabling all patients in need to receive HCT/CT.
美国移植与细胞治疗学会(ASTCT)和美国国家骨髓捐献计划(NMDP)ACCESS 计划第三次年度研讨会于 2024 年 7 月 23 日和 24 日举行。研讨会的内容旨在向造血细胞移植 (HCT) 和细胞治疗 (CT) 生态系统通报合作的进展和方向。会议回顾了会议的亮点,包括首届企业圆桌会议和宣传日、与非营利组织的新合作关系,以及提高认识委员会、贫困委员会和种族与民族不平等委员会的最新项目情况。此外,还介绍了青年教师和实习生沉浸计划在劳动力多样性和医生宣传方面所做的努力。最后,还提供了有关患者和护理人员参与以及社区参与的继续教育。进入第三年后,ASTCT-NMDP ACCESS 计划将从基层合作的基础建设过渡到有意识地为所有需要 HCT/CT 的患者减少障碍和改善结果差异。人们对 ACCESS 计划的热情和参与度仍然很高,要想在实现让所有需要 HCT/CT 的患者都能接受 HCT/CT 的目标方面持续取得进展,就需要这两方面的努力。
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Transplantation and Cellular Therapy
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