确定在多发性骨髓瘤挽救性移植中有效利用储存的自体 PBSCs 的候选者:谁最受益?

IF 1.1 Q4 HEMATOLOGY
Amany R. Keruakous, Laura Walker, Molly Denlinger, Mohammad A H Mian, Danielle Bradshaw, Vamsi Kota, Anand P. Jillella
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引用次数: 0

摘要

背景/目标:高剂量化疗(HD-CHT)后进行自体干细胞移植(ASCT)仍是符合条件的多发性骨髓瘤(MM)患者的黄金标准,即使治疗方案不断发展。临床试验证明了自体干细胞移植对多发性骨髓瘤的疗效,包括在长期缓解后作为挽救疗法的潜力。外周血干细胞(PBSCs)目前是ASCT的主要造血干细胞来源。在初始骨髓溶解调理后收集额外的PBSCs具有挑战性,这导致许多中心在初始治疗期间采用收集和储存多余PBSCs的做法,以支持串联移植或挽救治疗。面对新型、高效的治疗方法,如治疗复发/难治性 MM(RRMM)的双特异性抗体和细胞疗法,挽救性 ASCT 的使用可能会减少。尽管可以获得储存的PBSC移植物,但由于各种因素,包括表现状态下降和治疗相关的合并症,挽救性ASCT的利用率并不高。2013 年的一项成本利用分析显示,约 70% 的患者有长期冷冻保存的未使用 PBSC 产品,这在 ASCT 总费用中占了很大一部分。收集、冷冻保存和储存 PBSCs 的平均费用超过每人 2 万美元,用于第二次 ASCT 的未使用 PBSCs 费用超过 6700 美元。2016年的一项最新分析强调,挽救性ASCT的需求正在下降,十年来使用储存的PBSC移植物的患者不到10%。方法:为了解决骨髓瘤患者是否仍需备用干细胞的难题,该研究调查了减轻PBSC收集、处理和储存经济负担的策略。研究评估了2012年1月至2022年6月期间接受前线ASCT的MM患者,排除了计划进行串联移植的患者和接受单次ASCT且未储存细胞的患者。讨论结果在研究的 240 名患者中,采集 PBSC 时的中位年龄为 61 岁。值得注意的是,只有 7% 的患者接受了挽救性 ASCT,而近 90% 的挽救性 ASCT 受者在初次接受 ASCT 时年龄小于 61 岁。该研究显示,随着年龄的增长,挽救性 ASCT 的使用率呈下降趋势,这表明在老年患者(年龄大于 60 岁)中收集 PBSC 用于单次移植可能是一种具有成本效益的替代方法。大多数移植中心的目标是收集 10 × 106 CD34 + 细胞/公斤,而 65 岁以上的患者往往需要多次收集。在老年人中转向单次移植采集可降低成本和资源需求。此外,该研究还建议在采集当天实施多余 PBSC 处理或再利用策略,以避免额外的储存成本。总之,在MM患者中,挽救性ASCT的使用率不断下降,加上经济方面的考虑,强调了修订干细胞采集政策的必要性。结论:研究提倡考虑为老年患者进行单次移植PBSC采集,并有效管理多余的PBSC,以优化资源利用。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Identifying Candidates for Effective Utilization of Stored Autologous PBSCs in Salvage Transplantation for Multiple Myeloma: Who Benefits Most?
Background/Objectives: High-dose chemotherapy (HD-CHT) followed by autologous stem cell transplantation (ASCT) remains the gold standard for eligible multiple myeloma (MM) patients, even amidst evolving therapeutic options. Clinical trials have demonstrated ASCT’s efficacy in MM, including its potential as salvage therapy after prolonged remission. Peripheral blood stem cells (PBSCs) are now the primary source of hematopoietic stem cells for ASCT. Collecting additional PBSCs post-initial myeloablative conditioning is challenging, leading many centers to adopt the practice of collecting and storing excess PBSCs during initial therapy to support tandem transplants or salvage treatments. The use of salvage ASCT may diminish in the face of novel, highly effective treatments like bispecific antibodies and cellular therapies for relapsed/refractory MM (RRMM). Despite available stored PBSC grafts, salvage ASCTs are underutilized due to various factors, including declining performance status and therapy-related comorbidities. A cost utilization analysis from 2013 revealed that roughly 70% of patients had unused PBSC products in prolonged cryopreservation, costing a significant portion of total ASCT expenses. The average cost for collecting, cryopreserving, and storing PBSCs exceeded $20,000 per person, with more than $6700 spent on unused PBSCs for a second ASCT. A more recent analysis from 2016 underscored the declining need for salvage ASCT, with less than 10% of patients using stored PBSC grafts over a decade. Methods: To address the dilemma of whether backup stem cells remain necessary for myeloma patients, the study investigated strategies to reduce the financial burden of PBSC collection, processing, and storage. It evaluated MM patients undergoing frontline ASCT from January 2012 to June 2022, excluding those with planned tandem transplants and those who had a single ASCT with no stored cells. Discussion: Among the 240 patients studied, the median age at PBSC collection was 61. Notably, only 7% underwent salvage ASCT, with nearly 90% of salvage ASCT recipients being ≤ 61 years old at the time of initial ASCT. The study revealed a decreasing trend in salvage ASCT use with increasing age, suggesting that PBSC collection for a single transplant among elderly patients (>60 years old) could be a cost-effective alternative. Most transplant centers aimed to collect 10 × 106 CD34 + cells/kg, with patients over 65 often requiring multiple collection days. Shifting towards single-transplant collections among the elderly could reduce costs and resource requirements. Additionally, the study recommended implementing strategies for excess PBSC disposal or repurposing on the collection day to avoid additional storage costs. In summary, the decreasing utilization of salvage ASCT in MM, alongside financial considerations, underscores the need for revised stem cell collection policies. Conclusions: The study advocates considering single-transplant PBSC collections for elderly patients and efficient management of excess PBSCs to optimize resource utilization.
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来源期刊
Hematology Reports
Hematology Reports HEMATOLOGY-
CiteScore
0.90
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47
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10 weeks
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