前瞻性分析患有急性白血病患者进行异基因造血干细胞移植的障碍因素。
Prospective analysis to determine barriers to allogeneic hematopoietic cell transplantation in patients with acute leukemia.
发表日期:2023 Sep 09
作者:
Karthik Nath, Jasme Lee, Theresa A Elko, Lauren Levy, Elaina Preston, Sean M Devlin, Doris M Ponce, Richard J Lin, Brian C Shaffer, Christina Cho, Ioannis Politikos, Ann A Jakubowski, Jae H Park, Raajit Rampal, Miguel-Angel Perales, Martin S Tallman, Juliet N Barker, Ellin Berman, Roni Tamari, Eytan Stein, Sergio A Giralt, Boglarka Gyurkocza
来源:
AMERICAN JOURNAL OF HEMATOLOGY
摘要:
异基因造血细胞移植(allo-HCT)是一种潜在的治疗急性白血病患者的根治性治疗方法。然而,研究表明只有少数患者最终进行了allo-HCT。本前瞻性观察性研究的主要目标是确定符合推荐条件和适合进行HCT的患者中进行allo-HCT的比例以及为什么这些患者最终未接受移植。在2016年4月至2021年4月期间,我们在诱导/再诱导治疗时招募了新诊断或复发/难治性急性白血病的成年患者。诱导/再诱导早期阶段进行了初步移植准备和allo-HCT的推荐。在307名招募的患者中,建议进行allo-HCT的占85%(n=259),其中66%(n=170)接受了移植。供体来源包括54%配型不相关供体、20%配型相合同胞供体和HLA不相合的移植源,其中15%为脐血单位供体、8%为配型不相关供体和4%为半相合同胞供体。最常见的导致89名初步推荐的患者无法进行移植的原因是持续/复发疾病(70%),其次是早期患者死亡(10%)。在这一前瞻性研究中,我们报道了较高的allo-HCT比例,这可能归因于早期的移植转诊和准备工作。主要的allo-HCT障碍是疾病控制,其次是早期患者死亡。随着越来越多HLA不相合的移植源的可用性提高,供体不可得性不再是移植的障碍。进一步发展针对未达到缓解状态的患者的新型移植策略和改进诱导方案可能会导致allo-HCT的使用增加。 © 2023 Wiley Periodicals LLC.
Allogeneic hematopoietic cell transplantation (allo-HCT) is a potentially curative treatment for patients with acute leukemia. Despite this, studies have shown that only a minority of patients ultimately proceed to allo-HCT. The primary objective of this prospective, observational study was to identify the rate of allo-HCT in patients for whom it was recommended, and reasons why patients deemed appropriate and eligible for HCT did not subsequently undergo transplant. Between April 2016 and April 2021, adult patients with newly diagnosed or relapsed/refractory acute leukemia were enrolled at the time of induction/reinduction therapy. Initial transplantation workup and allo-HCT recommendations were made during the early phase of induction/reinduction. Of the 307 enrolled patients, allo-HCT was recommended to 85% (n = 259), of whom 66% (n = 170) underwent transplant. Donor sources comprised 54% human leukocyte antigen (HLA)-matched unrelated donors, 20% HLA-matched sibling donors and HLA-mismatched graft sources with 15% umbilical cord blood units, 8% HLA-mismatched unrelated donors, and 4% HLA-haploidentical donors. The most common reason for transplant disqualification in the 89 patients in whom it was initially recommended was persistent/relapsed disease (70%), followed by early patient death (10%). In this prospective study, we report a high allo-HCT rate, which may be due to early transplant referral and workup. The main allo-HCT barrier was disease control, followed by early patient death. With the increasing availability of HLA-mismatched graft sources, the lack of donor availability was not a transplant barrier. Further development of novel transplant strategies for patients not achieving remission and improvements in induction regimens could result in increased allo-HCT utilization.© 2023 Wiley Periodicals LLC.