Axicabtagene Ciloleucel后一年的真实世界患者报告和神经认知结果。

IF 3.6 3区 医学 Q2 HEMATOLOGY Transplantation and Cellular Therapy Pub Date : 2025-03-01 DOI:10.1016/j.jtct.2024.12.020
Aasha I. Hoogland , Xiaoyin Li , Karnav Modi , Taylor Welniak , Yvelise Rodriguez , Nathaly Irizarry-Arroyo , Laura B. Oswald , Julia T. Snider , Sally W. Wade , Julio Chavez , Salvatore Corallo , Margaret Booth-Jones , Michael D. Jain , Frederick L. Locke , Heather S.L. Jim
{"title":"Axicabtagene Ciloleucel后一年的真实世界患者报告和神经认知结果。","authors":"Aasha I. Hoogland ,&nbsp;Xiaoyin Li ,&nbsp;Karnav Modi ,&nbsp;Taylor Welniak ,&nbsp;Yvelise Rodriguez ,&nbsp;Nathaly Irizarry-Arroyo ,&nbsp;Laura B. Oswald ,&nbsp;Julia T. Snider ,&nbsp;Sally W. Wade ,&nbsp;Julio Chavez ,&nbsp;Salvatore Corallo ,&nbsp;Margaret Booth-Jones ,&nbsp;Michael D. Jain ,&nbsp;Frederick L. Locke ,&nbsp;Heather S.L. Jim","doi":"10.1016/j.jtct.2024.12.020","DOIUrl":null,"url":null,"abstract":"<div><div>Axicabtagene ciloleucel (axi-cel), a chimeric antigen receptor T-cell therapy, has significantly improved clinical outcomes in adult patients with relapsed/refractory large B-cell lymphoma. However, few studies have examined patient-reported outcomes (PROs) or neurocognitive performance in patients treated with axi-cel. Moreover, no longitudinal PRO study has reported on patients treated with axi-cel as standard of care in the United States, to our knowledge. This paper reports on real-world changes in PROs (i.e., quality of life [QOL] and perceived cognition) and objective neurocognitive performance before treatment with axi-cel and in the first year after. Patients scheduled to receive axi-cel as standard of care were recruited from a single cancer center between March 2020 and June 2022. QOL was assessed using the EORTC QLQ-C30 and EQ-5D-5L at baseline recruitment (ie, prior to conditioning chemotherapy before axi-cel), and at 7, 14, 30, 60, 90, 180, and 360 days after receiving axi-cel. Perceived cognition was assessed using the Patient-Reported Outcomes Measurement Information System Cognitive Function 4a scale. Objective neurocognitive performance was assessed using a battery of tests at baseline, and 30, 90, and 360 days after receiving axi-cel. Random-effects mixed models evaluated changes in QOL, perceived cognition, and neurocognitive performance using all available data. Clinically meaningful change in QOL was defined as a difference of 10 points on the EORTC QLQ-C30. Clinically meaningful change in perceived cognition or neurocognitive performance was defined as a difference of 5 points. On average, participants (<em>N</em> = 53) were 63 years of age (SD = 13), and predominantly male (62%), White (92%), and college graduates (60%). Participants reported statistically significant improvements from baseline to day 360 in overall QOL, physical functioning, role functioning, and social functioning (<em>P</em>s &lt; .05) after axi-cel, despite clinically significant worsening in the first 14 days. For role functioning and social functioning, improvements also met criteria for clinical significance. There were no statistically (<em>P</em>s &gt; .05) or clinically significant changes in perceived cognition over time. Despite some transient declines, neurocognitive performance generally returned to or exceeded baseline levels by day 360 (<em>P</em>s &lt; .01). However, visuospatial ability worsened by day 90 and did not recover to baseline levels by day 360 (<em>P</em> &lt; .0001). These real-world data suggest that axi-cel is associated with significant improvements in overall QOL in the first year after infusion. These data are generally consistent with, or exceed, improvements in QOL reported from clinical trials of axi-cel therapy. Despite transient worsening in the acute period after treatment, neurocognitive performance in most domains also recovered to pretreatment levels by 1 year after infusion. These findings extend previous research by reporting on patients’ perspectives on axi-cel therapy received as standard of care in the real-world setting and neurocognitive changes after treatment with axi-cel.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 3","pages":"Pages 157.e1-157.e13"},"PeriodicalIF":3.6000,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Real-World Patient-Reported and Neurocognitive Outcomes in the Year After Axicabtagene Ciloleucel\",\"authors\":\"Aasha I. Hoogland ,&nbsp;Xiaoyin Li ,&nbsp;Karnav Modi ,&nbsp;Taylor Welniak ,&nbsp;Yvelise Rodriguez ,&nbsp;Nathaly Irizarry-Arroyo ,&nbsp;Laura B. Oswald ,&nbsp;Julia T. Snider ,&nbsp;Sally W. Wade ,&nbsp;Julio Chavez ,&nbsp;Salvatore Corallo ,&nbsp;Margaret Booth-Jones ,&nbsp;Michael D. Jain ,&nbsp;Frederick L. Locke ,&nbsp;Heather S.L. Jim\",\"doi\":\"10.1016/j.jtct.2024.12.020\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div><div>Axicabtagene ciloleucel (axi-cel), a chimeric antigen receptor T-cell therapy, has significantly improved clinical outcomes in adult patients with relapsed/refractory large B-cell lymphoma. However, few studies have examined patient-reported outcomes (PROs) or neurocognitive performance in patients treated with axi-cel. Moreover, no longitudinal PRO study has reported on patients treated with axi-cel as standard of care in the United States, to our knowledge. This paper reports on real-world changes in PROs (i.e., quality of life [QOL] and perceived cognition) and objective neurocognitive performance before treatment with axi-cel and in the first year after. Patients scheduled to receive axi-cel as standard of care were recruited from a single cancer center between March 2020 and June 2022. QOL was assessed using the EORTC QLQ-C30 and EQ-5D-5L at baseline recruitment (ie, prior to conditioning chemotherapy before axi-cel), and at 7, 14, 30, 60, 90, 180, and 360 days after receiving axi-cel. Perceived cognition was assessed using the Patient-Reported Outcomes Measurement Information System Cognitive Function 4a scale. Objective neurocognitive performance was assessed using a battery of tests at baseline, and 30, 90, and 360 days after receiving axi-cel. Random-effects mixed models evaluated changes in QOL, perceived cognition, and neurocognitive performance using all available data. Clinically meaningful change in QOL was defined as a difference of 10 points on the EORTC QLQ-C30. Clinically meaningful change in perceived cognition or neurocognitive performance was defined as a difference of 5 points. On average, participants (<em>N</em> = 53) were 63 years of age (SD = 13), and predominantly male (62%), White (92%), and college graduates (60%). Participants reported statistically significant improvements from baseline to day 360 in overall QOL, physical functioning, role functioning, and social functioning (<em>P</em>s &lt; .05) after axi-cel, despite clinically significant worsening in the first 14 days. For role functioning and social functioning, improvements also met criteria for clinical significance. There were no statistically (<em>P</em>s &gt; .05) or clinically significant changes in perceived cognition over time. Despite some transient declines, neurocognitive performance generally returned to or exceeded baseline levels by day 360 (<em>P</em>s &lt; .01). However, visuospatial ability worsened by day 90 and did not recover to baseline levels by day 360 (<em>P</em> &lt; .0001). These real-world data suggest that axi-cel is associated with significant improvements in overall QOL in the first year after infusion. These data are generally consistent with, or exceed, improvements in QOL reported from clinical trials of axi-cel therapy. Despite transient worsening in the acute period after treatment, neurocognitive performance in most domains also recovered to pretreatment levels by 1 year after infusion. These findings extend previous research by reporting on patients’ perspectives on axi-cel therapy received as standard of care in the real-world setting and neurocognitive changes after treatment with axi-cel.</div></div>\",\"PeriodicalId\":23283,\"journal\":{\"name\":\"Transplantation and Cellular Therapy\",\"volume\":\"31 3\",\"pages\":\"Pages 157.e1-157.e13\"},\"PeriodicalIF\":3.6000,\"publicationDate\":\"2025-03-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Transplantation and Cellular Therapy\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://www.sciencedirect.com/science/article/pii/S266663672400839X\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"HEMATOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Transplantation and Cellular Therapy","FirstCategoryId":"3","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S266663672400839X","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"HEMATOLOGY","Score":null,"Total":0}
引用次数: 0

摘要

背景:Axicabtagene ciloleucel(轴细胞)是一种嵌合抗原受体(CAR) t细胞疗法,可显著改善复发/难治性大b细胞淋巴瘤(LBCL)成人患者的临床结果。然而,很少有研究检查患者报告的结果(PROs)或接受轴细胞治疗的患者的神经认知表现。此外,据我们所知,在美国,没有纵向PRO研究报道将axis -cel作为标准治疗的患者。目的:本文报道了在axis -cel治疗前和治疗后第一年的pro[即生活质量(QOL)和感知认知]和客观神经认知表现的现实世界变化。研究设计:计划在2020年3月至2022年6月期间从单个癌症中心招募接受轴细胞作为标准治疗的患者。使用EORTC QLQ-C30和EQ-5D-5L在基线招募(即在轴细胞前调节化疗前)和接受轴细胞后7、14、30、60、90、180和360天评估QOL。感知认知采用PROMIS认知功能4a量表进行评估。在基线、接受轴细胞治疗后30、90和360天,通过一系列测试评估客观神经认知表现。随机效应混合模型利用所有可用数据评估生活质量、感知认知和神经认知表现的变化。临床上有意义的生活质量变化定义为EORTC QLQ-C30差10分。感知认知或神经认知表现的临床意义改变被定义为5分的差异。结果:参与者(N=53)平均年龄为63岁(SD=13),主要为男性(62%),白人(92%)和大学毕业生(60%)。从基线到第360天,参与者报告了总体生活质量、身体功能、角色功能和社会功能的统计学显著改善(ps0.05),或随着时间的推移感知认知的临床显着变化。尽管有一些短暂的下降,但神经认知能力通常在第360天恢复到或超过基线水平。结论:这些真实世界的数据表明,在输注后的第一年,axis -cel与总体生活质量的显着改善有关。这些数据通常与轴细胞治疗临床试验报告的生活质量改善相一致或超过。尽管在治疗后的急性期有短暂的恶化,但大多数领域的神经认知表现在输注后一年内也恢复到治疗前的水平。这些发现扩展了先前的研究,报告了患者对轴细胞治疗作为现实世界标准治疗的看法,以及轴细胞治疗后神经认知的变化。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
查看原文
分享 分享
微信好友 朋友圈 QQ好友 复制链接
本刊更多论文
Real-World Patient-Reported and Neurocognitive Outcomes in the Year After Axicabtagene Ciloleucel
Axicabtagene ciloleucel (axi-cel), a chimeric antigen receptor T-cell therapy, has significantly improved clinical outcomes in adult patients with relapsed/refractory large B-cell lymphoma. However, few studies have examined patient-reported outcomes (PROs) or neurocognitive performance in patients treated with axi-cel. Moreover, no longitudinal PRO study has reported on patients treated with axi-cel as standard of care in the United States, to our knowledge. This paper reports on real-world changes in PROs (i.e., quality of life [QOL] and perceived cognition) and objective neurocognitive performance before treatment with axi-cel and in the first year after. Patients scheduled to receive axi-cel as standard of care were recruited from a single cancer center between March 2020 and June 2022. QOL was assessed using the EORTC QLQ-C30 and EQ-5D-5L at baseline recruitment (ie, prior to conditioning chemotherapy before axi-cel), and at 7, 14, 30, 60, 90, 180, and 360 days after receiving axi-cel. Perceived cognition was assessed using the Patient-Reported Outcomes Measurement Information System Cognitive Function 4a scale. Objective neurocognitive performance was assessed using a battery of tests at baseline, and 30, 90, and 360 days after receiving axi-cel. Random-effects mixed models evaluated changes in QOL, perceived cognition, and neurocognitive performance using all available data. Clinically meaningful change in QOL was defined as a difference of 10 points on the EORTC QLQ-C30. Clinically meaningful change in perceived cognition or neurocognitive performance was defined as a difference of 5 points. On average, participants (N = 53) were 63 years of age (SD = 13), and predominantly male (62%), White (92%), and college graduates (60%). Participants reported statistically significant improvements from baseline to day 360 in overall QOL, physical functioning, role functioning, and social functioning (Ps < .05) after axi-cel, despite clinically significant worsening in the first 14 days. For role functioning and social functioning, improvements also met criteria for clinical significance. There were no statistically (Ps > .05) or clinically significant changes in perceived cognition over time. Despite some transient declines, neurocognitive performance generally returned to or exceeded baseline levels by day 360 (Ps < .01). However, visuospatial ability worsened by day 90 and did not recover to baseline levels by day 360 (P < .0001). These real-world data suggest that axi-cel is associated with significant improvements in overall QOL in the first year after infusion. These data are generally consistent with, or exceed, improvements in QOL reported from clinical trials of axi-cel therapy. Despite transient worsening in the acute period after treatment, neurocognitive performance in most domains also recovered to pretreatment levels by 1 year after infusion. These findings extend previous research by reporting on patients’ perspectives on axi-cel therapy received as standard of care in the real-world setting and neurocognitive changes after treatment with axi-cel.
求助全文
通过发布文献求助,成功后即可免费获取论文全文。 去求助
来源期刊
CiteScore
7.00
自引率
15.60%
发文量
1061
审稿时长
51 days
期刊最新文献
INFECTIOUS ENTEROCOLITIS IN HEMATOPOIETIC CELL TRANSPLANT WITH POST-TRANSPLANT CYCLOPHOSPHAMIDE. Best Practices in Gene Therapy for Sickle Cell Disease and Transfusion-dependent β-Thalassemia. Outcome of Patients with IDH-mutated AML following Allogeneic Stem Cell Transplantation - a Retrospective Analysis on behalf of the German Registry for Hematopoietic Stem Cell Transplantation and Cell Therapy, DRST. Temporal evolution of functional immune reconstitution after allogeneic HSCT. Clinical Outcome of UCBT for Children with CAEBV: A Retrospective Analysis of a Single Center.
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
现在去查看 取消
×
提示
确定
0
微信
客服QQ
Book学术公众号 扫码关注我们
反馈
×
意见反馈
请填写您的意见或建议
请填写您的手机或邮箱
已复制链接
已复制链接
快去分享给好友吧!
我知道了
×
扫码分享
扫码分享
Book学术官方微信
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术
文献互助 智能选刊 最新文献 互助须知 联系我们:info@booksci.cn
Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。
Copyright © 2023 Book学术 All rights reserved.
ghs 京公网安备 11010802042870号 京ICP备2023020795号-1