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嵌合抗原受体T细胞(CAR-T)疗法期间患者报告结局和活动数据的纵向收集:可行性、可接受性及数据可视化

Longitudinal Collection of Patient-Reported Outcomes and Activity Data during CAR-T Therapy: Feasibility, Acceptability, and Data Visualization.

作者信息

Oswald Laura B, Li Xiaoyin, Carvajal Rodrigo, Hoogland Aasha I, Gudenkauf Lisa M, Hansen Doris K, Alsina Melissa, Locke Frederick L, Rodriguez Yvelise, Irizarry-Arroyo Nathaly, Robinson Edmondo J, Jim Heather S L, Gonzalez Brian D, Kirtane Kedar

机构信息

Department of Health Outcomes and Behavior, Moffitt Cancer Center, 12902 USF Magnolia Dive, MFC-HOB, Tampa, FL 33612, USA.

Department of Biostatistics and Bioinformatics, Moffitt Cancer Center, Tampa, FL 33612, USA.

出版信息

Cancers (Basel). 2022 May 31;14(11):2742. doi: 10.3390/cancers14112742.


DOI:10.3390/cancers14112742
PMID:35681722
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC9179384/
Abstract

Background: Clinicians must closely monitor patients for toxicities after chimeric antigen receptor T-cell therapy (CAR-T). Patient-reported outcomes (PROs) (e.g., toxicities, quality of life) and activity data (e.g., steps, sleep) may complement clinicians’ observations. This study tested the feasibility and acceptability of collecting PROs and activity data from patients with hematologic malignancies during CAR-T and explored preliminary data patterns. Methods: Participants wore a Fitbit tracker and completed PROs at several timepoints through 90-days post-infusion. Feasibility was assessed with a priori benchmarks for recruitment (≥50%), retention (≥70%), PRO completion (≥70%), and days wearing the Fitbit (≥50%). Acceptability was assessed with participant satisfaction (a priori benchmark > 2 on a 0−4 scale). Results: Participants (N = 12) were M = 66 years old (SD = 7). Rates of recruitment (68%), retention (83%), PRO completion (85%), and days wearing the Fitbit (85%) indicated feasibility. Satisfaction with completing the PROs (M = 3.2, SD = 0.5) and wearing the Fitbit (M = 2.9, SD = 0.5) indicated acceptability. Preliminary data patterns suggested that participants with better treatment response (vs. progressive disease) had a higher toxicity burden. Conclusions: Longitudinal PRO and activity data collection was feasible and acceptable. Data collected on a larger scale may be used to specify risk prediction models to identify predictors of severe CAR-T-related toxicities and inform early interventions.

摘要

背景:嵌合抗原受体T细胞疗法(CAR-T)后,临床医生必须密切监测患者的毒性反应。患者报告的结局(PROs)(如毒性反应、生活质量)和活动数据(如步数、睡眠)可补充临床医生的观察结果。本研究测试了在CAR-T治疗期间从血液系统恶性肿瘤患者中收集PROs和活动数据的可行性和可接受性,并探索了初步的数据模式。方法:参与者佩戴Fitbit追踪器,并在输注后90天内的多个时间点完成PROs。通过招募(≥50%)、留存率(≥70%)、PRO完成率(≥70%)和佩戴Fitbit的天数(≥50%)的先验基准评估可行性。通过参与者满意度(先验基准在0-4量表上>2)评估可接受性。结果:参与者(N=12)的平均年龄M=66岁(标准差SD=7)。招募率(68%)、留存率(83%)、PRO完成率(85%)和佩戴Fitbit的天数(85%)表明具有可行性。对完成PROs(M=3.2,SD=0.5)和佩戴Fitbit(M=2.9,SD=0.5)的满意度表明具有可接受性。初步数据模式表明,治疗反应较好(与疾病进展相比)的参与者毒性负担更高。结论:纵向PRO和活动数据收集是可行且可接受的。更大规模收集的数据可用于指定风险预测模型,以识别严重CAR-T相关毒性的预测因素并为早期干预提供信息。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/bb87/9179384/9fe4194d9b4c/cancers-14-02742-g003.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/bb87/9179384/060347e60d6f/cancers-14-02742-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/bb87/9179384/a71af95b14d3/cancers-14-02742-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/bb87/9179384/9fe4194d9b4c/cancers-14-02742-g003.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/bb87/9179384/060347e60d6f/cancers-14-02742-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/bb87/9179384/a71af95b14d3/cancers-14-02742-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/bb87/9179384/9fe4194d9b4c/cancers-14-02742-g003.jpg

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[2]
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[3]
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[4]
Essential Components of an Electronic Patient-Reported Symptom Monitoring and Management System: A Randomized Clinical Trial.

JAMA Netw Open. 2024-9-3

[5]
The acceptability of using wearable electronic devices to monitor physical activity of patients with Multiple Myeloma undergoing treatment: a systematic review.

Clin Hematol Int. 2024-7-29

[6]
Feasibility of collecting longitudinal patient-reported outcomes in individuals with relapsed or refractory large B-cell lymphoma who received chimeric antigen receptor T-cell (CART) therapy.

BMC Cancer. 2024-8-9

[7]
Toward a more patient-centered drug development process in clinical trials for patients with myelodysplastic syndromes/neoplasms (MDS): Practical considerations from the International Consortium for MDS (icMDS).

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[8]
Protocol for a mixed-methods study to develop and feasibility test a digital system for the capture of patient-reported outcomes (PROs) in patients receiving chimeric antigen receptor T-cell (CAR-T) therapies (the PRO-CAR-T study).

BMJ Open. 2024-3-29

[9]
Health-related quality of life in patients with hematologic malignancies treated with chimeric antigen receptor T-cell therapy: review and current progress.

Haematologica. 2024-8-1

[10]
Patient-Reported Outcomes among Multiple Myeloma Patients Treated with Standard of Care Idecabtagene Vicleucel.

Cancers (Basel). 2023-9-25

本文引用的文献

[1]
Axicabtagene Ciloleucel as Second-Line Therapy for Large B-Cell Lymphoma.

N Engl J Med. 2022-2-17

[2]
Early toxicity and clinical outcomes after chimeric antigen receptor T-cell (CAR-T) therapy for lymphoma.

J Immunother Cancer. 2021-8

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J Cancer Educ. 2021-7

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NPJ Digit Med. 2021-3-10

[8]
Biomarkers for Predicting Cytokine Release Syndrome following CD19-Targeted CAR T Cell Therapy.

J Immunol. 2021-4-1

[9]
Idecabtagene Vicleucel in Relapsed and Refractory Multiple Myeloma.

N Engl J Med. 2021-2-25

[10]
Five-Year Outcomes for Refractory B-Cell Lymphomas with CAR T-Cell Therapy.

N Engl J Med. 2021-2-18

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