评估移植和CAR-T治疗受者的生活质量及症状:美国血液和骨髓移植学会(ASTCT)生存特别兴趣小组的专家小组建议
Assessing Quality of Life and Symptoms in Transplantation and CAR-T Recipients: Expert Panel Recommendations from the Survivorship Special Interest Group of ASTCT.
作者信息
Banerjee Rahul, Amonoo Hermioni L, Barata Anna, Bhatt Neel S, Espinoza-Gutarra Manuel R, Jayani-Kosarzycki Reena V, Katz Hannah, Kennedy Vanessa E, Nawas Mariam, Steineck Angela, Wanjiku Chris, Costanzo Erin, Cusatis Rachel N, Knight Jennifer M, Schoemans Helene, Sidana Surbhi, Wood William A, Sung Anthony D, Lee Catherine J, Hamilton Betty K
机构信息
Fred Hutchinson Cancer Center, Seattle, WA, USA.
Brigham and Women's Hospital, Boston, MA, USA.
出版信息
Transplant Cell Ther. 2025 Jul 1. doi: 10.1016/j.jtct.2025.06.030.
Patient-reported outcomes (PROs) to measure quality of life (QOL) and other symptoms play an increasingly important role in clinical trials and regulatory approvals for hematopoietic cell transplantation (HCT) and chimeric antigen receptor T-cell (CAR-T) therapy. However, their adoption has been hindered by wide heterogeneity in the choice of PRO measures for clinical research, including the Functional Assessment of Cancer Therapy Bone Marrow Transplantation (FACT-BMT) and the European Organization for Research and Treatment of Cancer Quality of Life Core Questionnaire (EORTC QLQ-C30) inventories. Additionally, the potential for PRO integration into routine standard-of-care (SOC) practice for patients undergoing HCT or CAR-T therapy has not yet been realized. As part of a coordinated effort by three American Society for Transplantation and Cellular Therapy (ASTCT) Special Interest Groups, we developed best practices for PRO integration in adult and pediatric recipients of HCT and CAR-T therapy. We strongly encourage the use of Patient-Reported Outcomes Measurement Information System (PROMIS) or PRO version of Common Terminology Criteria for Adverse Events (PRO-CTCAE) instruments as the primary PRO measures for most HCT and CAR-T trials. Measures such as the PROMIS-29 inventory can be used for QOL assessments, while PRO-CTCAE item banks can be used for specific symptoms. Rationales for our strong recommendation to move from FACT-BMT and EORTC QLQ-C30 to PROMIS/PRO-CTCAE instruments include: (1) free licensing and ease of implementation, including in electronic medical records; (2) psychometric validation in a variety of oncologic settings, including during inpatient hospitalizations; (3) translation into multiple languages, with validation in both adult and pediatric settings; and (4) adoption into centrally-collected PRO protocols from the Center for International Blood and Marrow Transplant Research for both HCT and CAR-T recipients. Steps to operationalize these PRO measures are discussed, as are methods to migrate existing data from legacy PRO instruments. We similarly recommend the consideration of PRO integration into SOC clinical practice, including the development of threshold-based workflows to both personalize and standardize care in this setting. Other panel recommendations include the use of standardized timepoints for longitudinal PRO assessments and the inclusion of patient advocates when implementing PRO measures. Implementing these steps will improve the ability of PROs to improve outcomes for patients undergoing HCT or CAR-T therapy, both in trials and - more importantly - in SOC practice.
患者报告结局(PROs)用于衡量生活质量(QOL)和其他症状,在造血细胞移植(HCT)和嵌合抗原受体T细胞(CAR-T)治疗的临床试验及监管审批中发挥着越来越重要的作用。然而,临床研究中PRO测量方法的选择存在广泛异质性,包括癌症治疗功能评估骨髓移植(FACT-BMT)和欧洲癌症研究与治疗组织生活质量核心问卷(EORTC QLQ-C30)量表,这阻碍了它们的采用。此外,将PRO整合到接受HCT或CAR-T治疗患者的常规标准治疗(SOC)实践中的潜力尚未实现。作为美国移植与细胞治疗学会(ASTCT)三个特别兴趣小组协调努力的一部分,我们制定了将PRO整合到成人和儿童HCT及CAR-T治疗受者中的最佳实践。我们强烈鼓励在大多数HCT和CAR-T试验中,使用患者报告结局测量信息系统(PROMIS)或不良事件通用术语标准的PRO版本(PRO-CTCAE)工具作为主要的PRO测量方法。诸如PROMIS-29量表可用于QOL评估,而PRO-CTCAE条目库可用于特定症状评估。我们强烈建议从FACT-BMT和EORTC QLQ-C30转向PROMIS/PRO-CTCAE工具的理由包括:(1)免费许可且易于实施,包括在电子病历中;(2)在多种肿瘤环境中进行了心理测量学验证,包括住院期间;(3)已翻译成多种语言,并在成人和儿童环境中进行了验证;(4)国际血液和骨髓移植研究中心已将其纳入针对HCT和CAR-T受者的集中收集PRO方案。文中讨论了实施这些PRO测量方法的步骤,以及从传统PRO工具迁移现有数据的方法。我们同样建议考虑将PRO整合到SOC临床实践中,包括制定基于阈值的工作流程,以便在这种情况下实现个性化和标准化护理。其他小组建议包括在纵向PRO评估中使用标准化时间点,以及在实施PRO测量时纳入患者权益倡导者。实施这些步骤将提高PRO改善接受HCT或CAR-T治疗患者结局的能力,无论是在试验中,更重要的是在SOC实践中。
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