CAR-T(CAR-T)细胞疗法在非肿瘤性疾病中的应用
Chimeric antigen receptor T (CAR-T) cell therapy in non-oncological diseases.
CAR-T(CAR-T)细胞在血液系统恶性肿瘤中的应用推动了这种免疫治疗形式的显著进展。
CELL INTELLIGENCE · 肿瘤细胞治疗研究
肿瘤细胞治疗研究
英文原题:ASTCT Committee on Practice Guidelines Survey on Evaluation and Management of Relapsed/Refractory Multiple Myeloma after Failure of Chimeric Antigen Receptor T Cell Therapy.
ASTCT Committee on Practice Guidelines Survey on Evaluation and Management of Relapsed/Refractory Multiple Myeloma after Failure of Chimeric Antigen Receptor T Cell Therapy.
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CAR-T 细胞疗法(CAR-T)已革新复发和/或难治性多发性骨髓瘤(RRMM)的管理。然而,CAR-T 治疗失败并不少见,仍是重大治疗挑战。不同移植和细胞治疗项目在评估及处理RRMM患者CAR-T 治疗失败方面存在显著差异。美国移植与细胞治疗学会(ASTCT)实践指南委员会于2023年9月至12月开展在线横断面调查,以了解骨髓瘤、移植和细胞治疗医生在监测、诊断及管理CAR-T 治疗失败方面的实践模式。本调查旨在了解临床实践并确定进一步研究领域。调查邮件发送给1,311名ASTCT医生会员,其中80人(6.1%)完成调查。受访者中58%为白人、66%为男性,51%拥有超过10年临床经验;多数(89%)受访者隶属大学/教学中心,56%从事以骨髓瘤为主的移植和/或细胞治疗。
CAR-T 后通常每4周进行一次监测实验室检查;监测骨髓活检和/或影像学检查最常在3个月时进行。64%的受访者经常或总是考虑通过活检或影像学确认复发。CAR-T 治疗失败后最受欢迎的挽救方案为:复发发生在3个月内时采用靶向GPRC5D免疫疗法(30%);复发超过3个月时采用靶向BCMA双特异性疗法(32.5%)。41%的受访者认为CAR-T 后长期血细胞减少经常或总是妨碍后续治疗;53%曾采用干细胞增补缓解这一问题。中心间实践模式存在较大差异,凸显了开展协作研究和制定专家临床建议的必要性,以明确CAR-T 后疾病监测最佳实践、治疗失败的优化检查方案及挽救治疗选择。
Chimeric antigen receptor T cell therapy (CAR-T) has revolutionized the management of relapsed and/or refractory multiple myeloma (RRMM).
However, CAR-T treatment failure is not uncommon and remains a major therapeutic challenge. There is substantial variability across transplantation and cellular therapy programs in assessing and managing post-CAR-T failures in patients with RRMM. The American Society for Transplantation and Cellular Therapy (ASTCT) Committee on Practice Guidelines conducted an online cross-sectional survey between September 2023 and December 2023 to determine myeloma, transplantation, and cellular therapy physicians' practice patterns for the surveillance, diagnosis, and management of CAR-T failure. The intent of this survey was to understand clinical practice patterns and identify areas for further investigation. Email surveys were sent to 1311 ASTCT physician members, of whom 80 (6. 1%) completed the survey. The respondents were 58% white and 66% male, and 51% had >10 years of clinical experience. Most (89%) respondents were affiliated with a university/teaching center, and 56% had a myeloma-focused transplantation and/or cellular therapy practice.
Post-CAR-T surveillance laboratory studies were commonly done every 4 weeks, and surveillance bone marrow biopsies and/or imaging surveillance were most commonly done at 3 months. Sixty-four percent of the respondents would often or always consider biopsy or imaging to confirm relapse. The most popular post-CAR-T failure rescue regimen was GPRC5D-directed immunotherapy (30%) for relapses occurring 3 months and BCMA-directed bispecific therapies (32. 5%) for relapse at >3 months.
Forty-one percent of the respondents endorsed post-CAR-T prolonged cytopenia as being "often" or "always" a barrier to next-line therapy; 53% had offered stem cell boost as a mitigation approach. Substantial across-center variation in practice patterns raises the need for collaborative studies and expert clinical recommendations to describe best practices for post-CAR-T disease surveillance, optimal workup for treatment failure, and choice of rescue therapies.
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