Ahmed Nausheen, Wesson William, Mushtaq Muhammad Umair, Bansal Rajat, AbdelHakim Haitham, Bromert Sarah, Appenfeller Allison, Ghazal Batool Abu, Singh Anurag, Abhyankar Sunil, Ganguly Siddhartha, McGuirk Joseph, Abdallah Al-Ola, Shune Leyla
Division of Hematologic Malignancies & Cellular Therapeutics, University of Kansas Cancer Center, Westwood, KS, United States.
School of Medicine, University of Kansas, Kansas, KS, United States.
Front Oncol. 2023 Aug 3;13:1206715. doi: 10.3389/fonc.2023.1206715. eCollection 2023.
The first-in-class approved BCMA CAR-T therapy was idecabtagene vicleucel (ide-cel), approved in March 2021, for RRMM patients who progressed after 4 or more lines of therapy. Despite the promising outcomes, there were limited apheresis/production slots for ide-cel. We report outcomes of patients at our institution who were on the "waitlist" to receive ide-cel in 2021 and who could not secure a slot.
We conducted a retrospective review of RRMM patients evaluated at the University of Kansas Cancer Center for ide-cel from 3/2021-7/2021. A retrospective chart review was performed to determine patient and disease characteristics. Descriptive statistics were reported using medians for continuous variables. Survival analysis from initial consult was performed using Kaplan-Meier Survival estimator.
Forty patients were eligible and were on the "waitlist" for CAR-T. The median follow-up was 14 months (2-25mo). Twenty-four patients (60%) secured a production slot and 16 (40%) did not. The median time from consult to collection was 38 days (8-703). The median time from collection to infusion was 42 days (34-132 days). The median overall survival was higher in the CAR-T group (NR vs 9 mo, p<0.001).
Many patients who were eligible for ide-cel were not able to secure a timely slot in 2021. Mortality was higher in this group, due to a lack of comparable alternatives. Increasing alternate options as well as improvement in manufacturing and access is an area of high importance to improve RRMM outcomes.
首款获批的靶向B细胞成熟抗原（BCMA）的嵌合抗原受体T细胞（CAR-T）疗法是阿基仑赛注射液（ide-cel），于2021年3月获批用于接受过4线或更多线治疗后病情进展的复发/难治性多发性骨髓瘤（RRMM）患者。尽管疗效显著，但阿基仑赛注射液的单采/生产名额有限。我们报告了2021年在本机构等待接受阿基仑赛注射液治疗但未能获得名额的患者的治疗结果。
我们对2021年3月至7月在堪萨斯大学癌症中心接受阿基仑赛注射液评估的RRMM患者进行了回顾性研究。通过回顾病历确定患者和疾病特征。连续变量采用中位数进行描述性统计。使用Kaplan-Meier生存估计器从初次咨询开始进行生存分析。
40名患者符合条件并在CAR-T治疗的“等待名单”上。中位随访时间为14个月（2 - 25个月）。24名患者（60%）获得了生产名额，16名（40%）未获得。从咨询到采集的中位时间为38天（8 - 703天）。从采集到输注的中位时间为42天（34 - 132天）。CAR-T组的中位总生存期更高（未达到 vs 9个月，p<0.001）。
2021年，许多符合阿基仑赛注射液治疗条件的患者未能及时获得名额。由于缺乏可比的替代方案，该组患者的死亡率更高。增加替代方案以及改善生产和可及性是改善RRMM治疗结果的重要领域。
