Gehr Family Center for Leukemia Research, City of Hope, Duarte, California; Department of Hematology/Hematopoietic Cell Transplantation, City of Hope, Duarte, California.
Department of Computational and Quantitative Sciences, Beckman Research Institute, City of Hope, Duarte, California.
Transplant Cell Ther. 2024 Aug;30(8):788.e1-788.e9. doi: 10.1016/j.jtct.2024.06.013. Epub 2024 Jun 12.
CD19-targeted chimeric antigen receptor T cell (CAR-T) therapy has led to unprecedented rates of complete remission (CR) in children and adults with relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (B-ALL), yet the majority of adults relapse after initial response. One proposed method to extend the durability of remission in adults following response to CAR-T therapy is consolidation with allogeneic hematopoietic cell transplantation (alloHCT). Considering the limited published data for the utility of post CAR-T therapy consolidative alloHCT in r/r B-ALL, especially data related to patients receiving a second alloHCT, we sought to describe outcomes of patients with r/r B-ALL at our institution who received their first or second alloHCT following response to CAR-T therapy. We performed a retrospective analysis of adult patients with r/r B-ALL who responded to either investigational or standard of care (SOC) CD19-targeted CAR-T therapy and underwent consolidation with alloHCT while in CR without interim therapy. We identified 45 patients, of whom 26 (58%) and 19 (42%) received their first and second alloHCT as consolidation post CAR-T therapy, respectively. The median age was 31 years (range: 19-67) and 31 (69%) patients were Hispanic. Ph-like was the most common genetic subtype and comprised over half of cases (53%; n = 24). The median number of prior therapies pre-transplant was 5 (range: 2-7), and disease status at the time of alloHCT was CR1, CR2 or ≥CR3 in 7 (16%), 22 (49%) and 16 (35%) patients, respectively. The median time from CAR-T therapy until alloHCT was 93 (range: 42-262) days. The conditioning regimen was radiation-based myeloablative (MAC) in 22 (49%) patients. With a median follow-up of 2.47 years (range: 0.13-6.93), 2-year overall survival (OS), relapse free survival (RFS), cumulative incidence of relapse (CIR) and non-relapse mortality (NRM) were 57.3% (95% CI: 0.432-0.760), 56.2% (95% CI: 0.562-0.745), 23.3% (95% CI: 0.13-0.42), and 20.4% (95% CI: 0.109-0.384), respectively. Two-year OS (52% vs. 68%, P = .641), RFS (54% vs. 59%, P = .820), CIR (33.5% vs. 8.5%, P = .104), and NRM (12.5% vs. 32.2%, P = .120) were not significantly different between patients who underwent their first vs. second transplant, respectively. In univariate analysis, only Ph-like genotype was associated with inferior RFS (P = .03). AlloHCT post CAR-T response is associated with a relatively low early mortality rate and encouraging survival results in high-risk adults with r/r B-ALL, extending to the second alloHCT for fit and eligible patients.
CD19 靶向嵌合抗原受体 T 细胞 (CAR-T) 疗法在复发/难治性 (r/r) B 细胞急性淋巴细胞白血病 (B-ALL) 儿童和成人中导致了前所未有的完全缓解 (CR) 率,但大多数成人在初始反应后会复发。一种延长 CAR-T 治疗后缓解持久性的方法是在缓解后进行同种异体造血细胞移植 (alloHCT) 巩固治疗。考虑到关于 r/r B-ALL 患者在 CAR-T 治疗后接受巩固性 alloHCT 的效用的有限已发表数据,特别是与接受第二次 alloHCT 的患者相关的数据,我们试图描述在我们机构接受 r/r B-ALL 且在 CAR-T 治疗后缓解的患者接受第一次或第二次 alloHCT 的结果。我们对接受研究性或标准护理 (SOC) CD19 靶向 CAR-T 治疗且在无中间治疗的 CR 中接受 alloHCT 巩固治疗的 r/r B-ALL 成年患者进行了回顾性分析。我们确定了 45 名患者,其中 26 名 (58%)和 19 名 (42%)分别接受了他们的第一次和第二次 alloHCT 作为 CAR-T 治疗后的巩固治疗。中位年龄为 31 岁 (范围:19-67),31 名 (69%)患者为西班牙裔。Ph 样是最常见的遗传亚型,占半数以上病例 (53%;n = 24)。移植前中位数治疗次数为 5 次 (范围:2-7),alloHCT 时疾病状态分别为 CR1、CR2 或≥CR3 的患者为 7 名 (16%)、22 名 (49%)和 16 名 (35%)。从 CAR-T 治疗到 alloHCT 的中位时间为 93 天 (范围:42-262)。22 名 (49%)患者接受了基于辐射的清髓性 (MAC) 预处理方案。中位随访 2.47 年 (范围:0.13-6.93),2 年总生存率 (OS)、无复发生存率 (RFS)、累积复发率 (CIR) 和非复发死亡率 (NRM) 分别为 57.3% (95%CI:0.432-0.760)、56.2% (95%CI:0.562-0.745)、23.3% (95%CI:0.13-0.42)和 20.4% (95%CI:0.109-0.384)。两组 2 年 OS(52% vs. 68%,P =.641)、RFS(54% vs. 59%,P =.820)、CIR(33.5% vs. 8.5%,P =.104)和 NRM(12.5% vs. 32.2%,P =.120)在接受第一次与第二次移植的患者之间无显著差异。单变量分析显示,只有 Ph 样基因型与 RFS 较差相关 (P =.03)。CAR-T 治疗后alloHCT 与高危 r/r B-ALL 成人中相对较低的早期死亡率和令人鼓舞的生存结果相关,对于适合和有资格的患者,可延伸至第二次 alloHCT。