This retrospective observational study (NEUF) included adult patients with B-cell acute lymphoblastic leukemia (B-cell ALL) who had received blinatumomab for the treatment of minimal residual disease-positive (MRD+) or relapsed/refractory (R/R) B-cell ALL via an expanded access program (EAP). Patients were eligible if blinatumomab was initiated via the EAP between January 2014 and June 2017. Patients were followed from blinatumomab initiation until death, entry into a clinical trial, the end of follow-up, or the end of the study period (December 31, 2017), whichever occurred first. Of the 249 adult patients included, 109 were MRD+ (83 Philadelphia chromosome-negative [Ph−] and 26 Philadelphia chromosome-positive [Ph+]) and 140 had a diagnosis of R/R B-cell ALL (106 Ph− and 34 Ph+). In the MRD+ group, within the first cycle of blinatumomab treatment, 93% (n = 49/53) of Ph− and 64% (n = 7/11) of Ph+ patients with evaluable MRD achieved an MRD response (MRD <0.01%). Median overall survival (OS) was not reached over a median follow-up time of 18.5 months (Ph−, 18.8 [range: 5.1–34.8] months; Ph+, 16.5 [range: 1.8–31.6] months). In the R/R group, within two cycles of blinatumomab, 51% of Ph− and 41% of Ph+ patients achieved complete hematologic remission (CR/CRh/CRi), and 83% of Ph− and 67% of Ph+ MRD-evaluable patients in CR/CRh/CRi achieved an MRD response. Median (95% confidence interval) OS was 12.2 (7.3–24.2) months in the R/R Ph− subgroup and 16.3 (5.3–not estimated) months in the R/R Ph+ subgroup. This large, real-world data set of adults with B-cell ALL treated with blinatumomab confirms efficacy outcomes from published studies.
这项回顾性观察研究（NEUF）纳入了通过扩大准入项目接受贝林妥欧单抗治疗微小残留病阳性或复发/难治性B细胞急性淋巴细胞白血病的成年患者。患者需在2014年1月至2017年6月期间通过扩大准入项目开始接受贝林妥欧单抗治疗。随访时间从贝林妥欧单抗治疗开始直至患者死亡、进入临床试验、随访结束或研究截止日期（2017年12月31日），以先发生者为准。在纳入的249例成年患者中，109例为微小残留病阳性（其中83例费城染色体阴性，26例费城染色体阳性），140例诊断为复发/难治性B细胞急性淋巴细胞白血病（其中106例费城染色体阴性，34例费城染色体阳性）。在微小残留病阳性组中，首个贝林妥欧单抗治疗周期内，93%的可评估费城染色体阴性患者（49/53）和64%的可评估费城染色体阳性患者（7/11）达到微小残留病缓解（微小残留病<0.01%）。在中位随访时间为18.5个月期间，中位总生存期尚未达到（费城染色体阴性亚组为18.8个月[范围：5.1-34.8]，费城染色体阳性亚组为16.5个月[范围：1.8-31.6]）。在复发/难治性组中，两个贝林妥欧单抗治疗周期内，51%的费城染色体阴性患者和41%的费城染色体阳性患者达到完全血液学缓解，而达到完全血液学缓解的患者中，83%的费城染色体阴性患者和67%的费城染色体阳性患者实现微小残留病缓解。复发/难治性费城染色体阴性亚组的中位总生存期为12.2个月（95%置信区间：7.3-24.2），复发/难治性费城染色体阳性亚组为16.3个月（95%置信区间：5.3-未估计）。这项关于贝林妥欧单抗治疗B细胞急性淋巴细胞白血病成年患者的大规模真实世界数据集，证实了已发表研究中的疗效结果。
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