Center for Cell and Gene Therapy, Baylor College of Medicine, Houston Methodist Hospital, Texas Children's Hospital, Houston, TX.
Department of Pediatrics, Aflac Cancer and Blood Disorders Center of Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, GA.
Blood Adv. 2019 Sep 10;3(17):2571-2580. doi: 10.1182/bloodadvances.2019000371.
Cytomegalovirus (CMV) infections remain a major cause of morbidity and mortality after allogeneic hematopoietic stem cell transplantation (HSCT), and standard antiviral therapies are associated with significant side effects and development of drug-resistant mutants. Adoptively transferred donor-derived CMV-specific T cells (CMVSTs) can provide an alternative treatment modality with few side effects but are not widely available due to their patient-specific nature. Here we report the establishment and use of a bank of CMVSTs derived from just 8 CMV-seropositive donors, with HLA types representing the diverse US population, as an "off-the-shelf" therapy to treat drug-refractory infections. To date, we have screened 29 patients for study participation and identified a suitable line, with ≥2 of 8 shared HLA antigens, for 28 (96.6%) patients with a median of 4 shared HLA antigens. Of these, 10 patients with persistent/refractory CMV infections or disease were eligible for treatment; a single infusion of cells produced 3 partial responses and 7 complete responses, for a cumulative response rate of 100% (95% confidence interval, 69.2-100) with no graft-versus-host disease, graft failure, or cytokine release syndrome. Potential wider use of the tested CMVSTs across transplant centers is made more feasible by our ability to produce sufficient material to generate cells for >2000 infusions from a single donor collection. Our data indicate that a "mini" bank of CMVSTs prepared from just 8 well-chosen third-party donors can supply the majority of patients with an appropriately matched line that produces safe and effective anti-CMV activity post-HSCT.
巨细胞病毒 (CMV) 感染仍然是异基因造血干细胞移植 (HSCT) 后发病率和死亡率的主要原因,标准抗病毒治疗与显著的副作用和耐药突变体的发展有关。过继转移的供体来源的 CMV 特异性 T 细胞 (CMVSTs) 可以提供一种副作用较少的替代治疗方法,但由于其患者特异性,因此并不广泛应用。在这里,我们报告了从 8 名 CMV 血清阳性供体中建立和使用 CMVST 库的情况,这些供体的 HLA 类型代表了美国多样化的人群,作为一种“现成”的治疗方法来治疗耐药感染。迄今为止,我们已经筛选了 29 名患者参与研究,并确定了 28 名(96.6%)患者中有合适的谱系,这些患者有≥2 个 8 个共享 HLA 抗原,中位数为 4 个共享 HLA 抗原。其中,有 10 名持续性/难治性 CMV 感染或疾病患者符合治疗条件;单次输注细胞产生 3 个部分缓解和 7 个完全缓解,累积缓解率为 100%(95%置信区间,69.2-100),无移植物抗宿主病、移植物衰竭或细胞因子释放综合征。通过我们能够从单个供体采集产生足以用于超过 2000 次输注的细胞的能力,使得在移植中心更可行地使用经过测试的 CMVSTs。我们的数据表明,从仅 8 名精心挑选的第三方供体中制备的“迷你”CMVST 库可以为大多数患者提供适当匹配的谱系,从而在 HSCT 后产生安全有效的抗 CMV 活性。