肿瘤细胞治疗研究
英文原题：Expanded Natural Killer Cells Following Haploidentical HSCT for AML/MDS
这是一项 I/II 期注册临床试验，评估细胞治疗用于急性髓系白血病、骨髓增生异常综合征的安全性、可行性及初步疗效。当前状态：招募中。计划入组 10 例。试验地点：欧洲 · 巴塞尔（共 1 个中心）。登记号：NCT03300492。
不限性别 · ≥ 18 Years
纳入标准： 患者： • 年龄>18岁。 • 无 HLA 匹配的相关或无关供者。 • 患 AML 或 MDS-EB，且按巴塞尔大学医院干细胞移植团队指南适合 haplo-HSCT。 • 移植医生判断器官功能充分且无 haplo-HSCT 禁忌证。 • 有可用的相关半相合供者。 • 已签署书面知情同意。 供者： • 年龄>18岁，为半相合父母、兄弟姐妹或其他亲属。 • 按标准符合细胞捐献和单采条件。 • 已签署书面知情同意。 排除标准： 患者： • 急性早幼粒细胞白血病（APL）。 • 存在有临床意义的供者特异性抗 HLA 抗体（平均荧光强度>2,000）。 • 妊娠。 • 除 GVHD 预防外需使用免疫抑制剂。 供者：妊娠。
Inclusion Criteria: Patient: * \>18 years of age * No HLA-matched related or unrelated donor available * AML or MDS-EB with indication for a haplo-HSCT according to the guidelines of the University Hospital Basel Stem Cell Transplant Team * Judged by the transplant physicians to have adequate organ function and no contraindications to haplo-HSCT * Available related haploidentical donor * Written informed consent Donor: * \>18 years old, haploidentical parent, sibling or other relative * Donor suitable for cell donation and apheresis according to standard criteria * Written informed consent Exclusion Criteria: Patient: * APL diagnosis * Presence of relevant (mean fluorescence intensity \>2000) donor-specific anti-HLA antibodies * Pregnancy * Necessity of immunosuppression apart from GvHD prophylaxis Exclusion Criteria: Donor: • Pregnancy
以上为辅助阅读译文。是否适合入组须由主治医生判断，最终以登记平台与研究者确认为准。
主要终点：Incidence and severity of adverse events including GvHD and infections. · As defined by the CTCAE version 4.03 and the NIH Scoring of GvHD. · 1 year following haplo HSCT
次要终点：Progression-free survival (PFS)；Incidence of AML/MDS-EB complete morphological and molecular remission (CR) at day + 30, + 90, +180 and 1 year post allo-HSCT；Incidence of graft rejection；Number of NK cells given per kg body weight；Number of NK-DLI infusions applied
haplo-HSCT 后第+10、+15和+20天进行预防性免疫治疗，输注体外扩增 NK 细胞并逐步增加剂量。
本研究评估扩增自然杀伤（NK）细胞用于急性髓系白血病（AML）或骨髓增生异常综合征（MDS）患者半相合异基因造血干细胞移植（haplo-HSCT）后的应用。对于没有 HLA 匹配供者的 AML/MDS 患者，haplo-HSCT 是优选治疗方案。移植后使用环磷酰胺可使安全性接近 HLA 相合同胞供者移植，但 AML/MDS 复发仍是重要问题。NK 细胞可杀伤肿瘤细胞，尤其在 haplo-HSCT 环境下具有抗肿瘤活性。本研究中，接受 haplo-HSCT 的患者将在移植后接受各自干细胞供者来源的扩增 NK 细胞。主要目标是确定该方法的安全性和可行性，并评估 NK 细胞活性。
The study examines the application of expanded natural killer cells (NK cells) following haploidentical allogeneic hematopoietic stem cell transplantation (haplo-HSCT) for AML or MDS. Haplo-HSCT is a preferred treatment option for patients with AML or MDS without a HLA-matched donor. With administration of cyclophosphamide post-transplant , the safety of the procedure is similar to a HSCT from a HLA-identical donor. Relapse of AML/MDS represents a serious problem following haplo-HSCT. NK cells are immune cells able to destroy tumor cells. Their potency has been established particularly in the setting of a haplo-HSCT. In the current study, study participants undergoing haplo-HSCT will receive expanded NK cells from their respective stem-cell donors following haplo-HSCT. The primary goal of the study is to establish the safety and feasibility of this approach. In addition, the activity of the NK cells will be examined.
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