Department of Internal Medicine I, University Hospital Carl Gustav Carus, TU, Dresden, Germany.
Cellex Collection Center GmbH, Cologne, Germany.
Bone Marrow Transplant. 2021 Mar;56(3):635-645. doi: 10.1038/s41409-020-01053-4. Epub 2020 Oct 7.
We conducted a prospective clinical trial to investigate the safety and efficacy of plerixafor (P) in allogeneic peripheral blood stem cells (PBSC) donors with poor mobilization response to standard-dose granulocyte colony-stimulating factor (G-CSF), defined by <2 × 10 CD34 + cells/kg recipient body-weight (CD34+/kg RBW) after 1st apheresis. A single dose of 240 µg/kg P was injected subcutaneously at 10 p.m. on the day of the 1st apheresis. Thirty-seven allogeneic PBSC donors underwent study treatment. The median CD34+ count in peripheral blood was 15/µl on Day 1 after G-CSF alone, versus 44/µl on Day 2 after G-CSF plus P (p < 0.001). The median yield of CD34+ cells was 1.1 × 10 on Day 1 and 2.8 × 10 on Day 2. In contrast to a median yield of only 1.31 × 10 CD CD34+/kg RBW on Day 1, triggering study inclusion, a median of 3.74 × 10 CD CD34+/kg RBW were collected with G-CSF plus P on Day 2. Of 37 donors, 21 reached the target cell count of >4.5 × 10 CD34+/kg RBW (57%, 95%CI 40-73%). No donor experienced a severe adverse event requiring treatment. In conclusion, P might be considered on a case-by-case basis for healthy allogeneic donors with very poor stem cell mobilization success after G-CSF.
我们进行了一项前瞻性临床试验，以研究在对标准剂量粒细胞集落刺激因子（G-CSF）反应不佳的异基因外周血造血干细胞（PBSC）供者中，用培非格司亭（P）的安全性和疗效，其定义为首次单采后 1 天受体体重（RBW）<2×10 CD34+细胞/kg 的<2×10 CD34+细胞/kg RBW。在首次单采的前一天晚上 10 点，皮下注射 240µg/kg P 单次剂量。37 名异基因 PBSC 供者接受了研究治疗。G-CSF 单独治疗后第 1 天外周血中 CD34+计数中位数为 15/µl，而 G-CSF 加 P 后第 2 天为 44/µl（p<0.001）。CD34+细胞的中位产量第 1 天为 1.1×10，第 2 天为 2.8×10。与首次单采仅获得的中位数为 1.31×10 CD34+/kg RBW 相比，触发研究纳入标准，G-CSF 加 P 后第 2 天中位数为 3.74×10 CD34+/kg RBW。在 37 名供者中，有 21 名达到>4.5×10 CD34+/kg RBW 的目标细胞计数（57%，95%CI 40-73%）。没有供者出现需要治疗的严重不良事件。总之，对于 G-CSF 后非常低的干细胞动员成功的健康异基因供者，可以考虑个案使用 P。
