Division of Hematology, Oncology, Blood and Bone Marrow Transplant, Nationwide Children's Hospital, Columbus, Ohio, USA.
Department of Pediatrics, The Ohio State University College of Medicine, Columbus, Ohio, USA.
Transfusion. 2021 Mar;61(3):894-902. doi: 10.1111/trf.16260. Epub 2021 Jan 21.
Pleraxifor for peripheral blood stem cell (PBSC) mobilization in children with malignancies is often given following failure of standard mobilization (SM) rather than as a primary mobilizing agent.
In this retrospective multicenter study, we report the safety of plerixafor-based PBSC mobilization in children with malignancies and compare outcomes between patients who received plerixafor upfront with SM (Group A) with those who received plerixafor following failure of SM (Group B). In the latter pleraxifor was given either following a low peripheral blood (PB) CD34 (<20 cells/cu.mm) (Group B1) or as a second collection process due to an unsuccessful yield (CD34 + < 2 × 10 /kg) (Group B2) following failed SM and first apheresis attempts.
The study cohort (n = 47) with a median age of 8 (range 0.6-21) year, comprised 19 (40%) Group A and 28 (60%) Group B patients (B1 = 12 and B2 = 16). Pleraxifor mobilization was successful in 87.2% of patients, similar between Groups A and B (84.2% vs 89.2%) and resulted in a median 4-fold increase in PB CD34. Median number of apheresis attempts was 2 in Groups A and B1 but 4 in Group B2. In Group B2, median total CD34+ yield post-plerixafor was 9-fold higher than after SM (P = .0013). Mild to moderate transient adverse events affected 8.5% of patients. Among patients who proceeded to autologous transplant (n = 39), all but one engrafted.
Plerixafor-based PBSC collection was safe and effective in our cohort and supports consideration as a primary mobilizing agent in children with malignancies.
培瑞克昔福（plerixafor）通常用于治疗儿童恶性肿瘤患者在外周血造血干细胞（PBSC）动员失败后的补救性治疗，而非作为主要的动员剂。
在这项回顾性多中心研究中，我们报告了培瑞克昔福在恶性肿瘤儿童中的 PBSC 动员安全性，并比较了接受培瑞克昔福一线治疗与动员失败后接受培瑞克昔福治疗（SM）的患者（A 组）之间的结果。在后一组中，培瑞克昔福是在 PB 中 CD34 计数低（<20 个细胞/立方毫米）（B1 组），还是在 SM 和首次单采尝试失败后，由于产量不足（CD34+ <2×10 /kg）（B2 组）进行第二次采集过程时使用。
该研究队列（n = 47）中位年龄为 8 岁（范围 0.6-21 岁），包括 19 名（40%）A 组和 28 名（60%）B 组患者（B1 = 12 名，B2 = 16 名）。培瑞克昔福动员在 87.2%的患者中取得成功，A 组和 B 组之间相似（84.2% vs 89.2%），并导致 PB CD34 计数增加 4 倍。A 组和 B1 组的单采次数中位数为 2 次，但 B2 组为 4 次。在 B2 组中，培瑞克昔福治疗后 CD34+ 的总产量中位数是 SM 后 9 倍（P =.0013）。轻度至中度短暂不良反应影响了 8.5%的患者。在接受自体移植的患者（n = 39）中，除 1 例外，所有患者均植入成功。
在我们的队列中，培瑞克昔福为基础的 PBSC 采集是安全有效的，并支持将其作为恶性肿瘤儿童的主要动员剂。
