Department of Pathology and Cell Biology, Columbia University Medical Center, New York, New York 10032, USA.
Cytotherapy. 2012 Apr;14(4):467-72. doi: 10.3109/14653249.2012.658912. Epub 2012 Feb 20.
Plerixafor was recently approved for use in combination with granulocyte-colony-stimulating factor (G-CSF) for hematopoietic progenitor cell (HPC) collection by apheresis in adults with multiple myeloma (MM) or non-Hodgkin lymphoma (NHL). However, its efficacy in pediatric patients is not well-studied; thus, we report on our institutional experience with this population.
A retrospective observational analysis was performed using both stem cell-processing laboratory information as well as apheresis charts and medical records on all pediatric patients who received plerixafor as part of the mobilization regimen between December 2006 and December 2010. The primary outcome was collection yield. Secondary outcomes included the ability to undergo autologous hematopoietic stem cell transplantation (auto-HSCT) and engraftment status.
Eighteen HPC collections by apheresis representing seven mobilization courses were performed on five pediatric patients with poor mobilization status (three males, two females; median age 14 years). Median pre-harvest peripheral blood CD34(+) cell (PB CD34(+)) count was 6.88/μL. A strong correlation between pre-harvest PB CD34(+) count and collection yield was observed. Median total collection yield was 2.26 × 10(6) CD34(+) cells/kg. Four patients achieved a minimum collection of 2 × 10(6) CD34(+) cells/kg. Three patients underwent auto-HSCT with a median neutrophil and platelet engraftment of 12 and 34 days, respectively. No major adverse events with plerixafor administration or apheresis collections were reported.
Plerixafor in combination with G-CSF is a safe and potentially helpful mobilization agent in poor mobilizers. Further studies should be done to evaluate the true efficacy of plerixafor in the pediatric population.
plerixafor 最近被批准与粒细胞集落刺激因子（G-CSF）联合使用，用于多发性骨髓瘤（MM）或非霍奇金淋巴瘤（NHL）成人患者通过外周血造血干细胞采集（apheresis）采集造血祖细胞（HPC）。然而，其在儿科患者中的疗效尚未得到充分研究；因此，我们报告了我们在该人群中的机构经验。
使用干细胞处理实验室信息以及所有接受 plerixafor 作为动员方案一部分的儿科患者的外周血造血干细胞采集图表和病历进行回顾性观察分析。主要结局是采集产量。次要结局包括进行自体造血干细胞移植（auto-HSCT）的能力和植入状态。
对 5 名动员状态不佳的儿科患者（3 名男性，2 名女性；中位年龄 14 岁）进行了 18 次 HPC 采集，共进行了 7 次动员疗程。中位收获前外周血 CD34+细胞（PB CD34+）计数为 6.88/μL。观察到收获前 PB CD34+计数与采集产量之间存在很强的相关性。中位总采集产量为 2.26×106 CD34+细胞/kg。4 名患者达到了 2×106 CD34+细胞/kg 的最低采集量。3 名患者接受了自体 HSCT，中性粒细胞和血小板植入的中位时间分别为 12 天和 34 天。没有报道与 plerixafor 给药或外周血造血干细胞采集相关的重大不良事件。
plerixafor 联合 G-CSF 是动员不佳患者的一种安全且潜在有效的动员剂。应进一步研究评估 plerixafor 在儿科人群中的真正疗效。
