Memorial Sloan Kettering Cancer Center, New York, NY, United States of America.
New York Blood Center, New York, NY, United States of America.
Blood Cells Mol Dis. 2021 Sep;90:102588. doi: 10.1016/j.bcmd.2021.102588. Epub 2021 Jun 15.
We present data from our study of plerixafor mobilization (NCT02193191) relevant to the question of whether further dose escalation of plerixafor can address inconsistent adequacy of CD34+ mobilization for gene therapy of sickle cell disease (SCD). We found that, in the same patient, higher plerixafor dosing was associated with higher fold increases in PB CD34+ count, but not necessarily higher absolute CD34+ counts. Variation in pre-apheresis absolute CD34+ counts was related to intra-individual variation in baseline PB CD34+ counts and inter-individual variation in responsiveness to plerixafor. Overall, our results support further studies of continued dose escalation of plerixafor for autologous HPC collection in SCD.
培瑞克昔福动员（NCT02193191），即进一步提高培瑞克昔福剂量是否可以解决镰状细胞病（SCD）基因治疗中 CD34+动员不一致的问题。我们发现，在同一患者中，较高的培瑞克昔福剂量与 PB CD34+计数的更高倍数增加相关，但不一定与绝对 CD34+计数相关。预处理绝对 CD34+计数的变化与个体内基线 PB CD34+计数的变化以及对培瑞克昔福的反应性的个体间变化相关。总的来说，我们的结果支持进一步研究继续提高培瑞克昔福剂量，以用于 SCD 患者的自体造血细胞采集。
