Supplementary Material for: Mobilization and hematopoietic stem cell collection in poor mobilizing patients with lymphoma: Final results of the German OPTIMOB study

Introduction: Successful mobilization and collection of peripheral hematopoietic stem cells (HSCs) is necessary for lymphoma patients eligible for myeloablative chemotherapy with subsequent autologous stem cell transplantation (ASCT). Albeit granulocyte-colony stimulating factor alone or combined with chemotherapy are well established methods for HSC mobilization, up to 40% of the patients fail to mobilize (poor mobilizer, PM). Plerixafor (PLX) is commonly used in PM patients resulting in increased migration of HSCs into peripheral blood (PB) and thus improves the collection outcome. Methods: The prospective, multicenter, open-label, non-interventional OPTIMOB study assessed mobilization and collection parameter of patients with lymphoma or multiple myeloma to get deep insights in the treatment of those patients in clinical routine focusing on PM patients. PM were defined as follows: 1) no achievement of ≥ 20 CD34+ progenitor cells/µL before first apheresis, 2) PLX administration at any timepoint during the observational period, 3) reduction of the initially planned CD34+ progenitor cell yield as necessity due to failed mobilization or HSC collection, and 4) no performance of apheresis due to low CD34+ progenitor level. Primary objective of the study was to assess mobilization success by the proportion of PM patients achieving > 2 x 10^6 CD34+ progenitor cells/kg body weight on the first day of apheresis. Here, the data of the lymphoma cohort are presented. Results: Out of 238 patients with lymphoma documented in the study, 32% were classified as PM. 87% of them received PLX. Demographic data revealed no obvious differences between PM and good mobilizing (GM) patients. All patients were treated highly individualized prior to mobilization. The majority of all PM patients were able to undergo apheresis (95%) and reached their individual requested CD34+ progenitor cell target (72%). 57% of the PM patients achieved > 2.0 x 10^6 CD34+ progenitor cells/kg body weight on day 1 of apheresis and nearby 70% of them underwent ASCT. Median time to engraftment was similar in PM and GM patients of the lymphoma cohort. Conclusions: Majority of PM patients with lymphoma were successfully mobilized and underwent ASCT. Most of them received PLX during the study.

引言：对于符合清髓性化疗联合后续自体造血干细胞移植（autologous stem cell transplantation, ASCT）指征的淋巴瘤患者而言，成功动员并采集外周血造血干细胞（peripheral hematopoietic stem cells, HSCs）是必不可少的临床环节。尽管单用粒细胞集落刺激因子（granulocyte-colony stimulating factor）或联合化疗是目前公认的造血干细胞动员方案，但仍有高达40%的患者出现动员失败，这类患者被称为动员不良者（poor mobilizer, PM）。普乐沙福（Plerixafor, PLX）常用于动员不良者患者，可促进造血干细胞向外周血（peripheral blood, PB）迁移，从而改善干细胞采集效果。
方法：本项前瞻性、多中心、开放标签、非干预性的OPTIMOB研究，针对淋巴瘤或多发性骨髓瘤患者，重点聚焦动员不良者群体，评估其临床实践中的造血干细胞动员与采集参数，以期深入了解该类患者的诊疗现状。动员不良者的定义如下：① 首次单采术前未达到≥20个CD34+祖细胞/微升；② 观察期内任意时间点接受普乐沙福治疗；③ 因动员失败或造血干细胞采集失败，不得不下调初始计划的CD34+祖细胞采集量；④ 因CD34+祖细胞水平过低而未实施单采术。本研究的主要终点为：通过动员不良者中单采首日达到＞2×10^6个CD34+祖细胞/千克体重的患者占比，评估造血干细胞动员成功情况。本文将呈现淋巴瘤队列的相关数据。
结果：本研究共纳入238例淋巴瘤患者，其中32%被归类为动员不良者，该群体中87%接受了普乐沙福治疗。人口统计学数据显示，动员不良者与动员良好者（good mobilizing, GM）之间无显著差异。所有患者在动员前均接受了高度个体化的治疗方案。绝大多数动员不良者能够接受单采术（95%），且72%的患者达到了其个体化设定的CD34+祖细胞采集目标。57%的动员不良者在单采首日达到＞2.0×10^6个CD34+祖细胞/千克体重，其中近70%的患者后续接受了自体造血干细胞移植。淋巴瘤队列中，动员不良者与动员良好者的造血植入中位时间无显著差异。
结论：多数淋巴瘤合并动员不良的患者可成功完成造血干细胞动员并接受自体造血干细胞移植，其中大部分患者在研究期间接受了普乐沙福治疗。