Goto Hideki, Sawa Masashi, Fujiwara Shin-Ichiro, Ri Masaki, Ishida Tadao, Takeuchi Masahiro, Ishitsuka Kenji, Toyosaki Masako, Sunami Kazutaka, Tsukada Junichi, Sonoki Takashi, Shimogomi Aiko, Ichihashi Yuki, Ouchi Yoshiumi, Miyamoto Toshihiro, Hino Masayuki, Maeda Yoshinobu, Teshima Takanori
Department of Hematology, Graduate School of Medicine, Hokkaido University Faculty of Medicine, Sapporo, Japan.
Division of Laboratory and Transfusion Medicine, Hokkaido University Hospital, Sapporo, Japan.
Sci Rep. 2025 Apr 25;15(1):14523. doi: 10.1038/s41598-025-98453-7.
This phase 2 study evaluated the impact of pegfilgrastim, a single-dose, long-acting granulocyte colony-stimulating factor, on the steady-state mobilization of hematopoietic stem cells into peripheral blood in patients with multiple myeloma (MM) or malignant lymphoma (ML). Efficacy and safety, along with CD34-positive cell mobilization outcomes were assessed in patients with MM, who were randomly assigned to pegfilgrastim (n = 30) or daily filgrastim (n = 31), and ML (pegfilgrastim only, n = 13) cohorts. In the MM cohort, CD34-positive cell counts ≥ 2 × 10/kg were achieved in 100% of patients in the pegfilgrastim group and 96.7% in the filgrastim group (difference: 3.3%; 80% confidence interval: -0.9-7.5%), demonstrating the non-inferiority of pegfilgrastim to filgrastim. All patients in the ML cohort achieved ≥ 2 × 10/kg CD34-positive cell counts. The plerixafor administration rates in the MM cohort were 50.0% and 63.3% in the pegfilgrastim and filgrastim groups, respectively, and 91.7% in the ML cohort. There were no major differences in safety measures between the two groups. Although the sample size was small, particularly in the ML cohort, a single dose of pegfilgrastim demonstrated comparable efficacy and safety to daily doses of filgrastim, indicating its potential for clinical use while reducing patient burden.Trial Registration: jRCT2011210029, NCT05007652.
这项2期研究评估了聚乙二醇化非格司亭（一种单剂量长效粒细胞集落刺激因子）对多发性骨髓瘤（MM）或恶性淋巴瘤（ML）患者造血干细胞向外周血稳态动员的影响。在MM患者（随机分为聚乙二醇化非格司亭组[n = 30]或每日非格司亭组[n = 31]）和ML患者（仅聚乙二醇化非格司亭组，n = 13）队列中评估了疗效、安全性以及CD34阳性细胞动员结果。在MM队列中，聚乙二醇化非格司亭组100%的患者实现了CD34阳性细胞计数≥2×10⁶/kg，非格司亭组为96.7%（差异：3.3%；80%置信区间：-0.9-7.5%），表明聚乙二醇化非格司亭不劣于非格司亭。ML队列中的所有患者均实现了CD34阳性细胞计数≥2×10⁶/kg。MM队列中，聚乙二醇化非格司亭组和非格司亭组的普乐沙福给药率分别为50.0%和63.3%，ML队列中为91.7%。两组之间的安全措施没有重大差异。尽管样本量较小，尤其是在ML队列中，但单剂量聚乙二醇化非格司亭显示出与每日剂量非格司亭相当的疗效和安全性，表明其在减轻患者负担的同时具有临床应用潜力。试验注册号：jRCT2011210029，NCT05007652。
