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不同美法仑制剂预处理对淋巴肿瘤自体造血干细胞移植患者腹泻发生率及严重程度的影响

Effect of different melphalan formulation pretreatment on the incidence and severity of diarrhea in patients with lymphoid tumors undergoing autologous hematopoietic stem cell transplantation

  • 摘要:
    目的 探讨接受含丙二醇美法仑(propylene glycol-melphalan, PG-MEL)和不含丙二醇美法仑(PG-free MEL)预处理的患者在自体造血干细胞移植(autologous hematopoietic stem cell transplantation, AHSCT)中腹泻发生率及严重程度的差异。
    方法 回顾性分析2024年1月至2025年11月在复旦大学附属中山医院(厦门)血液科接受高剂量MEL(high dose MEL, HDM)预处理的30例AHSCT患者的临床资料。根据制剂分为PG-MEL组(n=15)和PG-free MEL组(n=15),比较两组患者腹泻发生率、发生时间、每日最大腹泻次数、持续时间、腹泻类型及严重程度,以及口腔黏膜炎、呕吐、感染、发热等相关不良事件及造血重建情况。
    结果 两组患者腹泻总体发生率均为93.3%,腹泻发生时间差异无统计学意义(4.0±2.0)d vs(3.0±2.0)d。与PG-MEL组相比,PG-free MEL组每日最大腹泻次数更多8(6,14)次 vs 6(4,6)次, P=0.009,3~4级腹泻比例更高(57.1% vs 14.3%, P=0.046),腹泻持续时间更长(8.0±4.0)d vs(5.0±2.0)d, P=0.033。两组口腔黏膜炎、呕吐、感染、发热等不良事件发生率、中性粒细胞及血小板植入时间差异无统计学意义。
    结论 接受PG-MEL与PG-free MEL的患者在AHSCT中的腹泻总体发生率相同,但PG-free MEL组腹泻更严重、持续时间更久。未发现两种制剂在其他不良反应及短期移植结局方面的差异。

     

    Abstract:
    Objective To explore the differences in the incidence and severity of diarrhea between patients receiving propylene glycol-melphalan (PG-MEL) or propylene glycol-free melphalan (PG-free MEL) pretreatment during autologous hematopoietic stem cell transplantation (AHSCT).
    Methods The clinical data of 30 patients who underwent AHSCT with high-dose MEL (HDM) pretreatment at the Department of Hematology, Zhongshan Hospital (Xiamen), Fudan University, from January 2024 to November 2025 were retrospectively analyzed. Patients were divided into the PG-MEL group (n=15) and the PG-free MEL group (n=15) according to the formulation. The incidence, onset time, maximum daily frequency, duration, type, and severity of diarrhea were compared between the two groups. Oral mucositis, vomiting, infection, fever, and other related adverse events, as well as hematopoietic recovery, were also observed.
    Results The overall incidence of diarrhea was 93.3% in both groups. There was no significant difference in the onset time of diarrhea between the two groups (4.0±2.0 d vs 3.0±2.0 d). Compared with the PG-MEL group, the PG-free MEL group had a higher maximum daily frequency of diarrhea (8 6,14 times vs 6 4,6 times, P=0.009), a higher proportion of grade 3–4 diarrhea (57.1% vs 14.3%, P=0.046), and a longer duration of diarrhea (8.0±4.0 d vs 5.0±2.0 d, P=0.033). No statistically significant differences were observed between the two groups in the incidence of other adverse events such as oral mucositis, vomiting, infection, fever, or neutrophil and platelet engraftment times.
    Conclusions PG-MEL and PG-free MEL showed the same overall incidence of diarrhea in AHSCT. However, the PG-free MEL group experienced more severe diarrhea and longer duration. There were no significant differences between the two formulations in other adverse reactions or short-term transplant outcomes.

     

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