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在多发性骨髓瘤的自体干细胞移植中使用不含丙二醇的美法仑,可获得可比的结果。

Comparable outcomes using propylene glycol-free melphalan for autologous stem cell transplantation in multiple myeloma.

机构信息

Mayo Clinic School of Medicine, Rochester, MN, USA.

Division of Hematology, Department of Medicine, Mayo Clinic, Rochester, MN, USA.

出版信息

Bone Marrow Transplant. 2019 Apr;54(4):587-594. doi: 10.1038/s41409-018-0302-6. Epub 2018 Aug 16.

DOI:10.1038/s41409-018-0302-6
PMID:30116014
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC6377862/
Abstract

Autologous stem cell transplantation (ASCT) remains a mainstay in the treatment of multiple myeloma (MM). While the procedure is generally safe, toxicities associated with high-dose melphalan conditioning are common and significantly affect patient quality of life. Recently, a propylene glycol-free melphalan formulation (PG-free MEL; Evomela®) was approved by the United States Food and Drug Administration as an ASCT-conditioning regimen for MM. PG-free MEL is more soluble and stable than propylene glycol-solubilized melphalan (PG-solubilized MEL; Alkeran®). As such, there is speculation that it could decrease toxicities and increase the efficacy of ASCT. We compared the outcomes of patients conditioned with PG-free MEL (n = 216) to PG-solubilized MEL (n = 200) at our institution. The baseline characteristics were similar between the two groups. After Day +0, there were no differences in terms of hospitalizations, neutropenic fevers, intravenous granisetron requirement, World Health Organization grade ≥ 2 oral/esophageal mucositis, intravenous fluid requirement, or narcotic requirement. However, PG-free MEL patients had a higher incidence of diarrhea, which was mostly C. difficile-negative (82% vs. 71%, P = 0.015*). Day + 100 hematologic responses and progression-free survival after ASCT were comparable. In summary, we demonstrate that switching to PG-free MEL did not significantly reduce short-term complications of ASCT or improve outcomes in MM.

摘要

自体干细胞移植 (ASCT) 仍然是多发性骨髓瘤 (MM) 治疗的主要方法。虽然该程序通常是安全的,但与大剂量马法兰预处理相关的毒性是常见的,并且会严重影响患者的生活质量。最近,一种无丙二醇马法兰制剂 (PG-free MEL; Evomela®) 被美国食品和药物管理局批准作为 MM 的 ASCT 预处理方案。PG-free MEL 比丙二醇溶解的马法兰 (PG-solubilized MEL; Alkeran®) 更具溶解性和稳定性。因此,有人推测它可以降低毒性并提高 ASCT 的疗效。我们比较了我们机构中使用 PG-free MEL (n=216) 和 PG-solubilized MEL (n=200) 预处理的患者的结局。两组的基线特征相似。在 Day+0 后,住院、中性粒细胞减少性发热、静脉注射格拉司琼需求、世界卫生组织 (WHO) 分级≥2 级口腔/食管粘膜炎、静脉输液需求或阿片类药物需求方面没有差异。然而,PG-free MEL 患者腹泻的发生率更高,其中大多数为艰难梭菌阴性 (82% vs. 71%,P=0.015*)。ASCT 后第 100 天的血液学反应和无进展生存期相似。总之,我们证明转换为 PG-free MEL 并没有显著降低 ASCT 的短期并发症或改善 MM 的结果。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1e4e/6377862/84b63018c68f/nihms1500760f3.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1e4e/6377862/b5d6c53a3ff6/nihms1500760f1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1e4e/6377862/e39ca8b7c042/nihms1500760f2.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1e4e/6377862/84b63018c68f/nihms1500760f3.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1e4e/6377862/b5d6c53a3ff6/nihms1500760f1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1e4e/6377862/e39ca8b7c042/nihms1500760f2.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1e4e/6377862/84b63018c68f/nihms1500760f3.jpg

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The evolution of stem-cell transplantation in multiple myeloma.多发性骨髓瘤中干细胞移植的发展历程。
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