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鲁索利替尼时代骨髓纤维化患者的移植:来自法语骨髓移植和细胞治疗学会的注册研究。

Transplantation for myelofibrosis patients in the ruxolitinib era: a registry study from the Société Francophone de Greffe de Moelle et de Thérapie Cellulaire.

机构信息

Service d'hématologie - greffe, Hôpital Saint Louis, APHP, Université de Paris, Paris, France.

Hematology Department, Clínica Universidad de Navarra, Pamplona, Spain.

出版信息

Bone Marrow Transplant. 2024 Jul;59(7):965-973. doi: 10.1038/s41409-024-02268-5. Epub 2024 Mar 21.

DOI:10.1038/s41409-024-02268-5
PMID:38514813
Abstract

In this SFGM-TC registry study, we report the results after stem cell transplantation (HSCT) in 305 myelofibrosis patients, in order to determine potential risk factors associated with outcomes, especially regarding previous treatment with ruxolitinib. A total of 102 patients were transplanted from an HLA-matched-sibling donor (MSD), and 143 patients received ruxolitinib. In contrast with previous studies, our results showed significantly worse outcomes for ruxolitinib patients regarding overall survival (OS) and non-relapse mortality (NRM), especially in the context of unrelated donors (URD). When exploring reasons for potential confounders regarding the ruxolitinib effect, an interaction between the type of donor and the use of ATG was found, therefore subsequent analyses were performed separately for each type of donor. Multivariable analyses did not confirm a significant negative impact of ruxolitinib in transplantation outcomes. In the setting of URD, only age and Fludarabine-Melphalan (FM) conditioning were associated with increased NRM. For MSD, only Karnoksfy <70% was associated with reduced OS. However, a propensity score analysis showed that ruxolitinib had a negative impact on OS but only in non-responding patients, consistent with previous data. To conclude, with all the precautions due to confounders and bias, ruxolitinib itself does not appear to increase mortality after HSCT.

摘要

在这项 SFGM-TC 注册研究中,我们报告了 305 例骨髓纤维化患者接受干细胞移植(HSCT)后的结果,以确定与结局相关的潜在风险因素,特别是与先前接受鲁索利替尼治疗有关的因素。共有 102 名患者从 HLA 匹配的同胞供体(MSD)接受移植,143 名患者接受了鲁索利替尼治疗。与之前的研究相比,我们的结果显示,接受鲁索利替尼治疗的患者在总体生存(OS)和非复发死亡率(NRM)方面的结局明显较差,尤其是在无关供体(URD)的情况下。当探索鲁索利替尼效应的潜在混杂因素的原因时,发现供体类型和使用 ATG 之间存在相互作用,因此随后分别对每种供体类型进行了分析。多变量分析并未证实鲁索利替尼对移植结局有显著的负面影响。在 URD 情况下,只有年龄和氟达拉滨-马法兰(FM)预处理与 NRM 增加相关。对于 MSD,只有 Karnoksfy <70%与 OS 降低相关。然而,倾向评分分析表明,鲁索利替尼对 OS 有负面影响,但仅在无反应的患者中,与之前的数据一致。总之,考虑到混杂因素和偏倚的所有预防措施,鲁索利替尼本身似乎不会增加 HSCT 后的死亡率。

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Myelofibrosis.骨髓纤维化。
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Allogeneic blood or marrow transplantation with haploidentical donor and post-transplantation cyclophosphamide in patients with myelofibrosis: a multicenter study.异基因血液或骨髓移植联合亲缘单倍体供者和移植后环磷酰胺治疗骨髓纤维化:一项多中心研究。
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