鲁索利替尼时代原发性骨髓纤维化移植候选者的决策分析。

IF 8.2 1区 医学 Q1 HEMATOLOGY Haematologica Pub Date : 2024-11-01 DOI:10.3324/haematol.2024.285256
Yosuke Okada, Hideki Nakasone, Shunto Kawamura, Kosuke Takano, Kazuki Yoshimura, Masaharu Tamaki, Akari Matsuoka, Takuto Ishikawa, Tomohiro Meno, Yuhei Nakamura, Masakatsu Kawamura, Junko Takeshita, Nozomu Yoshino, Yukiko Misaki, Machiko Kusuda, Aki Tanihara, Shun-Ichi Kimura, Shinichi Kako, Yoshinobu Kanda
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引用次数: 0

摘要

近年来,鲁索利替尼(ruxolitinib)治疗的进展可能会改善原发性骨髓纤维化(PMF)患者的生活质量和总生存期。因此,在鲁索利替尼时代,异基因造血细胞移植(HCT)的最佳时机仍有待阐明。我们构建了一个马尔可夫模型来模拟PMF移植候选者的5年临床过程,并比较了鲁索利替尼失败后立即HCT和延迟HCT的结果。由于年龄越大死亡风险越高,我们在亚组分析中分别对年龄小于60岁和≥60岁的患者进行了分析。无论患者年龄如何,在鲁索利替尼治疗失败后进行延迟 HCT 治疗的患者预期寿命都更长。关于质量调整生命年(QALYs),一项基线分析显示,在鲁索利替尼治疗失败后,立即进行HCT治疗的效果不如延迟进行HCT治疗的效果(2.19比2.26)。在年龄小于60岁的患者中,鲁索利替尼治疗失败后立即进行HCT治疗与延迟进行HCT治疗效果相当(2.31比2.31)。另一方面,在年龄≥60岁的患者中,鲁索利替尼治疗失败后立即进行造血干细胞移植不如延迟进行造血干细胞移植(1.98对2.21)。单向敏感性分析表明,即刻HCT后无慢性移植物抗宿主疾病而存活的效用是对QALYs影响最大的参数,该值高于0.836可逆转鲁索利替尼失败后延迟HCT的优越性。因此,鲁索利替尼失败后的延迟HCT有望优于即刻HCT,尤其是对于年龄≥60岁的患者,即使对于年龄小于60岁的患者,延迟HCT也是一种很有前景的策略。
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Decision analysis for transplant candidates with primary myelofibrosis in the ruxolitinib era.

The recent progress with ruxolitinib treatment might improve quality of life as well as overall survival in patients with primary myelofibrosis. Therefore, the optimal timing of allogeneic hematopoietic cell transplantation (HCT) in the ruxolitinib era remains to be elucidated. We constructed a Markov model to simulate the 5-year clinical course of transplant candidates with primary myelofibrosis and compared outcomes between those who underwent immediate HCT and those whose HCT was delayed until after ruxolitinib failure. Since older age was associated with an increased risk of mortality, we analyzed patients aged <60 and ≥60 years separately in subgroup analyses. Life expectancy was consistently longer in the groups undergoing delayed HCT after ruxolitinib failure regardless of the patients' age. Regarding quality-adjusted life years, a baseline analysis showed that immediate HCT was inferior to delayed HCT after ruxolitinib failure (2.19 vs. 2.26). In patients aged <60 years, immediate HCT was equivalent to delayed HCT after ruxolitinib failure (2.31 vs. 2.31). On the other hand, in patients aged ≥60 years, immediate HCT was inferior to delayed HCT after ruxolitinib failure (1.98 vs. 2.21). A one-way sensitivity analysis showed that the utility of being alive without chronic graft-versus-host disease after immediate HCT was the most influential parameter for quality-adjusted life years, and that a value higher than 0.836 could reverse the superiority of delayed HCT after ruxolitinib failure. As a result, delayed HCT after ruxolitinib failure is expected to be superior to immediate HCT, especially in patients aged ≥60 years, and is also a promising strategy even in those aged <60 years.

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来源期刊
Haematologica
Haematologica 医学-血液学
CiteScore
14.10
自引率
2.00%
发文量
349
审稿时长
3-6 weeks
期刊介绍: Haematologica is a journal that publishes articles within the broad field of hematology. It reports on novel findings in basic, clinical, and translational research. Scope: The scope of the journal includes reporting novel research results that: Have a significant impact on understanding normal hematology or the development of hematological diseases. Are likely to bring important changes to the diagnosis or treatment of hematological diseases.
期刊最新文献
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