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既往接受鲁索替尼治疗失败后的骨髓纤维化患者应用 Fedratinib 的真实世界应用:患者特征、治疗模式和临床结局。

Real-World Use of Fedratinib for Myelofibrosis Following Prior Ruxolitinib Failure: Patient Characteristics, Treatment Patterns, and Clinical Outcomes.

机构信息

The Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY.

Guy's and St Thomas' NHS Foundation Trust, London, UK.

出版信息

Clin Lymphoma Myeloma Leuk. 2024 Feb;24(2):122-132. doi: 10.1016/j.clml.2023.09.008. Epub 2023 Sep 29.

DOI:10.1016/j.clml.2023.09.008
PMID:37839939
Abstract

BACKGROUND

There is a lack of established clinical outcomes for patients with myelofibrosis (MF) receiving fedratinib following ruxolitinib failure. This study examined real-world patient characteristics, treatment patterns, and clinical outcomes of patients with MF treated with fedratinib following ruxolitinib failure in US clinical practice.

PATIENTS AND METHODS

This retrospective patient chart review included adults with a physician-reported diagnosis of MF, who initiated fedratinib after discontinuing ruxolitinib. Descriptive analyses characterized patient characteristics, clinical outcomes, and treatment patterns from MF diagnosis through ruxolitinib and fedratinib treatment.

RESULTS

Twenty-four physicians abstracted data for 150 eligible patients. Approximately 55.3% of the patients were male, 68.0% were White, and median age at MF diagnosis was 68 (range, 35-84) years. Median duration of ruxolitinib therapy was 7.6 (range, 0.7-65.5) months. At initiation of fedratinib, 88.0% of patients had palpable spleen and a mean spleen size of 16.0 (standard deviation [SD], 5.9) cm. Spleen size decreased by 19.4% to 13.2 (SD, 7.9) cm at month 3 (P = .0001) and by 53.4% to 7.2 (SD, 7.4) cm at month 6 (P = .01) of fedratinib treatment, respectively. Almost one-third (26.8%) of patients had achieved ≥ 50% spleen reduction by month 6. Mean number of symptoms also decreased significantly at month 3 (P < .0001) and month 6 (P = .01).

CONCLUSION

Fedratinib appears to deliver spleen and symptom benefits in real-world patients with MF previously treated with ruxolitinib.

摘要

背景

接受芦可替尼治疗失败后的患者使用非格司亭时,缺乏既定的临床结局。本研究在美国临床实践中检查了接受芦可替尼治疗失败后的骨髓纤维化(MF)患者使用非格司亭的真实世界患者特征、治疗模式和临床结局。

方法

这项回顾性患者病历审查包括经医生诊断患有 MF、停止使用芦可替尼后开始使用非格司亭的成年人。描述性分析从 MF 诊断到芦可替尼和非格司亭治疗,描述了患者特征、临床结局和治疗模式。

结果

24 名医生为 150 名符合条件的患者提取了数据。大约 55.3%的患者为男性,68.0%为白人,MF 诊断时的中位年龄为 68(范围,35-84)岁。芦可替尼治疗的中位持续时间为 7.6(范围,0.7-65.5)个月。开始使用非格司亭时,88.0%的患者脾脏可触及,平均脾脏大小为 16.0(标准差[SD],5.9)cm。脾脏大小在第 3 个月时缩小了 19.4%,至 13.2(SD,7.9)cm(P =.0001),在第 6 个月时缩小了 53.4%,至 7.2(SD,7.4)cm(P =.01)。大约三分之一(26.8%)的患者在第 6 个月时实现了≥50%的脾脏缩小。第 3 个月(P <.0001)和第 6 个月(P =.01)时,平均症状数也显著减少。

结论

非格司亭似乎为接受过芦可替尼治疗的骨髓纤维化患者提供了脾脏和症状方面的获益。

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