Gupta Vikas, Tomuleasa Ciprian, Barranco Lampón Gilberto Israel, Hou Hsin-An, Helbig Grzegorz, Vachhani Pankit, Symeonidis Argiris, Haznedaroglu Ibrahim, Galvez Kenny, Tatsch Fernando, Chopra Avijeet S, Zhang Meng, Vizkelety Tamas, Murray Bryan, Ross David M
Clinical Cancer Research Unit, Princess Margaret Cancer Centre, Toronto, ON, Canada.
Medfuture Research Center for Advanced Medicine, Iuliu Hatieganu University of Medicine and Pharmacy, Cluj-Napoca, Romania.
Blood Adv. 2025 Mar 11;9(5):1105-1116. doi: 10.1182/bloodadvances.2024014625.
Myelofibrosis (MF), a myeloproliferative neoplasm, was most commonly treated with hydroxyurea (HU) before approval of ruxolitinib (RUX), now the standard of care. Factors that influence real-world MF treatment patterns are not well understood. The METER study was a multi-country, retrospective chart review of MF treatment patterns, treatment effectiveness, and health care resource utilization. Of 997 eligible patients, 65.9% had primary MF, and 11.7% were transfusion dependent. Median time from diagnosis to the start of initial treatment (index date) was 29 days (interquartile range [IQR], 1-140). RUX was the most common first-line (1L) therapy (49.0%), followed by HU (40.2%); 48.5% of patients remained on 1L therapy through week 156. Seventy-seven patients underwent allogeneic stem cell transplantation; transplantation was uncommon at 1L, increasing from 2.2% at week 24 to 11.0% at week 156 in patients ≤70 years of age. Median overall survival was 79.1 months (95% confidence interval [95% CI], 70.8 to not estimable [NE]) in all patients, 142.3 months (95% CI, 74.1 to NE) for non-RUX patients, 77.6 months (95% CI, 64.2-85.9) for patients on RUX 1L therapy, and 72.6 months (95% CI, 62.0 to NE) for RUX 2L+ patients. Of patients who experienced ≥1 corresponding event, the median hospital length of stay (LoS; n = 520), intensive care unit LoS (n = 71), and number of transfusions (n = 375) were 16 days (IQR, 7-37), 5 days (IQR, 2-13), and 12 (IQR, 4-26), respectively. Despite improvements, there were numerous hospitalization and transfusion events among these patients in routine practice. This trial was registered at www.ClinicalTrials.gov as #NCT05444972.
骨髓纤维化(MF)是一种骨髓增殖性肿瘤,在芦可替尼(RUX)获批之前,羟基脲(HU)是其最常用的治疗药物,而现在RUX是标准治疗药物。影响实际临床中MF治疗模式的因素尚未完全明确。METER研究是一项多国回顾性图表审查研究,旨在分析MF的治疗模式、治疗效果及医疗资源利用情况。在997例符合条件的患者中,65.9%为原发性MF,11.7%依赖输血治疗。从诊断到开始初始治疗(索引日期)的中位时间为29天(四分位间距[IQR],1 - 140天)。RUX是最常见的一线(1L)治疗药物(49.0%),其次是HU(40.2%);48.5%的患者在第156周时仍接受1L治疗。77例患者接受了异基因干细胞移植;1L治疗时移植并不常见,≤70岁患者中,从第24周的2.2%增至第156周的11.0%。所有患者的中位总生存期为79.1个月(95%置信区间[95%CI],70.8至不可估计[NE]),未接受RUX治疗的患者为142.3个月(95%CI,74.1至NE),接受RUX 1L治疗的患者为77.6个月(95%CI,64.2 - 85.9),接受RUX 2L +治疗的患者为72.6个月(95%CI,62.0至NE)。在经历≥1次相应事件的患者中,中位住院时间(LoS;n = 520)、重症监护病房住院时间(n = 71)和输血次数(n = 375)分别为16天(IQR,7 - 37)、5天(IQR,2 - 13)和12次(IQR,4 - 26)。尽管有所改善,但在常规临床实践中,这些患者仍有大量住院和输血事件。该试验已在www.ClinicalTrials.gov上注册,注册号为#NCT05444972。