Cleveland Clinic Taussig Cancer Institute, Cleveland, Ohio, USA.
Incyte Corporation, Wilmington, Delaware, USA,
Acta Haematol. 2022;145(4):448-453. doi: 10.1159/000520440. Epub 2022 Jan 10.
Ruxolitinib is an FDA-approved treatment of intermediate- and high-risk myelofibrosis. In the phase 3 COMFORT studies, ruxolitinib reduced spleen volume in patients with myelofibrosis, with a median time to response of 3 months. However, nearly 20% of patients discontinued by month 4 with few treatment options available following discontinuation of ruxolitinib treatment. In this study, 2 independent patient care data sources were queried (Cardinal Health Oncology Provider Extended Network [OPEN] and HealthCore Integrated Research Environment [HIRE®]), and a retrospective review of medical charts was conducted. Patients aged ≥18 years with a diagnosis of myelofibrosis (primary or secondary), use of ruxolitinib for myelofibrosis, and documented physician-directed ruxolitinib interruption were included. Among 26 included patients, pre-interruption median (interquartile range [IQR]) ruxolitinib treatment duration was 123 (57-391, OPEN) and 110 (37-148, HIRE) days. Half the patients interrupted treatment within 3 months, commonly for adverse events (42% and 71%, respectively). After restarting ruxolitinib, median (IQR) re-treatment duration was 196 (54-553) and 166 (108-262) days, respectively. Consistent with previous reports, symptoms and spleen size improved in (OPEN/HIRE) 45%/43% and 40%/33% of evaluable patients, respectively. Further studies investigating the management of dose modifications and interruptions are needed to optimize benefit from ruxolitinib therapy.
芦可替尼是一种获得美国食品药品监督管理局批准的中高危骨髓纤维化治疗药物。在 3 期 COMFORT 研究中，芦可替尼可降低骨髓纤维化患者的脾脏体积，其反应中位数时间为 3 个月。然而，近 20%的患者在第 4 个月停药，在停止芦可替尼治疗后，可用的治疗选择很少。在这项研究中，我们查询了 2 个独立的患者护理数据源（Cardinal Health Oncology Provider Extended Network [OPEN] 和 HealthCore Integrated Research Environment [HIRE®]），并对医疗记录进行了回顾性审查。纳入的患者年龄≥18 岁，诊断为骨髓纤维化（原发性或继发性），使用芦可替尼治疗骨髓纤维化，并且有医生指导的芦可替尼中断记录。在纳入的 26 名患者中，中断前的中位（四分位间距[IQR]）芦可替尼治疗持续时间为 123（57-391，OPEN）和 110（37-148，HIRE）天。半数患者在 3 个月内中断治疗，通常是由于不良反应（分别为 42%和 71%）。重新开始芦可替尼治疗后，中位（IQR）再次治疗持续时间分别为 196（54-553）和 166（108-262）天。与之前的报告一致，（OPEN/HIRE）分别有 45%/43%和 40%/33%的可评估患者的症状和脾脏大小得到改善。需要进一步研究剂量调整和中断的管理，以优化芦可替尼治疗的获益。
