IRCCS Azienda Ospedaliero-Universitaria Di Bologna, Istituto Di Ematologia "Seràgnoli", Bologna, Italy.
Dipartimento Di Medicina Specialistica, Diagnostica E Sperimentale, Università Di Bologna, Bologna, Italy.
Ann Hematol. 2024 Jun;103(6):1931-1940. doi: 10.1007/s00277-024-05704-0. Epub 2024 Mar 13.
Ruxolitinib is beneficial in patients with myelofibrosis (MF) and polycythemia vera (PV). Information on ruxolitinib adherence is scant. The Ruxolitinib Adherence in Myelofibrosis and Polycythemia Vera (RAMP) prospective multicenter study (NCT06078319) included 189 ruxolitinib-treated patients. Patients completed the Adherence to Refills and Medications Scale (ARMS) and Distress Thermometer and Problem List (DTPL) at the earliest convenience, after registration in the study, and at later timepoints. At week-0, low adherence (ARMS > 14) and high distress (DT ≥ 4) were declared by 49.7% and 40.2% of patients, respectively. The main reason for low adherence was difficult ruxolitinib supply (49%), intentional (4.3%) and unintentional (46.7%) non-take. In multivariable regression analysis, low adherence was associated to male sex (p = 0.001), high distress (p < 0.001), and treatment duration ≥ 1 year (p = 0.03). Over time, rates of low adherence and high distress remained stable, but unintentional non-take decreased from 47.9% to 26.0% at week-48. MF patients with stable high adherence/low distress were more likely to obtain/maintain the spleen response at week-24. Low adherence to ruxolitinib represents an unmet clinical need that require a multifaceted approach, based on reason behind it (patients characteristics and treatment duration). Its recognition may help distinguishing patients who are truly refractory and those in need of therapy optimization.
芦可替尼对骨髓纤维化(MF)和真性红细胞增多症(PV)患者有益。关于芦可替尼依从性的信息很少。Ruxolitinib Adherence in Myelofibrosis and Polycythemia Vera(RAMP)前瞻性多中心研究(NCT06078319)纳入了 189 名接受芦可替尼治疗的患者。患者在研究注册后最早方便的时间点以及之后的时间点完成了Adherence to Refills and Medications Scale(ARMS)和Distress Thermometer and Problem List(DTPL)。在第 0 周,49.7%和 40.2%的患者分别报告低依从性(ARMS>14)和高痛苦(DT≥4)。低依从性的主要原因是芦可替尼供应困难(49%)、故意(4.3%)和非故意(46.7%)不服药。多变量回归分析显示,低依从性与男性(p=0.001)、高痛苦(p<0.001)和治疗持续时间≥1 年(p=0.03)有关。随着时间的推移,低依从性和高痛苦的发生率保持稳定,但在第 48 周时,非故意不服药的比例从 47.9%降至 26.0%。在第 24 周时,具有稳定高依从性/低痛苦的 MF 患者更有可能获得/维持脾脏反应。芦可替尼低依从性代表一种未满足的临床需求,需要采取多方面的方法,根据其背后的原因(患者特征和治疗持续时间)。对其的认识可能有助于区分真正难治的患者和需要优化治疗的患者。