Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA.
Bristol Myers Squibb, Princeton, NJ 08540, USA.
Future Oncol. 2023 Apr;19(11):763-773. doi: 10.2217/fon-2022-1298. Epub 2023 May 10.
To define ruxolitinib failure and develop parameters to guide transition to next-line therapy for patients with myelofibrosis. A modified Delphi panel with 14 hematologists-oncologists. Survey concepts included defining primary refractory status, loss of response, disease progression, intolerance and transition to next-line therapy. Ruxolitinib failure may be defined as no improvement in symptoms or spleen size, progressive disease or ruxolitinib intolerance, following a maximally tolerated dose for ≥3 months. Loss of spleen response 1 month after initial response may prompt discontinuation. Lack of evidence to inform transition to next-line therapy was noted; tapering ruxolitinib should be considered according to ruxolitinib dose and patient characteristics. Expert consensus was provided on defining ruxolitinib failure and transition to next-line therapy as summarized in this position paper, which may support considerations in the development of future clinical practice guidelines.
定义芦可替尼治疗失败,并制定指导骨髓纤维化患者进入下一线治疗的参数。采用改良 Delphi 法对 14 名血液肿瘤学家进行了问卷调查。调查概念包括定义原发性难治状态、无反应、疾病进展、不耐受以及向二线治疗的转变。芦可替尼治疗失败可能定义为在最大耐受剂量下≥3 个月后症状或脾脏大小无改善、疾病进展或芦可替尼不耐受。在初始反应后 1 个月时脾脏反应丧失可能提示停药。专家共识指出,缺乏向二线治疗过渡的证据;应根据芦可替尼剂量和患者特征考虑逐渐减少芦可替尼的剂量。本立场文件总结了芦可替尼治疗失败和向二线治疗过渡的定义,为专家共识提供了依据,这可能有助于制定未来临床实践指南的考虑。