Palandri Francesca, Tiribelli Mario, Benevolo Giulia, Tieghi Alessia, Cavazzini Francesco, Breccia Massimo, Bergamaschi Micaela, Sgherza Nicola, Polverelli Nicola, Crugnola Monica, Isidori Alessandro, Binotto Gianni, Heidel Florian H, Buccisano Francesco, Martino Bruno, Latagliata Roberto, Spinsanti Marco, Kallenberg Lydia, Palumbo Giuseppe Alberto, Abruzzese Elisabetta, Scaffidi Luigi, Cuneo Antonio, Cavo Michele, Vianelli Nicola, Bonifacio Massimiliano
Department of Experimental, Diagnostic and Specialty Medicine, Institute of Hematology 'L. and A. Seràgnoli', University of Bologna, S. Orsola-Malpighi Hospital, Bologna, Italy.
Division of Hematology and Stem Cell Transplantation, Azienda Sanitaria Universitaria Integrata, Udine, Italy.
Hematol Oncol. 2018 Feb;36(1):285-290. doi: 10.1002/hon.2429. Epub 2017 May 17.
Patients with myelofibrosis at intermediate-1 risk according to the International Prognostic Score System are projected to a relatively long survival; nonetheless, they may carry significant splenomegaly and/or systemic constitutional symptoms that hamper quality of life and require treatment. Since registrative COMFORT studies included only patients at intermediate-2/high International Prognostic Score System risk, safety and efficacy data in intermediate-1 patients are limited. We report on 70 intermediate-1 patients treated with ruxolitinib according to standard clinical practice that were evaluated for response using the 2013 IWG-MRT criteria. At 6 months, rates of spleen and symptoms response were 54.7% and 80% in 64 and 65 evaluable patients, respectively. At 3 months, ruxolitinib-induced grade 3 anemia and thrombocytopenia occurred in 40.6% and 2.9% of evaluable patients, respectively. Notably, 11 (15.9%) patients experienced at least one infectious event ≥grade 2. Most (82.6%) patients were still on therapy after a median follow-up of 27 months. These data support the need for standardized guidelines that may guide the decision to initiate ruxolitinib therapy in this risk category, balancing benefit expectations and potential adverse effects.
根据国际预后评分系统，处于中危-1风险的骨髓纤维化患者预计生存期相对较长；尽管如此，他们可能伴有明显的脾肿大和/或全身症状，这会影响生活质量并需要进行治疗。由于注册的COMFORT研究仅纳入了国际预后评分系统中危-2/高危风险的患者，因此中危-1患者的安全性和有效性数据有限。我们报告了70例按照标准临床实践接受鲁索替尼治疗的中危-1患者，这些患者根据2013年国际工作组-骨髓增殖性肿瘤（IWG-MRT）标准进行疗效评估。在6个月时，64例可评估患者的脾脏反应率为54.7%，65例可评估患者的症状反应率为80%。在3个月时，可评估患者中分别有40.6%和2.9%发生了鲁索替尼诱导的3级贫血和血小板减少。值得注意的是，11例（15.9%）患者发生了至少1次≥2级感染事件。中位随访27个月后，大多数（82.6%）患者仍在接受治疗。这些数据支持需要制定标准化指南，以指导在这一风险类别中启动鲁索替尼治疗的决策，平衡获益预期和潜在不良反应。
