Hematology, Department of Precision and Translational Medicine, Policlinico Umberto 1, Sapienza University, 00161 Rome, Italy.
Istituto di Ematologia "Seràgnoli", IRCCS Azienda Ospedaliero-Universitaria di Bologna, 40138 Bologna, Italy.
Curr Oncol. 2022 Jul 15;29(7):4970-4980. doi: 10.3390/curroncol29070395.
The development and approval of ruxolitinib, the first JAK1/2 inhibitor indicated to treat myelofibrosis, has improved patient outcomes, with higher spleen and symptoms responses, improved quality of life, and overall survival. Despite this, several unmet needs remain, including the absence of resistance criteria, suboptimal response, the timing of allogeneic transplant, and the management of patients in case of intolerance. Here, we report the results of the second survey led by the "MPN Lab" collaboration, which aimed to report physicians' perspectives on these topics. As in our first survey, physicians were selected throughout Italy, and we included those with extensive experience in treating myeloproliferative neoplasms and those with less experience representing clinical practice in the real world. The results presented here, summarized using descriptive analyses, highlight the need for a clear definition of response to ruxolitinib as well as recommendations to guide the management of ruxolitinib under specific conditions including anemia, thrombocytopenia, infections, and non-melanoma skin cancers.
芦可替尼的开发和批准，作为首个用于治疗骨髓纤维化的 JAK1/2 抑制剂，改善了患者的预后，提高了脾脏和症状反应，改善了生活质量，并延长了总生存期。尽管如此，仍存在一些未满足的需求，包括缺乏耐药标准、反应不理想、异基因移植的时机以及不耐受患者的管理。在这里，我们报告了由“MPN Lab”合作组织领导的第二次调查的结果，该调查旨在报告医生对这些问题的看法。与我们的第一次调查一样，我们在意大利各地选择了医生，并纳入了在治疗骨髓增生性肿瘤方面经验丰富的医生和在现实世界中具有丰富临床实践经验的医生。这里呈现的结果，使用描述性分析进行总结，强调了需要明确芦可替尼的反应定义，并建议在特定情况下指导芦可替尼的管理，包括贫血、血小板减少、感染和非黑色素瘤皮肤癌。
