Department of Experimental and Clinical Medicine, University of Florence, Florence, Italy.
CRIMM, Center for Research and Innovation of Myeloproliferative Neoplasms, Azienda Ospedaliero-Universitaria Careggi, Florence, Italy.
Cancer Med. 2023 Apr;12(7):8166-8171. doi: 10.1002/cam4.5618. Epub 2023 Jan 27.
Incorporating real-world data in the drug development process allows the improvement of health outcomes by providing better representation of actual patterns of drug safety and efficacy.
Here, we present the results of a retroprospective, observational real-life study of 154 patients with myelofibrosis treated with ruxolitinib in a real-life setting in seven Italian centers of the MYNERVA project.
Median drug exposure was 29 (range, 3-98) months. Discontinuation rate was 27% after a median time of 13 (range, 3-61). While hematological toxicities were in line with previous findings, infections occurred frequently, representing a not negligible cause of discontinuation and death. Anemia, symptoms, and spleen responses were obtained at any time in 23%, 91%, and 68% of patients, respectively; most patients achieved their responses by week 24. Larger splenomegaly and delayed treatment initiation correlated with lower spleen response at 24 weeks. Spleen response was associated with a superior overall survival, regardless of DIPSS. Of interest, both achievement and loss of spleen response had prognostic implications.
Overall, our findings provide insights on the efficacy and safety of ruxolitinib in a real-world, multicenter cohort of Italian MF patients.
在药物开发过程中纳入真实世界数据可以通过更好地体现药物安全性和疗效的实际模式来改善健康结果。
在这里，我们展示了一项回顾性、观察性真实世界研究的结果，该研究纳入了 154 例在意大利七个 MYNERVA 项目中心接受鲁索替尼治疗的骨髓纤维化患者。
中位药物暴露时间为 29 个月（范围为 3-98 个月）。中位停药时间为 13 个月（范围为 3-61 个月），停药率为 27%。虽然血液学毒性与先前的发现一致，但感染频繁发生，是导致停药和死亡的一个不可忽视的原因。贫血、症状和脾脏反应在 23%、91%和 68%的患者中分别在任何时间获得；大多数患者在第 24 周达到反应。较大的脾脏肿大和延迟治疗开始与 24 周时较低的脾脏反应相关。脾脏反应与总生存有关，与 DIPSS 无关。有趣的是，脾脏反应的获得和丧失都具有预后意义。
总体而言，我们的发现提供了关于鲁索替尼在意大利骨髓纤维化患者真实世界、多中心队列中的疗效和安全性的见解。
