Ellis Martin H, Koren-Michowitz Maya, Lavi Noa, Vannucchi Alessandro M, Mesa Ruben, Harrison Claire N
Hematology Institute, Meir Medical Center, Kfar Saba, Israel; Sackler School of Medicine, Tel Aviv University, Tel Aviv, Israel.
Department of Hematology, Asaf HaRofeh Medical Center, Tzrifin, Israel; Sackler School of Medicine, Tel Aviv University, Tel Aviv, Israel.
Leuk Res. 2017 Oct;61:6-9. doi: 10.1016/j.leukres.2017.08.002. Epub 2017 Aug 10.
Ruxolitinib is established as treatment for symptomatic myeloproliferative neoplasm (MPN)-associated myelofibrosis. The strict inclusion and exclusion criteria and dose modification rules that applied to the COMFORTI and II studies that led to the licensing of ruxolitinib are not always applicable to routine clinical practice. Thus physicians now face decisions regarding ruxolitinib use that were not addressed in these pivotal trials.
We performed an online survey of hematologists practicing in Europe, Israel, the United Kingdom and the United States. Demographic details regarding the physicians and their practice as relates to MPNs were collected. Management decisions pertaining to the use of ruxolitinib were obtained regarding 10 clinical scenarios relating to anemia, thrombocytopenia, frailty, infection and lack or loss of response to ruxolitnib in MF patients.
140 physicians responded to the survey. There were marked differences regarding their decisions for ruxolitinib administration in MF patients with or developing anemia or thrombocytopenia. Similarly there was little consensus regarding management of patients refractory or losing a response to ruxolitinib. There were differences between "MPN-focused" and "non-MPN-focused" physicians in certain areas.
Physician practices regarding management of MF patients experiencing ruxolitinib-related toxicities or in whom response to the drug is lost was variable. This was true of "MPN-focused" and "non-MPN-focused" physicians in certain cases. Physician education and experience in using ruxolitinib may improve patient management.
芦可替尼已被确立为有症状的骨髓增殖性肿瘤（MPN）相关骨髓纤维化的治疗药物。导致芦可替尼获批的COMFORT I和II研究中所应用的严格纳入和排除标准以及剂量调整规则并不总是适用于常规临床实践。因此，医生现在面临着这些关键试验中未涉及的关于芦可替尼使用的决策。
我们对在欧洲、以色列、英国和美国执业的血液科医生进行了一项在线调查。收集了医生的人口统计学细节及其与MPN相关的临床实践情况。针对骨髓纤维化（MF）患者贫血、血小板减少、身体虚弱、感染以及对芦可替尼缺乏反应或反应丧失的10种临床场景，获取了与芦可替尼使用相关的管理决策。
140名医生回复了调查。他们对于在患有或发生贫血或血小板减少的MF患者中给予芦可替尼的决策存在显著差异。同样，对于对芦可替尼难治或失去反应的患者的管理也几乎没有共识。在某些方面，“专注于MPN”的医生和“非专注于MPN”的医生之间存在差异。
对于经历芦可替尼相关毒性或对该药物失去反应的MF患者的管理，医生的做法存在差异。在某些情况下，“专注于MPN”的医生和“非专注于MPN”的医生都是如此。医生在使用芦可替尼方面的教育和经验可能会改善患者管理。
