摘要目的 探讨芦可替尼(RUX)联合PTD方案(泼尼松+沙利度胺+达那唑)治疗骨髓纤维化(MF)的疗效和安全性.方法 对符合2016年WHO的MF诊断标准的MF患者,采用芦可替尼(RUX)联合PTD方案治疗,并评价该方案的临床疗效、血细胞计数变化和不良反应.结果 7例MF患者纳入研究,其中原发性骨髓纤维化(PMF)6例,原发性血小板增多症后骨髓纤维化(post-ET MF)1例;JAK2V617F突变4例,CARL突变1例,MPLW515突变1例,以上3种始动基因突变均阴性1例.7例患者治疗前均有肋缘下可触及的脾肿大.7例患者中5例获得临床改善,2例疾病稳定.7例患者均有不同程度的临床症状改善,6例患者有不同程度的脾脏缩小.3例患者接受芦可替尼单药治疗,均出现不同程度的血红蛋白浓度和血小板计数下降,改用芦可替尼联合PTD方案后血红蛋白浓度和血小板计数均明显上升.所有7例接受芦可替尼联合PTD方案治疗患者的血红蛋白浓度中位上升值为30(18～54)g/L,血小板计数中位上升值为116(13～ 369)×109/L,其中3例患者因血小板计数明显上升而调高芦可替尼剂量.全部7例患者均未发生血液学不良反应,未发生Ⅲ/Ⅳ级非血液学不良反应,Ⅰ/Ⅱ级非血液学不良反应包括便秘、腹胀、下肢水肿、丙氨酸转氨酶升高和齿龈感染各1例.结论 芦可替尼联合泼尼松、沙利度胺和达那唑治疗MF患者,可减少芦可替尼的血液学不良反应,使血红蛋白和血小板计数上升、提高临床疗效,且安全性好.

abstractsObjective To evaluate the efficacy and tolerability of ruxolitinib combined with prednisone,thalidomide and danazol for treatment of in myelofibrosis (MF).Methods Patients of MF according to the WHO 2016 criteria,received ruxolitinib (RUX) combined with prednisone,thalidomide and danazol (PTD).The response,changes of blood counts and adverse events were evaluated.Results Six PMF and one post-ET MF patients were enrolled.Four patients presented JAK2V617F mutation,one CALR mutation,one MPL mutation,one triple-negative.Responses per IWG-MRT criteria were clinical improvement in 5 patients,stable disease in 2 ones,spleen response in 6 ones.All of 7 patients were symptomatic responses,four patients achieved at least 50％ improvement from baseline on MPN-SAF TSS.Three patients initially treated with RUX alone,all of 3 patients experienced treatment-associated anemia and thrombocytopenia.Then these 3 patients received RUX combined with PTD,both hemoglobin and platelet increased significantly.Four patients initially treated with RUX combined with PTD.Increased levels of hemoglobin and platelet were seen in all of 7 patients received RUX combined with PTD with maximum increased hemoglobin of 30(18-54) g/L and maximum increased platelets of 116(13-369)× 109/L,respectively from baseline.The treatment dose of RUX increased due to improved platelet count in 3 patients.The frequent non-hematologic adverse events grade 1-2 were constipation,abdominal distension,crura edema and increased ALT.Conclusions RUX combined with PTD for treatment of MF may modulate initial hematologic toxicity observed when RUX alone,and may increase response due to improved levels of hemoglobin or platelet.