BACKGROUND Graft-vs-host disease (GVHD) is a major cause of mortality after allogeneic hematopoietic stem cell transplantation.Some patients have steroid-refractory(SR) GVHD.AIM To evaluate the effect and safety of ru...BACKGROUND Graft-vs-host disease (GVHD) is a major cause of mortality after allogeneic hematopoietic stem cell transplantation.Some patients have steroid-refractory(SR) GVHD.AIM To evaluate the effect and safety of ruxolitinib add-on in the treatment of patients with SR acute (a) and chronic (c) GVHD.METHODS We retrospectively analyzed 38 patients administered ruxolitinib add-on to standard immunosuppressive therapy for SR-aGVHD or SR-cGVHD following allogeneic hematopoietic stem cell transplantation.Ruxolitinib was administered5-10 mg/d depending on disease severity,patient status,and the use of antifungal drugs.Overall response rate,time to best response,malignancy relapse rate,infection rate,and treatment-related adverse events were assessed.RESULTS The analysis included 10 patients with SR-aGVHD (gradeⅢ/Ⅳ,n=9) and 28patients with SR-cGVHD (moderate/severe,n=24).For the SR-aGVHD and SRcGVHD groups,respectively:Median number of previous GVHD therapies was 2(range:1-3) and 2 (1-4);median follow-up was 2.5 (1.5-4) and 5 (1.5-10) mo;median time to best response was 1 (0.5-2.5) and 3 (1-9.5) mo;and overall response rate was 100%(complete response:80%) and 82.1%(complete response:10.7%) with a response observed in all GVHD-affected organs.The malignancy relapse rates for the SR-aGVHD and SR-cGVHD groups were 10.0%and 10.7%,respectively.Reactivation rates for cytomegalovirus,Epstein-Barr virus,and varicella-zoster virus,respectively,were 30.0%,10.0%,and 0%for the SR-aGVHD group and 0%,14.3%,and 7.1%for the SR-cGVHD group.CONCLUSION Ruxolitinib add-on was effective and safe as salvage therapy for SR-GVHD.展开更多
该文报告1例青年男性患者,同种异基因造血干细胞移植后出现移植物抗宿主病(graft versus host disease,GVHD)多脏器受累,临床表现多样,急慢性病程重叠发生:急性GVHD表现以高胆红素血症伴或不伴转氨酶升高、血性水样便为主;慢性GVHD以广...该文报告1例青年男性患者,同种异基因造血干细胞移植后出现移植物抗宿主病(graft versus host disease,GVHD)多脏器受累,临床表现多样,急慢性病程重叠发生:急性GVHD表现以高胆红素血症伴或不伴转氨酶升高、血性水样便为主;慢性GVHD以广泛皮肤色素脱失、口腔黏膜溃疡、病甲等表现突出,还有膜性肾病、多浆膜腔积液及肺限制性通气功能不全等慢性GVHD表现。GVHD诊断主要依靠病史和临床表现,鉴别排查感染、药物及肿瘤等因素影响,易漏诊误诊,需多学科诊治。文献回顾提示造血干细胞移植后男性受者女性供者、外周血造血干细胞移植者较骨髓移植者慢性GVHD发生风险高,但移植物抗白血病效应存在。目前糖皮质激素联合或不联合钙调磷酸酶抑制剂是GVHD的一线治疗方案,总体患者预后不佳。展开更多
文摘BACKGROUND Graft-vs-host disease (GVHD) is a major cause of mortality after allogeneic hematopoietic stem cell transplantation.Some patients have steroid-refractory(SR) GVHD.AIM To evaluate the effect and safety of ruxolitinib add-on in the treatment of patients with SR acute (a) and chronic (c) GVHD.METHODS We retrospectively analyzed 38 patients administered ruxolitinib add-on to standard immunosuppressive therapy for SR-aGVHD or SR-cGVHD following allogeneic hematopoietic stem cell transplantation.Ruxolitinib was administered5-10 mg/d depending on disease severity,patient status,and the use of antifungal drugs.Overall response rate,time to best response,malignancy relapse rate,infection rate,and treatment-related adverse events were assessed.RESULTS The analysis included 10 patients with SR-aGVHD (gradeⅢ/Ⅳ,n=9) and 28patients with SR-cGVHD (moderate/severe,n=24).For the SR-aGVHD and SRcGVHD groups,respectively:Median number of previous GVHD therapies was 2(range:1-3) and 2 (1-4);median follow-up was 2.5 (1.5-4) and 5 (1.5-10) mo;median time to best response was 1 (0.5-2.5) and 3 (1-9.5) mo;and overall response rate was 100%(complete response:80%) and 82.1%(complete response:10.7%) with a response observed in all GVHD-affected organs.The malignancy relapse rates for the SR-aGVHD and SR-cGVHD groups were 10.0%and 10.7%,respectively.Reactivation rates for cytomegalovirus,Epstein-Barr virus,and varicella-zoster virus,respectively,were 30.0%,10.0%,and 0%for the SR-aGVHD group and 0%,14.3%,and 7.1%for the SR-cGVHD group.CONCLUSION Ruxolitinib add-on was effective and safe as salvage therapy for SR-GVHD.
文摘该文报告1例青年男性患者,同种异基因造血干细胞移植后出现移植物抗宿主病(graft versus host disease,GVHD)多脏器受累,临床表现多样,急慢性病程重叠发生:急性GVHD表现以高胆红素血症伴或不伴转氨酶升高、血性水样便为主;慢性GVHD以广泛皮肤色素脱失、口腔黏膜溃疡、病甲等表现突出,还有膜性肾病、多浆膜腔积液及肺限制性通气功能不全等慢性GVHD表现。GVHD诊断主要依靠病史和临床表现,鉴别排查感染、药物及肿瘤等因素影响,易漏诊误诊,需多学科诊治。文献回顾提示造血干细胞移植后男性受者女性供者、外周血造血干细胞移植者较骨髓移植者慢性GVHD发生风险高,但移植物抗白血病效应存在。目前糖皮质激素联合或不联合钙调磷酸酶抑制剂是GVHD的一线治疗方案,总体患者预后不佳。
