Acute and chronic steroid-refractory graft-versus-host disease (srGVHD) is a life-threatening complication of allogeneic stem cell transplantation. There are a number of reports on case series describing efficacy of ruxolitinib in both acute and chronic srGVHD. We conducted a prospective study (NCT02997280) in 75 patients with srGVHD (32 acute, 43 chronic, 41 adults, and 34 children). Patients with chronic GVHD had severe disease in 83% of cases, and acute GVHD patients had grade III–IV disease in 66% of cases. The overall response rate (ORR) was 75% (95% CI 57–89%) in acute GVHD and 81% (95% CI 67–92%) in chronic. Overall survival was 59% (95% CI 49–74%) in acute group and 85% (95% CI 70–93%). The major risk factors for lower survival were grade III–IV gastrointestinal involvement (29% vs 93%, p = 0.0001) in acute form and high disease risk score in chronic (65% vs 90%, p = 0.038). Toxicity was predominantly hematologic with 79% and 44% of grade III–IV neutropenia in acute and chronic groups, respectively. There was no difference between adults and children in terms of ORR (p = 0.31, p = 0.35), survival (p = 0.44, p = 0.12) and toxicity (p > 0.93). The study demonstrated that ruxolitinib is an effective option in acute and chronic srGVHD and can be used both in adults and children.

急性和慢性类固醇难治性移植物抗宿主病 (srGVHD) 是异基因干细胞移植的危及生命的并发症。有许多关于描述鲁索替尼对急性和慢性 srGVHD 疗效的病例系列报告。我们对 75 名 srGVHD 患者（32 名急性、43 名慢性、41 名成人和 34 名儿童）进行了一项前瞻性研究 (NCT02997280)。83% 的慢性 GVHD 患者患有严重疾病，66% 的急性 GVHD 患者患有 III-IV 级疾病。急性 GVHD 的总缓解率 (ORR) 为 75% (95% CI 57–89%)，慢性 GVHD 为 81% (95% CI 67–92%)。急性组的总生存率为 59%（95% CI 49-74%）和 85%（95% CI 70-93%）。较低生存率的主要危险因素是 III-IV 级胃肠道受累（29% vs 93%，p = 0.0001) 急性形式和慢性高疾病风险评分 (65% vs 90%, p  = 0.038)。毒性主要是血液学毒性，急性组和慢性组分别有 79% 和 44% 的 III-IV 级中性粒细胞减少。成人和儿童在 ORR ( p  = 0.31, p  = 0.35)、生存 ( p  = 0.44, p  = 0.12) 和毒性 ( p  > 0.93)方面没有差异。该研究表明，鲁索替尼是治疗急性和慢性 srGVHD 的有效选择，可用于成人和儿童。