Background

Chronic Graft versus Host Disease (GvHD) is a serious complication of allogeneic hematopoietic stem cell transplant that severely impacts quality of life and long-term survival. About 50-to-60 % of patients treated with steroids require a further line of therapy due to lack of sustained response. Ruxolitinib, a JAK1/2 inhibitor, has recently been approved for the treatment of acute GvHD.

Methods

We aimed to retrospectively evaluate ruxolitinib efficacy and safety in a cohort of patients diagnosed with moderate (25 %) or severe (75 %) steroid-refractory or steroid-dependent chronic GvHD. Response evaluation was performed at three and six months.

Results

Thirty-six patients received ruxolitinib after a median of three previous lines (range, r 1–11) for a median of 8.6 months (r 1–51.6). Cutaneous GvHD was the most frequent presentation. We observed an overall response of 59 % (CR 9%, PR 50 %) at three months and 62 % (CR 15 %, PR 46 %) at six months. Two patients had hematologic disease recurrence and were censored at relapse; no other permanent discontinuation due to adverse events were documented. Cutaneous, oral, genital and ocular GvHD significantly improved after treatment. 2-year overall survival and 2-year transplant related mortality were 74 % and 19 % respectively. Ruxolitinib was associated with a significant reduction of steroid dose.

Conclusion

Ruxolitinib was confirmed to be a safe and effective option as salvage treatment also for advanced stages of chronic GvHD. Longer follow up is needed to evaluate durability of response. Prospective analyses on larger cohorts are ongoing.

中文翻译：

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Ruxolitinib 治疗慢性类固醇难治性移植物抗宿主病：单中心经验

背景

慢性移植物抗宿主病 (GvHD) 是同种异体造血干细胞移植的严重并发症，严重影响生活质量和长期生存。由于缺乏持续反应，大约 50% 至 60% 的接受类固醇治疗的患者需要进一步的治疗。Ruxolitinib 是一种 JAK1/2 抑制剂，最近已被批准用于治疗急性 GvHD。

方法

我们旨在对一组诊断为中度 (25 %) 或重度 (75 %) 类固醇难治性或类固醇依赖性慢性 GvHD 的患者进行回顾性评估鲁索替尼的疗效和安全性。在三个月和六个月时进行了反应评估。

结果

36 名患者在接受 ruxolitinib 治疗后接受了中位 3 线（范围，r 1–11），中位时间为 8.6 个月（r 1–51.6）。皮肤 GvHD 是最常见的表现。我们观察到三个月时的总体缓解率为 59%（CR 9%，PR 50%），六个月时为 62%（CR 15%，PR 46%）。两名患者出现血液系统疾病复发，并在复发时进行审查；没有其他因不良事件而永久停药的记录。治疗后皮肤、口腔、生殖器和眼部 GvHD 显着改善。2 年总生存率和 2 年移植相关死亡率分别为 74% 和 19%。Ruxolitinib 与类固醇剂量的显着减少有关。

结论

Ruxolitinib 被证实是一种安全有效的选择，可作为晚期慢性 GvHD 的抢救治疗。需要更长时间的随访来评估反应的持久性。正在进行对更大队列的前瞻性分析。