Ravindranath Archita, Selvarajan Sushil, Lionel Sharon, Devasia Anup Joseph, Korula Anu, N Aboobacker Fouzia, M Lakshmi Kavitha, Balasubramanian Poonkuzhali, George Biju, Mathews Vikram, Abraham Aby, Kulkarni Uday
Department of Haematology, Christian Medical College Vellore, Tamil Nadu, India.
Blood Cell Ther. 2025 Jun 27;8(3):217-224. doi: 10.31547/bct-2024-033. eCollection 2025 Aug 25.
Acute graft-versus-host disease (aGVHD) is a life-threatening complication that can develop after allogeneic hematopoietic stem cell transplantation. Patients with steroid-refractory aGVHD (SR-aGVHD) have an extremely poor prognosis. Ruxolitinib is an approved treatment for SR-aGVHD. However, there is a paucity of real-world data on the clinical outcomes of patients with SR-aGVHD treated with ruxolitinib. We conducted a retrospective analysis using hospital records of the clinical outcomes of patients who underwent stem cell transplantation at our center between January 2021 and December 2022 and developed steroid-refractory aGVHD which was treated with ruxolitinib. During the study period, 381 patients underwent allogeneic stem cell transplantation at our center. Amongst these, 160 (42.0%) developed aGVHD. Of these, 59 (36.8%) had SR-aGVHD and 40 were treated with ruxolitinib. Ruxolitinib therapy was administered after a median of 6 days (range 3-29) from onset of aGVHD. Amongst the 28 patients who survived at day 28 (12 died before the day 28 response could be assessed), a total of 16 patients (57.1%) attained a response (complete response (n=12) or partial response (n=4)). Infectious complications were the most common adverse event (n=39; 97.5%), followed by severe cytopenia (grades 3 to 4) in 25 (62.5%) patients. The median follow-up of the cohort was 5 months (range 1 to 29 months). At the last follow up, 30 (75%) patients died; 3 patients died of progression of steroid-refractory aGVHD, 14 died of progression of aGVHD with infection, 10 died of underlying infection and 3 had disease relapse. From our real-world analysis, we conclude that though the outcomes in patients with SR-aGVHD responding to ruxolitinib are encouraging, there is still a large unmet need for novel strategies for improving outcomes and reducing infection-related mortality, even while there is access to ruxolitinib.
急性移植物抗宿主病（aGVHD）是异基因造血干细胞移植后可能发生的一种危及生命的并发症。患有类固醇难治性aGVHD（SR-aGVHD）的患者预后极差。芦可替尼是一种获批用于治疗SR-aGVHD的药物。然而，关于接受芦可替尼治疗的SR-aGVHD患者临床结局的真实世界数据较少。我们使用了本中心2021年1月至2022年12月期间接受干细胞移植且发生了接受芦可替尼治疗的类固醇难治性aGVHD患者的医院记录进行了一项回顾性分析。在研究期间，381例患者在本中心接受了异基因干细胞移植。其中，160例（42.0%）发生了aGVHD。在这些患者中，59例（36.8%）患有SR-aGVHD，40例接受了芦可替尼治疗。芦可替尼治疗在aGVHD发病后中位6天（范围3 - 29天）开始。在第28天存活的28例患者中（12例在第28天反应可评估前死亡），共有16例患者（57.1%）获得反应（完全缓解（n = 12）或部分缓解（n = 4））。感染并发症是最常见的不良事件（n = 39；97.5%），其次是25例（62.5%）患者出现严重血细胞减少（3至4级）。该队列的中位随访时间为5个月（范围1至29个月）。在最后一次随访时，30例（75%）患者死亡；3例死于类固醇难治性aGVHD进展，14例死于aGVHD伴感染进展，10例死于基础感染，3例疾病复发。从我们的真实世界分析中，我们得出结论，尽管对芦可替尼有反应的SR-aGVHD患者的结局令人鼓舞，但即使有芦可替尼可用，对于改善结局和降低感染相关死亡率的新策略仍有很大未满足的需求。
