Pediatric Hematology Oncology and Stem Cell Transplant, Department of Pediatrics, Indiana University School of Medicine, Indianapolis, Indiana, USA.
Riley Hospital for Children at Indiana University Health, Indianapolis, Indiana, USA.
Pediatr Blood Cancer. 2025 Jan;72(1):e31396. doi: 10.1002/pbc.31396. Epub 2024 Nov 1.
Veno-occlusive disease (VOD) is a life-threatening endotheliopathy that can occur after stem cell transplant (SCT). Numerous risk factors contribute to the development of VOD during SCT, and the role of prophylactic defibrotide (DF) in mitigating these risks remains unclear.
We compare not only the incidence of VOD development, but also the severity of VOD and survival outcomes between patients who did and did not develop VOD and did or did not receive prophylactic DF.
In this single-center retrospective study of 58 pediatric SCT patients from 2008 to 2022, we compare the demographics, risk profiles, and outcomes within three cohorts: Group 1: prophylactic DF and no VOD (n = 5), Group 2: prophylactic DF and development of VOD (n = 6), and Group 3: treatment DF for patients who developed VOD (n = 47).
Patients with VOD who did not receive prophylactic DF had higher severity classification of disease at onset (very severe 80.9% vs. 66.7%, p = .592) and at maximum severity (very severe 89.4% vs. 83.3%, p = .532), as opposed to mild, moderate, or severe categorization compared to those who did not receive prophylactic DF. Patients who developed VOD and did not receive prophylactic DF had a lower 1-year survival probability compared to those who received prophylactic DF and still developed VOD (51.1% vs. 75% alive at 1 year, excluding the two subjects without adequate follow-up time, p = .266).
Although, not statistically significant in our small retrospective study, there is potential overall survival and decreased VOD severity benefits of prophylactic DF.
静脉闭塞性疾病（VOD）是一种危及生命的血管内皮病，可发生在干细胞移植（SCT）后。许多危险因素导致 SCT 期间发生 VOD，而预防性地使用去纤维蛋白（DF）来减轻这些风险的作用尚不清楚。
我们不仅比较了 VOD 发展的发生率，还比较了 VOD 发展和未发展、是否接受预防性 DF 的患者的 VOD 严重程度和生存结果。
在这项对 2008 年至 2022 年间的 58 名儿科 SCT 患者进行的单中心回顾性研究中，我们比较了三个队列的人口统计学、风险概况和结果：第 1 组：预防性 DF 且无 VOD（n=5）、第 2 组：预防性 DF 且发生 VOD（n=6）、和第 3 组：VOD 发生后使用治疗性 DF 的患者（n=47）。
未接受预防性 DF 的 VOD 患者在发病时（非常严重 80.9% vs. 66.7%，p=0.592）和达到最大严重程度时（非常严重 89.4% vs. 83.3%，p=0.532）的疾病严重程度分类更高，而与未接受预防性 DF 的患者相比，他们的严重程度更轻、中度或重度。与那些未接受预防性 DF 且仍发生 VOD 的患者相比，发生 VOD 且未接受预防性 DF 的患者 1 年生存率较低（51.1% vs. 1 年时 75%存活，不包括两个随访时间不足的患者，p=0.266）。
尽管在我们的小型回顾性研究中没有统计学意义，但预防性 DF 可能具有总体生存和降低 VOD 严重程度的益处。
