Rahim Mahvish Q, Nichols Cydney, Althouse Sandra, Rahrig April L
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 Transplant. 2025 Feb;29(1):e70003. doi: 10.1111/petr.70003.
Veno-occlusive disease (VOD) and transplant-associated thrombotic microangiopathy (TA-TMA) remain a diagnostic and therapeutic challenge for patients undergoing hematopoietic stem cell transplant (HSCT). Both VOD and TA-TMA share an underlying etiology of microvascular endothelial damage. Potential under-recognition of TA-TMA in the context of VOD leaves HSCT recipients vulnerable to additional endothelial damage, and risk of end-organ failure.
A cohort of 44 pediatric HSCT recipients diagnosed with VOD between 2010and 2019 were retrospectively evaluated for the development of TA-TMA within 1 week before and 2 months after VOD diagnosis. Patients were classified into three categories: sole diagnosis of VOD (VOD), concurrent clinical diagnosis of TA-TMA during the VOD course (VOD+TA-TMA), and patients with VOD who on retrospective review satisfied criteria for diagnosis of TA-TMA (VOD+rTA-TMA).
A total of 42 patients were evaluated and 50% of the patients were diagnosed clinically with TA-TMA (5) or where retrospectively identified to have TA-TMA (16). There was no difference in the severity of the course of VOD between the three groups based on need for intubation, dialysis, and pediatric intensive care unit (PICU) care. Patients in the VOD only group had the highest survival at 1 year (66.7%, n = 14) compared with patients in the VOD+TA-TMA group (60%, n = 3) and VOD+rTA-TMA group (62.5%, n = 10), p = 0.9582.
Better understanding of the association between these two endotheliopathies is essential to improve diagnosis, treatment, and prevention of potentially fatal adverse outcomes in transplant recipients.
对于接受造血干细胞移植（HSCT）的患者而言，静脉闭塞性疾病（VOD）和移植相关血栓性微血管病（TA-TMA）仍然是诊断和治疗方面的挑战。VOD和TA-TMA都存在微血管内皮损伤这一潜在病因。在VOD背景下，TA-TMA可能未得到充分认识，这使得HSCT受者更容易受到额外的内皮损伤以及终末器官衰竭的风险。
回顾性评估了2010年至2019年间被诊断为VOD的44名儿科HSCT受者，以确定在VOD诊断前1周和诊断后2个月内TA-TMA的发生情况。患者被分为三类：单纯VOD诊断（VOD）、VOD病程中同时临床诊断为TA-TMA（VOD+TA-TMA）以及回顾性审查符合TA-TMA诊断标准的VOD患者（VOD+rTA-TMA）。
共评估了42例患者，其中50%的患者临床诊断为TA-TMA（5例）或回顾性确定患有TA-TMA（16例）。根据插管、透析和儿科重症监护病房（PICU）护理需求，三组之间VOD病程的严重程度没有差异。仅VOD组患者1年生存率最高（66.7%，n = 14），而VOD+TA-TMA组（60%，n = 3）和VOD+rTA-TMA组（62.5%，n = 10），p = 0.9582。
更好地理解这两种内皮病之间的关联对于改善移植受者潜在致命不良结局的诊断、治疗和预防至关重要。
