Posterior Reversible Encephalopathy Syndrome in Pediatric Hematopoietic Stem Cell Transplantation with Beta Major Thalassemia: The Association between the PRES Occurrence and Class of Beta Major Thalassemia

Abstract

AbstractIntroductionAllogeneic hematopoietic stem cell transplantation (HSCT) is the only definitive curative option for β‐major thalassemia patients (β‐MT). Posterior reversible encephalopathy syndrome (PRES) is a pervasive neurological complication which typically occurs following HSCT. β‐MT patients are prone to a higher PRES incidence due to long‐term immunosuppression; thus, it is imperative that these patients are closely monitored for PRES after HSCT.Patients and methodsWe included 148 pediatric patients with β‐MT who underwent HSCT between March 2015 and August 2022 in Children's Medical Center. Patients in this study were divided into two groups. The association between PRES and class of β‐MT and other risk factors were assessed and the overall survival rate was determined.ResultsFourteen out of 112 patients (12%) with class I and II β‐MT developed PRES. However, PRES occurred in 11 out of 36 patients (30.5%) with β‐MT‐III. Our results indicated that there was a significant association between class III β‐MT and the occurrence of (P = .004). Additionally, acute graft‐versus‐host disease (aGVHD) occurred in 80% and 44.7% of patients in the PRES and non‐PRES groups, respectively (P = .001). The results of the Kaplan‐Meier analysis revealed that the 5‐year overall survival (OS) was 75.6% in the PRES group versus 95% in the non‐PRES group, which was statistically significant (P = .001).ConclusionBased on our results, pediatric β‐MT III patients are at a higher risk of developing PRES. Additionally, pediatric β‐MT patients with a history of aGVHD, regardless of disease class, are more likely to develop PRES. Considering these results, PRES has a higher chance of being the etiology of symptoms and should be considered more often in these patients.