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.

Introduction: Allogeneic 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 Methods: We 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.

Results: Fourteen 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).

Conclusion: Based 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.