Hemorrhagic cystitis in pediatric severe aplastic anemia undergoing haploidentical hematopoietic stem cell transplantation: incidence, risk factors and outcomes.

Abstract

Background: Hemorrhagic cystitis (HC) is a common complication of hematopoietic stem cell transplantation (HSCT) and may adversely affect the prognosis of patients. However, the risk factors associated with HC and its influence on prognosis remain unclear in pediatric Severe aplastic anemia (SAA) patients who underwent haploidentical HSCT (haplo-HSCT).

Methods: Clinical data from 116 SAA patients who received haplo-HSCT based on the 'Beijing Protocol' at the Children's Hospital of Soochow University between 2018 and 2023 were examined retrospectively. Potential risk factors were identified by univariate and multivariate logistic regression, and the effect of HC on overall survival (OS) was analyzed by Kaplan-Meier curves and log-rank tests.

Results: 32 out of 116 patients (27.6%) developed HC and the median time to onset of HC was 12 days (range: 1-157 days) after HSCT. In multivariate analysis, Very SAA (VSAA) (OR = 3.47, 95% CI: 1.15-10.44), II-IV acute graft versus host disease (aGVHD) (OR = 2.75, 95% CI: 1.05-7.18) and pre-transplant iron overload (OR = 3.90, 95% CI: 1.27-11.94) were regarded as risk factors. Compared to the non-HC group and mild HC group, the severe HC group had the worst 2-year OS rates (non-HC: 94.0% ± 2.6%; mild HC: 96.0% ± 3.9%; severe HC: 71.4% ± 1.7%, P = 0.047).

Conclusion: For pediatric SAA patients, VSAA, II-IV aGVHD, and pre-transplant iron overload elevate the risk of HC following haplo-HSCT. The development of severe HC can affect the clinical outcomes of patients.