Objectives: To explore the risk factors for hemorrhagic cystitis (HC) in children with β-thalassemia major (TM) undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT).
Methods: A retrospective analysis was conducted on clinical data of 247 children with TM who underwent allo-HSCT at Shenzhen Children's Hospital from January 2021 to November 2022. The children were divided into an HC group (91 cases) and a non-HC group (156 cases) based on whether HC occurred after operation. Multivariable logistic regression analysis was used to explore the risk factors for HC, and the receiver operating characteristic curve was used to analyze the predictive efficacy of related factors for HC.
Results: Among the 247 TM patients who underwent allo-HSCT, the incidence of HC was 36.8% (91/247). Univariate analysis showed age, incompatible blood types between donors and recipients, occurrence of acute graft-versus-host disease (aGVHD), positive urine BK virus deoxyribonucleic acid (BKV-DNA), and ≥2 viral infections were associated with the development of HC after allo-HSCT (P<0.05). Multivariable analysis revealed that incompatible blood types between donors and recipients (OR=3.171, 95%CI: 1.538-6.539), occurrence of aGVHD (OR=2.581, 95%CI: 1.125-5.918), and positive urine BKV-DNA (OR=21.878, 95%CI: 9.633-49.687) were independent risk factors for HC in children with TM who underwent allo-HSCT. The receiver operating characteristic curve analysis showed that positive urine BKV-DNA alone or in combination with two other risk factors (occurrence of aGVHD, incompatible blood types between donors and recipients) had a certain accuracy in predicting the development of HC after allo-HSCT (area under the curve >0.8, P<0.05).
Conclusions: Incompatible blood types between donors and recipients, occurrence of aGVHD, and positive urine BKV-DNA are risk factors for HC after allo-HSCT in children with TM. Regular monitoring of urine BKV-DNA has a positive significance for early diagnosis and treatment of HC.
目的: 探讨重型β地中海贫血（β-thalassemia major, TM）患儿异基因造血干细胞移植（allogeneic hematopoietic stem cell transplantation, allo-HSCT）后并发出血性膀胱炎（hemorrhagic cystitis, HC）的危险因素。方法: 回顾性分析2021年1月—2022年11月在深圳市儿童医院进行allo-HSCT的247例TM患儿的临床资料，以术后是否并发HC，分为HC组（91例）和非HC组（156例），采用多因素logistic回归分析探讨HC发生的危险因素，并采用受试者操作特征曲线分析相关因素预测HC的效能。结果: 247例allo-HSCT TM患儿中，HC发生率为36.8%（91/247）。单因素分析显示，年龄、供受者血型不一致、发生急性移植物抗宿主病（acute graft-versus-host disease, aGVHD）、尿BK病毒核酸（BK virus deoxyribonucleic acid, BKV-DNA）阳性和≥2种病毒感染与患儿allo-HSCT后并发HC有关（P<0.05）。多因素分析显示，供受者血型不一致（OR=3.171，95%CI：1.538~6.539）、发生aGVHD（OR=2.581，95%CI：1.125~5.918）和尿BKV-DNA阳性（OR=21.878，95%CI：9.633~49.687）是allo-HSCT TM患儿并发HC的独立危险因素。受试者操作特征曲线分析显示，单一尿BKV-DNA阳性或联合其他2种危险因素（发生aGVHD、供受者血型不一致）预测allo-HSCT后并发HC具有一定的准确性（曲线下面积>0.8，P<0.05）。结论: 供受者血型不一致、发生aGVHD和尿BKV-DNA阳性是TM患儿allo-HSCT后并发HC的独立危险因素，定期监测尿BKV-DNA对HC的早期诊断及治疗具有积极意义。.
Keywords: Allogeneic hematopoietic stem cell transplantation; Child; Hemorrhagic cystitis; Risk factor; β-thalassemia major.