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单倍体相合造血干细胞移植治疗小儿重型再生障碍性贫血并发出血性膀胱炎:发病率、危险因素及预后

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

作者信息

Cui Kai, Zhang Senlin, Liang Mingchu, He ChenChen, Chen Jie, Wei Yufeng, Hu Shaoyan, Li Jie

机构信息

Department of Hematology and Oncology, Children'S Hospital of Soochow University, No. 92, Zhongnan Street, Suzhou, 215000, China.

Department of Nephrology and Immunology, Children'S Hospital of Soochow University, No. 92, Zhongnan Street, Suzhou, 215000, China.

出版信息

BMC Pediatr. 2025 Mar 26;25(1):234. doi: 10.1186/s12887-025-05505-y.

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.

摘要

背景

出血性膀胱炎(HC)是造血干细胞移植(HSCT)的常见并发症,可能对患者预后产生不利影响。然而,在接受单倍体HSCT(haplo-HSCT)的儿童重型再生障碍性贫血(SAA)患者中,与HC相关的危险因素及其对预后的影响仍不清楚。

方法

回顾性分析2018年至2023年期间在苏州大学附属儿童医院接受基于“北京方案”的单倍体HSCT的116例SAA患者的临床资料。通过单因素和多因素逻辑回归确定潜在危险因素,并通过Kaplan-Meier曲线和对数秩检验分析HC对总生存(OS)的影响。

结果

116例患者中有32例(27.6%)发生HC,HC发病的中位时间为HSCT后12天(范围:1-157天)。在多因素分析中,极重型SAA(VSAA)(OR = 3.47,95% CI:1.15-10.44)、II-IV级急性移植物抗宿主病(aGVHD)(OR = 2.75,95% CI:1.05-7.18)和移植前铁过载(OR = 3.90,95% CI:1.27-11.94)被视为危险因素。与非HC组和轻度HC组相比,重度HC组的2年OS率最差(非HC:94.0%±2.6%;轻度HC:96.0%±3.9%;重度HC:71.4%±1.7%,P = 0.047)。

结论

对于儿童SAA患者,VSAA、II-IV级aGVHD和移植前铁过载会增加单倍体HSCT后发生HC的风险。重度HC的发生会影响患者的临床结局。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/dcf5/11938558/97fe27025aa8/12887_2025_5505_Fig1_HTML.jpg

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