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巴林出国进行骨髓移植的儿科患者的特征与结局:一项十年回顾性队列研究。

Characteristics and outcomes of Bahraini pediatric patients sent abroad for bone marrow transplantation: A ten-year retrospective cohort study.

作者信息

Isa Hasan M, Bucheeri Shahd T, Aldoseri Jarrah Y, Redha Ayah A, Mubarak Abdulla F, Altamimi Shaikha A, AlOraibi Ameera A, Alshaikh Mohamed I

机构信息

Department of Pediatrics, College of Medicine and Medical Sciences, Arabian Gulf University, Manama 26671, Bahrain.

Department of Pediatrics, Salmaniya Medical Complex, Manama 26671, Bahrain.

出版信息

World J Transplant. 2025 Jun 18;15(2):100065. doi: 10.5500/wjt.v15.i2.100065.

Abstract

BACKGROUND

Bone marrow transplantation (BMT) is a breakthrough procedure for patients with hematological and oncological conditions, particularly when all other treatments fail. Its indications vary between patients and settings, and its outcomes depend on the donor type, transplantation facility, and center expertise. In countries where transplantation facilities are lacking, sending patients abroad for transplantation might be a safe and effective alternative to leaving the patient to face eventual disease morbidity or even mortality if the procedure is not performed locally. However, studies evaluating BMT abroad are scarce.

AIM

To assess the clinical characteristics of patients who underwent BMT overseas and analyze the factors affecting their survival outcomes.

METHODS

We conducted a retrospective cohort study of all Bahraini pediatric patients who underwent BMT between 2013 and 2024. Medical records from Salmaniya Medical Complex and Overseas Treatment Office were reviewed. Patient demographics, transplant indications, donor type, transplantation type, overseas centers, complications, and outcomes (overall and 5-year survival rates) were analyzed. Clinical characteristics and outcomes were compared using test, Student's -test, Mann-Whitney test, and Kaplan-Meier method. Univariate and multivariate analyses were used to estimate survival predictors.

RESULTS

Of the 75 listed patients, 62 (82.7%) underwent BMT and were included, 10 (13.3%) did not, and 3 (4.0%) were awaiting transplantation. Most patients were male ( = 33, 53.2%). The mean age at transplantation was 7.8 ± 4.9 years. The main indication for treatment was acute myeloid leukemia (AML) ( = 15, 36.6%). Six patients (9.7%) required re-transplantation. Of the 68 transplants, 60 (88.2%) involved conditioning, mostly a combination of fludarabine and total body irradiation ( = 7, 11.7%). Most patients underwent allogeneic transplantation ( = 48, 77.4%), primarily from related donors ( = 47/48, 97.9%). The most common complication was infection ( = 51, 79.7%). Follow-up averaged 3.3 ± 2.5 years. The overall survival rate was 77.4%. Survival odds were better for non-AML patients and Middle Eastern centers ( = 0.015 and = 0.032, respectively).

CONCLUSION

Bahraini males with AML primarily underwent allogeneic BMT. Non-AML patients and those transplanted in the Middle East had better survival rates, despite high complication rates.

摘要

背景

骨髓移植(BMT)对于患有血液系统疾病和肿瘤疾病的患者而言是一种突破性的治疗方法,尤其是在所有其他治疗均告失败的情况下。其适应症因患者和治疗环境而异,其治疗结果取决于供体类型、移植机构以及中心的专业水平。在缺乏移植机构的国家,如果不在当地进行该手术,让患者出国进行移植可能是一种安全有效的替代方案,可避免患者最终面临疾病的发病甚至死亡。然而,评估海外骨髓移植的研究很少。

目的

评估在海外接受骨髓移植的患者的临床特征,并分析影响其生存结果的因素。

方法

我们对2013年至2024年间所有接受骨髓移植的巴林儿科患者进行了一项回顾性队列研究。查阅了萨勒曼尼亚医疗中心和海外治疗办公室的病历。分析了患者的人口统计学特征、移植适应症、供体类型、移植类型、海外中心、并发症以及结果(总生存率和5年生存率)。使用卡方检验、学生t检验、曼-惠特尼U检验和卡普兰-迈耶方法比较临床特征和结果。采用单因素和多因素分析来评估生存预测因素。

结果

在列出的75例患者中,62例(82.7%)接受了骨髓移植并被纳入研究,10例(13.3%)未接受,3例(4.0%)正在等待移植。大多数患者为男性(n = 33,53.2%)。移植时的平均年龄为7.8±4.9岁。主要治疗适应症为急性髓系白血病(AML)(n = 15,36.6%)。6例患者(9.7%)需要再次移植。在68例移植中,60例(88.2%)涉及预处理,主要是氟达拉滨和全身照射的联合使用(n = 7,11.7%)。大多数患者接受了异基因移植(n = 48,77.4%),主要来自相关供体(47/48,97.9%)。最常见的并发症是感染(n = 51,79.7%)。随访平均为3.3±2.5年。总生存率为77.4%。非AML患者和中东中心的生存几率更高(分别为P = 0.015和P = 0.032)。

结论

患有AML的巴林男性主要接受了异基因骨髓移植。尽管并发症发生率很高,但非AML患者以及在中东接受移植的患者生存率更高。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/8ac0/11886298/d24d29a5886c/100065-g001.jpg

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