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小儿急性淋巴细胞白血病患者造血干细胞移植的获得情况:一项基于人群的分析。

Access to Hematopoietic Stem Cell Transplantation among Pediatric Patients with Acute Lymphoblastic Leukemia: A Population-Based Analysis.

机构信息

Division of Pediatric Oncology, Blood and Marrow Transplant, Alberta Children's Hospital, Calgary, Alberta, Canada.

Pediatric Oncology Group of Ontario, Toronto, Ontario and Dalla Lana School of Public Health, University of Toronto, Toronto, Ontario, Canada.

出版信息

Biol Blood Marrow Transplant. 2019 Jun;25(6):1172-1178. doi: 10.1016/j.bbmt.2019.02.009. Epub 2019 Feb 12.

DOI:10.1016/j.bbmt.2019.02.009
PMID:30769192
Abstract

Access to hematopoietic stem cell transplantation (HSCT) in pediatric acute lymphoblastic leukemia (ALL) primarily depends on disease-related factors but may be influenced by social and economic determinants. We included all children aged < 15 years with newly diagnosed ALL in Canada between 2001 and 2018 using the Cancer in Young People in Canada national registry. We examined factors potentially associated with the likelihood of receiving HSCT using univariate and multivariable logistic regression models. A total of 3992 patients with newly diagnosed ALL were included. Three hundred twenty-five (8.1%) received an HSCT and formed the transplant cohort. In multivariable analysis factors independently associated with an increased odds of receiving HSCT were male sex (odds ratio [OR], 1.42; 95% confidence interval [CI], 1.05 to 1.93), initial WBC ≥ 50,000 × 10/L (OR, 1.58; 95% CI, 1.09 to 2.28), mixed phenotype acute leukemia relative to B-precursor ALL (OR, 34.32; 95% CI, 16.64 to 70.79), T cell relative to B-precursor ALL (OR, 1.77; 95% CI, 1.07 to 2.91), unfavorable relative to standard cytogenetics (OR, 3.96; 95% CI, 2.56 to 6.12), and relapse before HSCT (OR, 32.77; 95%, 23.89 to 44.96). No association was found between race, neighborhood income quintile or region at diagnosis, and receipt of HSCT. Diagnosis at an HSCT treating center (OR, 1.51; 95% CI, 1.09 to 2.09) and residential distance from the ALL treating center (OR, 1.84 for ≥300 km compared with <100 km; 95% CI, 1.17 to 2.91) were associated with higher odds of receiving HSCT. In a publically funded healthcare system, children with ALL had equitable access to HSCT, which was largely governed by biologic disease-related factors. Patients diagnosed at an HSCT performing center and patients who live farthest away from their treatment center had higher odds of receiving HSCT, although the effect was small, possibly suggesting preferential referral to HSCT for some patients.

摘要

在儿科急性淋巴细胞白血病(ALL)中,造血干细胞移植(HSCT)的获得主要取决于疾病相关因素,但可能受到社会经济决定因素的影响。我们使用加拿大癌症青年全国登记处,纳入了 2001 年至 2018 年间加拿大所有新诊断为 ALL 的年龄<15 岁的儿童。我们使用单变量和多变量逻辑回归模型检查了与接受 HSCT 可能性相关的潜在因素。共纳入 3992 例新诊断为 ALL 的患者。其中 325 例(8.1%)接受了 HSCT,形成了移植组。多变量分析显示,男性(比值比[OR],1.42;95%置信区间[CI],1.05 至 1.93)、初始白细胞计数≥50,000×10/L(OR,1.58;95%CI,1.09 至 2.28)、混合表型急性白血病相对于 B 前体 ALL(OR,34.32;95%CI,16.64 至 70.79)、T 细胞相对于 B 前体 ALL(OR,1.77;95%CI,1.07 至 2.91)、不良核型相对于标准核型(OR,3.96;95%CI,2.56 至 6.12)和 HSCT 前复发(OR,32.77;95%CI,23.89 至 44.96)与接受 HSCT 的可能性增加相关。种族、居住地收入五分位数或诊断时所在地区与 HSCT 的接受情况之间没有关联。在接受 HSCT 的治疗中心诊断(OR,1.51;95%CI,1.09 至 2.09)和居住地与 ALL 治疗中心的距离(OR,≥300km 与<100km 相比为 1.84;95%CI,1.17 至 2.91)与接受 HSCT 的可能性增加相关。在一个公共资助的医疗保健系统中,ALL 患儿获得 HSCT 的机会均等,这主要由与疾病相关的生物学因素决定。在进行 HSCT 的中心诊断的患者和离治疗中心最远的患者接受 HSCT 的可能性更高,尽管效果很小,但可能表明某些患者更倾向于接受 HSCT 治疗。

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