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血液系统恶性肿瘤儿童和青少年患者参与临床试验的差异

Disparities in Clinical Trial Participation in Children and Adolescent Patients With a Hematologic Malignancy.

作者信息

Monroe Caitlin, Muñiz Joshua P, Leong Traci, Lewis Rebecca Williamson, Castellino Sharon M

机构信息

Aflac Cancer and Blood Disorders Center of Children's Healthcare of Atlanta (CHOA), Atlanta, Georgia, USA.

Division of Pediatric Hematology/Oncology, Department of Pediatrics, Emory University, Atlanta, Georgia, USA.

出版信息

Pediatr Blood Cancer. 2025 Jun;72(6):e31641. doi: 10.1002/pbc.31641. Epub 2025 Mar 7.

Abstract

BACKGROUND

Low socioeconomic groups and racial/ethnic minorities continue to experience pediatric cancer outcome disparities, and remain underrepresented in clinical trials. It is vital to understand why underrepresentation exists and to address it in order to generalize trial findings to all groups. This study examined institutional disparities in clinical trial offerings and enrollment for children and adolescents with hematologic malignancies.

PROCEDURE

We conducted a single-institution retrospective analysis of clinical trial participation in patients less than 18 years old with newly diagnosed hematologic malignancies between 2011 and 2017. Patient demographics (e.g., parental primary language, race) were abstracted, and patient address at diagnosis was geocoded to characterize neighborhood socioeconomic status. Endpoints were frontline therapeutic clinical trial offering and enrollment. Multivariable logistic regression was constructed to examine predictors of trial enrollment.

RESULTS

Among 464 trial-eligible patients, 90.1% were offered clinical trial participation, of which 85% enrolled. There was no significant difference in enrollment by age, sex, parental primary language, neighborhood socioeconomic status, or rurality. However, non-Hispanic Black patients [OR: 0.4 (95% CI: 0.20-0.8), p = 0.01] and patients with lymphoma [OR: 0.15 (95% CI: 0.04-0.6), p = 0.01] were less likely to enroll on a clinical trial in our adjusted analysis.

CONCLUSIONS

Despite a high institutional clinical trial enrollment rate for eligible patients, we found racial and disease-type disparities. Further work is needed to more granularly determine reasons for not offering trial participation or for not enrolling. By better-defining barriers to clinical trial enrollment, targeted institution-level interventions can be created to improve trial enrollment and reduce outcome disparities.

摘要

背景

社会经济地位较低的群体以及种族/少数族裔在儿童癌症治疗结果方面仍存在差异,并且在临床试验中的代表性仍然不足。了解代表性不足的原因并加以解决,对于将试验结果推广至所有群体至关重要。本研究调查了血液系统恶性肿瘤儿童和青少年在临床试验提供和入组方面的机构差异。

程序

我们对2011年至2017年间新诊断为血液系统恶性肿瘤的18岁以下患者的临床试验参与情况进行了单机构回顾性分析。提取了患者的人口统计学特征(如父母的母语、种族),并对诊断时的患者地址进行地理编码,以描述社区的社会经济地位。研究终点为一线治疗性临床试验的提供和入组情况。构建多变量逻辑回归模型以研究试验入组的预测因素。

结果

在464名符合试验条件的患者中,90.1%的患者获得了参与临床试验的机会,其中85%的患者入组。在年龄、性别、父母母语、社区社会经济地位或是否为农村地区方面,入组情况没有显著差异。然而,在我们的校正分析中,非西班牙裔黑人患者[比值比:0.4(95%置信区间:0.20 - 0.8),p = 0.01]和淋巴瘤患者[比值比:0.15(95%置信区间:0.04 - 0.6),p = 0.01]入组临床试验的可能性较小。

结论

尽管符合条件的患者在机构层面的临床试验入组率较高,但我们发现了种族和疾病类型方面的差异。需要进一步开展工作,更细致地确定不提供试验参与机会或不入组的原因。通过更好地界定临床试验入组的障碍,可以制定有针对性的机构层面干预措施,以提高试验入组率并减少治疗结果差异。

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