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美国严重甲型血友病患者免疫耐受诱导的种族和民族差异。

Race and ethnicity and the success of immune tolerance induction among people with severe haemophilia A in the United States.

机构信息

Department of Hematology and Medical Oncology, Emory University School of Medicine, Atlanta, Georgia, USA.

Hemophilia of Georgia Center for Bleeding & Clotting Disorders of Emory, Emory University School of Medicine, Atlanta, Georgia, USA.

出版信息

Haemophilia. 2024 May;30(3):628-637. doi: 10.1111/hae.14980. Epub 2024 Mar 10.

Abstract

INTRODUCTION

Immune tolerance induction (ITI) is the only treatment to eradicate inhibitors in people with severe haemophilia A with inhibitors. Since the risk of inhibitor development is greater among Black and Hispanic persons, it has been hypothesized that race and ethnicity may influence ITI success. Limited studies have evaluated this hypothesis.

AIM

To examine the success of ITI according to race and ethnicity.

METHODS

Participants who entered the Community Counts (CC) Registry between 2013 and 2017, were aged ≥3 years at study entry, and received ITI were included (n = 559). The proportion of participants with successful ITI was examined with adjusted prevalence ratios (aPRs) and corresponding 95% confidence intervals (95% CIs).

RESULTS

Among 559 participants, 56.9%, 19.1%, 18.1% and 4.3% were Non-Hispanic (NH) White, NH Black, Hispanic and Asian, respectively, and 1.7% were coded as other or missing. Approximately 80% of Hispanic, NH Black and NH White participants had good/very good prognosis, defined as having a pre-ITI peak inhibitor of < 200 Bethesda Units per millilitre. Nearly 60% of participants (59.7%) achieved successful ITI, 20.7% and 19.5% experienced partially successful or failed ITI, respectively. Successful ITI was non-significantly lower in NH Black (54.2%; aPR = 0.95, 95% CI 0.62-1.44) and Hispanic (55.4%; aPR = 0.89, 95% CI 0.71-1.13) relative to NH White participants (62.6%).

CONCLUSION

In this study, 60% of participants in the CC Registry had successful ITI, consistent with previous studies. The proportion with successful ITI was generally comparable across racial and ethnic groups with similar prognosis. These findings do not support the hypothesis that ITI response varies according to race or ethnicity.

摘要

简介

免疫耐受诱导(ITI)是治疗具有抑制剂的重度血友病 A 患者抑制剂的唯一方法。由于黑人及西班牙裔人群发生抑制剂的风险更高,因此有人假设种族和民族可能会影响 ITI 的成功。有限的研究评估了这一假设。

目的

根据种族和民族研究 ITI 的成功。

方法

2013 年至 2017 年期间入组社区计数(CC)登记处的参与者,在研究入组时年龄≥3 岁,接受 ITI 治疗,被纳入研究(n=559)。使用调整后的患病率比(aPR)及其相应的 95%置信区间(95%CI)检查具有成功 ITI 的参与者比例。

结果

在 559 名参与者中,分别有 56.9%、19.1%、18.1%和 4.3%为非西班牙裔(NH)白人、NH 黑人、西班牙裔和亚洲人,1.7%被编码为其他或缺失。大约 80%的西班牙裔、NH 黑人和 NH 白人参与者预后良好/非常好,定义为 ITI 前峰值抑制剂<200 贝塞斯达单位/毫升。近 60%的参与者(59.7%)实现了成功的 ITI,分别有 20.7%和 19.5%经历了部分成功或失败的 ITI。与 NH 白人参与者(62.6%)相比,NH 黑人(54.2%;aPR=0.95,95%CI 0.62-1.44)和西班牙裔(55.4%;aPR=0.89,95%CI 0.71-1.13)的成功 ITI 比例显著较低。

结论

在这项研究中,CC 登记处 60%的参与者 ITI 成功,与以往研究一致。具有成功 ITI 的比例在具有相似预后的不同种族和民族群体中基本相当。这些发现不支持 ITI 反应根据种族或民族而不同的假设。

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