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生物制剂在高嗜酸性粒细胞综合征中使用的类固醇节省效益及各亚型的重大疾病负担

Steroid-sparing benefits of biologic use in hypereosinophilic syndrome and substantial disease burden across subtypes.

作者信息

Hwee Jeremiah, Huynh Lynn, da Costa Wilson, Rothenberg Marc E, Duh Mei Sheng, Alfonso-Cristancho Rafael

机构信息

Epidemiology, GSK, Mississauga, ON, Canada.

Analysis Group, Inc., Boston, MA, United States.

出版信息

Front Allergy. 2025 May 23;6:1605397. doi: 10.3389/falgy.2025.1605397. eCollection 2025.

DOI:10.3389/falgy.2025.1605397
PMID:40486501
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC12143263/
Abstract

BACKGROUND

Limited data exist on the burden of myeloproliferative, lymphocytic and idiopathic subtypes of hypereosinophilic syndrome (M-HES, L-HES and I-HES) and the characteristics of patients with HES receiving biologic therapies. This analysis aimed to further characterize these subtypes and explore the impact of biologics in a real-world European setting.

METHODS

This was a subgroup analysis of a retrospective, non-interventional, chart review (GSK ID: 214657) across five European countries. Index date was first clinical visit during January 2015-December 2019 (after or at time of HES diagnosis). Patients with HES aged ≥6 years with ≥1-year follow-up from index were included. Demographics, disease characteristics, diagnostic assessments, comorbidities, types of treatment, clinical manifestations, clinical outcomes and HES-related healthcare resource utilization were summarized for HES overall and subtypes. Oral corticosteroid (OCS) use and clinical manifestations/outcomes were assessed 12-months pre- and post-biologics.

RESULTS

The analysis included 280 patients with I-HES ( = 155), M-HES ( = 66), L-HES ( = 42) and chronic eosinophilic leukemia ( = 2). The most common clinical manifestations were fatigue (54.2% I-HES, 52.4% L-HES, 42.4% M-HES), skin itch (36.4% M-HES, 35.7% L-HES, 33.5% I-HES) and pain (31.0% L-HES, 30.3% M-HES, 27.1% I-HES). Biologic use was highest with L-HES (64.3%), followed by I-HES (43.9%) and M-HES (34.8%). Clinical response rates were highest for the I-HES subtype (75.5%; 66.7% L-HES, 63.6% M-HES). Hospitalizations were highest for L-HES (45.2%; 30.3% M-HES, 25.8% I-HES). The annualized rate of OCS prescriptions reduced by 56.8% (0.44-0.19 per person-year) and the proportion of patients with ≥1 clinical response increased 3.6-fold (6.5%-23.4%) between the pre- and post-biologics periods.

CONCLUSIONS

All HES subtypes had a substantial disease burden and were commonly associated with fatigue, skin itch and pain. I-HES appeared to be more responsive to treatment than L-HES and M-HES. Biologic use for HES led to more patients experiencing clinical responses and was OCS-sparing.

摘要

背景

关于嗜酸性粒细胞增多综合征的骨髓增殖性、淋巴细胞性和特发性亚型(M-HES、L-HES和I-HES)的负担以及接受生物疗法的嗜酸性粒细胞增多综合征患者的特征,现有数据有限。本分析旨在进一步描述这些亚型,并探讨生物制剂在欧洲实际环境中的影响。

方法

这是一项对五个欧洲国家进行的回顾性、非干预性病历审查(GSK ID:214657)的亚组分析。索引日期为2015年1月至2019年12月期间的首次临床就诊(嗜酸性粒细胞增多综合征诊断之后或之时)。纳入年龄≥6岁、自索引日期起有≥1年随访的嗜酸性粒细胞增多综合征患者。总结了嗜酸性粒细胞增多综合征总体及其亚型的人口统计学、疾病特征、诊断评估、合并症、治疗类型、临床表现、临床结局以及与嗜酸性粒细胞增多综合征相关的医疗资源利用情况。在生物制剂治疗前和治疗后12个月评估口服糖皮质激素(OCS)的使用情况以及临床表现/结局。

结果

分析纳入了280例I-HES患者(n = 155)、M-HES患者(n = 66)、L-HES患者(n = 42)和慢性嗜酸性粒细胞白血病患者(n = 2)。最常见的临床表现为疲劳(I-HES患者中占54.2%,L-HES患者中占52.4%,M-HES患者中占42.4%)、皮肤瘙痒(M-HES患者中占36.4%,L-HES患者中占35.7%,I-HES患者中占33.5%)和疼痛(L-HES患者中占31.0%,M-HES患者中占30.3%,I-HES患者中占27.1%)。L-HES患者生物制剂的使用率最高(64.3%),其次是I-HES患者(43.9%)和M-HES患者(34.8%)。I-HES亚型的临床缓解率最高(75.5%;L-HES患者为66.7%,M-HES患者为63.6%)。L-HES患者的住院率最高(45.2%;M-HES患者为30.3%,I-HES患者为25.8%)。在生物制剂治疗前和治疗后期间,OCS处方的年化率降低了56.8%(从每人每年0.44降至0.19),有≥1次临床缓解的患者比例增加了3.6倍(从6.5%增至23.4%)。

结论

所有嗜酸性粒细胞增多综合征亚型都有相当大 的疾病负担,并且通常与疲劳、皮肤瘙痒和疼痛相关。I-HES似乎比L-HES和M-HES对治疗更敏感。嗜酸性粒细胞增多综合征使用生物制剂使更多患者出现临床缓解,并且减少了OCS的使用。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/218b/12143263/2e360adbc7b6/falgy-06-1605397-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/218b/12143263/d5b45fe49fcb/falgy-06-1605397-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/218b/12143263/2e360adbc7b6/falgy-06-1605397-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/218b/12143263/d5b45fe49fcb/falgy-06-1605397-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/218b/12143263/2e360adbc7b6/falgy-06-1605397-g002.jpg

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