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中重度急性移植物抗宿主病患者的治疗模式及临床结局:一项多中心病历回顾研究

Treatment Patterns and Clinical Outcomes of Patients with Moderate to Severe Acute Graft-Versus-Host Disease: A Multicenter Chart Review Study.

作者信息

Michonneau David, Devillier Raynier, Keränen Mikko, Rubio Marie Thérèse, Nicklasson Malin, Labussière-Wallet Hélène, Carre Martin, Huynh Anne, Viayna Elisabet, Roset Montserrat, Finzi Jonathan, Pfeiffer Minja, Thunström Daniel, Lara Núria, Sabatelli Lorenzo, Chevallier Patrice, Itälä-Remes Maija

机构信息

Hôpital Saint-Louis, Université de Paris, 1 Av. Claude Vellefaux, 75010 Paris, France.

Institute Paoli-Calmettes, 232 Boulevard de Sainte-Marguerite, 13009 Marseille, France.

出版信息

Hematol Rep. 2024 May 6;16(2):283-294. doi: 10.3390/hematolrep16020028.

Abstract

Acute graft-versus-host disease (aGVHD) remains a barrier to successful allogeneic hematopoietic stem cell transplantation (HSCT) outcomes. Contemporary comprehensive analyses of real-world clinical outcomes among patients who develop aGVHD post-HSCT are needed to better understand the unmet needs of this patient population. This multicenter, retrospective chart review describes treatment patterns and clinical outcomes among patients (≥18 years old) from Finland, Sweden, and France who developed grades II-IV aGVHD after their first HSCT (January 2016-June 2017). From 13 participating centers, 151 patients were included. The median (Q1, Q3) age at HSCT was 56 (45, 62) years old. One line of aGVHD treatment was received by 47.7%, and the most common first-line treatment was methylprednisolone (alone or in a combination regimen, 74.2%; monotherapy, 25.8%). Among patients treated with methylprednisolone, 79.5% achieved a complete or partial response. The median (Q1, Q3) number of treatment lines was 2.0 (1.0, 3.0). The median (Q1, Q3) time to obtain an aGVHD diagnosis from transplant was 29.5 (21.0, 44.0) days, and 14.5 (7.0, 34.0) days to achieve the best response for 110 evaluable patients. At 6 and 12 months, 53.6% and 49.0%, respectively, achieved a complete response. Chronic GVHD occurred in 37.7% of patients, and aGVHD reoccurred in 26.5%. Following aGVHD diagnosis, mortality rates were 30.0% at 6 months and 37.3% at 12 months. Findings from this study demonstrate a continuing unmet need for new therapies that control aGVHD and improve mortality.

摘要

急性移植物抗宿主病(aGVHD)仍然是异基因造血干细胞移植(HSCT)取得成功结果的一个障碍。需要对HSCT后发生aGVHD的患者的真实世界临床结果进行当代综合分析,以更好地了解该患者群体未满足的需求。这项多中心回顾性图表审查描述了来自芬兰、瑞典和法国的患者(≥18岁)在首次HSCT(2016年1月至2017年6月)后发生II-IV级aGVHD的治疗模式和临床结果。来自13个参与中心,共纳入151例患者。HSCT时的中位(Q1,Q3)年龄为56(45,62)岁。47.7%的患者接受了一线aGVHD治疗,最常见的一线治疗是甲泼尼龙(单独或联合方案,74.2%;单药治疗,25.8%)。在接受甲泼尼龙治疗的患者中,79.5%实现了完全或部分缓解。治疗线数的中位(Q1,Q3)数为2.0(1.0,3.0)。从移植到获得aGVHD诊断的中位(Q1,Q3)时间为29.5(21.0,44.0)天,110例可评估患者达到最佳缓解的时间为14.5(7.0,34.0)天。在6个月和12个月时,分别有53.6%和49.0%的患者实现了完全缓解。37.7%的患者发生了慢性GVHD,26.5%的患者aGVHD复发。在aGVHD诊断后,6个月时的死亡率为30.0%,12个月时为37.3%。这项研究的结果表明,对于控制aGVHD和提高死亡率的新疗法,仍存在未满足的需求。

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