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应对小儿急性淋巴细胞白血病中欧文氏菌天冬酰胺酶短缺的综合策略。

A comprehensive strategy to address shortage of Erwinia asparaginase in pediatric acute lymphoblastic leukemia.

作者信息

Vagrecha Anshul, Tao Vincent, Corless Rosemarie, Colon Cassandra, Redner Arlene, Atlas Mark

机构信息

Department of Pediatrics, Division of Hematology/Oncology and Cellular Therapy, Cohen Children's Medical Center, New Hyde Park, NY, USA.

Department of Pediatrics, Zucker School of Medicine at Hofstra/Northwell, Hempstead, NY, USA.

出版信息

Expert Rev Clin Pharmacol. 2023 Jul-Dec;16(8):763-769. doi: 10.1080/17512433.2023.2223970. Epub 2023 Jun 15.

DOI:10.1080/17512433.2023.2223970
PMID:37294084
Abstract

BACKGROUND

Pegylated form of E. coli derived asparaginase (PEG) is a crucial component of pediatric ALL therapy. Patients who develop a hypersensitivity (HSR) reaction with PEG receive an alternative form - Erwinia asparaginase (EA). However, an international shortage in 2017 had made it challenging to treat these patients. We have developed a comprehensive strategy to address this need.

PATIENTS AND METHODS

This is a single center, retrospective analysis. All patients receiving PEG were premedicated to reduce infusion reactions. Patients who developed HSR underwent PEG desensitization. Patients were compared to historic controls.

RESULTS

Fifty-six patients were treated within the study period. There was no difference in the frequency of reactions before and after the adoption of universal premedication ( = 0.78). Eight patients (14.2%) developed either ≥ Grade 2 HSR or silent inactivation and 5 patients (62.5%) successfully underwent desensitization. The remaining three patients received EA asparaginase. This intervention led to a decrease in PEG substitution, with 3 patients (5.3%) requiring EA compared to 8 patients (15.09%) in the pre-intervention period. ( = 0.11) PEG desensitization was more cost effective than EA administration.

CONCLUSION

PEG desensitization is a safe, cost effective, and practical alternative in children with ALL and a Grade 2 or higher HSR.

摘要

背景

大肠杆菌来源的聚乙二醇化天冬酰胺酶(PEG)是儿童急性淋巴细胞白血病(ALL)治疗的关键组成部分。对PEG发生超敏反应(HSR)的患者接受另一种形式的天冬酰胺酶——欧文氏菌天冬酰胺酶(EA)。然而,2017年的国际短缺使得治疗这些患者具有挑战性。我们制定了一项综合策略来满足这一需求。

患者与方法

这是一项单中心回顾性分析。所有接受PEG治疗的患者均进行了预处理以减少输液反应。发生HSR的患者接受了PEG脱敏治疗。将患者与历史对照组进行比较。

结果

在研究期间共治疗了56例患者。采用通用预处理前后反应频率无差异(P = 0.78)。8例患者(14.2%)发生了≥2级HSR或沉默失活事件,5例患者(62.5%)成功进行了脱敏治疗。其余3例患者接受了EA天冬酰胺酶治疗。这一干预措施导致PEG替代率下降,干预后3例患者(5.3%)需要EA,而干预前为8例患者(15.09%)(P = 0.11)。PEG脱敏治疗比EA给药更具成本效益。

结论

对于患有ALL且发生2级或更高等级HSR的儿童,PEG脱敏是一种安全、具有成本效益且实用的替代方法。

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