静脉注射免疫球蛋白疗法:卡塔尔十年间的使用模式及治疗反应

Intravenous immunoglobulin therapy: usage patterns and response to treatment in Qatar over ten years.

作者信息

Taha Salma A, Thalappil Sherin, Ali Ramzy M, Fatima Haajra, Imameldin Asaad Omer A, Aqel Sami, Abdelaal Ahmed M, Siepmann Timo, Barlinn Jessica, Al-Nesf Maryam A

机构信息

Allergy and Immunology Division, Department of Medicine, Hamad Medical Corporation, Doha, Qatar.

General Internal Medicine Division, Department of Medical Education, Hamad Medical Corporation, Doha, Qatar.

出版信息

Front Immunol. 2024 Dec 2;15:1481079. doi: 10.3389/fimmu.2024.1481079. eCollection 2024.

Abstract

BACKGROUND

IVIg is a blood-derived antibody product initially designed as a replacement therapy in inborn errors of immunity (IEIs). However, over the last 50 years, IVIg has been used to treat a growing range of autoimmune, autoinflammatory, and secondary immunodeficiency disorders. The US FDA has licensed IVIg for use in the treatment of nine clinical indications; although, IVIg global usage extends to off-label indications with variable treatment responses. Data from Qatar on the use of IVIg is scarce; thus, hampering the formulation of local policies. This study aimed to examine the utilization patterns, clinical indications, and safety profile of IVIg usage in Qatar; a nation with a predominantly young population, and to investigate the response rates to short- and long-term IVIg treatment, as well as explore associations between age at first IVIg dose, clinical indication, and treatment response.

METHODS

A retrospective chart review was conducted of patients who received IVIg between March 2009, and March 2019, in Hamad General Hospital, Qatar. Demographics, immediate adverse effects of IVIg, and treatment response were collected. IVIg clinical indications were categorized into FDA- and/or EMA-approved, those supported by international guidelines; those approved as second-line therapy, and those with low or no supportive evidence.

RESULTS

IVIg was used for 63 indications during the 10-years. The age of patients skewed towards a younger demographic (median (IQR) 24 (44-6) years); however, no significant differences in response to short- and long-term treatment between age groups were observed. Of the 841 patients, 62% received IVIg in concordance with international recommendations, while 14% bestowed the treatment for indications with low or no supportive evidence. Immediate IVIg adverse effects were documented in 4% of patients in all of the infusions received, with headaches being the most prevalent (1.8%). Variable treatment responses were observed, with the highest recovery reported in immune thrombocytopenic purpura (35%), followed by transverse myelitis (28%).

CONCLUSION

This study provided crucial insights into IVIg utilization, safety, and treatment outcomes in Qatar's young population. Despite variability in treatment responses and off-label use, adherence to international recommendations remained eminent. Further research is warranted to inform local guidelines and optimize IVIg therapy outcomes.

摘要

背景

静脉注射免疫球蛋白(IVIg)是一种血液来源的抗体产品,最初设计用于先天性免疫缺陷病(IEIs)的替代治疗。然而,在过去50年里,IVIg已被用于治疗越来越多的自身免疫性、自身炎症性和继发性免疫缺陷疾病。美国食品药品监督管理局(FDA)已批准IVIg用于九种临床适应症的治疗;尽管如此,IVIg在全球的使用范围已扩展到一些治疗反应各异的非适应症情况。卡塔尔关于IVIg使用的数据很少,这阻碍了当地政策的制定。本研究旨在调查卡塔尔IVIg的使用模式、临床适应症和安全性;卡塔尔主要是年轻人口国家,并调查短期和长期IVIg治疗的反应率,以及探讨首次使用IVIg的年龄、临床适应症和治疗反应之间的关联。

方法

对2009年3月至2019年3月期间在卡塔尔哈马德总医院接受IVIg治疗的患者进行回顾性病历审查。收集人口统计学资料、IVIg的即刻不良反应和治疗反应。IVIg的临床适应症分为FDA和/或欧洲药品管理局(EMA)批准的、有国际指南支持的、批准作为二线治疗的以及支持证据少或无支持证据的。

结果

在这10年中,IVIg用于63种适应症。患者年龄倾向于年轻人群(中位数(四分位间距)24(44 - 6)岁);然而,各年龄组在短期和长期治疗反应方面未观察到显著差异。在841例患者中,62%的患者按照国际建议接受IVIg治疗,而14%的患者将其用于支持证据少或无支持证据的适应症。在所有接受输液的患者中,4%记录有IVIg即刻不良反应,其中头痛最为常见(1.8%)。观察到不同的治疗反应,免疫性血小板减少性紫癜的恢复率最高(35%),其次是横贯性脊髓炎(28%)。

结论

本研究为卡塔尔年轻人群中IVIg的使用、安全性和治疗结果提供了重要见解。尽管治疗反应存在差异且存在非适应症使用情况,但遵循国际建议仍然很突出。有必要进一步开展研究以指导当地指南并优化IVIg治疗结果。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/092d/11646857/9ffa3698d8cb/fimmu-15-1481079-g001.jpg

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