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一项关于英国重症肌无力患者特征、治疗模式及医疗资源利用情况的回顾性观察研究。

A retrospective observational study on characteristics, treatment patterns, and healthcare resource use of patients with myasthenia gravis in England.

作者信息

van Enkhuizen Jordy, Binns Jean, Betts April, Hosnijeh Fatemeh Saberi, Alexander Myriam, McCormack Mark, Jacob Saiju

机构信息

UCB Pharma, 216 Bath Road, Slough, Berkshire SL1 3WE, UK.

UCB Pharma, Slough, UK.

出版信息

Ther Adv Neurol Disord. 2024 Apr 16;17:17562864241237495. doi: 10.1177/17562864241237495. eCollection 2024.

DOI:10.1177/17562864241237495
PMID:38634003
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11022674/
Abstract

BACKGROUND

There are limited data on the real-world healthcare resource use (HCRU) and management costs of myasthenia gravis (MG) in England.

OBJECTIVE

This study aims to assess the burden of disease for patients with MG in England.

DESIGN

A retrospective, observational cohort study of adult patients diagnosed with MG, using data from the Hospital Episode Statistics data warehouse.

METHODS

Patients with a first-ever recorded diagnosis of MG between 30 June 2015 and 30 June 2020 were followed up until 30 June 2021 or death, whichever occurred first. Post-diagnosis patient characteristics, treatment patterns, HCRU, and costs were described. Costs were evaluated using National Health Service reference costs.

RESULTS

A total of 9087 patients with a median follow-up time of 2.9 years (range, 1.7-4.3 years) were included. The mean age at diagnosis was 66.5 years and 53% of the patients were male. A large proportion of patients (72.8%) were admitted as inpatients during follow-up with a mean number of 1.3 admissions. Patients hospitalized for MG-related complications spent a mean of 9.7 days per patient-year in the hospital. During follow-up, 599 (6.6% of the total cohort) and 163 (1.8%) patients had a record of rescue therapy with intravenous immunoglobulin (IVIg) and plasma exchange (PLEX), respectively. Rituximab was administered to 81 (0.9%) patients and 268 (2.9%) patients underwent thymectomy. In those patients receiving rescue therapy or rituximab, >10% received at least three cycles of the same treatment. The average annual cost of hospital admissions across all patients treated with IVIg, PLEX, and rituximab were £907,072, £689,979, and £146,726, respectively.

CONCLUSION

A majority of MG patients required hospitalization or accident and emergency attendance, resulting in high HCRU and costs. A subset of patients required rescue therapy (including IVIg and PLEX), rituximab administration, ventilation, or thymectomy.

摘要

背景

关于英国重症肌无力(MG)的实际医疗资源使用(HCRU)和管理成本的数据有限。

目的

本研究旨在评估英国MG患者的疾病负担。

设计

一项对诊断为MG的成年患者进行的回顾性观察队列研究,使用医院事件统计数据仓库的数据。

方法

对2015年6月30日至2020年6月30日首次记录诊断为MG的患者进行随访,直至2021年6月30日或死亡,以先发生者为准。描述诊断后患者的特征、治疗模式、HCRU和成本。使用国民保健服务参考成本评估成本。

结果

共纳入9087例患者,中位随访时间为2.9年(范围1.7 - 4.3年)。诊断时的平均年龄为66.5岁,53%的患者为男性。大部分患者(72.8%)在随访期间住院,平均住院次数为1.3次。因MG相关并发症住院的患者平均每人每年住院9.7天。随访期间,分别有599例(占总队列的6.6%)和163例(1.8%)患者有静脉注射免疫球蛋白(IVIg)和血浆置换(PLEX)的抢救治疗记录。81例(0.9%)患者接受了利妥昔单抗治疗,268例(2.9%)患者接受了胸腺切除术。在接受抢救治疗或利妥昔单抗治疗的患者中,超过10%的患者接受了至少三个周期的相同治疗。接受IVIg、PLEX和利妥昔单抗治疗的所有患者的平均每年住院费用分别为907,072英镑、689,979英镑和146,726英镑。

结论

大多数MG患者需要住院治疗或急诊就诊,导致高HCRU和成本。一部分患者需要抢救治疗(包括IVIg和PLEX)、利妥昔单抗治疗、通气或胸腺切除术。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9e7d/11022674/b54e9dd05886/10.1177_17562864241237495-fig1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9e7d/11022674/b54e9dd05886/10.1177_17562864241237495-fig1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9e7d/11022674/b54e9dd05886/10.1177_17562864241237495-fig1.jpg

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