在治疗抗-HMGCR 免疫介导的坏死性肌病时,早期启动 IVIG 与晚期启动 IVIG 的益处。

IF 3.7 2区 医学 Q1 RHEUMATOLOGY Arthritis Care & Research Pub Date : 2024-07-25 DOI:10.1002/acr.25406
Kyle Sharf, Toan Do, Daniela Ghetie, Dongseok Choi, Nizar Chahin
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引用次数: 0

摘要

背景/目的:免疫介导坏死性肌病(IMNM)是一种难治性疾病,尤其是在诊断和治疗延误的病例中,永久性肌肉损伤会增加发病率。自身免疫性坏死性肌病的一个亚型与 3-羟基-3-甲基戊二酰辅酶 A 还原酶(HMGCR)抗体有关。治疗方法包括停用他汀类药物,并联合使用免疫抑制剂和免疫调节治疗。我们的研究旨在通过我们肌炎中心的抗-HMGCR IMNM 患者队列,提供有关静脉注射免疫球蛋白(IVIG)早期启动与晚期启动结果的纵向数据:我们对俄勒冈健康与科学大学(OHSU)肌炎中心在2016年9月至2022年10月期间确诊的31名抗HMGCR-IMNM成人患者进行了回顾性病历审查,并审查了他们的体格检查、血清学实验室数据和治疗情况,包括泼尼松的使用情况以及治疗0个月(开始使用IVIG前的评估)、3个月、6个月和12个月的治疗反应。我们将这一群体分为在确诊抗-HMGCR IMNM 后 6 个月或 6 个月之前接受 IVIG 治疗的群体和在确诊后 6 个月之后接受 IVIG 治疗的群体,前者称为 "非延迟 "群体,后者称为 "延迟 "群体。根据 2016 ENMC 标准,抗-HMGCR IMNM 的诊断标准是:血清 CK 升高、近端肌无力和抗-HMGCR 抗体三者缺一不可。我们根据2016年ACR/EULAR肌炎反应标准,采用有限总改善评分(TIS)评估治疗反应:在总共 31 名患者中,19 人被纳入非延迟队列,12 人被纳入延迟队列。两组患者从出现症状到确诊的时间相当,但延迟组患者从确诊到接受 IVIG 治疗的时间明显较长(p 值 0.999),但延迟组患者在 0 个月时点的血清 CK 预期较低(p 值 0.016)。在 0 个月的时间点上,9 名非延迟患者(47%)需要使用助行器或轮椅,而 8 名延迟患者(66%)需要使用助行器或轮椅。非延迟患者的 MMT8 在 12 个月间隔期内有明显改善(P 值 结论:非延迟患者的 MMT8 在 12 个月间隔期内有明显改善(P 值 结论:非延迟患者的 MMT8 在 12 个月间隔期内有明显改善):尽管我们的研究结果存在局限性,但它为越来越多的证据提供了依据,这些证据表明,对于抗 HMGCR IMNM 患者来说,IVIG 可被证明是早期积极诱导疗法的重要补充。延迟 IVIG 治疗可能会导致永久性残余乏力和长期残疾。
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Benefits of Early Versus Late Initiation of Intravenous Immunoglobulin in the Treatment of Patients With Anti–3-Hydroxy-3-Methylglutaryl-Coenzyme A Reductase Immune-Mediated Necrotizing Myopathy

Objective

No clinical trials have been conducted to establish optimal and effective treatment in patients with immune-mediated necrotizing myopathy (IMNM), which can have a refractory course with increased morbidity from permanent muscle damage, especially in patients who experience delay in diagnosis and treatment. A subset of autoimmune necrotizing myopathy is associated with antibodies against 3-hydroxy-3-methylglutaryl-coenzyme A reductase (HMGCR). Treatment involves withdrawing statins and using a combination of immunosuppressant and immunomodulatory treatment. Our study aims to provide longitudinally collected data on outcomes of early versus late initiation of intravenous Ig (IVIG) using our myositis center cohort of patients with anti-HMGCR IMNM.

Methods

We conducted a retrospective chart review of 31 adult patients of the Oregon Health and Science University Myositis Center who were diagnosed with anti-HMGCR IMNM from September 2016 through October 2022 and reviewed physical examination, serologic laboratory data, and their treatment including prednisone reception as well as treatment response at 0 (the evaluation immediately before IVIG initiation), 3, 6, and 12 months on treatment. We divided this cohort into those who received IVIG at or before six months after receiving the diagnosis of anti-HMGCR IMNM and refer this as the cohort with nondelayed treatment, and those who received IVIG after six months following their diagnosis, which we referred to as the cohort with delayed treatment. Diagnosis of anti-HMGCR IMNM was defined as per the 2016 European Neuromuscular Centre criteria as having all three of elevated serum creatine kinase (CK), proximal muscle weakness, and anti-HMGCR antibodies. We evaluated the response to treatment by using a limited total improvement score (TIS) as per 2016 American College of Rheumatology/EULAR myositis response criteria.

Results

Among the 31 total patients, 19 were included within the cohort with nondelayed treatment, and 12 within the cohort with delayed treatment. The two cohorts had a comparable amount of time between the onset of symptoms and diagnosis; however, the cohort with delayed treatment had a significantly longer time between diagnosis and IVIG treatment (P < 0.001). At disease onset, cohorts had a comparable serum CK (P > 0.999), but patients with delayed treatment had an expected lower serum CK (P = 0.016) at the 0-month time point. At the 0-month time point, nine of the patients with nondelayed treatment (47%) required the use of a walker or wheelchair, whereas eight of the cohort with delayed treatment (66%) did. Patients who received nondelayed treatment demonstrated significant improvement in manual muscle testing 8 at the 12-month intervals (P < 0.001). Average serum CK values of all patients measured at the 3, 6, and 12 months did not significantly differ between the groups with nondelayed and delayed treatment. TIS improved more in the group with nondelayed treatment than in the group with delayed treatment (P = 0.002 at 3 months, P = 0.019 at 6 months, and P = 0.001 at 12 months). Seven patients in the group with delayed treatment had permanent residual muscle weakness requiring walker or wheelchair use at 12 months, whereas none of the patients in the group with nondelayed treatment did.

Conclusion

Though our results have limitations, they contribute to a growing body of evidence that suggests that IVIG may prove to be a valuable addition to an early and aggressive induction regimen in patients afflicted by anti-HMGCRIMNM, particularly those with moderate to severe weakness requiring the use of a wheelchair or walking aids. Delay in IVIG treatment may lead to the development of permanent residual weakness and long-term disability.

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来源期刊
CiteScore
9.40
自引率
6.40%
发文量
368
审稿时长
3-6 weeks
期刊介绍: Arthritis Care & Research, an official journal of the American College of Rheumatology and the Association of Rheumatology Health Professionals (a division of the College), is a peer-reviewed publication that publishes original research, review articles, and editorials that promote excellence in the clinical practice of rheumatology. Relevant to the care of individuals with rheumatic diseases, major topics are evidence-based practice studies, clinical problems, practice guidelines, educational, social, and public health issues, health economics, health care policy, and future trends in rheumatology practice.
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