41PTreatment outcomes in idiopathic inflammatory myopathies based on pathology and autoantibody profiles: a single-center study of 127 cases

IF 2.8 4区医学 Q2 CLINICAL NEUROLOGY

Neuromuscular Disorders Pub Date : 2025-09-01 DOI:10.1016/j.nmd.2025.105504

N. Eura, Y. Nishimori, A. Yamanaka, T. Shiota, H. Tanaka, T. Ohashi, H. Shimizu, M. Yamaoka, N. Yamada, N. Iguchi, A. Tanaka, M. Sugata, H. Nanaura, T. Kiriyama, K. Sugie

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Abstract

Idiopathic inflammatory myopathies (IIMs) are autoimmune disorders affecting skeletal muscle and various organs. Although myositis-specific autoantibodies (MSAs) aid in diagnosis, treatment strategies remain suboptimal. Large-scale, subtype-specific outcome studies are limited. We retrospectively analyzed 127 IIM patients who underwent muscle biopsy at our institution between 2009 and 2023. Inclusion required pathological confirmation, excluding inclusion body myositis and secondary myositis related to collagen diseases or immune checkpoint inhibitors. MSAs were detected in 97 patients (76%). Among MSA-positive cases, dermatomyositis (DM: 27.1%) and immune-mediated necrotizing myopathy (IMNM: 27.1%) were most common, followed by antisynthetase syndrome (ASS: 14.4%) and polymyositis (PM: 2.7%). DM was linked to anti-TIF1-γ, MDA5, Mi-2, and NXP-2 antibodies (in that order); IMNM to SRP, HMGCR, and AMA-M2. HMGCR-positive patients had the highest CK levels. Interstitial lung disease (ILD) was seen in 90% of MDA5, 67% of Mi-2, and 50% of SRP cases. Malignancies occurred in all antibody subtypes except MDA5, NXP-2, and HMGCR, and originated from diverse organ systems. Seven of 13 traceable malignancy cases died within 3 years of IIM diagnosis. Notably, AMA-M2-positive patients showed a high frequency of respiratory failure unrelated to ILD (40%) and cardiac events such as arrhythmias or heart failure (89%). All patients received glucocorticoids. Immunosuppressants were used in 80% of MDA5, 60% of HMGCR, and 47% of ASS cases. The most frequently used immunosuppressant was tacrolimus, followed by methotrexate and azathioprine. IVIg was limited to four severe cases. At 3 years, the mean prednisolone dose was 8.4 ± 5.5 mg; only SRP-positive patients reached <5 mg/day. Orthopedic complications such as fractures occurred in 9.2%. This study highlights clinical diversity and treatment challenges in IIM. Despite improved diagnostics, more effective, tailored therapies are urgently needed.

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基于病理和自身抗体谱的特发性炎性肌病治疗结果：一项127例病例的单中心研究

特发性炎症性肌病（IIMs）是一种影响骨骼肌和各器官的自身免疫性疾病。虽然肌炎特异性自身抗体（msa）有助于诊断，但治疗策略仍然不理想。大规模的、特定亚型的结果研究是有限的。我们回顾性分析了2009年至2023年间在我院接受肌肉活检的127例IIM患者。纳入需要病理确认，排除包涵体肌炎和与胶原蛋白疾病或免疫检查点抑制剂相关的继发性肌炎。97例（76%）患者检出msa。在msa阳性病例中，最常见的是皮肌炎（DM: 27.1%）和免疫介导的坏死性肌病（IMNM: 27.1%），其次是抗合成酶综合征（ASS: 14.4%）和多发性肌炎（PM: 2.7%）。DM与抗tif1 -γ、MDA5、Mi-2和NXP-2抗体相关（按顺序）；IMNM到SRP， HMGCR和AMA-M2。hmgcr阳性患者CK水平最高。间质性肺疾病（ILD）见于90%的MDA5、67%的Mi-2和50%的SRP病例。恶性肿瘤发生在除MDA5、NXP-2和HMGCR以外的所有抗体亚型中，并且起源于不同的器官系统。13例可追溯恶性肿瘤中有7例在IIM诊断后3年内死亡。值得注意的是，ama - m2阳性患者表现出与ILD无关的呼吸衰竭（40%）和心律失常或心力衰竭（89%）等心脏事件的高频率。所有患者均接受糖皮质激素治疗。80%的MDA5、60%的HMGCR和47%的ASS病例使用免疫抑制剂。最常用的免疫抑制剂是他克莫司，其次是甲氨蝶呤和硫唑嘌呤。IVIg仅限于4例严重病例。3年时，泼尼松龙的平均剂量为8.4±5.5 mg；只有srp阳性患者达到5mg /天。骨折等骨科并发症发生率为9.2%。这项研究强调了IIM的临床多样性和治疗挑战。尽管诊断方法有所改进，但迫切需要更有效、更有针对性的治疗方法。

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来源期刊

Neuromuscular Disorders 医学-临床神经学

CiteScore

4.60

自引率

3.60%

发文量

543

审稿时长

53 days

期刊介绍： This international, multidisciplinary journal covers all aspects of neuromuscular disorders in childhood and adult life (including the muscular dystrophies, spinal muscular atrophies, hereditary neuropathies, congenital myopathies, myasthenias, myotonic syndromes, metabolic myopathies and inflammatory myopathies). The Editors welcome original articles from all areas of the field: • Clinical aspects, such as new clinical entities, case studies of interest, treatment, management and rehabilitation (including biomechanics, orthotic design and surgery). • Basic scientific studies of relevance to the clinical syndromes, including advances in the fields of molecular biology and genetics. • Studies of animal models relevant to the human diseases. The journal is aimed at a wide range of clinicians, pathologists, associated paramedical professionals and clinical and basic scientists with an interest in the study of neuromuscular disorders.