Long-term observational study of sporadic inclusion body myositis
Long-term observational study of sporadic inclusion body myositis
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DOI：
10.1093/brain/awr213
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发表时间：
2011-11-01
期刊：
影响因子：
14.5
通讯作者：
Hilton-Jones, David
中科院分区：
文献类型：
--
作者：
Benveniste, Olivier;Guiguet, Marguerite;Hilton-Jones, David
我们描述了一项对散发性包涵体肌炎患者进行的长期观察性研究，并提出了一种易于在临床上执行的散发性包涵体肌炎虚弱综合指数。两组患者（巴黎和牛津）的数据收集是在诊所访视期间完成的（52%），或通过提取既往病历（48%）完成的。纳入了136例患者[57%为男性，发病时61岁（四分位距55-69）]。在最后一次就诊时，所有患者都有肌无力（近端英国医学研究理事会量表< 3/5的患者占48%，远端英国医学研究理事会量表< 3/5的患者占40%，吞咽问题的患者占46%）。在随访期间，75%的患者有明显的行走困难，37%的患者使用轮椅（从发病开始的中位持续时间为14年）。散发性包涵体肌炎虚弱综合指数与握力（相关系数：0.47; P < 0.001）和Rivermead活动指数（相关系数：0.85; P < 0.001）相关，随病程延长而显著降低（相关系数：-0.47; P < 0.001）。死亡风险仅受首次出现症状时年龄较大的影响。71例（52%）患者接受免疫抑制治疗[91.5%的患者接受泼尼松，64.8%的患者接受其他免疫调节药物（静脉注射免疫球蛋白、甲氨蝶呤或硫唑嘌呤）联合治疗，中位持续时间为40.8个月]。在最后一次评估中，接受治疗的患者在残疾量表（Walton P = 0.007，Rivermead活动指数P = 0.004）和散发性包涵体肌炎虚弱综合指数（P = 0.04）上受到更严重的影响。接受免疫抑制治疗的患者中，第一阶段疾病进展至行走障碍的速度更快（风险比= 2.0，P = 0.002）。这项研究证实，散发性包涵体肌炎是缓慢进行性的，但不是致命的，免疫抑制治疗不能改善其自然病程，从而证实了从较小的研究结果。此外，我们的研究结果表明，免疫抑制剂药物治疗可能会适度加剧残疾的进展。散发性包涵体肌炎虚弱综合指数可能是未来临床试验的一个有价值的结局指标，但需要进一步评估和验证。
We describe a long-term observational study of a large cohort of patients with sporadic inclusion body myositis and propose a sporadic inclusion body myositis weakness composite index that is easy to perform during a clinic. Data collection from two groups of patients (Paris and Oxford) was completed either during a clinic visit (52%), or by extraction from previous medical records (48%). One hundred and thirty-six patients [57% males, 61 (interquartile range 55-69) years at onset] were included. At the last visit all patients had muscle weakness (proximal British Medical Research Council scale < 3/5 in 48%, distal British Medical Research Council scale < 3/5 in 40%, swallowing problems in 46%). During their follow-up, 75% of patients had significant walking difficulties and 37% used a wheelchair (after a median duration from onset of 14 years). The sporadic inclusion body myositis weakness composite index, which correlated with grip strength (correlation coefficient: 0.47; P < 0.001) and Rivermead Mobility Index (correlation coefficient: 0.85; P < 0.001), decreased significantly with disease duration (correlation coefficient: -0.47; P < 0.001). The risk of death was only influenced by older age at onset of first symptoms. Seventy-one (52%) patients received immunosuppressive treatments [prednisone in 91.5%, associated (in 64.8%) with other immunomodulatory drugs (intravenous immunoglobulins, methotrexate or azathioprine) for a median duration of 40.8 months]. At the last assessment, patients who had been treated were more severely affected on disability scales (Walton P = 0.007, Rivermead Mobility Index P = 0.004) and on the sporadic inclusion body myositis weakness composite index (P = 0.04). The first stage of disease progression towards handicap for walking was more rapid among patients receiving immunosuppressive treatments (hazard ratio = 2.0, P = 0.002). This study confirms that sporadic inclusion body myositis is slowly progressive but not lethal and that immunosuppressive treatments do not ameliorate its natural course, thus confirming findings from smaller studies. Furthermore, our findings suggest that immunosuppressant drug therapy could have modestly exacerbated progression of disability. The sporadic inclusion body myositis weakness composite index might be a valuable outcome measure for future clinical trials, but requires further assessment and validation.