A 12-year follow-up in sporadic inclusion body myositis: an end stage with major disabilities
A 12-year follow-up in sporadic inclusion body myositis: an end stage with major disabilities
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DOI：
10.1093/brain/awr217
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发表时间：
2011-11-01
期刊：
影响因子：
14.5
通讯作者：
Badrising, Umesh A.
中科院分区：
文献类型：
--
作者：
Cox, Fieke M.;Titulaer, Maarten J.;Badrising, Umesh A.
散发性包涵体肌炎被认为是一种缓慢进行性肌病。然而，长期随访数据尚未获得。随访数据对于告知患者其预后和为临床试验选择适当的结局指标非常重要。我们对64例参与荷兰国家流行病学研究的散发性包涵体肌炎患者进行了随访研究。记录病史，并在基线和首次门诊访视后12年（中位数）进行手动和定量肌肉测试以及实验室检查。记录所有死亡患者的死亡日期和原因。46例患者在随访期间死亡，2例患者选择不参加，1例患者失访。其余15例存活患者的平均病程为20年，并在第二个时间点进行临床评价。根据手动肌肉测试和定量肌肉测试，力量的平均下降分别为每年3.5%和5.4%。这种下降在下肢最为明显，这也是最弱的四肢。预期寿命正常，为81岁，但日常生活活动明显受限。在随访时，发现所有患者都使用轮椅，其中7人（47%）完全依赖轮椅。呼吸系统疾病是最常见的死亡原因。在3名患者中，要求安乐死，在另外3名患者中，应用持续深度镇静。在6名患者（13%）中使用了临终护理干预措施，这一事实反映了这种疾病终末期的严重残疾和生活质量下降。散发性包涵体肌炎是一种慢性进行性疾病，由于广泛的肌肉无力，在疾病的终末期导致严重残疾。
Sporadic inclusion body myositis is considered to be a slowly progressive myopathy. Long-term follow-up data are, however, not yet available. Follow-up data are important with a view to informing patients about their prognosis and selecting appropriate outcome measures for clinical trials. We performed a follow-up study of 64 patients with sporadic inclusion body myositis who participated in a national epidemiological study in the Netherlands. Case histories were recorded, and manual and quantitative muscle tests as well as laboratory tests were performed at baseline and 12 years (median) after the first out-patient visit. Date and cause of death were recorded for all deceased patients. Forty-six patients died during the follow-up period, two patients chose not to participate and one patient was lost to follow-up. The remaining 15 surviving patients had a mean disease duration of 20 years and were clinically evaluated at the second time point. The mean decline in strength was 3.5 and 5.4% per year according to the manual muscle testing and quantitative muscle testing, respectively. This decline was most pronounced in the lower legs, which were also the weakest extremities. Life expectancy was normal at 81 years, but activities of daily life were clearly restricted. At follow-up, all patients were found to be using a wheelchair, seven of them (47%) being completely wheelchair-bound. Disorders of the respiratory system were the most common cause of death. In three patients, euthanasia was requested and in another three, continuous deep sedation was applied. The fact that end-of-life care interventions were used in six patients (13%) reflects the severe disability and loss of quality of life at the end stage of this disease. Sporadic inclusion body myositis is a chronic progressive disorder, leading to major disabilities at the end stage of the disease due to extensive muscle weakness.