Defining Clinical Improvement in Adult and Juvenile Myositis.
Defining Clinical Improvement in Adult and Juvenile Myositis.
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发表时间：
2003-03
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通讯作者：
L. Rider;E. Giannini;M. Harris-Love;G. Joe;D. Isenberg;C. Pilkington;P. Lachenbruch;F. Miller
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作者：
L. Rider;E. Giannini;M. Harris-Love;G. Joe;D. Isenberg;C. Pilkington;P. Lachenbruch;F. Miller
对于特发性炎症性肌病(IIM)的结果测量和试验设计问题缺乏共识，这阻碍了临床试验的进行和解释。为了着手解决这些问题，一个由70多名成人和儿科神经学家、风湿病专家、康复医学医生、统计学家和患者支持小组负责人组成的多专业小组，称为国际肌炎结局评估合作研究小组(IMACS)，正在致力于就肌炎临床试验的疾病活动和损害评估达成共识。作为这一持续进行的国际努力的一部分，该小组的成员于2001年11月在一家名为“确定成人和青少年肌炎临床改进”的工作场所举行了会议。该工作组的一个目标是审查最近发表的关于肌炎临床试验中疾病结果评估的初步核心集测量的有效性和响应性的当前数据，并确定每个核心集测量中具有临床意义的变化程度。尽管成人和青少年发作性肌炎的临床表现、自然病史和治疗反应不同，但这些疾病的专家们达成了共识，即在每项核心指标中，对成人和青少年肌炎具有临床意义的改善程度是相同的。对于肌肉力量和身体功能领域，至少15%的改善是临床显著的，而对于医生和患者的全球评估以及肌肉外评估，至少20%的改善被认为是具有临床意义的，而对于血清肌肉相关酶水平，至少需要30%的改善才是临床重要的。这次研讨会是计划就IIM临床试验的实施和报告达成多学科、国际共识的几个研讨会中的第一个。
The lack of consensus regarding outcome measures and trial design issues in the idiopathic inflammatory myopathies (IIM) is inhibiting the conduct and interpretation of clinical trials. To begin to address these problems, a multispecialty group of over 70 adult and pediatric neurologists, rheumatologists, rehabilitation medicine physicians, statisticians, and patient support group leaders, called the International Myositis Outcome Assessment Collaborative Study Group (IMACS), is engaged in developing consensus on the assessment of disease activity and damage for myositis clinical trials. As part of this ongoing international effort, members of this group met in November 2001 at a work-shop entitled "Defining Clinical Improvement in Adult and Juvenile Myositis." A goal of the work-shop was to review current data on the validity and responsiveness of the recently published proposed preliminary core set measures for disease outcome assessment in clinical trials for myositis and to define the degree of change in each core set measure that is clinically meaningful. Despite differences in the clinical presentations, natural history and responses to therapy between adult onset and juvenile onset myositis, expert specialists in these diseases came to a consensus that the amount of improvement that is clinically meaningful in each core measure is the same for adult and juvenile myositis. For the domains of muscle strength and physical function, a minimum of 15% improvement is clinically significant, whereas for the physician and patient global assessments, as well as the extramuscular assessment, a minimum of 20% improvement is considered clinically meaningful, and for serum levels of muscle associated enzymes, at least 30% improvement is needed to be clinically important. This workshop is the first of several planned to develop multidisciplinary, international consensus on the conduct and reporting of IIM clinical trials.