EXERCISE INCREASES UTROPHIN PROTEIN EXPRESSION IN THE mdx MOUSE MODEL OF DUCHENNE MUSCULAR DYSTROPHY
EXERCISE INCREASES UTROPHIN PROTEIN EXPRESSION IN THE mdx MOUSE MODEL OF DUCHENNE MUSCULAR DYSTROPHY
复制标题
DOI：
10.1002/mus.24151
复制
发表时间：
2014-06-01
期刊：
影响因子：
3.4
通讯作者：
Kostek, Matthew C.
中科院分区：
文献类型：
--
作者：
Gordon, Bradley S.;Lowe, Dawn A.;Kostek, Matthew C.
简介：在准备临床试验时，我们检查强直性肌营养不良健康指数 (MDHI) 的有效性、可靠性和患者理解。方法：最初，我们与 278 名强直性肌营养不良 1 型 (DM1) 患者合作，确定了与 MDHI 最相关的问题。接下来，我们使用因素分析、患者访谈和重测可靠性评估来完善和评估该工具。最后，我们确定了 MDHI 区分已知 DM1 参与者组的能力。结果：最终 MDHI 中的问题代表 DM1 健康状况的 17 个领域。所有分量表的内部一致性都是可以接受的。 MDHI 具有较高的重测信度 (ICC = 0.95)，并且能够区分具有不同疾病严重程度的 DM1 患者组。结论：MDHI 的初步评估提供的证据表明，它作为评估患者报告健康状况的结果指标是有效且可靠的。这些结果表明，使用 MDHI 可以有效地测量 DM1 健康的重要方面。肌肉神经 49：906-914，2014
Introduction: In preparation for clinical trials we examine the validity, reliability, and patient understanding of the Myotonic Dystrophy Health Index (MDHI). Methods: Initially we partnered with 278 myotonic dystrophy type-1 (DM1) patients and identified the most relevant questions for the MDHI. Next, we used factor analysis, patient interviews, and test-retest reliability assessments to refine and evaluate the instrument. Lastly, we determined the capability of the MDHI to differentiate between known groups of DM1 participants. Results: Questions in the final MDHI represent 17 areas of DM1 health. The internal consistency was acceptable in all subscales. The MDHI had a high test-retest reliability (ICC = 0.95) and differentiated between DM1 patient groups with different disease severities. Conclusions: Initial evaluation of the MDHI provides evidence that it is valid and reliable as an outcome measure for assessing patient-reported health. These results suggest that important aspects of DM1 health may be measured effectively using the MDHI. Muscle Nerve 49: 906-914, 2014