From the Department of Neurology (G.T., K.R., Y.C., A.S., J. Fong, H.H., J.K., J.K., B.M., A.B., H.R., K.R.), Memory and Aging Center, University of California, San Francisco; Case Western Reserve University (B.A.), Cleveland, OH; Mayo Clinic (D.B., J. Fields, L.F., N.G.-R., K.K., D. Knopman, J.S., B.B.), Rochester, MN; University of California (G.C., M.M., E.R.), Los Angeles; Department of Neurology (B.D.), Massachusetts General Hospital, Harvard Medical School, Boston; University of Washington (K.D.-R.), Seattle; Washington University (N.G.), St. Louis, MO; University of Pennsylvania (M.G., D.I., K.R.), Philadelphia; University of British Columbia (G.-Y.H., I.M.), Vancouver, Canada; Columbia University (E.H.), New York, NY; University of North Carolina (D. Kaufer), Chapel Hill; University of Texas Southwestern (D. Kerwin), Dallas; Department of Neuroscience (I.L.), Parkinson and Other Movement Disorder Center, University of California, San Diego; Mayo Clinic (R.R.), Jacksonville, FL; University of Alabama at Birmingham (E.R.); University of Toronto (C.T.), Ontario, Canada; and Mesulam Center for Cognitive Neurology and Alzheimer's Disease (S.W.), Northwestern University, Chicago, IL.
Neurology. 2020 Jun 2;94(22):e2384-e2395. doi: 10.1212/WNL.0000000000009451. Epub 2020 May 5.
To investigate whether the Revised Self-Monitoring Scale (RSMS), an informant measure of socioemotional sensitivity, is a potential clinical endpoint for treatment trials for patients with behavioral variant frontotemporal dementia (bvFTD).
We investigated whether RSMS informant ratings reflected disease severity in 475 participants (71 bvFTD mutation+, 154 bvFTD mutation-, 12 behavioral mild cognitive impairment [MCI] mutation+, 98 asymptomatic mutation+, 140 asymptomatic mutation-). In a subset of 62 patients (20 bvFTD mutation+, 35 bvFTD mutation-, 7 MCI mutation+) who had at least 2 time points of T1-weighted images available on the same 3T scanner, we examined longitudinal changes in RSMS score over time and its correspondence to progressive gray matter atrophy.
RSMS score showed a similar pattern in mutation carriers and noncarriers, with significant drops at each stage of progression from asymptomatic to very mild, mild, moderate, and severe disease = 140.10, < 0.001) and a significant slope of decline over time in patients with bvFTD ( = 0.004, 95% confidence interval [CI] -1.90 to -0.23). More rapid declines on the RSMS corresponded to faster gray matter atrophy predominantly in the salience network (SN), and RSMS score progression best predicted thalamic volume in very mild and mild disease stages of bvFTD. Higher RSMS score predicted more caregiver burden ( < 0.001, 95% CI -0.30 to -0.11).
The RSMS is sensitive to progression of both socioemotional symptoms and SN atrophy in patients with bvFTD and corresponds directly to caregiver burden. The RSMS may be useful in both neurologic practice and clinical trials aiming to treat behavioral symptoms of patients with bvFTD.
研究修订后的自我监测量表（RSMS）作为一种社会情感敏感性的知情者测量指标，是否可作为行为变异额颞叶痴呆（bvFTD）患者治疗试验的潜在临床终点。
我们调查了 RSMS 知情者评分是否反映了 475 名参与者（71 名 bvFTD 突变阳性，154 名 bvFTD 突变阴性，12 名行为性轻度认知障碍 [MCI] 突变阳性，98 名无症状突变阳性，140 名无症状突变阴性）的疾病严重程度。在 62 名患者（20 名 bvFTD 突变阳性，35 名 bvFTD 突变阴性，7 名 MCI 突变阳性）中，他们至少有 2 次在同一 3T 扫描仪上的 T1 加权图像，我们检查了 RSMS 评分随时间的纵向变化及其与进行性灰质萎缩的对应关系。
RSMS 评分在突变携带者和非携带者中表现出相似的模式，从无症状到非常轻度、轻度、中度和重度疾病的每个阶段都有显著下降（ = 140.10，<0.001），并且 bvFTD 患者的时间斜率明显下降（ = 0.004，95%置信区间 [CI] -1.90 至-0.23）。RSMS 评分的快速下降与主要在突显网络（SN）中的更快灰质萎缩相对应，并且 RSMS 评分的进展最好预测了 bvFTD 的非常轻度和轻度疾病阶段的丘脑体积。更高的 RSMS 评分预测了更大的 caregiver负担（<0.001，95%CI -0.30 至-0.11）。
RSMS 对 bvFTD 患者的社会情感症状和 SN 萎缩的进展均敏感，并直接对应于 caregiver负担。RSMS 可能在神经病学实践和旨在治疗 bvFTD 患者行为症状的临床试验中都有用。
