Comprehensive cross-sectional and longitudinal analyses of plasma neurofilament light across FTD spectrum disorders.
Comprehensive cross-sectional and longitudinal analyses of plasma neurofilament light across FTD spectrum disorders.
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DOI：
10.1016/j.xcrm.2022.100607
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发表时间：
2022-04-19
影响因子：
14.3
通讯作者：
Petrucelli, Leonard
中科院分区：
文献类型：
--
作者：
Gendron, Tania F.;Heckman, Michael G.;White, Launia J.;Veire, Austin M.;Pedraza, Otto;Burch, Alexander R.;Bozoki, Andrea C.;Dickerson, Bradford C.;Domoto-Reilly, Kimiko;Foroud, Tatiana;Forsberg, Leah K.;Galasko, Douglas R.;Ghoshal, Nupur;Graff-Radford, Neill R.;Grossman, Murray;Heuer, Hilary W.;Huey, Edward D.;Hsiung, Ging-Yuek R.;Irwin, David J.;Kaufer, Daniel, I;Leger, Gabriel C.;Litvan, Irene;Masdeu, Joseph C.;Mendez, Mario F.;Onyike, Chiadi U.;Pascual, Belen;Ritter, Aaron;Roberson, Erik D.;Rojas, Julio C.;Tartaglia, Maria Carmela;Wszolek, Zbigniew K.;Rosen, Howard;Boeve, Bradley F.;Boxer, Adam L.;Petrucelli, Leonard
额颞叶痴呆 (FTD) 治疗的发展因缺乏易感性、诊断性和预后生物标志物而受到阻碍。血液神经丝光 (NfL) 显示出作为生物标志物的前景，但研究主要集中在核心 FTD 综合征上，通常将具有不同诊断的患者分组。为了加快 NfL 的临床转化，我们利用 ARTFL LEFFTDS 纵向额颞叶变性 (ALLFTD) 研究资源，对 FTD 综合征和症状前 FTD 突变携带者的血浆 NfL 进行全面调查。我们发现，在所有研究的综合征中，包括轻度病例，血浆 NfL 均升高；表型转变前症状前突变携带者增加；并与疾病严重程度指标相关。通过促进识别有表型转化风险的个体以及 FTD 的早期诊断，血浆 NfL 可以帮助选择预防或早期治疗试验的参与者。此外，其预后效用将改善患者护理、临床试验效率和治疗结果估计。额颞叶痴呆 (FTD) 症状出现之前，血浆 NfL 水平会增加 血浆 NfL 可以促进早期 FTD 诊断 血浆 NfL 水平与 FTD 疾病严重程度的临床指标相关 血浆 NfL 有希望作为 FTD 的易感性和预后生物标志物 Gendron 等人。研究表明，具有额颞叶痴呆 (FTD) 遗传风险的个体在症状出现前，血浆神经丝光 (NfL) 会增加，并且与疾病严重程度的临床指标相关。因此，血浆 NfL 有希望作为一种易感性和预后生物标志物，可以改善患者护理和 FTD 治疗开发。
Frontotemporal dementia (FTD) therapy development is hamstrung by a lack of susceptibility, diagnostic, and prognostic biomarkers. Blood neurofilament light (NfL) shows promise as a biomarker, but studies have largely focused only on core FTD syndromes, often grouping patients with different diagnoses. To expedite the clinical translation of NfL, we avail ARTFL LEFFTDS Longitudinal Frontotemporal Lobar Degeneration (ALLFTD) study resources and conduct a comprehensive investigation of plasma NfL across FTD syndromes and in presymptomatic FTD mutation carriers. We find plasma NfL is elevated in all studied syndromes, including mild cases; increases in presymptomatic mutation carriers prior to phenoconversion; and associates with indicators of disease severity. By facilitating the identification of individuals at risk of phenoconversion, and the early diagnosis of FTD, plasma NfL can aid in participant selection for prevention or early treatment trials. Moreover, its prognostic utility would improve patient care, clinical trial efficiency, and treatment outcome estimations. Plasma NfL levels increase prior to frontotemporal dementia (FTD) symptom onset Plasma NfL can facilitate an earlier FTD diagnosis Plasma NfL levels associate with clinical indicators of FTD disease severity Plasma NfL shows promise as a susceptibility and prognostic biomarker for FTD Gendron et al. show that plasma neurofilament light (NfL) increases before symptom onset in individuals with a genetic risk of frontotemporal dementia (FTD) and associates with clinical indicators of disease severity. Plasma NfL thus holds promise as a susceptibility and prognostic biomarker that can improve patient care and FTD treatment development.
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影响因子：
5.3
作者：
Rojas JC;Karydas A;Bang J;Tsai RM;Blennow K;Liman V;Kramer JH;Rosen H;Miller BL;Zetterberg H;Boxer AL
通讯作者：
Boxer AL
影响因子：
9.9
作者：
Alcolea, Daniel;Vilaplana, Eduard;Lleo, Alberto
通讯作者：
Lleo, Alberto
影响因子：
9.9
作者：
Rojas JC;Wang P;Staffaroni AM;Heller C;Cobigo Y;Wolf A;Goh SM;Ljubenkov PA;Heuer HW;Fong JC;Taylor JB;Veras E;Song L;Jeromin A;Hanlon D;Yu L;Khinikar A;Sivasankaran R;Kieloch A;Valentin MA;Karydas AM;Mitic LL;Pearlman R;Kornak J;Kramer JH;Miller BL;Kantarci K;Knopman DS;Graff-Radford N;Petrucelli L;Rademakers R;Irwin DJ;Grossman M;Ramos EM;Coppola G;Mendez MF;Bordelon Y;Dickerson BC;Ghoshal N;Huey ED;Mackenzie IR;Appleby BS;Domoto-Reilly K;Hsiung GR;Toga AW;Weintraub S;Kaufer DI;Kerwin D;Litvan I;Onyike CU;Pantelyat A;Roberson ED;Tartaglia MC;Foroud T;Chen W;Czerkowicz J;Graham DL;van Swieten JC;Borroni B;Sanchez-Valle R;Moreno F;Laforce R;Graff C;Synofzik M;Galimberti D;Rowe JB;Masellis M;Finger E;Vandenberghe R;de Mendonça A;Tagliavini F;Santana I;Ducharme S;Butler CR;Gerhard A;Levin J;Danek A;Otto M;Sorbi S;Cash DM;Convery RS;Bocchetta M;Foiani M;Greaves CV;Peakman G;Russell L;Swift I;Todd E;Rohrer JD;Boeve BF;Rosen HJ;Boxer AL;ALLFTD and GENFI consortia
通讯作者：
ALLFTD and GENFI consortia
DOI：
10.1136/jnnp-2021-326914
发表时间：
2021-12
期刊：
Journal of neurology, neurosurgery, and psychiatry
影响因子：
--
作者：
Saracino D;Dorgham K;Camuzat A;Rinaldi D;Rametti-Lacroux A;Houot M;Clot F;Martin-Hardy P;Jornea L;Azuar C;Migliaccio R;Pasquier F;Couratier P;Auriacombe S;Sauvée M;Boutoleau-Bretonnière C;Pariente J;Didic M;Hannequin D;Wallon D;French Research Network on FTD/FTD-ALS;PREV-DEMALS and Predict-PGRN study groups;Colliot O;Dubois B;Brice A;Levy R;Forlani S;Le Ber I
通讯作者：
Le Ber I
影响因子：
9.9
作者：
Sjögren, M;Rosengren, L;Wallin, A
通讯作者：
Wallin, A