Predicting Progression in Parkinson's Disease Using Baseline and 1-Year Change Measures

被引：18

作者：

Chahine, Lana M. ^{[1
]}

Siderowf, Andrew ^{[2
,12
]}

Barnes, Janel ^{[3
]}

Seedorff, Nicholas ^{[3
]}

Caspell-Garcia, Chelsea ^{[3
,13
]}

Simuni, Tanya ^{[4
,15
]}

Coffey, Christopher S. ^{[3
]}

Galasko, Douglas ^{[5
,18
]}

Mollenhauer, Brit ^{[6
,7
]}

Arnedo, Vanessa ^{[8
]}

Daegele, Nichole ^{[9
]}

Frasier, Mark ^{[8
]}

Tanner, Caroline ^{[10
,14
]}

Kieburtz, Karl ^{[7
,11
]}

Marek, Kenneth ^{[9
]}

Seibyl, John ^{[9
]}

Coffey, Christopher ^{[13
]}

Tosun-Turgut, Duygu ^{[14
]}

Shaw, Leslie

Trojanowski, John ^{[12
]}

Singleton, Andrew ^{[16
]}

Toga, Arthur ^{[17
]}

Chahine, Lana ^{[19
]}

Poewe, Werner ^{[20
]}

Foroud, Tatiana ^{[21
]}

Poston, Kathleen ^{[22
]}

Sherer, Todd

Chowdhury, Sohini

Kopil, Catherine

Casaceli, Cynthia ^{[23
,24
]}

Dorsey, Ray ^{[23
,24
]}

Wilson, Renee ^{[23
,24
]}

Mahes, Sugi ^{[23
,24
]}

Salerno, Christina ^{[9
]}

Crawford, Karen ^{[17
]}

Casalin, Paola ^{[25
]}

Malferrari, Giulia ^{[25
]}

Weisz, Mali Gani ^{[26
]}

Orr-Urtreger, Avi ^{[26
]}

Montine, Thomas ^{[22
]}

Russell, David ^{[9
]}

Dahodwala, Nabila ^{[12
]}

Giladi, Nir ^{[26
]}

Factor, Stewart ^{[27
]}

Hogarth, Penelope ^{[28
]}

Standaert, David ^{[29
]}

Hauser, Robert ^{[30
]}

Jankovic, Joseph ^{[31
]}

Saint-Hilaire, Marie ^{[32
]}

Richard, Irene ^{[33
]}

机构：

[1] Univ Pittsburgh, Dept Neurol, 3471 Fifth Ave, Pittsburgh, PA 15213 USA

[2] Univ Penn, Dept Neurol, Perelman Sch Med, Philadelphia, PA 19104 USA

[3] Univ Iowa, Coll Publ Hlth, Dept Biostat, Iowa City, IA USA

[4] Northwestern Univ, Feinberg Sch Med, Dept Neurol, Chicago, IL 60611 USA

[5] Univ Calif San Diego, Dept Neurol, San Diego, CA 92103 USA

[6] Univ Med Ctr Goettingen, Dept Neurol, Gottingen, Germany

[7] Paracelsus Elena Klin, Kassel, Germany

[8] Michael J Fox Fdn, New York, NY USA

[9] Inst Neurodegenerat Disorders, New Haven, CT USA

[10] Univ Calif San Francisco, Dept Neurol, San Francisco, CA USA

[11] Univ Rochester, Med Ctr, Dept Neurol, Rochester, NY 14642 USA

[12] Univ Penn, Philadelphia, PA 19104 USA

[13] Univ Iowa, Iowa City, IA USA

[14] Univ Calif San Francisco, San Francisco, CA 94143 USA

[15] Northwestern Univ, Chicago, IL 60611 USA

[16] NIA, NIH, Bethesda, MD 20892 USA

[17] Univ Southern Calif, Lab Neuroimaging LONI, Los Angeles, CA USA

[18] Univ Calif San Diego, San Diego, CA 92103 USA

[19] Univ Pittsburgh, Pittsburgh, PA 15213 USA

[20] Innsbruck Med Univ, Innsbruck, Austria

[21] Indiana Univ, Indianapolis, IN 46204 USA

[22] Stanford Univ, Stanford, CA 94305 USA

[23] Michael J Fox Fdn Parkinsons Res, New York, NY USA

[24] Univ Rochester, Clin Trials Coordinat Ctr, Rochester, NY USA

[25] BioRep, Milan, Italy

[26] Tel Aviv Med Ctr & Sch Med, Tel Aviv, Israel

[27] Emory Univ Med, Atlanta, GA USA

[28] Oregon Hlth & Sci Univ, Portland, OR 97201 USA

[29] Univ Alabama Birmingham, Birmingham, AL USA

[30] Univ S Florida, Tampa, FL USA

[31] Baylor Coll Med, Houston, TX 77030 USA

[32] Boston Univ, Boston, MA 02215 USA

[33] Univ Rochester, Rochester, NY USA

[34] Banner Res Inst, Sun City, AZ USA

[35] Cleveland Clin, Cleveland, OH 44106 USA

[36] Univ Tubingen, Tubingen, Germany

[37] Johns Hopkins Univ, Baltimore, MD USA

[38] Univ Salerno, Salerno, Italy

[39] Univ Cincinnati, Cincinnati, OH USA

[40] Macquarie Univ, Sydney, NSW, Australia

[41] Columbia Univ, New York, NY USA

[42] Parkinsons Inst, Sunnyvale, CA USA

[43] Beth Israel Deaconess Med Ctr, New York, NY 10003 USA

[44] Univ Washington, Seattle, WA 98195 USA

[45] Parkinsons Dis & Movement Disorders Ctr Boca Rato, Boca Raton, FL USA

[46] Hosp Pitie Salpetriere, Paris, France

[47] Hosp Donostia, San Sebastian, Spain

[48] Hosp Clin Barcelona, Barcelona, Spain

[49] Imperial Coll London, London, England

[50] Kings Coll London, London, England

来源：

JOURNAL OF PARKINSONS DISEASE | 2019年 / 9卷 / 04期

基金：

美国国家卫生研究院;

关键词：

Parkinson's disease; biomarkers; disease progression; surrogate endpoint; SLEEP BEHAVIOR DISORDER; PROGNOSTIC-FACTORS; RATING-SCALE; MOTOR PROGRESSION; IDENTIFICATION; BOOTSTRAP; SPECT; TAU;

D O I：

10.3233/JPD-181518

中图分类号：

Q189 [神经科学];

学科分类号：

071006 ;

摘要：

Background: Improved prediction of Parkinson's disease (PD) progression is needed to support clinical decision-making and to accelerate research trials. Objectives: To examine whether baseline measures and their 1-year change predict longer-term progression in early PD. Methods: Parkinson's Progression Markers Initiative study data were used. Participants had disease duration <= 2 years, abnormal dopamine transporter (DAT) imaging, and were untreated with PD medications. Baseline and 1-year change in clinical, cerebrospinal fluid (CSF), and imaging measures were evaluated as candidate predictors of longer-term (up to 5 years) change in Movement Disorders Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) score and DAT specific binding ratios (SBR) using linear mixed-effects models. Results: Among 413 PD participants, median follow-up was 5 years. Change in MDS-UPDRS from year-2 to last follow-up was associated with disease duration (beta = 0.351; 95%CI = 0.146, 0.555), male gender (beta = 3.090; 95%CI = 0.310, 5.869), and baseline (beta = -0.199; 95%CI = -0.315, -0.082) and 1-year change (beta = 0.540; 95%CI = 0.423, 0.658) in MDS-UPDRS; predictors in the model accounted for 17.6% of the variance in outcome. Predictors of percent change in mean SBR from year-2 to last follow-up included baseline rapid eye movement sleep behavior disorder score (beta = -0.6229; 95%CI = -1.2910, 0.0452), baseline (beta = 7.232; 95%CI = 2.268, 12.195) and 1-year change (beta = 45.918; 95%CI = 35.994,55.843) in mean striatum SBR, and 1-year change in autonomic symptom score (beta = -0.325;95%CI = -0.695, 0.045); predictors in the model accounted for 44.1% of the variance. Conclusions: Baseline clinical, CSF, and imaging measures in early PD predicted change in MDS-UPDRS and dopamine-transporter binding, but the predictive value of the models was low. Adding the short-term change of possible predictors improved the predictive value, especially for modeling change in dopamine-transporter binding.

引用

页码：665 / 679

页数：15

共 29 条

[1] Confirmatory double-blind, parallel-group, placebo-controlled study of efficacy and safety of edaravone (MCI-186) in amyotrophic lateral sclerosis patients [J].

Abe, Koji ;

Itoyama, Yasuto ;

Sobue, Gen ;

Tsuji, Shoji ;

Aoki, Masashi ;

Doyu, Manabu ;

Hamada, Chikuma ;

Kondo, Kazuoki ;

Yoneoka, Takatomo ;

Akimoto, Makoto ;

Yoshino, Hiide .

AMYOTROPHIC LATERAL SCLEROSIS AND FRONTOTEMPORAL DEGENERATION, 2014, 15 (7-8) :610-617

[2] BOOTSTRAP INVESTIGATION OF THE STABILITY OF A COX REGRESSION-MODEL [J].

ALTMAN, DG ;

ANDERSEN, PK .

STATISTICS IN MEDICINE, 1989, 8 (07) :771-783

[3] Structural Brain Alterations Associated with Rapid Eye Movement Sleep Behavior Disorder in Parkinson's Disease [J].

Boucetta, Soufiane ;

Salimi, Ali ;

Dadar, Mahsa ;

Jones, Barbara E. ;

Collins, D. Louis ;

Thien Thanh Dang-Vu .

SCIENTIFIC REPORTS, 2016, 6

[4] THE BOOTSTRAP AND IDENTIFICATION OF PROGNOSTIC FACTORS VIA COX PROPORTIONAL HAZARDS REGRESSION-MODEL [J].

CHEN, CH ;

GEORGE, SL .

STATISTICS IN MEDICINE, 1985, 4 (01) :39-46

[5] Dopamine Transporter Neuroimaging as an Enrichment Biomarker in Early Parkinson's Disease Clinical Trials: A Disease Progression Modeling Analysis [J].

Conrado, Daniela J. ;

Nicholas, Timothy ;

Tsai, Kuenhi ;

Macha, Sreeraj ;

Sinha, Vikram ;

Stone, Julie ;

Corrigan, Brian ;

Bani, Massimo ;

Muglia, Pierandrea ;

Watson, Ian A. ;

Kern, Volker D. ;

Sheveleva, Elena ;

Marek, Kenneth ;

Stephenson, Diane T. ;

Romero, Klaus .

CTS-CLINICAL AND TRANSLATIONAL SCIENCE, 2018, 11 (01) :63-70

[6] Clinical criteria for subtyping Parkinson's disease: biomarkers and longitudinal progression [J].

Fereshtehnejad, Seyed-Mohammad ;

Zeighami, Yashar ;

Dagher, Alain ;

Postuma, Ronald B. .

BRAIN, 2017, 140 :1959-1976

[7] Movement disorder society-sponsored revision of the unified Parkinson's disease rating scale (MDS-UPDRS): Process, format, and clinimetric testing plan [J].

Goetz, Christopher G. ;

Fahn, Stanley ;

Martinez-Martin, Pablo ;

Poewe, Werner ;

Sampaio, Cristina ;

Stebbins, Glenn T. ;

Stern, Matthew B. ;

Tilley, Barbara C. ;

Dodel, Richard ;

Dubois, Bruno ;

Holloway, Robert ;

Jankovic, Joseph ;

Kulisevsky, Jaime ;

Lang, Anthony E. ;

Lees, Andrew ;

Leurgans, Sue ;

LeWitt, Peter A. ;

Nyenhuis, David ;

Olanow, C. Warren ;

Rascol, Olivier ;

Schrag, Anette ;

Teresi, Jeanne A. ;

van Hilten, Jacobus J. ;

LaPelle, Nancy .

MOVEMENT DISORDERS, 2007, 22 (01) :41-47

[8] CSF tau and β-amyloid predict cerebral synucleinopathy in autopsied Lewy body disorders [J].

Irwin, David J. ;

Xie, Sharon X. ;

Coughlin, David ;

Nevler, Naomi ;

Akhtar, Rizwan S. ;

McMillan, Corey T. ;

Lee, Edward B. ;

Wolk, David A. ;

Weintraub, Daniel ;

Chen-Plotkin, Alice ;

Duda, John E. ;

Spindler, Meredith ;

Siderowf, Andrew ;

Hurtig, Howard, I ;

Shaw, Leslie M. ;

Grossman, Murray ;

Trojanowski, John Q. .

NEUROLOGY, 2018, 90 (12) :E1038-E1046

[9] Association of Cerebrospinal Fluid β-Amyloid 1-42, T-tau, P-tau181, and α-Synuclein Levels With Clinical Features of Drug-Naive Patients With Early Parkinson Disease [J].

Kang, Ju-Hee ;

Irwin, David J. ;

Chen-Plotkin, Alice S. ;

Siderowf, Andrew ;

Caspell, Chelsea ;

Coffey, Christopher S. ;

Waligorska, Teresa ;

Taylor, Pegg ;

Pan, Sarah ;

Frasier, Mark ;

Marek, Kenneth ;

Kieburtz, Karl ;

Jennings, Danna ;

Simuni, Tanya ;

Tanner, Caroline M. ;

Singleton, Andrew ;

Toga, Arthur W. ;

Chowdhury, Sohini ;

Mollenhauer, Brit ;

Trojanowski, John Q. ;

Shaw, Leslie M. .

JAMA NEUROLOGY, 2013, 70 (10) :1277-1287

[10] Large-scale identification of clinical and genetic predictors of motor progression in patients with newly diagnosed Parkinson's disease: a longitudinal cohort study and validation [J].

Latourelle, Jeanne C. ;

Beste, Michael T. ;

Hadzi, Tiffany C. ;

Miller, Robert E. ;

Oppenheim, Jacob N. ;

Valko, Matthew P. ;

Wuest, Diane M. ;

Church, Bruce W. ;

Khalil, Iya G. ;

Hayete, Boris ;

Venuto, Charles S. .

LANCET NEUROLOGY, 2017, 16 (11) :908-916

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