A review of disease progression models of Parkinson's disease and applications in clinical trials

Movement Disorders

Volumn 31, Issue 7, 2016, Pages 947-956

  Venuto, Charles S ^a   Potter, Nicholas B ^a   Ray Dorsey, E ^a   Kieburtz, Karl ^a  

^a UNIVERSITY OF ROCHESTER MEDICAL CENTER   (United States)

Author keywords

disease model; disease progression; Parkinson's disease; pharmacometrics; UPDRS

Indexed keywords

DISEASE COURSE; DISEASE SIMULATION; HUMAN; PARKINSON DISEASE; PRIORITY JOURNAL; REVIEW; TIME; UNIFIED PARKINSON DISEASE RATING SCALE;

EID: 84977556275     PISSN: 08853185     EISSN: 15318257     Source Type: Journal    
DOI: 10.1002/mds.26644     Document Type: Review

References (64)

1. National Institute of Neurological Disorders and Stroke. Parkinson's disease 2014 advancing research improving lives conference recommendations report to the National Advisory Neurological Disorders and Stroke Council. January 30, 2014. Available at: http://www.ninds.nih.gov/research/parkinsonsweb/PD2014/.
2. Mould DR, Denman NG, Dufull S. Using disease progression models as a tool to detect drug effect. Clin Pharmacol Ther 2007;82(1):81-86.
3. Samtani MN, Raghavan N, Shi Y, et al; Alzheimer's Disease Neuroimaging Initiative. Disease progression model in subjects with mild cognitive impairment from the Alzheimer's disease neuroimaging initiative: CSF biomarkers predict population subtypes. Br J Clin Pharmacol 2013;75(1):146-161.
4. Passey C, Kimko H, Nandy P, Kagan L. Osteoarthritis disease progression model using six year follow-up data from the osteoarthritis initiative. J Clin Pharmacol 2015; 55(3): 269–278.
5. Atchison TB, Massman PJ, Doody RS. Baseline cognitive function predicts rate of decline in basic-care abilities of individuals with dementia of the Alzheimer's type. Arch Clin Neuropsychol 2007;22(1):99-107.
6. Food and Drug Administration Center for Drug Evaluation Research (2012). Guidance for Industry: Enrichment strategies for clinical trials to support approval of human drugs and biological products. Silver Spring, MD.
7. Milligan PA, Brown MJ, Marchant B, et al. Model-based drug development: a rational approach to efficiently accelerate drug development. Clin Pharmacol Ther 2013;93(6):502-514.
8. Romero K, Ito K, Rogers JA, et al. Alzheimer's Disease Neuroimaging Initiative; Coalition Against Major Diseases. The future is now: model-based clinical trial design for Alzheimer's disease. Clin Pharmacol Ther 2015;97(3):210-214.
9. Holford NH, Peace KE. Methodologic aspects of a population pharmacodynamic model for cognitive effects in Alzheimer patients treated with tacrine. Proc Natl Acad Sci U S A 1992;89(23):11466-11470.
10. Ito K, Ahadieh S, Corrigan B, French J, Fullerton T, Tensfeldt T; Alzheimer's Disease Working Group. Disease progression meta-analysis model in Alzheimer's disease. Alzheimers Dement 2010;6(1):39-53.
11. Samtani MN, Farnum M, Lobanov V, et al; Alzheimer's Disease Neuroimaging Initiative. An improved model for disease progression in patients from the Alzheimer's disease neuroimaging initiative. J Clin Pharmacol 2012;52(5):629-644.
12. Perelson AS, Nelson PW. Mathematical analysis of HIV-I dynamics in vivo. SIAM Rev 1999;41(1):3-44.
13. Perelson AS. Modelling viral and immune system dynamics. Nat Rev Immunol 2002;2(1):28-36. Review.
14. Chatterjee A, Guedj J, Perelson AS. Mathematical modelling of HCV infection: what can it teach us in the era of direct-acting antiviral agents? Antivir Ther 2012;17(6 Pt B):1171-1182.
15. Dixit NM, Layden-Almer JE, Layden TJ, Perelson AS. Modelling how ribavirin improves interferon response rates in hepatitis C virus infection. Nature 2004;432(7019):922-924.
16. Richards M, Marder K, Cote L, Mayeux R. Interrater reliability of the unified Parkinson's disease rating scale motor examination. Mov Disord 2004;9(1):89-91.
17. Martinez-Martin P, Gil-Nagel A, Gracia M, Gomez JB, Martinez-Sarries J, Bermejo F. Unified Parkinson's disease rating scale characteristics and structure. Mov Disord 2004;9(1):76-82.
18. Guimaraes P, Kieburtz K, Goetz CG, et al. Non-linearity of Parkinson's disease progression: implications for sample size calculations in clinical trials. Clin Trials 2005;2(6):509-518.
19. Parkinson Study Group. Pramipexole vs levodopa as initial treatment for Parkinson disease. JAMA 2000;284(15):1931-1938.
20. Korczyn AD, Brunt ER, Larsen JP, Nagy Z, Poewe WH, Ruggieri S. A 3-year randomized trial of ropinirole and bromocriptine in early Parkinson's disease. The 053 Study Group. Neurology 1999;53(2):364-370.
21. Maetzler W, Liepelt I, Berg D. Progression of Parkinson's disease in the clinical phase: potential markers. Lancet Neurol 2009;8(12):1158-1171.
22. Lopez IC, Ruiz PJ, Del Pozo SV, Bernardos VS. Motor complications in Parkinson's disease: ten year follow-up study. Mov Disord 2010;25:2735-2739.
23. DATATOP: a multicenter controlled clinical trial in early Parkinson's disease. Parkinson Study Group. Arch Neurol. 1989;46(10):1052-1060.
24. Shoulson I. DATATOP: a decade of neuroprotective inquiry. Parkinson Study Group. Deprenyl and Tocopherol Antioxidative Therapy of Parkinsonism. Ann Neurol 1998;44(3 Suppl 1):S160-S166.
25. Pålhagen S, Heinonen E, Hägglund J, Kaugesaar T, Mäki-Ikola O, Palm R; Swedish Parkinson Study Group. Selegiline slows the progression of the symptoms of Parkinson disease. Neurology 2006;66(8):1200-1206.
26. Elm JJ; NINDS NET-PD Investigators. Design innovations and baseline findings in a long-term Parkinson's trial: the National Institute of Neurological Disorders and Stroke Exploratory Trials in Parkinson's Disease Long-Term Study-1. Mov Disord 2012;27(12):1513-1521.
27. Holford NH, Chan PL, Nutt JG, Kieburtz K, Shoulson I; Parkinson Study Group. Disease progression and pharmacodynamics in Parkinson disease - evidence for functional protection with levodopa and other treatments. J Pharmacokinet Pharmacodyn 2006;33(3):281-311.
28. Parkinson Study Group. Effects of tocopherol and deprenyl on the progression of disability in early Parkinson's disease. N Engl J Med 1993;328(3):176-183
29. Effect of deprenyl on the progression of disability in early Parkinson's disease. The Parkinson Study Group. N Engl J Med 1989;321(20):1364-1371.
30. Parkinson Study Group. Impact of deprenyl and tocopherol treatment on PD in DATATOP subjects not requiring levodopa. Ann Neurol 1996;39:9-36.
31. Parkinson Study Group. Impact of deprenyl and tocopherol treatment on PD in DATATOP subjects requiring levodopa. Ann Neurol 1996;39:37-45.
32. Shoulson I, Oakes D, Fahn S, et al. Impact of sustained deprenyl (selegiline) in levodopa-treated Parkinson's disease: a randomized placebo-controlled extension of the deprenyl and tocopherol antioxidative therapy of parkinsonism trial. Ann Neurol 2002;51(5):604-612.
33. Forys U, Marciniak-Czochra A. Logistic equations in tumour growth modelling. Int J Appl Math Comput Sci 2003;13(3):317-325.
34. Rogers JA, Polhamus D, Gillespie WR, et al. Combining patient-level and summary-level data for Alzheimer's disease modeling and simulation: a β regression meta-analysis. J Pharmacokinet Pharmacodyn 2012;39(5):479-498.
35. Sun W, Laughren TP, Zhu H, Hochhaus G, Wang Y. Development of a placebo effect model combined with a dropout model for bipolar disorder. J Pharmacokinet Pharmacodyn 2013;40(3):359-368.
36. Björnsson MA, Friberg LE, Simonsson US. Performance of nonlinear mixed effects models in the presence of informative dropout. AAPS J 2015;17(1):245-255.
37. Olanow CW. Levodopa: effect on cell death and the natural history of Parkinson's disease. Mov Disord 2015;30(1):37-44.
38. Bhattaram VA, Siddiqui O, Kapcala LP, Gobburu JV. Endpoints and analyses to discern disease-modifying drug effects in early Parkinson's disease. AAPS J 2009;11(3):456-464.
39. Lee JY, Gobburu JV. Bayesian quantitative disease-drug-trial models for Parkinson's disease to guide early drug development. AAPS J 2011;13(4):508-518.
40. Goetz CG, Tanner CM, Shannon KM. Progression of Parkinson's disease without levodopa. Neurology 1987;37(4):695-698.
41. Poewe WH, Wenning GK. The natural history of Parkinson's disease. Ann Neurol 1998;44:S1-S9.
42. Goetz CG, Stebbins GT, Blasucci LM. Differential progression of motor impairment in levodopa-treated Parkinson's disease. Mov Disord 2000;15:479-484.
43. Alves G, Wentzel-Larsen T, Aarsland D, Larsen JP. Progression of motor impairment and disability in Parkinson's disease: a population based-study. Neurology 2005;65(9):1436-1441.
44. Schrag A, Dodel R, Spottke A, Bornschein B, Siebert U, Quinn NP. Rate of clinical progression in Parkinson's disease. A prospective study. Mov Disord 2007;22(7):938-945.
45. Fahn S, Oakes D, Shoulson I, et al; Parkinson Study Group. Levodopa and the progression of Parkinson's disease. N Engl J Med 2004;351(24):2498-2508.
46. Parkinson Study Group. A controlled trial of rasagiline in early Parkinson disease: the TEMPO Study. Arch Neurol 2002;59(12):1937-1943.
47. O'Hagan A. The Bayesian approach to statistics. In: Rudas, T. Handbook of Probability: Theory and Applications. Thousand Oaks, CA: SAGE Publications, Inc.; 2008.
48. Reinoso G, Allen JC Jr, Au WL, Seah SH, Tay KY, Tan LC. Clinical evolution of Parkinson's disease and prognostic factors affecting motor progression: 9-year follow-up study. Eur J Neurol 2015;22(3):457-463.
49. Aarsland D, Muniz G, Matthews F. Nonlinear decline of mini-mental state examination in Parkinson's disease. Mov Disord 2011;26(2):334-337.
50. Vu TC, Nutt JG, Holford NH. Progression of motor and nonmotor features of Parkinson's disease and their response to treatment. Br J Clin Pharmacol 2012;74(2):267-283.
51. Kuramoto L, Cragg J, Nandhagopal R, et al. The nature of progression in Parkinson's disease: an application of non-linear, multivariate, longitudinal random effects modelling. PLoS One 2013;8(10):e76595.
52. Nandhagopal R, Kuramoto L, Schulzer M, et al. Longitudinal progression of sporadic Parkinson's disease: a multi-tracer positron emission tomography study. Brain 2009;132(Pt 11):2970-2979.
53. Kordower JH, Olanow CW, Dodiya HB, et al. Disease duration and the integrity of the nigrostriatal system in Parkinson's disease. Brain 2013;136(Pt 8):2419-2431.
54. Woodcock, J. Biomarker Letter of Support. Silver Spring, MD: FDA: CDER, March 16, 2015.
55. Dorsey ER, Venuto C, Venkataraman V, Harris DA, Kieburtz K. Novel methods and technologies for 21st-century clinical trials: a review. JAMA Neurol 2015;72(5):582-588.
56. Holford NH, Nutt JG. Interpreting the results of Parkinson's disease clinical trials: time for a change. Mov Disord 2011;26(4):569-577.
57. Olanow CW, Rascol O, Hauser R, et al. A double-blind, delayed-start trial of rasagiline in Parkinson's disease. N Engl J Med 2009;361(13):1268-1278.
58. Chan PLS, Nutt JG, Holford NHG. Levodopa slows progression of Parkinson's disease. External validation by clinical trial simulation. Pharmaceut Res 2007;24(4):791-802.
59. Goetz CG, Leurgans S, Raman R; Parkinson Study Group. Placebo-associated improvements in motor function: comparison of subjective and objective sections of the UPDRS in early Parkinson's disease. Mov Disord 2002;17(2):283-288.
60. Goetz CG, Wuu J, McDermott MP, et al. Placebo response in Parkinson's disease: comparisons among 11 trials covering medical and surgical interventions. Mov Disord 2008;23(5):690-699.
61. Ito K, Ahadieh S, Corrigan B, French J, Fullerton T, Tensfeldt T, Alzheimer's Disease Working Group. Disease progression meta-analysis model in Alzheimer's disease. Alzheimers Dement 2010;6(1):39-53.
62. EMA. Human Medicines and Evaluation. Qualification opinion of a novel data driven model of disease progression and trial evaluation in mild and moderate Alzheimer's disease. s.l. EMA; 2013.
63. Küffner R, Zach N, Norel R, et al. Crowdsourced analysis of clinical trial data to predict amyotrophic lateral sclerosis progression. Nat Biotechnol 2015;33(1):51-7.
64. Woodcock J, Woosley R. The FDA Critical Path Initiative and its influence on new drug development. Annu Rev Med 2008;59:1-12.