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Application of longitudinal item response theory models to modeling Parkinson's disease progression.

Publication ,  Journal Article
Zou, H; Aggarwal, V; Stebbins, GT; Müller, MLTM; Cedarbaum, JM; Pedata, A; Stephenson, D; Simuni, T; Luo, S
Published in: CPT Pharmacometrics Syst Pharmacol
October 2022

The Movement Disorder Society revised version of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS) parts 2 and 3 reflect patient-reported functional impact and clinician-reported severity of motor signs of Parkinson's disease (PD), respectively. Total scores are common clinical outcomes but may obscure important time-based changes in items. We aim to analyze longitudinal disease progression based on MDS-UPRDS parts 2 and 3 item-level responses over time and as functions of Hoehn & Yahr (H&Y) stages 1 and 2 for subjects with early PD. The longitudinal item response theory (IRT) modeling is a novel statistical method addressing limitations in traditional linear regression approaches, such as ignoring varying item sensitivities and the sum score balancing out improvements and declines. We utilized a harmonized dataset consisting of six studies with 3573 subjects with early PD and 14,904 visits, and mean follow-up time of 2.5 years (±1.57). We applied both a unidimensional (each part separately) and multidimensional (both parts combined) longitudinal IRT models. We assessed the progression rates for both parts, anchored to baseline H&Y stages 1 and 2. Both the uni- and multidimensional longitudinal IRT models indicate significant worsening time effects in both parts 2 and 3. Baseline H&Y stage 2 was associated with significantly higher baseline severities, but slower progression rates in both parts, as compared with stage 1. Patients with baseline H&Y stage 1 demonstrated slower progression in part 2 severity compared to part 3, whereas patients with baseline H&Y stage 2 progressed faster in part 2 than part 3. The multidimensional model had a superior fit compared to the unidimensional models and it had excellent model performance.

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Published In

CPT Pharmacometrics Syst Pharmacol

DOI

EISSN

2163-8306

Publication Date

October 2022

Volume

11

Issue

10

Start / End Page

1382 / 1392

Location

United States

Related Subject Headings

  • Severity of Illness Index
  • Parkinson Disease
  • Mental Status and Dementia Tests
  • Humans
  • Disease Progression
  • 3214 Pharmacology and pharmaceutical sciences
  • 3208 Medical physiology
  • 1116 Medical Physiology
  • 1115 Pharmacology and Pharmaceutical Sciences
 

Citation

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Zou, H., Aggarwal, V., Stebbins, G. T., Müller, M. L. T. M., Cedarbaum, J. M., Pedata, A., … Luo, S. (2022). Application of longitudinal item response theory models to modeling Parkinson's disease progression. CPT Pharmacometrics Syst Pharmacol, 11(10), 1382–1392. https://doi.org/10.1002/psp4.12853
Zou, Haotian, Varun Aggarwal, Glenn T. Stebbins, Martijn L. T. M. Müller, Jesse M. Cedarbaum, Anne Pedata, Diane Stephenson, Tanya Simuni, and Sheng Luo. “Application of longitudinal item response theory models to modeling Parkinson's disease progression.CPT Pharmacometrics Syst Pharmacol 11, no. 10 (October 2022): 1382–92. https://doi.org/10.1002/psp4.12853.
Zou H, Aggarwal V, Stebbins GT, Müller MLTM, Cedarbaum JM, Pedata A, et al. Application of longitudinal item response theory models to modeling Parkinson's disease progression. CPT Pharmacometrics Syst Pharmacol. 2022 Oct;11(10):1382–92.
Zou, Haotian, et al. “Application of longitudinal item response theory models to modeling Parkinson's disease progression.CPT Pharmacometrics Syst Pharmacol, vol. 11, no. 10, Oct. 2022, pp. 1382–92. Pubmed, doi:10.1002/psp4.12853.
Zou H, Aggarwal V, Stebbins GT, Müller MLTM, Cedarbaum JM, Pedata A, Stephenson D, Simuni T, Luo S. Application of longitudinal item response theory models to modeling Parkinson's disease progression. CPT Pharmacometrics Syst Pharmacol. 2022 Oct;11(10):1382–1392.
Journal cover image

Published In

CPT Pharmacometrics Syst Pharmacol

DOI

EISSN

2163-8306

Publication Date

October 2022

Volume

11

Issue

10

Start / End Page

1382 / 1392

Location

United States

Related Subject Headings

  • Severity of Illness Index
  • Parkinson Disease
  • Mental Status and Dementia Tests
  • Humans
  • Disease Progression
  • 3214 Pharmacology and pharmaceutical sciences
  • 3208 Medical physiology
  • 1116 Medical Physiology
  • 1115 Pharmacology and Pharmaceutical Sciences