Constructing a Personalized Treatment Rule for Initial Therapy in Early Parkinson's Disease.
Constructing a Personalized Treatment Rule for Initial Therapy in Early Parkinson's Disease.
Where did the research take place?
The study site has not been established. Author addresses may differ from where the research occurred.
New York City, US · Author affiliation
Department of Biostatistics and Computational Biology, University of Rochester, Rochester, New York, USA.Location evidence
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Original abstract
BackgroundDopaminergic therapies such as levodopa and dopamine receptor agonists (DRA) improve motor function in people with Parkinson's disease. These therapies are also linked to the advent of motor complications such as dyskinesias and wearing-off episodes.ObjectivesWe illustrate a method that creates a personalized treatment rule that takes patient-specific information and provides a recommended first-line therapy for Parkinson's disease that will provide the best mean improvement in motor function while constraining the probability of a motor complication within the first 2 years of therapy below a level mutually deemed to be the maximum acceptable risk by the patient and clinician.MethodsWe apply a machine learning technique that simultaneously optimizes for benefit and risk outcomes to a harmonized clinical dataset based on the CALM-PD and STEADY-PD III randomized clinical trials. This generates a decision rule for allocating patients to levodopa or a DRA, based on a specified risk threshold. We evaluate the individualized decision rule by comparing the mean benefit and risk outcomes under the decision rule to the mean outcomes from policies that assign all patients to either levodopa or a DRA.ResultsThe optimal decision rule improves the mean change from baseline in MDS-UPDRS (Movement Disorder Society Unified Parkinson's Disease Rating Scale) motor (Part 3) score compared to assigning all patients to a DRA and provides a smaller mean probability of motor complications than assigning all patients to levodopa. More data are required to further develop and validate this decision rule.ConclusionsAn optimal decision rule can provide improved data adaptive treatment decisions that balance benefit and risk outcomes given a maximum acceptable risk.