English
Background of the study
Both Continuous intrajejunal Levodopa Infusion (CLI) and Deep Brain Stimulation (DBS) are accepted therapies for the treatment of advanced Parkinson’s disease (PD). Neurologists and patients tend to prefer the more expensive CLI although a scientific rationale is lacking. To determine the optimal treatment in advanced PD, a comparative study of CLI and DBS is warranted.
Objective of the study
The objective of the study is to realize an efficient allocation of resources to the available treatment options in advanced PD, while guaranteeing the highest standard of care.
Secondary objectives are
- to quantify the need for and cost of healthcare and the health benefits in both groups
- to assess the patients motor and non-motor symptoms, quality of life and daily functioning in both DBS and CLI
- to register and compare adverse effects and complications in both groups and verify patients’ and treating physicians’ conceptions about the different treatments
Study design
Prospective, randomized, open label multicenter trial, with two additional patient preference treatment arms (“patient preference randomized trial”).
Study population
Patients with PD who, despite optimal pharmacological treatment, have severe response fluctuations, dyskinesias, painful dystonia, or bradykinesia. A total of 66 patients will be randomized, aim is to include 180 patients will in the patient preference arms.
Intervention
Patients will be randomized to CLI or DBS. For CLI treatment, a tube will be placed in the jejunum via a percutaneous endoscopic gastrostomy (PEG). This tube is connected to an external pump that delivers the levodopa-gel. For DBS treatment, 2 electrodes will be implanted in the brain. The electrodes are connected to an implanted pulse generator, which will be placed subcutaneously in the subclavian area.
Primary study parameters
The primary health economic outcomes of the randomized trial are the costs per unit on the PDQ-39 and the costs per QALY for the cost-effectiveness and cost-utility analyses respectively.
Secundary study parameters
Secondary clinical outcomes are quality of life (PDQ-39), PD motor symptoms (UPDRS), dyskinesias (CDRS), 3-day motor symptom diary), adverse effects and complications, treatment failure, non-motor symptoms such as autonomic functions and sleep (Non Motor Symptom Checklist, Rotterdam Symptom Checklist), PD-medication, disability, functional health status (ALDS), patient and physician preferences, patient satisfaction, caregiver burden, neuropsychological and psychiatric assessment, stopping treatment, starting with the alternative than initially started treatment, and medical and non-medical care costs (iMCQ, iPCQ).