Objective
The purposes of this protocol are:
To provide DBS therapy and follow-up management
To maintain a cohort of patients treated with DBS who can participate in other NIH protocols addressing the efficacy of functional surgery and the relevant physiology.
To collect physiology, programming, and efficacy data related to DBS therapy and motor and cognitive function in these patient populations. All the data collected will be an outcome of standard of care and all analyses will be retrospective.
All treatment under this protocol will be based on the current standard of care for DBS therapy. All tests and study procedures will be administered per standard and routine clinical care; however, the schedule of procedures has been standardized for research purposes. Subjects may be enrolled in the study to support participation in other DBS protocols.
Study Population
Patients 18 years and older with medically refractory PD, dystonia, and/or ET may participate in this study. Other indications will be added with subsequent amendments if FDA approval of deep brain stimulation is extended to other conditions.
Study Design
The treatment that is rendered in this protocol is standard of care for PD, dystonia, and ET. Patients confirmed to have medically refractory PD, dystonia or ET will be offered DBS as a therapeutic option per standard of care procedures and routine clinical care. Patients will be evaluated for their eligibility for the procedure and the risk/benefit balance for surgical therapy will be assessed (Lang et al., 2006). After completing the evaluation, a decision will be made on recommending the procedure. At that point the patients will be referred for the surgical intervention to the NIH Surgical Neurology Branch or to collaborating surgeons in the community. If the surgery is performed at the NIH, the Neurology DBS team can be involved in surgical planning, target selection, intraoperative physiology recording and testing, as specified under SNB protocols. After the surgery, the patients will be followed in the NIH DBS clinic and the DBS programming will be initiated and performed as outlined below. The patients will be followed up for at least two years, and then they will have the option to transfer their care back to the neurologists in the community or continue care with the NIH Neurology team until care in the community is available.
In addition, patients can be enrolled in the protocol at various points in relation to DBS surgery.
Data regarding the movement disorder of the subject and observations of their standard of care treatment will be collected. Data may be used for future research questions that are related to subjects movement disorder and/or treatment.
Outcome Measures
1. To evaluate effects of DBS before and after surgery using clinically-generated data on:
1. Severity of PD motor symptoms (measured by the UPDRS III scale)
2. Changes in dystonia severity (measured by the Burke-Fahn-Marsden (BFM) dystonia rating scale (Burke et al., 1985)
3. Changes in tremor severity (measured by the Tremor Rating Scale (TRS))
4. Levels of effective drug therapy for PD patients using the Levodopa Equivalent Drug Dosing (LEDD)
5. Changes in behavior, performance of activities of daily living and complications of therapy as measured by the UPDRS I, II, and IV scales
2. To evaluate effects of DBS before and after surgery on quality of life:
1. For PD patients (measured by UPDRS part II and other scales such as the PDQ-39)
2. For the Dystonia and ET patients (measured by the SF-12 scale)
3. To evaluate radiographic correlation of DBS electrode position and clinical changes
4. To evaluate neurophysiological mechanisms of DBS and relevant basal ganglia physiology
All data collected will be done as standard of care and all analysis will be retrospective.
