Unilateral focused ultrasound subthalamotomy in early Parkinson's disease: a pilot study.

BACKGROUND

Unilateral focused ultrasound subthalamotomy (FUS-STN) improves motor features of Parkinson's disease (PD) in moderately advanced patients. The less invasive nature of FUS makes its early application in PD feasible. We aim to assess the safety and efficacy of unilateral FUS-STN in patients with PD of less than 5 years from diagnosis (early PD).

METHODS

Prospective, open-label study. Eligible patients with early PD had highly asymmetrical cardinal features. The primary outcome was safety, defined as treatment-related adverse events at 6 months. Secondary outcomes included efficacy, assessed as motor improvement in the Movement Disorders Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS), motor fluctuations, non-motor symptoms, daily living activities, quality of life, medication and patients' impression of change.

RESULTS

Twelve patients with PD (median age 52.0 (IQR 49.8-55.3) years, median time from diagnosis 3.0 (2.1-3.9) years) underwent unilateral FUS-STN. Within 2 weeks after treatment, five patients developed dyskinesia on the treated side, all resolved after levodopa dose adjustment. One patient developed mild contralateral motor weakness which fully resolved in 4 weeks. One patient developed dystonic foot and another hand and foot dystonia. The latter impaired gait and became functionally disabling initially. Both cases were well controlled with botulinum toxin injections. The off-medication motor MDS-UPDRS score for the treated side improved at 12 months by 68.7% (from 14.5 to 4.0, p=0.002), and the total motor MDS-UPDRS improved by 49.0% (from 26.5 to 13.0, p=0.002). Eleven patients (92%) reported global improvement 12 months after treatment.

CONCLUSION

Unilateral FUS-STN may be safe and effective to treat motor manifestations in patients with early PD. A larger confirmatory trial is warranted.

TRIAL REGISTRATION NUMBER

NCT04692116.

Informations de copyright

© Author(s) (or their employer(s)) 2023. No commercial re-use. See rights and permissions. Published by BMJ.

Déclaration de conflit d'intérêts

Competing interests: RMF has received speaker honoraria from Insightec, Bial, Zambon and Boston Scientific, and reimbursement of travel expenses to attend scientific conferences from Insightec and Bial. ENV was supported in 2021 by a fellowship from the Movement Disorders Group of the Spanish Neurology Society (Sociedad Española de Neurología) granted by Zambon and has received honoraria for lectures from Zambon and Palex. RRR has received speaker honoraria from Insightec and Zambon. MdA has received speaker honoraria from Insightec and Boston Scientific and reimbursement of travel expenses to attend scientific conferences from Boston Scientific and Medtronic. JAPP declares no conflicts of interest nor additional disclosures to report. DMM declares no conflicts of interest nor additional disclosures to report. PG declares no conflicts of interest nor additional disclosures to report. TJC has received speaker honoraria from Insightec and Zambon. DPB declares no conflicts of interest nor additional disclosures to report. AD declares no conflicts of interest nor additional disclosures to report. JUMM has received speaker honoraria from Insightec, Bial, Zambon, UCB Pharma, Lundbeck and Italfarmaco, and reimbursement of travel expenses to attend scientific conferences from Insightec and Bial. CGS has received speaker honoraria from Exeltis, Esteve and Fundación ACE, and a grant from Asociacion Madrileña de Neurologia, funded by Bial. MM has received speaker honoraria from Teva Pharmaceutical Industries and Novartis and reimbursement of travel expenses to attend scientific conferences from Lundbeck and Cerevel Therapeutics. JAO has received honoraria for lecturing and reimbursement of travel expenses to attend scientific meetings by Insightec.