• Raúl Martínez-Fernández, Jorge U Máñez-Miró, Rafael Rodríguez-Rojas, Marta Del Álamo, Binit B Shah, Frida Hernández-Fernández, José A Pineda-Pardo, Mariana H G Monje, Beatriz Fernández-Rodríguez, Scott A Sperling, David Mata-Marín, Pasqualina Guida, Fernando Alonso-Frech, Ignacio Obeso, Carmen Gasca-Salas, Lydia Vela-Desojo, W Jeffrey Elias, and José A Obeso.
• From HM Centro Integral en Neurociencias AC (CINAC), University Hospital HM Puerta del Sur, CEU San Pablo University, Mostoles (R.M.-F., J.U.M.-M., R.R.-R., M.A., F.H.-F., J.A.P.-P., M.H.G.M., B.F.-R., D.M.-M., P.G., F.A.-F., I.O., C.G.-S., L.V.-D., J.A.O.), and the Network Center for Biomedical Research on Neurodegenerative Diseases, Carlos III Institute, Madrid (R.M.-F., R.R.-R., M.A., F.H.-F., J.A.P.-P., D.M.-M., P.G., F.A.-F., I.O., C.G.-S., L.V.-D., J.A.O.) - both in Spain; and the University of Virginia Health Sciences Center, Charlottesville (B.B.S., S.A.S., W.J.E.).
• N. Engl. J. Med. 2020 Dec 24; 383 (26): 2501-2513.

BackgroundThe subthalamic nucleus is the preferred neurosurgical target for deep-brain stimulation to treat cardinal motor features of Parkinson's disease. Focused ultrasound is an imaging-guided method for creating therapeutic lesions in deep-brain structures, including the subthalamic nucleus.MethodsWe randomly assigned, in a 2:1 ratio, patients with markedly asymmetric Parkinson's disease who had motor signs not fully controlled by medication or who were ineligible for deep-brain stimulation surgery to undergo focused ultrasound subthalamotomy on the side opposite their main motor signs or a sham procedure. The primary efficacy outcome was the between-group difference in the change from baseline to 4 months in the Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) motor score (i.e., part III) for the more affected body side (range, 0 to 44, with higher scores indicating worse parkinsonism) in the off-medication state. The primary safety outcome (procedure-related complications) was assessed at 4 months.ResultsAmong 40 enrolled patients, 27 were assigned to focused ultrasound subthalamotomy (active treatment) and 13 to the sham procedure (control). The mean MDS-UPDRS III score for the more affected side decreased from 19.9 at baseline to 9.9 at 4 months in the active-treatment group (least-squares mean difference, 9.8 points; 95% confidence interval [CI], 8.6 to 11.1) and from 18.7 to 17.1 in the control group (least-squares mean difference, 1.7 points; 95% CI, 0.0 to 3.5); the between-group difference was 8.1 points (95% CI, 6.0 to 10.3; P<0.001). Adverse events in the active-treatment group were dyskinesia in the off-medication state in 6 patients and in the on-medication state in 6, which persisted in 3 and 1, respectively, at 4 months; weakness on the treated side in 5 patients, which persisted in 2 at 4 months; speech disturbance in 15 patients, which persisted in 3 at 4 months; facial weakness in 3 patients, which persisted in 1 at 4 months; and gait disturbance in 13 patients, which persisted in 2 at 4 months. In 6 patients in the active-treatment group, some of these deficits were present at 12 months.ConclusionsFocused ultrasound subthalamotomy in one hemisphere improved motor features of Parkinson's disease in selected patients with asymmetric signs. Adverse events included speech and gait disturbances, weakness on the treated side, and dyskinesia. (Funded by Insightec and others; ClinicalTrials.gov number, NCT03454425.).Copyright © 2020 Massachusetts Medical Society.

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