The role of pallidotomy in the precision medicine era

The use of radiofrequency pallidotomy (RP) for medically refractory dystonia has markedly declined since the introduction of deep brain stimulation (DBS). Conditions such as severe cognitive impairment, poor nutritional status, or acquired dystonia-common in children with severe dystonia-may limit DBS eligibility or benefit. In these cases, RP may represent a valuable alternative. Evidence on RP in this population remains scarce, mostly based on small case-series with limited follow-up. We retrospectively analyzed RP safety, feasibility and long-term outcomes in children with medically refractory dystonia.

Records of patients who underwent RP at Bambino Gesù Children's Hospital were reviewed. Outcomes were retrospectively assessed through the Clinical Global Impression-Improvement scale. For patients with a history of status dystonicus (SD), recurrence was used as outcome measure.

Eighteen patients underwent 21 procedures. Sixteen received bilateral simultaneous pallidotomy; two unilateral pallidotomy after GPi-DBS removal due to infection. Three patients required repeated surgery for recurrent-SD. Ten had acquired dystonia (cerebral palsy, CP), five genetically confirmed dystonia, three idiopathic dystonia. Three were followed for three months or less; in the others, mean follow-up was 6.62 ± 3.65 years. Three months postoperatively, 12 of 16 patients improved (1 "very much improved," 2 "much improved," 9 "minimally improved"), 2 were unchanged and 2 minimally worsened. At last follow-up (15 patients), 1 remained "very much improved," 2 "minimally improved," 3 "unchanged," 6"minimally worsened," 3 "much worsened." Among six patients treated for ongoing-SD, crises abated over 50.7 ± 27.8 days. SD recurred in 8 patients (5 CP, 1 genetic, 2 idiopathic) after a mean of 20.3 ± 19.8 months.

RP emerges as a feasible rescue-therapy for severe medically refractory SD. Most patients show meaningful short-term improvement, but beneficial effects tend to decline over time, with high rate of SD recurrence-particularly in dyskinetic-CP. This may reflect suboptimal lesions' location and/or size, progressive disease course or re-emergence of dystonia through different networks. In this light, RP should be considered when DBS is contraindicated, the risk of hardware-related complications outweighs the advantages of an adjustable system, or in patients with limited life expectancy, where an irreversible yet effective rescue intervention is clinically justified.