The device was used for an off label indication; other relevant device(s) are: product id: neu_unknown_lead.Gonzalez, v., cif, l., milesi, c., cambonie, g., de antonio rubio, i., cyprien, f., chan seng, e., roujeau, t., coubes, p.Long-term clinical outcome of deep brain stimulation in pkan syndrome.European journal of paediatric neurology (2017) 21 supplement 1 (e179).If information is provided in the future, a supplemental report will be issued.[(b)(4)].
|
Abstract: objective: to describe long-term clinical outcome of a series of patients suffering from pkan syndrome treated with deep brain stimulation (dbs).Pkan syndrome is an autosomal recessive neurodegenerative disorder secondary to pank2 gene mutations, manifesting by severe generalized dystonia and spasticity.Classical childhood-onset phenotype is often associated with fatal outcome.To date, several case reports have been published to describe the therapeutic outcome of globus pallidus internus (gpi) and more recently subthalamic nucleus dbs in pkan syndrome.No pharmacological treatment has yet been approved to treat this condition.Methods: we conducted a retrospective systematic review of patients with documented diagnosis of pkan syndrome treated with dbs in our center.Clinical outcome was measured with bfmdrs scale.All patients were implanted with mri-guided targeting without microelectrode recordings.Results: we identified 18 patients in our database corresponding to inclusion criteria: 5 adults and 13 children (m:f =10:8).Age at surgery was 28.601 ± 7 years for adults and 11.9 ± 2.6 for children.All patients underwent gpi dbs; 5 patients were implanted with a second pair of leads during follow-up (thalamic vim/vop target (n = 4) and stn (n = 1)).Intrathecal baclofen pump was used to treat severe associated spasticity in 4 patients.Four patients required tube feeding due to severe dysphagia.Postoperative clinical management was complicated by several device-related adverse events: lead fracture, lead dysfunction (7 patients).Six patients developed status dystonicus.Five patients died despite intensive care unit management and advanced surgical therapies (2.8 ± 2 yr after surgery).Mean follow-up after surgery for the other patients was 10.9 ± 4.6 years.Conclusion: gpi dbs was effective in long-term treatment of severe dystonia in pkan syndrome in our series (mean follow-up: 8.6 years).Classical childhood-onset of disease and severe gpi atrophy were associated with a more severe clinical condition with high prevalence of status dystonicus despite dbs therapy.Reported events: 1.6 patients who were implanted with gpi-dbs for pkan developed status dystonicus despite dbs therapy.The authors noted that classical childhood-onset of the disease and severe gpi atrophy were associated with more severe clinical condition with high prevalence of status dystonicus.It was not possible to ascertain specific device information from the article or to match the reported event with any previously reported event.
|