Subthalamic Nuclei Stimulation in Patients With Pantothenate Kinase-Associated Neurodegeneration (PKAN)
adolescent; subthalamic nucleus; follow up; scoring system; human; article; female; male; adult; middle aged; dystonia; case report; treatment outcome; brain depth stimulation; Deep brain stimulation; neurologic disease assessment; Burke Fahn Marsden Dystonia Rating Scale movement rating scale; fluency disorder; globus pallidus internus; neurodegeneration with brain iron accumulation/th [Therapy]; neurologic examination; pantothenate kinase-associated neurodegeneration; subthalamic nuclei; tone and motor problems; IND; surgical intervention; subthalamic nuclei stimulation
Introduction: Pantothenate kinase-associated neurodegeneration (PKAN) is a rare autosomal recessive genetic disease that leads to extrapyramidal symptoms, such as dystonia, ataxia, dysarthria, and involuntary movements. Treatment of PKAN with deep brain stimulation (DBS) has been reported, but mainly focuses on targeting the globus pallidus internus (GPi). Subthalamic nuclei (STN) may also be a potential target for treatment of PKAN. Methods: In this study, we reviewed three patients with PKAN (two with typical PKAN and one with atypical PKAN) treated by bilateral STN stimulation and present a review of the literature. All patients received neurological evaluation using the Burke-Fahn-Marsden Dystonia Rating Scale-movement (BFMDRS) scoring system before and after surgery. Patients were then subject to regular clinical follow-ups (ranging from 22 to 44 months). Results: The mean stimulation amplitude, pulse width and frequency was 2.65 +/- 0.45 V, 91.7 +/- 21.9 mus, and 146.7 +/- 12.5 Hz, respectively. BFMDRS scores were improved in all patients after surgery, ranging from 41.6 to 73.1%. Improvements of appendicular symptoms ranged from 46.2 to 94.1%, and improvements of axial symptoms ranged from 27.3 to 33.3%. No side effects were reported in patients 1 and 2; whereas patient 3 exhibited a mild decline in verbal fluency one year after surgery. Conclusion: STN stimulation could serve as a candidate DBS target in the treatment of PKAN, especially for patients with prominent appendicular symptoms. Copyright © 2017 International Neuromodulation Society
Liu Z; Liu Y; Yang Y; Wang L; Dou W; Guo J; Wang Y; Guo Y; Wan X; Ma W; Wang R
Neuromodulation
2017
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<a href="http://doi.org/10.1111/ner.12549" target="_blank" rel="noreferrer noopener">10.1111/ner.12549</a>
Predictors of Specialized Pediatric Palliative Care Involvement and Impact on Patterns of End-of-Life Care in Children With Cancer
Purpose The impact of specialized pediatric palliative care (SPPC) teams on patterns of end-of-life care is unknown. We sought to determine (1) which children with cancer access SPPC and (2) the impact of accessing SPPC on the risk of experiencing high-intensity end-of-life care (intensive care unit admission, mechanical ventilation, or in-hospital death). Methods Using a provincial childhood cancer registry, we assembled a retrospective cohort of Ontario children with cancer who died between 2000 and 2012 and received care through pediatric institutions with an SPPC team. Patients were linked to population-based administrative data capturing inpatient, outpatient, and emergency visits. Children were classified as having SPPC, general palliative care, or no palliative care on the basis of SPPC clinical databases, physician billing codes, or inpatient diagnosis codes. Results Of the 572 children, 166 (29%) received care from an SPPC team for at least 30 days before death, and 100 (17.5%) received general palliative care. SPPC involvement was significantly less likely for children with hematologic cancers (OR, 0.3; 95% CI, 0.3 to 0.4), living in the lowest income areas (OR, 0.4; 95% CI, 0.2 to 0.8), and living further from the treatment center (OR, 0.5; 95% CI, 0.4 to 0.5). SPPC was associated with a five-fold decrease in odds of intensive care unit admission (OR, 0.2; 95% CI, 0.1 to 0.4), whereas general palliative care had no impact. Similar associations were seen with all secondary indicators. Conclusion When available, SPPC, but not general palliative care, is associated with lower intensity care at the end of life for children with cancer. However, access remains uneven. These results provide the strongest evidence to date supporting the creation of SPPC teams.
Widger K; Sutradhar R; Rapoport A; Vadeboncoeur C; Zelcer S; Kassam A; Nelson K; Liu Y; Wolfe J; Earle CC; Pole JD; Gupta S
Journal of Clinical Oncology
2018
<a href="http://doi.org/%2010.1200/jco.2017.75.6312" target="_blank" rel="noreferrer noopener">10.1200/jco.2017.75.6312</a>