Tags

Type your tag names separated by a space and hit enter

Clinical phenotypes associated with outcomes following deep brain stimulation for childhood dystonia.
J Neurosurg Pediatr 2019; :1-9JN

Abstract

OBJECTIVE

Although deep brain stimulation (DBS) is an accepted treatment for childhood dystonia, there is significant heterogeneity in treatment response and few data are available to identify ideal surgical candidates.

METHODS

Data were derived from a systematic review and individual patient data meta-analysis of DBS for dystonia in children that was previously published. Outcomes were assessed using the Burke-Fahn-Marsden Dystonia Rating Scale for movement (BFMDRS-M) and for disability (BFMDRS-D). The authors used partial least squares, bootstrapping, and permutation statistics to extract patterns of contributions of specific preoperative characteristics to relationship with distinct outcomes, in all patients and in patients with primary and secondary dystonia separately.

RESULTS

Of 301 children undergoing DBS for dystonia, 167 had primary dystonia, 125 secondary dystonia, and 9 myoclonus dystonia. Three dissociable preoperative phenotypes (latent variables) were identified and associated with the following: 1) BFMDRS-M at last follow-up; 2) relative change in BFMDRS-M score; and 3) relative change in BFMDRS-D score. The phenotype of patients with secondary dystonia, with a high BFMDRS-M score and truncal involvement, undergoing DBS at a younger age, was associated with a worse postoperative BFMDRS-M score. Children with primary dystonia involving the trunk had greater improvement in BFMDRS-M and -D scores. Those with primary dystonia of shorter duration and proportion of life with disease, undergoing globus pallidus DBS, had greater improvements in BFMDRS-D scores at long-term follow-up.

CONCLUSIONS

In a comprehensive, data-driven, multivariate analysis of DBS for childhood dystonia, the authors identified novel and dissociable patient phenotypes associated with distinct outcomes. The findings of this report may inform surgical candidacy for DBS.

Authors+Show Affiliations

1Division of Neurosurgery, Department of Surgery, and.2Faculty of Medicine, University of Toronto, Toronto, Ontario.3Faculty of Medicine, Université de Montréal, Montréal, Québec.1Division of Neurosurgery, Department of Surgery, and.2Faculty of Medicine, University of Toronto, Toronto, Ontario.1Division of Neurosurgery, Department of Surgery, and. 4Division of Neurosurgery, Sunnybrook Health Sciences Centre, Toronto, Ontario.1Division of Neurosurgery, Department of Surgery, and. 5Division of Neurosurgery, Toronto Western Hospital, Toronto, Ontario; and.1Division of Neurosurgery, Department of Surgery, and. 5Division of Neurosurgery, Toronto Western Hospital, Toronto, Ontario; and.1Division of Neurosurgery, Department of Surgery, and. 5Division of Neurosurgery, Toronto Western Hospital, Toronto, Ontario; and.1Division of Neurosurgery, Department of Surgery, and. 6Division of Neurosurgery, The Hospital for Sick Children, Toronto, Ontario, Canada.

Pub Type(s)

Journal Article

Language

eng

PubMed ID

31299640

Citation

Badhiwala, Jetan H., et al. "Clinical Phenotypes Associated With Outcomes Following Deep Brain Stimulation for Childhood Dystonia." Journal of Neurosurgery. Pediatrics, 2019, pp. 1-9.
Badhiwala JH, Karmur B, Elkaim LM, et al. Clinical phenotypes associated with outcomes following deep brain stimulation for childhood dystonia. J Neurosurg Pediatr. 2019.
Badhiwala, J. H., Karmur, B., Elkaim, L. M., Alotaibi, N. M., Morgan, B. R., Lipsman, N., ... Ibrahim, G. M. (2019). Clinical phenotypes associated with outcomes following deep brain stimulation for childhood dystonia. Journal of Neurosurgery. Pediatrics, pp. 1-9. doi:10.3171/2019.5.PEDS1973.
Badhiwala JH, et al. Clinical Phenotypes Associated With Outcomes Following Deep Brain Stimulation for Childhood Dystonia. J Neurosurg Pediatr. 2019 Jul 12;1-9. PubMed PMID: 31299640.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Clinical phenotypes associated with outcomes following deep brain stimulation for childhood dystonia. AU - Badhiwala,Jetan H, AU - Karmur,Brij, AU - Elkaim,Lior M, AU - Alotaibi,Naif M, AU - Morgan,Benjamin R, AU - Lipsman,Nir, AU - De Vloo,Philippe, AU - Kalia,Suneil K, AU - Lozano,Andres M, AU - Ibrahim,George M, Y1 - 2019/07/12/ PY - 2019/01/30/received PY - 2019/05/08/accepted PY - 2019/7/13/entrez PY - 2019/7/13/pubmed PY - 2019/7/13/medline KW - BFMDRS-D = Burke-Fahn-Marsden Dystonia Rating Scale for disability KW - BFMDRS-M = BFMDRS for movement KW - DBS = deep brain stimulation KW - GPi = globus pallidus internus KW - IPD = individual patient data KW - LV = latent variable KW - PKAN = pantothenate kinase–associated neurodegeneration KW - PLS = partial least squares KW - SVD = singular-value decomposition KW - clinical outcomes KW - deep brain stimulation KW - dystonia KW - functional neurosurgery KW - movement disorders KW - neuromodulation SP - 1 EP - 9 JF - Journal of neurosurgery. Pediatrics JO - J Neurosurg Pediatr N2 - OBJECTIVE: Although deep brain stimulation (DBS) is an accepted treatment for childhood dystonia, there is significant heterogeneity in treatment response and few data are available to identify ideal surgical candidates. METHODS: Data were derived from a systematic review and individual patient data meta-analysis of DBS for dystonia in children that was previously published. Outcomes were assessed using the Burke-Fahn-Marsden Dystonia Rating Scale for movement (BFMDRS-M) and for disability (BFMDRS-D). The authors used partial least squares, bootstrapping, and permutation statistics to extract patterns of contributions of specific preoperative characteristics to relationship with distinct outcomes, in all patients and in patients with primary and secondary dystonia separately. RESULTS: Of 301 children undergoing DBS for dystonia, 167 had primary dystonia, 125 secondary dystonia, and 9 myoclonus dystonia. Three dissociable preoperative phenotypes (latent variables) were identified and associated with the following: 1) BFMDRS-M at last follow-up; 2) relative change in BFMDRS-M score; and 3) relative change in BFMDRS-D score. The phenotype of patients with secondary dystonia, with a high BFMDRS-M score and truncal involvement, undergoing DBS at a younger age, was associated with a worse postoperative BFMDRS-M score. Children with primary dystonia involving the trunk had greater improvement in BFMDRS-M and -D scores. Those with primary dystonia of shorter duration and proportion of life with disease, undergoing globus pallidus DBS, had greater improvements in BFMDRS-D scores at long-term follow-up. CONCLUSIONS: In a comprehensive, data-driven, multivariate analysis of DBS for childhood dystonia, the authors identified novel and dissociable patient phenotypes associated with distinct outcomes. The findings of this report may inform surgical candidacy for DBS. SN - 1933-0715 UR - https://www.unboundmedicine.com/medline/citation/31299640/Clinical_phenotypes_associated_with_outcomes_following_deep_brain_stimulation_for_childhood_dystonia L2 - https://thejns.org/doi/10.3171/2019.5.PEDS1973 DB - PRIME DP - Unbound Medicine ER -