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1.
J Neurosurg Pediatr ; 33(5): 417-427, 2024 May 01.
Article in English | MEDLINE | ID: mdl-38335514

ABSTRACT

OBJECTIVE: The Hydrocephalus Clinical Research Network (HCRN) conducted a prospective study 1) to determine if a new, better-performing version of the Endoscopic Third Ventriculostomy Success Score (ETVSS) could be developed, 2) to explore the performance characteristics of the original ETVSS in a modern endoscopic third ventriculostomy (ETV) cohort, and 3) to determine if the addition of radiological variables to the ETVSS improved its predictive abilities. METHODS: From April 2008 to August 2019, children (corrected age ≤ 17.5 years) who underwent a first-time ETV for hydrocephalus were included in a prospective multicenter HCRN study. All children had at least 6 months of clinical follow-up and were followed since the index ETV in the HCRN Core Data Registry. Children who underwent choroid plexus cauterization were excluded. Outcome (ETV success) was defined as the lack of ETV failure within 6 months of the index procedure. Kaplan-Meier curves were constructed to evaluate time-dependent variables. Multivariable binary logistic models were built to evaluate predictors of ETV success. Model performance was evaluated with Hosmer-Lemeshow and Harrell's C statistics. RESULTS: Seven hundred sixty-one children underwent a first-time ETV. The rate of 6-month ETV success was 76%. The Hosmer-Lemeshow and Harrell's C statistics of the logistic model containing more granular age and etiology categorizations did not differ significantly from a model containing the ETVSS categories. In children ≥ 12 months of age with ETVSSs of 50 or 60, the original ETVSS underestimated success, but this analysis was limited by a small sample size. Fronto-occipital horn ratio (p = 0.37), maximum width of the third ventricle (p = 0.39), and downward concavity of the floor of the third ventricle (p = 0.63) did not predict ETV success. A possible association between the degree of prepontine adhesions on preoperative MRI and ETV success was detected, but this did not reach statistical significance. CONCLUSIONS: This modern, multicenter study of ETV success shows that the original ETVSS continues to demonstrate good predictive ability, which was not substantially improved with a new success score. There might be an association between preoperative prepontine adhesions and ETV success, and this needs to be evaluated in a future large prospective study.


Subject(s)
Hydrocephalus , Third Ventricle , Ventriculostomy , Humans , Ventriculostomy/methods , Hydrocephalus/surgery , Hydrocephalus/diagnostic imaging , Female , Male , Third Ventricle/surgery , Third Ventricle/diagnostic imaging , Child , Child, Preschool , Prospective Studies , Infant , Treatment Outcome , Adolescent , Neuroendoscopy/methods , Follow-Up Studies
2.
J Stroke Cerebrovasc Dis ; 32(10): 107282, 2023 Oct.
Article in English | MEDLINE | ID: mdl-37659190

ABSTRACT

BACKGROUND: The objective of this study was to compare procedural and clinical outcomes in patients with acute ischemic stroke (AIS) treated via transradial access (TRA) mechanical thrombectomy (MT) versus conventional transfemoral access (TFA). METHODS: We performed a retrospective analysis of consecutive patients with AIS treated with TRA versus TFA MT at our tertiary comprehensive stroke center. Access choice was individualized based on occlusion site, aortic and arch anatomy. Outcomes were extracted from our institutional stroke registry and included procedural time, Thrombolysis in Cerebral Infarction (TICI) reperfusion score, NIHSS, 90-day mRS and 90-day mortality. Comparisons were performed using Student t-Test and Fischer's exact test as appropriate. RESULTS: 175 mechanical thrombectomies were performed during the study interval; 39 (22%) were performed via TRA and 136 (79%) TFA. Access to reperfusion time was 36.3 ± 24.5 minutes in the TRA group and 21.9 ± 17.6 in the TFA group (p<0.001). The proportion of patients with a TICI reperfusion score of 2b or 3 was similar in both groups (TRA: 34 (87%) vs. TFA: 121 (89%) p=0.559. The median 90-day mRS was similar between both groups (p=0.170), as was the 90-day mortality (p = 0.509). CONCLUSIONS: While TFA is faster in our cohort, TFA and TRA are both safe and effective for MT in acute ischemic stroke. While TFA remains mainstay, TRA can be valuable in variant anatomy despite its technical limitations. Individualizing access based on advanced imaging and patient factors may improve practice; however, updates in catheter and access technology are necessary to optimize outcomes with TRA.


Subject(s)
Ischemic Stroke , Stroke , Humans , Retrospective Studies , Cerebral Infarction , Stroke/diagnostic imaging , Stroke/therapy , Thrombectomy/adverse effects
3.
Interv Neuroradiol ; : 15910199231175348, 2023 May 17.
Article in English | MEDLINE | ID: mdl-37198900

ABSTRACT

BACKGROUND: For stent-retriever (SR) thrombectomy, technical developments such as the Push and Fluff technique (PFT) appear to have a significant impact on procedural success. This study aimed to (1) quantify the enhancement in clot traction when using PFT as compared to the standard unsheathing technique (SUT) and (2) to evaluate the performance of PFT in new versus established users of the technique. METHODS: Operators were divided between established PFT and SUT users. Each experiment was labeled according to the SR size, utilized technique, and operator experience. A three-dimensional-printed chamber with a clot simulant was used. After each retriever deployment, the SR wire was connected to a force gauge. Tension was applied by pulling the gauge until clot disengagement. The maximal force was recorded. RESULTS: A total of 167 experiments were performed. The median overall force to disengage the clot was 1.11 pounds for PFT and 0.70 pounds for SUT (an overall 59.1% increment with PFT; p < 0.001). The PFT effect was consistent across different retriever sizes (69% enhancement with the 3 × 32mm device, 52% with the 4 × 28mm, 65% with the 4 × 41mm, 47% with the 6 × 37mm). The ratio of tension required for clot disengagement with PFT versus SUT was comparable between physicians who were PFT versus SUT operators (1.595 [0.844] vs. 1.448 [1.021]; p: 0.424). The PFT/SUT traction ratio remained consistent from passes 1 to 4 of each technique in SUT users. CONCLUSION: PFT led to reproduceable improvement in clot engagement with an average ∼60% increase in clot traction in this model and was found not to have a significant learning curve.

4.
Childs Nerv Syst ; 39(4): 887-894, 2023 04.
Article in English | MEDLINE | ID: mdl-36633680

ABSTRACT

PURPOSE: To determine whether intraoperative adjunctive EVD placement in patients with a posterior fossa tumor (PFT) led to improved surgical, radiographic, and clinical outcomes compared to those who did not receive an EVD. METHODS: Patients were grouped as those who underwent routine intraoperative adjunctive EVD insertion and those who did not at time of PFT resection. Patients who pre-operatively required a clinically indicated EVD insertion were excluded. Comparative analyses between both groups were conducted to evaluate clinical, radiological, and pathological outcomes. Odds ratios (ORs) with corresponding 95% confidence intervals (CIs) were computed for post-operative outcomes. RESULTS: Fifty-five selected patients were included, 15 who had an EVD placed at the time of PFT resection surgery, and 40 who did not. Children without an EVD did not experience a higher rate of complications or poorer post-operative outcomes compared to those with an EVD placed during resection surgery. There was no significant difference in the degree of gross total resection (p = 0.129), post-operative CSF leak (p = 1.000), and post-operative hemorrhage (p = 0.554) between those with an EVD and those without. The frequency of new cranial nerve deficits post-operatively was higher in those with an EVD (40%) compared to those without (3%, p = 0.001). There was a trend towards more frequently observed post-operative hydrocephalus in the EVD group (p = 0.057). CONCLUSION: The routine use of EVD as an intraoperative adjunct in clinically stable pediatric patients with posterior fossa tumors and hydrocephalus may not be associated with improved radiological or clinical outcomes.


Subject(s)
Brain Neoplasms , Hydrocephalus , Infratentorial Neoplasms , Humans , Child , Retrospective Studies , Ventriculostomy/adverse effects , Postoperative Complications/etiology , Brain Neoplasms/surgery , Infratentorial Neoplasms/complications , Infratentorial Neoplasms/diagnostic imaging , Infratentorial Neoplasms/surgery , Hydrocephalus/etiology , Hydrocephalus/surgery , Drainage/adverse effects
5.
Surg Neurol Int ; 13: 177, 2022.
Article in English | MEDLINE | ID: mdl-35509556

ABSTRACT

Background: Acute ischemic stroke (AIS) due to cervical internal carotid artery (cICA) occlusion is challenging to treat, with the lower revascularization rates, higher risk for complications, and poor response to thrombolytic therapy compared to isolated intracranial occlusions. While emergent revascularization through mechanical thrombectomy (MT) improves outcomes, the impact of tissue plasminogen activator (tPA) on outcomes in this subgroup of patients remains unclear. The objective of this study is to report our preliminary experience in treating AIS with cICA occlusions secondary to severe atherosclerotic stenosis and to establish the need for further clinical studies to determine the optimal intervention strategy for these lesions. Methods: Data were collected on patients who presented with acute cICA occlusion who underwent MT and either acute or staged carotid angioplasty and stenting. We compare patients who received tPA to those who did not, analyzing revascularization times, outcomes, and complications between the two populations, and discuss how this influenced our preferred treatment approach. Results: Twenty-one patients met inclusion criteria, seven of who received tPA and 14 did not receive tPA before surgical intervention. Procedural and functional outcomes were similar between the two populations. TPA administration correlated with a higher rate of vessel reocclusion in staged procedures and trended toward higher rates of symptomatic ICH and 90-day mortality. Conclusion: Emergent revascularization with acute cICA stenting carries advantages, but its safety is precluded by tPA administration. We suggest a trial which randomizes patients with cICA occlusions to receiving either tPA or dual antiplatelet therapy before surgical intervention, aiming to ultimately improved outcomes in these patients.

6.
Neurosurg Clin N Am ; 33(2): 161-167, 2022 Apr.
Article in English | MEDLINE | ID: mdl-35346448

ABSTRACT

Leveraging from the interventional cardiology experience, the transradial access (TRA) for neurointervention has also started to become more used for both diagnostic and therapeutic procedures. A growing body of evidence is showing a superiority of the TRA compared with the conventional transfemoral access (TFA) in terms of access site complications (ACSs), patient satisfaction and preference, hospital length of stay, and cost. Outcomes via the transradial are noninferior, and at times superior, in select neuroendovascular procedures. Future advancements in technology with radial-specific catheters and further operator experience will aid in the full adoption of the TRA for endovascular procedures.


Subject(s)
Endovascular Procedures , Radial Artery , Endovascular Procedures/methods , Humans , Radial Artery/surgery , Retrospective Studies
7.
World Neurosurg ; 146: e1226-e1235, 2021 02.
Article in English | MEDLINE | ID: mdl-33271377

ABSTRACT

BACKGROUND: Intracerebral hemorrhage (ICH) is a neurosurgical emergency. Combined decompressive hemicraniectomy (DHC) and minimally invasive parafascicular surgery (MIPS) may provide a practical method of managing subcortical ICH. OBJECTIVE: 1) To present a case series of combined DHC-MIPS for the treatment of subcortical-based ICH; 2) to describe technical nuances of DHC-MIPS; and 3) to provide a literature overview of MIPS for ICH. METHODS: The following inclusion criteria were used: 1) Glasgow Coma Scale (GCS) score <3-4; 2) admission within 6 hours of onset; 3) increased intracranial pressure caused by hemorrhage; 4) patient unresponsive to medical management; 5) hemorrhage >30 cm3; 6) subcortical location; and 7) midline shift (mm). Before DHC, sulcal cannulation used the following coordinates: intersection of tragus-frontal bone and midpoint of midpupillary line and midline; coronal suture: 3-4 cm posterior to this point). RESULTS: Three patients were selected: a 62-year old woman, a 45-year old woman, and a 36-year-old man. GCS and ICH scores on admission were 7 and 3, 3 and 4, and 3 and 4, respectively. ICH was located in left basal ganglia in patients 1 and 3 and right basal ganglia in patient 2, all with intraventricular extension. ICH volume was 81.7, 68.2, and 42.3 cm3, respectively. The postoperative GCS score was 11, 10, and 6, respectively. There were no intraoperative complications or mortalities. Evacuation was within 15 minutes in all patients. The modified Rankin Scale score was 3, 4, and 5, respectively, with semi-independence in case 1. CONCLUSIONS: Combined DHC-MIPS, with the use of craniometric points, can provide a unique and simple surgical option for the management of subcortical ICH.


Subject(s)
Basal Ganglia Hemorrhage/surgery , Decompressive Craniectomy/methods , Drainage/methods , Intracranial Hypertension/surgery , Minimally Invasive Surgical Procedures/methods , Adult , Basal Ganglia Hemorrhage/complications , Cerebral Ventricles , Drainage/instrumentation , Female , Glasgow Coma Scale , Humans , Intracranial Hypertension/etiology , Male , Middle Aged , Minimally Invasive Surgical Procedures/instrumentation , Prefrontal Cortex , Surgical Instruments
8.
Surg Neurol Int ; 10: 179, 2019.
Article in English | MEDLINE | ID: mdl-31583176

ABSTRACT

BACKGROUND: Cerebral pseudoaneurysm formation associated with ventricular catheterization is an exceedingly rare complication that results from direct catheter-induced injury to a vessel. We report a case of intracerebral pseudoaneurysm formation associated with ventricular catheterization in a patient with hydrocephalus following aneurysmal subarachnoid hemorrhage. CASE DESCRIPTION: The patient presented with aneurysmal subarachnoid hemorrhage and underwent partial endovascular embolization of the offending wide-necked basilar tip aneurysm with the plan for a Stage 2 stent-assisted coiling after initial recovery. Before discharge, a ventriculoperitoneal shunt (VPS) was placed for postaneurysmal hydrocephalus. Three weeks later, she presented with intraparenchymal and intraventricular hemorrhage. Angiography revealed a cortical aneurysm contiguous to the ventricular catheter of the VPS. She underwent microsurgical excision of the aneurysm, and a new VPS was placed after resolution of the intraventricular hemorrhage. She later underwent the second stage of the treatment and had an excellent neurological recovery to an independent state. CONCLUSION: Iatrogenic intracerebral pseudoaneurysm formation is an exceedingly rare complication of ventricular catheterization but is associated with significant mortality. Identifying a pseudoaneurysm in this context warrants prompt and definitive treatment with microsurgical or endovascular treatment.

9.
J Clin Neurosci ; 70: 79-84, 2019 Dec.
Article in English | MEDLINE | ID: mdl-31466905

ABSTRACT

PURPOSE: The present study aims to determine the tumor-related, clinical, and demographic factors associated with extent of resection (EOR) and post-operative outcomes in JPA patients. METHODS: All patients with JPA, identified from a single-center brain tumour data base, were included in this retrospective analysis. Pre-operative MRI scans were reviewed by a single neurosurgeon blinded to the EOR. JPA cases that exhibited no residual tumor post-operatively were assigned to the GTR group, all other tumors were assigned to the

Subject(s)
Astrocytoma/surgery , Brain Neoplasms/surgery , Neurosurgical Procedures/methods , Adolescent , Astrocytoma/pathology , Brain Neoplasms/pathology , Child , Child, Preschool , Cohort Studies , Female , Humans , Infant , Male , Neoplasm, Residual/pathology , Neurosurgical Procedures/adverse effects , Retrospective Studies , Treatment Outcome
10.
Lancet Child Adolesc Health ; 2(3): 191-204, 2018 03.
Article in English | MEDLINE | ID: mdl-30169254

ABSTRACT

BACKGROUND: MRI and laboratory features have been incorporated into international diagnostic criteria for multiple sclerosis. We assessed the pattern of MRI lesions and contributions of cerebrospinal fluid (CSF) and serum antibody findings that best identifies children with multiple sclerosis, and the applicability of international diagnostic criteria in the paediatric context. METHODS: In this prospective cohort study, detailed clinical assessments, serum and CSF studies, and MRI scans were done in youth (aged 0·46-17·87 years) with incidental acquired demyelinating syndrome. Participants were examined prospectively to identify relapsing disease. All MRI scans were assessed using a validated scoring method. A random forest classifier identified imaging and laboratory features that best predicted a multiple sclerosis or monophasic outcome. Performance of the 2001, 2010, and 2017 international McDonald criteria for the diagnosis of multiple sclerosis, the 2016 MRI in multiple sclerosis (MAGNIMS) criteria, and our 2011 proposed (Verhey) criteria were determined; performance was adjudicated with generalised linear models. FINDINGS: Between Sept 1, 2004, and June 30, 2017, we included 324 participants with median follow-up of 72 months (range 6-150), 71 (22%) participants with multiple sclerosis, 237 (73%) with monophasic acquired demyelinating syndrome, 14 (4%) with relapsing non-multiple sclerosis, and two (1%) with alternative diagnoses. We scored 2391 brain, 444 spinal, and 67 dedicated orbital MRI scans. One or more T1 hypointense lesions plus one or more periventricular lesions (Verhey criteria) best predicted multiple sclerosis outcome. Performance of the 2017 McDonald criteria was comparable to the 2010 McDonald criteria and was easier to adjudicate. The ability of CSF oligoclonal bands to substitute for the requirement for both enhancing and non-enhancing lesions in the 2017 McDonald criteria improved its performance compared with the 2010 criteria. Myelin oligodendrocyte testing at baseline did not improve performance of the 2017 McDonald criteria. INTERPRETATION: The 2017 McDonald criteria for the diagnosis of multiple sclerosis, as applied at the time of incident attack, perform well in identifying children and youth with multiple sclerosis, indicating that the same diagnostic criteria for multiple sclerosis apply across the age span. The presence of so-called black holes on MRI and periventricular lesions at baseline (Verhey criteria) also effectively distinguish children with multiple sclerosis from children with monophasic demyelination. The presence of CSF oligoclonal bands improve diagnostic accuracy. Myelin oligodendrocyte glycoprotein antibodies identify children with acute disseminated encephalomyelitis, and those with relapsing non-multiple sclerosis, most of whom do not meet 2017 McDonald criteria at onset. FUNDING: The Multiple Sclerosis Scientific Research Foundation and The Children's Hospital of Philadelphia.


Subject(s)
Magnetic Resonance Imaging , Multiple Sclerosis/diagnosis , Adolescent , Child , Child, Preschool , Cohort Studies , Female , Humans , Infant , Internationality , Male , Multiple Sclerosis/blood , Multiple Sclerosis/cerebrospinal fluid , Multiple Sclerosis/diagnostic imaging , Prospective Studies
11.
World Neurosurg ; 113: 58-61, 2018 May.
Article in English | MEDLINE | ID: mdl-29408346

ABSTRACT

BACKGROUND: Subdural hematoma, without any radiographic evidence of subarachnoid hemorrhage, is a rare presentation of a ruptured intracranial aneurysm. Even more rare is the occurrence of a pure subdural hematoma caused by a ruptured cortical saccular aneurysm. We report the eighth case of pure subdural hematoma secondary to a ruptured nonmycotic cortical berry aneurysm. CASE DESCRIPTION: We report a case of pure subdural hematoma secondary to a ruptured true saccular aneurysm of a cortical artery branch. The lesion was carefully delineated with computed tomography (CT) angiography (CTA) and cerebral angiography, and successfully treated with hematoma evacuation and clip ligation. The patient demonstrates no neurologic deficits 6 months after surgery, and CTA results remain negative. CONCLUSIONS: In the context of a presentation of spontaneous subdural hematoma, intracerebral aneurysm rupture should be considered as a possible etiology. Prompt vascular imaging with careful evaluation of the entire cerebral vasculature, including the cortical vessels, should be considered.


Subject(s)
Aneurysm, Ruptured/diagnostic imaging , Aneurysm, Ruptured/surgery , Hematoma, Subdural, Acute/diagnostic imaging , Hematoma, Subdural, Acute/surgery , Intracranial Aneurysm/diagnostic imaging , Intracranial Aneurysm/surgery , Aged , Aneurysm, Ruptured/complications , Diagnosis, Differential , Hematoma, Subdural, Acute/etiology , Humans , Intracranial Aneurysm/complications , Male
12.
Neurology ; 88(18): 1744-1750, 2017 May 02.
Article in English | MEDLINE | ID: mdl-28381515

ABSTRACT

OBJECTIVE: To investigate how monophasic acquired demyelinating syndromes (ADS) affect age-expected brain growth over time. METHODS: We analyzed 83 pediatric patients imaged serially from initial demyelinating attack: 18 with acute disseminated encephalomyelitis (ADEM) and 65 with other monophasic ADS presentations (monoADS). We further subdivided the monoADS group by the presence (n = 33; monoADSlesion) or absence (n = 32; monoADSnolesion) of T2 lesions involving the brain at onset. We used normative data to compare brain volumes and calculate age- and sex-specific z scores, and used mixed-effect models to investigate their relationship with time from demyelinating illness. RESULTS: Children with monophasic demyelination (ADEM, non-ADEM with brain lesions, and those without brain involvement) demonstrated reduced age-expected brain growth on serial images, driven by reduced age-expected white matter growth. Cortical gray matter volumes were not reduced at onset but demonstrated reduced age-expected growth afterwards in all groups. Brain volumes differed from age- and sex-expected values to the greatest extent in children with ADEM. All patient groups failed to recover age-expected brain growth trajectories. CONCLUSIONS: Brain volume, and more importantly age-expected brain growth, is negatively affected by acquired demyelination, even in the absence of chronicity, implicating factors other than active inflammation as operative in this process.


Subject(s)
Brain/diagnostic imaging , Brain/growth & development , Demyelinating Autoimmune Diseases, CNS/diagnostic imaging , Adolescent , Child , Child, Preschool , Female , Follow-Up Studies , Gray Matter/diagnostic imaging , Gray Matter/growth & development , Humans , Longitudinal Studies , Magnetic Resonance Imaging , Male , Organ Size , Prospective Studies , Young Adult
13.
J Int Neuropsychol Soc ; 22(10): 1050-1060, 2016 11.
Article in English | MEDLINE | ID: mdl-27903328

ABSTRACT

OBJECTIVES: The aim of this study was to describe cognitive, academic, and psychosocial outcomes after an incident demyelinating event (acquired demyelinating syndromes, ADS) in childhood and to investigate the contribution of brain lesions and confirmed MS diagnosis on outcome. METHODS: Thirty-six patients with ADS (mean age=12.2 years, SD=2.7, range: 7-16 years) underwent brain MRI scans at presentation and at 6-months follow-up. T2-weighted lesions on MRI were assessed using a binary classification. At 6-months follow-up, patients underwent neuropsychological evaluation and were compared with 42 healthy controls. RESULTS: Cognitive, academic, and behavioral outcomes did not differ between the patients with ADS and controls. Three of 36 patients (8.3%) were identified with cognitive impairment, as determined by performance falling ≤1.5 SD below normative values on more than four independent tests in the battery. Poor performance on a visuomotor integration task was most common, observed among 6/32 patients, but this did not differ significantly from controls. Twelve of 36 patients received a diagnosis of MS within 3 years post-ADS. Patients with MS did not differ from children with monophasic ADS in terms of cognitive performance at the 6-months follow-up. Fatigue symptoms were reported in 50% of patients, irrespective of MS diagnosis. Presence of brain lesions at onset and 6 months post-incident demyelinating event did not associate with cognitive outcome. CONCLUSIONS: Children with ADS experience a favorable short-term neurocognitive outcome, even those confirmed to have MS. Longitudinal evaluations of children with monophasic ADS and MS are required to determine the possibility of late-emerging sequelae and their time course. (JINS, 2016, 22, 1050-1060).


Subject(s)
Cognitive Dysfunction/diagnosis , Demyelinating Diseases/diagnosis , Multiple Sclerosis/diagnosis , Adolescent , Child , Cognitive Dysfunction/diagnostic imaging , Cognitive Dysfunction/etiology , Cognitive Dysfunction/physiopathology , Demyelinating Diseases/complications , Demyelinating Diseases/diagnostic imaging , Demyelinating Diseases/physiopathology , Female , Follow-Up Studies , Humans , Magnetic Resonance Imaging , Male , Multiple Sclerosis/complications , Multiple Sclerosis/diagnostic imaging , Multiple Sclerosis/physiopathology
14.
Mult Scler Int ; 2014: 262350, 2014.
Article in English | MEDLINE | ID: mdl-24883205

ABSTRACT

Multiple sclerosis (MS) is a chronic inflammatory and neurodegenerative disease that manifests as acute relapses and progressive disability. As a primary endpoint for clinical trials in MS, disability is difficult to both characterize and measure. Furthermore, the recovery from relapses and the rate of disability vary considerably among patients. Given these challenges, investigators have developed and studied the performance of various outcome measures and surrogate endpoints in MS clinical trials. This review defines the outcome measures and surrogate endpoints used to date in MS clinical trials and presents challenges in the design of both adult and pediatric trials.

15.
Pediatr Neurol ; 50(1): 38-48, 2014 Jan.
Article in English | MEDLINE | ID: mdl-24370172

ABSTRACT

BACKGROUND: The impact of childhood epilepsy can only be appreciated by understanding that epilepsy comprises a set of complex neurobehavioral conditions with significant social consequences, and not simply disorders of recurrent seizures. Our objective is to describe the hypotheses and methodology behind a large prospective longitudinal study that is based on a conceptual framework for understanding health outcomes. The study will quantify the specific influences--direct, mediating or moderating--that various epilepsy, comorbid, child, and family variables exert on health over the early life course. METHODS: The target population is 8- to 14-year-old children with epilepsy and their caregivers from across Canada. Children, caregivers, and health professionals are completing 17 measures at five visits over a 28-month period. We have selected measures based on content, the source of the items, psychometric properties, and provisions for child self-report. Our cross-sectional and longitudinal design includes a relational model for structural equation modeling of specific biomedical and psychosocial variables with hierarchical direction of influence. To measure change over time, we will use hierarchical linear modeling. SIGNIFICANCE: This article reports the framework for interpreting future data. We believe that it will help researchers consider their methodology and encourage them to plan and execute longitudinal studies. Furthermore, the article will help clinical readers identify what to look for when evaluating outcomes research. It is our belief that the next generation of research to understand life-course effect in the lives of children and youth with chronic conditions and their families must occur over real time.


Subject(s)
Epilepsy/epidemiology , Epilepsy/therapy , Outcome Assessment, Health Care , Adolescent , Canada/epidemiology , Child , Epilepsy/psychology , Female , Humans , Longitudinal Studies , Male , Observation , Quality of Life
16.
J Child Neurol ; 29(5): 654-65, 2014 May.
Article in English | MEDLINE | ID: mdl-24092896

ABSTRACT

Tumefactive demyelinating lesions can be difficult to distinguish from tumors. Clinical and magnetic resonance imaging features of children with tumefactive demyelination and supratentorial brain tumors were compared. Patients were identified through a 23-site national demyelinating disease study, and from a single-site neuroradiology database. For inclusion, lesions met at least 1 of 3 criteria: maximal cross-sectional diameter >20 mm, local or global cerebral mass effect, or presence of perilesional edema. Thirty-one children with tumefactive demyelination (5 with solitary lesions) were identified: 27 of 189 (14.3%) from the demyelinating disease study and 4 from the database. Thirty-three children with tumors were identified. Children with tumefactive demyelination were more likely to have an abnormal neurologic examination and polyfocal neurologic deficits compared to children with tumors. Tumefactive demyelination was distinguished from tumor by the presence of multiple lesions, absence of cortical involvement, and decrease in lesion size or detection of new lesions on serial imaging.


Subject(s)
Brain Neoplasms/diagnosis , Brain/pathology , Demyelinating Diseases/diagnosis , Child , Cohort Studies , Female , Humans , Magnetic Resonance Imaging , Male , Outcome Assessment, Health Care , Statistics, Nonparametric , Time Factors
17.
Neurology ; 81(14): 1215-21, 2013 Oct 01.
Article in English | MEDLINE | ID: mdl-23966255

ABSTRACT

OBJECTIVE: To estimate sample sizes for pediatric multiple sclerosis (MS) trials using new T2 lesion count, annualized relapse rate (ARR), and time to first relapse (TTFR) endpoints. METHODS: Poisson and negative binomial models were fit to new T2 lesion and relapse count data, and negative binomial time-to-event and exponential models were fit to TTFR data of 42 children with MS enrolled in a national prospective cohort study. Simulations were performed by resampling from the best-fitting model of new T2 lesion count, number of relapses, or TTFR, under various assumptions of the effect size, trial duration, and model parameters. RESULTS: Assuming a 50% reduction in new T2 lesions over 6 months, 90 patients/arm are required, whereas 165 patients/arm are required for a 40% treatment effect. Sample sizes for 2-year trials using relapse-related endpoints are lower than that for 1-year trials. For 2-year trials and a conservative assumption of overdispersion (ϑ), sample sizes range from 70 patients/arm (using ARR) to 105 patients/arm (TTFR) for a 50% reduction in relapses, and 230 patients/arm (ARR) to 365 patients/arm (TTFR) for a 30% relapse reduction. Assuming a less conservative ϑ, 2-year trials using ARR require 45 patients/arm (60 patients/arm for TTFR) for a 50% reduction in relapses and 145 patients/arm (200 patients/arm for TTFR) for a 30% reduction. CONCLUSION: Six-month phase II trials using new T2 lesion count as an endpoint are feasible in the pediatric MS population; however, trials powered on ARR or TTFR will need to be 2 years in duration and will require multicentered collaboration.


Subject(s)
Brain/pathology , Clinical Trials as Topic/standards , Magnetic Resonance Imaging/methods , Multiple Sclerosis/diagnosis , Research Design/standards , Adolescent , Binomial Distribution , Child , Clinical Protocols/standards , Female , Humans , Magnetic Resonance Imaging/instrumentation , Male , Models, Statistical , Multiple Sclerosis/pathology , Poisson Distribution , Prospective Studies , Recurrence , Sample Size , Time Factors
18.
Mult Scler ; 19(10): 1359-62, 2013 Sep.
Article in English | MEDLINE | ID: mdl-23620544

ABSTRACT

The 2010 McDonald criteria allow the diagnosis of multiple sclerosis (MS) at first attack in children and adults provided that the first attack symptoms are typical of MS and that the magnetic resonance imaging (MRI) conforms to prescribed features. We evaluate whether meeting the 2010 McDonald criteria at onset correlates with a more aggressive clinical course in a cohort of pediatric MS patients. The Expanded Disability Status Scale (EDSS) and annualized relapse rate were not associated with positivity for 2010 McDonald criteria at onset. The 2010 McDonald criteria identify children with similar MS features to those identified by clinical or MRI evidence of dissemination over time.


Subject(s)
Central Nervous System/pathology , Multiple Sclerosis/pathology , Age of Onset , Child , Disease Progression , Female , Humans , Magnetic Resonance Imaging , Male
19.
Handb Clin Neurol ; 112: 999-1017, 2013.
Article in English | MEDLINE | ID: mdl-23622308

ABSTRACT

Acute nontraumatic myelopathies of childhood include inflammatory, infectious, and vascular etiologies. Inflammatory immune-mediated disorders of the spinal cord can be categorized as idiopathic isolated transverse myelitis, neuromyelitis optica, and multiple sclerosis. In recent years, human T-cell lymphotropic virus type 1, West Nile virus, enterovirus-71, and Lyme disease have been increasingly recognized as infectious etiologies of myelopathy, and poliomyelitis remains an important etiology in world regions where vaccination programs have not been universally available. Vascular etiologies include vasculopathies (systemic lupus erythematosus, small vessel primary angiitis of the central nervous system), arteriovenous malformations, and spinal cord infarction (fibrocartilaginous embolism, diffuse hypoxic ischemia-mediated infarction). Vascular myelopathies are less common than inflammatory and infectious myelopathies, but are more likely to lead to devastating clinical deficits. Current therapeutic strategies include acute anti-inflammatory treatment and rehabilitation. Stem cell transplantation, nerve graft implantation, and stimulation of endogenous repair mechanisms represent promising strategies for spinal cord repair.


Subject(s)
Central Nervous System Infections/diagnosis , Myelitis, Transverse/diagnosis , Neuromyelitis Optica/diagnosis , Spinal Cord Diseases/diagnosis , Vascular Diseases/diagnosis , Central Nervous System Infections/complications , Diagnosis, Differential , Humans , Myelitis, Transverse/etiology , Neuromyelitis Optica/etiology , Spinal Cord/blood supply , Spinal Cord Diseases/etiology , Vascular Diseases/complications
20.
Neuroimaging Clin N Am ; 23(2): 217-26.e1-7, 2013 May.
Article in English | MEDLINE | ID: mdl-23608686

ABSTRACT

Magnetic resonance (MR) imaging is one of the most important paraclinical tools for the diagnosis of multiple sclerosis (MS), and monitoring of disease progression and treatment response. This article provides clinicians and neuroradiologists caring for children with demyelinating disorders with a suggested standard MR imaging acquisition and reporting protocol, and defines a standard lexicon for lesion features typical of MS in children. As there is considerable overlap between the MR imaging features of pediatric- and adult-onset MS, the recommendations provided herein may be of relevance to radiologists and clinicians caring for adults with multiple sclerosis.


Subject(s)
Brain/pathology , Magnetic Resonance Imaging/standards , Multiple Sclerosis/diagnosis , Multiple Sclerosis/pathology , Optic Nerve/pathology , Spinal Cord/pathology , Age Factors , Algorithms , Artifacts , Child , Diffusion Magnetic Resonance Imaging/standards , Disease Progression , Documentation/standards , Humans , Image Enhancement/standards , Image Interpretation, Computer-Assisted/standards , Nerve Fibers, Myelinated/pathology
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