Successful treatment of hemorrhagic brainstem cavernous malformation with hematoma evacuation and postoperative propranolol.

In this case, we describe an evident hemorrhagic brainstem cavernous malformation successfully treated with a planned sequence of surgical evacuation of the hematoma followed by postoperative propranolol therapy. In contrast to common practice, the cavernoma itself was not resected. A nearly 3-year-old male presented with altered mental status, gait disturbance, and facial palsy. CT and MRI demonstrated a large acute pontine hematoma. A large nearby vein suggested cavernous malformation. He was initially treated conservatively but a repeat CT scan demonstrated further expansion of hematoma and he was taken emergently to the OR. Due to the sensitive location of the hematoma in the pons, we planned to evacuate the hematoma without resecting any of the presumed cavernoma. Instead, we planned to treat the cavernoma with propranolol. Postoperatively, the patient's condition improved and was still improving at hospital discharge 2 weeks later. Six-month follow-up MRI showed no cavernoma with only hemosiderin at the site of the evacuated hematoma. This is the first reported case of a hemorrhagic brainstem cavernous malformation treated with a planned sequence of hematoma evacuation followed by propranolol without an attempt to resect the cavernoma.

The utility of magnetic resonance imaging in pediatric trauma patients suspected of having cervical spine injuries.

BACKGROUND: Pediatric cervical spine injuries (CSI) are rare but potentially devastating sequelae of blunt trauma. Existing protocols to evaluate children at risk for CSI frequently incorporate computed topography (CT) and magnetic resonance imaging (MRI); however, the clinical value of performing both remains unclear. METHODS: Single-center retrospective review of pediatric trauma patients who underwent both CT and MRI of the cervical spine between 2001 and 2015. Based on radiographic findings, CT and MRI results were grouped into one of three categories: no injury, stable injury, or unstable injury. Radiographic instability was defined by disruption of two or more contiguous spinal columns while radiographic stability was defined by any other acute cervical spine abnormality on imaging. Clinical instability was defined by the need for surgical intervention (halo or spinal fusion), with the remaining patients, including children discharged in a cervical collar, considered clinically stable. RESULTS: In total, 221 children met inclusion criteria, with a median age of 9 (interquartile range, 3-14). The Glasgow Coma Scale (GCS) score of the cohort was 9 (interquartile range, 4-15). Thirty-three (14.9%) children had clinically unstable injuries, requiring surgical intervention. Among the 160 (72.4%) children with no injury on CT, MRI identified no injury in 84 (52.5%) cases, a stable injury in 76 (47.5%) cases, and an unstable injury in none. Among the 21 children with stable injuries on CT, MRI findings were concordant in 17 (81.0%) cases. In four (19.0%) cases, a spinal column injury was identified on CT and appeared to be stable, but later deemed unstable on MRI. Forty (18.1%) patients had an unstable injury on CT with 100% MRI concordance. CONCLUSION: In pediatric trauma patients suspected of having a CSI, a normal cervical spine CT is sufficient to rule out a clinically significant CSI as no child with a normal cervical CT was found to be radiographically or clinically unstable. LEVEL OF EVIDENCE: Diagnostic Test, level III.

Selection of children with ultra-severe traumatic brain injury for neurosurgical intervention.

OBJECTIVE: A recent retrospective study of severe traumatic brain injury (TBI) in pediatric patients showed similar outcomes in those with a Glasgow Coma Scale (GCS) score of 3 and those with a score of 4 and reported a favorable long-term outcome in 11.9% of patients. Using decision tree analysis, authors of that study provided criteria to identify patients with a potentially favorable outcome. The authors of the present study sought to validate the previously described decision tree and further inform understanding of the outcomes of children with a GCS score 3 or 4 by using data from multiple institutions and machine learning methods to identify important predictors of outcome. METHODS: Clinical, radiographic, and outcome data on pediatric TBI patients (age < 18 years) were prospectively collected as part of an institutional TBI registry. Patients with a GCS score of 3 or 4 were selected, and the previously published prediction model was evaluated using this data set. Next, a combined data set that included data from two institutions was used to create a new, more statistically robust model using binomial recursive partitioning to create a decision tree. RESULTS: Forty-five patients from the institutional TBI registry were included in the present study, as were 67 patients from the previously published data set, for a total of 112 patients in the combined analysis. The previously published prediction model for survival was externally validated and performed only modestly (AUC 0.68, 95% CI 0.47, 0.89). In the combined data set, pupillary response and age were the only predictors retained in the decision tree. Ninety-six percent of patients with bilaterally nonreactive pupils had a poor outcome. If the pupillary response was normal in at least one eye, the outcome subsequently depended on age: 72% of children between 5 months and 6 years old had a favorable outcome, whereas 100% of children younger than 5 months old and 77% of those older than 6 years had poor outcomes. The overall accuracy of the combined prediction model was 90.2% with a sensitivity of 68.4% and specificity of 93.6%. CONCLUSIONS: A previously published survival model for severe TBI in children with a low GCS score was externally validated. With a larger data set, however, a simplified and more robust model was developed, and the variables most predictive of outcome were age and pupillary response.

Understanding United States Investigational Device Exemption Studies-Clinical Relevance and Importance for Healthcare Economics.

INTRODUCTION: The US Food and Drug Administration allows a previously unapproved device to be used clinically to collect safety and effectiveness data under their Investigational Device Exemption (IDE) category. The process usually falls under 3 different trial categories: noninferiority, equivalency, and superiority. To confidently inform our patients, understanding the basic concepts of these trials is paramount. The purpose of this manuscript was to provide a comprehensive review of these topics using recently published IDE trials and economic analyses of cervical total disc replacement as illustrative examples. CASE STUDY MOBI-C ARTIFICIAL CERVICAL DISC: In 2006, an IDE was initiated to study the safety and effectiveness of total disc replacement controlled against the standard of care, anterior cervical discectomy, and fusion. Under the IDE, randomized controlled trials comparing both 1 and 2 level cervical disease were completed. The sponsor designed the initial trial as noninferiority; however, using adaptive methodology, superiority could be claimed in the 2-level investigation. REVIEWING HEALTHCARE ECONOMICS: Healthcare economics are critical in medical decision making and reimbursement practices. Once both cost- and quality-adjusted life-year (QALY) are known for each patient, the incremental cost-effectiveness ratio is calculated. Willingness-to-pay is controversial, but a commonly cited guideline considers interventions costing below 20 000 $/QALY strongly cost effective and more than 100 000 $/QALY as not cost effective. CONCLUSION: While large Food and Drug Administration IDE studies are often besieged by complex statistical considerations and calculations, it is fundamentally important that clinicians understand at least the terminology and basic concepts on a practical level.

Medical necessity of routine admission of children with mild traumatic brain injury to the intensive care unit.

OBJECTIVE Approximately 475,000 children are treated for traumatic brain injury (TBI) in the US each year; most are classified as mild TBI (Glasgow Coma Scale [GCS] Score 13-15). Patients with positive findings on head CT, defined as either intracranial hemorrhage or skull fracture, regardless of severity, are often transferred to tertiary care centers for intensive care unit (ICU) monitoring. This practice creates a significant burden on the health care system. The purpose of this investigation was to derive a clinical decision rule (CDR) to determine which children can safely avoid ICU care. METHODS The authors retrospectively reviewed patients with mild TBI who were ≤ 16 years old and who presented to a Level 1 trauma center between 2008 and 2013. Data were abstracted from institutional TBI and trauma registries. Independent covariates included age, GCS score, pupillary response, CT characteristics, and Injury Severity Score. A composite outcome measure, ICU-level care, was defined as cardiopulmonary instability, transfusion, intubation, placement of intracranial pressure monitor or other invasive monitoring, and/or need for surgical intervention. Stepwise logistic regression defined significant predictors for model inclusion with p < 0.10. The authors derived the CDR with binary recursive partitioning (using a misclassification cost of 20:1). RESULTS A total of 284 patients with mild TBI were included in the analysis; 40 (14.1%) had ICU-level care. The CDR consisted of 5 final predictor variables: midline shift > 5 mm, intraventricular hemorrhage, nonisolated head injury, postresuscitation GCS score of < 15, and cisterns absent. The CDR correctly identified 37 of 40 patients requiring ICU-level care (sensitivity 92.5%; 95% CI 78.5-98.0) and 154 of 244 patients who did not require an ICU-level intervention (specificity 63.1%; 95% CI 56.7-69.1). This results in a negative predictive value of 98.1% (95% CI 94.1-99.5). CONCLUSIONS The authors derived a clinical tool that defines a subset of pediatric patients with mild TBI at low risk for ICU-level care. Although prospective evaluation is needed, the potential for improved resource allocation is significant.