Faculty Bibliography

Objective: We report the feasibility and outcomes of endoscopic resection of select intraventricular tumors in children. Methods: The clinical characteristics of 11 children with solid intraventricular tumors who underwent tumor resection were reviewed. 12 procedures were performed. Results: Gross total resection was achieved in 11 of 12 cases (92%). Maximal diameter ranged from 9-26 mm (mean 16.6 mm). Pathology included subependymal giant cell astrocytomas (SEGA), ependymomas, non-germinomatous germ cell tumor (NGGCT) and pilocytic astrocytoma. Mean follow-up was 35 months (range 10-109 months). All patients returned to their neurological baseline following surgery. Local tumor recurrence occurred in one patient and distant recurrence in another. Complications occurred in one patient, no permanent morbidity or mortality occurred. Hydrocephalus was present preoperatively in 5 cases and was treated with tumor removal alone or with additional endoscopic third ventriculostomy. No patient required a ventriculoperitoneal shunt. Conclusion: Neuroendoscopic gross-total resection of solid intraventricular tumors is a safe procedure in carefully selected pediatric patients

Rhabdomyomatous mesenchymal hamartoma (RMH) is a rare, benign, congenital tumor of the dermis and subcutaneous tissue comprised of skeletal muscle and adipose and adnexal elements. Although the majority of cases are an incidental finding in otherwise healthy patients, some have been reported in association with other anomalies. We present a full-term boy evaluated on day 2 of life for two lesions located on the midline of the lower back and right buttock that each appeared clinically as an atrophic, pink plaque. Ultrasound of the midline lesion revealed an underlying lipomyelomeningocele with a tethered cord in the spinal canal. Histopathology of the right buttock cutaneous lesion was consistent with a diagnosis of RMH. Surgical excision was performed on the midline intradural lipoma and the lesion on the buttock was monitored clinically. Repeat biopsy of this site at 1 year of age revealed complete spontaneous regression. This case highlights three interesting features: the association with an occult spinal dysraphism lipomyelomeningocele and tethered cord, the clinical presentation of an atrophic plaque as opposed to the more commonly reported raised lesions, and the phenomenon of spontaneous regression of the lesion. Most importantly, this final feature of regression in our patient suggests that, in the absence of symptoms, clinical observation of RMH lesions is warranted for spontaneous regression for 1 to 2 years provided that no functional deficit is noted and that the cutaneous or deeper lesions are not causing a medical problem.

Learning Objectives: The purpose of this study was to identify trends in postoperative transfusion practice in children undergoing cranial vault reconstruction. We hypothesize that young age is a risk factors for blood product transfusion requirement. Methods: A retrospective chart review was performed for all patients undergoing fronto-orbital advancement or cranial vault reconstruction at a single, medium-sized, academic tertiary pediatric hospital from June 14, 2011 to June 4, 2014. Diagnosis, procedure type, age, postoperative transfusion, hemoglobin level, platelet count, and international normalized ratio (INR) were recorded. Results: 90 patients were included in the analysis. Patient's age in months (mo) and years (yrs) ranged from 2 mo to 16 yrs. 25 patients (28%) were transfused with packed red blood cells (pRBC) and 4 patients (4%) were transfused with fresh frozen plasma (FFP). Median age of the total population was 0.8 yrs. Median age of patients receiving pRBC, FFP, and no transfusions were 0.7, 0.9, and 0.8 yrs, respectively. There may have been a trend toward younger age in patients that received pRBC transfusion when compared to patients receiving no transfusion (median 0.7 vs 0.8, p = 0.12, Wilcoxon rank-sum test). The hemoglobin nadir was 5.2 g/dL and 6 g/dL for patients who were transfused and who were not transfused with pRBCs, respectively. The mean hemoglobin level indicating need for pRBC transfusion was 6.85 + 1.07 g/dL and the mean INR indicating need for FFP transfusion was 1.38 + 0.05. Conclusions: This observational study of pediatric patients undergoing cranial vault reconstruction shows a trend that patients receiving postoperative pRBC transfusions were younger than patients that were not transfused. Further studies are needed to assess what impact age, hemoglobin nadir, and receipt of transfusion in the postoperative period has on short- and long-term postoperative outcomes

BACKGROUND: Activation of the RAS-RAF-MEK-ERK signaling pathway is thought to be the key driver of pediatric low-grade astrocytoma (PLGA) growth. Sorafenib is a multikinase inhibitor targeting BRAF, VEGFR, PDGFR, and c-kit. This multicenter phase II study was conducted to determine the response rate to sorafenib in patients with recurrent or progressive PLGA. METHODS: Key eligibility criteria included age >/=2 years, progressive PLGA evaluable on MRI, and at least one prior chemotherapy treatment. Sorafenib was administered twice daily at 200 mg/m2/dose (maximum of 400 mg/dose) in continuous 28-day cycles. MRI, including 3-dimensional volumetric tumor analysis, was performed every 12 weeks. BRAF molecular testing was performed on tumor tissue when available. RESULTS: Eleven patients, including 3 with neurofibromatosis type 1 (NF1), were evaluable for response; 5 tested positive for BRAF duplication. Nine patients (82%) came off trial due to radiological tumor progression after 2 or 3 cycles, including 3 patients with confirmed BRAF duplication. Median time to progression was 2.8 months (95% CI, 2.1-31.0 months). Enrollment was terminated early due to this rapid and unexpectedly high progression rate. Tumor tissue obtained from 4 patients after termination of the study showed viable pilocytic or pilomyxoid astrocytoma. CONCLUSIONS: Sorafenib produced unexpected and unprecedented acceleration of tumor growth in children with PLGA, irrespective of NF1 or tumor BRAF status. In vitro studies with sorafenib indicate that this effect is likely related to paradoxical ERK activation. Close monitoring for early tumor progression should be included in trials of novel agents that modulate signal transduction.