Hannah E. Goldstein

Cerebrospinal Fluid Shunt Failure in Children Is Associated with Medical Complexity and Shunt Position, Not Shunt Technology: A Multicenter Prospective Study from the Hydrocephalus Clinical Research Network

Vijay M. Ravindra, John R.W. Kestle, Hailey Jensen, Abhaya V. Kulkarni et autres

OBJECTIVES: To investigate patterns of cerebrospinal fluid (CSF) shunt failure and to determine whether new risk factors have arisen as care has evolved. STUDY DESIGN: This prospective study examined first-time shunt insertions in children <18 years at 14 Hydrocephalus Clinical Research Network …

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Comparative multicenter evaluation of thalamic neuromodulation for treatment‐resistant epilepsy in children

Samuel A. Tenhoeve, Nebras M. Warsi, Allison M. Ludwick, Adam C. Glaser et autres

OBJECTIVE: Use of neuromodulation strategies targeting thalamic nuclei, including deep brain stimulation (DBS) and responsive neurostimulation (RNS), for treatment of pediatric drug-resistant epilepsy (DRE) is increasing, despite limited evidence for efficacy and safety. We present the initial results from the Comparative Multicenter …

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Intracerebroventricular B7-H3-targeting chimeric antigen receptor T cells for non-pontine diffuse midline glioma and recurrent/refractory pediatric CNS tumors: A phase 1 trial

Rebecca Ronsley, Wenjun Huang, Ethan Rohlf, Kristy Seidel et autres

BACKGROUND: High-grade central nervous system (CNS) tumors carry a poor prognosis with limited curative options if first-line therapy fails. B7-H3 is expressed in many of these tumors, and chimeric antigen receptor (CAR) T cell therapy is an emerging immunotherapeutic strategy. METHODS: BrainChild-03 …

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413 Thalamic Neuromodulation for Pediatric Drug-Resistant Epilepsy: A Case Series Evaluating Deep Brain Stimulation and Responsive Neurostimulation

Dominic Nistal, Adriel Barrios-Anderson, Scott Boop, Benjamin Edmonds et autres

INTRODUCTION: Non-lesional drug-resistant epilepsy (DRE) in children is difficult to manage, with limited treatment options. Recent advances in deep brain stimulation (DBS) and responsive neurostimulation (RNS) have shown seizure reduction in adults, particularly with thalamic targets for generalized multifocal epilepsy. However, their …

Endoscopic Endonasal Transsphenoidal Surgery for of Pediatric Sellar Lesions (Pituitary Neuroendocrine Tumors and Rathke's Cleft Cysts): Radiographic and Biochemical Outcomes from a Single-Center Series

Thomas Hanks, Dominic Nistal, Jessica Eaton, Adriel Barrios-Anderson et autres

Introduction: Pituitary neuroendocrine tumors (PitNETs) and Rathkes Cleft cysts (RCC) in children are rare and distinct from more common pediatric sellar lesions such as craniopharyngioma. Presentation is often due to endocrinopathies or visual dysfunction secondary to optic chiasm compression. Surgical resection and …

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Use of a Multiportal Endoscopic Transnasal and Transorbital Approach for Pediatric Skull Base Neoplasms: Case Series and Literature Review

Adriel Barrios-Anderson, Zirun Zhao, Christopher Seward, Tyler Lazaro et autres

Background: Lesions of the pediatric skull base can involve multiple sinonasal and intracranial compartments, posing unique surgical challenges due to limited pneumatization, immature bony structures, and the need to balance oncologic control with functional preservation and cosmetic outcomes. Combining endoscopic endonasal and …

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Two-Surgeon, Multi-Portal Endoscopic Transnasal and Transmaxillary Approach for Resection of Juvenile Nasopharyngeal Angiofibroma: A Case Series

Zirun Zhao, Adriel Barrios-Anderson, Chris Seward, Jeremy S. Ruthberg et autres

Background: Juvenile nasopharyngeal angiofibroma (JNA) is a rare, benign hypervascular tumor, typically occurring in adolescent males. Despite the benign nature, these lesions have propensity for rapid expansion, erosion of the skull base and sinonasal bony structures, and significant epistaxis. Surgical resection via …

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Hemispherectomy in infants: an institutional experience with 21 patients

Dominic Nistal, Robert T. Buckley, Scott Boop, Hannah E. Goldstein et autres

OBJECTIVE: Drug-resistant epilepsy (DRE) can arise early in life due to multiple structural abnormalities. In patients with the disorder, seizures can be difficult to control and can significantly impair neurodevelopment. Hemispherectomy is well described as a treatment for refractory hemispheric-onset epilepsy, but …

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Phase 1 study of ABI ‐009 ( nab ‐rapamycin) for surgically refractory epilepsy ( RaSuRE )

Koko Hall, Russell P. Saneto, Stephanie Randle, Mark S. Wainwright et autres

Abstract Objective Seizures that are refractory to medical and surgical therapy increase the risk of morbidity and mortality in children with epilepsy. Novel therapeutic trials for this population remain sparse and suboptimal. This Phase 1 study evaluates the (1) safety, tolerability, and …

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Novel surgical approach for intraventricular cerliponase alfa enzyme replacement therapy via central venous access device (CVAD) port in neuronal ceroid lipofuscinosis type 2 (CLN2) disease

Scott Boop, Dominic Nistal, Adriel Barrios-Anderson, Walter B. Cherny et autres

INTRODUCTION: Neuronal ceroid lipofuscinosis type 2 (CLN2) is a genetic disease caused by deficiency of the enzyme tripeptidyl peptidase 1 (TPP1), resulting in seizures, vision/cognitive decline, and early death. Cerliponase alfa is an enzyme replacement therapy approved as treatment for CLN2 disease, …

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Indomethacin‐responsive refractory headache: Two case reports in children after hemispherectomy for Rasmussen's encephalitis

Jessica Hauser Chatterjee, Priya Monrad, Hannah E. Goldstein, Dennis Shaw et autres

BACKGROUND: Indomethacin-responsive headaches occur in youth and include primary headache syndromes such as hemicrania continua and paroxysmal hemicrania. Both are trigeminal autonomic cephalalgias (TACs). In pediatrics TACs are rare. Moreover, they may differ in their phenotypes and response to indomethacin compared to …

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Intracerebroventricular B7-H3-targeting CAR T cells for diffuse intrinsic pontine glioma: a phase 1 trial

Nicholas A. Vitanza, Rebecca Ronsley, Michelle Choe, Kristy Seidel et autres

Abstract Diffuse intrinsic pontine glioma (DIPG) is a fatal central nervous system (CNS) tumor that confers a median survival of 11 months. As B7-H3 is expressed on pediatric CNS tumors, we conducted BrainChild-03, a single-center, dose-escalation phase 1 clinical trial of repetitive …

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