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Full-Text Articles in Neurology

Age And Functional Outcomes Post-Neurologic Insult In Patients Attending Inpatient Rehabilitation, Jonathan Bowman Spt, Elena Crooks Pt, Dpt, Phd, Doug Weeks Phd, Kimberly Honn Phd Jan 2020

Age And Functional Outcomes Post-Neurologic Insult In Patients Attending Inpatient Rehabilitation, Jonathan Bowman Spt, Elena Crooks Pt, Dpt, Phd, Doug Weeks Phd, Kimberly Honn Phd

2020 Symposium Posters

Introduction

Neurologic insults such as strokes and traumatic brain injuries (TBI) affect over 1 million Americans every year. The lack of current knowledge informing accurate prognoses causes victims and their loved ones distress, and is a focus of much research. The purpose of this study was to determine whether patient age at time of insult could predict change in functional outcomes during inpatient rehabilitation.

Methods

Subjects were patients of an inpatient rehabilitation facility (IRF) post-stroke or TBI. The Functional Independence Measure (FIM) assessed functional independence and cognitive status at admission and discharge from the IRF. The Montebello Rehabilitation Factor Score …


Machine Learning Prediction Of Glioblastoma Patient One-Year Survival, Andrew Du '20, Warren Mcgee, Jane Y. Wu Jan 2020

Machine Learning Prediction Of Glioblastoma Patient One-Year Survival, Andrew Du '20, Warren Mcgee, Jane Y. Wu

Student Publications & Research

Glioblastoma (GBM) is a grade IV astrocytoma formed primarily from cancerous astrocytes and sustained by intense angiogenesis. GBM often causes non-specific symptoms, creating difficulty for diagnosis. This study aimed to utilize machine learning techniques to provide an accurate one-year survival prognosis for GBM patients using clinical and genomic data from the Chinese Glioma Genome Atlas. Logistic regression (LR), support vector machines (SVM), random forest (RF), and ensemble models were used to identify and select predictors for GBM survival and to classify patients into those with an overall survival (OS) of less than one year and one year or greater. With …


Polio To The Rescue; New Frontiers In Glioblastoma Treatment, Syed Sarmad Bukhari, Aneela Darbar, Muhammad Shahzad Shamim Jan 2020

Polio To The Rescue; New Frontiers In Glioblastoma Treatment, Syed Sarmad Bukhari, Aneela Darbar, Muhammad Shahzad Shamim

Section of Neurosurgery

Lack of substantial progress in effective treatment of glioblastomamultiforme has led investigators to explore unconventional treatment options including the use of tumour treating fields and oncolyticvirotherapy. Herein the authors have reviewed some of the recent literature on oncolyticvirotherapy for the treatment of glioblastomamultiforme.


Rna Sequencing Of Human Peripheral Nerve In Response To Injury: Distinctive Analysis Of The Nerve Repair Pathways., Andrew S. Welleford, Jorge E. Quintero, Nader El Seblani, Eric Blalock, Sumedha Gunewardena, Steven M. Shapiro, Sean M. Riordan, Peter Huettl, Zain Guduru, John A. Stanford, Craig G. Van Horne, Greg A. Gerhardt Jan 2020

Rna Sequencing Of Human Peripheral Nerve In Response To Injury: Distinctive Analysis Of The Nerve Repair Pathways., Andrew S. Welleford, Jorge E. Quintero, Nader El Seblani, Eric Blalock, Sumedha Gunewardena, Steven M. Shapiro, Sean M. Riordan, Peter Huettl, Zain Guduru, John A. Stanford, Craig G. Van Horne, Greg A. Gerhardt

Manuscripts, Articles, Book Chapters and Other Papers

The development of regenerative therapies for central nervous system diseases can likely benefit from an understanding of the peripheral nervous system repair process, particularly in identifying potential gene pathways involved in human nerve repair. This study employed RNA sequencing (RNA-seq) technology to analyze the whole transcriptome profile of the human peripheral nerve in response to an injury. The distal sural nerve was exposed, completely transected, and a 1 to 2 cm section of nerve fascicles was collected for RNA-seq from six participants with Parkinson's disease, ranging in age between 53 and 70 yr. Two weeks after the initial injury, another …


The Comparative Efficacy Of Amantadine Vs. Lcig Therapy For The Treatment Of Levodopa-Induced Dyskinesia In Patients With Advanced Parkinson's Disease, Katelynn Timony Jan 2020

The Comparative Efficacy Of Amantadine Vs. Lcig Therapy For The Treatment Of Levodopa-Induced Dyskinesia In Patients With Advanced Parkinson's Disease, Katelynn Timony

Capstone Showcase

Introduction: Though levodopa-carbidopa therapy remains the gold standard of Parkinson’s disease (PD) treatment to this day, patients often experience increased dyskinetic activity as a result of long-term treatment; known as levodopa-induced dyskinesia (LID); the mechanism of which is not completely understood. In this review, the efficacy of two adjunct treatments for LID are compared: 1) the concomitant administration of amantadine, an antiviral medication with antidyskinetic properties; and 2) a new form of levodopa administration in the form of an intestinal gel delivered straight via a device much like an insulin pump.

Methods: Two systematic literature searches of primary, peer-reviewed articles …


Comparing Open And Fetoscopic Fetal Surgical Repairs Of Myelomeningocele, Kyra Isaacs Jan 2020

Comparing Open And Fetoscopic Fetal Surgical Repairs Of Myelomeningocele, Kyra Isaacs

Capstone Showcase

Introduction: There have been great advances in the treatment of myelomeningocele (MMC) spina bifida in the past 20 years. An open surgical repair in-utero has been shown to reduce mortality and morbidity, specifically decreasing the rate of shunt placement and hindbrain herniation compared to a postnatal surgical approach. However, this surgery introduced risks to the mother that were never a consideration in previous surgical approaches.

Methods: This paper compares research on new minimally invasive fetoscopic surgical techniques to open fetal surgery in the prenatal repair of MMC. Searches in PubMed and Clinical Key were conducted to produce papers published on …


Prevalence And Predictors Of Chronic Pain Intensity And Disability Among Adults With Sickle Cell Disease, Nadine Matthie, Coretta Jenerette, Ashley Gibson, Sudeshna Paul, Melinda Higgins, Lakshmanan Krishnamurti Jan 2020

Prevalence And Predictors Of Chronic Pain Intensity And Disability Among Adults With Sickle Cell Disease, Nadine Matthie, Coretta Jenerette, Ashley Gibson, Sudeshna Paul, Melinda Higgins, Lakshmanan Krishnamurti

Faculty, Staff and Student Publications

Among 170 adults with sickle cell disease, we evaluated chronic pain impact and disability prevalence, assessed age and gender differences, and identified psychosocial predictors of chronic pain intensity and disability. Most participants had a high level of disability. Chronic pain intensity and disability were significantly associated with pain catastrophizing and chronic pain self-efficacy, and worsened with age. Further research is needed to confirm study findings and develop interventions, including palliative care approaches that address catastrophizing and disability, particularly for young women and middle-aged adults with sickle cell disease. Moreover, consistent clinical assessment of chronic pain and psychosocial health should be …


Utilization Of A Continuous Pericapsular Nerve Group (Peng) Block With An Opioid-Sparing Repair Of A Femoral Neck Fracture In A Pediatric Patient, Karla Wyatt, Moustafa Zidane, Chyong-Jy Joyce Liu Jan 2020

Utilization Of A Continuous Pericapsular Nerve Group (Peng) Block With An Opioid-Sparing Repair Of A Femoral Neck Fracture In A Pediatric Patient, Karla Wyatt, Moustafa Zidane, Chyong-Jy Joyce Liu

Faculty, Staff and Students Publications

In the pediatric population, femoral neck fractures are usually associated with high-impact trauma and often present with pain in the groin area. Regional anesthesia can offer adjunctive therapy for acute pain management. Various techniques have been employed to circumvent pain related to hip fractures and resultant hip surgery. Neuraxial, lumbar plexus, caudal, epidural, fascia iliaca, and femoral continuous nerve block techniques are advantageous in mitigating hip pain. However, these approaches require patient repositioning during placement and carry the potential for motor blockade with resultant weakness. A newly described method, the Pericapsular Nerve Group (PENG) block, allows for analgesia of the …


Alzheimer's And Amyloid Beta: Amyloidogenicity And Tauopathy Via Dyshomeostatic Interactions Of Amyloid Beta, Jordan Tillinghast Dec 2019

Alzheimer's And Amyloid Beta: Amyloidogenicity And Tauopathy Via Dyshomeostatic Interactions Of Amyloid Beta, Jordan Tillinghast

Senior Honors Theses

This paper reviews functions of Amyloid-β (Aβ) in healthy individuals compared to the consequences of aberrant Aβ in Alzheimer’s disease (AD). As extraneuronal Aβ accumulation and plaque formation are characteristics of AD, it is reasonable to infer a pivotal role for Aβ in AD pathogenesis. Establishing progress of the disease as well as the mechanism of neurodegeneration from AD have proven difficult (Selkoe, 1994). This thesis provides evidence suggesting the pathogenesis of AD is due to dysfunctional neuronal processes involving Aβ’s synaptic malfunction, abnormal interaction with tau, and disruption of neuronal homeostasis. Significant evidence demonstrates that AD symptoms are partially …


Is It Fair To Compare? A Patient And Family Experience Of Two Healthcare Systems And Neurosurgical Teams Within A Two-Week Period, Laura Miller Cpxp Nov 2019

Is It Fair To Compare? A Patient And Family Experience Of Two Healthcare Systems And Neurosurgical Teams Within A Two-Week Period, Laura Miller Cpxp

Patient Experience Journal

As the mother of a 28-year-old son with cerebral palsy and hydrocephalus, and as a healthcare consultant focused on patient experience and professional development, I have a unique perspective and skill set. Recently he experienced symptoms that included an excruciating headache, neck pain and lethargy. Fearing his ventriculoperitoneal shunt had malfunctioned, he went to the emergency room and was later admitted on the neuro inpatient floor for a three-day hospitalization. His original shunt had been placed in 1991, and he never had an issue with until August 2018. While in the hospital, he was informed that he was no longer …


Awakening From A Medical Mystery: One Patient’S Experience Of Being Undiagnosed, Dwane Unruh Nov 2019

Awakening From A Medical Mystery: One Patient’S Experience Of Being Undiagnosed, Dwane Unruh

Patient Experience Journal

This personal narrative pleads for a supportive and comprehensive system or sub-system similar to that which exists for cancer patients, to deal with undiagnosed illnesses. By describing the torment of living with a debilitating illness that medicine could not easily recognize, then by contrasting this experience with my wife’s experience of the cancer care system, and by referring along the way to lessons learned many years ago from reading the works of the inciteful neurologist, Dr. Oliver Sacks, I hope to inspire the medical system to develop a separate, supportive and comprehensive system to deal with the undiagnosed. As it …


Acute Diagnosis Of Wilson’S Disease In A Teenage Patient, Sarah Irvin, Ryan Mccarthy Oct 2019

Acute Diagnosis Of Wilson’S Disease In A Teenage Patient, Sarah Irvin, Ryan Mccarthy

Marshall Journal of Medicine

Wilson’s Disease, a rare autosomal recessive genetic disease, is caused by a mutation in the ATP7B enzyme gene. Without this enzyme, copper builds up in the brain, liver, and cornea causing a multitude of symptoms. It is important to consider Wilson’s disease because the prognosis is dependent on timely diagnosis. This is an interesting case of a 19-year-old male who presented with suicidal thoughts and rapid weight loss. After many months and an extensive work-up, Wilson’s Disease was diagnosed. Due to his rapid decline, he was transferred to a larger university healthcare center where he is currently enrolled in clinical …


Guillain Barre Syndrome In A Patient With Sickle Cell Anemia, Kunjan Udani Md, Pooja Patel, Dveet Patel, Hajra Awwab, Nino Balanchivadze Oct 2019

Guillain Barre Syndrome In A Patient With Sickle Cell Anemia, Kunjan Udani Md, Pooja Patel, Dveet Patel, Hajra Awwab, Nino Balanchivadze

Internal Medicine

No abstract provided.


Peripheral Nerve Stimulator For Treating Nummular Headaches And Occipital Neuralgia, Bruce Dixon Do, Stephen Pyles Md, Ettore Crimi Md Oct 2019

Peripheral Nerve Stimulator For Treating Nummular Headaches And Occipital Neuralgia, Bruce Dixon Do, Stephen Pyles Md, Ettore Crimi Md

Anesthesiology

No abstract provided.


Spinal Cord Stimulator For Treating Pudendal Neuralgia, Bruce Dixon Do, Stephen Pyles Md, Ettore Crimi Md Oct 2019

Spinal Cord Stimulator For Treating Pudendal Neuralgia, Bruce Dixon Do, Stephen Pyles Md, Ettore Crimi Md

Anesthesiology

No abstract provided.


Use Of Botulinum Toxin In Central Nervous System Disorders, Julie Puvogel, Paige Torbet, Jourdan Ujlaki, Rebecca Worden, Lindsey Peters Oct 2019

Use Of Botulinum Toxin In Central Nervous System Disorders, Julie Puvogel, Paige Torbet, Jourdan Ujlaki, Rebecca Worden, Lindsey Peters

Pharmacy and Wellness Review

Botulinum toxin is a neurotoxin that is produced by Clostridium botulinum. At one time, this toxin was only seen as a lethal substance, but now scientists have found many medical uses for it. There are eight distinctive toxins (A-H), but only A and B currently have clinical uses. Botulinum toxin A has three different versions that are U.S. Food and Drug Administration (FDA) approved: onabotulinumtoxinA (Botox®), abobotulinumtoxinA (Dysport®), incobotulinumtoxinA (Xeomin®). Botulinum toxin B is also FDA approved as rimabotulinumtoxinB (Myobloc®). The toxins work by inducing reversible, local, dose-dependent chemodenervation by inhibiting acetylcholine release from presynaptic terminals. These drugs are approved …


Zika Virus Induced Neurological Disorders And Impacts On Public Health, Alexandra Herman, Martha Zimmerman, Olivia Vanscoy, Olivia Henton, Lindsey Peters Oct 2019

Zika Virus Induced Neurological Disorders And Impacts On Public Health, Alexandra Herman, Martha Zimmerman, Olivia Vanscoy, Olivia Henton, Lindsey Peters

Pharmacy and Wellness Review

Zika virus is a public health emergency of international concern. Zika virus (ZIKV) is a mosquito-borne pathogen that is carried by the Aedes genus. Zika is spread through direct bite and nonvector transmission. Most individuals infected with Zika will be asymptomatic, but some may present nonspecific viral symptoms. A rising number of neurological disorders in newborns whose mothers were infected with ZIKV during pregnancy have been reported due to recent outbreaks. Neurological disorders affect both the central and peripheral nervous systems and can result from bacterial, viral, fungal or parasitic infections of the nervous system or from the immune response …


Belsomra®: A Novel Dual Orexin Receptor Antagonist For The Treatment Of Insomnia, Shane Bogusz, Steven Blake, Michaela Wolford, Victoria Cho, Manoranjan D'Souza Oct 2019

Belsomra®: A Novel Dual Orexin Receptor Antagonist For The Treatment Of Insomnia, Shane Bogusz, Steven Blake, Michaela Wolford, Victoria Cho, Manoranjan D'Souza

Pharmacy and Wellness Review

Insomnia is a disease state characterized by a persistent difficulty in falling asleep, and results in enormous health-related and economic costs to both the individual and society. Several medications are currently available for the treatment of insomnia; however, these medications are associated with several limitations including anterograde amnesia, dependence, withdrawal symptoms upon stopping the medication and rebound insomnia. The U.S. Food and Drug Administration recently approved suvorexant (Belsomra®) as a treatment for insomnia. Suvorexant is a first-in-class dual orexin receptor antagonist for the treatment of insomnia. This review will first describe the Diagnostic and Statistical Manual of Mental Disorders (DSM-5) …


Moyamoya Disease Presenting With Stroke, Timothy Collins, Chimezie Ubbaonu, Phuong Vo Oct 2019

Moyamoya Disease Presenting With Stroke, Timothy Collins, Chimezie Ubbaonu, Phuong Vo

Internal Medicine

No abstract provided.


Current Trials And Therapies For The Treatment Of Amyotrophic Lateral Sclerosis And Frontotemporal Dementia, Adam Smith, Angela Chu, Lucy Wagala, Hannah Stewart, Lindsey Peters Oct 2019

Current Trials And Therapies For The Treatment Of Amyotrophic Lateral Sclerosis And Frontotemporal Dementia, Adam Smith, Angela Chu, Lucy Wagala, Hannah Stewart, Lindsey Peters

Pharmacy and Wellness Review

An area of health care that provides many more questions than answers includes neurodegenerative disorders. Amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig's disease, and frontotemporal dementia (FTD) are both diseases about which we know very little. However, ALS and FTD affect nearly 30,000 and 60,000 Americans respectively. Currently, there is not a cure for ALS or FTD and treatment options are aimed toward symptom management. Much of the pathophysiology of these diseases is unknown, but we do know there are genetic implications, specifically in SOD1, TARDBP and c90RF72. These mutations lead to cognitive deficits, muscle weakness and, eventually, …


Neurotransmitter, Fall 2019, George Washington Institute For Neuroscience Neurological Institute, George Washington University Hospital Oct 2019

Neurotransmitter, Fall 2019, George Washington Institute For Neuroscience Neurological Institute, George Washington University Hospital

Neurotransmitter

No abstract provided.


The Hapless State Of Amyotrophic Lateral Sclerosis In India: A Comprehensive Look At Life And Medical Services For Als Patients In Rural Himachal Pradesh, Carson J. Bergström Oct 2019

The Hapless State Of Amyotrophic Lateral Sclerosis In India: A Comprehensive Look At Life And Medical Services For Als Patients In Rural Himachal Pradesh, Carson J. Bergström

Independent Study Project (ISP) Collection

Amyotrophic lateral sclerosis (ALS) or Lou Gehrig’s disease is a neurodegenerative disorder that leads to paralysis. The disease requires a high amount of medical intervention and interdisciplinary focus to achieve quality of life for patients. This study looks at ALS patient’s lifestyles, their access to these medical devices, Indian therapeutic approaches and policy that impacts patients in Shimla, Himachal Pradesh. As caretakers have a critical part in the care for this disease, their lives were also considered in the case studies. It was found that ALS patients are not experiencing access to medical devices necessary for life because of physical …


Neurologic Manifestations Of Auto-Immune Thyroiditis - A Rare Case Of Hashimoto's Encephalopathy, Mark Cohen, Roberta Santos, Yassar Chakfe Sep 2019

Neurologic Manifestations Of Auto-Immune Thyroiditis - A Rare Case Of Hashimoto's Encephalopathy, Mark Cohen, Roberta Santos, Yassar Chakfe

Neurology

No abstract provided.


Post-Dural Puncture Headache Complicated By Subdural Hematomas - A Case Report, Mark Cohen Md, Mays Alani Md, Amir Touray Md, Saif Ullah Md Sep 2019

Post-Dural Puncture Headache Complicated By Subdural Hematomas - A Case Report, Mark Cohen Md, Mays Alani Md, Amir Touray Md, Saif Ullah Md

Neurology

Post-dural puncture headache (PDPH) is a potential iatrogenic complication of procedures which disrupt meningeal integrity, particularly in obstetric patients, which are perhaps the highest-risk group. Common clinical manifestations include severe diffuse headache, neck stiffness, and nausea and vomiting; these symptoms, however, are non-specific and PDPH remains a diagnosis of exclusion that can be ascertained with a thorough history and proper consideration of other etiologies.


Oxidative Phosphorylation: A Critical Feature And Novel Therapeutic Target In Melanoma Brain Metastases, Grant Fischer Aug 2019

Oxidative Phosphorylation: A Critical Feature And Novel Therapeutic Target In Melanoma Brain Metastases, Grant Fischer

Dissertations and Theses (Open Access)

We recently showed via RNA-sequencing (RNA-seq) analysis of clinical samples that melanoma brain metastases (MBMs) have higher expression of oxidative phosphorylation (OXPHOS) genes (including PPARGC1A or PGC1α) than patient-matched extracranial metastases (ECMs). Thus, the central hypothesis of this dissertation is that OXPHOS plays a critical role in the pathogenesis of MBMs.

RNA-seq analysis identified increased expression of OXPHOS genes in intracranial (ICr) vs. subcutaneous (SQ) xenografts of 4 different human melanoma cell lines. Increased OXPHOS in the ICr xenografts was confirmed by direct metabolite analysis and [U-13C]-glucose tracing analysis. Together, these studies indicate that the brain TME …


Association Of Office And Ambulatory Blood Pressure With Mortality And Cardiovascular Outcomes, Wen-Yi Yang, Jesus D. Melgarejo, Lutgarde Thijs, Zhen-Yu Zhang, José Boggia, Fang-Fei Wei, Tine W. Hansen, Kei Asayama, Gladys E. Maestre, Takayoshi Ohkubo Aug 2019

Association Of Office And Ambulatory Blood Pressure With Mortality And Cardiovascular Outcomes, Wen-Yi Yang, Jesus D. Melgarejo, Lutgarde Thijs, Zhen-Yu Zhang, José Boggia, Fang-Fei Wei, Tine W. Hansen, Kei Asayama, Gladys E. Maestre, Takayoshi Ohkubo

School of Medicine Publications

Importance Blood pressure (BP) is a known risk factor for overall mortality and cardiovascular (CV)-specific fatal and nonfatal outcomes. It is uncertain which BP index is most strongly associated with these outcomes.

Objective To evaluate the association of BP indexes with death and a composite CV event.

Design, Setting, and Participants Longitudinal population-based cohort study of 11 135 adults from Europe, Asia, and South America with baseline observations collected from May 1988 to May 2010 (last follow-ups, August 2006-October 2016).

Exposures Blood pressure measured by an observer or an automated office machine; measured for 24 hours, during the day or …


Short Amylin Receptor Antagonist Peptides Improve Memory Deficits In Alzheimer’S Disease Mouse Model, Rania Soudy, Ryoichi Kimura, Aarti Patel, Wen Fu, Kamaljit Kaur, David Westaway, Jing Yang, Jack Jhamandas Jul 2019

Short Amylin Receptor Antagonist Peptides Improve Memory Deficits In Alzheimer’S Disease Mouse Model, Rania Soudy, Ryoichi Kimura, Aarti Patel, Wen Fu, Kamaljit Kaur, David Westaway, Jing Yang, Jack Jhamandas

Pharmacy Faculty Articles and Research

Recent evidence supports involvement of amylin and the amylin receptor in the pathogenesis of Alzheimer’s disease (AD). We have previously shown that amylin receptor antagonist, AC253, improves spatial memory in AD mouse models. Herein, we generated and screened a peptide library and identified two short sequence amylin peptides (12–14 aa) that are proteolytically stable, brain penetrant when administered intraperitoneally, neuroprotective against Aβ toxicity and restore diminished levels of hippocampal long term potentiation in AD mice. Systemic administration of the peptides for five weeks in aged 5XFAD mice improved spatial memory, reduced amyloid plaque burden, and neuroinflammation. The common residue SQELHRLQTY …


Perianal Necrotizing Fasciitis With Retroperitoneal Extension: A Case Report From Pakistan, Mirza Zain Baig, Abeer Aziz, Umme E Hani Abdullah, Muhammad Salman Khalil, Sumiya Abbasi Jul 2019

Perianal Necrotizing Fasciitis With Retroperitoneal Extension: A Case Report From Pakistan, Mirza Zain Baig, Abeer Aziz, Umme E Hani Abdullah, Muhammad Salman Khalil, Sumiya Abbasi

Section of Neurosurgery

Necrotizing fasciitis is a rare but potentially fatal condition. It is defined as a rapidly spreading infection of the subcutaneous soft tissue. Extension into the retroperitoneum may further complicate this deadly condition. We report a case of a 45-year-old gentleman who presented to our institute with perianal necrotizing fasciitis with extension into the retroperitoneum. He was managed with antibiotics and prompt surgical debridement. Our patient had a positive outcome which may be due to the fact that we had a high clinical suspicion, on the basis of which we opted for early operative management rather than delaying definitive treatment by …


A Technique For Approximating Transition Rates From Published Survival Analyses, Markian A. Pahuta, Joel Werier, Eugene K. Wai, Roy A. Patchell, Doug Coyle Jul 2019

A Technique For Approximating Transition Rates From Published Survival Analyses, Markian A. Pahuta, Joel Werier, Eugene K. Wai, Roy A. Patchell, Doug Coyle

Neurology Faculty Publications

Background: Quality-adjusted-life-years (QALYs) are used to concurrently quantify morbidity and mortality within a single parameter. For this reason, QALYs can facilitate the discussion of risks and benefits during patient counseling regarding treatment options. QALYs are often calculated using partitioned-survival modelling. Alternatively, QALYs can be calculated using more flexible and informative state-transition models populated with transition rates estimated using multistate modelling (MSM) techniques. Unfortunately the latter approach is considered not possible when only progression-free survival (PFS) and overall survival (OS) analyses are reported.

Methods: We have developed a method that can be used to estimate approximate transition rates from published PFS …


Recommendations For The Design Of Therapeutic Trials For Neonatal Seizures., Janet S. Soul, Ronit Pressler, Marilee Allen, Geraldine Boylan, Heike Rabe, Ron Portman, Pollyanna Hardy, Sarah Zohar, Klaus Romero, Brian Tseng, Varsha Bhatt-Mehta, Cecil Hahn, Scott Denne, Stephane Auvin, Alexander Vinks, John Lantos, Neil Marlow, Jonathan M. Davis, International Neonatal Consortium Jun 2019

Recommendations For The Design Of Therapeutic Trials For Neonatal Seizures., Janet S. Soul, Ronit Pressler, Marilee Allen, Geraldine Boylan, Heike Rabe, Ron Portman, Pollyanna Hardy, Sarah Zohar, Klaus Romero, Brian Tseng, Varsha Bhatt-Mehta, Cecil Hahn, Scott Denne, Stephane Auvin, Alexander Vinks, John Lantos, Neil Marlow, Jonathan M. Davis, International Neonatal Consortium

Manuscripts, Articles, Book Chapters and Other Papers

Although seizures have a higher incidence in neonates than any other age group and are associated with significant mortality and neurodevelopmental disability, treatment is largely guided by physician preference and tradition, due to a lack of data from well-designed clinical trials. There is increasing interest in conducting trials of novel drugs to treat neonatal seizures, but the unique characteristics of this disorder and patient population require special consideration with regard to trial design. The Critical Path Institute formed a global working group of experts and key stakeholders from academia, the pharmaceutical industry, regulatory agencies, neonatal nurse associations, and patient advocacy …