Medicine and Health Sciences Commons^™

Improving The Spiritual Dimension Of Whole Person Care: Reaching National And International Consensus, Christina M. Puchalski, Robert Vitillo, Sharon K. Hull, Nancy Reller

Medicine Faculty Publications

Two conferences, Creating More Compassionate Systems of Care (November 2012) and On Improving the Spiritual Dimension of Whole Person Care: The Transformational Role of Compassion, Love and Forgiveness in Health Care (January 2013), were convened with the goals of reaching consensus on approaches to the integration of spirituality into health care structures at all levels and development of strategies to create more compassionate systems of care. The conferences built on the work of a 2009 consensus conference, Improving the Quality of Spiritual Care as a Dimension of Palliative Care. Conference organizers in 2012 and 2013 aimed to identify consensus-derived care …

Controlling Cytomegalovirus: Helping The Immune System Take The Lead, Patrick J. Hanley, Catherine M. Bollard

Pediatrics Faculty Publications

Cytomegalovirus, of the Herpesviridae family, has evolved alongside humans for thousands of years with an intricate balance of latency, immune evasion, and transmission. While upwards of 70% of humans have evidence of CMV infection, the majority of healthy people show little to no clinical symptoms of primary infection and CMV disease is rarely observed during persistent infection in immunocompetent hosts. Despite the fact that the majority of infected individuals are asymptomatic, immunologically, CMV hijacks the immune system by infecting and remaining latent in antigen-presenting cells that occasionally reactivate subclinically and present antigen to T cells, eventually causing the inflation of …

Clinical Management Of Skin And Soft Tissue Infections In The U.S. Emergency Departments, Rakesh Mistry, Daniel J. Shapiro, Monika Goyal, Theoklis Zaoutis, Jeffrey Gerber, Catherine Liu, Adam L. Hersh

Emergency Medicine Faculty Publications

Community-associated methicillin resistant Staphylococcus aureus (CA-MRSA) has emerged as the most common cause of skin and soft-tissue infections (SSTI) in the United States. A nearly three-fold increase in SSTI visit rates had been documented in the nation's emergency departments (ED). The objective of this study was to determine characteristics associated with ED performance of incision and drainage (I+D) and use of adjuvant antibiotics in the management of skin and soft tissue infections (SSTI).

METHODS:

Cross-sectional study of the National Hospital Ambulatory Medical Care Survey, a nationally representative database of ED visits from 2007-09. Demographics, rates of I+D, and adjuvant antibiotic …

Medial Temporal Lobe Roles In Human Path Integration, Naohide Yamamoto, John W. Philbeck, Adam J. Woods, Daniel A. Gajewski, Joeanna C. Arthur, Samuel J. Potolicchio, Lucien M. Levy, Anthony J. Caputy

Neurological Surgery Faculty Publications

Path integration is a process in which observers derive their location by integrating self-motion signals along their locomotion trajectory. Although the medial temporal lobe (MTL) is thought to take part in path integration, the scope of its role for path integration remains unclear. To address this issue, we administered a variety of tasks involving path integration and other related processes to a group of neurosurgical patients whose MTL was unilaterally resected as therapy for epilepsy. These patients were unimpaired relative to neurologically intact controls in many tasks that required integration of various kinds of sensory self-motion information. However, the same …

Manganese-Containing Prussian Blue Nanoparticles For Imaging Of Pediatric Brain Tumors, Matthieu F. Dumont, Sridevi Yadavilli, Raymond Sze, Javad Nazarian, Rohan Fernandes

Radiology Faculty Publications

Pediatric brain tumors (PBTs) are a leading cause of death in children. For an improved prognosis in patients with PBTs, there is a critical need to develop molecularly-specific imaging agents to monitor disease progression and response to treatment. In this paper, we describe manganese-containing Prussian blue nanoparticles as agents for molecular magnetic resonance imaging (MRI) and fluorescence-based imaging of PBTs. Our core-shell nanoparticles consist of a core lattice structure that incorporates and retains paramagnetic Mn²⁺ ions, and generates MRI contrast (both negative and positive). The biofunctionalized shell is comprised of fluorescent avidin, which serves the dual purpose of enabling …

Response To Androgen Therapy In Patients With Dyskeratosis Congenita, Payal P. Khincha, Ingrid M. Wentzensen, Neelam Giri, Blanche P. Alter, Sharon A. Savage

Pediatrics Faculty Publications

Dyskeratosis congenita (DC) is an inherited bone marrow failure syndrome and telomere biology disorder characterized by dysplastic nails, reticular skin pigmentation and oral leucoplakia. Androgens are a standard therapeutic option for bone marrow failure in those patients with DC who are unable to undergo haematopoietic stem cell transplantation, but there are no systematic data on its use in those patients. We evaluated haematological response and side effects of androgen therapy in 16 patients with DC in our observational cohort study. Untreated DC patients served as controls. Seventy percent of treated DC patients had a haematological response with red blood cell …

Culture-Independent Evaluation Of The Appendix And Rectum Microbiomes In Children With And Without Appendicitis, Hope T. Jackson, Emmanuel F. Mongodin, Katherine P. Davenport, Claire M. Fraser, Anthony D. Sandler, Steven L. Zeichner

Surgery Faculty Publications

Purpose

The function of the appendix is largely unknown, but its microbiota likely contributes to function. Alterations in microbiota may contribute to appendicitis, but conventional culture studies have not yielded conclusive information. We conducted a pilot, culture-independent 16S rRNA-based microbiota study of paired appendix and rectal samples.

Methods

We collected appendix and rectal swabs from 21 children undergoing appendectomy, six with normal appendices and fifteen with appendicitis (nine perforated). After DNA extraction, we amplified and sequenced 16S rRNA genes and analyzed sequences using CLoVR. We identified organisms differing in relative abundance using ANOVA (p

Results

We identified 290 taxa in …

Neuroimaging Of Unusual Glioblastoma Using Diffusion Tensor Imaging, Akshita Mehta, Lucien M. Levy, M. Isabel Almira-Suarez, Raymond Tu

GW Research Days 2014

Background: Gliosarcoma refers to an uncommon astrocytic tumor of the central nervous system. These tumors include both glial and mesenchymal components by definition, and are extremely malignant. Gliosarcomas are particular to tumors with distinct gliomatous and sarcomatous constituents, and are distinguished from gliobastomas which have undergone mesenchymal metaplasia. Gliosarcomas encompass 2-8% of all glioblastoma cases and tend to occupy the supratentorial regions of the brain, especially the temporal lobe. Rare infratentorial lesions including the cerebellar hemisphere, intraventricular, and multi-focal tumors have also been reported. Accurate neuroimaging diagnosis is critical and diffusion tensor imaging (DTI) and spectroscopy can be useful to …

Improving Anatomical Knowledge Retention In Medical Students Entering The Surgery And Obstetrics And Gynecology Clinical Rotations By Nesting Interactive Modules, Samantha Ahle, Jill Krapf, Kirsten Marie Brown, Juliet Lee, Gisela Butera, Ellen Goldman, Myriam Ferzli, Rosalyn A. Jurjus

GW Research Days 2014

Background: It is well documented in the medical education literature that students retain minimal anatomy knowledge when transitioning from pre-clinical didactics to clinical application on the wards. In a previous study at our own institution we were able to quantify this knowledge deficit in students on surgical clerkships, specifically surgery and obstetrics and gynecology (OB/GYN). Using our previous data, we designed an integrated, nested clinical teaching model utilizing e-modules to review clinically relevant surgical anatomy during their clinical rotations.

Objective: The goal of this study is to evaluate the impact of a newly designed method of teaching clinically relevant anatomy …

Bilateral Laparoscopic Partial Adrenalectomy: A Case Presentation And Review Of The Literature, Sean Satey, Nathan Richards, Frederick J. Brody

Surgery Faculty Publications

Laparoscopic adrenalectomy is the standard of care for benign adrenal diseases. Partial adrenalectomy has been performed for various tumors to preserve adrenal function while avoiding steroid supplements postoperatively. This article discusses a patient that underwent bilateral laparoscopic partial adrenalectomies for bilateral pheochromocytomas and reviews the literature on partial adrenalectomy. A 40-year-old woman with a recent diagnosis of hypertension and an emergent Caesarian section for pre-eclampsia presented to an outside clinic with labile blood pressure, diaphoresis, palpitations, and headache. Physical examination was unremarkable with no bruits appreciated in the neck or abdomen. Medical management of her hypertension was initiated with minimal …

Childhood Achalasia: A Comprehensive Review Of Disease, Diagnosis And Therapeutic Management, Ashanti L. Franklin, Mikael Petrosyan, Timothy D. Kane

Surgery Faculty Publications

Achalasia is an esophageal motility disorder characterized by failure of lower esophageal sphincter (LES) relaxation and is rare in children. The most common symptoms are vomiting, dysphagia, regurgitation, and weight loss. Definitive diagnosis is made with barium swallow study and esophageal manometry. In adults, endoscopic biopsy is recommended to exclude malignancy however; it is not as often indicated in children. Medical management often fails resulting in recurrent symptoms and the ultimate definitive treatment is surgical. Laparoscopic Heller myotomy with or without an anti-reflux procedure is the treatment of choice and has become standard of care for children with achalasia. Peroral …

Transoral Incisionless Fundoplication Effective In Eliminating Gerd Symptoms In Partial Responders To Proton Pump Inhibitor Therapy At 6 Months: The Tempo Randomized Clinical Trial, Karim S. Trad, William E. Barnes, Gilbert Simoni, Ahmad B. Shughoury, Peter G. Mavrelis, Mamoon Raza, Jeffrey Heise, Daniel G. Turgeon, Mark A. Fox

Surgery Faculty Publications

Background. Incomplete control of troublesome regurgitation and extraesophageal manifestations of chronic gastroesophageal reflux disease (GERD) is a known limitation of proton pump inhibitor (PPI) therapy. This multicenter randomized study compared the efficacy of transoral incisionless fundoplication (TIF) against PPIs in controlling these symptoms in patients with small hiatal hernias.

Methods. Between June and August 2012, 63 patients were randomized at 7 US community hospitals. Patients in the PPI group were placed on maximum standard dose (MSD). Patients in the TIF group underwent esophagogastric fundoplication using the EsophyX₂ device. Primary outcome was elimination of daily troublesome regurgitation or …

Redefining Frequent Emergency Department Users, Amy Waldner, Maria Raven, Danielle Lazar, Jesse Pines

Center for Health Care Quality

Frequent ED users are perceived to be a costly population that often abuse or misuse ED services due to a combination of unmet social needs and medical conditions that, in theory, could be treated outside of the ED at a lower cost. The reality is that factors contributing to frequent ED use are more varied and complex than originally believed.

The Impact Of Salsalate Treatment On Serum Levels Of Advanced Glycation End Products In Type 2 Diabetes., Joshua I Barzilay, Kathleen A. Jablonski, Vivian Fonseca, Steven E Shoelson, Allison B Goldfine, Christopher Strauch, Vincent M Monnier, Tinsal-T2d Research Consortium.

GW Biostatistics Center

OBJECTIVE Salsalate is a nonacetylated salicylate that lowers glucose levels in people with type 2 diabetes (T2D). Here we examined whether salsalate also lowered serum-protein-bound levels of early and advanced glycation end products (AGEs) that have been implicated in diabetic vascular complications. RESEARCH DESIGN AND METHODS Participants were from the Targeting Inflammation Using Salsalate for Type 2 Diabetes (TINSAL-T2D) study, which examined the impact of salsalate treatment on hemoglobin A1c (HbA1c) and a wide variety of other parameters. One hundred eighteen participants received salsalate, 3.5 g/day for 48 weeks, and 109 received placebo. Early glycation product levels (HbA1c and fructoselysine …

Genetic Risk Of Progression To Type 2 Diabetes And Response To Intensive Lifestyle Or Metformin In Prediabetic Women With And Without A History Of Gestational Diabetes Mellitus., Shannon D Sullivan, Kathleen A. Jablonski, Jose C Florez, Dana Dabelea, Paul W Franks, Sam Dagogo-Jack, Catherine Kim, William C Knowler, Costas A Christophi, Robert Ratner, Diabetes Prevention Program Research Group.

GW Biostatistics Center

OBJECTIVE The Diabetes Prevention Program (DPP) trial investigated rates of progression to diabetes among adults with prediabetes randomized to treatment with placebo, metformin, or intensive lifestyle intervention. Among women in the DPP, diabetes risk reduction with metformin was greater in women with prior gestational diabetes mellitus (GDM) compared with women without GDM but with one or more previous live births.

RESEARCH DESIGN AND METHODS We asked if genetic variability could account for these differences by comparing β-cell function and genetic risk scores (GRS), calculated from 34 diabetes-associated loci, between women with and without histories of GDM.

RESULTS β-Cell function was …

Ultrasound-Enhanced Ocular Delivery Of Dexamethasone Sodium Phosphate: An In Vivo Study, Marjan Nabili, Aditi Shenoy, Shawn Chawla, Sankaranarayana P. Mahesh, Craig Geist, Vesna Zderic

Ophthalmology Faculty Publications

Background

The eye's unique anatomy and its physiological and anatomical barriers can limit effective drug delivery into the eye.

Methods

An in vivo study was designed to determine the effectiveness and safety of ultrasound application in enhancing drug delivery in a rabbit model. Permeability of a steroid ophthalmic drug, dexamethasone sodium phosphate, was investigated in ultrasound- and sham-treated cases. For this study, an eye cup filled with dexamethasone sodium phosphate was placed on the cornea. Ultrasound was applied at intensity of 0.8 W/cm² and frequency of 400 or 600 kHz for 5 min. The drug concentration in aqueous humor …

International Telemedicine Consultations For Neurodevelopmental Disabilities, Phillip L. Pearl, Craig A. Sable, Sarah Helen Evans, Joseph Knight, Parker Cunningham, Gaetano R. Lotrecchiano, Andrea L. Gropman, Sheela Stuart, Penny J. Glass, Anne Conway, Issam Ramadan, Tania Paiva, Mark L. Batshaw, Roger J. Packer

Neurology Faculty Publications

Background: A telemedicine program was developed between the Children's National Medical Center (CNMC) in Washington, DC, and the Sheikh Khalifa Bin Zayed Foundation in the United Arab Emirates (UAE). A needs assessment and a curriculum of on-site training conferences were devised preparatory to an ongoing telemedicine consultation program for children with neurodevelopmental disabilities in the underserved eastern region of the UAE.

Materials and Methods: Weekly telemedicine consultations are provided by a multidisciplinary faculty. Patients are presented in the UAE with their therapists and families. Real-time (video over Internet protocol; average connection, 768 kilobits/s) telemedicine conferences are held weekly following previews …

Indirect Cyclopexy For Treatment Of A Chronic Traumatic Cyclodialysis Cleft With Hypotony, Nisha Chadha, Tania Lamba, David A. Belyea, Kunal Y. Merchant

Ophthalmology Faculty Publications

Cyclodialysis cleft is a rare clinical finding and therefore, reports on surgical repair techniques in the literature are limited. Additionally, hypotony can make repair technically challenging. We share a novel, simple surgical approach to management of a case of chronic traumatic cyclodialysis cleft with a successful outcome.

Microfluidic-Enabled Liposomes Elucidate Size-Dependent Transdermal Transport, Renee R. Hood, Eric L. Kendall, Mariana Junqueira, Wyatt N. Vreeland, Zenaide M.N. Quezado, Julia C. Finkel, Don L. Devoe

Pediatrics Faculty Publications

Microfluidic synthesis of small and nearly-monodisperse liposomes is used to investigate the size-dependent passive transdermal transport of nanoscale lipid vesicles. While large liposomes with diameters above 105 nm are found to be excluded from deeper skin layers past the stratum corneum, the primary barrier to nanoparticle transport, liposomes with mean diameters between 31–41 nm exhibit significantly enhanced penetration. Furthermore, multicolor fluorescence imaging reveals that the smaller liposomes pass rapidly through the stratum corneum without vesicle rupture. These findings reveal that nanoscale liposomes with well-controlled size and minimal size variance are excellent vehicles for transdermal delivery of functional nanoparticle drugs.

The Temporal Version Of The Pediatric Sepsis Biomarker Risk Model, Hector R. Wong, Scott L. Weiss, John S. Giuliano Jr., Mark S. Wainwright, Natalie Z. Cvijanovich, Robert J. Freishtat, +19 Additional Authors

Pediatrics Faculty Publications

Background

PERSEVERE is a risk model for estimating mortality probability in pediatric septic shock, using five biomarkers measured within 24 hours of clinical presentation.

Objective

Here, we derive and test a temporal version of PERSEVERE (tPERSEVERE) that considers biomarker values at the first and third day following presentation to estimate the probability of a “complicated course”, defined as persistence of ≥2 organ failures at seven days after meeting criteria for septic shock, or death within 28 days.

Methods

Biomarkers were measured in the derivation cohort (n = 225) using serum samples obtained during days 1 and 3 of septic shock. …

Challenging Situations In Family Centered Rounds: Making The Best Out Of Worst Case Scenarios, Priti Bhansali, Kerry Moss, Christiane Corriveau, Geeta Singhal, Robert Kavanagh, Jeffrey Simmons

E-Learning Modules

This resource is a workshop intended for both seasoned and novice health care providers who are interested in becoming prepared for challenging scenarios that may be encountered during FCR. Case scenarios of challenging situations relating to discussing sensitive topics, correcting errors, and teaching learners in front of both parents and patients are presented. Tangible opportunities to role model communication skills, efficiency, and teaching skills for trainees are highlighted. The session will conclude with a question and answer session with a multi-specialty panel to share experiences and strategies for addressing the unexpected during FCR. By the end of the workshop, participants …

Genomics In Premature Infants: A Non-Invasive Strategy To Obtain High-Quality Dna, Mariam M. Said, Clint D. Cappiello, Joseph M. Devaney, Daniele S. Podini, Alana L. Beres, Stanislav Vukmanovic, Khodayar Rais-Bahrami, Naomi L. Luban, Anthony D. Sandler, Zohreh Tatari-Calderone

Pediatrics Faculty Publications

We used a cost-effective, non-invasive method to obtain high-quality DNA from buccal epithelial-cells (BEC) of premature infants for genomic analysis. DNAs from BEC were obtained from premature infants with gestational age ≤ 36 weeks. Short terminal repeats (STRs) were performed simultaneously on DNA obtained from the buccal swabs and blood from the same patient. The STR profiles demonstrated that the samples originated from the same individual and exclude any contamination by external DNAs. Whole exome sequencing was performed on DNAs obtained from BEC on premature infants with and without necrotizing enterocolitis, and successfully provided a total number of reads and …

Do Not Judge A Kidney By Its Pelvis: A Rare Case Of An Unobstructed Dilated Renal Pelvis, Aaron D. Martin, Craig A. Peters

Urology Faculty Publications

We present a rare case of an unobstructed dilated renal pelvis in a newborn female. Prenatal imaging documented a large abdominal cyst in a stable fetus. Postnatal imaging confirmed persistence of this large cyst but with an unclear etiology. The child was taken to surgery by the pediatric surgeons with concern for a possible harmful nonurologic diagnosis. Intraoperative findings were that of a severely dilated renal pelvis; however, in the absence of an expected ureteropelvic junction obstruction. Reduction pyeloplasty without interference of the ureteropelvic junction proved successful.

Trends In Antibiotic Resistance In Coagulase-Negative Staphylococci In The United States, 1999 To 2012, Larissa S. May, Eili Y. Klein, Richard E. Rothman, Ramanan Laxminarayan

Emergency Medicine Faculty Publications

Coagulase-negative staphylococci (CoNS) are important bloodstream pathogens that are typically resistant to multiple antibiotics. Despite the concern about increasing resistance, there have been no recent studies describing the national prevalence of CoNS pathogens. We used national resistance data over a period of 13 years (1999 to 2012) from The Surveillance Network (TSN) to determine the prevalence of and assess the trends in resistance for Staphylococcus epidermidis, the most common CoNS pathogen, and all other CoNS pathogens. Over the course of the study period, S. epidermidis resistance to ciprofloxacin and clindamycin increased steadily from 58.3% to 68.4% and from 43.4% to …

Child And Adolescent Psychiatrists' Attitudes And Practices Prescribing Second Generation Antipsychotics, Angie Mae Rodday, Susan K. Parsons, Christoph U. Correll, Adelaide S. Robb, Bonnie T. Zima, Tully S. Saunders, Laurel K. Leslie

Psychiatry and Behavioral Sciences Faculty Publications

Objective: The purpose of this study was to examine psychiatrists' attitudes and practices in prescribing second-generation antipsychotics (SGA) to children and adolescents (referred to here as “children”) and identify factors associated with off-label SGA use.

Methods: A survey was mailed to a national, randomly selected sample of 1600 child and adolescent psychiatrists identified by the American Medical Association. Multivariable logistic regression was used to identify factors, including psychiatrists' characteristics, practice characteristics, and psychiatrists' attitudes, that are associated with off-label SGA use (i.e., SGAs used in children with attention-deficit/hyperactivity disorder (ADHD), oppositional defiant disorder, conduct disorder, or nonbipolar mood disorders).

Results: …

Long-Term Safety Of Ospemifene (52-Week Extension) In The Treatment Of Vulvar And Vaginal Atrophy In Hysterectomized Postmenopausal Women, James A. Simon, David J. Portman, R. Garn Mabey, The Ospemifene Study Group

Obstetrics and Gynecology Faculty Publications

Objective

To examine the long-term safety of oral ospemifene, a non-estrogen tissue-selective estrogen agonist/antagonist, for the treatment of moderate to severe dyspareunia, a symptom of vulvar and vaginal atrophy (VVA) due to menopause.

Study design

This multicenter, long-term, open-label, safety extension study was conducted in women without a uterus aged 40–80 years (N = 301) who received oral ospemifene 60 mg/day for 52 weeks. Participants either continued their 60-mg/day ospemifene dose from the initial 12-week pivotal efficacy study or switched from blinded placebo or ospemifene 30 mg/day to open-label ospemifene 60 mg/day. The 52-week open-label extension period plus initial …

A Melanin-Independent Interaction Between Mc1r And Met Signalling Pathways Is Required For Hgf-Dependent Melanoma, Agnieszka Wolnicka-Głubisz, Faith M. Strickland, Albert Wielgus, Miriam Anver, Glenn Merlino, Edward C. De Fabo, Frances P. Noonan

Microbiology, Immunology, and Tropical Medicine Faculty Publications

Melanocortin 1 receptor (MC1R) signaling stimulates black eumelanin production through a cAMP-dependent pathway. MC1R polymorphisms can impair this process, resulting in a predominance of red phaeomelanin. The red hair, fair skin and UV sensitive phenotype is a well-described melanoma risk factor. MC1R polymorphisms also confer melanoma risk independent of pigment. We investigated the effect of Mc1r deficiency in a mouse model of UV-induced melanoma. C57BL/6-Mc1r+/+-HGF transgenic mice have a characteristic hyperpigmented black phenotype with extra-follicular dermal melanocytes located at the dermal/epidermal junction. UVB induces melanoma, independent of melanin pigmentation, but UVA-induced and spontaneous melanomas are dependent on black eumelanin. We …

Gaps And Opportunities In Refractory Status Epilepticus Research In Children: A Multi-Center Approach By The Pediatric Status Epilepticus Research Group (Pserg), Ivan Sanchez Fernandez, Nicholas S. Abend, Satish Agadi, Sookee An, Ravindra Arya, Jessica L. Carpenter, Kevin E. Chapman, William D. Gaillard, Tracy A. Glauser, David B. Golstein, Joshua L. Goldstein, Howard P. Goodkin, Cecil D. Hahn, Erin L. Heinzen, Mohamad A. Mikati, Katrina Peariso, John P. Pestian, Margie Ream, James J. Riviello, Robert C. Tasker, Korwyn Williams, Tobias Loddenkemper

Neurology Faculty Publications

PURPOSE:

Status epilepticus (SE) is a life-threatening condition that can be refractory to initial treatment. Randomized controlled studies to guide treatment choices, especially beyond first-line drugs, are not available. This report summarizes the evidence that guides the management of refractory convulsive SE (RCSE) in children, defines gaps in our clinical knowledge and describes the development and works of the 'pediatric Status Epilepticus Research Group' (pSERG).

METHODS:

A literature review was performed to evaluate current gaps in the pediatric SE and RCSE literature. In person and online meetings helped to develop and expand the pSERG network.

RESULTS:

The care of pediatric …

Phase 2 Randomized, Flexible Crossover, Double-Blinded, Placebo-Controlled Trial Of The Farnesyltransferase Inhibitor Tipifarnib In Children And Young Adults With Neurofibromatosis Type 1 And Progressive Plexiform Neurofibromas, Brigitte C. Widemann, Eva Dombi, Andrea Gillespie, Pamela L. Wolters, Jean Belasco, Aerang Kim, +13 Additional Authors

Pediatrics Faculty Publications

Background

RAS is dysregulated in neurofibromatosis type 1 (NF1) related plexiform neurofibromas (PNs). The activity of tipifarnib, which blocks RAS signaling by inhibiting its farnesylation, was tested in children and young adults with NF1 and progressive PNs.

Methods

Patients aged 3–25 years with NF1-related PNs and imaging evidence of tumor progression were randomized in a double-blinded fashion to receive tipifarnib (200 mg/m² orally every 12 h) or placebo (phase A) and crossed over to the opposite treatment arm at the time of tumor progression (phase B). PN volumes were measured with MRI, and progression was defined as ≥20% volume …

Spirituality And Health, Arndt Bussing, Klaus Baumann, Niels Christian Hvidt, Harold G. Koenig, Christina M. Puchalski, John Swinton

Medicine Faculty Publications

No abstract provided.