Digital Commons Network^™

Prevalence Of Trypanosoma Cruzi, The Etiologic Agent Of Chagas Disease, Infection In Texas Skunks (Mammalia: Mephitidae), Bonnie E Gulas-Wroblewski, Rodion Gorchakov, Rebecca B Kairis, Robert C Dowler, Kristy O Murray

Faculty, Staff and Students Publications

Background:

Chagas disease is one of the world's most neglected tropical diseases, infecting over six million people across the Americas. The hemoparasite Trypanosoma cruzi is the etiological agent for the disease, circulating in domestic, peridomestic, and sylvatic transmission cycles that are maintained by triatomine vectors and a diversity of wild and synanthropic hosts. Public health and wildlife management interventions targeting the interruption of T. cruzi transmission rely on an understanding of the dynamics driving the ecology of this zoonotic pathogen. One wildlife host that purportedly plays a role in the transmission of Chagas disease within the southern United States is …

Participant Perceptions Of Changes In Psychosocial Domains Following Participation In An Adaptive Deep Brain Stimulation Trial, Amanda R Merner, Kristin Kostick-Quenet, Tiffany A Campbell, Michelle T Pham, Clarissa E Sanchez, Laura Torgerson, Jill Robinson, Stacey Pereira, Simon Outram, Barbara A Koenig, Philip A Starr, Aysegul Gunduz, Kelly D Foote, Michael S Okun, Wayne Goodman, Amy L Mcguire, Peter Zuk, Gabriel Lázaro-Muñoz

Faculty, Staff and Students Publications

BACKGROUND: There has been substantial controversy in the neuroethics literature regarding the extent to which deep brain stimulation (DBS) impacts dimensions of personality, mood, and behavior.

OBJECTIVE/HYPOTHESIS: Despite extensive debate in the theoretical literature, there remains a paucity of empirical data available to support or refute claims related to the psychosocial changes following DBS.

METHODS: A mixed-methods approach was used to examine the perspectives of patients who underwent DBS regarding changes to their personality, authenticity, autonomy, risk-taking, and overall quality of life.

RESULTS: Patients (n = 21) who were enrolled in adaptive DBS trials for Parkinson's disease, essential tremor, obsessive-compulsive …

Perceived Care Partner Burden At 1-Year Post-Injury And Associations With Emotional Awareness, Functioning, And Empathy After Tbi: A Tbi Model Systems Study, Daniel W Klyce, Kritzianel Merced, Alexander Erickson, Dawn M Neumann, Flora M Hammond, Angelle M Sander, Jennifer A Bogner, Tamara Bushnik, Joyce S Chung, Jacob A Finn

Faculty, Staff and Students Publications

BACKGROUND: People with traumatic brain injury (TBI) can lack awareness of their own emotions and often have problems with emotion dysregulation, affective disorders, and empathy deficits. These impairments are known to impact psychosocial behaviors and may contribute to the burden experienced by care partners of individuals with TBI.

OBJECTIVE: To examine the associations of emotional awareness, emotional functioning, and empathy among participants with TBI with care partner burden.

METHOD: This multisite, cross-sectional, observational study used data from 90 dyads (participants with TBI and their care partner) 1-year post-injury. Participants with TBI completed the Difficulty with Emotional Regulation Scale (DERS; Awareness, …

Prefrontal Network Engagement By Deep Brain Stimulation In Limbic Hubs, Anusha Allawala, Kelly R Bijanki, Denise Oswalt, Raissa K Mathura, Joshua Adkinson, Victoria Pirtle, Ben Shofty, Meghan Robinson, Matthew T Harrison, Sanjay J Mathew, Wayne K Goodman, Nader Pouratian, Sameer A Sheth, David A Borton

Faculty, Staff and Students Publications

Prefrontal circuits in the human brain play an important role in cognitive and affective processing. Neuromodulation therapies delivered to certain key hubs within these circuits are being used with increasing frequency to treat a host of neuropsychiatric disorders. However, the detailed neurophysiological effects of stimulation to these hubs are largely unknown. Here, we performed intracranial recordings across prefrontal networks while delivering electrical stimulation to two well-established white matter hubs involved in cognitive regulation and depression: the subcallosal cingulate (SCC) and ventral capsule/ventral striatum (VC/VS). We demonstrate a shared frontotemporal circuit consisting of the ventromedial prefrontal cortex, amygdala, and lateral orbitofrontal …

Transcriptomic Profiles Of Multiple Organ Dysfunction Syndrome Phenotypes In Pediatric Critical Influenza, Tanya Novak, Jeremy Chase Crawford, Georg Hahn, Mark W Hall, Simone A Thair, Margaret M Newhams, Janet Chou, Peter M Mourani, Keiko M Tarquinio, Barry Markovitz, Laura L Loftis, Scott L Weiss, Renee Higgerson, Adam J Schwarz, Neethi P Pinto, Neal J Thomas, Rainer G Gedeit, Ronald C Sanders, Sidharth Mahapatra, Bria M Coates, Natalie Z Cvijanovich, Kate G Ackerman, David W Tellez, Patrick Mcquillen, Stephen C Kurachek, Steven L Shein, Christoph Lange, Paul G Thomas, Adrienne G Randolph

Faculty, Staff and Students Publications

BACKGROUND: Influenza virus is responsible for a large global burden of disease, especially in children. Multiple Organ Dysfunction Syndrome (MODS) is a life-threatening and fatal complication of severe influenza infection.

METHODS: We measured RNA expression of 469 biologically plausible candidate genes in children admitted to North American pediatric intensive care units with severe influenza virus infection with and without MODS. Whole blood samples from 191 influenza-infected children (median age 6.4 years, IQR: 2.2, 11) were collected a median of 27 hours following admission; for 45 children a second blood sample was collected approximately seven days later. Extracted RNA was hybridized …

Cc16 Drives Vla-2-Dependent Splunc1 Expression, Natalie Iannuzo, Holly Welfley, Nicholas C Li, Michael D L Johnson, Joselyn Rojas-Quintero, Francesca Polverino, Stefano Guerra, Xingnan Li, Darren A Cusanovich, Paul R Langlais, Julie G Ledford

Faculty, Staff and Students Publications

RATIONALE: CC16 (Club Cell Secretory Protein) is a protein produced by club cells and other non-ciliated epithelial cells within the lungs. CC16 has been shown to protect against the development of obstructive lung diseases and attenuate pulmonary pathogen burden. Despite recent advances in understanding CC16 effects in circulation, the biological mechanisms of CC16 in pulmonary epithelial responses have not been elucidated.

OBJECTIVES: We sought to determine if CC16 deficiency impairs epithelial-driven host responses and identify novel receptors expressed within the pulmonary epithelium through which CC16 imparts activity.

METHODS: We utilized mass spectrometry and quantitative proteomics to investigate how CC16 deficiency …

Burnout During The Covid-19 Pandemic: A Report On Pediatric Residents, Linessa M Zuniga, Abigail Schuh, Alan Schwartz, Patricia Seo-Mayer, Rachel Cramton, Kira Sieplinga, Ruchi Kaushik, Lauren Nassetta, Jason H Homme, Jessica Babal, John D Mahan, Maneesh Batra

Faculty, Staff and Students Publications

Background and objective: The Pediatric Resident Burnout and Resilience Consortium (PRB-RSC) has described the epidemiology of burnout in pediatric residents since 2016. We hypothesized burnout rates during the pandemic would increase. We explored resident burnout during the COVID-19 pandemic and its relationship to resident perception of workload, training, personal life, and local COVID burden.

Methods: Since 2016, PRB-RSC has sent an annual, confidential survey to over 30 pediatric and medicine-pediatrics residencies. In 2020 and 2021, seven questions were added to explore the relationship of COVID-19 and perceptions of workload, training, and personal life.

Results: In 2019, 46 programs participated, 22 …

Lox Upregulates Fak Phosphorylation To Promote Metastasis In Osteosarcoma, Xiang Chen, Margaret Clement, M John Hicks, Poonam Sarkar, M Waleed Gaber, Tsz-Kwong Man

Faculty, Staff and Students Publications

Osteosarcoma is a malignant bone tumor that commonly occurs in the pediatric population. Despite the use of chemotherapy and surgery, metastasis remains to be the leading cause of death in patients with osteosarcoma. We have previously reported that cytoplasmic mislocalization of p27 is associated with a poor outcome in osteosarcoma. In this study, we further show that lysyl oxidase (LOX) expression was associated with p27 mislocalization. LOX is an enigmatic molecule that acts as a tumor suppressor or a metastatic promoter; however, its role in osteosarcoma is still unclear. Hence, we performed both

Resident Education And Virtual Medicine: A Faculty Development Session To Enhance Trainee Skills In The Realm Of Telemedicine, Anna E Rueda, Ana C Monterrey, Margaret Wood, Linessa Zuniga, Betty Del Rio Rodriguez

Faculty, Staff and Students Publications

INTRODUCTION: COVID-19 accelerated the use of telemedicine. Subsequently, clinical sites began conducting virtual visits. Academic institutions implemented telemedicine for patient care and simultaneously had to teach residents the logistics and best practices. To meet this need, we developed a training session for faculty focused on telemedicine best practices and teaching telemedicine in the pediatric realm.

METHODS: We designed this training session based on institutional and society guidelines and faculty experience with telemedicine. Objectives included telemedicine documentation, triage, counseling, and ethical issues in telemedicine. We conducted all sessions in a 60-minute or 90-minute format over a virtual platform for small and …

Combined Immunodeficiency Caused By Pathogenic Variants In The Zap70 C-Terminal Sh2 Domain, Cédric Mongellaz, Rita Vicente, Lenora M Noroski, Nelly Noraz, Valérie Courgnaud, Javier Chinen, Emilia Faria, Valérie S Zimmermann, Naomi Taylor

Faculty, Staff and Students Publications

INTRODUCTION: ZAP-70, a protein tyrosine kinase recruited to the T cell receptor (TCR), initiates a TCR signaling cascade upon antigen stimulation. Mutations in the ZAP70 gene cause a combined immunodeficiency characterized by low or absent CD8+ T cells and nonfunctional CD4+ T cells. Most deleterious missense ZAP70 mutations in patients are located in the kinase domain but the impact of mutations in the SH2 domains, regulating ZAP-70 recruitment to the TCR, are not well understood.

METHODS:Genetic analyses were performed on four patients with CD8 lymphopenia and a high resolution melting screening for ZAP70 mutations was developed. The impact of SH2 …

Review Of Acute Kidney Injury And Progression To Chronic Kidney Disease In Pediatric Patients Undergoing Hematopoietic Cell Transplant, Kim T Vuong, Catherine Joseph, Joseph R Angelo

Faculty, Staff and Students Publications

While acute kidney injury (AKI) after hematopoietic cell transplant (HCT) has been well-described in pediatric patients, literature regarding the long term renal consequences of HCT-related AKI, the development of chronic kidney disease (CKD), and CKD care in pediatric patients post-HCT is limited. CKD affects almost 50% of patients after HCT with multifactorial etiology including infection, nephrotoxic medications, transplant-associated thrombotic microangiopathy, graft-versus-host disease, and sinusoidal obstruction syndrome. As renal function declines in CKD, eventually progressing to end stage kidney disease (ESKD), mortality increases and is more than 80% among patients requiring dialysis. Using society guidelines and current literature, this review summarizes …

A Pregnant Patient Presents With Leakage Of Vaginal Fluid In The Second Trimester, Alison N Goulding, Jennifer R Mckinney, Diana Crabtree Sokol, Anitra D Beasley

Faculty, Staff and Students Publications

Morning Report is a time-honored tradition where physicians-in-training present cases to their colleagues and clinical experts to collaboratively examine an interesting patient presentation. The Morning Report section seeks to carry on this tradition by presenting a patient’s chief concern and story, inviting the reader to develop a differential diagnosis and discover the diagnosis alongside the authors of the case.

This report examines the story of a 43-year-old (gravida 3, para 2) woman who sought evaluation for leakage of vaginal fluid at 15 weeks of gestation. Using questions, physical examination, and testing, an illness script for the presentation emerges. As her …

Effectiveness Of Elbasvir/Grazoprevir Plus Ribavirin For Hepatitis C Virus Genotype 1a Infection And Baseline Ns5a Resistance, Deanna D Hill, Jennifer R Kramer, Kassie R Chaffin, T Christopher Mast, Michael N Robertson, Fasiha Kanwal, Barbara A Haber

Faculty, Staff and Students Publications

Introduction and objectives: In clinical trials, patients with hepatitis C virus (HCV) genotype (GT)1a infection and baseline resistance-associated substitutions (RASs) at amino acid positions 28, 30, 31, or 93 receiving elbasvir/grazoprevir for 12 weeks achieved lower rates of sustained virologic response (SVR) than those without baseline RASs. SVR rates in patients with RASs were improved when elbasvir/grazoprevir treatment duration was extended from 12 to 16 weeks and administered concomitantly with ribavirin.

Materials and methods: This was a retrospective, observational analysis using electronic health record abstraction. Patients with HCV GT1a infection and RASs at positions 28, 30, 31, or 93 who …

Proceedings Of The 2023 Santa Fe Bone Symposium: Progress And Controversies In The Management Of Patients With Skeletal Diseases, E Michael Lewiecki, Teresita Bellido, John P Bilezikian, Jacques P Brown, Azeez Farooki, Christopher S Kovacs, Brendan Lee, William D Leslie, Michael R Mcclung, Mark L Prasarn, Deborah E Sellmeyer

Faculty, Staff and Students Publications

The Santa Fe Bone Symposium (SFBS) held its 23rd annual event on August 5-6, 2023, in Santa Fe, New Mexico, USA. Attendees participated in-person and remotely, representing many states and countries. The program included plenary presentations, panel discussions, satellite symposia, a Project ECHO workshop, and a session on healthcare policy and reimbursement for fracture liaison programs. A broad range of topics were addressed, including transitions of osteoporosis treatments over a lifetime; controversies in vitamin D; update on Official Positions of the International Society for Clinical Densitometry; spine surgery and bone health; clinical applications of bone turnover markers; basic bone biology …

The Neurobiology Of Duration Of Untreated Psychosis: A Comprehensive Review, Anthony W Zoghbi, Jeffrey A Lieberman, Ragy R Girgis

Faculty, Staff and Students Publications

Duration of untreated psychosis (DUP) is defined as the time from the onset of psychotic symptoms until the first treatment. Studies have shown that longer DUP is associated with poorer response rates to antipsychotic medications and impaired cognition, yet the neurobiologic correlates of DUP are poorly understood. Moreover, it has been hypothesized that untreated psychosis may be neurotoxic. Here, we conducted a comprehensive review of studies that have examined the neurobiology of DUP. Specifically, we included studies that evaluated DUP using a range of neurobiologic and imaging techniques and identified 83 articles that met inclusion and exclusion criteria. Overall, 27 …

Codon-Optimized Tdp-43 Mediates Neurodegeneration In A Drosophila Model Of Als/Ftld, Tanzeen Yusuff, Ya-Chu Chang, Tzu-Kang Sang, George R Jackson, Shreyasi Chatterjee

Faculty, Staff and Students Publications

Transactive response DNA binding protein-43 (TDP-43) is known to mediate neurodegeneration associated with amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration (FTLD). The exact mechanism by which TDP-43 exerts toxicity in the brains, spinal cord, and lower motor neurons of affected patients remains unclear. In a novel Drosophila melanogaster model, we report gain-of-function phenotypes due to misexpression of insect codon-optimized version of human wild-type TDP-43 (CO-TDP-43) using both the binary GAL4/UAS system and direct promoter fusion constructs. The CO-TDP-43 model showed robust tissue specific phenotypes in the adult eye, wing, and bristles in the notum. Compared to non-codon optimized …

Unmasking Barriers In The Delivery Of Preconception Counseling And Contraception Provision For Patients With Type 1 Or Type 2 Diabetes, Layla A Abushamat, Lauren Sayres, Rebecca Jeffers, Chloe Nielsen, Linda A Barbour, Adnin Zaman

Faculty, Staff and Students Publications

No abstract provided.

Editorial: Hemostasis In Ecmo And Vad, Volume Ii, Jun Teruya, Jean M Connors, Barbara Zieger

Faculty, Staff and Students Publications

No abstract provided.

Existential Distress In Family Caregivers: Scoping Review Of Meaning-Making Interventions, Natalie S. Mcandrew, Tamryn F. Gray, Lyndsey Wallace, Kelly Calkins, Jill L. Guttormson, Eric Harding, Allison J. Applebaum

College of Nursing Faculty Research and Publications

Objectives: Family and friend caregivers often feel overwhelmed by and ill-prepared for their responsibilities. Many feel helpless living with uncertainty about the outcome of the patient's illness, which leads to existential distress. Supportive care interventions that address existential distress by promoting meaning and purpose buffer the negative effects of caregiver burden and promote resilience and growth. The purpose of this scoping review is to describe the depth and breadth of available interventions targeting caregiver existential distress.

Methods: We followed the Joanna Briggs Institute's scoping review methods and applied the Preferred Reporting Items for Systematic Reviews and Meta-Analyses Extension checklist. SCOPUS, …

Sprinting In A Marathon: Nursing Staff And Nurse Leaders Make Meaning Of Practicing In Covid-19 Devoted Units Pre-Vaccine, Natalie S. Mcandrew, William E. Rosa, Kaylen M. Moore, Jacqueline Christianson, Tala Abuzahra, Megann Mussatti, Colleen Mccracken, Amy R. Newman, Kelly Calkins, Susan Breakwell, Katie Klink, Jill L. Guttormson

College of Nursing Faculty Research and Publications

Objective

To describe the lived experience of nursing staff and nurse leaders working in COVID-19 devoted units (intensive care or medical unit) prior to vaccine availability.

Research Design

Qualitative phenomenological design with a focus group approach.

Methods

The study team recruited a convenience sample of nursing staff (nurses, and nursing assistants/nurse technicians) and nurse leaders (managers, assistant nurse managers, clinical nurse specialists, and nurse educators) at an academic medical center in the midwestern United States. Focus groups and individual interviews were conducted to encourage participants to describe their (1) experiences as nursing professionals, (2) coping strategies, and (3) perspectives about …

Sickle Cell Virtual Support Group Program For Adults: An Implementation Evaluation, Dora L. Clayton-Jones, Lee Za Ong, Abir K. Bekhet, Weneaka D. Jones, Elodie Ontala, James Griffin, Ethlean Peacock, Kristin Haglund

College of Nursing Faculty Research and Publications

Sickle cell disease (SCD) is associated with medical and psychosocial challenges. SCD in adulthood is characterized by increased morbidity and mortality, vulnerability, inadequate self-management preparation, and limited social support. This study evaluated the implementation of a virtual support group during the COVID-19 pandemic using an intervention parameters framework evaluation. Participants included six adults living with SCD. Content analysis was conducted for each of the six intervention parameters, and satisfaction scores were summarized. Participants positively evaluated all intervention parameters of the virtual support group. Findings confirmed that a community-engaged intervention supports self-management and establishes key social networks for adult participants.

A Swimming Program For Children With Autism Spectrum Disorders: Assessing Critical Parameters From Caregivers’ Perspectives, Abir K. Bekhet, Norah L. Johnson, Tana Karenke, Amy V. Van Hecke

College of Nursing Faculty Research and Publications

One in 54 individuals is diagnosed with autism spectrum disorders (ASD). Swimming can impact the physical and mental well-being of persons with ASD, which in turn may improve their caregivers’ well-being. The purpose of this qualitative evaluation study is to assess the necessity, acceptability, feasibility, fidelity, and beginning effectiveness of a 12-session swimming training program (STP) at an indoor university pool designed to teach persons with ASD how to swim, and to teach caregivers precautions for safety around water. Participants included 7 boys, and 3 girls with ASD [mean age 7.05 years (SD 1.17)] and their caregivers. Results indicated that …

Physical Activity, Inflammation, And Physical Function In Older Adults: Results From The Health & Retirement Study, Jeanne L. Hlebichuk, Randall J. Gretebeck, Mauricio Garnier-Villarreal, Linda B. Piacentine, Maharaj Singh, Kimberlee A. Gretebeck

College of Nursing Faculty Research and Publications

Physical function declines with aging due to physical and biological changes. The biological process of aging has been associated with increases in systemic inflammation and a greater risk for chronic conditions. In older adults, physical activity aids in maintenance of function. However, the influence of inflammatory biomarkers and adiposity on physical activity and physical function needs to be further explored. Methods: A cross-sectional secondary data analysis from Wave 13 of the Health & Retirement Study (HRS) core biennial data and Venous Blood Study (VBS) was conducted. Structural equation modeling was used to establish the model and test the relationships. Results: …

Exploring The Feasibility Of Intrapartum Gbs Collection To Identify Residual Gbs In A Pilot Study Of An Antenatal Probiotic Intervention, Emily Malloy, Lisa Hanson, Leona Vandevusse, Karen Robinson, Maharaj Singh, Marie Forgie

College of Nursing Faculty Research and Publications

Background: We aimed to explore the feasibility of collecting intrapartum maternal Group B Streptococcus (GBS) colonization and immediate post-birth neonatal GBS colonization cultures for use in a larger trial and to identify cases of residual GBS, which were hypothesized to be less common in the probiotics group.

Methods: This sub-study added additional outcome measures to the parent study to identify intrapartum and neonatal colonization and compare between probiotic and placebo groups and to identify cases of residual GBS. Intrapartum maternal vaginal and rectal GBS cultures were collected at the time of admission to a hospital for labor and to give …

University Of Central Florida Libraries, Annual Report 2021-2022, University Libraries

Libraries' Documents

No abstract provided.

Sik3 And Wnk Converge On Fray To Regulate Glial K+ Buffering And Seizure Susceptibility, Lorenzo Lones, Aaron Diantonio

2020-Current year OA Pubs

Glial cells play a critical role in maintaining homeostatic ion concentration gradients. Salt-inducible kinase 3 (SIK3) regulates a gene expression program that controls K+ buffering in glia, and upregulation of this pathway suppresses seizure behavior in the eag, Shaker hyperexcitability mutant. Here we show that boosting the glial SIK3 K+ buffering pathway suppresses seizures in three additional molecularly diverse hyperexcitable mutants, highlighting the therapeutic potential of upregulating glial K+ buffering. We then explore additional mechanisms regulating glial K+ buffering. Fray, a transcriptional target of the SIK3 K+ buffering program, is a kinase that promotes K+ uptake by activating the Na+/K+/Cl- …

Multi-Omic Association Study Identifies Dna Methylation-Mediated Genotype And Smoking Exposure Effects On Lung Function In Children Living In Urban Settings, Matthew Dapas, Michelle A. Gill, Et Al

2020-Current year OA Pubs

Impaired lung function in early life is associated with the subsequent development of chronic respiratory disease. Most genetic associations with lung function have been identified in adults of European descent and therefore may not represent those most relevant to pediatric populations and populations of different ancestries. In this study, we performed genome-wide association analyses of lung function in a multiethnic cohort of children (n = 1,035) living in low-income urban neighborhoods. We identified one novel locus at the TDRD9 gene in chromosome 14q32.33 associated with percent predicted forced expiratory volume in one second (FEV1) (p = 2.4x10-9; βz = -0.31, …

A Novel Biosignature Identifies Patients With Dcis With High Risk Of Local Recurrence After Breast Conserving Surgery And Radiation Therapy, Frank A Vicini, G Bruce Mann, Chirag Shah, Sheila Weinmann, Michael C Leo, Pat Whitworth, Rachel Rabinovitch, Mylin A Torres, Julie A Margenthaler, David Dabbs, Jess Savala, Steven C Shivers, Karuna Mittal, Fredrik Wärnberg, Troy Bremer

2020-Current year OA Pubs

PURPOSE: There is an unmet need to identify women diagnosed with ductal carcinoma in situ (DCIS) with a low risk of in-breast recurrence (IBR) after breast conserving surgery (BCS), which could omit radiation therapy (RT), and also to identify those with elevated IBR risk remaining after BCS plus RT. We evaluated a novel biosignature for a residual risk subtype (RRt) to help identify patients with elevated IBR risk after BCS plus RT.

METHODS AND MATERIALS: Women with DCIS treated with BCS with or without RT at centers in the US, Australia, and Sweden (n = 926) were evaluated. Patients were …

The Mutational Landscape In Chronic Myelomonocytic Leukemia And Its Impact On Allogeneic Hematopoietic Cell Transplantation Outcomes: A Center For Blood And Marrow Transplantation Research (Cibmtr) Analysis, Matthew Mei, Ravi Vij, Et Al.

2020-Current year OA Pubs

Somatic mutations are recognized as an important prognostic factor in chronic myelomonocytic leukemia (CMML). However, limited data are available regarding their impact on outcomes after allogeneic hematopoietic cell transplantation (HCT). In this registry analysis conducted in collaboration with the Center for International Blood and Marrow Transplantation Registry database/sample repository, we identified 313 adult patients with CMML (median age: 64 years, range, 28- 77) who underwent allogeneic HCT during 2001-2017 and had an available biospecimen in the form of a peripheral blood sample obtained prior to the start of conditioning. In multivariate analysis, a CMML-specific prognostic scoring system (CPSS) score of …

Design Of Effisayil™ 2: A Randomized, Double-Blind, Placebo-Controlled Study Of Spesolimab In Preventing Flares In Patients With Generalized Pustular Psoriasis, Akimichi Morita, Siew Eng Choon, Hervé Bachelez, Milan J Anadkat, Slaheddine Marrakchi, Min Zheng, Tsen-Fang Tsai, Hamida Turki, Harry Hua, Sushmita Rajeswari, Christian Thoma, A David Burden

2020-Current year OA Pubs

INTRODUCTION: Generalized pustular psoriasis (GPP) is a rare autoinflammatory skin disease characterized by flares of widespread erythema with sterile pustules, and can be relapsing with recurrent flares, or persistent with intermittent flares. Spesolimab, a humanized anti-interleukin-36 (IL-36) receptor monoclonal antibody, targets the key IL-36 pathogenetic pathway in GPP. A previous study showed that spesolimab treatment led to rapid pustular and skin clearance in patients with GPP flares, which was sustained for up to 12 weeks. This study investigates the long-term effects of spesolimab on GPP flares, for which no specific treatments are currently available. The Effisayil™ 2 study will assess …