Medicine and Health Sciences Commons^™

An Activity Program For Individuals With Intellectual And Developmental Disabilities, Rebecca Wright, Keisa Boykin

Fall 2023 Virtual OTD Capstone Symposium

According to the Diagnostic and Statistical Manual of Mental Disorders intellectual and developmental disabilities (IDD) are characterized by onset in the developmental period and include both intellectual and adaptive functioning deficits in conceptual, social, or practical domains that limit functioning in one or more areas of daily life (American Psychiatric Association [APA], 2013). Occupational therapists provide interventions for individuals with IDD however there are gaps surrounding occupation-based leisure and recreation activities (Kalijaca, et al 2019). Purpose: The purpose of this project is to create an activity program for individuals with IDD to increase participation and performance in leisure and recreation …

A Occupational Therapy Guidebook For Teachers On Sensory Processing And Sensory Integration, Kaitlyn Stanley, Keisa Boykin, Heidi Woolley

Fall 2023 Virtual OTD Capstone Symposium

Children with sensory processing (SP) challenges find simple classroom tasks overwhelming and can present with deficits in behavior management, emotional regulation, and occupations due to the inability to self-regulate the sensory input from their environment. In addition, deficits in SP may impact a child's ability to maintain engagement and participation in the learning process. If these SP needs are not met through the school, the child lacks the adequate tools to be successful in learning, which is considered occupational injustice (AOTA, 2020). By providing teachers with the adequate educational tools to understand sensory processing and sensory integration (SP-SI), identify and …

Prevalence Of Age-Related Macular Degeneration And Associated Factors In Indian Cohort In A Tertiary Care Setting, Jacqueline Hamati, Sai Prashanthi, Raja Narayanan, Niroj Sahoo, Anthony Vipin Das, Padmaja Kumari Rani, Umesh Chandra Behera, Rohit Khanna, Gudlavalleti V.S. Murthy

Student Papers, Posters & Projects

PURPOSE: To report a big data analysis of risk and protective factors in patients with AMD, as well as report on the age-adjusted prevalence in a geriatric Indian cohort in a hospital setting.

METHODS: This retrospective, observational study of all patients older than 60 years of age. Multiple logistic regression was performed for the binary outcome and the presence of AMD. Variables analyzed include age, gender, socioeconomic status, occupation, urban-rural-metropolitan distribution, self-reported history of diabetes mellitus (DM), hypertension (HTN), or coronary artery disease (CAD), ocular comorbidities, history of cataract surgery, and presenting VA. Odds ratios (OR) and 99% confidence intervals …

Pelaksanaan Tanggung Jawab Sosial Perusahaan Melalui Program Pemulihan Mata Pencaharian Oleh Lembaga X, Muhammad Hanif Ridho, Mochammad Rizqy Maulana, Muhamad Azami Nasri

Jurnal Ilmu Kesejahteraan Sosial

This study examines the implementation of corporate social responsibility through the Livelihood Restoration Program (LRP) by Institution X. The study employs a qualitative approach with descriptive research methodology. Data collection techniques include in-depth interviews and literature review. The study is motivated by land acquisition conducted by PT Y, which affected the livelihoods of the local community. Consequently, PT Y initiated livelihood restoration for the affected community through the LRP managed by Institution X. The findings indicate that Institution X has two primary roles: providing consultancy services and facilitating the implementation of the LRP. First, as a consultancy agency, Institution X …

Program Penanganan Fakir Miskin Di Desa Mulyasari Kota Banjar Provinsi Jawa Barat, Aep Rusmana, Eri Susanto, Muhammad Rai Jemadila, Nabila Aulia Putri

Jurnal Ilmu Kesejahteraan Sosial

Poverty is a condition where a person cannot fulfill their basic needs to achieve a more dignified life. This research aims to explain the program for handling the poor in Mulyasari Village which is directed at three things, namely: 1) managing social problems, 2) mechanisms for meeting needs, and 3) providing opportunities for advancement for the poor. The method used in this research is a descriptive qualitative method with a phenomenological tradition. Determining the research sample used purposive sampling technique. There were 6 research informants consisting of Mulyasari Village officials, poor families who received social welfare services, and Community Social …

Keterlibatan Serikat Pekerja Dalam Perubahan Kebijakan Pengupahan Melalui Advokasii Sosial, Zeni Tri Lestari, Arif Wibowo

Jurnal Ilmu Kesejahteraan Sosial

This research is about the involvement of trade unions in changing wage policies through social advocacy from the social welfare perspective. The urgency of conducting this research is to reveal how KASBI's involvement as a worker's union encourages changes in wage policies to ensure better labor welfare through advocacy against the Job Creation Law and advocacy against Minister of Manpower Regulation Number 2 of 2022. This research uses a descriptive qualitative method. Data were collected from February to June 2023 through online and offline interviews involving three informants selected by the purposive sampling technique. The research results reveal that KASBI …

Pengembangan Sistem Informasi Desa Dalam Upaya Penanggulangan Kemiskinan Di Desa Rarang Selatan, Meita Annisa, Bambang Shergi Laksmono

Jurnal Ilmu Kesejahteraan Sosial

Indonesia continues to face challenges in poverty alleviation, with extreme poverty still prevalent in the country. Despite having a social protection system aimed at reducing poverty and cushioning the poor and vulnerable from crises, its implementation often encounters difficulties, particularly in obtaining valid and verified data sources. This study explores how the development of the Village Information System (SID) can contribute to poverty alleviation in Rarang Selatan Village, West Nusa Tenggara Province (NTB). When SID was introduced in 2016, Rarang Selatan was a newly established village classified as underdeveloped. By 2022, the village had progressed to the category of an …

Dean's Report- Fall 2023, Marina K. Holz

GSBMS Dean’s Report

No abstract provided.

A Word From The Writing Team (October 2023), Pam Walter, Mfa, Liz Declan, Ma, Mfa

A Word From the Writing Team (Newsletter)

This issue includes:

• Join Us for First Friday Writing Retreats Starting October 6^th
• Creative Writing Publication Opportunity: LGBTQ+ Theme
• Publication Spotlight
• Reminders
• Consultations Available
• Scott Memorial Library Renovations
• Wiley Open Access Fees Waived for Jefferson Authors

Increasing Vaccination Rates Of 23-Valent Pneumococcal Polysaccharide Vaccine Among Patients At High-Risk For Invasive Pneumococcal Disease., Edward Lyon, Tracey Wetzel, Ann L. Wirtz, Douglas Swanson, Rachel Moran, Jessica Peters, Christine Symes, Liset Olarte, Rana El Feghaly

Posters

Introduction: Pneumococcal disease causes significant morbidity and mortality in children. Routine childhood immunizations protect for 13 or 15 pneumococcal serotypes via two pneumococcal conjugated vaccines. Patients with immunocompromising and chronic medical conditions are at high risk of invasive pneumococcal disease. A 23-valent pneumococcal polysaccharide vaccine (PPSV23) is recommended in these patients to protect against more pneumococcal serotypes, but many patients have not received it. Our AIM is to increase PPSV23 vaccination rates among eligible patients in both the inpatient and outpatient infectious diseases (ID) settings from a baseline of 44% to 55% by October 2024. Methods: In collaboration with Children’s …

Une Com Research And Scholarship Fall Forum October 2023, Une Com Research & Scholarship

Osteopathic Student Research Symposia

Program including abstracts from the University of New England College of Osteopathic Medicine’s 2023 Research and Scholarship Fall Forum.

Supplemental content: List of award winners.

Effects Of Thermocycling And Hydrolytic Aging On The Flexural Strength Of Additively Manufactured Restorative Materials, Steven Mustafa Abu Al Tamn

Master's Theses (2009 -)

Introduction: Additive manufacturing is being integrated into various aspects of clinical prosthodontics due to its potential for use in various indications and the promise of improved clinical efficiency and properties.Objective: The purpose of this in-vitro study was to evaluate the flexural strength of two 3D-printed resin composites in relation to a conventionally fabricated resin composite after being subjected to two different artificial aging methods: thermocycling and distilled water storage. Material and methods: A total of 220 samples were fabricated (N=220); 72 samples were fabricated from Integrity composite (Dentsply Sirona) and 148 samples were fabricated from two 3D-printed composite materials MFH …

Long-Term Assessment Of Skeletal And Dental Asymmetry After Conventional And Mini-Screw Assisted Rapid Palatal Expansion, Brett Barton

Master's Theses (2009 -)

Objective: To evaluate the long-term skeletal and dental asymmetry following Mini-screw Assisted Rapid Palatal Expansion (MARPE), Rapid Palatal Expansion (RPE) and controls with 3D cone beam computed tomography (CBCT) analysis. Methods: 180 CBCT scans were evaluated retrospectively for 60 patients at pre-treatment (T1), post-expansion (T2), and post-treatment (T3). The patients were randomly assigned into 3 groups – MARPE, RPE, and controls. Anterior Nasal Spine (ANS), Posterior Nasal Spine (PNS), Zygomaticomaxillary Point (ZMA), Anterior Nasal Cavity Width (ANCW), Posterior Nasal Cavity Width (PNCW), External Maxillary Width (EMW), Palatal Width (PW), Maxillary Width (MMW), Maxillary Canine Width (MCW) were measured on the …

A Single Center Analysis Of Patients Undergoing Liver Resection For Intrahepatic Cholangiocarcinoma, Nazli Begum Ozturk, Laith H. Jamil

Conference Presentation Abstracts

Background: Intrahepatic cholangiocarcinoma (iCCA) is the second most common primary liver cancer with increasing rates of incidence and mortality. Surgical resection with negative margins and regional lymphadenectomy is the mainstay of the treatment for patients with resectable-iCCA. Because of its rarity and low resectability rate of iCCA, prognostic markers and outcomes of patients who undergo liver resection are not well defined. We aimed to evaluate the characteristics of patients undergoing liver resection for iCCA at our institution over ten years. Methods: All patients with iCCA who had liver resection between 3/2013-3/2023 at our institution were included in the study. An …

Cheiloscopy Examination And Classification Of Lip Prints With And Without Parafunctional Oral Habits: A Cross-Sectional Observation Study, Emily Smith Regan

Dental Hygiene Theses & Dissertations

Problem: Lip prints are unique and have potential for use as a human identifier. The purpose of this study was to observe possible cheiloscopy differences of individuals with and without parafunctional oral habits. Additionally, inter-rater reliability (IRR) of lip print examiners was observed. Methods: This IRB approved blinded cross-sectional observational study collected lip prints from sixty-six individuals using lipstick and adhesive tape to transfer lip prints to white bond paper for viewing purposes. Each set of included lip prints was divided into quadrants and dichotomized as those with or without an oral parafunctional habit. Each quadrant sample was manually analyzed …

Nightmares And Self-Injury Among High-Risk Adolescents: Examining The Role Of Emotion Regulation, Kinjal K. Patel

Psychology Theses & Dissertations

Self-injurious thoughts and behaviors (SITBs) are a public health concern among youth. SITB rates rise drastically during adolescence -- a critical and sensitive developmental period characterized by dramatic changes in biological and social systems. Prior research has identified distal risk factors which tell us who is at risk for SITBs. Emerging research suggests proximal risk factors may be useful to identify when an individual may be at risk for SITBs. One promising proximal risk factor for SITBs is sleep problems. Although the relation between sleep problems and SITBs has been widely documented, little is known about the short-term, proximal links …

Specialized Astrocytes Mediate Glutamatergic Gliotransmission In The Cns, Roberta De Ceglia, Ada Ledonne, David Gregory Litvin, Barbara Lykke Lind, Giovanni Carriero, Emanuele Claudio Latagliata, Erika Bindocci, Maria Amalia Di Castro, Iaroslav Savtchouk, Ilaria Vitali, Anurag Ranjak, Mauro Congiu, Tara Canonica, William Wisden, Kenneth Harris, Manuel Mameli, Nicola Mercuri, Ludovic Telley, Andrea Volterra

Biomedical Sciences Faculty Research and Publications

Multimodal astrocyte–neuron communications govern brain circuitry assembly and function. For example, through rapid glutamate release, astrocytes can control excitability, plasticity and synchronous activity of synaptic networks, while also contributing to their dysregulation in neuropsychiatric conditions. For astrocytes to communicate through fast focal glutamate release, they should possess an apparatus for Ca2+-dependent exocytosis similar to neurons. However, the existence of this mechanism has been questioned owing to inconsistent data and a lack of direct supporting evidence. Here we revisited the astrocyte glutamate exocytosis hypothesis by considering the emerging molecular heterogeneity of astrocytes and using molecular, bioinformatic and imaging approaches, together with …

Web-Grading-A Tool To Test Personal Grading Of Renal And Prostate Cancer, Glen Kristiansen, Matthias Schmid, Lars Egevad, Hemamali Samaratunga, Murali Varma, Kaan Inam, Hans-Jürgen Thiesen, Brett Delahunt, Yulin Dai

Faculty, Staff and Student Publications

Only a few pathologists have the opportunity to verify their personal grading through objective assessment. This study introduces a web-based grading platform to facilitate and validate the grading of renal cell carcinoma and prostate cancer. Two representative images of two clinically annotated cohorts of 100 cases each of prostate and renal cell carcinoma were used. Each participant was asked to grade a tumor series utilizing a three tiered grading system. Finally, a Kaplan-Meier curve was drawn, and the log-rank test was used for statistical testing of the p-value. The grading of 22 participants (68%) achieved prognostic significance. Further analysis highlighted …

Recent Insights Into The Pathogenesis And Therapeutic Targets Of Chronic Liver Diseases, Yankai Wen, Lichun Ma, Cynthia Ju

Faculty, Staff and Student Publications

Viral hepatitis, alcohol-associated liver disease (ALD) and non-alcoholic fatty liver disease (NAFLD) are the three major causes of chronic liver diseases, which account for approximately 2 million deaths per year worldwide. The current direct-acting antiviral drugs and vaccinations have effectively reduced and ameliorated viral hepatitis infection, but there are still no effective drug treatments for ALD, NAFLD and liver cancer due to the poor understanding of their pathogenesis. To better understand the pathogenesis, the fifth Chinese American Liver Society/Society of Chinese Bioscientists in America Hepatology Division Annual Symposium, which was held virtually on 21-22 October 2022, focused on the topics …

Discoverpath: A Knowledge Refinement And Retrieval System For Interdisciplinarity On Biomedical Research, Yu-Neng Chuang, Guanchu Wang, Chia-Yuan Chang, Kwei-Herng Lai, Daochen Zha, Ruixiang Tang, Fan Yang, Alfredo Costilla Reyes, Kaixiong Zhou, Xiaoqian Jiang, Xia Hu

Faculty, Staff and Student Publications

The exponential growth in scholarly publications necessitates advanced tools for efficient article retrieval, especially in interdisciplinary fields where diverse terminologies are used to describe similar research. Traditional keyword-based search engines often fall short in assisting users who may not be familiar with specific terminologies. To address this, we present a knowledge graph based paper search engine for biomedical research to enhance the user experience in discovering relevant queries and articles. The system, dubbed DiscoverPath, employs Named Entity Recognition (NER) and part-of-speech (POS) tagging to extract terminologies and relationships from article abstracts to create a KG. To reduce information overload, DiscoverPath …

Visualization Of Patient-Generated Health Data: A Scoping Review Of Dashboard Designs, Edna Shenvi, Aziz Boxwala, Dean Sittig, Courtney Zott, Edwin Lomotan, James Swiger, Prashila Dullabh

Faculty, Staff and Student Publications

BACKGROUND: Patient-centered clinical decision support (PC CDS) aims to assist with tailoring decisions to an individual patient's needs. Patient-generated health data (PGHD), including physiologic measurements captured frequently by automated devices, provide important information for PC CDS. The volume and availability of such PGHD is increasing, but how PGHD should be presented to clinicians to best aid decision-making is unclear.

OBJECTIVES: Identify best practices in visualizations of physiologic PGHD, for designing a software application as a PC CDS tool.

METHODS: We performed a scoping review of studies of PGHD dashboards that involved clinician users in design or evaluations. We included only …

Co-Targeting Bcl-Xl And Bcl-2 By Protac 753b Eliminates Leukemia Cells And Enhances Efficacy Of Chemotherapy By Targeting Senescent Cells, Yannan Jia, Lina Han, Cassandra L Ramage, Zhe Wang, Connie C Weng, Lei Yang, Simona Colla, Helen Ma, Weiguo Zhang, Michael Andreeff, Naval Daver, Nitin Jain, Naveen Pemmaraju, Kapil Bhalla, Satu Mustjoki, Peiyi Zhang, Guangrong Zheng, Daohong Zhou, Qi Zhang, Marina Konopleva

Faculty, Staff and Student Publications

BCL-XL and BCL-2 are key anti-apoptotic proteins and validated cancer targets. 753B is a novel BCL-XL/BCL-2 proteolysis targeting chimera (PROTAC) that targets both BCL-XL and BCL-2 to the von Hippel-Lindau (VHL) E3 ligase, leading to BCLX L/BCL-2 ubiquitination and degradation selectively in cells expressing VHL. Because platelets lack VHL expression, 753B spares on-target platelet toxicity caused by the first-generation dual BCL-XL/BCL-2 inhibitor navitoclax (ABT-263). Here, we report pre-clinical single-agent activity of 753B against different leukemia subsets. 753B effectively reduced cell viability and induced dose-dependent degradation of BCL-XL and BCL-2 in a subset of hematopoietic cell lines, acute myeloid leukemia (AML) …

Combining Clinical And Biological Data To Predict Progressive Pulmonary Fibrosis In Patients With Systemic Sclerosis Despite Immunomodulatory Therapy, Elizabeth R Volkmann, Holly Wilhalme, Shervin Assassi, Grace Hyun J Kim, Jonathan Goldin, Masataka Kuwana, Donald P Tashkin, Michael D Roth

Faculty, Staff and Student Publications

OBJECTIVE: Progressive pulmonary fibrosis (PPF) is the leading cause of death in systemic sclerosis (SSc). This study aimed to develop a clinical prediction nomogram using clinical and biological data to assess risk of PPF among patients receiving treatment of SSc-related interstitial lung disease (SSc-ILD).

METHODS: Patients with SSc-ILD who participated in the Scleroderma Lung Study II (SLS II) were randomized to treatment with either mycophenolate mofetil (MMF) or cyclophosphamide (CYC). Clinical and biological parameters were analyzed using univariable and multivariable logistic regression, and a nomogram was created to assess the risk of PPF and validated by bootstrap resampling.

RESULTS: Among …

Evolving Availability And Standardization Of Patient Attributes For Matching, Yu Deng, Lacey P Gleason, Adam Culbertson, Xiaotian Chen, Elmer V Bernstam, Theresa Cullen, Ramkiran Gouripeddi, Christopher Harle, David F Hesse, Jacob Kean, John Lee, Tanja Magoc, Daniella Meeker, Toan Ong, Jyotishman Pathak, Marc Rosenman, Laura K Rusie, Akash J Shah, Lizheng Shi, Aaron Thomas, William E Trick, Shaun Grannis, Abel Kho

Faculty, Staff and Student Publications

Variation in availability, format, and standardization of patient attributes across health care organizations impacts patient-matching performance. We report on the changing nature of patient-matching features available from 2010-2020 across diverse care settings. We asked 38 health care provider organizations about their current patient attribute data-collection practices. All sites collected name, date of birth (DOB), address, and phone number. Name, DOB, current address, social security number (SSN), sex, and phone number were most commonly used for cross-provider patient matching. Electronic health record queries for a subset of 20 participating sites revealed that DOB, first name, last name, city, and postal codes …

An Efficient Strategy For Evaluating New Non-Invasive Screening Tests For Colorectal Cancer: The Guiding Principles, Robert S Bresalier, Carlo Senore, Graeme P Young, James Allison, Robert Benamouzig, Sally Benton, Patrick M M Bossuyt, Luis Caro, Beatriz Carvalho, Han-Mo Chiu, Veerle M H Coupé, Willemijn De Klaver, Clasine Maria De Klerk, Evelien Dekker, Sunil Dolwani, Callum G Fraser, William Grady, Lydia Guittet, Samir Gupta, Stephen P Halloran, Ulrike Haug, Geir Hoff, Steven Itzkowitz, Tim Kortlever, Anastasios Koulaouzidis, Uri Ladabaum, Beatrice Lauby-Secretan, Mārcis Leja, Bernard Levin, Theodore Robert Levin, Finlay Macrae, Gerrit A Meijer, Joshua Melson, Colm O'Morain, Susan Parry, Linda Rabeneck, David F Ransohoff, Roque Sáenz, Hiroshi Saito, Silvia Sanduleanu-Dascalescu, Robert E Schoen, Kevin Selby, Harminder Singh, Robert J C Steele, Joseph J Y Sung, Erin Leigh Symonds, Sidney J Winawer, Members Of The World Endoscopy Colorectal Cancer Screening New Test Evaluation Expert Working Group

Faculty, Staff and Student Publications

OBJECTIVE: New screening tests for colorectal cancer (CRC) are rapidly emerging. Conducting trials with mortality reduction as the end point supporting their adoption is challenging. We re-examined the principles underlying evaluation of new non-invasive tests in view of technological developments and identification of new biomarkers.

DESIGN: A formal consensus approach involving a multidisciplinary expert panel revised eight previously established principles.

RESULTS: Twelve newly stated principles emerged. Effectiveness of a new test can be evaluated by comparison with a proven comparator non-invasive test. The faecal immunochemical test is now considered the appropriate comparator, while colonoscopy remains the diagnostic standard. For a …

Zyxin Inhibits The Epithelial-Mesenchymal Transition Process In Gastric Cancer By Upregulating Sirt1, Jing Lou, Sha Geng, Wei He, Song-Bai Liu, Xinghong Shi, Ying Chang, Shiyuan Han, Panting Qian, Hesham M Amin, Yao-Hua Song, Yangxin Li, Jin Zhou

Faculty, Staff and Student Publications

Tumor development relies on the stemness of cancer stem cells, which is regulated by environmental cues. Previous studies have shown that zyxin can inhibit the expression of genes for embryonic stem cell status. In the present study, the expression levels of zyxin protein in cancer tissues and adjacent noncancerous tissues from 73 gastric cancer patients with different clinical stages were analyzed by Western blot. We showed that the relative expression levels of zyxin in gastric cancer tissues (cancer tissues/adjacent tissues) were significantly downregulated in advanced clinical stages. Overexpression of zyxin inhibited the stemness and epithelial-mesenchymal transition (EMT) processes in gastric …

Therapeutic Effects Of Combining Curcumin And Swimming In Osteoarthritis Using A Rat Model, Mona M Saber, Manal Moustafa Mahmoud, Hesham M Amin, Reham M Essam

Faculty, Staff and Student Publications

Osteoarthritis (OA) is a common debilitating degenerative disease of the elderly. We aimed to study the therapeutic effects of combining curcumin and swimming in monosodium iodoacetate (MIA)-induced OA in a rat model. The rats were divided into 5 groups (n = 9). Group 1 received saline and served as a control group. Groups 2-5 were injected intra-articularly in the right knee with 100 μL MIA. One week later, groups 3 and 5 were started on daily swimming sessions that gradually increased to 20-mins per session, and for groups 4 and 5, oral curcumin was administered at a dose of 200 …

Evaluation Of The Natural History Of Disease By Combining Incident And Prevalent Cohorts: Application To The Nun Study, Daewoo Pak, Jing Ning, Richard J Kryscio, Yu Shen

Faculty, Staff and Student Publications

The Nun study is a well-known longitudinal epidemiology study of aging and dementia that recruited elderly nuns who were not yet diagnosed with dementia (i.e., incident cohort) and who had dementia prior to entry (i.e., prevalent cohort). In such a natural history of disease study, multistate modeling of the combined data from both incident and prevalent cohorts is desirable to improve the efficiency of inference. While important, the multistate modeling approaches for the combined data have been scarcely used in practice because prevalent samples do not provide the exact date of disease onset and do not represent the target population …

A Self-Configuring Deep Learning Network For Segmentation Of Temporal Bone Anatomy In Cone-Beam Ct Imaging, Andy S Ding, Alexander Lu, Zhaoshuo Li, Manish Sahu, Deepa Galaiya, Jeffrey H Siewerdsen, Mathias Unberath, Russell H Taylor, Francis X Creighton

Faculty, Staff and Student Publications

OBJECTIVE: Preoperative planning for otologic or neurotologic procedures often requires manual segmentation of relevant structures, which can be tedious and time-consuming. Automated methods for segmenting multiple geometrically complex structures can not only streamline preoperative planning but also augment minimally invasive and/or robot-assisted procedures in this space. This study evaluates a state-of-the-art deep learning pipeline for semantic segmentation of temporal bone anatomy.

STUDY DESIGN: A descriptive study of a segmentation network.

SETTING: Academic institution.

METHODS: A total of 15 high-resolution cone-beam temporal bone computed tomography (CT) data sets were included in this study. All images were co-registered, with relevant anatomical structures …

Garetosmab In Fibrodysplasia Ossificans Progressiva: A Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial, Maja Di Rocco, Eduardo Forleo-Neto, Robert J Pignolo, Richard Keen, Philippe Orcel, Thomas Funck-Brentano, Christian Roux, Sami Kolta, Annalisa Madeo, Judith S Bubbear, Jacek Tabarkiewicz, Małgorzata Szczepanek, Javier Bachiller-Corral, Angela M Cheung, Kathryn M Dahir, Esmée Botman, Pieter G Raijmakers, Mona Al Mukaddam, Lianne Tile, Cynthia Portal-Celhay, Neena Sarkar, Peijie Hou, Bret J Musser, Anita Boyapati, Kusha Mohammadi, Scott J Mellis, Andrew J Rankin, Aris N Economides, Dinko Gonzalez Trotter, Gary A Herman, Sarah J O'Meara, Richard Delgizzi, David M Weinreich, George D Yancopoulos, E Marelise W Eekhoff, Frederick S Kaplan

Faculty, Staff and Student Publications

Fibrodysplasia ossificans progressiva (FOP) is a rare disease characterized by heterotopic ossification (HO) in connective tissues and painful flare-ups. In the phase 2 LUMINA-1 trial, adult patients with FOP were randomized to garetosmab, an activin A-blocking antibody (n = 20) or placebo (n = 24) in period 1 (28 weeks), followed by an open-label period 2 (28 weeks; n = 43). The primary end points were safety and for period 1, the activity and size of HO lesions. All patients experienced at least one treatment-emergent adverse event during period 1, notably epistaxis, madarosis and skin abscesses. Five deaths …