Pediatrics Commons^™

Congenital Abnormalities Of The Lower Airways And Lungs, Christopher M. Oermann

Presentations

Goals and Objectives. Participants will be able to: Describe the 5 stages of fetal lung development. Summarize the prenatal identification and management of congenital abnormalities of the lower airways and lung (CALAL). Explain the classification of CALAL. Compare the clinical characteristics, pathology, and management of common CALAL. Discuss the long term prognosis of CALAL

New Mexico Children's Alliance Children's Advocacy Centers Needs Assessment, Theresa H. Cruz, Leona Woelk, Ivy Vitanzos Cervantes, Emily Guerra

Reports & Documents

No abstract provided.

Risk Factors For Nonaccidental Burns In Children, Dalya M Ferguson, Tayler D Parker, Vanessa E Marino, Elisa I Garcia, Seyed A Arshad, Pranali S Kamat, Caroline M Anding, Kuojen Tsao, Rebecca G Girardet, Mary T Austin

Faculty, Staff and Student Publications

BACKGROUND: The relative influences of baseline risk factors for pediatric nonaccidental burns have not been well described. We evaluated baseline characteristics of pediatric nonaccidental burn patients and their primary caretakers.

METHODS: A single-center retrospective cohort study was conducted of pediatric (age < 17) burn patients from July 1, 2013, to June 30, 2018. The primary outcome was nonaccidental burn, defined as burn secondary to abuse or neglect as determined by the inpatient child protection team or Child Protective Services. Univariate and multivariate analyses were performed.

RESULTS: Of 489 burn patients, 47 (9.6%) suffered nonaccidental burns. Nonaccidental burn patients more frequently had a history of Child Protective Services involvement (48.9% vs 9.7%, P < .001), as did their primary caretakers (59.6% vs 10.9%, P < .001). Non-Hispanic black children had higher rates of Child Protective Services referral (50.7% vs 26.7%, P < .001) and nonaccidental burn diagnosis (18.9% vs 5.6%, P < .001) than children of other races/ethnicities. On multivariate analysis, caretaker involvement with CPS (odds ratio 7.53, 95% confidence interval 3.38-16.77) and non-Hispanic black race/ethnicity (odds ratio 3.28, 95% confidence interval 1.29-8.36) were associated with nonaccidental burn.

CONCLUSION: Caretaker history of Child Protective Services involvement and non-Hispanic black race/ethnicity were associated with increased odds of pediatric nonaccidental burn. Prospective research is necessary to determine whether these represent true risk …

Atrx Deletion In Neurons Leads To Sexually Dimorphic Dysregulation Of Mir-137 And Spatial Learning And Memory Deficits., Renee J. Tamming, Vanessa Dumeaux, Yan Jiang, Sarfraz Shafiq, Luana Langlois, Jacob Ellegood, Lily R. Qiu, Jason P. Lerch, Nathalie G. Bérubé

Paediatrics Publications

ATRX gene mutations have been identified in syndromic and non-syndromic intellectual disabilities in humans. ATRX is known to maintain genomic stability in neuroprogenitor cells, but its function in differentiated neurons and memory processes remains largely unresolved. Here, we show that the deletion of neuronal Atrx in mice leads to distinct hippocampal structural defects, fewer presynaptic vesicles, and an enlarged postsynaptic area at CA1 apical dendrite-axon junctions. We identify male-specific impairments in long-term contextual memory and in synaptic gene expression, linked to altered miR-137 levels. We show that ATRX directly binds to the miR-137 locus and that the enrichment of the …

Host Immune Response To Enterovirus And Parechovirus Systemic Infections In Children., Anjana Sasidharan, Wail M. Hassan, Christopher J. Harrison, Ferdaus Hassan, Rangaraj Selvarangan

Manuscripts, Articles, Book Chapters and Other Papers

Background: Enterovirus (EV) and parechovirus type A3 (PeV-A3) cause infections ranging from asymptomatic to life-threatening. Host immune responses in children, particularly innate responses to PeV-A3, remain largely unknown. The aim of this study was to determine aspects of the cytokine/chemokine responses to EV and PeV-A3 in cerebrospinal fluid (CSF) and plasma obtained from children with systemic/central nervous system infection.

Methods: A total of 74 salvaged CSF samples (27 with EV, 23 with PeV-A3, and 24 with neither EV nor PeV-A3) and 35 paired blood samples (10 with EV, 14 with PeV-A3, and 11 with neither) were studied. Concentrations of cytokines …

Transient Hyponatremia Of Prematurity Caused By Mild Bartter Syndrome Type Ii: A Case Report., Subhrata Verma, Rahul Chanchlani, Victoria Mok Siu, Guido Filler

Paediatrics Publications

BACKGROUND: Bartter syndrome subtypes are a group of rare renal tubular diseases characterized by impaired salt reabsorption in the tubule, specifically the thick ascending limb of Henle's loop. Clinically, they are characterized by the association of hypokalemic metabolic alkalosis, hypercalciuria, nephrocalcinosis, increased levels of plasma renin and aldosterone, low blood pressure and vascular resistance to angiotensin II. Bartter syndrome type II is caused by mutations in the renal outer medullary potassium channel (ROMK) gene (KCNJ1), can present in the newborn period and typically requires lifelong therapy.

CASE PRESENTATION: We describe a case of a prematurely born female infant presenting with …

Evolving Role For Pharmacotherapy In Nafld/Nash, Suzanna L. Attia, Samir Softic, Marialena Mouzaki

Pediatrics Faculty Publications

Nonalcoholic fatty liver disease (NAFLD) is a highly prevalent, dynamic disease that occurs across the age spectrum and can lead to cirrhosis and hepatocellular carcinoma. There are currently no US Food and Drug Administration (FDA) approved treatments for NAFLD; however, this is a field of active research. This review summarizes emerging pharmacotherapies for the treatment of adult and pediatric NAFLD. Investigated pharmacotherapies predominantly target bile acid signaling, insulin resistance, and lipid handling within the liver. Three drugs have gone on to phase III trials for which results are available. Of those, obeticholic acid is the single agent that demonstrates promise …

Effects Of A Postnatal Atrx Conditional Knockout In Neurons On Autism-Like Behaviours In Male And Female Mice., Nicole Martin-Kenny, Nathalie G Bérubé

Paediatrics Publications

BACKGROUND: Alpha-thalassemia/mental retardation, X-linked, or ATRX, is an autism susceptibility gene that encodes a chromatin remodeler. Mutations of ATRX result in the ATR-X intellectual disability syndrome and have been identified in autism spectrum disorder (ASD) patients. The mechanisms by which ATRX mutations lead to autism and autistic-like behaviours are not yet known. To address this question, we generated mice with postnatal Atrx inactivation in excitatory neurons of the forebrain and performed a battery of behavioural assays that assess autistic-like behaviours.

METHODS: Male and female mice with a postnatal conditional ablation of ATRX were generated using the Cre/lox system under the …

Seasonal Influence On Postoperative Hip Fracture Complications: Retrospective Cohort Of More Than 1000 Patients From A Tertiary-Care University Hospital, Obada Hasan, Mashal Amin, Fatima Mahmood, Amna Rabbani, Umar Rabbani, Shahryar Noordin

Department of Paediatrics and Child Health

Background: Hip fractures account for one of the most debilitating conditions affecting the general population amongst the developed and developing nations. Investigators aimed to study the influence of the ongoing season i.e. whether patients operated upon in summer or winter, on post-operative complications occurring within 30 days of surgical procedure for hip fractures at a tertiary care hospital and level 1 trauma center.
Materials and methods: Investigators conducted a non-funded, non-commercialized retrospective cohort of 1045 patients with hip fractures managed surgically. Primary exposure was surgical procedures undertaken during the summer months (April-September). Our primary outcome was determining post-operative complication rates …

Do New Trainees Pose A Threat To Postoperative Complications After Hip Fracture Surgeries? Retrospective Cohort Of 1045 Patients Over A Decade At A University Hospital, Obada Hasan, Mashal Amin, Umar Rabbani, Amna Rabbani, Fatima Mahmood, Shahryar Noordin

Department of Paediatrics and Child Health

Introduction: Induction of new residents and surgical trainees in most institutes occurs once a year. Fresh residents with no experience, may pose a threat to the surgical procedure outcome and there can be a potential increase in patients' morbidity and mortality as a result of this turnover. Literature is inconclusive about this effect. Our aim was to study the "new residents' induction effect" on postoperative complications after hip fracture surgeries.
Methodology: This is non funded non commercialized study from a university hospital. Investigators studied a retrospective cohort of 1045 adult hip fracture patients who were operated at our tertiary care …

Education And Safety For Children With Autism To Prevent Elopement (E.S.C.A.P.E.) Kits, Silvia Pereira-Smith, Jessie Montezuma, Nicole Vonada, Laura A. Carpenter, Andrea Boan

MUSC Conference Presentations

Per a study published in 2019, two-thirds of patients with autism seen by the Division of Developmental-Behavioral Pediatrics (DBP) at MUSC engaged in elopement behavior, a potentially life-threatening behavior also known as wandering, that can lead to serious injury or death. E.S.C.A.P.E. kits were created by Dr. Pereira and the MUSC DBP autism research team in 2017 to help prevent elopement. Original kits included a door/window alarm, child ID kit, stop sign visual aids, ID bracelet, and printed materials with additional prevention tips. Initial survey data showed that 17 of 22 caregivers who completed surveys after receiving a kit reported …

Failure To Thrive - An Overlooked Manifestation Of Kmt2b-Related Dystonia: A Case Presentation., Andrew Ng, Serena Galosi, Lisa Salz, Terence Wong, Caitlin Schwager, Shivarajan Amudhavalli, Rose N. Gelineau-Morel, Shimul Chowdhury, Rady Children’S Institute For Genomic Medicine Investigators, Jennifer Friedman

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: KMT2B-related dystonia is a recently described form of childhood onset dystonia that may improve with deep brain stimulation. Prior reports have focused on neurologic features including prominent bulbar involvement without detailing general health consequences that may result from orolingual dysfunction. We describe a family with novel KMT2B mutation with several members with failure to thrive to highlight this non-neurologic, but consequential impact of mutation in this gene.

CASE PRESENTATION: We present a case of a 15-year old female who was admitted and evaluated for failure to thrive. On exam, she had severe speech dysfluency, limited ability to protrude the …

Diabetes Conversation Map - A Novel Tool For Diabetes Management Self-Efficacy Among Type 2 Diabetes Patients In Pakistan: A Randomized Controlled Trial, Rubina Qasim, Sarfaraz Masih, Mohammad Tahir Yousafzai, Hakim Shah, Abdul Manan, Yousaf Shah, Muhammad Yaqoob, Abida Razzaq, Ajmal Khan, Atiya Rahman Khan Rohilla

Department of Paediatrics and Child Health

Background: This study aimed to measure the effect of diabetes education using the novel method of "diabetes conversation map (DCM)" as compared to routine counselling (RC) on diabetes management self-efficacy (DMSE) among patients living with type 2 diabetes in Karachi, Pakistan.
Methods: A parallel arm randomized controlled trial among patients with type 2 diabetes aged 30-60 years, with HbA1c > 7%, diagnosed for at least 5 yrs., was conducted at the national institute of diabetes and endocrinology in Karachi, Pakistan. A total 123 type 2 diabetes patients were randomized into DCM (n = 62) or RC (n = 61). Four weekly …

Pediatric Outpatient Utilization By Differing Medicaid Payment Models In The United States., Therese L Canares, Ari Friedman, Jonathan Rodean, Rebecca R Burns, Deena Berkowitz, Matt Hall, Elizabeth Alpern, Amanda Montalbano

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: In the United States (US), Medicaid capitated managed care costs are controlled by optimizing patients' healthcare utilization. Adults in capitated plans utilize primary care providers (PCP) more than emergency departments (ED), compared to fee-for-service (FFS). Pediatric data are lacking. We aim to determine the association between US capitated and FFS Medicaid payment models and children's outpatient utilization.

METHODS: This retrospective cohort compared outpatient utilization between two payment models of US Medicaid enrollees aged 1-18 years using Truven's 2014 Marketscan Medicaid database. Children enrolled > 11 months were included, and were excluded for eligibility due to disability/complex chronic condition, lack of …

Pathogenic Variants In Kptn Gene Identified By Clinical Whole-Genome Sequencing, Isabelle Thiffault, Andrea Atherton, Bryce Heese, Ahmed Abdelmoity, Kailash Pawar, Emily G. Farrow, Lee Zellmer, Neil A. Miller, Sarah E. Soden, Carol J. Saunders

Manuscripts, Articles, Book Chapters and Other Papers

Status epilepticus is not rare in critically ill intensive care unit patients, but its diagnosis is often delayed or missed. The mortality for convulsive status epilepticus is dependent on the underlying aetiologies and the age of the patients and thus varies from study to study. In this context, effective molecular diagnosis in a pediatric patient with a genetically heterogeneous phenotype is essential. Homozygous or compound heterozygous variants in KPTN have been recently associated with a syndrome typified by macrocephaly, neurodevelopmental delay, and seizures. We describe a comprehensive investigation of a 9-yr-old male patient who was admitted to the intensive care …

Investigation Of Japanese Encephalitis Virus As A Cause Of Acute Encephalitis In Southern Pakistan, April 2015-January 2018, Tazeen Fatima, Abida Rais, Erum Khan, Susan L. Hills, Trudy V. Chambers, Aneeta Hotwani, Shahida Qureshi, Saad Shafqat, Saima Malik, Farah Qamar, Fatima Mir, Anthony A. Marfin, Anita K. M. Zaidi, Asif Raza Khowaja, Sadia Shakoor

Department of Pathology and Laboratory Medicine

Background: Japanese encephalitis (JE) occurs in fewer than 1% of JE virus (JEV) infections, often with catastrophic sequelae including death and neuropsychiatric disability. JEV transmission in Pakistan was documented in 1980s and 1990s, but recent evidence is lacking. Our objective was to investigate JEV as a cause of acute encephalitis in Pakistan.
Methods: Persons aged ≥1 month with possible JE admitted to two acute care hospitals in Karachi, Pakistan from April 2015 to January 2018 were enrolled. Cerebrospinal fluid (CSF) or serum samples were tested for JEV immunoglobulin M (IgM) using the InBios JE DetectTM assay. Positive or equivocal samples …

Impact Of Conflict On Maternal And Child Health Service Delivery: A Country Case Study Of Afghanistan, Mohammed Shafiq Mirzazada, Zahra Ali Padhani, Sultana Jabeen, Malika Fatima, Arjumand Rizvi, Uzair Ansari, Jai K. Das, Zulfiqar Ahmed Bhutta

Woman and Child Health

Introduction: Since decades, the health system of Afghanistan has been in disarray due to ongoing conflict. We aimed to explore the direct effects of conflict on provision of reproductive, maternal, newborn, child and adolescent health and nutrition (RMNCAH&N) services and describe the contextual factors influencing these services.
Method: We conducted a quantitative analysis of secondary data on RMNCAH&N indicators and undertook a supportive qualitative study to help understand processes and contextual factors. For quantitative analysis, we stratified the various provinces of Afghanistan into minimal-, moderate- and severe conflict categories based on battle-related deaths from Uppsala Conflict Data Program (UCDP) and …

Facing Real-World Challenges Of Immunogenicity In Pediatric Inflammatory Bowel Disease., Kyle Gress, Julie A. Bass, Ryan S Funk, Ryan Morrow, Rachel Hasenkamp, Valentina Shakhnovich

Manuscripts, Articles, Book Chapters and Other Papers

No abstract provided.

Idiopathic Pulmonary Hemosiderosis - A Rare Cause Of Chronic Anemia, Ayesha Butt, Rashida Ahmed, Muhammad Dawood Amir Sheikh, Omar Khan, Nousheen Iqbal, Arshalooz Rahman, Javaid Khan

Medical College Documents

Idiopathic pulmonary hemosiderosis (IPH) is a rare disease marked by alveolar bleeding and accumulation of hemosiderin in the lungs. Here we present three cases of IPH. The first case is of a 26-year-old male with anemia, hemoptysis and dyspnea. Bronchoscopy confirmed diffuse alveolar hemorrhage (DAH). A diagnosis of IPH was made after ruling out other causes of DAH and observing good response to steroids. The patient's condition improved with prednisolone and azathioprine. The second case is of 26-year-old female with severe anemia. Imaging suggested IPH and lung biopsy confirmed it. She died shortly afterwards. The third case is of a …

Psychometric Properties Of Segmental Assessment Of Trunk Control In Infants And Toddlers With Down Syndrome., Megan Flores, Katy Mitchell, Christina Bickley, Carolyn P. Da Silva

Physical Therapy Collection

Purpose: This study investigated infants and toddlers with Down syndrome (DS) to determine: reliability of the Segmental Assessment of Trunk Control (SATCo), concurrent validity of the SATCo with the Gross Motor Function Measure (GMFM), and whether age and SATCo score predict GMFM score.

Methods: Eighteen infants and toddlers with DS were tested on the SATCo by 2 physical therapist (PT) raters. One PT rater administered the GMFM. After 2 weeks, PT raters rescored their recorded SATCo sessions. A third PT rater also scored the SATCo videos.

Results: Interrater reliability of the SATCo was moderate to good and …

Intergrowth-21st Project International Inter-Nda Standards For Child Development At 2 Years Of Age: An International Prospective Population-Based Study, Michelle Fernandes, José Villar, Alan Stein, Eleonora Staines Urias, Cutberto Garza, Cesar G. Victora, Fernando C. Barros, Enrico Bertino, Manorama Purwar, Maria Carvalho, Zulfiqar Ahmed Bhutta

Woman and Child Health

Objectives: To describe the construction of the international INTERGROWTH-21st Neurodevelopment Assessment (INTER-NDA) standards for child development at 2 years by reporting the cognitive, language, motor and behaviour outcomes in optimally healthy and nourished children in the INTERGROWTH-21st Project.
Design: Population-based cohort study, the INTERGROWTH-21st Project.
Setting: Brazil, India, Italy, Kenya and the UK.
Participants: 1181 children prospectively recruited from early fetal life according to the prescriptive WHO approach, and confirmed to be at low risk of adverse perinatal and postnatal outcomes.
Primary measures: Scaled INTER-NDA domain scores for cognition, language, fine and gross motor skills and behaviour; vision outcomes measured …

Fri0547 The Effect Of Corrected Inflammation, Oxidative Stress And Endothelial Dysfunction On Fmd Levels In Patients With Selected Chronic Diseases: A Quasi-Experimental Study., Mahmut Ilker Yilmaz, Micol Romano, Mustafa Kemal Basarali, Abdelbaset Elzagallaai, Murat Karaman, Zeynep Demir, Muhammet Fatih Demir, Fatih Akcay, Melik Seyrek, Nuri Haksever, David Piskin, Rolando Cimaz, Michael Rieder, Erkan Demirkaya

Paediatrics Publications

While the pathophysiology of chronic disorders varies there are three basic mechanisms - inflammation, oxidative stress and endothelial dysfunction - that are common in many chronic diseases. However, the failure of these mechanisms to work synchronously can lead to morbidity complicating the course of many chronic diseases. We analyzed data of 178 patients from cohorts with selected chronic diseases in this quasi-experimental study. Endothelial dysfunction was determined by flow-mediated dilatation (FMD) and asymmetric dimethylarginine (ADMA) levels. Serum ADMA, high sensitive C-reactive protein (hs-CRP), serum PTX3, malondialdehyde (MDA), Cu/Zn-superoxide dismutase (Cu/Zn-SOD), glutathione peroxidase (GSH-Px) levels and FMD were studied in baseline …

Teachers’ Perceptions And Understanding Of Children’S Fluid Intake, Kristy Howells, Tara Coppinger

Publications

No public health data exists on elementary teachers’ perceptions of both their own fluid intake and of their elementary school aged children’s fluid intake. A total of 271 (20 males, 251 females) teachers in developed areas of Australia, Belgium, England, Ireland, United Arab Emirates, and the United States of America completed an online questionnaire (Feb–Mar 2019) on: (i) their fluid intake, (ii) their perception and understanding of children’s fluid intake and (iii) barriers in the school day that they felt prevented school children consuming fluids. Overall, the data indicated that teachers consume considerably lower amounts than recommended themselves, but have …

Evaluation Of Pediatric Patients Diagnosed With Lyme Disease In The Lehigh Valley, Katiemarie Vottero Do, Jessica Phan, Kris Rooney Md, Tibisay Villalobos Md, Kristin M. Held Wheatley Pharmd, Bcop

Department of Pediatrics

No abstract provided.

Progressive Stridor: Extraintestinal Airway Manifestations In A Pediatric Patient With Inflammatory Bowel Disease, Kaitlyn Kuntzman Do, Sri Kiran Chennupati Md, Catherine April Bingham Md, Naser Tolaymat Md, Lauren Greenawald Do

Department of Pediatrics

No abstract provided.

The Effect Of Corrected Inflammation, Oxidative Stress And Endothelial Dysfunction On Fmd Levels In Patients With Selected Chronic Diseases: A Quasi-Experimental Study., Mahmut Ilker Yilmaz, Micol Romano, Mustafa Kemal Basarali, Abdelbaset Elzagallaai, Murat Karaman, Zeynep Demir, Muhammet Fatih Demir, Fatih Akcay, Melik Seyrek, Nuri Haksever, David Piskin, Rolando Cimaz, Michael Rieder, Erkan Demirkaya

Paediatrics Publications

While the pathophysiology of chronic disorders varies there are three basic mechanisms - inflammation, oxidative stress and endothelial dysfunction - that are common in many chronic diseases. However, the failure of these mechanisms to work synchronously can lead to morbidity complicating the course of many chronic diseases. We analyzed data of 178 patients from cohorts with selected chronic diseases in this quasi-experimental study. Endothelial dysfunction was determined by flow-mediated dilatation (FMD) and asymmetric dimethylarginine (ADMA) levels. Serum ADMA, high sensitive C-reactive protein (hs-CRP), serum PTX3, malondialdehyde (MDA), Cu/Zn-superoxide dismutase (Cu/Zn-SOD), glutathione peroxidase (GSH-Px) levels and FMD were studied in baseline …

Prevalence And Clinical Features Of Inflammatory Bowel Diseases Associated With Monogenic Variants, Identified By Whole-Exome Sequencing In 1000 Children At A Single Center, Eileen Crowley, Neil Warner, Jie Pan, Sam Khalouei, Abdul Elkadri, Karoline Fiedler, Justin Foong, Andrei L Turinsky, Dana Bronte-Tinkew, Shiqi Zhang

Paediatrics Publications

BACKGROUND & AIMS: A proportion of infants and young children with inflammatory bowel diseases (IBDs) have subtypes associated with a single gene variant (monogenic IBD). We aimed to determine the prevalence of monogenic disease in a cohort of pediatric patients with IBD.

METHODS: We performed whole-exome sequencing analyses of blood samples from an unselected cohort of 1005 children with IBD, aged 0-18 years (median age at diagnosis, 11.96 years) at a single center in Canada and their family members (2305 samples total). Variants believed to cause IBD were validated using Sanger sequencing. Biopsies from patients were analyzed by immunofluorescence and …

Randomized Phase 2 Trial And Open-Label Extension Of Domagrozumab In Duchenne Muscular Dystrophy., Kathryn R. Wagner, Hoda Z. Abdel-Hamid, Jean K. Mah, Craig Campbell, Michela Guglieri, Francesco Muntoni, Yasuhiro Takeshima, Craig M. Mcdonald, Anna Kostera-Pruszczyk, Peter Karachunski, Russell J. Butterfield, Eugenio Mercuri, Chiara Fiorillo, Enrico S. Bertini, Cuixia Tian, Jeffery Statland, Alesia B. Sadosky, Vivek S. Purohit, Sarah P. Sherlock, Jeffrey P. Palmer, Michael Binks, Lawrence Charnas, Shannon Marraffino, Brenda L. Wong

Paediatrics Publications

We report results from a phase 2, randomized, double-blind, 2-period trial (48 weeks each) of domagrozumab and its open-label extension in patients with Duchenne muscular dystrophy (DMD). Of 120 ambulatory boys (aged 6 to <16 >years) with DMD, 80 were treated with multiple ascending doses (5, 20, and 40 mg/kg) of domagrozumab and 40 treated with placebo. The primary endpoints were safety and mean change in 4-stair climb (4SC) time at week 49. Secondary endpoints included other functional tests, pharmacokinetics, and pharmacodynamics. Mean (SD) age was 8.4 (1.7) and 9.3 (2.3) years in domagrozumab- and placebo-treated patients, respectively. Difference in mean …

Authorizing Medical Cannabis For Children., Michael Rieder

Paediatrics Publications

No abstract provided.

Determining A Bayesian Predictive Power Stopping Rule For Futility In A Non-Inferiority Trial With Binary Outcomes., Anna Heath, Martin Offringa, Petros Pechlivanoglou, Juan David Rios, Terry P Klassen, Naveen Poonai, Eleanor Pullenayegum

Paediatrics Publications

Background/Aims: Non-inferiority trials investigate whether a novel intervention, which typically has other benefits (i.e., cheaper or safer), has similar clinical effectiveness to currently available treatments. In situations where interim evidence in a non-inferiority trial suggests that the novel treatment is truly inferior, ethical concerns with continuing randomisation to the "inferior" intervention are raised. Thus, if interim data indicate that concluding non-inferiority at the end of the trial is unlikely, stopping for futility should be considered. To date, limited examples are available to guide the development of stopping rules for non-inferiority trials.

Methods: We used a Bayesian predictive power approach to …