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1.
Pediatr Qual Saf ; 6(4): e439, 2021.
Article in English | MEDLINE | ID: mdl-34345752

ABSTRACT

INTRODUCTION: Neurodevelopmental surveillance is critical for high-risk infants following neonatal intensive care discharge and is traditionally performed in-person. COVID-19 interruption of regular surveillance necessitated a rapid development of telehealth models for effective and standardized care. METHODS: We used implementation science and lean methodologies to develop an effective telehealth neurodevelopmental surveillance program for high-risk infants. Interventions included reorganization of visit flow processes and a telehealth toolkit for standardized neurological and developmental assessments. We tested and improved our intervention through plan-do-study-act cycles, value-added analysis, and parent- and provider-satisfaction questionnaires. Process metrics (standard elements, subspecialty referrals, diagnostic tests, and prescriptions ordered) were compared in group-level analyses between telehealth patients (N = 97) March 16, 2020-July 1, 2020 and a matched in-person cohort at the same period the previous year. Run charts examined shifts in balancing measures (provider efficiency and missed visits) over 8 weeks before and after implementation. RESULTS: Primary outcomes were visit completion (100%), patient parent satisfaction (>90% strongly agreed or agreed telehealth procedures were valuable and easy to use) and ability to accurately diagnose cerebral palsy (no statistical difference with comparison visits). Providers (N = 6) rated telehealth experiences favorably. Process metrics indicated no differences between telehealth and in-person visits (all P > 0.05). Following telehealth implementation, provider efficiency increased to near baseline (median 88.9% versus 91.7%) and median missed visits decreased to 0% from 20% (in-person). CONCLUSIONS: Implementation of telehealth for neurodevelopmental surveillance in a tertiary high-risk infant follow-up clinic successfully provided standardized and timely care during stay-at-home orders; broader telehealth applications may overcome access barriers in this field.

2.
J Med Chem ; 64(11): 7241-7260, 2021 06 10.
Article in English | MEDLINE | ID: mdl-34028270

ABSTRACT

Mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) ion channel are established as the primary causative factor in the devastating lung disease cystic fibrosis (CF). More recently, cigarette smoke exposure has been shown to be associated with dysfunctional airway epithelial ion transport, suggesting a role for CFTR in the pathogenesis of chronic obstructive pulmonary disease (COPD). Here, the identification and characterization of a high throughput screening hit 6 as a potentiator of mutant human F508del and wild-type CFTR channels is reported. The design, synthesis, and biological evaluation of compounds 7-33 to establish structure-activity relationships of the scaffold are described, leading to the identification of clinical development compound icenticaftor (QBW251) 33, which has subsequently progressed to deliver two positive clinical proofs of concept in patients with CF and COPD and is now being further developed as a novel therapeutic approach for COPD patients.


Subject(s)
Aminopyridines/chemistry , Cystic Fibrosis Transmembrane Conductance Regulator/metabolism , Administration, Oral , Aminopyridines/metabolism , Aminopyridines/therapeutic use , Animals , Cystic Fibrosis/drug therapy , Cystic Fibrosis Transmembrane Conductance Regulator/antagonists & inhibitors , Cystic Fibrosis Transmembrane Conductance Regulator/genetics , Disease Models, Animal , Drug Evaluation, Preclinical , Gene Deletion , Half-Life , Humans , Protein Binding , Pulmonary Disease, Chronic Obstructive/drug therapy , Rats , Rats, Sprague-Dawley , Solubility , Structure-Activity Relationship
3.
BMC Pediatr ; 19(1): 81, 2019 03 19.
Article in English | MEDLINE | ID: mdl-30890132

ABSTRACT

BACKGROUND: Premature infants are at risk for abnormal sensory development due to brain immaturity at birth and atypical early sensory experiences in the Neonatal Intensive Care Unit. This altered sensory development can have downstream effects on other more complex developmental processes. There are currently no interventions that address rehabilitation of sensory function in the neonatal period. METHODS: This study is a randomized controlled trial of preterm infants enrolled at 32-36 weeks postmenstrual age to either standard care or standard care plus multisensory intervention in order to study the effect of multisensory intervention as compared to standard care alone. The study population will consist of 100 preterm infants in each group (total n = 200). Both groups will receive standard care, consisting of non-contingent recorded parent's voice and skin-to-skin by parent. The multisensory group will also receive contemporaneous holding and light pressure containment for tactile stimulation, playing of the mother's voice contingent on the infant's pacifier sucking for auditory stimulation, exposure to a parent-scented cloth for olfactory stimulation, and exposure to carefully regulated therapist breathing that is mindful and responsive to the child's condition for vestibular stimulation. The primary outcome is a brain-based measure of multisensory processing, measured using time locked-EEG. Secondary outcomes include sensory adaptation, tactile processing, speech sound differentiation, motor and language function, measured at one and two years corrected gestational age. DISCUSSION: This is the first randomized controlled trial of a multisensory intervention using brain-based measurements in order to explain the causal effects of the multisensory intervention on neural processing changes to mediate neurodevelopmental outcomes in former preterm infants. In addition to contributing a critical link in our understanding of these processes, the protocolized multisensory intervention in this study is therapist administered, parent supported and leverages simple technology. Thus, this multisensory intervention has the potential to be widely implemented in various NICU settings, with the opportunity to potentially improve neurodevelopment of premature infants. TRIAL REGISTRATION: NIH Clinical Trials ( clinicaltrials.gov ): NCT03232931 . Registered July 2017.


Subject(s)
Infant, Premature , Language Development , Motor Skills , Neurodevelopmental Disorders/prevention & control , Electroencephalography , Female , Humans , Infant, Newborn , Intensive Care Units, Neonatal , Male , Nervous System Physiological Phenomena , Parents
4.
Med Educ Online ; 20: 28851, 2015.
Article in English | MEDLINE | ID: mdl-26344391

ABSTRACT

BACKGROUND: The role of peer teachers in interprofessional education has not been extensively studied. This study is designed to determine if peer-teacher-led problem-based seminars can influence medical and pharmacy students' perceptions of interprofessional education. METHODS: Undergraduate medical and pharmacy students participated in one-hour problem-based learning seminars held over the course of 16 weeks. A case-control study design was used to compare perceptions of interprofessional education between students who participated in seminars and students who did not participate in seminars. The validated Interdisciplinary Education Perception Scale (IEPS) was used to assess perceptions of interprofessional education and was distributed to medical and pharmacy students at the conclusion of 16 weeks of seminars. A two-tailed t-test was used to determine significance between groups. A survey was also distributed to all students regarding perceived barriers to involvement in interprofessional education training. RESULTS: In total, 97 students responded to IEPS (62 medical, 35 pharmacy). Data showed significantly higher perception of professional cooperation among medical students (p=0.006) and pharmacy students (p=0.02) who attended interprofessional seminars compared to those who did not attend. One hundred and nine students responded to the survey regarding perceived barriers to interprofessional education, with the two most common barriers being: 'I am not aware of interprofessional education opportunities' (61.5%) and 'I do not have time to participate' (52.3%). CONCLUSION: Based on this data we believe peer-teacher-led problem-based interprofessional seminars can be used to increase medical and pharmacy students' perceived need for professional cooperation. Currently, major barriers to interprofessional education involvement are awareness and time commitment. Undergraduate health professions education can incorporate student-led seminars to improve interprofessional education.


Subject(s)
Interprofessional Relations , Peer Group , Students, Medical/psychology , Students, Pharmacy/psychology , Clinical Competence , Cooperative Behavior , Female , Humans , Male , Perception , Problem-Based Learning , Professional Role , Time Factors
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