Background: Elexacaftor/tezacaftor/ivacaftor (ETI) is a highly effective therapy that improves lung disease in people with cystic fibrosis (pwCF), but its effect on glucose tolerance and insulin secretion is unclear.
Methods: PROMISE is a multicenter prospective, observational study of ETI in pwCF ≥12 years and at least one F508del allele. The PROMISE Endocrine sub-study (PROMISE-ENDO) enrolled participants at 10 CF Centers where hemoglobin A1c (HbA1c) was collected and 3-hour oral glucose tolerance tests (OGTT) conducted to examine glucose tolerance, glucose excursions, insulin secretory rates (deconvolution of C-peptide) and sensitivity (oral minimal model) prior to ETI and 12-18 months (mos) and 24-30 mos following ETI initiation.
Transplanted islet functional β-cell mass is measured by the β-cell secretory capacity derived from the acute insulin response to glucose-potentiated arginine (AIRpot), however, data are limited beyond one-year post-transplant for individuals with type 1 diabetes. We evaluated changes in β-cell secretory capacity in a single-center longitudinal analysis and examined relationships with measures of islet cell hormone metabolism and clinical measures of graft function (mixed-meal tolerance test [MMTT] C-peptide, BETA-2 score, and continuous glucose monitoring [CGM]). Eleven individuals received purified human pancreatic islets over one or two intra-portal infusions to achieve insulin-independence and were followed over a median (IQR) 6 (5-7) years.
View Article and Find Full Text PDFBackground: Prenatal glucocorticoids are one of the most widely proposed prenatal programming mechanisms, yet few studies exist that measure fetal cortisol via neonatal hair. Neonatal hair provides a window into the fetal experience and represents cortisol accumulation in the third trimester of pregnancy. In the current study, we test the links between two types of anxiety over the course of gestation (pregnancy-related anxiety and general anxiety) with neonatal hair cortisol.
View Article and Find Full Text PDFContext: Elexacaftor/tezacaftor/ivacaftor (ETI; Trikafta) enhances aberrant cystic fibrosis transmembrane conductance regulator function and may improve the insulin secretory defects associated with a deterioration in clinical outcomes in pancreatic insufficient cystic fibrosis (PI-CF).
Objective: This longitudinal case-control study assessed changes in β-cell function and secretory capacity measures over 2 visits in individuals with PI-CF who were initiated on ETI after the baseline visit (2012-2018) and (1) restudied between 2019 and 2021 (ETI group) vs (2) those restudied between 2015 and 2018 and not yet treated with cystic fibrosis transmembrane conductance regulator modulator therapy (controls).
Methods: Nine ETI participants (mean ± SD age, 25 ± 5 years) and 8 matched controls were followed up after a median (interquartile range) 5 (4-7) and 3 (2-3) years, respectively (P < .
This paper presents two studies conducted to develop and evaluate a new pragmatic measure of therapist adherence to Dialectical Behavior Therapy (DBT): the DBT Adherence Checklist for Individual Therapy (DBT AC-I). Study 1 used item response analysis to select items from the gold standard DBT Adherence Coding Scale (DBT ACS) using archival data from 1271 DBT sessions. Items were then iteratively refined based on feedback from 33 target end-users to ensure relevance, usability, and understandability.
View Article and Find Full Text PDFImportance: Prenatal depression is prevalent with negative consequences for both the mother and developing fetus. Brief, effective, and safe interventions to reduce depression during pregnancy are needed.
Objective: To evaluate depression improvement (symptoms and diagnosis) among pregnant individuals from diverse backgrounds randomized to brief interpersonal psychotherapy (IPT) vs enhanced usual care (EUC).
Repeated hypoglycemia exposure leads to impaired awareness of hypoglycemia (IAH) and the development of defective counterregulatory responses. To date, only pancreas or islet transplantation has demonstrated normalization of hypoglycemia awareness and the endogenous glucose production (EGP) response to defend against insulin-induced hypoglycemia in long-standing type 1 diabetes (T1D). This study aims to validate clinical metrics of IAH (Clarke score), hypoglycemia severity (HYPO score), glycemic lability (lability index), and continuous glucose monitoring (CGM) as predictors of absent autonomic symptom (AS) recognition and defective glucose counterregulation during insulin-induced hypoglycemia, thus enabling early identification of individuals with compromised physiologic defense against clinically significant hypoglycemia.
View Article and Find Full Text PDFIdentification of new medications for alcohol use disorder (AUD) is important for improving treatment options. Baclofen, a GABA agonist, has been identified as a potential pharmacotherapy for AUD. In a 16-week double-blind, randomized, placebo-controlled trial, we investigated 30 and 90 mg/day of baclofen compared to placebo and examined effects of dose, sex, and level of pretreatment drinking.
View Article and Find Full Text PDFThe Dialectical Behavior Therapy Prolonged Exposure (DBT PE) protocol improves DBT's effects on PTSD in research settings, but its effectiveness in community settings is largely unknown. This pilot nonrandomized controlled trial examined DBT with and without DBT PE in four public mental health agencies. Patients (N = 35, 12-56 years old, 80.
View Article and Find Full Text PDFThe Dialectical Behavior Therapy Adherence Coding Scale (DBT ACS) is an observer-rated measure used to evaluate the extent to which therapists deliver individual and group DBT with adherence to the manual. Despite its frequent use in clinical trials of DBT, relatively little is known about its psychometric properties. The present study utilized data from six clinical trials conducted in research and community settings with a variety of patient populations.
View Article and Find Full Text PDFBackground: Enrichment strategies improve therapeutic targeting and trial efficiency, but enrichment factors for sepsis trials are lacking. We determined whether concentrations of soluble tumor necrosis factor receptor-1 (sTNFR1), interleukin-8 (IL8), and angiopoietin-2 (Ang2) could identify sepsis patients at higher mortality risk and serve as prognostic enrichment factors.
Methods: In a multicenter prospective cohort study of 400 critically ill septic patients, we derived and validated thresholds for each marker and expressed prognostic enrichment using risk differences (RD) of 30-day mortality as predictive values.
Objective: Primary care has been promoted as a setting to identify and manage adolescent depression. This study examined primary care-based adolescent depression identification and follow-up care when elevated symptoms were identified.
Methods: Data came from a large pediatric care network with an organizational recommendation to screen for depression at age 16 well-visits using an electronic health record (EHR)-integrated standardized measure.
Previous research has demonstrated the effectiveness of school-based depression prevention programs in reducing depressive symptoms and improving functioning. This study examined whether these programs have positive effects on school-related outcomes. Students at 10 middle and high schools in New Jersey were randomized to weekly sessions of Interpersonal Psychotherapy - Adolescent Skills Training (IPT-AST) or group counseling (GC).
View Article and Find Full Text PDFResearch on routine outcome monitoring in psychotherapy settings is plentiful but not without implementation obstacles. In fact, there is a relative dearth of real-time outcome monitoring in substance use treatment settings. Numerous barriers to the development and implementation of clinical decision support tools and outcome monitoring of substance use patients, including the need to establish expected trajectories of change and use of reliable change indices have been identified (Goodman, McKay, & DePhilippis, 2013 ).
View Article and Find Full Text PDFImportance: Identification of moderators of the response to naltrexone hydrochloride treatment for alcohol dependence could improve clinical care for patients with alcohol use disorders.
Objective: To investigate the preliminary finding that the sweet-liking (SL) phenotype interacts with a high level of craving for alcohol and is associated with an improved response to naltrexone in alcohol dependence.
Design, Setting, And Participants: This 12-week double-blind, randomized, placebo-controlled clinical trial was conducted from February 1, 2010, to April 30, 2012, in an academic outpatient medical center.
Objective: An empirically derived prediction model was developed in a private practice setting to monitor on-track and off-track weekly treatment progress in an intensive outpatient program (IOP).
Method: The predictive equation was derived as a function of the baseline measure and time. The formulae for the predictive equations were derived from two groups of psychiatric patients (N = 400 each) in an IOP diagnosed with major depression.