ABSTRACT
This study evaluated the effects of a staff training package on the frequency (rate) of trial presentations to children diagnosed with autism. The training consisted of a combination of repeated timings, modeling plus frequency building, and modeling in vivo with the client plus frequency building. The experimenters implemented 20-min training sessions or frequency-building sessions with staff that used 1-min timings for trial presentation in each phase. Training resulted in a higher frequency of trials across all 3 participants in the modeling and feedback phase.
ABSTRACT
BACKGROUND: Clinician reporting of symptomatic adverse events (AEs) in phase I trials uses the Common Terminology Criteria for Adverse Events (CTCAE). The utility of the patient-reported outcomes (PROs) version of the CTCAE (PRO-CTCAE) in this setting is unknown. This prospective, observational study compared patient- and clinician-reported symptomatic AEs in phase I patients. METHODS: Phase I study-eligible patients at Princess Margaret were surveyed with the PRO-CTCAE full-item library (78 symptomatic AEs) at baseline (BL), mid-cycle 1, and mid-cycle 2 (C2). Patient and trial characteristics, best response, and survival data were collected. Presence or absence of patient- (PRO-CTCAE) or clinician-reported symptomatic AEs were compared (kappa) at defined timepoints and overall (BL+ mid-cycle 1 + C2). RESULTS: Of 292 patients approached from May 2017 to January 2019, a total of 265 (90.8%) were consented, with 243 (91.7%) evaluable and 552 PRO-CTCAE surveys (completion rate = 98.7%) included in analyses. Evaluation of overall patient-reported symptomatic AEs identified 50 PRO-CTCAE and 11 CTCAE items with 10% or greater reporting frequency. Nineteen CTCAE items were reported as 1% or less despite matched PRO-CTCAE items reporting as 10% or greater. Underreported categories included sexual health, bodily emissions, and cognition. Clinician- relative to patient-reporting frequency (ratio) demonstrated 9 symptomatic AEs with a 50-fold or more lower clinician reporting rate. Overall patient-clinician agreement for individual symptomatic AEs ranged from poor (κ = 0.00-0.19) to moderate (κ = 0.40-0.59), with discordance driven by lack of clinician reporting. Dyspnea (κ = 0.54) and peripheral neuropathy (κ = 0.63) at BL and limb edema (κ = 0.55) at C2 demonstrated the highest patient-clinician agreement. CONCLUSIONS: Poor to moderate patient-clinician agreement for symptomatic AEs suggests clinician underreporting in phase I trials. Analyses of severity and interference PRO categories are ongoing.
Subject(s)
Antineoplastic Agents , Neoplasms , Patient Reported Outcome Measures , Antineoplastic Agents/adverse effects , Clinical Trials, Phase I as Topic , Cognition , Humans , Neoplasms/drug therapy , Prospective Studies , Surveys and QuestionnairesSubject(s)
Adenoviridae Infections , Adoptive Transfer , Infant, Newborn, Diseases , T-Lymphocytes , Adenoviridae Infections/immunology , Adenoviridae Infections/pathology , Adenoviridae Infections/therapy , Female , Humans , Infant, Newborn , Infant, Newborn, Diseases/immunology , Infant, Newborn, Diseases/pathology , Infant, Newborn, Diseases/therapy , Infant, Premature , T-Lymphocytes/immunology , T-Lymphocytes/transplantationABSTRACT
Venous thromboembolism (VTE) is being increasingly recognized in children with sickle cell disease (SCD). In a retrospective cohort study, we identified bilateral central venous catheter (CVC) placement as an independent risk factor for VTE. At our institution, the only indication for bilateral CVC placement in children with SCD is erythrocytapheresis. To investigate the impact of erythrocytapheresis on coagulation, we measured levels of natural anticoagulants in 11 patients with SCD on chronic erythrocytapheresis, immediately before and after apheresis. We demonstrated a statistically significant reduction in most parameters. Additional studies are needed to further investigate the exact etiology and clinical impact of this acute decrease.
