Method for Using Rubric Ratings on Fishbone Diagrams to Compare Case Studies

Background: In multi-case study program evaluations, the large amount of qualitative data that are generated from interviews can be difficult to utilize. This is particularly so when inference must be made as to why some cases succeed and some fail. Purpose: This paper shows a method for comparing multiple evaluation sites by using a rubric to define ratings for relevant factors, and an Ishikawa fishbone diagram as a model to show relationships among those factors. We show how this technique identified reasons for differences in outcomes among the sites. Setting: The evaluation setting was a large-scale safety innovation in the U.S. railroad industry. Four cases were considered—two passenger railroads and two freight railroads. Intervention: The Confidential Close Call Reporting System (C3RS) program allowed railroad workers to confidentially submit “close calls” which were reviewed by a team made up of labor, management, and the Federal Railroad administration to determine ways to improve safety. Research design: Multiple comparative case study, Ishikawa root and contributing cause modeling, evaluative rubric scoring, and data visualization techniques. Data collection & analysis: Interview data were collected from four pilot railroad sites, each of which participated in a five-year test of C3RS. Testing periods overlapped, with the entire evaluation lasting about 12 years. Findings: The method of using Ishikawa fishbone diagrams with ratings from an evaluative rubric was an effective method to summarize, analyze, and present large quantities of qualitative data. The approach succeeded in explaining degrees of success and failure across the sites. The sponsor and industry stakeholders were able to understand the analysis and the findings, and to develop deep insight into how to promote successful implementation. Keywords: Multiple comparative case studies; qualitative methodology; qualitative coding; data visualization; fishbone diagrams; Ishikawa diagrams; evaluative rubrics; close calls; near miss; data visualization

The need for establishing a universal CTG sizing method in myotonic dystrophy type 1

The number of cytosine-thymine-guanine (CTG) repeats (‘CTG expansion size’) in the 3′untranslated region (UTR) region of the dystrophia myotonica-protein kinase (DMPK) gene is a hallmark of myotonic dystrophy type 1 (DM1), which has been related to age of disease onset and clinical severity. However, accurate determination of CTG expansion size is challenging due to its characteristic instability. We compared five different approaches (heat pulse extension polymerase chain reaction [PCR], long PCR-Southern blot [with three different primers sets—1, 2 and 3] and small pool [SP]-PCR) to estimate CTG expansion size in the progenitor allele as well as the most abundant CTG expansion size, in 15 patients with DM1. Our results indicated variability between the methods (although we found no overall differences between long PCR 1 and 2 and SP-PCR, respectively). While keeping in mind the limited sample size of our patient cohort, SP-PCR appeared as the most suitable technique, with an inverse significant correlation found between CTG expansion size of the progenitor allele, as determined by this method, and age of disease onset (r = −0.734, p = 0.016). Yet, in light of the variability of the results obtained with the different methods, we propose that an international agreement is needed to determine which is the most suitable method for assessing CTG expansion size in DM1

Heritability of non-speech auditory processing skills

Recent insight into the genetic bases for autism spectrum disorder, dyslexia, stuttering, and language disorders suggest that neurogenetic approaches may also reveal at least one etiology of auditory processing disorder (APD). A person with an APD typically has difficulty understanding speech in background noise despite having normal pure-tone hearing sensitivity. The estimated prevalence of APD may be as high as 10% in the pediatric population, yet the causes are unknown and have not been explored by molecular or genetic approaches. The aim of our study was to determine the heritability of frequency and temporal resolution for auditory signals and speech recognition in noise in 96 identical or fraternal twin pairs, aged 6–11 years. Measures of auditory processing (AP) of non-speech sounds included backward masking (temporal resolution), notched noise masking (spectral resolution), pure-tone frequency discrimination (temporal fine structure sensitivity), and nonsense syllable recognition in noise. We provide evidence of significant heritability, ranging from 0.32 to 0.74, for individual measures of these non-speech-based AP skills that are crucial for understanding spoken language. Identification of specific heritable AP traits such as these serve as a basis to pursue the genetic underpinnings of APD by identifying genetic variants associated with common AP disorders in children and adults

Effects of antiplatelet therapy on stroke risk by brain imaging features of intracerebral haemorrhage and cerebral small vessel diseases: subgroup analyses of the RESTART randomised, open-label trial

Background Findings from the RESTART trial suggest that starting antiplatelet therapy might reduce the risk of recurrent symptomatic intracerebral haemorrhage compared with avoiding antiplatelet therapy. Brain imaging features of intracerebral haemorrhage and cerebral small vessel diseases (such as cerebral microbleeds) are associated with greater risks of recurrent intracerebral haemorrhage. We did subgroup analyses of the RESTART trial to explore whether these brain imaging features modify the effects of antiplatelet therapy

Obeticholic acid for the treatment of non-alcoholic steatohepatitis: interim analysis from a multicentre, randomised, placebo-controlled phase 3 trial

Background Non-alcoholic steatohepatitis (NASH) is a common type of chronic liver disease that can lead to cirrhosis. Obeticholic acid, a farnesoid X receptor agonist, has been shown to improve the histological features of NASH. Here we report results from a planned interim analysis of an ongoing, phase 3 study of obeticholic acid for NASH. Methods In this multicentre, randomised, double-blind, placebo-controlled study, adult patients with definite NASH,non-alcoholic fatty liver disease (NAFLD) activity score of at least 4, and fibrosis stages F2–F3, or F1 with at least oneaccompanying comorbidity, were randomly assigned using an interactive web response system in a 1:1:1 ratio to receive oral placebo, obeticholic acid 10 mg, or obeticholic acid 25 mg daily. Patients were excluded if cirrhosis, other chronic liver disease, elevated alcohol consumption, or confounding conditions were present. The primary endpointsfor the month-18 interim analysis were fibrosis improvement (≥1 stage) with no worsening of NASH, or NASH resolution with no worsening of fibrosis, with the study considered successful if either primary endpoint was met. Primary analyses were done by intention to treat, in patients with fibrosis stage F2–F3 who received at least one dose of treatment and reached, or would have reached, the month 18 visit by the prespecified interim analysis cutoff date. The study also evaluated other histological and biochemical markers of NASH and fibrosis, and safety. This study is ongoing, and registered with ClinicalTrials.gov, NCT02548351, and EudraCT, 20150-025601-6. Findings Between Dec 9, 2015, and Oct 26, 2018, 1968 patients with stage F1–F3 fibrosis were enrolled and received at least one dose of study treatment; 931 patients with stage F2–F3 fibrosis were included in the primary analysis (311 in the placebo group, 312 in the obeticholic acid 10 mg group, and 308 in the obeticholic acid 25 mg group). The fibrosis improvement endpoint was achieved by 37 (12%) patients in the placebo group, 55 (18%) in the obeticholic acid 10 mg group (p=0·045), and 71 (23%) in the obeticholic acid 25 mg group (p=0·0002). The NASH resolution endpoint was not met (25 [8%] patients in the placebo group, 35 [11%] in the obeticholic acid 10 mg group [p=0·18], and 36 [12%] in the obeticholic acid 25 mg group [p=0·13]). In the safety population (1968 patients with fibrosis stages F1–F3), the most common adverse event was pruritus (123 [19%] in the placebo group, 183 [28%] in the obeticholic acid 10 mg group, and 336 [51%] in the obeticholic acid 25 mg group); incidence was generally mild to moderate in severity. The overall safety profile was similar to that in previous studies, and incidence of serious adverse events was similar across treatment groups (75 [11%] patients in the placebo group, 72 [11%] in the obeticholic acid 10 mg group, and 93 [14%] in the obeticholic acid 25 mg group). Interpretation Obeticholic acid 25 mg significantly improved fibrosis and key components of NASH disease activity among patients with NASH. The results from this planned interim analysis show clinically significant histological improvement that is reasonably likely to predict clinical benefit. This study is ongoing to assess clinical outcomes

Method for Using Rubric Ratings on Fishbone Diagrams to Compare Case Studies

Background: In multi-case study program evaluations, the large amount of qualitative data that are generated from interviews can be difficult to utilize. This is particularly so when inference must be made as to why some cases succeed and some fail. Purpose: This paper shows a method for comparing multiple evaluation sites by using a rubric to define ratings for relevant factors, and an Ishikawa fishbone diagram as a model to show relationships among those factors. We show how this technique identified reasons for differences in outcomes among the sites. Setting: The evaluation setting was a large-scale safety innovation in the U.S. railroad industry. Four cases were considered—two passenger railroads and two freight railroads. Intervention: The Confidential Close Call Reporting System (C3RS) program allowed railroad workers to confidentially submit “close calls” which were reviewed by a team made up of labor, management, and the Federal Railroad administration to determine ways to improve safety. Research design: Multiple comparative case study, Ishikawa root and contributing cause modeling, evaluative rubric scoring, and data visualization techniques. Data collection & analysis: Interview data were collected from four pilot railroad sites, each of which participated in a five-year test of C3RS. Testing periods overlapped, with the entire evaluation lasting about 12 years. Findings: The method of using Ishikawa fishbone diagrams with ratings from an evaluative rubric was an effective method to summarize, analyze, and present large quantities of qualitative data. The approach succeeded in explaining degrees of success and failure across the sites. The sponsor and industry stakeholders were able to understand the analysis and the findings, and to develop deep insight into how to promote successful implementation. Keywords: Multiple comparative case studies; qualitative methodology; qualitative coding; data visualization; fishbone diagrams; Ishikawa diagrams; evaluative rubrics; close calls; near miss; data visualization

Program evaluation in social research

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