Cost Utility of Voretigene Neparvovec for Biallelic RPE65-Mediated Inherited Retinal Disease

Objective

The gene therapy voretigene neparvovec (VN) is the first Food and Drug Administration–approved treatment for vision loss owing to the ultra-rare RPE65-mediated inherited retinal disorders. We modeled the cost-utility of VN compared with standard of care (SoC).

Did quality of life for older cancer survivors improve with the turn of the century in the United States?

ObjectivesAlthough survival after a cancer diagnosis has improved considerably over the past 20 years, little is known about trends in health-related quality-of-life (HRQOL) for older prostate, breast, and lung cancer survivors.MethodsUsing a population-based registry with longitudinal patient reported outcomes (the National Cancer Institute Surveillance, Epidemiology and End Results database linked to Medicare Health Outcomes Survey), we analyzed Medicare Advantage patients diagnosed with cancer during 1998–2011, who completed surveys regarding HRQOL through 2013. ‘Early Era’ patients were treated during 1998–2003; ‘Late Era’ patients were treated during 2006–2011. After propensity score matching, post-diagnosis changes in health utility (HU), Physical Component Summary (PCS) and Mental Component Summary (MCS) scores were calculated and compared between the two eras.ResultWe identified 208 older patients with prostate, 276 with breast and 76 with lung cancer who were treated in the ‘Early Era’ and matched to equal numbers in the ‘Late Era’. Mean age of patients in early and late era was 72 and 73 years, respectively. The mean post-diagnosis decline in health utility for patients treated in the ‘Late Era’ was not significantly different from the ‘Early Era’ for any cancer (Prostate [early vs. late]: ?0.06 vs. -0.03, p = .09; Breast: ?0.03 vs. ?0.04, p = .65; Lung: ?0.07 vs. ?0.07, p = .95); nor for Physical Component Summary or Mental Component Summary scores.ConclusionOlder patients treated for prostate, breast or lung cancer in the later era reported similar outcomes of changes in HRQOL compared to earlier era patients.     

Correlative polarizing light and scanning electron microscopy for the assessment of talc in pelvic region lymph nodes

Perineal talc use is associated with ovarian carcinoma in many case-control studies. Such talc may migrate to pelvic organs and regional lymph nodes, with both clinical and legal significance. Our goal was to differentiate talc in pelvic lymph nodes due to exposure, versus contamination with talc in the laboratory. We studied 22 lymph nodes from ovarian tumor patients, some of which had documented talc exposure, to quantify talc using digestion of tissue taken from paraffin blocks and scanning electron microscopy/energy dispersive X-ray analysis (SEM/EDX). Talc particles correlated significantly with surface contamination assessments using polarized light microscopy. After adjusting for surface contamination, talc burdens in nodes correlated strongly with perineal talc use. In a separate group of lymph nodes, birefringent particles within the same plane of focus as the tissues in histological sections were highly correlated with talc particles within the tissue by in situ SEM/EDX (r = 0.80; p < 0.0001). We conclude that since talc can be a surface contaminant from tissue collection/preparation, digestion measurements may be influenced by contamination. Instead, because they preserve anatomic landmarks and permit identification of particles in cells and tissues, polarized light microscopy and in situ SEM/EDX are recommended to assess talc in lymph nodes.     

Evaluation of Training Program for the Maternal and Child Health Workforce at Tulane University

Objectives Despite significant investments in Maternal and Child Health (MCH), the United States still lags behind other countries in key MCH indicators. A well-trained workforce is needed to improve MCH. The Division of MCH Workforce Development of HRSA’s Maternal and Child Health Bureau provides funding to schools of Public Health to support Centers of Excellence in MCH, which is focused on preparing the next generation of MCH leaders through specialized training and mentorship. One such center, the Tulane Center of Excellence in MCH (CEMCH), is housed at the Tulane University School of Public Health and Tropical Medicine. This study evaluated the perceived effectiveness and acceptability of the CEMCH leadership training program. Methods A mixed-methods approach was used, consisting of semi-structured interviews and quantitative surveys which were analyzed through inductive methods based in grounded theory and non-parametric methods respectively. Results Results indicated an overall high level of program satisfaction by all stakeholders. Mentorship and personal attention emerged as an important benefit for both former and current Scholars. The opportunity to gain real-world understanding of MCH work through program activities was an added benefit, although these activities also presented the most challenges. Community stakeholders generally did not view the program as providing immediate organizational benefit, but recognized the distal benefit of contributing to a well-trained MCH workforce. Conclusions for Practice These results will be used to inform other MCH training programs and strengthen Tulane’s CEMCH. A well-trained MCH workforce is essential to improving MCH, and high-quality training its foundation.     

Rationale for the use of a single fixed intravenous dolasetron dose for the prevention of cisplatin-induced nausea and vomiting

Dolasetron mesilate is a selective 5-HT₃ receptor antagonist that prevents chemotherapy-induced and postoperative nausea and vomiting. For the majority of patients in intravenous dolasetron trials for chemotherapy-induced nausea and vomiting, dosing has been based on body weight (mg/kg). The approved weight-based dose is 1.8 mg/kg based on results of controlled clinical trials. However, trials of dolasetron evaluating oral doses for prevention of chemotherapy-induced emesis, and intravenous doses for prevention and treatment of postoperative emesis have used a fixed milligram dose. To identify an appropriate intravenous fixed milligram dose for the prevention of chemotherapy-induced nausea and vomiting, this analysis was performed to derive efficacy results for fixed milligram doses from pooled results obtained with dosing based on body weight. Intravenous dolasetron doses for 1,598 patients treated on a mg/kg basis (0.3, 0.6, 1.2, 1.8, 2.4, 3.0 and 5.0 mg/kg) in 14 clinical trials were converted to fixed milligram doses based on weight. Fixed-dose groups were established at doses of 50, 75, 100, 125, 150, and 200 mg. Doses less than or equal to the midpoint between two dose groups were included in the lower dose group. Pooled results showed that the 100 mg intravenous dolasetron dose group (who received actual doses of 88–112 mg) produced the highest rate (53%) of complete response (0 emetic episodes and no rescue medication in the 24-h period following initiation of chemotherapy).     

A Network Approach to Studying the Associations Between Posttraumatic Stress Disorder Symptoms and Dissociative Experiences

In recent years, there has been a growing recognition of a dissociative subtype of posttraumatic stress disorder (D-PTSD), characterized by experiences of depersonalization (DP) and derealization (DR), among individuals with PTSD. Little is known, however, about how experiences of DP and/or DR are associated with the experience of other PTSD symptoms. The central aim of the present paper was to explore the associations among DP, DR, and other PTSD symptoms by means of a network analysis of cross-sectional data for 557 participants whose overall self-reported PTSD symptom severity warranted a probable PTSD diagnosis. Three notable findings emerged: (a) a strong association between DP and DR, (b) the identification of DP as the most central symptom in the network, and (c) the discovery that clusters of symptoms in the network were roughly consistent with DSM-5 PTSD criteria. We discuss these findings in light of some considerations, including the nature of our sample and the limits of interpreting cross-sectional network models.