27 research outputs found
A National Spinal Muscular Atrophy Registry for Real-World Evidence.
BACKGROUND: Spinal muscular atrophy (SMA) is a devastating rare disease that affects individuals regardless of ethnicity, gender, and age. The first-approved disease-modifying therapy for SMA, nusinursen, was approved by Health Canada, as well as by American and European regulatory agencies following positive clinical trial outcomes. The trials were conducted in a narrow pediatric population defined by age, severity, and genotype. Broad approval of therapy necessitates close follow-up of potential rare adverse events and effectiveness in the larger real-world population.
METHODS: The Canadian Neuromuscular Disease Registry (CNDR) undertook an iterative multi-stakeholder process to expand the existing SMA dataset to capture items relevant to patient outcomes in a post-marketing environment. The CNDR SMA expanded registry is a longitudinal, prospective, observational study of patients with SMA in Canada designed to evaluate the safety and effectiveness of novel therapies and provide practical information unattainable in trials.
RESULTS: The consensus expanded dataset includes items that address therapy effectiveness and safety and is collected in a multicenter, prospective, observational study, including SMA patients regardless of therapeutic status. The expanded dataset is aligned with global datasets to facilitate collaboration. Additionally, consensus dataset development aimed to standardize appropriate outcome measures across the network and broader Canadian community. Prospective outcome studies, data use, and analyses are independent of the funding partner.
CONCLUSION: Prospective outcome data collected will provide results on safety and effectiveness in a post-therapy approval era. These data are essential to inform improvements in care and access to therapy for all SMA patients
What Behaviors and Personality Traits Diminish Perceptions of Attractiveness?
Extensive research has been conducted on the factors that determine whether an individual is perceived as attractive. Researchers have examined the relationship between physical attributes such as body shape, body mass index, facial symmetry, height, skin color, hair length, breast size, chest hair and perceptions of attractiveness (e.g., Choi, Kim, & Pasnak, 2014; Dagnino, Navajas, & Sigman, 2012; Swami, Furnham, & Joshi, 2008). Researchers have also studied the relationship between both positive and negative personality traits (e.g., honesty, intelligence, rudeness, sense of humor, lack of empathy, narcissism, manipulative) and perceptions of attractiveness (e.g., Jonason & Webster, 2010; Lewandowski, 2007; Whitbourne, 2013). The present study will seek to advance our understanding of attractiveness by examining the extent to which behaviors and personality traits that are perceived negatively by society diminish the perceived attractiveness of an individual. Participants will complete an anonymous, online survey and will first provide demographic information (e.g., gender, age, ethnicity, relationship status, area type (rural, suburban, urban), religious orientation, open-mindedness when identifying individuals whom they find attractive). Participants will then be asked to imagine there is a man or woman to whom they find themselves extremely attracted and evaluate the extent to which their perceptions of attractiveness would diminish if they were to learn that the man or woman engaged in behaviors or displayed personality traits viewed negatively by society. Analyses will examine gender differences in perceptions of these behaviors and personality traits and identify the behaviors and personality traits associated with the greatest diminishment in perceived attractiveness
Relationship of tissue dimensions and three captive bolt placements on cadaver heads from mature swine (Sus scrofa domesticus) > 200 kg body weight
Three penetrating captive bolt (PCB) placements were tested on cadaver heads from swine with estimated body weight (BW) >200 kg (sows = 232.9 ± 4.1 kg; boars = 229.3 ± 2.6 kg). The objectives were to determine tissue depth, cross-sectional brain area, visible brain damage (BD), regions of BD, and bolt-brain contact; and determine relationships between external head dimensions and tissue depth at each placement. A Jarvis PAS – Type P 0.25R PCB with a Long Stunning Rod Nosepiece Assembly and 3.5 gr power loads was used at the following placements on heads from 111 sows and 46 boars after storage at 2-4° C for approximately 62 h before treatment: FRONTAL (F) – 3.5 cm superior to the optic orbits at midline, TEMPORAL (T) – at the depression posterior to the lateral canthus of the eye within the plane between the lateral canthus and the base of the ear, or BEHIND EAR (BE) – directly caudal to the pinna of the ear on the same plane as the eyes and targeting the middle of the opposite eye. For sows, the bolt path was in the plane of the brain for 42/42 (100%, 95% CI: 91.6-100.0%) F heads, 39/40 (97.5%, 95% CI: 86.8-99.9%) T heads, and 34/39 (87.5%, 95% CI: 72.6-95.7%) BE heads; for the heads that could reliably be assessed for BD damage was detected in 25/26 (96.2%, 95% CI: 80.4-99.9%) F heads, 24/35 (68.6%, 95% CI: 50.7-83.2%) T heads, and 5/40 (12.5%, 95% CI: 4.2-26.8%) BE heads. For boars, the bolt path was in the plane of the brain for 17/17 (100.0%, 95% CI: 80.5-100.0%) F heads, 18/18 (100.0%, 95% CI: 81.5-100.0%) T heads, and 14/14 (100.0%, 95% CI: 76.8-100.0%) BE heads; damage was detected in 11/12 (91.7%, 95% CI: 61.5-99.8%) F heads, 2/15 (13.3%, 95% CI: 1.7-40.5%) T heads, and 7/14 (50.0%, 95% CI: 23.0-77.0%) BE heads. Tissue depth was reported as mean ± standard error followed by 95% one-sided upper reference limit (URL). For sows, total tissue thickness was different (P 200 kg BW, F placement may be more effective than T or BE due to less soft tissue thickness, which may reduce concussive force. The brain was within the plane of bolt travel for 100% of F heads with brain damage for 96.2% and 91.7% of F sow and boar heads, respectively.This is a pre-copyedited, author-produced version of an article accepted for publication in Journal of Animal Science following peer review. The version of record: Anderson, Karly N., Kaysie J. Allen, Angela Baysinger, Madonna Benjamin, Jennifer Berger, James R. Claus, Brian J. Greco et al. "Relationship of tissue dimensions and three captive bolt placements on cadaver heads from mature swine (Sus scrofa domesticus)> 200 kg body weight." 99, no. 12 Journal of Animal Science (2021) is available online at DOI: 10.1093/jas/skab327. Copyright The Author(s) 2021. Posted with permission
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Living kidney donors with HIV: experience and outcomes from a case series by the HOPE in Action Consortium.
BACKGROUND: Living kidney donation is possible for people living with HIV (PLWH) in the United States within research studies under the HIV Organ Policy Equity (HOPE) Act. There are concerns that donor nephrectomy may have an increased risk of end-stage renal disease (ESRD) in PLWH due to HIV-associated kidney disease and antiretroviral therapy (ART) nephrotoxicity. Here we report the first 3 cases of living kidney donors with HIV under the HOPE Act in the United States. METHODS: Within the HOPE in Action Multicenter Consortium, we conducted a prospective study of living kidney donors with HIV. Pre-donation, we estimated the 9-year cumulative incidence of ESRD, performed genetic testing of apolipoprotein L1 (APOL1), excluding individuals with high-risk variants, and performed pre-donation kidney biopsies (HOPE Act requirement). The primary endpoint was ≥grade 3 nephrectomy-related adverse events (AEs) in year one. Post-donation, we monitored glomerular filtration rate (measured by iohexol/Tc-99m DTPA [mGFR] or estimated with serum creatinine [eGFR]), HIV RNA, CD4 count, and ART. FINDINGS: There were three donors with two-four years of follow-up: a 35 year-old female, a 52 year-old male, and a 47 year-old male. Pre-donation 9-year estimated cumulative incidence of ESRD was 3.01, 8.01, and 7.76 per 10,000 persons, respectively. In two donors with APOL1 testing, no high-risk variants were detected. Biopsies from all three donors showed no kidney disease. Post-donation, two donors developed nephrectomy-related ≥grade 3 AEs: a medically-managed ileus and a laparoscopically-repaired incisional hernia. GFR declined from 103 to 84 mL/min/1.73 m2 at four years (mGFR) in donor 1, from 77 to 52 mL/min/1.73 m2 at three years (eGFR) in donor 2, and from 65 to 39 mL/min/1.73 m2 at two years (eGFR) in donor 3. HIV RNA remained <20 copies/mL and CD4 count remained stable in all donors. INTERPRETATION: The first three living kidney donors with HIV under the HOPE Act in the United States have had promising outcomes at two-four years, providing proof-of-concept to support living donation from PLWH to recipients with HIV. FUNDING: National Institute of Allergy and Infectious Diseases, National Institutes of Health
Outcomes of SOT Recipients With COVID-19 in Different Eras of COVID-19 Therapeutics.
BACKGROUND: Few reports have focused on newer coronavirus disease 2019 (COVID-19) therapies (remdesivir, dexamethasone, and convalescent plasma) in solid organ transplant recipients; concerns had been raised regarding possible adverse impact on allograft function or secondary infections. METHODS: We studied 77 solid organ transplant inpatients with COVID-19 during 2 therapeutic eras (Era 1: March-May 2020, 21 patients; and Era 2: June-November 2020, 56 patients) and 52 solid organ transplant outpatients. RESULTS: In Era 1, no patients received remdesivir or dexamethasone, and 4 of 21 (19.4%) received convalescent plasma, whereas in Era 2, remdesivir (24/56, 42.9%), dexamethasone (24/56, 42.9%), and convalescent plasma (40/56, 71.4%) were commonly used. Mortality was low across both eras, 4 of 77 (5.6%), and rejection occurred in only 2 of 77 (2.8%) inpatients; infections were similar in hypoxemic patients with or without dexamethasone. Preexisting graft dysfunction was associated with greater need for hospitalization, higher severity score, and lower survival. Acute kidney injury was present in 37.3% of inpatients; renal function improved more rapidly in patients who received remdesivir and convalescent plasma. Post-COVID-19 renal and liver function were comparable between eras, out to 90 d. CONCLUSIONS: Newer COVID-19 therapies did not appear to have a deleterious effect on allograft function, and infectious complications were comparable