Publications by authors named "M Pera"

510 Publications

Age related treatment effect in type II Spinal Muscular Atrophy pediatric patients treated with nusinersen.

Neuromuscul Disord 2021 Apr 2. Epub 2021 Apr 2.

Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy. Electronic address:

Previous natural history studies suggest that type II SMA patients remain stable over one year but show some progression over two years. Since nusinersen approval, there has been increasing attention to identify more specific age-related changes. The aim of the study was to establish 12-month changes in a cohort of pediatric type II SMA treated with nusinersen and to establish possible patterns of treatment effect in relation to different variables such as age, baseline value and SMN2 copy number. The Hammersmith Functional Motor Scale Expanded and the Revised Upper Limb Module were performed at T0 and 12 months after treatment (T12). Data in treated patients were compared to available data in untreated patients collected by the same evaluators.Seventy-seven patients of age between 2.64 and 17.88 years (mean:7.47, SD:3.79) were included. On t-test there was an improvement, with increased mean scores between T0 and T12 on both scales (p < 0.001). Using multivariate linear regression analysis, age and baseline scores were predictive of changes on both scales (p < 0.05) while SMN2 copy number was not. Differences were also found between study cohort and untreated data on both scales (p < 0.001). At 12 months, an increase in scores was observed in all the age subgroups at variance with natural history data. Our real-world data confirm the treatment effect of nusinersen in pediatric type II SMA patients and that the data interpretation should take into account different variables. These data confirm and expand the ones already reported in the Cherish study.
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http://dx.doi.org/10.1016/j.nmd.2021.03.012DOI Listing
April 2021

Use of buprenorphine for those with employer-sponsored insurance during the initial phase of the COVID-19 pandemic.

J Subst Abuse Treat 2021 Apr 8;129:108384. Epub 2021 Apr 8.

RAND Corporation, Santa Monica, CA, United States of America.

Objective: To quantify weekly rates of use of buprenorphine for those with employer-based insurance and whether the rate differs based on county-level measures of race, historical fatal drug overdose rate, and COVID-19 case rate.

Methods: We used 2020 pharmaceutical claims for 4.8 million adults from a privately insured population to examine changes in the use of buprenorphine to treat opioid use disorder in 2020 during the onset of the COVID-19 pandemic. We quantified variation by examining changes in use rates across counties based on their fatal drug overdose rate in 2018, number of COVID-19 cases per capita, and percent nonwhite.

Results: Weekly use of buprenorphine was relatively stable between the first week of January (0.6 per 10,000 enrollees, 95%CI = 0.2 to 1.1) and the last week of August (0.8 per 10,000 enrollees, 95%CI = 0.4 to 1.3). We did not find evidence of any consistent change in use of buprenorphine by county-level terciles for COVID-19 rate as of August 31, 2020, age-adjusted fatal drug overdose rate, and percent nonwhite. Use was consistently higher for counties in the highest tercile of county age-adjusted fatal drug overdose rate when compared to counties in the lowest tercile of county age-adjusted fatal drug overdose rate.

Discussion: Our results provide early evidence that new federal- and state-level policies may have steadied the rate of using buprenorphine for those with employer-based insurance during the pandemic.
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http://dx.doi.org/10.1016/j.jsat.2021.108384DOI Listing
April 2021

Updates for contributors: Rigor and reproducibility in stem cell research.

Authors:
Martin Pera

Stem Cell Reports 2021 May 8. Epub 2021 May 8.

Editor-in-Chief, Stem Cell Reports.

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http://dx.doi.org/10.1016/j.stemcr.2021.05.001DOI Listing
May 2021

Biomedical and societal impacts of in vitro embryo models of mammalian development.

Stem Cell Reports 2021 May;16(5):1021-1030

Department of Genetics, University of Cambridge, Cambridge CB2 3EH, UK.

In recent years, a diverse array of in vitro cell-derived models of mammalian development have been described that hold immense potential for exploring fundamental questions in developmental biology, particularly in the case of the human embryo where ethical and technical limitations restrict research. These models open up new avenues toward biomedical advances in in vitro fertilization, clinical research, and drug screening with potential to impact wider society across many diverse fields. These technologies raise challenging questions with profound ethical, regulatory, and social implications that deserve due consideration. Here, we discuss the potential impacts of embryo-like models, and their biomedical potential and current limitations.
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http://dx.doi.org/10.1016/j.stemcr.2021.03.023DOI Listing
May 2021

Patient-reported outcomes after oesophagectomy in the multicentre LASER study.

Br J Surg 2021 May 11. Epub 2021 May 11.

Oesophago-gastric Centre, Churchill Hospital, University of Oxford, Oxford, UK.

Background: Data on the long-term symptom burden in patients surviving oesophageal cancer surgery are scarce. The aim of this study was to identify the most prevalent symptoms and their interactions with health-related quality of life.

Methods: This was a cross-sectional cohort study of patients who underwent oesophageal cancer surgery in 20 European centres between 2010 and 2016. Patients had to be disease-free for at least 1 year. They were asked to complete a 28-symptom questionnaire at a single time point, at least 1 year after surgery. Principal component analysis was used to assess for clustering and association of symptoms. Risk factors associated with the development of severe symptoms were identified by multivariable logistic regression models.

Results: Of 1081 invited patients, 876 (81.0 per cent) responded. Symptoms in the preceding 6 months associated with previous surgery were experienced by 586 patients (66.9 per cent). The most common severe symptoms included reduced energy or activity tolerance (30.7 per cent), feeling of early fullness after eating (30.0 per cent), tiredness (28.7 per cent), and heartburn/acid or bile regurgitation (19.6 per cent). Clustering analysis showed that symptoms clustered into six domains: lethargy, musculoskeletal pain, dumping, lower gastrointestinal symptoms, regurgitation/reflux, and swallowing/conduit problems; the latter two were the most closely associated. Surgical approach, neoadjuvant therapy, patient age, and sex were factors associated with severe symptoms.

Conclusion: A long-term symptom burden is common after oesophageal cancer surgery.
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http://dx.doi.org/10.1093/bjs/znab124DOI Listing
May 2021