bluuebunny/crossref_metadata_2025_split huggingface dataset:
Total marrow and lymphoid irradiation as conditioning in haploidentical transplant with posttransplant cyclophosphamide
Authors: Al Malki Monzr M. , Palmer Joycelynne , Tsai Ni-Chun , Mokhtari Sally , Hui Susanta , Tsai Weimin , Aldoss Ibrahim , Ali Haris , Aribi Ahmed , Cao Thai , Mei Mathew , Sandhu Karamjeet S. , Siddiqi Tanya , Forman Stephen J. , Nakamura Ryotaro , Marcucci Guido , Stein Anthony , Wong Jeffrey Y. C. , Rosenthal Joseph 
Published: July 2022

Abstract
Posttransplant cyclophosphamide (PTCy) platform has shown low rates of graft-versus-host disease (GVHD) and nonrelapse mortality (NRM) after haploidentical hematopoietic cell transplantation (HaploHCT). However, because of the limited disease control, relapse rate remains a major cause of treatment failure in high-risk patients. Total marrow and lymphoid irradiation (TMLI) allows for delivery of high radiation to bone marrow and other targeted structures, without increasing off-target radiation exposure and toxicity to end organs. In this phase 1 trial, 31 patients with high-risk and/or active primary refractory leukemias or myelodysplastic syndrome underwent peripheral blood stem cell HaploHCT with TMLI, fludarabine, and cyclophosphamide as the conditioning regimen. Radiation dose was escalated in increments of 200 cGy (1200-2000 cGy). GVHD prophylaxis was PTCy with tacrolimus/mycophenolate mofetil. Grade 2 toxicities by the Bearman scale were mucositis (n = 1), hepatic (n = 3), gastrointestinal (n = 5), and cardiac (n = 2). One patient (1800 cGy) experienced grade 3 pulmonary toxicity (dose-limiting toxicity). At a follow-up duration of 23.9 months for the whole cohort; 2-year NRM was 13%. Cumulative incidence of day 100 grade 2 to 4 and 3 to 4 acute GVHD was 52% and 6%, respectively. Chronic GVHD at 2 years was 35%. For patients treated with 2000 cGy, with a median follow-up duration of 12.3 months, 1-year relapse/progression, progression-free survival, and overall survival rates were 17%, 74%, and 83%, respectively. In conclusion, HaploHCT-TMLI with PTCy was safe and feasible in our high-risk patient population with promising outcomes.
Complications associated with removal of airway devices under deep anesthesia in children: an analysis of the Wake Up Safe database
Authors: Vitale Lisa , Rodriguez Briana , Baetzel Anne , Christensen Robert , Haydar Bishr 
Published: July 2022

Abstract
Background
Previous studies examining removal of endotracheal tubes and supraglottic devices under deep anesthesia were underpowered to identify rare complications. This study sought to report all adverse events associated with this practice found in a large national database of pediatric anesthesia adverse events.

Methods
An extract of an adverse events database created by the Wake Up Safe database, a multi-institutional pediatric anesthesia quality improvement initiative, was performed for this study. It was screened to identify anesthetics with variables indicating removal of airway devices under deep anesthesia. Three anesthesiologists screened the data to identify events where this practice possibly contributed to the event. Event data was extracted and collated.

Results
One hundred two events met screening criteria and 66 met inclusion criteria. Two cardiac etiology events were identified, one of which resulted in the patient’s demise. The remaining 97% of events were respiratory in nature (64 events), including airway obstruction, laryngospasm, bronchospasm and aspiration. Some respiratory events consisted of multiple distinct events in series. Nineteen respiratory events resulted in cardiac arrest (29.7%) of which 15 (78.9%) were deemed preventable by local anesthesiologists performing independent review. Respiratory events resulted in intensive care unit admission (37.5%), prolonged intubation and temporary neurologic injury but no permanent harm. Provider and patient factors were root causes in most events. Upon investigation, areas for improvement identified included improving patient selection, ensuring monitoring, availability of intravenous access, and access to emergency drugs and equipment until emergence.

Conclusions
Serious adverse events have been associated with this practice, but no respiratory events were associated with long-term harm.
Study protocol of a phase II study to evaluate safety and efficacy of neo-adjuvant pembrolizumab and radiotherapy in localized rectal cancer
Authors: Corrò Claudia , Buchs Nicolas C. , Tihy Matthieu , Durham-Faivre André , Bichard Philippe , Frossard Jean-Louis , Puppa Giacomo , McKee Thomas , Roth Arnaud , Zilli Thomas , Trembleau Christelle , Di Marco Mariagrazia , Dutoit Valérie , Dietrich Pierre-Yves , Ris Frédéric , Koessler Thibaud 
Published: July 2022

Abstract
Background
Reshaping the tumor microenvironment by novel immunotherapies represents a key strategy to improve cancer treatment. Nevertheless, responsiveness to these treatments is often correlated with the extent of T cell infiltration at the tumor site. Remarkably, microsatellite stable rectal cancer is characterized by poor T cell infiltration and, therefore, does not respond to immune checkpoint blockade. To date, the only available curative option for these patients relies on extensive surgery. With the aim to broaden the application of promising immunotherapies, it is necessary to develop alternative approaches to promote T cell infiltration into the tumor microenvironment of these tumors. In this regard, recent evidence shows that radiotherapy has profound immunostimulatory effects, hinting at the possibility of combining it with immunotherapy. The combination of long-course chemoradiotherapy and immune checkpoint inhibition was recently shown to be safe and yielded promising results in rectal cancer, however short-course radiotherapy and immune checkpoint inhibition have never been tested in these tumors.

Methods
Our clinical trial investigates the clinical and biological impact of combining pembrolizumab with short-course radiotherapy in the neo-adjuvant treatment of localized rectal cancer. This phase II non-randomized study will recruit 25 patients who will receive short-course preoperative radiotherapy (5 Gy × 5 days) and four injections of pembrolizumab starting on the same day and on weeks 4, 7 and 10. Radical surgery will be performed three weeks after the last pembrolizumab injection. Our clinical trial includes an extensive translational research program involving the transcriptomic and proteomic analysis of tumor and blood samples throughout the course of the treatment.

Discussion
Our study is the first clinical trial to combine short-course radiotherapy and immune checkpoint inhibition in rectal cancer, which could potentially result in a major breakthrough in the treatment of this cancer. Additionally, the translational research program will offer insights into immunological changes within the tumor and blood and their correlation with patient outcome. Taken together, our work will help optimizing future treatment combinations and, possibly, better selecting patients.

Trial registration
This study was registered with www.clinicaltrial.gov: NCT04109755. Registration date: June, 2020.
Prognostic impact of pretreatment T790M mutation on outcomes for patients with resected, EGFR-mutated, non-small cell lung cancer
Authors: Matsumoto Yoshiya , Kawaguchi Tomoya , Watanabe Masaru , Isa Shun-ichi , Ando Masahiko , Tamiya Akihiro , Kubo Akihito , Kitagawa Chiyoe , Yoshimoto Naoki , Koh Yasuhiro 
Published: July 2022

Abstract
Background
Many previous studies have demonstrated that minor-frequency pretreatment T790M mutation (preT790M) could be detected by ultrasensitive methods in a considerable number of treatment-naïve, epidermal growth factor receptor (EGFR)-mutated, non-small cell lung cancer (NSCLC) cases. However, the impact of preT790M in resected cases on prognosis remains unclear.

Methods
We previously reported that preT790M could be detected in 298 (79.9%) of 373 surgically resected, EGFR-mutated NSCLC patients. Therefore, we investigated the impact of preT790M on recurrence-free survival (RFS) and overall survival (OS) in this cohort by multivariate analysis. All patients were enrolled from July 2012 to December 2013, with follow-up until November 30, 2017.

Results
The median follow-up time was 48.6 months. Using a cutoff value of the median preT790M allele frequency, the high-preT790M group (n = 151) had significantly shorter RFS (hazard ratio [HR] = 1.51, 95% confidence interval [CI]: 1.01–2.25, P = 0.045) and a tendency for a shorter OS (HR = 1.87, 95% CI: 0.99–3.55, P = 0.055) than the low-preT790M group (n = 222). On multivariate analysis, higher preT790M was independently associated with shorter RFS (high vs low, HR = 1.56, 95% CI: 1.03–2.36, P = 0.035), irrespective of advanced stage, older age, and male sex, and was also associated with shorter OS (high vs low, HR = 2.16, 95% CI: 1.11–4.20, P = 0.024) irrespective of advanced stage, older age, EGFR mutation subtype, and history of adjuvant chemotherapy.

Conclusions
Minor-frequency, especially high-abundance of, preT790M was an independent factor associated with a poor prognosis in patients with surgically resected, EGFR-mutated NSCLC.
Physical activity and healthcare utilization in France: evidence from the European Health Interview Survey (EHIS) 2014
Authors: Jemna Dănuț-Vasile , David Mihaela , Depret Marc-Hubert , Ancelot Lydie 
Published: July 2022

AbstractBackgroundA growing need and focus on preventing and controlling the diseases and promoting a healthier lifestyle is more evident at global, regional, and national levels. In this respect, it is well-known the positive association between physical activity and population’s health, but also its negative association with the demand of healthcare, which could lead to lower spending on healthcare systems. In France, a lack of physical activity, a high prevalence of sedentary behaviours, and a continuous deterioration of these behaviours are observed since 2006. Therefore, promoting and increasing physical activities could contribute to major societal issues. Within this context, the study aims to analyse how the use of different healthcare services are related to physical activity in a nationally representative sample of French population.MethodsThe data used was retrieved from the second wave of the EHIS-ESPS 2014. The relationship between physical activity and healthcare utilization, controlled by a set of socioeconomic, demographic, and health behaviour factors, was explored both at the level of the entire population and separately for two age groups (less than 65 years, 65 years and older), employing probit and recursive multivariate probit models.ResultsOur findings underline that the relation between healthcare utilization and physical activity depends on the type of healthcare services and age group. In this respect, only among adult respondents, we observe a significant negative association between physical activity and prescribed medicines consumption and day hospitalization, while preventive services use is positively related to physical activity. Common to both age groups, the positive association of physical activity with general physician services and non-prescribed medicines reveal that moderately and highly active adults and elders may be more health conscious and therefore may seek referrals to generalist and other prevention measures more frequently than their inactive counterparts. This explanation is also sustained by the negative association between physical activity and overnight hospitalization or home healthcare services.ConclusionsThis study highlights the double role of physical activity on health as preventive measure and treatment and thus support the implementation of public health policies aimed at increasing the level of physical activity in French population.
Study protocol for a hybrid type 1 effectiveness-implementation trial testing virtual tobacco treatment in oncology practices [Smokefree Support Study 2.0]
Authors: Goshe Brett M. , Rasmussen Autumn W. , Wagner Lynne I. , Sicks JoRean D. , Gareen Ilana F. , Carlos Ruth C. , Herman Benjamin A. , Walter Angela Wangari , Regan Susan , Levy Douglas E. , Mahon Irene , Muzikansky Alona , Neil Jordan M. , Lui Michelle , Dilip Deepika , Malloy Laura , Gonzalez Irina , Finkelstein-Fox Lucy , McCann Caitlin , Perez Elissa , Ostroff Jamie S. , Park Elyse R. 
Published: July 2022

AbstractBackgroundPersistent smoking among patients diagnosed with cancer is associated with adverse clinical outcomes, yet an evidence-based tobacco use intervention has not been well-integrated into cancer care in community oncology settings. This paper describes the protocol of a nation-wide clinical trial conducted by the ECOG-ACRIN National Cancer Institute (NCI) Community Oncology Research Program (NCORP) Research Base to assess the effectiveness of a virtual tobacco treatment intervention and the process of implementing tobacco treatment in NCORP community oncology settings.Methods/designThis two-arm, multisite (n: 49 NCORP sites) hybrid type 1 effectiveness-implementation randomized controlled trial compares the effectiveness of a Virtual Intervention Treatment (VIT) versus an Enhanced Usual Control (EUC) among English and Spanish speaking patients recently diagnosed with cancer, reporting current smoking and receiving care at a participating NCORP Community or Minority/Underserved Site. The VIT includes up to 11 virtual counseling sessions with a tobacco treatment specialist and up to 12 weeks of nicotine replacement therapy (NRT). The EUC arm receives a referral to the NCI Quitline. The primary study outcome is biochemically confirmed 7-day point prevalence smoking abstinence. Moderators of treatment effect will be assessed. The study evaluates implementation processes from participating NCORP site staff via survey, administrative, and focus group data, including reach, acceptability, appropriateness, fidelity, feasibility, adoption, cost and sustainability outcomes.DiscussionThis trial will generate findings about the effectiveness of an evidence-based virtual tobacco treatment intervention targeting patients diagnosed with cancer and illuminate barriers and facilitators that influence implementing tobacco treatment into community oncology settings nationally. In the era of COVID-19, virtual care solutions are vital for maximizing access and utilization of tobacco treatment delivery.Trial registrationClinicalTrials.gov (NCT03808818) on January 18th, 2019; Last update posted: May 21st, 2020.
A systematic review of patient-reported outcome measures in paediatric endocrinology
Authors: McGee Richard G. , Zhang Edward Y. B. , Tan Jennifer J. G. , Cheung Aiden C. K. , Garvey Matthew P. 
Published: July 2022

Abstract
Context
Patient reported outcome measures (PROMs) are useful tools in paediatric endocrinology to gauge health status in children, especially since they are often unable to clearly communicate it themselves. We aimed to systematically search and appraise all available PROMs relevant to paediatric endocrinology and provide a curated resource for health professionals to utilise.

Evidence acquisition
We identified PROMs in paediatric endocrinology by systematically searching the Cochrane Library, MEDLINE, World Health Organisation International Clinical Trials Registry Platform, and the Cumulative Index to Nursing and Allied Health Literature on May 20, 2022. Additional studies were located through hand searching and content area expert contributions. We assessed the quality of each PROM using the COSMIN risk of bias checklist.

Evidence synthesis
We identified 5003 papers in the initial search. After applying exclusion criteria we included seven PROMs in the review. Six PROMs were specific to Type I Diabetes and one to Hypothyroidism. We gave all studies an overall COSMIN score of ‘inadequate’ due to poorly detailed PROM development.

Conclusion
The scope and quality of PROMs in paediatric endocrinology is limited. Further research and development of PROM tools are required in paediatric endocrinology to allow for improved patient care.
Evaluation of a training programme for Pharmacist Independent Prescribers in a care home medicine management intervention
Authors: Birt L. , Dalgarno L. , Bond C. M. , Holland R. , Alldred D. P. , Hughes C. , Blyth A. , Watts L. , Wright D. J. ,   on behalf of the CHIPPS team
Published: July 2022

Abstract
Background
The provision of independent prescribing rights for United Kingdom (UK) pharmacists has enabled them to prescribe within their area of competence. The aim of this study was to evaluate an evidence-based training programme designed to prepare Pharmacist Independent Prescribers (PIPs) to safely and effectively assume responsibility for pharmaceutical care of older people in care homes in the UK, within a randomised controlled trial.

Methods
The training and competency assessment process included two training days, professional development planning against a bespoke competency framework, mentor support, and a viva with an independent General Practitioner (GP). Data on the PIPs’ perceptions of the training were collected through evaluation forms immediately after the training days and through online questionnaires and interviews after delivery of the 6-month intervention. Using a mixed method approach each data set was analysed separately then triangulated providing a detailed evaluation of the process. Kaufman’s Model of Learning Evaluation guided interpretations.

Results
All 25 PIPs who received the training completed an evaluation form (N = 25). Post-intervention questionnaires were completed by 16 PIPs and 14 PIPs took part in interviews. PIPs reported the training days and mentorship enabled them to develop a personalised portfolio of competence in preparation for discussion during a viva with an independent GP. Contact with the mentor reduced as PIPs gained confidence in their role. PIPs applied their new learning throughout the delivery of the intervention leading to perceived improvements in residents’ quality of life and medicines management. A few PIPs reported that developing a portfolio of competence was time intensive, and that further training on leadership skills would have been beneficial.

Conclusions
The bespoke training programme was fit for purpose. Mentorship and competency assessment were resource intensive but appropriate. An additional benefit was that many PIPs reported professional growth beyond the requirement of the study.

Trial registration
The definitive RCT was registered with the ISRCTN registry (registration number ISRCTN 17,847,169).
Study of the factors influencing the use of MyData platform based on personal health record data sharing system
Authors: Choi Wona , Chang Se-Hyun , Yang Yoon-Sik , Jung Surin , Lee Seo-Joon , Chun Ji-Won , Kim Dai-Jin , Lee Woonjeong , Choi In Young 
Published: July 2022

Abstract
Background
The application of telemedicine and electronic health (eHealth) technology has grown in importance during the COVID-19 pandemic, and a new approach in personal data management and processing MyData, has emerged. Data portability and informational self-determination are fundamental concepts of MyData. This study analysed the factors that influence acceptance of the MyData platform, which, reflects the right to self-determine personal data.

Methods
The study involved participants having experience using the MyData platform, and the key factors of the unified theory of acceptance and use of technology were used in the research model (performance expectancy, effort expectancy, social influence, facilitation condition and behavioural intention to use). The questionnaire comprided 27 items, and system usage log data were used to confirm that behavioural intention to use affected actual use behaviour through structural equation modeling.

Results
In total, 1153 participants completed the survey. The goodness of fit in the structural equation model indices indicates that the data fit the research model well. Performance expectancy, social influence, and facilitating conditions had direct effects on behavioural intention to use. We used system usage log data to confirm that behavioural intention to use positively affected actual use behaviour. The impact of the main factors in the unified theory of acceptance and use of technology was not moderated by age or gender, except for performance expectancy.

Conclusions
This study is the first to examine the factors influencing the use of the MyData platform based on the personal health record data sharing system in Korea. In addition, the study confirmed the use behaviour of the MyData platform utilising the system’s actual usage log for each function and analysing the effect of the intention of use on actual use. Our study serves as a significant foundation for the acceptance of data portability and sharing concepts. It also lays the foundation for expanding the data economy and ecosystem in the pandemic era.
LZTR1 molecular genetic overlap with clinical implications for Noonan syndrome and schwannomatosis
Authors: Farncombe Kirsten M. , Thain Emily , Barnett-Tapia Carolina , Sadeghian Hamid , Kim Raymond H. 
Published: July 2022

AbstractBackgroundNoonan syndrome (NS) is a genetic disorder characterized by developmental delays, typical facial gestalt and cardiovascular defects.LZTR1variants have been recently described in patients with NS and schwannomatosis, but the association, inheritance pattern and management strategy has not been fully elucidated. Here, we review the contribution ofLZTR1in NS and describe a patient with a novel, likely pathogenic variant inLZTR1.Case presentationA female patient was diagnosed with clinical NS at 8 months of age. She presented in adulthood when a brain and spine MRI identified plexiform neurofibromas; however, she did not meet the clinical criteria for Neurofibromatosis type 1. No pathogenic variants were identified through molecular genetic analysis ofNF1,SPRED1and a multigene NS panel. Whole exome sequencing at age 23 identified a novel de novo likely pathogenic heterozygous variant in theLZTR1gene denoted as c.743G>A (p.Gly248Glu). Serial MRIs have shown stable imaging findings and the patient is being followed clinically by cardiology, neurology and medical genetics.ConclusionsWe identified a novel mutation in theLZTR1gene, not previously reported in association with NS. This report provides additional evidence to support for the assessment of schwannomatosis in patients withLZTR1-NS and may have overlap with Neurofibromatosis type 1.