Pub Date : 2019-05-01DOI: 10.1164/ajrccm-conference.2019.199.1_meetingabstracts.a1307
L. Evitt, R. Follows, J. Bentley, W. Williams, H. Shalhoub, M. Celone, R. Maltzahn
{"title":"Mepolizumab Prefilled Autoinjector and Prefilled Syringe Real World Use: The Patient Experience","authors":"L. Evitt, R. Follows, J. Bentley, W. Williams, H. Shalhoub, M. Celone, R. Maltzahn","doi":"10.1164/ajrccm-conference.2019.199.1_meetingabstracts.a1307","DOIUrl":"https://doi.org/10.1164/ajrccm-conference.2019.199.1_meetingabstracts.a1307","url":null,"abstract":"","PeriodicalId":7013,"journal":{"name":"A32. ASTHMA: CLINICAL STUDIES II","volume":"252 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2019-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"76779331","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2019-05-01DOI: 10.1164/ajrccm-conference.2019.199.1_meetingabstracts.a1333
B. Kuti, D. Kuti, O. S. Smith
{"title":"Relationship Between Serum Inflammatory Cytokines and 25-Hydroxy Vitamin D in Nigerian Children with Asthma","authors":"B. Kuti, D. Kuti, O. S. Smith","doi":"10.1164/ajrccm-conference.2019.199.1_meetingabstracts.a1333","DOIUrl":"https://doi.org/10.1164/ajrccm-conference.2019.199.1_meetingabstracts.a1333","url":null,"abstract":"","PeriodicalId":7013,"journal":{"name":"A32. ASTHMA: CLINICAL STUDIES II","volume":"34 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2019-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"74479561","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2019-05-01DOI: 10.1164/ajrccm-conference.2019.199.1_meetingabstracts.a1319
C. Cappelletti, A. Maes, K. Rossman, E. Kerwin, C. Reisner
{"title":"A Dose Ranging Study of Albuterol Sulfate MDI in Co-Suspension Delivery Technology (AS MDI; PT007) in Patients with Asthma","authors":"C. Cappelletti, A. Maes, K. Rossman, E. Kerwin, C. Reisner","doi":"10.1164/ajrccm-conference.2019.199.1_meetingabstracts.a1319","DOIUrl":"https://doi.org/10.1164/ajrccm-conference.2019.199.1_meetingabstracts.a1319","url":null,"abstract":"","PeriodicalId":7013,"journal":{"name":"A32. ASTHMA: CLINICAL STUDIES II","volume":"49 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2019-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"82338482","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2018-09-01DOI: 10.1136/JNNP-2018-EHDN.131
S. Mason, Wei-Li Kuan, M. Baddeley, R. Barker
Advances in technology have changed the way in which we spend and manage our money. For most people this has been a liberating development but for the thousands of people living with dementia this poses serious challenges to their ability to live independently and puts them at increased risk of financial abuse. A loss of financial autonomy and monetary mismanagement is a characteristic feature of Huntington’s disease (HD), however, it is currently unclear what drives these problems. We know that patients experience disruption to the functional integrity of the frontostriatal circuitry leading to a characteristic dysexecutive syndrome that can be accompanied by behavioral problems such as impulsivity, poor risk assessment and emotional changes. There is also evidence that they can struggle to interact socially, but the extent to which these problems relate to their financial vulnerability is unknown. In this study we adopted both theory and methodology from the field of social economics to help provide an insight into the reasons why patients with HD are vulnerable to financial abuse. The results indicate that patient’s generosity on social economics tests such as the Dictator, Ultimatum, Trust and Public Goods Games increases with advancing HD whilst their ability to adjust their risk taking behavior in the context of relevant information deteriorates. Furthermore, performance strongly correlated with their ability to accurately interpret the feelings and beliefs of their confederates. This suggests that both an increased tendency to make risky decisions and difficulty identifying spurious financial offers or disingenuous people may be important factors that promote HD patient’s susceptibility to financial abuse.
{"title":"F27 Factors that influence financial vulnerability in huntington’s disease","authors":"S. Mason, Wei-Li Kuan, M. Baddeley, R. Barker","doi":"10.1136/JNNP-2018-EHDN.131","DOIUrl":"https://doi.org/10.1136/JNNP-2018-EHDN.131","url":null,"abstract":"Advances in technology have changed the way in which we spend and manage our money. For most people this has been a liberating development but for the thousands of people living with dementia this poses serious challenges to their ability to live independently and puts them at increased risk of financial abuse. A loss of financial autonomy and monetary mismanagement is a characteristic feature of Huntington’s disease (HD), however, it is currently unclear what drives these problems. We know that patients experience disruption to the functional integrity of the frontostriatal circuitry leading to a characteristic dysexecutive syndrome that can be accompanied by behavioral problems such as impulsivity, poor risk assessment and emotional changes. There is also evidence that they can struggle to interact socially, but the extent to which these problems relate to their financial vulnerability is unknown. In this study we adopted both theory and methodology from the field of social economics to help provide an insight into the reasons why patients with HD are vulnerable to financial abuse. The results indicate that patient’s generosity on social economics tests such as the Dictator, Ultimatum, Trust and Public Goods Games increases with advancing HD whilst their ability to adjust their risk taking behavior in the context of relevant information deteriorates. Furthermore, performance strongly correlated with their ability to accurately interpret the feelings and beliefs of their confederates. This suggests that both an increased tendency to make risky decisions and difficulty identifying spurious financial offers or disingenuous people may be important factors that promote HD patient’s susceptibility to financial abuse.","PeriodicalId":7013,"journal":{"name":"A32. ASTHMA: CLINICAL STUDIES II","volume":"48 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2018-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"84791118","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2018-09-01DOI: 10.1136/JNNP-2018-EHDN.170
T. McLean
Enroll-HD is a global research platform. Key elements of the platform’s infrastructure include global study management and governance, standardised informed consent forms (ICFs) and site contracts, uniform clinical site training, an integrated EDC and study database and a user-friendly comprehensive webportal. This infrastructure supports the Enroll-HD study, a prospective, observational, longitudinal registry study of HD, currently with >17,000 participants who have performed standardised clinical assessments and biosample collections from annual visits at over 160 clinical sites in 17 countries. Resources and services of the platform that are made available to the HD research community include easily accessible periodic clinical datasets and associated biosamples. Consequent research output may be used to support future clinical studies, including protocol design. A team of subject matter experts (SMEs) with experience in activities such as ICF development and translation, site agreements and financial payments, insurance, assessment scale licencing, IRB/ethics submissions, site staff training and certification via the platform training portal, database construction, and data monitoring is available to assist in the implementation of clinical studies. Long term working relationships with the participating clinical sites allow for well-informed clinical trial site selection and efficient feasibility as well as ongoing support for issue resolution and trial facilitation. The Enroll-HD platform’s substantial registry of participants allows for powerful in-silico screening to support participant recruitment. A toolkit of platform resources, procedures and template documents is in preparation. The provision of platform services, including the SME support, is managed by a team of Project Resource and Service Managers (PRSM Team). This team also facilitates the interface between clinical study and trial project teams or researchers and the Enroll-HD Platform.
{"title":"F70 Enroll-hd platform services","authors":"T. McLean","doi":"10.1136/JNNP-2018-EHDN.170","DOIUrl":"https://doi.org/10.1136/JNNP-2018-EHDN.170","url":null,"abstract":"Enroll-HD is a global research platform. Key elements of the platform’s infrastructure include global study management and governance, standardised informed consent forms (ICFs) and site contracts, uniform clinical site training, an integrated EDC and study database and a user-friendly comprehensive webportal. This infrastructure supports the Enroll-HD study, a prospective, observational, longitudinal registry study of HD, currently with >17,000 participants who have performed standardised clinical assessments and biosample collections from annual visits at over 160 clinical sites in 17 countries. Resources and services of the platform that are made available to the HD research community include easily accessible periodic clinical datasets and associated biosamples. Consequent research output may be used to support future clinical studies, including protocol design. A team of subject matter experts (SMEs) with experience in activities such as ICF development and translation, site agreements and financial payments, insurance, assessment scale licencing, IRB/ethics submissions, site staff training and certification via the platform training portal, database construction, and data monitoring is available to assist in the implementation of clinical studies. Long term working relationships with the participating clinical sites allow for well-informed clinical trial site selection and efficient feasibility as well as ongoing support for issue resolution and trial facilitation. The Enroll-HD platform’s substantial registry of participants allows for powerful in-silico screening to support participant recruitment. A toolkit of platform resources, procedures and template documents is in preparation. The provision of platform services, including the SME support, is managed by a team of Project Resource and Service Managers (PRSM Team). This team also facilitates the interface between clinical study and trial project teams or researchers and the Enroll-HD Platform.","PeriodicalId":7013,"journal":{"name":"A32. ASTHMA: CLINICAL STUDIES II","volume":"6 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2018-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"89840391","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2018-09-01DOI: 10.1136/JNNP-2018-EHDN.160
Paul Zeun, J. Lowe, K. Osborne-Crowley, C. O’Callaghan, E. Johnson, S. Gregory, A. Nair, Kate Fayer, F. B. Rodrigues, C. Estévez-Fraga, E. Wild, Gary Zhang, C. Sampaio, T. Robbins, G. Rees, R. Scahill, B. Sahakian, S. Tabrizi
HD-YAS will study a cohort of young adult HD Gene Expansion Carriers (HDGEC) decades before expected symptom onset to characterize the very earliest signs of disease-related brain changes and identify whether there is any identifiable early functional impairment. Currently there is no detailed characterization of such a young adult HDGEC cohort and this represents the earliest time point after predictive genetic testing in which to gain disease insights. HD-YAS will be important in determining the earliest potential time window for therapeutic intervention. HD-YAS will recruit 120 participants-60 premanifest HDGECs and 60 gene negative or family controls-and will use a cross-sectional comparison with one visit per participant to assess the earliest time-point at which neurodegeneration can be detected. Participants will undergo 3T Volumetric MRI, rsfMRI and task fMRI, NODDI, and cognitive assessments including the CANTAB and EMOTICOM batteries, and biosamples such as CSF, blood and DNA will be collected to investigate biomarkers. Participants will be 18–40 years old and at-risk individuals must have a predictive genetic test; either carry the HD gene (gene-carrier) or not carry the HD gene (control). Individuals must not show any clinical symptoms of HD, have a disease burden score of ≤240, and must be willing and able to comply with the study visit and study procedures. HD-YAS began in August 2017 and is expected to complete by February 2019. HD-YAS collaborates with Enroll-HD, UK clinical genetics services and HD charity groups to identify potentially eligible participants throughout the UK. HD-YAS is funded by Wellcome, with CSF collection funded by CHDI. HD-YAS investigators – Participant Identification Site Investigators; Dr Oliver Quarrell, Dr Nayana Lahiri, Dr Andrea Nemeth, Dr Mary Porteous, Dr Elisabeth Rosser, Dr David Craufurd, Dr Rhona MacLeod, Dr Deborah Ruddy, Dr Roger Barker, Dr Simon Holden, Dr Hugh Rickards, Dr Anne Rosser, Dr Emma Hobson, Prof Angus Clarke, Dr Katherine Lachlan, Dr Reza Kiani, Dr Timothy Harrower. Thank you to the HDA, HDYO, SHA.
{"title":"F59 Huntington’s disease young adult study (HD-YAS)","authors":"Paul Zeun, J. Lowe, K. Osborne-Crowley, C. O’Callaghan, E. Johnson, S. Gregory, A. Nair, Kate Fayer, F. B. Rodrigues, C. Estévez-Fraga, E. Wild, Gary Zhang, C. Sampaio, T. Robbins, G. Rees, R. Scahill, B. Sahakian, S. Tabrizi","doi":"10.1136/JNNP-2018-EHDN.160","DOIUrl":"https://doi.org/10.1136/JNNP-2018-EHDN.160","url":null,"abstract":"HD-YAS will study a cohort of young adult HD Gene Expansion Carriers (HDGEC) decades before expected symptom onset to characterize the very earliest signs of disease-related brain changes and identify whether there is any identifiable early functional impairment. Currently there is no detailed characterization of such a young adult HDGEC cohort and this represents the earliest time point after predictive genetic testing in which to gain disease insights. HD-YAS will be important in determining the earliest potential time window for therapeutic intervention. HD-YAS will recruit 120 participants-60 premanifest HDGECs and 60 gene negative or family controls-and will use a cross-sectional comparison with one visit per participant to assess the earliest time-point at which neurodegeneration can be detected. Participants will undergo 3T Volumetric MRI, rsfMRI and task fMRI, NODDI, and cognitive assessments including the CANTAB and EMOTICOM batteries, and biosamples such as CSF, blood and DNA will be collected to investigate biomarkers. Participants will be 18–40 years old and at-risk individuals must have a predictive genetic test; either carry the HD gene (gene-carrier) or not carry the HD gene (control). Individuals must not show any clinical symptoms of HD, have a disease burden score of ≤240, and must be willing and able to comply with the study visit and study procedures. HD-YAS began in August 2017 and is expected to complete by February 2019. HD-YAS collaborates with Enroll-HD, UK clinical genetics services and HD charity groups to identify potentially eligible participants throughout the UK. HD-YAS is funded by Wellcome, with CSF collection funded by CHDI. HD-YAS investigators – Participant Identification Site Investigators; Dr Oliver Quarrell, Dr Nayana Lahiri, Dr Andrea Nemeth, Dr Mary Porteous, Dr Elisabeth Rosser, Dr David Craufurd, Dr Rhona MacLeod, Dr Deborah Ruddy, Dr Roger Barker, Dr Simon Holden, Dr Hugh Rickards, Dr Anne Rosser, Dr Emma Hobson, Prof Angus Clarke, Dr Katherine Lachlan, Dr Reza Kiani, Dr Timothy Harrower. Thank you to the HDA, HDYO, SHA.","PeriodicalId":7013,"journal":{"name":"A32. ASTHMA: CLINICAL STUDIES II","volume":"74 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2018-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"74560605","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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