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  1. AU="Michael Pugliese"
  2. AU="Amador-Sánchez, Yoarhy A"
  3. AU="Lanbo Shi"
  4. AU="Gregg, R J"
  5. AU="Zou, Peiyuan"
  6. AU="Sasha Stevenson"
  7. AU="Boncompagni, Alessandra"
  8. AU="Lewis, Annisa L"
  9. AU="Daniel Freilich"
  10. AU="Glascock, Abigail L"
  11. AU="Gordon Bernard"
  12. AU="Lv, Mengwen"
  13. AU="Rottman Pietrzak, Kathleen A"
  14. AU=Panczak Radoslaw
  15. AU="Hosseini, Seyed Mohammad Hadi"
  16. AU="Noda, Haruna"
  17. AU="Raoul, Cédric"
  18. AU=Wissing Silke AU=Wissing Silke
  19. AU="Chun-Lin Yang"
  20. AU="Romine, Kyle A"
  21. AU="Cunsolo, Vincenzo"
  22. AU="Ba, Aboubacar"
  23. AU="Prisca, Mirandolina"
  24. AU="Perez, Tate"
  25. AU="Bakkaloglu, Sevan"
  26. AU="Guernieri, Rebecca L"
  27. AU="Xing, Z Y"
  28. AU="Yu-Heng Cheng"
  29. AU=Freeman Richard B Jr
  30. AU="Wang, Qi-En"
  31. AU="Mallamaci, M"
  32. AU="Turk, Yael R"
  33. AU="Tinto, Monica"
  34. AU="Selvendiran, Karuppaiyah" AU="Selvendiran, Karuppaiyah"
  35. AU="Enns, Murray W"
  36. AU="Yaohua Yang" AU="Yaohua Yang"

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  1. Artikel ; Online: Physician benzodiazepine self-use prior to and during the COVID-19 pandemic in Ontario, Canada

    Peter Tanuseputro / Tetyana Kendzerska / Claire E Kendall / Michael Pugliese / Daniel Myran / Christina Milani / Jennifer M Hensel / Manish Sood

    BMJ Open, Vol 13, Iss

    a population-level cohort study

    2023  Band 4

    Abstract: Objectives The aim of this study was to investigate physician benzodiazepine (BZD) self-use pre-COVID-19 pandemic and to examine changes in BZD self-use during the first year of the pandemic.Design Population-based retrospective cohort study using linked ...

    Abstract Objectives The aim of this study was to investigate physician benzodiazepine (BZD) self-use pre-COVID-19 pandemic and to examine changes in BZD self-use during the first year of the pandemic.Design Population-based retrospective cohort study using linked routinely collected administrative health data comparing the first year of the pandemic to the period before the pandemic.Setting Province of Ontario, Canada between March 2016 and March 2021.Participants Intervention Onset of the COVID-19 pandemic in March 2020.Outcomes measures The primary outcome measure was the receipt of one or more prescriptions for BZD, which was captured via the Narcotics Monitoring System.Results In a cohort of 30 798 physicians (mean age 42, 47.8% women), we found that during the year before the pandemic, 4.4% of physicians had 1 or more BZD prescriptions. Older physicians (6.8% aged 50+ years), female physicians (5.1%) and physicians with a prior mental health (MH) diagnosis (12.4%) were more likely than younger (3.7% aged <50 years), male physicians (3.8%) and physicians without a prior MH diagnosis (2.9%) to have received 1 or more BZD prescriptions. The first year of the COVID-19 pandemic was associated with a 10.5% decrease (adjusted OR (aOR) 0.85, 95% CI: 0.80 to 0.91) in the number of physicians with 1 or more BZD prescriptions compared with the year before the pandemic. Female physicians were less likely to reduce BZD self-use (aORfemale=0.90, 95% CI: 0.83 to 0.98) compared with male physicians (aORmale=0.79, 95% CI: 0.72 to 0.87, pinteraction=0.046 during the pandemic. Physicians presenting with an incident MH visit had higher odds of filling a BZD prescription during COVID-19 compared with the prior year.Conclusions Physicians’ BZD prescriptions decreased during the first year of the COVID-19 pandemic in Ontario, Canada. These findings suggest that previously reported increases in mental distress and MH visits among physicians during the pandemic did not lead to greater self-use of BZDs.
    Schlagwörter Medicine ; R
    Thema/Rubrik (Code) 900
    Sprache Englisch
    Erscheinungsdatum 2023-04-01T00:00:00Z
    Verlag BMJ Publishing Group
    Dokumenttyp Artikel ; Online
    Datenquelle BASE - Bielefeld Academic Search Engine (Lebenswissenschaftliche Auswahl)

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  2. Artikel ; Online: Mobile tablet-based therapies following stroke

    Michael Pugliese / Tim Ramsay / Dylan Johnson / Dar Dowlatshahi

    PLoS ONE, Vol 13, Iss 1, p e

    A systematic scoping review of administrative methods and patient experiences.

    2018  Band 0191566

    Abstract: Stroke survivors are often left with deficits requiring rehabilitation to recover function and yet, many are unable to access rehabilitative therapies. Mobile tablet-based therapies (MTBTs) may be a resource-efficient means of improving access to timely ... ...

    Abstract Stroke survivors are often left with deficits requiring rehabilitation to recover function and yet, many are unable to access rehabilitative therapies. Mobile tablet-based therapies (MTBTs) may be a resource-efficient means of improving access to timely rehabilitation. It is unclear what MTBTs have been attempted following stroke, how they were administered, and how patients experienced the therapies. The review summarizes studies of MTBTs following stroke in terms of administrative methods and patient experiences to inform treatment feasibility.Articles were eligible if they reported the results of an MTBT attempted with stroke participants. Six research databases were searched along with grey literature sources, trial registries, and article references. Intervention administration details and patient experiences were summarized.The search returned 903 articles of which 23 were eligible for inclusion. Most studies were small, observational, and enrolled chronic stroke patients. Interventions commonly targeted communication, cognition, or fine-motor skills. Therapies tended to be personalized based on patient deficits using commercially available applications. The complexity of therapy instructions, fine-motor requirements, and unreliability of internet or cellular connections were identified as common barriers to tablet-based care.Stroke patients responded positively to MTBTs in both the inpatient and home settings. However, some support from therapists or caregivers may be required for patients to overcome barriers to care. Feasibility studies should continue to identify the administrative methods that minimize barriers to care and maximize patient adherence to prescribed therapy regiments.
    Schlagwörter Medicine ; R ; Science ; Q
    Thema/Rubrik (Code) 616
    Sprache Englisch
    Erscheinungsdatum 2018-01-01T00:00:00Z
    Verlag Public Library of Science (PLoS)
    Dokumenttyp Artikel ; Online
    Datenquelle BASE - Bielefeld Academic Search Engine (Lebenswissenschaftliche Auswahl)

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  3. Artikel ; Online: Mobile tablet-based therapies following stroke

    Michael Pugliese / Dylan Johnson / Dar Dowlatshahi / Tim Ramsay

    Systematic Reviews, Vol 6, Iss 1, Pp 1-

    a systematic scoping review protocol of attempted interventions and the challenges encountered

    2017  Band 7

    Abstract: Abstract Background Stroke is a growing global epidemic limiting the ability of millions to function independently due to post-stroke deficits and complications. Although specialized stroke rehabilitation improves the recovery of functional abilities, ... ...

    Abstract Abstract Background Stroke is a growing global epidemic limiting the ability of millions to function independently due to post-stroke deficits and complications. Although specialized stroke rehabilitation improves the recovery of functional abilities, accessing rehabilitation services has become increasingly challenging as the number of stroke survivors continues to increase and rehabilitation resources remain scarce. Mobile tablet-based therapies (MTBTs) may be a resource-efficient platform for providing stroke rehabilitation services. The feasibility and challenges of offering MTBTs to stroke survivors should be well understood before expensive, large-scale clinical trials are undertaken to study treatment efficacy. Method A systematic scoping review will be conducted to describe attempted MTBTs following stroke and the challenges encountered by survivors and study staff. Studies of interest will evaluate MTBTs offered to adult stroke patients in response to post-stroke complications or deficits. Journal databases, gray literature sources, clinical trial registries, relevant organizational websites, and reference lists of eligible studies will be searched to identify suitable studies. Study characteristics, barriers to care, methodological challenges, patient-reported outcomes, and health outcomes will be extracted to describe MTBTs and understand the challenges encountered in context. Results will be presented using descriptive statistics, tables, figures, and narrative description to summarize the scope of the field. Discussion Trends in MTBT feasibility and common challenges will be discussed to summarize major findings and highlight research gaps. Solutions to common challenges experienced by intervention participants and study staff will be proposed. Implications for the conduct of randomized clinical trials of MTBT efficacy and the appropriateness of a systematic review and meta-analysis of completed trials will be discussed. Systematic review registration uO Research ( http://hdl.handle.net/10393/35696 ...
    Schlagwörter Stroke rehabilitation ; mHealth ; iPad ; Tablet computer ; CVA ; Disability ; Medicine ; R
    Thema/Rubrik (Code) 306
    Sprache Englisch
    Erscheinungsdatum 2017-11-01T00:00:00Z
    Verlag BMC
    Dokumenttyp Artikel ; Online
    Datenquelle BASE - Bielefeld Academic Search Engine (Lebenswissenschaftliche Auswahl)

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  4. Artikel ; Online: Trends in outpatient and inpatient visits for separate ambulatory-care-sensitive conditions during the first year of the COVID-19 pandemic

    Tetyana Kendzerska / David T. Zhu / Michael Pugliese / Douglas Manuel / Mohsen Sadatsafavi / Marcus Povitz / Therese A. Stukel / Teresa To / Shawn D. Aaron / Sunita Mulpuru / Melanie Chin / Claire E. Kendall / Kednapa Thavorn / Rebecca Robillard / Andrea S. Gershon

    Frontiers in Public Health, Vol

    a province-based study

    2023  Band 11

    Abstract: BackgroundThe COVID-19 pandemic led to global disruptions in non-urgent health services, affecting health outcomes of individuals with ambulatory-care-sensitive conditions (ACSCs).MethodsWe conducted a province-based study using Ontario health ... ...

    Abstract BackgroundThe COVID-19 pandemic led to global disruptions in non-urgent health services, affecting health outcomes of individuals with ambulatory-care-sensitive conditions (ACSCs).MethodsWe conducted a province-based study using Ontario health administrative data (Canada) to determine trends in outpatient visits and hospitalization rates (per 100,000 people) in the general adult population for seven ACSCs during the first pandemic year (March 2020–March 2021) compared to previous years (2016–2019), and how disruption in outpatient visits related to acute care use. ACSCs considered were chronic obstructive pulmonary disease (COPD), asthma, angina, congestive heart failure (CHF), hypertension, diabetes, and epilepsy. We used time series auto-regressive integrated moving-average models to compare observed versus projected rates.ResultsFollowing an initial reduction (March–May 2020) in all types of visits, primary care outpatient visits (combined in-person and virtual) returned to pre-pandemic levels for asthma, angina, hypertension, and diabetes, remained below pre-pandemic levels for COPD, and rose above pre-pandemic levels for CHF (104.8 vs. 96.4, 95% CI: 89.4–104.0) and epilepsy (29.6 vs. 24.7, 95% CI: 22.1–27.5) by the end of the first pandemic year. Specialty visits returned to pre-pandemic levels for COPD, angina, CHF, hypertension, and diabetes, but remained above pre-pandemic levels for asthma (95.4 vs. 79.5, 95% CI: 70.7–89.5) and epilepsy (53.3 vs. 45.6, 95% CI: 41.2–50.5), by the end of the year. Virtual visit rates increased for all ACSCs. Among ACSCs, reductions in hospitalizations were most pronounced for COPD and asthma. CHF-related hospitalizations also decreased, albeit to a lesser extent. For angina, hypertension, diabetes, and epilepsy, hospitalization rates reduced initially, but returned to pre-pandemic levels by the end of the year.ConclusionThis study demonstrated variation in outpatient visit trends for different ACSCs in the first pandemic year. No outpatient visit trends resulted in ...
    Schlagwörter COVID-19 pandemic ; ambulatory care sensitive conditions ; outpatient visits ; inpatient visits ; ARIMA ; trends ; Public aspects of medicine ; RA1-1270
    Thema/Rubrik (Code) 333
    Sprache Englisch
    Erscheinungsdatum 2023-12-01T00:00:00Z
    Verlag Frontiers Media S.A.
    Dokumenttyp Artikel ; Online
    Datenquelle BASE - Bielefeld Academic Search Engine (Lebenswissenschaftliche Auswahl)

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  5. Artikel ; Online: Patient and family engagement in the development of core outcome sets for two rare chronic diseases in children

    Shelley M. Vanderhout / Maureen Smith / Nicole Pallone / Kylie Tingley / Michael Pugliese / Pranesh Chakraborty / Sylvia Stockler / Martin Offringa / Nancy Butcher / Stuart G. Nicholls / Beth K. Potter

    Research Involvement and Engagement, Vol 7, Iss 1, Pp 1-

    2021  Band 8

    Abstract: Plain English summary Core outcome sets (COS) are lists of outcomes agreed upon by a group of people to be measured and reported in studies about certain diseases and populations. Core outcomes are meant to represent what is useful to study from the ... ...

    Abstract Plain English summary Core outcome sets (COS) are lists of outcomes agreed upon by a group of people to be measured and reported in studies about certain diseases and populations. Core outcomes are meant to represent what is useful to study from the perspectives of health care providers, researchers and patients. For researchers who seek to include patients in the development of a COS, there is little guidance about how to do this well. We recently developed COS for two rare diseases in children, medium-chain acyl-CoA dehydrogenase deficiency and phenylketonuria. We did this by reviewing available information from published research, surveying health care providers, researchers, and patients, and eventually coming to agreement during a workshop. We included two adult patient partner co-researchers who helped design the COS study and co-developed the patient engagement strategy. These partners formed relationships with seven adult family advisors, who helped ensure that materials were accessible, participated in outcome selection, and helped select tools to measure core outcomes. Strategies we used to engage patient partners included a) training about both the scientific research process and how to help other researchers in the future, and b) frequent communication about study progress and how family advisor feedback was used. Also, we made sure that the impacts of power imbalances between health care providers, researchers and patients were low. Our approach to patient engagement in COS development for two rare conditions in children proved to be both feasible and considered valuable by all study team members, including patient partners and family advisors. To include patient perspectives and values, future COS developers may take a similar approach.
    Schlagwörter Core outcome sets ; Medium-chain acyl-CoA dehydrogenase deficiency ; Phenylketonuria ; Patient engagement ; Consensus ; Medicine ; R ; Medicine (General) ; R5-920
    Thema/Rubrik (Code) 360
    Sprache Englisch
    Erscheinungsdatum 2021-09-01T00:00:00Z
    Verlag BMC
    Dokumenttyp Artikel ; Online
    Datenquelle BASE - Bielefeld Academic Search Engine (Lebenswissenschaftliche Auswahl)

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  6. Artikel ; Online: RecoverNow

    Michael Pugliese / Tim Ramsay / Rany Shamloul / Karen Mallet / Lise Zakutney / Dale Corbett / Sean Dukelow / Grant Stotts / Michel Shamy / Kumanan Wilson / Julien Guerinet / Dar Dowlatshahi

    PLoS ONE, Vol 14, Iss 1, p e

    A mobile tablet-based therapy platform for early stroke rehabilitation.

    2019  Band 0210725

    Abstract: INTRODUCTION:Stroke survivors frequently experience a range of post-stroke deficits. Specialized stroke rehabilitation improves recovery, especially if it is started early post-stroke. However, resource limitations often preclude early rehabilitation. ... ...

    Abstract INTRODUCTION:Stroke survivors frequently experience a range of post-stroke deficits. Specialized stroke rehabilitation improves recovery, especially if it is started early post-stroke. However, resource limitations often preclude early rehabilitation. Mobile technologies may provide a platform for stroke survivors to begin recovery when they might not be able to otherwise. The study objective was to demonstrate the feasibility of RecoverNow, a tablet-based stroke recovery platform aimed at delivering speech and cognitive therapy. METHODS:We recruited a convenience sample of 30 acute stroke patients to use RecoverNow for up to 3 months. Allied health professionals assigned specific applications based on standard of care assessments. Participants were encouraged to take home the RecoverNow tablets upon discharge from acute care. The study team contacted participants to return for a follow-up interview 3 months after enrollment. The primary outcome of interest was feasibility, defined using 5 facets: recruitment rate, adherence rate, retention rate, the proportion of successful follow-up interventions, and protocol deviations. We tracked barriers to tablet-based care as a secondary outcome. RESULTS:We successfully recruited 30 of 62 eligible patients in 15 weeks (48% recruitment rate). Participants were non-adherent to tablet-based therapy inside and outside of acute care, using RecoverNow for a median of 12 minutes a day. Retention was high with 23 of 30 patients participating in follow-up interviews (77% retention rate) and all but 3 of the 23 interviews (87%) were successfully completed. Only 2 major protocol deviations occurred: one enrollment failure and one therapy protocol violation. Barriers to tablet-based care were frequently encountered by study participants with many expressing the assigned applications were either too easy or too difficult. CONCLUSIONS:Acute stroke patients are interested in attempting tablet-based stroke rehabilitation and are easily recruited early post-stroke. However, tablet-based ...
    Schlagwörter Medicine ; R ; Science ; Q
    Thema/Rubrik (Code) 150
    Sprache Englisch
    Erscheinungsdatum 2019-01-01T00:00:00Z
    Verlag Public Library of Science (PLoS)
    Dokumenttyp Artikel ; Online
    Datenquelle BASE - Bielefeld Academic Search Engine (Lebenswissenschaftliche Auswahl)

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  7. Artikel ; Online: Evaluation of the quality of clinical data collection for a pan-Canadian cohort of children affected by inherited metabolic diseases

    Kylie Tingley / Monica Lamoureux / Michael Pugliese / Michael T. Geraghty / Jonathan B. Kronick / Beth K. Potter / Doug Coyle / Kumanan Wilson / Michael Kowalski / Valerie Austin / Catherine Brunel-Guitton / Daniela Buhas / Alicia K. J. Chan / Sarah Dyack / Annette Feigenbaum / Alette Giezen / Sharan Goobie / Cheryl R. Greenberg / Shailly Jain Ghai /
    Michal Inbar-Feigenberg / Natalya Karp / Mariya Kozenko / Erica Langley / Matthew Lines / Julian Little / Jennifer MacKenzie / Bruno Maranda / Saadet Mercimek-Andrews / Connie Mohan / Aizeddin Mhanni / Grant Mitchell / John J. Mitchell / Laura Nagy / Melanie Napier / Amy Pender / Murray Potter / Chitra Prasad / Suzanne Ratko / Ramona Salvarinova / Andreas Schulze / Komudi Siriwardena / Neal Sondheimer / Rebecca Sparkes / Sylvia Stockler-Ipsiroglu / Yannis Trakadis / Lesley Turner / Clara Van Karnebeek / Hilary Vallance / Anthony Vandersteen / Jagdeep Walia

    Orphanet Journal of Rare Diseases, Vol 15, Iss 1, Pp 1-

    lessons learned from the Canadian Inherited Metabolic Diseases Research Network

    2020  Band 12

    Abstract: Abstract Background The Canadian Inherited Metabolic Diseases Research Network (CIMDRN) is a pan-Canadian practice-based research network of 14 Hereditary Metabolic Disease Treatment Centres and over 50 investigators. CIMDRN aims to develop evidence to ... ...

    Abstract Abstract Background The Canadian Inherited Metabolic Diseases Research Network (CIMDRN) is a pan-Canadian practice-based research network of 14 Hereditary Metabolic Disease Treatment Centres and over 50 investigators. CIMDRN aims to develop evidence to improve health outcomes for children with inherited metabolic diseases (IMD). We describe the development of our clinical data collection platform, discuss our data quality management plan, and present the findings to date from our data quality assessment, highlighting key lessons that can serve as a resource for future clinical research initiatives relating to rare diseases. Methods At participating centres, children born from 2006 to 2015 who were diagnosed with one of 31 targeted IMD were eligible to participate in CIMDRN’s clinical research stream. For all participants, we collected a minimum data set that includes information about demographics and diagnosis. For children with five prioritized IMD, we collected longitudinal data including interventions, clinical outcomes, and indicators of disease management. The data quality management plan included: design of user-friendly and intuitive clinical data collection forms; validation measures at point of data entry, designed to minimize data entry errors; regular communications with each CIMDRN site; and routine review of aggregate data. Results As of June 2019, CIMDRN has enrolled 798 participants of whom 764 (96%) have complete minimum data set information. Results from our data quality assessment revealed that potential data quality issues were related to interpretation of definitions of some variables, participants who transferred care across institutions, and the organization of information within the patient charts (e.g., neuropsychological test results). Little information was missing regarding disease ascertainment and diagnosis (e.g., ascertainment method – 0% missing). Discussion Using several data quality management strategies, we have established a comprehensive clinical database that provides ...
    Schlagwörter Inherited metabolic diseases ; Observational research ; Registry science ; Data quality ; Database ; Sustainability ; Medicine ; R
    Thema/Rubrik (Code) 310
    Sprache Englisch
    Erscheinungsdatum 2020-04-01T00:00:00Z
    Verlag BMC
    Dokumenttyp Artikel ; Online
    Datenquelle BASE - Bielefeld Academic Search Engine (Lebenswissenschaftliche Auswahl)

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  8. Artikel ; Online: Outcomes in pediatric studies of medium-chain acyl-coA dehydrogenase (MCAD) deficiency and phenylketonuria (PKU)

    Michael Pugliese / Kylie Tingley / Andrea Chow / Nicole Pallone / Maureen Smith / Alvi Rahman / Pranesh Chakraborty / Michael T. Geraghty / Julie Irwin / Laure Tessier / Stuart G. Nicholls / Martin Offringa / Nancy J. Butcher / Ryan Iverson / Tammy J. Clifford / Sylvia Stockler / Brian Hutton / Karen Paik / Jessica Tao /
    Becky Skidmore / Doug Coyle / Kathleen Duddy / Sarah Dyack / Cheryl R. Greenberg / Shailly Jain Ghai / Natalya Karp / Lawrence Korngut / Jonathan Kronick / Alex MacKenzie / Jennifer MacKenzie / Bruno Maranda / John J. Mitchell / Murray Potter / Chitra Prasad / Andreas Schulze / Rebecca Sparkes / Monica Taljaard / Yannis Trakadis / Jagdeep Walia / Beth K. Potter / Canadian Inherited Metabolic Diseases Research Network

    Orphanet Journal of Rare Diseases, Vol 15, Iss 1, Pp 1-

    a review

    2020  Band 15

    Abstract: Abstract Background Inherited metabolic diseases (IMDs) are a group of individually rare single-gene diseases. For many IMDs, there is a paucity of high-quality evidence that evaluates the effectiveness of clinical interventions. Clinical effectiveness ... ...

    Abstract Abstract Background Inherited metabolic diseases (IMDs) are a group of individually rare single-gene diseases. For many IMDs, there is a paucity of high-quality evidence that evaluates the effectiveness of clinical interventions. Clinical effectiveness trials of IMD interventions could be supported through the development of core outcome sets (COSs), a recommended minimum set of standardized, high-quality outcomes and associated outcome measurement instruments to be incorporated by all trials in an area of study. We began the process of establishing pediatric COSs for two IMDs, medium-chain acyl-CoA dehydrogenase (MCAD) deficiency and phenylketonuria (PKU), by reviewing published literature to describe outcomes reported by authors, identify heterogeneity in outcomes across studies, and assemble a candidate list of outcomes. Methods We used a comprehensive search strategy to identify primary studies and guidelines relevant to children with MCAD deficiency and PKU, extracting study characteristics and outcome information from eligible studies including outcome measurement instruments for select outcomes. Informed by an established framework and a previously published pediatric COS, outcomes were grouped into five, mutually-exclusive, a priori core areas: growth and development, life impact, pathophysiological manifestations, resource use, and death. Results For MCAD deficiency, we identified 83 outcomes from 52 articles. The most frequently represented core area was pathophysiological manifestations, with 33 outcomes reported in 29/52 articles (56%). Death was the most frequently reported outcome. One-third of outcomes were reported by a single study. The most diversely measured outcome was cognition and intelligence/IQ for which eight unique measurement instruments were reported among 14 articles. For PKU, we identified 97 outcomes from 343 articles. The most frequently represented core area was pathophysiological manifestations with 31 outcomes reported in 281/343 articles (82%). Phenylalanine concentration was ...
    Schlagwörter PKU ; MCAD deficiency ; Core outcome sets ; Rare diseases ; Patient-oriented outcomes ; Medicine ; R
    Thema/Rubrik (Code) 306
    Sprache Englisch
    Erscheinungsdatum 2020-01-01T00:00:00Z
    Verlag BMC
    Dokumenttyp Artikel ; Online
    Datenquelle BASE - Bielefeld Academic Search Engine (Lebenswissenschaftliche Auswahl)

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  9. Artikel ; Online: Establishing core outcome sets for phenylketonuria (PKU) and medium-chain Acyl-CoA dehydrogenase (MCAD) deficiency in children

    Beth K. Potter / Brian Hutton / Tammy J. Clifford / Nicole Pallone / Maureen Smith / Sylvia Stockler / Pranesh Chakraborty / Pauline Barbeau / Chantelle M. Garritty / Michael Pugliese / Alvi Rahman / Becky Skidmore / Laure Tessier / Kylie Tingley / Doug Coyle / Cheryl R. Greenberg / Lawrence Korngut / Alex MacKenzie / John J. Mitchell /
    Stuart Nicholls / Martin Offringa / Andreas Schulze / Monica Taljaard / In collaboration with the Canadian Inherited Metabolic Diseases Research Network

    Trials, Vol 18, Iss 1, Pp 1-

    study protocol for systematic reviews and Delphi surveys

    2017  Band 10

    Abstract: Abstract Background Inherited metabolic diseases (IMD) are a large group of rare single-gene disorders that are typically diagnosed early in life. There are important evidence gaps related to the comparative effectiveness of therapies for IMD, which are ... ...

    Abstract Abstract Background Inherited metabolic diseases (IMD) are a large group of rare single-gene disorders that are typically diagnosed early in life. There are important evidence gaps related to the comparative effectiveness of therapies for IMD, which are in part due to challenges in conducting randomized controlled trials (RCTs) for rare diseases. Registry-based RCTs present a unique opportunity to address these challenges provided the registries implement standardized collection of outcomes that are important to patients and their caregivers and to clinical providers and healthcare systems. Currently there is no core outcome set (COS) for studies evaluating interventions for paediatric IMD. This protocol outlines a study that will establish COS for each of two relatively common IMD in children, phenylketonuria (PKU) and medium-chain acyl-CoA dehydrogenase (MCAD) deficiency. Methods This two-part study is registered with the Core Outcome Measures in Effectiveness Trials (COMET) initiative. Part 1 includes a rapid review and development of an evidence map to identify a comprehensive listing of outcomes reported in past studies of PKU and MCAD deficiency. The review follows established methods for knowledge synthesis, including a comprehensive search strategy, two stages of screening citations against inclusion/exclusion criteria by two reviewers working independently, and extraction of important data elements from eligible studies, including details of the outcomes collected and outcome measurement instruments. The review findings will inform part 2 of our study, a set of Delphi surveys to establish consensus on the highest priority outcomes for each condition. Healthcare providers, families of children with PKU or MCAD deficiency, and health system decision-makers will be invited to participate in two to three rounds of Delphi surveys. The design of the surveys will involve parents of children with IMD who are part of a family advisory forum. Discussion This protocol is a crucial step in developing the capacity ...
    Schlagwörter Inherited metabolic diseases ; Core outcome set ; Registry-based randomized trials ; PKU ; MCAD deficiency ; Delphi ; Medicine (General) ; R5-920
    Thema/Rubrik (Code) 360
    Sprache Englisch
    Erscheinungsdatum 2017-12-01T00:00:00Z
    Verlag BMC
    Dokumenttyp Artikel ; Online
    Datenquelle BASE - Bielefeld Academic Search Engine (Lebenswissenschaftliche Auswahl)

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