Browsing by Author "Korngut, Lawrence"
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- ItemOpen AccessAdult patient perspectives of the unknowns of living with epilepsy - results from a focus group study.(2019-11-24) Lee, Jeanie Y. Y.; Gelfand, Jennifer; Khan, Sundus; Crooks, Rachel E.; Josephson, Colin B.; Wiebe, Samuel; Patten, Scott B.; Korngut, Lawrence; Smith, Eric Edward; Roach, Pamela M.Background/Objectives: Epilepsy is one of the most common and debilitating neurological conditions that affects nearly 50 million people worldwide, yet there remains a stigma around this condition, which can impact the information-seeking behaviours of patients. As the Brain and Mental Health Research Clinics develop a website about registry-based research, including patient-facing areas, it is important to understand how patients look for information, and the types of information they are seeking out. The objective of this study was to encourage conversation and understand the patient perspectives of existing knowledge gaps between epilepsy patients and the resources they use to obtain information. Methods: A total of thirteen patients (mean (SD) age = 46.4 (16.1) years) from the Calgary Comprehensive Epilepsy Program Registry and four caregivers participated in one of the three focus groups completed in order to meet our aims. There were eight female and five male patients. A semi-structured guide was used to understand the patients’ experiences, top concerns, informational resources currently used, and resources or knowledge that patients felt are lacking. The focus groups were audio-recorded and transcribed verbatim. Thematic content analysis was conducted by two researchers who independently open-coded the transcripts using NVivo 11. The final analysis was done by team discussion and ongoing analysis of the codes to create themes and sub-themes. Results: The major themes that emerged from the data included: 1) daily management; 2) resources; and 3) medications and treatment. For daily management, the participants reported concerns about the effects of epilepsy on day-to-day activities such as driving, working, and the barriers they face in society due to their perceived lack of awareness and education about seizure management in the general public. The participants felt negatively impacted by the stigma and compared their experience with epilepsy with other disorders such as cancer or diabetes which they feel are much more accepted in society. The geographical location of the patient also plays a role in the support they receive for epilepsy management, with participants citing challenges and feelings of isolation in rural areas. To acquire more information about epilepsy, participants reported that they primarily asked their physicians or searched online. However, despite the conveniences of the internet, some individuals felt the volume and variation of quality of online information was overwhelming. Instead, they would prefer to go to trusted resources that are provided by healthcare professionals or websites affiliated with hospitals or universities. Updated information on medication, side effects, and research are examples of resources the patients would like to see provided on such websites. Conclusion: Overall, it is clear from our focus groups that resources and support for self-management and day-to-day living for individuals with epilepsy is paramount to reduce knowledge gaps. Not only is it important to provide daily management and medication information to patients through trusted organizational resources, but it is equally important to increase public awareness about epilepsy and seizure disorders to reduce the stigma attached to these conditions.
- ItemOpen AccessCorrection to: Eight years after an international workshop on myotonic dystrophy patient registries: case study of a global collaboration for a rare disease(2019-08-15) Wood, Libby; Bassez, Guillaume; Bleyenheuft, Corinne; Campbell, Craig; Cossette, Louise; Jimenez-Moreno, Aura C; Dai, Yi; Dawkins, Hugh; Díaz-Manera, Jordi; Dogan, Celine; el Sherif, Rasha; Fossati, Barbara; Graham, Caroline; Hilbert, James; Kastreva, Kristinia; Kimura, En; Korngut, Lawrence; Kostera-Pruszczyk, Anna; Lindberg, Christopher; Lindvall, Bjorn; Luebbe, Elizabeth; Lusakowska, Anna; Mazanec, Radim; Meola, Giovani; Orlando, Liannna; Takahashi, Masanori P; Peric, Stojan; Puymirat, Jack; Rakocevic-Stojanovic, Vidosava; Rodrigues, Miriam; Roxburgh, Richard; Schoser, Benedikt; Segovia, Sonia; Shatillo, Andriy; Thiele, Simone; Tournev, Ivailo; van Engelen, Baziel; Vohanka, Stanislav; Lochmüller, HannsThe original version of this article [1] unfortunately included an error to an author’s name. Author Jordi Díaz-Manera was erroneously presented as Jorge Alberto Diaz Manera. The correct author name has been included in the author list of this Correction article.
- ItemOpen AccessThe Effect of Cigarette Smoking as a Modifiable Risk Factor for Secondary Progression in Multiple Sclerosis(2020-07-28) Javizian, Omid; Koch, Marcus W.; Patten, Scott B.; Korngut, LawrenceObjective: To investigate the influence of cigarette smoking on disease progression and disability accumulation in multiple sclerosis (MS). Methods: Kaplan-Meier analyses and Cox proportional hazard modelling were used to evaluate the influence of cigarette smoking on the time to- and age at onset of secondary progressive MS (SPMS), and on time from disease onset to Expanded Disability Status Scale (EDSS) 4.0 and 6.0 as well as the time between EDSS 4.0 to 6.0 in 763 patients with SPMS. Results: No significant associations between cigarette smoking and disease progression as measured by time to- and age at onset of SPMS, or disability accumulation, as measured by time to and age at EDSS 4.0 and 6.0 were identified. Conclusion: Our investigation of a large and well-characterized population-based SPMS cohort suggests that cigarette smoking does not influence disease progression or disability accumulation in SPMS patients.
- ItemOpen AccessEight years after an international workshop on myotonic dystrophy patient registries: case study of a global collaboration for a rare disease(2018-09-05) Wood, Libby; Bassez, Guillaume; Bleyenheuft, Corinne; Campbell, Craig; Cossette, Louise; Jimenez-Moreno, Aura C; Dai, Yi; Dawkins, Hugh; Manera, Jorge A D; Dogan, Celine; el Sherif, Rasha; Fossati, Barbara; Graham, Caroline; Hilbert, James; Kastreva, Kristinia; Kimura, En; Korngut, Lawrence; Kostera-Pruszczyk, Anna; Lindberg, Christopher; Lindvall, Bjorn; Luebbe, Elizabeth; Lusakowska, Anna; Mazanec, Radim; Meola, Giovani; Orlando, Liannna; Takahashi, Masanori P; Peric, Stojan; Puymirat, Jack; Rakocevic-Stojanovic, Vidosava; Rodrigues, Miriam; Roxburgh, Richard; Schoser, Benedikt; Segovia, Sonia; Shatillo, Andriy; Thiele, Simone; Tournev, Ivailo; van Engelen, Baziel; Vohanka, Stanislav; Lochmüller, HannsAbstract Background Myotonic Dystrophy is the most common form of muscular dystrophy in adults, affecting an estimated 10 per 100,000 people. It is a multisystemic disorder affecting multiple generations with increasing severity. There are currently no licenced therapies to reverse, slow down or cure its symptoms. In 2009 TREAT-NMD (a global alliance with the mission of improving trial readiness for neuromuscular diseases) and the Marigold Foundation held a workshop of key opinion leaders to agree a minimal dataset for patient registries in myotonic dystrophy. Eight years after this workshop, we surveyed 22 registries collecting information on myotonic dystrophy patients to assess the proliferation and utility the dataset agreed in 2009. These registries represent over 10,000 myotonic dystrophy patients worldwide (Europe, North America, Asia and Oceania). Results The registries use a variety of data collection methods (e.g. online patient surveys or clinician led) and have a variety of budgets (from being run by volunteers to annual budgets over €200,000). All registries collect at least some of the originally agreed data items, and a number of additional items have been suggested in particular items on cognitive impact. Conclusions The community should consider how to maximise this collective resource in future therapeutic programmes.
- ItemOpen AccessEstablishing core outcome sets for phenylketonuria (PKU) and medium-chain Acyl-CoA dehydrogenase (MCAD) deficiency in children: study protocol for systematic reviews and Delphi surveys(2017-12-19) Potter, Beth K; Hutton, Brian; Clifford, Tammy J; Pallone, Nicole; Smith, Maureen; Stockler, Sylvia; Chakraborty, Pranesh; Barbeau, Pauline; Garritty, Chantelle M; Pugliese, Michael; Rahman, Alvi; Skidmore, Becky; Tessier, Laure; Tingley, Kylie; Coyle, Doug; Greenberg, Cheryl R; Korngut, Lawrence; MacKenzie, Alex; Mitchell, John J; Nicholls, Stuart; Offringa, Martin; Schulze, Andreas; Taljaard, MonicaAbstract 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 to launch RCTs with meaningful outcomes that address comparative effectiveness questions in the field of paediatric IMD. Such trials will contribute high-quality evidence to inform decision-making by patients and their family members, clinicians, and policy-makers.
- ItemOpen AccessIntranasal Insulin for Treatment of Diabetic Polyneuropathy(2013-11-19) Korngut, Lawrence; Wiebe, Samuel; Jetté, NathalieIntranasal insulin administration is a novel approach to slow the progression of diabetic polyneuropathy (DPN). We performed a pilot randomized controlled trial of intranasal insulin in 12 type 1 diabetes mellitus patients with DPN to assess safety. We administered intranasal insulin for 6 weeks using biweekly dose-escalation up to 160 IU/d or intranasal saline. The primary outcome measure was frequency of hypoglycaemia. Frequency of mild (mHG) and serious hypoglycaemic (sHG) events was recorded. Secondary outcomes included clinical (Utah Early Neuropathy Score (UENS)) and laboratory (corneal confocal microscopy and electrophysiology) measures. There were no differences in glycemia between groups after supervised initial administration. The 40 IU/d and 80 IU/d doses were safe and well tolerated with comparable mHG events between groups. One intranasal insulin subject suffered a sHG at home while receiving 160 IU/d. Intranasal insulin was safe and well tolerated at 40 and 80 IU/d.
- ItemOpen AccessOutcomes in pediatric studies of medium-chain acyl-coA dehydrogenase (MCAD) deficiency and phenylketonuria (PKU): a review(2020-01-14) Pugliese, Michael; Tingley, Kylie; Chow, Andrea; Pallone, Nicole; Smith, Maureen; Rahman, Alvi; Chakraborty, Pranesh; Geraghty, Michael T; Irwin, Julie; Tessier, Laure; Nicholls, Stuart G; Offringa, Martin; Butcher, Nancy J; Iverson, Ryan; Clifford, Tammy J; Stockler, Sylvia; Hutton, Brian; Paik, Karen; Tao, Jessica; Skidmore, Becky; Coyle, Doug; Duddy, Kathleen; Dyack, Sarah; Greenberg, Cheryl R; Ghai, Shailly J; Karp, Natalya; Korngut, Lawrence; Kronick, Jonathan; MacKenzie, Alex; MacKenzie, Jennifer; Maranda, Bruno; Mitchell, John J; Potter, Murray; Prasad, Chitra; Schulze, Andreas; Sparkes, Rebecca; Taljaard, Monica; Trakadis, Yannis; Walia, Jagdeep; Potter, Beth KAbstract 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 the most frequently reported outcome. Sixteen percent of outcomes were reported by a single study. Similar to MCAD deficiency, the most diversely measured PKU outcome was cognition and intelligence/IQ with 39 different instruments reported among 82 articles. Conclusions Heterogeneity of reported outcomes and outcome measurement instruments across published studies for both MCAD deficiency and PKU highlights the need for COSs for these diseases, to promote the use of meaningful outcomes and facilitate comparisons across studies.
- ItemOpen AccessPerspectives on neurological patient registries: a literature review and focus group study(BioMed Central, 2013-11-09) Korngut, Lawrence; MacKean, Gail; Casselman, Lisa; Johnston, Megan; Day, Lundy; Lam, Darren; Lorenzetti, Diane; Warner, Janet; Jetté, Nathalie; Pringsheim, Tamara
- ItemOpen AccessThe Impact of Enrollment in a Specialized Interdisciplinary Neuropathic Pain Clinic(2011-01-01) Garven, Alex; Brady, Shauna; Wood, Susan; Hatfield, Melinda; Bestard, Jennifer; Korngut, Lawrence; Toth, CoryBACKGROUND: Chronic pain clinics have been created because of the increasing recognition of chronic pain as a very common, debilitating condition that requires specialized care. Neuropathic pain (NeP) is a multifaceted, specialized form of chronic pain that often requires input from multiple disciplines for assessment and management.OBJECTIVE: To determine the impact of an interdisciplinary clinic for evaluation and treatment of patients with NeP.METHODS: Patients with heterogeneous etiologies for NeP were prospectively evaluated using an interdisciplinary approach every six months. Diagnostic evaluation, comorbidity evaluation, education, and pharmacological and/or nonpharmacological management were completed. Severity (visual analogue scale) and features of pain (Modified Brief Pain Inventory), sleep difficulties (Medical Outcomes Study – Sleep Scale), mood/anxiety disruption (Hospital Anxiety and Depression Scale), quality of life (European Quality-of-Life Five-Domain index), health care resources use, patient satisfaction (Pain Treatment Satisfaction Scale and Neuropathic Pain Symptom Inventory) and self-perceived change in well-being (Patient Global Impression of Change scale) were examined at each visit.RESULTS: Pain severity only decreased after one year of follow-up, while anxiety and quality-of-life indexes improved after six months. Moderate improvements of sleep disturbance, less frequent medication use and reduced health care resource use were observed during enrollment at the NeP clinic.DISCUSSION: Despite the limitations of performing a real-world, uncontrolled study, patients with NeP benefit from enrollment in a small interdisciplinary clinic. Education and a complete diagnostic evaluation are hypothesized to lead to improvements in anxiety and, subsequently, pain severity. Questions remain regarding the long-term maintenance of these improvements and the optimal structure of specialized pain clinics.