scholarly journals Pompe Disease: Diagnosis and Management. Evidence-Based Guidelines from a Canadian Expert Panel

Author(s):  
Mark Tarnopolsky ◽  
Hans Katzberg ◽  
Basil J. Petrof ◽  
Sandra Sirrs ◽  
Harvey B. Sarnat ◽  
...  

AbstractPompe disease is a lysosomal storage disorder caused by a deficiency of the enzyme acid alpha-glucosidase. Patients have skeletal muscle and respiratory weakness with or without cardiomyopathy. The objective of our review was to systematically evaluate the quality of evidence from the literature to formulate evidence-based guidelines for the diagnosis and management of patients with Pompe disease. The literature review was conducted using published literature, clinical trials, cohort studies and systematic reviews. Cardinal treatment decisions produced seven management guidelines and were assigned a GRADE classification based on the quality of evidence in the published literature. In addition, six recommendations were made based on best clinical practices but with insufficient data to form a guideline. Studying outcomes in rare diseases is challenging due to the small number of patients, but this is in particular the reason why we believe that informed treatment decisions need to consider the quality of the evidence.

2013 ◽  
Vol 127 (4) ◽  
pp. 364-367 ◽  
Author(s):  
N Su ◽  
P P Cheang ◽  
H Khalil

AbstractBackground:Chronic sinusitis is the most common routine presentation for a general ENT surgeon. The 2007 ‘Primary Care Guidelines: European Position Paper on the Primary Care Diagnosis and Management of Rhinosinusitis and Nasal Polyps’ aimed to deliver evidence-based guidelines for the diagnosis and management of rhinosinusitis in specialist and primary care.Objective:The aim of this audit was to assess the information provided in the referral letters to the ENT department regarding patients with potential rhinosinusitis, and compare this to the information required for the rhinology care pathways.Method:We evaluated one month of referrals to the ENT department.Results:The quality of information in the referral letters was poor. Only 22 per cent of patient referrals included basic information about symptoms, duration and treatment.Conclusion:We plan to investigate why general practitioners are not complying with the pathway. In addition, the pathways will be more widely disseminated via the ‘Map of Medicine’ (an online resource for general practitioners). This should facilitate the receipt of the best evidence-based treatment for patients prior to referral to secondary care.


Transfusion ◽  
2021 ◽  
Author(s):  
Arwa Z. Al‐Riyami ◽  
Rachel Jug ◽  
Ursula La Rocca ◽  
Homa Keshavarz ◽  
Denise Landry ◽  
...  

BMJ ◽  
2021 ◽  
pp. e066045 ◽  
Author(s):  
Liang Yao ◽  
Muhammad Muneeb Ahmed ◽  
Gordon H Guyatt ◽  
Peijing Yan ◽  
Xu Hui ◽  
...  

Abstract Objective To investigate whether alignment of strength of recommendations with quality of evidence differs in consensus based versus evidence based guidelines. Design Empirical analysis. Data source Guidelines developed by the American College of Cardiology and the American Heart Association (ACC/AHA) and the American Society of Clinical Oncology (ASCO) up to 27 March 2021. Study selection Recommendations were clearly categorised as consensus or evidence based, were separated from the remainder of the text, and included both the quality of evidence and the strength of the recommendations. Data extraction Paired authors independently extracted the recommendation characteristics, including type of recommendation (consensus or evidence based), grading system used for developing recommendations, strength of the recommendation, and quality of evidence. The study team also calculated the number of discordant recommendations (strong recommendations with low quality evidence) and inappropriate discordant recommendations (those that did not meet grading of recommendations assessment, development, and evaluation criteria of appropriateness). Results The study included 12 ACC/AHA guidelines that generated 1434 recommendations and 69 ASCO guidelines that generated 1094 recommendations. Of the 504 ACC/AHA recommendations based on low quality evidence, 200 (40%) proved to be consensus based versus 304 (60%) evidence based; of the 404 ASCO recommendations based on low quality evidence, 292 (72%) were consensus based versus 112 (28%) that were evidence based. In both ACC/AHA and ASCO guidelines, the consensus approach yielded more discordant recommendations (ACC/AHA: odds ratio 2.1, 95% confidence interval 1.5 to 3.1; ASCO: 2.9, 1.1 to 7.8) and inappropriate discordant recommendations (ACC/AHA: 2.6, 1.7 to 3.7; ASCO: 5.1, 1.6 to 16.0) than the evidence based approach. Conclusion Consensus based guidelines produce more recommendations violating the evidence based medicine principles than evidence based guidelines. Ensuring appropriate alignment of quality of evidence with the strength of recommendations is key to the development of “trustworthy” guidelines.


2018 ◽  
Vol 32 (3) ◽  
pp. 135-143 ◽  
Author(s):  
Katerina Pavenski ◽  
Simon Stanworth ◽  
Mark Fung ◽  
Erica M. Wood ◽  
Joanne Pink ◽  
...  

2013 ◽  
Author(s):  
Kimberly D. Becker ◽  
Dana Darney ◽  
Celene Domitrovich ◽  
Catherine Bradshaw ◽  
Nicholas S. Ialongo

2022 ◽  
Author(s):  
Dedi Ardinata

Evidence-based medicine (EBM), which emphasizes that medical decisions must be based on the most recent best evidence, is gaining popularity. Individual clinical expertise is combined with the best available external clinical evidence derived from systematic research in the practice of EBM. The key and core of EBM is the hierarchical system for categorizing evidence. The Grading of Recommendations, Assessment, Development and Evaluations (GRADE) system divides evidence quality into four categories: high, moderate, low, and very low. GRADE is based on the lowest quality of evidence for any of the outcomes that are critical to making a decision, reducing the risk of mislabeling the overall evidence quality, when evidence for a critical outcome is lacking. This principle is also used in acupuncture as a complementary and integrative treatment modality, but incorporating scientific evidence is more difficult due to a number of factors. The goal of this chapter is to discuss how to establish a clinical evidence system for acupuncture, with a focus on the current quality of evidence for a variety of conditions or diseases.


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