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Browsing by Subject "Duchenne Muscular Dystrophy"
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Item Fat Embolism Syndrome in Duchenne Muscular Dystrophy Patients: Early Recognition and Aggressive Therapy(Hindawi, 2018-06-04) Murphy, Lee D.; Yabrodi, Mouhammad; Lutfi, Riad; Pediatrics, School of MedicineWe describe two pediatric patients with Duchenne muscular dystrophy that presented with acute neurologic deterioration and hypoxic respiratory failure requiring mechanical ventilation. These cases fulfill the clinical criteria for Fat Embolism Syndrome. Early recognition and aggressive supportive therapy with mechanical ventilation, right ventricular afterload reduction, and blood transfusion led to survival without any residual effects from the event. Fat Embolism Syndrome needs to be considered early in the course of patients with Duchenne muscular dystrophy who present with respiratory and neurological symptoms.Item Leveraging Cardiac Magnetic Resonance Imaging to Assess Skeletal Muscle Progression in Duchenne Muscular Dystrophy(Elsevier, 2022) Kaslow, Jacob A.; Sokolow, Andrew G.; Donnelly, Thomas; Buchowski, Maciej S.; Damon, Bruce M.; Markham, Larry W.; Burnette, W. Bryan; Soslow, Jonathan H.; Pediatrics, School of MedicineDuchenne Muscular Dystrophy (DMD) is characterized by muscle deterioration and progressive weakness. As a result, patients with DMD have significant cardiopulmonary morbidity and mortality that worsens with age and loss of ambulation. Since most validated muscle assessments require ambulation, new functional measures of DMD progression are needed. Despite several evaluation methods available for monitoring disease progression, the relationship between these measures is unknown. We sought to assess the correlation between imaging metrics obtained from cardiac magnetic resonance imaging (CMR) and functional assessments including quantitative muscle testing (QMT), spirometry, and accelerometry. Forty-nine patients with DMD were enrolled and underwent CMR, accelerometry and QMT at baseline, 1-year and 2-year clinic visits with temporally associated pulmonary function testing obtained from the medical record. Imaging of the upper extremity musculature (triceps and biceps) demonstrated the most robust correlations with accelerometry (p<0.03), QMT (p<0.02) and spirometry (p<0.01). T1-mapping of serratus anterior muscle showed a similar, but slightly weaker relationship with accelerometry and QMT. T2-mapping of serratus anterior demonstrated weak indirect correlation with aspects of accelerometry. These images are either routinely obtained in standard CMR or can be added to a protocol and may allow for a more comprehensive assessment of a patient’s disease progression.Item Participant Experiences with a New Online Modified-Delphi Approach for Engaging Patients and Caregivers in Developing Clinical Guidelines(2019) Armstrong, Courtney; Grant, Sean; Kinnett, Kathi; Denger, Brian; Martin, Ann; Coulter, Ian; Booth, Marika; Khodyakov, Dmitry; Social and Behavioral Sciences, School of Public HealthBackground Patient engagement in clinical practice guideline (CPG) development has increased significantly in recent years. However, only a few patients and caregivers join CPG development groups. Objective To describe participant experiences with a novel online, scalable approach for patient and caregiver engagement in CPG development. Design We developed and tested the RAND/PPMD Patient-Centeredness Method (RPM), a novel online modified-Delphi approach to patient engagement in CPG development that consists of an optional idea generation round and two rating rounds interspersed with an online discussion round. Setting and Participants Using the online ExpertLens™ system, we ran 2 concurrent panels of patients and caregivers of individuals with Duchenne Muscular Dystrophy (DMD). Main Outcome Measures We surveyed all 95 panel participants about their participation and satisfaction with the process. We also conducted telephone interviews with 25 participants. Results Participants expressed satisfaction with various ExpertLens™ features, noting that the system fostered lively interaction among them. Panelists also appreciated participating in an educational, interactive and convenient discussion forum that allowed them to share their opinions on the importance and acceptability of different recommendations. The RPM was viewed as empowering by patients and their caregivers who felt it would be useful for CPG developers. Discussion and Conclusion The results of our study show the overall participant satisfaction with a novel, scalable, online approach to engaging patients and caregivers in CPG development, which allows them to share their perspectives and lived experiences using a rigorous, systematic and iterative way that is similar to how clinicians provide their input.Item The RAND/PPMD Patient-Centeredness Method: a novel online approach to engaging patients and their representatives in guideline development(2019) Khodyakov, Dmitry; Denger, Brian; Grant, Sean; Kinnett, Kathi; Armstrong, Courtney; Martin, Ann; Peay, Holly; Coulter, Ian; Hazlewood, Glen; Social and Behavioral Sciences, School of Public HealthAlthough clinical practice guidelines (CPGs) provide recommendations for how best to treat a typical patient with a given condition, patients and their representatives are not always engaged in CPG development. Despite the agreement that patient participation may improve the quality and utility of CPGs, there is no systematic, scalable method for engaging patients and their representatives, as well as no consensus on what exactly patients and their representatives should be asked to do during CPG development. To address these gaps, an interdisciplinary team of researchers, patient representatives, and clinicians developed the RAND/PPMD Patient-Centeredness Method (RPM) - a novel online approach to engaging patients and their representatives in CPG development. The RPM is an iterative approach that allows patients and their representatives to provide input by (1) generating ideas; (2) rating draft recommendations on two criteria (importance and acceptability); (3) explaining and discussing their ratings with other participants using online, asynchronous, anonymous, moderated discussion boards, and (4) revising their responses if needed. The RPM was designed to be consistent with the RAND/UCLA Appropriateness Method used by clinicians and researchers to develop CPG, while helping patients and their representative rate outcome importance and recommendation acceptability - two key components of the GRADE Evidence to Decision (EtD) framework. With slight modifications, the RPM has the potential to explore consensus among key stakeholders on other dimensions of the EtD, including feasibility, equity, and resource use.