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1.
Mov Disord Clin Pract ; 10(9): 1377-1387, 2023 Sep.
Artículo en Inglés | MEDLINE | ID: mdl-37772308

RESUMEN

Background: Treating functional movement disorder (FMD) with motor retraining is effective but resource intensive. Objectives: Identify patient, disease, and program variables associated with favorable treatment outcomes. Methods: Retrospective review of the 1 week intensive outpatient FMD program at Mayo Clinic in Minnesota from February 2019 to August 2021. Outcomes included patient-reported measures (Canadian Occupational Performance Measure-Performance and Satisfaction subscales [COPM-P and COPM-S, range 0-10] and Global Rating of Change [GROC, -7 to +7]) and a retrospective investigator-rated scale (0-3, worse/not improved to significantly improved/resolved). Linear regression models identified variables predicting favorable outcomes. Results: Participants (n = 201, 74% female, mean age = 46) had median FMD duration of 24 months. The commonest FMD subtypes were gait disorder (65%), tremor (41%) and weakness (17%); 53% had ≥2 subtypes. Most patients (88%) completed a therapeutic screening process before program entry. Patient-reported outcomes at the end of the week improved substantially (COPM-P average change 3.8 ± 1.9; GROC post-program average 5.5 ± 1.7). Available investigator-rated outcomes from short-term follow-up were also positive (102/122 [84%] moderately to significantly improved/resolved). Factors predicting greater improvement in COPM-P were completing therapeutic screening, higher number of non-motor symptoms, shorter FMD duration, earlier program entry, lower baseline COPM scores, and (among screened patients) higher GROC between therapeutic screening and program start. Conclusion: Patients with diverse FMD subtypes improved substantially over a 1 week period. Utilization of therapeutic screening and greater improvement between therapeutic screening and program start were novel predictors of favorable outcomes. Non-motor symptoms did not preclude positive responses, although patients with predominant non-motor burden were excluded.

3.
J Inherit Metab Dis ; 44(2): 502-514, 2021 03.
Artículo en Inglés | MEDLINE | ID: mdl-32677106

RESUMEN

BACKGROUND: (+)-Epicatechin (EPI) induces mitochondrial biogenesis and antioxidant metabolism in muscle fibers and neurons. We aimed to evaluate safety and efficacy of (+)-EPI in pediatric subjects with Friedreich's ataxia (FRDA). METHODS: This was a phase II, open-label, baseline-controlled single-center trial including 10 participants ages 10 to 22 with confirmed FA diagnosis. (+)-EPI was administered orally at 75 mg/d for 24 weeks, with escalation to 150 mg/d at 12 weeks for subjects not showing improvement of neuromuscular, neurological or cardiac endpoints. Neurological endpoints were change from baseline in Friedreich's Ataxia Rating Scale (FARS) and 8-m timed walk. Cardiac endpoints were changes from baseline in left ventricular (LV) structure and function by cardiac magnetic resonance imaging (MRI) and echocardiogram, changes in cardiac electrophysiology, and changes in biomarkers for heart failure and hypertrophy. RESULTS: Mean FARS/modified (m)FARS scores showed nonstatistically significant improvement by both group and individual analysis. FARS/mFARS scores improved in 5/9 subjects (56%), 8-m walk in 3/9 (33%), 9-peg hole test in 6/10 (60%). LV mass index by cardiac MRI was significantly reduced at 12 weeks (P = .045), and was improved in 7/10 (70%) subjects at 24 weeks. Mean LV ejection fraction was increased at 24 weeks (P = .008) compared to baseline. Mean maximal septal thickness by echocardiography was increased at 24 weeks (P = .031). There were no serious adverse events. CONCLUSION: (+)-EPI was well tolerated over 24 weeks at up to 150 mg/d. Improvement was observed in cardiac structure and function in subset of subjects with FRDA without statistically significant improvement in primary neurological outcomes. SYNOPSIS: A (+)-epicatechin showed improvement of cardiac function, nonsignificant reduction of FARS/mFARS scores, and sustained significant upregulation of muscle-regeneration biomarker follistatin.


Asunto(s)
Antioxidantes/administración & dosificación , Catequina/administración & dosificación , Ataxia de Friedreich/tratamiento farmacológico , Corazón/diagnóstico por imagen , Adolescente , Niño , Ecocardiografía , Femenino , Ataxia de Friedreich/fisiopatología , Humanos , Imagen por Resonancia Magnética , Masculino , Estudios Prospectivos , Índice de Severidad de la Enfermedad , Resultado del Tratamiento , Caminata
4.
Am J Phys Med Rehabil ; 83(10): 785-90, 2004 Oct.
Artículo en Inglés | MEDLINE | ID: mdl-15385789

RESUMEN

OBJECTIVE: To characterize current musculoskeletal (MSK) education experiences in physical medicine and rehabilitation residency programs and to identify perceived barriers to providing more extensive MSK education experiences. In addition, to establish utilization patterns for the PASSOR Physical Examination Core Competencies List. DESIGN: Between March and November 2003, all 81 physical medicine and rehabilitation residency program directors were asked to complete an MSK education survey developed by the authors. RESULTS: A total of 69 of 81 program directors (86%) responded after multiple contacts. The most frequently utilized MSK education formats were MSK lecture series, MSK departmental conferences, and physical examination workshops. Potential barriers to expanded MSK education included money, time, and staff number. Given unlimited resources, most residency programs would greatly increase utilization of visiting lecturers, CD-ROMs/DVDs, objective structured clinical examinations, and physical examination videos. Of the 30 program directors who recalled receiving the Core Competencies List, only 40% (12 of 30) have formally integrated the list into their residency training. Barriers to implementation included logistical challenges and lack of direction regarding implementation. CONCLUSIONS: Residency program directors indicate a strong interest in expanding resident MSK education through the use of CD-ROMs/DVDs, physical examination videos, objective structured clinical examinations, and visiting lecturer programs. CD-ROMs/DVDs and videos represent particularly attractive educational formats for supplementing resident MSK education due to the advantages of central production, nominal costs, widespread distribution, multimedia capabilities, and accessibility. These educational formats should be considered for targeted educational initiatives to enhance resident MSK education, regardless of residency program size or resources.


Asunto(s)
Internado y Residencia/estadística & datos numéricos , Medicina Física y Rehabilitación/educación , Medicina Física y Rehabilitación/estadística & datos numéricos , Rehabilitación/educación , Rehabilitación/estadística & datos numéricos , Competencia Clínica/normas , Curriculum/estadística & datos numéricos , Conocimientos, Actitudes y Práctica en Salud , Humanos , Enfermedades Musculoesqueléticas/rehabilitación , Sistema Musculoesquelético , Enseñanza/métodos , Estados Unidos
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