Bayesian model of disease progression in GNE myopathy.

One Sentence Summary: A Bayesian repeated measures model based on quantitative muscle strength data from a prospective Natural History Study was developed to determine disease progression and design clinical trials for GNE myopathy, a rare and slowly progressive muscle disease. GNE myopathy is a rare muscle disease characterized by slowly progressive weakness and atrophy of skeletal muscles. To address the significant challenges of defining the natural history and designing clinical trials for GNE myopathy, we developed a Bayesian latent variable repeated measures model to determine disease progression. The model is based on longitudinal quantitative muscle strength data collected as part of a prospective Natural History Study. The GNE Myopathy Progression Model provides an understanding of disease progression that would have otherwise required a natural history of unfeasible duration. "Disease age," the model-generated measure of disease progression, highly correlates with a variety of clinical, functional and patient-reported outcomes. With the incorporation of a treatment effect parameter to the GNE Disease Progression Model, we describe a novel GNE Myopathy Disease Modification Analysis that significantly increases power and reduces the number of subjects required to test the effectiveness of novel therapies when compared to more traditional analysis methods. The GNE Myopathy Disease Progression Model and Disease Modification Analysis can be applied to muscle diseases with prospectively collected muscle strength data, and a variety of rare and slowly progressive diseases.

Distribution and severity of weakness among patients with polymyositis, dermatomyositis and juvenile dermatomyositis.

OBJECTIVE: To describe the distribution and severity of muscle weakness using manual muscle testing (MMT) in 172 patients with PM, DM and juvenile DM (JDM). The secondary objectives included characterizing individual muscle group weakness and determining associations of weakness with functional status and myositis characteristics in this large cohort of patients with myositis. METHODS: Strength was assessed for 13 muscle groups using the 10-point MMT and expressed as a total score, subscores based on functional and anatomical regions, and grades for individual muscle groups. Patient characteristics and secondary outcomes, such as clinical course, muscle enzymes, corticosteroid dosage and functional status were evaluated for association with strength using univariate and multivariate analyses. RESULTS: A gradient of proximal weakness was seen, with PM weakest, DM intermediate and JDM strongest among the three myositis clinical groups (P < or = 0.05). Hip flexors, hip extensors, hip abductors, neck flexors and shoulder abductors were the muscle groups with the greatest weakness among all three clinical groups. Muscle groups were affected symmetrically. CONCLUSIONS: Axial and proximal muscle impairment was reflected in the five weakest muscles shared by our cohort of myositis patients. However, differences in the pattern of weakness were observed among all three clinical groups. Our findings suggest a greater severity of proximal weakness in PM in comparison with DM.

Nonsurgical management of the foot and ankle affected by rheumatoid arthritis.

Rheumatoid arthritis frequently affects foot and ankle function, leading to pain, difficulty with ambulation, and disability. The purpose of this article is to describe common foot and ankle deformities associated with rheumatoid arthritis and present state-of-the-art nonsurgical management strategies. Physical impairments thought to be commonly associated with limitations of function and practical interventions for alleviating those impairments or reducing the impact of the impairment on ambulation are identified. Examples of rehabilitation interventions discussed include prescription footwear, custom and premolded foot orthoses, hindfoot orthoses, ankle-foot orthoses, shoe modifications, therapeutic exercises, and patient education. Early and aggressive attempts at prevention, delay, or correction of foot and ankle pathomechanics related to rheumatoid arthritis may play a key role in helping patients maintain an active ambulatory lifestyle.

Phonophoresis versus ultrasound in the treatment of common musculoskeletal conditions.

PURPOSE: The purpose of this study was to determine whether the pain response after phonophoresis (PH) differs from the pain response after ultrasound (US) alone. METHODS: Forty-nine subjects with soft tissue injuries including epicondylitis, tendinitis, and tenosynovitis were randomly assigned (double blinded technique) to PH or US treatment groups. Both groups received 8 min of continuous US at 1.5 w x cm(-2), three times per week for 3 wk. For the PH group a gel containing 0.05% fluocinonide was used as a coupling agent. An identical gel absent the steroid was used for the US group. Subjects indicated their pain level by marking on a visual analog scale (VAS) at the start of treatment and at the end of weeks 1, 2, and 3. Pressure algometry was used to note tolerance to direct pressure over the target tissue. ANOVA for repeated measures was used to analyze data. RESULTS: At the end of 3 wk of treatment, both groups combined showed a significant decrease in pain level and an increase in pressure tolerance (P < 0.05), but there were no differences between groups from the onset of treatment to the end of week 3 (VAS: US 5.5-1.9, PH 5.0-2.0; algometry (involved limb): US 4.7 lb-7.1 lb, PH 5.1 lb-6.6 lb). CONCLUSIONS: We conclude that US results in decreased pain and increased pressure tolerance in these selected soft tissue injuries. The addition of PH with fluocinonide does not augment the benefits of US used alone.

Postsurgical hindfoot deformity of a patient with rheumatoid arthritis treated with custom-made foot orthoses and shoe modifications.

This case report describes the treatment of a 73-year-old woman with long-standing, severe rheumatoid arthritis, using custom-made foot orthoses and shoe modifications. The patient was referred for physical therapy 4 weeks after triple arthrodesis of her right foot. Her primary complaint was periodic unsteadiness during gait, which necessitated the use of a cane at all times. Other problems included a lower-extremity length discrepancy and right foot pain. Physical therapy included fabrication of bilateral semirigid foot orthoses, shoe modifications, gait training, and instruction in strengthening exercises. After treatment, the patient reported feeling steady during walking without a cane, and she was able to resume community activities without right foot pain. Computerized movement analysis of gait revealed that the use of orthoses and modified footwear reduced weight bearing and movement-pattern abnormalities, as compared with barefoot walking. The findings in this case show that physical therapy including foot orthoses, shoe modifications, gait training, and strengthening exercises can be instrumental in the postsurgical rehabilitation of a patient with severe rheumatoid arthritis.