20 research outputs found

    Clinical Meaningfulness of the Changes in Muscle Performance and Physical Function Associated With Testosterone Administration in Older Men With Mobility Limitation

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    Context. Testosterone in Older Men with Mobility Limitations Trial determined the effects of testosterone on muscle performance and physical function in older men with mobility limitation. Trial's Data and Safety Monitoring Board recommended enrollment cessation due to increased frequency of adverse events in testosterone arm. The changes in muscle performance and physical function were evaluated in relation to participant's perception of change. Methods. Men aged 65 years and older, with mobility limitation, total testosterone 100-350 ng/dL, or free testosterone less than 50 pg/mL, were randomized to placebo or 10 g testosterone gel daily for 6 months. Primary outcome was leg-press strength. Secondary outcomes included chest-press strength, stair-climb, 40-m walk, muscle mass, physical activity, self-reported function, and fatigue. Proportions of participants exceeding minimally important difference in study arms were compared. Results. Of 209 randomized participants, 165 had follow-up efficacy measures. Mean (SD) age was 74 (5.4) years and short physical performance battery score 7.7 (1.4). Testosterone arm exhibited greater improvements in leg-press strength, chest-press strength and power, and loaded stair-climb than placebo. Compared with placebo, significantly greater proportion of men receiving testosterone improved their leg-press and chest-press strengths (43% vs 18%, p = .01) and stair-climbing power (28% vs 10%, p = .03) more than minimally important difference. Increases in leg-press strength and stair-climbing power were associated with changes in testosterone levels and muscle mass. Physical activity, walking speed, self-reported function, and fatigue did not change. Conclusions. Testosterone administration in older men with mobility limitation was associated with patient-important improvements in muscle strength and stair-climbing power. Improvements in muscle strength and only some physical function measures should be weighed against the risk of adverse events in this populatio

    Indirect estimation of the prevalence of spinal muscular atrophy Type I, II, and III in the United States

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    Abstract Background Spinal muscular atrophy (SMA) is a progressive, devastating disease and a leading inherited cause of infant mortality. The limited population-based literature is confined to small regional studies. Estimates of prevalence are needed to characterize the burden of SMA and to understand trends in prevalence by disease type as new treatments become available. The reported estimates of SMA genotype prevalence at birth consistently range from 8.5–10.3 per 100,000 live births, with a mid-range estimate of 9.4 per 100,000. Among infants born with an SMA genotype, it is reported that ~58% will develop SMA Type I, 29% will develop Type II, and 13% will develop Type III, respectively. Results Using evidence from peer-reviewed literature for SMA birth prevalence, age at symptom onset, and SMA type-specific survival, and incorporating United States vital statistics, we constructed life tables to estimate prevalence for SMA Types I, II, and III in the United States. We estimated the number of prevalent cases in the US to be 8526, 9429, and 10,333 based on a birth prevalence of 8.5, 9.4, and 10.3, respectively (the lower, midpoint, and upper ends of the reported range). Assuming the midpoint of 9.4 and US-reported survival, the type-specific population prevalence estimates were 1610 for SMA Type I, 3944 for SMA Type II, and 3875 for SMA Type III. Evidence-based estimates of the number of people living with SMA in the United States in the published literature were previously unavailable. Conclusions In the absence of a survey or other means to directly estimate prevalence in the US population, estimates can be calculated indirectly using a life table

    Additional file 1: Table S1. of Indirect estimation of the prevalence of spinal muscular atrophy Type I, II, and III in the United States

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    Summary of contemporary published estimates of SMA birth prevalence. Table showing summary of contemporary published estimates of SMA birth prevalence. (DOCX 23 kb

    Additional file 2: Table S2. of Indirect estimation of the prevalence of spinal muscular atrophy Type I, II, and III in the United States

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    Summary of survival probabilities for patients with SMA Type I in the United States. Table showing summary of survival probabilities for patients with SMA Type I in the United States. (DOCX 20 kb

    Additional file 3: Table S3. of Indirect estimation of the prevalence of spinal muscular atrophy Type I, II, and III in the United States

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    Summary of survival probabilities for patients with SMA Type II in the United States. Table showing summary of survival probabilities for patients with SMA Type II in the United States. (DOCX 14 kb
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