Staging and Surveillance of Myxoid Liposarcoma: Follow-up Assessment and the Metastatic Pattern of 169 Patients Suggests Inadequacy of Current Practice Standards.

BACKGROUND: Unlike other sarcoma subtypes, myxoid liposarcoma (MLS) has a propensity for extra-pulmonary metastases. Computed tomography (CT) scan of the chest, abdomen, and pelvis has become an accepted practice for surveillance. However, recent literature suggests that this may be inadequate. This study aimed to assess the ability of current imaging methods to detect metastases adequately in this population. METHODS: The study identified 169 patients with MLS diagnosed between 2000 and 2016. The timing and location of metastases, the reasons leading to the MLS diagnosis, and the imaging methods were recorded. The locations of metastases were classified into the following categories: pulmonary, soft tissue, bone, retroperitoneal, intraperitoneal, solid organ, and lymph node. RESULTS: An initial diagnosis of metastasis was made at presentation with staging CT scan for 3 (10 %) of 31 patients, with a follow-up surveillance CT scan for 15 (48 %) of the patients or with subsequent imaging obtained in response to patient-reported symptoms for 13 (42 %) of the patients. The proportions of patients who had metastases in each location were as follows: soft tissue (84 %), pulmonary (68 %), intraabdominal (48 %), solid organ (48 %), bone (45 %), lymph node (32 %), and retroperitoneal (29 %). Although 14 patients had bone metastases, only 1 patient had a sclerotic/blastic presentation visualized on CT scan, and the diagnosis for the remaining 13 patients was determined by magnetic resonance imaging (MRI). CONCLUSION: Due to metastatic disease identified outside surveillance imaging for 58 % of the patients, the diversity of locations, and the significant failure of CT and bone scan to identify bone metastases, this study questioned the adequacy of CT scan for surveillance of MLS. Consideration should be given to the use of whole-body MRI for detection of metastasis in MLS.

Comparison of results for body-mounted inertial sensor assessment with final lameness determination in 1,224 equids.

OBJECTIVE: To compare results for initial body-mounted inertial sensor (BMIS) measurement of lameness in equids trotting in a straight line with definitive findings after full lameness evaluation. ANIMALS: 1,224 equids. PROCEDURES: Lameness measured with BMIS equipment while trotting in a straight line was classified into categories of none, forelimb only, hind limb only, and 8 patterns of combined forelimb and hind limb lameness (CFHL). Definitive findings after full lameness evaluation were established in most horses and classified into types (no lameness, forelimb- or hind limb-only lameness, CFHL, or lameness not localized to the limbs). Observed proportions of lameness type in equids with definitive findings for each initial BMIS-assessed category were compared with hypothetical expected proportions through χ2 goodness-of-fit analysis. RESULTS: The most common initial BMIS-assessed lameness category was CFHL (693/1,224 [56.6%]), but this was the least common definitive finding (94/ 862 [10.9%]). The observed frequency of no lameness after full lameness evaluation was greater than expected only when initial BMIS measurements indicated no lameness. The observed frequency of forelimb-only lameness was greater than expected when initially measured as forelimb-only lameness and for CFHL categories consistent with the diagonal movement principle of compensatory lameness. Observed frequency of hind limb-only lameness was greater than expected when initially measured as hind limb-only lameness and for CFHL categories consistent with the sagittal movement principle of compensatory lameness. Equids initially assessed as having no lameness had the highest (103/112 [92%]) and those assessed as CFHL pattern 7 (forelimb with contralateral hind limb impact-only lameness) had the lowest (36/66 [55%]) rates of definitive findings. CONCLUSIONS AND CLINICAL RELEVANCE: In equids, results of initial straight-line trotting evaluations with a BMIS system did not necessarily match definitive findings but may be useful in planning the remaining lameness evaluation.

A Simple Method for Estimating the Economic Cost of Productivity Loss Due to Blindness and Moderate to Severe Visual Impairment.

PURPOSE: To estimate the annual loss of productivity from blindness and moderate to severe visual impairment (MSVI) using simple models (analogous to how a rapid assessment model relates to a comprehensive model) based on minimum wage (MW) and gross national income (GNI) per capita (US$, 2011). METHODS: Cost of blindness (COB) was calculated for the age group ≥50 years in nine sample countries by assuming the loss of current MW and loss of GNI per capita. It was assumed that all individuals work until 65 years old and that half of visual impairment prevalent in the ≥50 years age group is prevalent in the 50-64 years age group. For cost of MSVI (COMSVI), individual wage and GNI loss of 30% was assumed. Results were compared with the values of the uncorrected refractive error (URE) model of productivity loss. RESULTS: COB (MW method) ranged from $0.1 billion in Honduras to $2.5 billion in the United States, and COMSVI ranged from $0.1 billion in Honduras to $5.3 billion in the US. COB (GNI method) ranged from $0.1 million in Honduras to $7.8 billion in the US, and COMSVI ranged from $0.1 billion in Honduras to $16.5 billion in the US. Most GNI method values were near equivalent to those of the URE model. CONCLUSION: Although most people with blindness and MSVI live in developing countries, the highest productivity losses are in high income countries. The global economy could improve if eye care were made more accessible and more affordable to all.

The Global Burden of Potential Productivity Loss from Uncorrected Presbyopia.

PURPOSE: The onset of presbyopia in middle adulthood results in potential losses in productivity among otherwise healthy adults if uncorrected or undercorrected. The economic burden could be significant in lower-income countries, where up to 94% of cases may be uncorrected or undercorrected. This study estimates the global burden of potential productivity lost because of uncorrected functional presbyopia. DESIGN: Population data from the US Census Bureau were combined with the estimated presbyopia prevalence, age of onset, employment rate, gross domestic product (GDP) per capita in current US dollars, and near vision impairment disability weights from the Global Burden of Disease 2010 study to estimate the global loss of productivity from uncorrected and undercorrected presbyopia in each country in 2011. To allow comparison with earlier work, we also calculated the loss with the conservative assumption that the contribution to productivity extends only up to 50 years of age. PARTICIPANTS: The economic modeling did not require the use of subjects. METHODS: We estimated the number of cases of uncorrected or undercorrected presbyopia in each country among the working-age population. The number of working-age cases was multiplied by the labor force participation rate, the employment rate, a disability weight, and the GDP per capita to estimate the potential loss of GDP due to presbyopia. MAIN OUTCOME MEASURES: The outcome being measured is the lost productivity in 2011 US dollars resulting from uncorrected or undercorrected presbyopia. RESULTS: There were an estimated 1.272 billion cases of presbyopia worldwide in 2011. A total of 244 million cases, uncorrected or undercorrected among people aged <50 years, were associated with a potential productivity loss of US $11.023 billion (0.016% of global GDP). If all those people aged <65 years are assumed to be productive, the potential productivity loss would be US $25.367 billion or 0.037% of global GDP. Correcting presbyopia to the level achieved in Europe would reduce the burden to US $1.390 billion (0.002% of global GDP). CONCLUSIONS: Even with conservative assumptions regarding the productive population, presbyopia is a significant burden on productivity, and correction would have a significant impact on productivity in lower-income countries.

Real cost of recycled spectacles.

PURPOSE: Many programs aimed at mitigating the problem of uncorrected refractive error and the resulting avoidable blindness use recycled (donated) spectacles as a seemingly inexpensive expedient. This article analyses the costs and benefits of recycled spectacles and compares them with alternative methodologies. Although well intentioned, it is argued that recycled spectacles will neither suit many of those affected by uncorrected refractive error nor provide a cost saving solution to the problem. Although this is not the first argument against the use of recycled spectacles, there has been no accurate costing of their delivery. This article assesses the real cost of delivery of recycled spectacles. METHODS: The useable quantity of recycled spectacles was determined by examining two separate batches of donated spectacles. These data were used in the calculation of the cost of delivery. The metric used for comparison was only cost (i.e., it was a cost minimization analysis) because it was deemed that recycled spectacles and ready-made spectacles were the same mode of correction fundamentally. RESULTS: Only 7% of the 275 recycled spectacles analyzed were suitable for use. The relatively small proportion of useable spectacles contributed to the high societal cost of delivering recycled spectacles, which was found to be U.S.$20.49, more than twice the cost of supplying ready-made spectacles. CONCLUSIONS: Recycled spectacles are not a cost-saving method of correcting refractive error and should be discouraged as a strategy for eliminating uncorrected refractive error in developing countries.

Assessment of repeatability of a wireless, inertial sensor-based lameness evaluation system for horses.

OBJECTIVE: To determine repeatability of a wireless, inertial sensor-based lameness evaluation system in horses. ANIMALS: 236 horses. PROCEDURES: Horses were from 2 to 29 years of age and of various breeds and lameness disposition. All horses were instrumented with a wireless, inertial sensor-based motion analysis system on the head (accelerometer), pelvis (midline croup region [accelerometer]), and right forelimb (gyroscope) before evaluation in 2 consecutive trials, approximately 5 minutes apart, as the horse was trotted in a straight line. Signal-processing algorithms generated overall trial asymmetry measures for vertical head and pelvic movement and stride-by-stride differences in head and pelvic maximum and minimum positions between right and left sides of each stride. Repeatability was determined, and trial difference was determined for groups of horses with various numbers of strides for which data were collected per trial. RESULTS: Inertial sensor-based measures of torso movement asymmetry were repeatable. Repeatability for measures of torso asymmetry for determination of hind limb lameness was slightly greater than that for forelimb lameness. Collecting large numbers of strides degraded stride-to-stride repeatability but did not degrade intertrial repeatability. CONCLUSIONS AND CLINICAL RELEVANCE: The inertial sensor system used to measure asymmetry of head and pelvic movement as an aid in the detection and evaluation of lameness in horses trotting in a straight line was sufficiently repeatable to investigate for clinical use.

The humanistic burden of hereditary angioedema: Impact on health-related quality of life, productivity, and depression.

Hereditary angioedema (HAE) is a rare, autosomal dominant disorder characterized by recurrent acute attacks of swelling of the larynx, abdomen, and periphery. This study was designed to assess the humanistic burden of illness associated with HAE. HAE burden was assessed via a web-based survey of patients that solicited information on attack characterization, treatment, side effects, pain, and functional and emotional burden of disease management. In addition to HAE-specific sections, the survey used three standardized instruments to compare HAE patient data to normative (healthy) and chronic disease populations: the 12-Item Short Form (SF-12) Health Survey, the Work Productivity and Activity Impairment-General Health (WPAI-GH) questionnaire, and the Hamilton Depression Inventory-Short Form (HDI-SF). A total of 457 HAE patients responded to the survey (response rate, ∼19%). Patients reported significantly poorer health-related quality of life versus population norms, based on the SF-12 Physical Component Summary (mean, 43.7 versus 49.6; p < 0.001) and Mental Component Summary (mean, 42.6 versus 49.4; p < 0.001). HAE patients also had higher mean HDI-SF scores than population norms (8.1 ± 6.5 versus 3.1 ± 3.0; p < 0.001), with 42.5% of HAE patients scoring >8.5, indicative of depressive symptomatology. Productivity was also markedly impaired in all WPAI-GH categories, including 34% overall work impairment. Because of their most recent HAE attack, workers lost a mean of 3.3 days; students lost a mean of 1.9 days. HAE results in considerable humanistic burden to patients across physical and mental health domains; negatively impacts education, career, and work productivity; and compounds the substantial economic burdens that are reported separately.

Economic costs associated with acute attacks and long-term management of hereditary angioedema.

BACKGROUND: Hereditary angioedema (HAE) is a rare autosomal dominant disorder characterized by recurrent acute attacks of swelling of the larynx, abdomen, and periphery. OBJECTIVE: To assess the economic burden associated with acute attacks and long-term management of HAE. METHODS: Burden was assessed via a Web-based survey of HAE patients (> or = 18 years old) that solicited information on attack characterization, short-term treatment, long-term disease management, impact on work, and patient costs. A standardized instrument, the Work Productivity and Activity Impairment questionnaire, was included to assess impact on work productivity. Standard medical costs and US average wage costs were assigned to survey items to assess direct medical and indirect costs, respectively. RESULTS: Total annual per-patient costs are estimated at $42,000 for the average HAE patient, with costs totaling $14,000 for patients with mild attacks, $27,000 for patients with moderate attacks, and $96,000 for patients with severe attacks. Hospital costs account for 67% of direct medical costs. Respondents reported high rates of missed work, lost productivity, and lost income, contributing to indirect costs totaling $16,000 annually for the average patient. Almost all costs increase with disease severity, although the distribution varies with severity: indirect costs account for 75% of costs for patients with mild attacks, whereas emergency department and hospital costs account for 68% of costs for patients with severe attacks. CONCLUSIONS: HAE results in considerable economic burden to patients, payers, and society in terms of direct medical and indirect costs and compounds the substantial humanistic burdens, which will be reported separately.

Assessing the economic challenges posed by orphan drugs.

Historically, patients with rare diseases have been underserved by commercial drug development. In several jurisdictions, specific legislation has been enacted to encourage the development of drugs for rare diseases (orphan drugs), which would otherwise not be commercially viable. However, because of the small market, these drugs are often very expensive. Under the standard methods of health technology assessment (HTA) incorporating economic evaluation, orphan drugs do not usually prove to be cost-effective and this, coupled with their high cost, means that funding and patient access may be limited. However, these restrictions may not be in line with societal preferences. Therefore, this study discusses whether the standard methods of HTA are adequate for assisting decisions on patient access to and funding of orphan drugs and outlines a research agenda to help understand the societal value of orphan drugs and issues surrounding their development, funding, and use.

Health and economic consequences of sevelamer use for hyperphosphatemia in patients on hemodialysis.

OBJECTIVES: The safety and efficacy of sevelamer hydrochloride in binding phosphate in patients with end-stage renal disease and its ability to attenuate the progression of cardiac calcification have been well documented but not the longer-term health and economic implications. Thus, a model of the predicted long-term consequences of sevelamer compared with calcium-based binders (acetate and carbonate) was developed. METHODS: Long-term cardiovascular implications of 1 year of treatment with phosphate binders in patients on hemodialysis are estimated based on the patient's demographics, comorbidities, and physiologic and renal parameters. The initial calcification score and expected changes over 1 year are derived using regression equations developed from the Treat-to-Goal study and translated to cardiovascular disease risk based on equations developed from a long-term cohort study. In this article, the implications of cardiovascular disease for life expectancy and medical costs are accounted for from a US payer perspective. RESULTS: The cardioprotective effect of sevelamer over 1 year is estimated to result in a 12% reduction in cardiovascular events compared with calcium acetate. In a population of 100 patients, the savings of 205,600 dollars accrued due to avoiding nine cardiovascular events with sevelamer, largely offset the increased binder costs, leading to a favorable cost-effectiveness ratio of about 2200 dollars per (discounted) life-year gained. CONCLUSIONS: Although both binders provide equivalent phosphate binding capacity, the results indicate that the advantage of 1 year of treatment with sevelamer in attenuating the progression of calcification has important clinical and economic consequences, suggesting that this provides good value for money.