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Glucocorticoid treatment is recommended as a standard of care in Duchenne muscular dystrophy; however, few studies have assessed the long-term benefits of this treatment. We examined the long-term effects of glucocorticoids on milestone-related disease progression across the lifespan and survival in patients with Duchenne muscular dystrophy.For this prospective cohort study, we enrolled male patients aged 2-28 years with Duchenne muscular dystrophy at 20 centres in nine countries. Patients were followed up for 10 years. We compared no glucocorticoid treatment or cumulative treatment duration of less than 1 month versus treatment of 1 year or longer with regard to progression of nine disease-related and clinically meaningful mobility and upper limb milestones. We used Kaplan-Meier analyses to compare glucocorticoid treatment groups for time to stand from supine of 5 s or longer and 10 s or longer, and loss of stand from supine, four-stair climb, ambulation, full overhead reach, hand-to-mouth function, and hand function. Risk of death was also assessed. This study is registered with ClinicalTrials.gov, number NCT00468832.440 patients were enrolled during two recruitment periods (2006-09 and 2012-16). Time to all disease progression milestone events was significantly longer in patients treated with glucocorticoids for 1 year or longer than in patients treated for less than 1 month or never treated (log-rank p0·0001). Glucocorticoid treatment for 1 year or longer was associated with increased median age at loss of mobility milestones by 2·1-4·4 years and upper limb milestones by 2·8-8·0 years compared with treatment for less than 1 month. Deflazacort was associated with increased median age at loss of three milestones by 2·1-2·7 years in comparison with prednisone or prednisolone (log-rank p0·012). 45 patients died during the 10-year follow-up. 39 (87%) of these deaths were attributable to Duchenne-related causes in patients with known duration of glucocorticoids usage. 28 (9%) deaths occurred in 311 patients treated with glucocorticoids for 1 year or longer compared with 11 (19%) deaths in 58 patients with no history of glucocorticoid use (odds ratio 0·47, 95% CI 0·22-1·00; p=0·0501).In patients with Duchenne muscular dystrophy, glucocorticoid treatment is associated with reduced risk of losing clinically meaningful mobility and upper limb disease progression milestones across the lifespan as well as reduced risk of death.US Department of Education/National Institute on Disability and Rehabilitation Research; US Department of Defense; National Institutes of Health/National Institute of Arthritis and Musculoskeletal and Skin Diseases; and Parent Project Muscular Dystrophy.

We describe five patients with Duchenne muscular dystrophy who presented with acute neurologic and respiratory symptoms following minor trauma. Four of the five deteriorated rapidly and died within 36 h after falling. X-rays for fractures were negative. Four of the five patients were taking corticosteroids daily. All five patients fulfilled the clinical criteria for Fat Embolism Syndrome. Autopsy findings were consistent with fat embolism in two cases. Fat Embolism Syndrome needs to be considered in patients with Duchenne muscular dystrophy following minor trauma even without fractures. Early recognition of Fat Embolism Syndrome and aggressive resuscitation are important to improve survival. This report serves as an important reminder that seatbelts need to be used at all times.

Tumefactive demyelinating lesions may be misdiagnosed as brain neoplasms or abscesses. In this paper, we present four cases of pediatric tumefactive demyelination. Twelve cases of pediatric tumefactive demyelination previously reported in the English literature are also summarized. We describe the neuroimaging characteristics and clinical presentation of tumefactive demyelination and how these features may be used in differentiating demyelination from other mass lesions.

COSTS ASSOCIATED WITH HCV AND RELATED COMPLICATIONS IN THE UNITED STATES FROM A MANAGED CARE PAYER’S PERSPECTIVE McAdam Marx C1, Hane CA2, Biskupiak J1, Deniz B3, McGarry L4, Brixner D1 1University of Utah, Salt Lake City, UT, USA, 2Ingenix, Eden Prairie, MN, USA, 3Vertex Pharmaceuticals Incorporated, Cambridge, MA, USA, 4i3 Innovus, Medford, MA, USA OBJECTIVES: Estimate current healthcare costs for HCV and its consequences in a large, US managed care organization (MCO). METHODS: Patients with ICD-9 diagnosis codes for Hepatitis C viral (HCV) infection (1st diagnosis index date), age 18 years with 6 months of continuous enrollment were identified in a large, MCO claims database from 1/1/2002 to 3/31/2010. HCV patients were matched 1: 10 to patients without an HCV diagnosis or advanced liver disease (ALD), based on gender, age, index year (where synthetic index date enrollment median post-enrollment days to case index date), hospital referral region (HRR) state, pre-index healthcare costs, alcoholism, HIV/AIDS, and modified Charlson Comorbidity Index. Cases were stratified by disease state: chronic HCV without liver involvement (CHCV), compensated cirrhosis (CC), decompensated cirrhosis (DC), hepatocellular carcinoma (HCC), or liver transplant. Mean per-member per-year (PMPY) costs were estimated post-index (total post-index cost of all patients /[sum of all postindex days/365]). Incremental PMPY costs for HCV patients vs. matched controls were estimated overall and by each disease state. RESULTS: 34,597 HCV patients were matched to 330,435 controls. Mean age of cases was 49.9 ( 8.5) years; 62% were female; 78% had C-HCV, 4% CC, 12% DC, 3% HCC, and 3% transplant. Incremental costs vs. controls overall were $9,681 PMPY. Incremental PMPY costs for patients with ALD were DC: $27,845, HCC: $43,671, and transplant: $93,609. For patients without ALD, incremental PMPYs were C-HCV: $5,870, and CC: $5,330. Incremental drug costs for HCV treatment were $2,739 overall, ranging from $1,893$8,736 for different states. C-HCV and CC drug costs were $2,659 and $3,102, respectively. CONCLUSIONS: Current estimates of HCV cost burden to MCOs were higher than previously reported and increased substantially with progression to ALD. The higher estimated costs of managing chronic HCV were likely due to high non-liver related costs among HCV patients or imprecise coding of CC.