Pompe disease results from lysosomal acid α-glucosidase deficiency, which leads to cardiomyopathy in all infantile-onset and occasional late-onset patients. Cardiac assessment is important for its diagnosis and management. This article presents unpublished cardiac findings, concomitant medications, and cardiac efficacy and safety outcomes from the ADVANCE study; trajectories of patients with abnormal left ventricular mass z score at enrolment; and post hoc analyses of on-treatment left ventricular mass and systolic blood pressure z scores by disease phenotype, GAA genotype, and “fraction of life” (defined as the fraction of life on pre-study 160 L production-scale alglucosidase alfa). ADVANCE evaluated 52 weeks’ treatment with 4000 L production-scale alglucosidase alfa in ≥1-year-old United States of America patients with Pompe disease previously receiving 160 L production-scale alglucosidase alfa. M-mode echocardiography and 12-lead electrocardiography were performed at enrolment and Week 52. Sixty-seven patients had complete left ventricular mass z scores, decreasing at Week 52 (infantile-onset patients, change −0.8 ± 1.83; 95% confidence interval −1.3 to −0.2; all patients, change −0.5 ± 1.71; 95% confidence interval −1.0 to −0.1). Patients with “fraction of life” <0.79 had left ventricular mass z score decreasing (enrolment: +0.1 ± 3.0; Week 52: −1.1 ± 2.0); those with “fraction of life” ≥0.79 remained stable (enrolment: −0.9 ± 1.5; Week 52: −0.9 ± 1.4). Systolic blood pressure z scores were stable from enrolment to Week 52, and no cohort developed systemic hypertension. Eight patients had Wolff–Parkinson–White syndrome. Cardiac hypertrophy and dysrhythmia in ADVANCE patients at or before enrolment were typical of Pompe disease. Four-thousand L alglucosidase alfa therapy maintained fractional shortening, left ventricular posterior and septal end-diastolic thicknesses, and improved left ventricular mass z score.

Trial registry: ClinicalTrials.gov Identifier: NCT01526785 https://clinicaltrials.gov/ct2/show/NCT01526785.

Social Media Statement: Post hoc analyses of the ADVANCE study cohort of 113 children support ongoing cardiac monitoring and concomitant management of children with Pompe disease on long-term alglucosidase alfa to functionally improve cardiomyopathy and/or dysrhythmia.

Hereditary transthyretin-mediated (hATTR) amyloidosis is a progressive disease caused by mutations in the TTR gene leading to multisystem organ dysfunction. Pathogenic TTR aggregation, misfolding, and fibrillization lead to deposition of amyloid in multiple body organs and frequently involve the peripheral nerve system and the heart. Common neurologic manifestations include: sensorimotor polyneuropathy (PN), autonomic neuropathy, small-fiber PN, and carpal tunnel syndrome. Many patients have significant progression due to diagnostic delays as hATTR PN is not considered within the differential diagnosis. Recently, two effective novel disease-modifying therapies, inotersen and patisiran, were approved by Health Canada for the treatment of hATTR PN. Early diagnosis is crucial for the timely introduction of these disease-modifying treatments that reduce impairments, improve quality of life, and extend survival. In this guideline, we aim to improve awareness and outcomes of hATTR PN by making recommendations directed to the diagnosis, monitoring, and treatment in Canada.

For clinicians, primary care physicians (PCPs), and other health professionals treating patients with any ailment, including depression, the first task in addressing nonadherence to treatment is to detect it. Patients conceal treatment nonadherence because it is a socially undesirable behavior, and patients want their clinicians or PCPs to think that they are “good” patients. They may hope that their clinicians will be understanding, but assume that clinicians will adopt a judgmental attitude. There is evidence that, as a result of these psychological barriers, self-report and physician interview assessments have poor correlation with adherence as measured by virtually any other methodology. Three studies have been conducted that dramatically indicate the force of this need to conceal nonadherence.

The purpose was to compare the Spanish language picture version of the Free and Cued Selective Reminding Test with Immediate Recall (pFCSRT+IR) and the Mini Mental State Exam (MMSE) in identifying very mild dementia among Spanish speaking Latino patients. The tests and an independent diagnostic assessment were administered to 112 Latino patients free of medically diagnosed dementia from an urban primary care clinic. Receiver operating characteristic (ROC) curves and the area under the curve (AUC) were used to examine differences in the operating characteristics of the pFCSRT+IR and the MMSE. Cut scores were manipulated to equate sensitivities (specificities) at clinically relevant values to compare differences in specificities (sensitivities) using the Pearson Chi Square test. Youden’s index was used to select the optimal cut scores. Twenty-four of the 112 primary care patients (21%) received a research dementia diagnosis, indicating a substantial burden of unrecognized dementia. MMSE scores but not free recall scores were associated with years of education in patients free of dementia. AUC was significantly higher for free recall than for MMSE. Free recall performed significantly better than the MMSE in sensitivity and in specificity. Using optimal cut scores, patients with impaired free recall were 10 times more likely to have dementia than patients with intact recall, and patients with impaired MMSE scores were 4.5 times more likely to have dementia than patients with intact scores. These results suggest that the Spanish language pFCSRT+IR may be an effective tool for dementia screening in educationally diverse Latino primary care populations. (JINS, 2014, 20, 1–8)

Radiogenic iodine is one of the more difficult fission products to capture and immobilize during the reprocessing of spent nuclear fuel.

However, for metallic fuels reprocessed by electrometallurgical treatment, it is believed that the majority of fission-product iodine is retained during the various processing steps. Spent fuel from the Experimental Breeder Reactor II (EBR-II) at the Idaho National Laboratory (INL) is being treated by a combination of electrochemical and pyrometallurgical methods to deactivate the bond sodium of the fuel, recover uranium, and immobilize fission products for disposal. This paper discusses the progress of various strategies and experiments to confirm the expected retention of iodine during the electrometallurgical treatment of EBR-II spent fuel. This includes surveys of previous observations and measurements, and the direct measurement of iodine from various process samples. Current measurements are aimed at iodine determination in the bond sodium and plenum regions of the fuel, refined iodine measurements in electrorefiner salt, and the retention of iodine during waste form production.

Near the end of his important and challenging essay, Martin J. Sklar briefly considers an alternative path of development to the corporate-liberal reorganization that he identifies with the era between the 1890s and 1916. “A statist resolution might have taken hold,” Sklar writes,

had the American capitalist class, or its corporate sector, been less developed in its market powers and proficiencies and hence more dependent on the state for its wealth and power; had the liberal republican tradition of the supremacy of society over the state (the sovereignty of the people) been weaker; had the working class been less imbued with that republican ideology, less developed, and hence more inclined to statist rather than associative-constitutional ideas and principles; had the corporate sector of the capitalist class sought and found alliance with a statist-oriented sector of the working class or a statist-oriented petty bourgeoisie, especially in the farm and rural population; had the corporate sector of the capitalist class sought and found alliance with civilian or military professionals, technicians, administrators, and managers—or a “managerial class”—looking to the state as a base of power. (p. 210)